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Clinical Trials Unit

Stanford University School of Medicine's Center for Advanced Dermatologic Investigation is the Dermatology Department's clinical trials unit. 

The Center is home to 12-15 ongoing clinical studies, investigating the safety and efficacy of new and currently available drugs and over-the-counter medications.  The Center works with Stanford's own panel on medical research, leading pharmaceutical companies,and the Food and Drug Administration to safely and ethically expand the medical field's knowledge of dermatologic treatments.  New studies begin regularly, and the Center continues to recruit patients with skin aging, sun damage, skin cancer (including basal cell carcinomas), psoriasis, atopic dermatitis, rosacea, and other dermatologic diseases for ongoing studies. 

Skin Aging Studies

We seek to understand the human aging processes as it relates to skin on a fundamental level. To this end, our studies focus on clinical and translational research efforts ranging from: (1) the analysis of gene changes which predispose individuals to exceptionally youthful skin to (2) molecular signatures that may be biomarkers for aging skin to (3) the careful study of new candidate agents which might affect the skin aging process.

Nonmelanoma Skin Cancer

Recent advances in our understanding of basal cell skin cancer biology have enabled the development of cutting edge study drugs which combat tumor growth. We are currently home to a number of clinical trials at the forefront of potential therapy for advanced or metastatic basal cell cancer. In addition, we seek to understand the biology of basal cell skin cancers and to identify molecular predictors for treatment success.

Acne Rosecea

This is a common and frustrating chronic inflammatory condition of the face, usually affecting older individuals. The causes of this complex condition are the subject of much study. Our clinical studies seek to identify new topical or oral medications to improve the symptoms of acne rosacea.

Contact

For more information, please email dermtrials@stanford.edu

Featured Clinical Trials

  • The Impact of Pediatric Skin Disorders: The "Big" Study

    This study leverages the availability of a validated series of instruments to measure the effect of highly visible, chronic skin disorders, including atopic dermatitis (AD), on patients 8 years of age and above in causing stigma and psychiatric issues, particularly anxiety and depression.

    Investigator

    • Joyce Teng, MD, PhD
    Not accepting patients at this time View Details
  • Topical Remetinostat Gel as Neoadjuvant Therapy in Patients With Squamous Cell Carcinoma (SCC)

    The primary purpose of this study is to determine if 8 weeks of topical remetinostat applied three times daily will suppress Squamous Cell Carcinoma.

    Not accepting patients at this time View Details
  • Topical Remetinostat in Treating Patient With Cutaneous Basal Cell Cancer

    This phase 2 trial studies how well remetinostat works in treating patients with skin basal cell cancer. Remetinostat may slow the growth of basal cell cancer cells.

    Not accepting patients at this time View Details
  • Efficacy and Safety Study of Apremilast (CC-10004) in Pediatric Subjects From 6 Through 17 Years of Age With Moderate to Severe Plaque Psoriasis

    This is a Phase 3, multicenter, randomized, placebo-controlled, double-blind study of the efficacy and safety of apremilast (CC-10004) in pediatric subjects with moderate to severe plaque psoriasis.

    At least 230 pediatric subjects (ages 6 through 17 years) will be randomized 2:1 to receive either apremilast or placebo for the first 16 weeks and then all subjects will receive apremilast during the 36 week Extension Phase for a total of 52 weeks. Randomization to apremilast arm or placebo arm will be stratified by age group (6 to 11 years or 12 to 17 years). Subjects will receive apremilast treatment of either 20 mg twice daily (BID) or 30 mg BID, depending on weight. This Phase 3 study is being conducted to evaluate the safety and efficacy of apremilast in the treatment of pediatric subjects.

    Investigator

    • Joyce Teng, MD, PhD
    Not accepting patients at this time View Details

Psoriasis Clinical Trials

  • Daratumumab, Pomalidomide, and Dexamethasone (DPd) in Relapsed/Refractory Light Chain Amyloidosis Patients Previously Exposed to Daratumumab

    This study will test the hypothesis that in patients with previous daratumumab exposure, combination therapy of daratumumab, pomalidomide, and dexamethasone (DPd) will yield higher complete remission (CR) rates in relapsed/refractory amyloidosis than historical pomalidomide/dexamethasone treatment.

    Investigator

    • Michaela Liedtke
    Now accepting new patients View Details
  • Development of 3D Multi-Parametric Ultrasound (MPUS) as a Decision Support Tool for Patients

    This clinical trial studies how well 3-dimensional multi-parametric ultrasound (3D MPUS) imaging works as a decision-support tool for patients with liver tumors undergoing therapy. Continuous and dynamic imaging of patients undergoing therapy is required to monitor early-phase treatment response. 3D-MPUS is an inexpensive and safe method, which may provide complementary quantitative functional (perfusion) and tissue characterization information to anatomical radiological assessment or blood biomarkers.

    Investigator

    • Aya Kamaya, MD
    Now accepting new patients View Details
  • Dose Optimization and Expansion Study of DFV890 in Adult Patients With Myeloid Diseases

    Study CDFV890G12101 is an open-label, phase 1b, multicenter study with a randomized two-dose optimization part, and a dose expansion part consisting of two groups evaluating DFV890 in patients with myeloid diseases. The purpose of this study is to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, efficacy and recommended dose for single agent DFV890 in patients with lower risk (LR: very low, low or intermediate risk) myelodysplastic syndromes (LR MDS) and lower risk chronic myelomonocytic leukemia (LR CMML).

    Investigator

    • Peter Greenberg
    Now accepting new patients View Details
  • T-DM1 and Tucatinib Compared with T-DM1 Alone in Preventing Relapses in People with High Risk HER2-Positive Breast Cancer, the CompassHER2 RD Trial

    This phase III trial studies how well trastuzumab emtansine (T-DM1) and tucatinib work in preventing breast cancer from coming back (relapsing) in patients with high risk, HER2 positive breast cancer. T-DM1 is a monoclonal antibody, called trastuzumab, linked to a chemotherapy drug, called DM1. Trastuzumab is a form of targeted therapy because it attaches to specific molecules (receptors) on the surface of cancer cells, known as HER2 receptors, and delivers DM1 to kill them. Tucatinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Giving T-DM1 and tucatinib may work better in preventing breast cancer from relapsing in patients with HER2 positive breast cancer compared to T-DM1 alone.

    Investigator

    • Melinda L. Telli, M.D.
    Now accepting new patients View Details
  • Telephone-based Physical Activity Coaching or Self Monitored Physical Activity to Improve Physical Function in Older Adults Who Are Undergoing Surgery for Lung Cancer and Their Caregivers

    This clinical trial compares telephone-based physical activity coaching to self monitored physical activity for improving physical function in older adults who are undergoing surgery for lung cancer and their caregivers. Lung cancer surgery in older adults is associated with functional declines and unique challenges. Performing physical activity around the time of surgery has been shown to improve functional outcomes in patients and exercise programs delivered via telehealth may improve access and convenience for patients and minimize participant burden. Telephone-based physical activity coaching may improve physical functioning for older adults with lung cancer who are undergoing surgery.

    Investigator

    • Natalie Shaubie Lui
    Now accepting new patients View Details
  • Testing Nivolumab and Ipilimumab Immunotherapy With or Without the Targeted Drug Cabozantinib in Recurrent, Metastatic, or Incurable Nasopharyngeal Cancer

    This phase II trial tests how well nivolumab and ipilimumab immunotherapy with or without cabozantinib works in treating patients with nasopharyngeal cancer that has come back (after a period of improvement) (recurrent), has spread from where it first started (primary site) to other places in the body (metastatic), or for which no treatment is currently available (incurable). Immunotherapy with monoclonal antibodies, such as nivolumab and ipilimumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Cabozantinib is in a class of medications called kinase inhibitors. It works by blocking the action of an abnormal protein that signals cancer cells to multiply. This helps slow or stop the spread of cancer cells. Giving immunotherapy with nivolumab and ipilimumab and targeted therapy with cabozantinib may help shrink and stabilize nasopharyngeal cancer.

    Now accepting new patients View Details
  • Telehealth 2.0: Evaluating Effectiveness and Engagement Strategies for CPT-Text for PTSD

    There is a pressing need to increase capacity to treat PTSD related to or exacerbated by the COVID-19 pandemic. Texting-based therapy holds promise to increase capacity and reduce barriers to delivering evidence-based treatments (EBTs), but ongoing engagement in digital mental health interventions is low. This study will compare a texting-based EBT for PTSD to culturally-informed texting-based treatment for PTSD as usual, and it will also compare a unique incentive strategy to typical platform reminders aimed to prevent early discontinuation in therapy. This online study is open to individuals who live in 13 different states.

    Investigator

    • Shannon Wiltsey Stirman
    Now accepting new patients View Details
  • TACTIVE-U: A Study to Learn About the Study Medicine (Vepdegestrant) When Given With Other Medicines in People With Advanced or Metastatic Breast Cancer (Sub-Study B)

    The purpose of this clinical trial is to learn about the safety and effects of the study medicine (called ARV-471) when given together with other medicines for the potential treatment of advanced or metastatic breast cancer.

    This study is seeking participants who have breast cancer that:

    * is advanced, may have spread to other organs (metastatic) and cannot be fully treated by surgery or radiation therapy* is sensitive to hormonal therapy (it is called estrogen receptor positive); and* is no longer responding to previous treatments

    This study is divided into separate sub-studies.

    For Sub-Study B:

    All participants will receive ARV-471 and a medicine called ribociclib. ARV-471 and ribociclib will be given at the same time by mouth, at home, 1 time a day.

    The experiences of people receiving the study medicine will be examined. This will help determine if the study medicine is safe and effective.

    Participants will continue to take ARV-471 and ribociclib until their cancer is no longer responding, or side effects become too severe. They will have visits at the study clinic about every 4 weeks.

    Investigator

    • Melinda L. Telli, M.D.
    Now accepting new patients View Details
  • TACTIVE-U: A Study to Learn About the Study Medicine (Vepdegestrant) When Given With Other Medicines in People With Advanced or Metastatic Breast Cancer. (Sub-Study C)

    The purpose of this study is to learn about the safety and effects of the study medicine called vepdegestrant. The safety and effects of vepdegestrant will be see when given with other medicines. Vepdegestrant is studied to see if it can be a possible treatment for advanced metastatic breast cancer. This type of cancer would have spread from where it started (breast) to other parts of the body and would be tough to treat.

    The study is seeking for participants who have breast cancer that:

    * is hard to treat (advanced) and may have spread to other organs (metastatic). is sensitive to hormonal therapy (it is called estrogen receptor positive).* is no longer responding to treatments taken before starting this study.

    This study is divided into separate sub-studies.

    For Sub-Study C:

    All the participants will receive vepdegestrant and a medicine called samuraciclib.

    Vepdegestrant and samuraciclib will be taken once in a day by mouth. The medicines will be taken at home. The experience of people receiving the study medicines will be studied. This will help see if the study medicine is safe and effective.

    Participant will continue to take vepdegestrant and samuraciclib until:

    * their cancer is no longer responding, or* side effects become too severe.

    They will have visits at the study clinic about every 4 weeks.

    Investigator

    • Melinda L. Telli, M.D.
    Now accepting new patients View Details
  • Type 1 Diabetes and the Brain in Children

    The investigators have previously studied a group of young children with T1D using brain MRI, age-appropriate neurocognitive testing and continuous glucose monitoring, followed for 18 months. The investigators observed significant differences in gray matter volumes and white matter microstructure in the children with diabetes as compared to controls. These differences appeared to increase over time, with slower rates of brain growth in the T1D group (Mazelli, et al, Diabetes 2014; Barnea-Goraly, et al, Diabetes Care 2014; Mauras, et al, Diabetes 2015). In this new protocol the investigators will include the same children with T1D and healthy controls previously studied and recruit new similar subjects to replace those lost by attrition. The investigators will be using structural and functional brain MRI, neurocognitive testing and measures of glycemic control, to determine if changes in the brain persist or worsen over longitudinal follow up, and whether these changes are associated with measures of glycemic control and neurocognitive metrics as these children grow and progress through puberty.

    Investigator

    • Allan L. Reiss
    Now accepting new patients View Details
  • The Myelin Disorders Biorepository Project

    The Myelin Disorders Biorepository Project (MDBP) seeks to collect and analyze clinical data and biological samples from leukodystrophy patients worldwide to support ongoing and future research projects. The MDBP is one of the world's largest leukodystrophy biorepositories, having enrolled nearly 2,000 affected individuals since it was launched over a decade ago.

    Researchers working in the biorepository hope to use these materials to uncover new genetic etiologies for various leukodystrophies, develop biomarkers for use in future clinical trials, and better understand the natural history of these disorders. The knowledge gained from these efforts may help improve the diagnostic tools and treatment options available to patients in the future.

    Investigator

    • Keith Van Haren, MD
    Now accepting new patients View Details
  • Trial of Zolpidem for Sleep in Children with Autism

    The purpose of this study is to examine the effect of zolpidem on sleep in children and adolescents with Autism Spectrum Disorder (ASD). Zolpidem is a nonbenzodiazepine GABAa receptor agonist drug that acts as a hypnotic. To accomplish this, the investigators will use a randomized double-blind placebo-controlled crossover 8-week study design to examine the effect of zolpidem on sleep physiology as assessed by polysomnography (PSG), actigraphy, circadian rhythm, and clinical measures.

    Investigator

    • Antonio Hardan, M.D.
    Now accepting new patients View Details
  • Trial of Acebilustat for the Treatment of Upper Arm Lymphedema

    This study is designed to investigate the response of unilateral upper extremity (arm) lymphedema, during pharmacologic treatment of lymphedema with oral placebo and oral acebilustat. Participants will receive "study drug" (Acebilustat or placebo), for 9 months. For 3 of these months, the participant will receive placebo; for 6 of these months, the participant will receive active ingredient, acebilustat. The study is blinded which means that the participant will not be told which study pill they are taking.

    Investigators

    • Stanley G. Rockson, MD
    • Mark Nicolls
    Now accepting new patients View Details
  • Testing Multi-Level Remote Physical Activity Interventions in a National Sample of Older Women: The WHISH EnCore Trial

    While older women are disproportionately affected by chronic diseases and conditions associated with aging, including both physical and cognitive impairments, that can be alleviated or delayed by regular physical activity, few physical activity programs have been developed specifically with their needs in mind. This research aims to evaluate, in insufficiently active older women from the national WHISH pragmatic trial, the effects of a technology-driven "citizen science" approach to environmental physical activity barriers called Our Voice plus the ongoing "light-touch" remote physical activity educational program, compared to the "light-touch" remote physical activity educational program plus a control educational intervention that creates awareness around human and planetary health. This study will add important information on the benefits and trade-offs of combining these remotely delivered and practical behavioral health approaches to promote physical and cognitive health for the fast-expanding demographic group of U.S. older women.

    Investigator

    • Abby C. King
    Now accepting new patients View Details
  • Study to Evaluate Percussive Ventilation Breathhold to Improve Lung Stereotactic Ablative Radiotherapy

    The purpose of this research is to evaluate and determine the rate of successful administration of Percussive ventilation breathhold (PVB) to allow for more accurate radiotherapy for lung tumors.

    Now accepting new patients View Details
  • Study of Axicabtagene Ciloleucel Versus Standard of Care Therapy in Participants With Relapsed/Refractory Follicular Lymphoma

    The goal of this clinical study is test how well the study drug, axicabtagene ciloleucel, works in participants with relapsed/refractory follicular lymphoma

    Investigator

    • Matthew Frank
    Now accepting new patients View Details
  • Safety and Efficacy Study of Epcoritamab in Subjects With Relapsed/Refractory Chronic Lymphocytic Leukemia and Richter's Syndrome

    The study is a global, multi-center safety and efficacy trial of epcoritamab, an antibody also known as EPKINLY™ and GEN3013 (DuoBody®-CD3xCD20). Epcoritamab will either be studied as:

    * Monotherapy, or* Combination therapy:

    * epcoritamab + venetoclax * epcoritamab + lenalidomide * epcoritamab + R-CHOP (i.e., rituximab, cyclophosphamide, doxorubicin hydrochloride, vincristine (Oncovin®) and prednisone).

    The study includes patients with relapsed/refractory chronic lymphocytic leukemia (R/R CLL)/small lymphocytic lymphoma (SLL) and patients with Richter's Syndrome (RS).

    Study participants with R/R CLL/SLL are treated either with epcoritamab as monotherapy or epcoritamab + venetoclax. Study participants with RS are treated either with epcoritamab as monotherapy or epcoritamab + lenalidomide or epcoritamab + R-CHOP. The trial consists of two parts, a dose-escalation phase (phase Ib) and an expansion phase (phase II). Patients with RS are only included in the expansion phase.

    Epcoritamab will be injected subcutaneously (under the skin). Standard-of-care and combination treatments (venetoclax, lenalidomide, and R-CHOP) will be given either orally (by mouth) or intravenously (in a vein).

    Study details include:

    * Study duration will be up to 5 years.* The treatment duration for each participant will be between 18 months (1.5 years) and 24 months (2 years), depending upon the treatment arm assigned.* The visit frequency will be either weekly, every other week, or monthly, depending upon the part of the study.

    All participants will receive active drug; no one will be given placebo.

    Investigator

    • Bita Fakhri, MD, MPH
    Now accepting new patients View Details
  • Understanding Allergies and Sensitizations in Healthy and Allergic Individuals

    The purpose of this study is to strengthen our ability to accurately diagnose allergies and understand cellular, humoral, genetic components and physiological changes in allergic disease

    Investigator

    • R. Sharon Chinthrajah
    Now accepting new patients View Details
  • Study of Aspirin in Patients with Vestibular Schwannoma

    This is a phase II prospective, randomized, double-blind, longitudinal study evaluating whether the administration of aspirin can delay or slow tumor growth and maintain or improve hearing in VS patients.

    Investigator

    • Konstantina M. Stankovic, MD, PhD, FACS
    Now accepting new patients View Details
  • Study of BDC-3042 as Single Agent and in Combination With Cemiplimab in Patients With Advanced Malignancies

    A first-in-human study using BDC-3042 as a single agent and in combination with cemiplimab in patients with advanced malignancies

    Now accepting new patients View Details
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Dermatology Clinical Trials

  • Daratumumab, Pomalidomide, and Dexamethasone (DPd) in Relapsed/Refractory Light Chain Amyloidosis Patients Previously Exposed to Daratumumab

    This study will test the hypothesis that in patients with previous daratumumab exposure, combination therapy of daratumumab, pomalidomide, and dexamethasone (DPd) will yield higher complete remission (CR) rates in relapsed/refractory amyloidosis than historical pomalidomide/dexamethasone treatment.

    Investigator

    • Michaela Liedtke
    Now accepting new patients View Details
  • Development of 3D Multi-Parametric Ultrasound (MPUS) as a Decision Support Tool for Patients

    This clinical trial studies how well 3-dimensional multi-parametric ultrasound (3D MPUS) imaging works as a decision-support tool for patients with liver tumors undergoing therapy. Continuous and dynamic imaging of patients undergoing therapy is required to monitor early-phase treatment response. 3D-MPUS is an inexpensive and safe method, which may provide complementary quantitative functional (perfusion) and tissue characterization information to anatomical radiological assessment or blood biomarkers.

    Investigator

    • Aya Kamaya, MD
    Now accepting new patients View Details
  • Dose Optimization and Expansion Study of DFV890 in Adult Patients With Myeloid Diseases

    Study CDFV890G12101 is an open-label, phase 1b, multicenter study with a randomized two-dose optimization part, and a dose expansion part consisting of two groups evaluating DFV890 in patients with myeloid diseases. The purpose of this study is to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, efficacy and recommended dose for single agent DFV890 in patients with lower risk (LR: very low, low or intermediate risk) myelodysplastic syndromes (LR MDS) and lower risk chronic myelomonocytic leukemia (LR CMML).

    Investigator

    • Peter Greenberg
    Now accepting new patients View Details
  • T-DM1 and Tucatinib Compared with T-DM1 Alone in Preventing Relapses in People with High Risk HER2-Positive Breast Cancer, the CompassHER2 RD Trial

    This phase III trial studies how well trastuzumab emtansine (T-DM1) and tucatinib work in preventing breast cancer from coming back (relapsing) in patients with high risk, HER2 positive breast cancer. T-DM1 is a monoclonal antibody, called trastuzumab, linked to a chemotherapy drug, called DM1. Trastuzumab is a form of targeted therapy because it attaches to specific molecules (receptors) on the surface of cancer cells, known as HER2 receptors, and delivers DM1 to kill them. Tucatinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Giving T-DM1 and tucatinib may work better in preventing breast cancer from relapsing in patients with HER2 positive breast cancer compared to T-DM1 alone.

    Investigator

    • Melinda L. Telli, M.D.
    Now accepting new patients View Details
  • Telephone-based Physical Activity Coaching or Self Monitored Physical Activity to Improve Physical Function in Older Adults Who Are Undergoing Surgery for Lung Cancer and Their Caregivers

    This clinical trial compares telephone-based physical activity coaching to self monitored physical activity for improving physical function in older adults who are undergoing surgery for lung cancer and their caregivers. Lung cancer surgery in older adults is associated with functional declines and unique challenges. Performing physical activity around the time of surgery has been shown to improve functional outcomes in patients and exercise programs delivered via telehealth may improve access and convenience for patients and minimize participant burden. Telephone-based physical activity coaching may improve physical functioning for older adults with lung cancer who are undergoing surgery.

    Investigator

    • Natalie Shaubie Lui
    Now accepting new patients View Details
  • Testing Nivolumab and Ipilimumab Immunotherapy With or Without the Targeted Drug Cabozantinib in Recurrent, Metastatic, or Incurable Nasopharyngeal Cancer

    This phase II trial tests how well nivolumab and ipilimumab immunotherapy with or without cabozantinib works in treating patients with nasopharyngeal cancer that has come back (after a period of improvement) (recurrent), has spread from where it first started (primary site) to other places in the body (metastatic), or for which no treatment is currently available (incurable). Immunotherapy with monoclonal antibodies, such as nivolumab and ipilimumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Cabozantinib is in a class of medications called kinase inhibitors. It works by blocking the action of an abnormal protein that signals cancer cells to multiply. This helps slow or stop the spread of cancer cells. Giving immunotherapy with nivolumab and ipilimumab and targeted therapy with cabozantinib may help shrink and stabilize nasopharyngeal cancer.

    Now accepting new patients View Details
  • Telehealth 2.0: Evaluating Effectiveness and Engagement Strategies for CPT-Text for PTSD

    There is a pressing need to increase capacity to treat PTSD related to or exacerbated by the COVID-19 pandemic. Texting-based therapy holds promise to increase capacity and reduce barriers to delivering evidence-based treatments (EBTs), but ongoing engagement in digital mental health interventions is low. This study will compare a texting-based EBT for PTSD to culturally-informed texting-based treatment for PTSD as usual, and it will also compare a unique incentive strategy to typical platform reminders aimed to prevent early discontinuation in therapy. This online study is open to individuals who live in 13 different states.

    Investigator

    • Shannon Wiltsey Stirman
    Now accepting new patients View Details
  • TACTIVE-U: A Study to Learn About the Study Medicine (Vepdegestrant) When Given With Other Medicines in People With Advanced or Metastatic Breast Cancer (Sub-Study B)

    The purpose of this clinical trial is to learn about the safety and effects of the study medicine (called ARV-471) when given together with other medicines for the potential treatment of advanced or metastatic breast cancer.

    This study is seeking participants who have breast cancer that:

    * is advanced, may have spread to other organs (metastatic) and cannot be fully treated by surgery or radiation therapy* is sensitive to hormonal therapy (it is called estrogen receptor positive); and* is no longer responding to previous treatments

    This study is divided into separate sub-studies.

    For Sub-Study B:

    All participants will receive ARV-471 and a medicine called ribociclib. ARV-471 and ribociclib will be given at the same time by mouth, at home, 1 time a day.

    The experiences of people receiving the study medicine will be examined. This will help determine if the study medicine is safe and effective.

    Participants will continue to take ARV-471 and ribociclib until their cancer is no longer responding, or side effects become too severe. They will have visits at the study clinic about every 4 weeks.

    Investigator

    • Melinda L. Telli, M.D.
    Now accepting new patients View Details
  • TACTIVE-U: A Study to Learn About the Study Medicine (Vepdegestrant) When Given With Other Medicines in People With Advanced or Metastatic Breast Cancer. (Sub-Study C)

    The purpose of this study is to learn about the safety and effects of the study medicine called vepdegestrant. The safety and effects of vepdegestrant will be see when given with other medicines. Vepdegestrant is studied to see if it can be a possible treatment for advanced metastatic breast cancer. This type of cancer would have spread from where it started (breast) to other parts of the body and would be tough to treat.

    The study is seeking for participants who have breast cancer that:

    * is hard to treat (advanced) and may have spread to other organs (metastatic). is sensitive to hormonal therapy (it is called estrogen receptor positive).* is no longer responding to treatments taken before starting this study.

    This study is divided into separate sub-studies.

    For Sub-Study C:

    All the participants will receive vepdegestrant and a medicine called samuraciclib.

    Vepdegestrant and samuraciclib will be taken once in a day by mouth. The medicines will be taken at home. The experience of people receiving the study medicines will be studied. This will help see if the study medicine is safe and effective.

    Participant will continue to take vepdegestrant and samuraciclib until:

    * their cancer is no longer responding, or* side effects become too severe.

    They will have visits at the study clinic about every 4 weeks.

    Investigator

    • Melinda L. Telli, M.D.
    Now accepting new patients View Details
  • Type 1 Diabetes and the Brain in Children

    The investigators have previously studied a group of young children with T1D using brain MRI, age-appropriate neurocognitive testing and continuous glucose monitoring, followed for 18 months. The investigators observed significant differences in gray matter volumes and white matter microstructure in the children with diabetes as compared to controls. These differences appeared to increase over time, with slower rates of brain growth in the T1D group (Mazelli, et al, Diabetes 2014; Barnea-Goraly, et al, Diabetes Care 2014; Mauras, et al, Diabetes 2015). In this new protocol the investigators will include the same children with T1D and healthy controls previously studied and recruit new similar subjects to replace those lost by attrition. The investigators will be using structural and functional brain MRI, neurocognitive testing and measures of glycemic control, to determine if changes in the brain persist or worsen over longitudinal follow up, and whether these changes are associated with measures of glycemic control and neurocognitive metrics as these children grow and progress through puberty.

    Investigator

    • Allan L. Reiss
    Now accepting new patients View Details
  • The Myelin Disorders Biorepository Project

    The Myelin Disorders Biorepository Project (MDBP) seeks to collect and analyze clinical data and biological samples from leukodystrophy patients worldwide to support ongoing and future research projects. The MDBP is one of the world's largest leukodystrophy biorepositories, having enrolled nearly 2,000 affected individuals since it was launched over a decade ago.

    Researchers working in the biorepository hope to use these materials to uncover new genetic etiologies for various leukodystrophies, develop biomarkers for use in future clinical trials, and better understand the natural history of these disorders. The knowledge gained from these efforts may help improve the diagnostic tools and treatment options available to patients in the future.

    Investigator

    • Keith Van Haren, MD
    Now accepting new patients View Details
  • Trial of Zolpidem for Sleep in Children with Autism

    The purpose of this study is to examine the effect of zolpidem on sleep in children and adolescents with Autism Spectrum Disorder (ASD). Zolpidem is a nonbenzodiazepine GABAa receptor agonist drug that acts as a hypnotic. To accomplish this, the investigators will use a randomized double-blind placebo-controlled crossover 8-week study design to examine the effect of zolpidem on sleep physiology as assessed by polysomnography (PSG), actigraphy, circadian rhythm, and clinical measures.

    Investigator

    • Antonio Hardan, M.D.
    Now accepting new patients View Details
  • Trial of Acebilustat for the Treatment of Upper Arm Lymphedema

    This study is designed to investigate the response of unilateral upper extremity (arm) lymphedema, during pharmacologic treatment of lymphedema with oral placebo and oral acebilustat. Participants will receive "study drug" (Acebilustat or placebo), for 9 months. For 3 of these months, the participant will receive placebo; for 6 of these months, the participant will receive active ingredient, acebilustat. The study is blinded which means that the participant will not be told which study pill they are taking.

    Investigators

    • Stanley G. Rockson, MD
    • Mark Nicolls
    Now accepting new patients View Details
  • Testing Multi-Level Remote Physical Activity Interventions in a National Sample of Older Women: The WHISH EnCore Trial

    While older women are disproportionately affected by chronic diseases and conditions associated with aging, including both physical and cognitive impairments, that can be alleviated or delayed by regular physical activity, few physical activity programs have been developed specifically with their needs in mind. This research aims to evaluate, in insufficiently active older women from the national WHISH pragmatic trial, the effects of a technology-driven "citizen science" approach to environmental physical activity barriers called Our Voice plus the ongoing "light-touch" remote physical activity educational program, compared to the "light-touch" remote physical activity educational program plus a control educational intervention that creates awareness around human and planetary health. This study will add important information on the benefits and trade-offs of combining these remotely delivered and practical behavioral health approaches to promote physical and cognitive health for the fast-expanding demographic group of U.S. older women.

    Investigator

    • Abby C. King
    Now accepting new patients View Details
  • Study to Evaluate Percussive Ventilation Breathhold to Improve Lung Stereotactic Ablative Radiotherapy

    The purpose of this research is to evaluate and determine the rate of successful administration of Percussive ventilation breathhold (PVB) to allow for more accurate radiotherapy for lung tumors.

    Now accepting new patients View Details
  • Study of Axicabtagene Ciloleucel Versus Standard of Care Therapy in Participants With Relapsed/Refractory Follicular Lymphoma

    The goal of this clinical study is test how well the study drug, axicabtagene ciloleucel, works in participants with relapsed/refractory follicular lymphoma

    Investigator

    • Matthew Frank
    Now accepting new patients View Details
  • Safety and Efficacy Study of Epcoritamab in Subjects With Relapsed/Refractory Chronic Lymphocytic Leukemia and Richter's Syndrome

    The study is a global, multi-center safety and efficacy trial of epcoritamab, an antibody also known as EPKINLY™ and GEN3013 (DuoBody®-CD3xCD20). Epcoritamab will either be studied as:

    * Monotherapy, or* Combination therapy:

    * epcoritamab + venetoclax * epcoritamab + lenalidomide * epcoritamab + R-CHOP (i.e., rituximab, cyclophosphamide, doxorubicin hydrochloride, vincristine (Oncovin®) and prednisone).

    The study includes patients with relapsed/refractory chronic lymphocytic leukemia (R/R CLL)/small lymphocytic lymphoma (SLL) and patients with Richter's Syndrome (RS).

    Study participants with R/R CLL/SLL are treated either with epcoritamab as monotherapy or epcoritamab + venetoclax. Study participants with RS are treated either with epcoritamab as monotherapy or epcoritamab + lenalidomide or epcoritamab + R-CHOP. The trial consists of two parts, a dose-escalation phase (phase Ib) and an expansion phase (phase II). Patients with RS are only included in the expansion phase.

    Epcoritamab will be injected subcutaneously (under the skin). Standard-of-care and combination treatments (venetoclax, lenalidomide, and R-CHOP) will be given either orally (by mouth) or intravenously (in a vein).

    Study details include:

    * Study duration will be up to 5 years.* The treatment duration for each participant will be between 18 months (1.5 years) and 24 months (2 years), depending upon the treatment arm assigned.* The visit frequency will be either weekly, every other week, or monthly, depending upon the part of the study.

    All participants will receive active drug; no one will be given placebo.

    Investigator

    • Bita Fakhri, MD, MPH
    Now accepting new patients View Details
  • Understanding Allergies and Sensitizations in Healthy and Allergic Individuals

    The purpose of this study is to strengthen our ability to accurately diagnose allergies and understand cellular, humoral, genetic components and physiological changes in allergic disease

    Investigator

    • R. Sharon Chinthrajah
    Now accepting new patients View Details
  • Study of Aspirin in Patients with Vestibular Schwannoma

    This is a phase II prospective, randomized, double-blind, longitudinal study evaluating whether the administration of aspirin can delay or slow tumor growth and maintain or improve hearing in VS patients.

    Investigator

    • Konstantina M. Stankovic, MD, PhD, FACS
    Now accepting new patients View Details
  • Study of BDC-3042 as Single Agent and in Combination With Cemiplimab in Patients With Advanced Malignancies

    A first-in-human study using BDC-3042 as a single agent and in combination with cemiplimab in patients with advanced malignancies

    Now accepting new patients View Details
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Pediatric Dermatology Clinical Trials

  • Trametinib in the Treatment of Complicated Extracranial Arterial Venous Malformation

    Arteriovenous malformation (AVM) is a congenital vascular anomaly that progresses throughout life and causes complications including tissue destruction due to rapid overgrowth, bleeding, functional deficits, severe deformity and cardiac failure. Unfortunately, traditional managements have transient benefits with more than 90 recurrence rate within a year. Therefore, there is a significant unmet medical need. The purpose of this study is to assess the safety and efficacy of Trametinib in children and adults with Extracranial Arteriovenous Malformation (AVM).

    Investigators

    • Joyce Teng, MD, PhD
    • Huy M. Do, MD
    Now accepting new patients View Details
  • Characteristics of Patients With Recessive Dystrophic Epidermolysis Bullosa

    Recessive dystrophic epidermolysis bullosa (RDEB) is a disease caused by genetic mutations in the gene for type VII collagen. Patients with RDEB develop large, severely painful blisters and open wounds from minor trauma to their skin. We are screening subjects with RDEB to evaluate characteristics of the subjects and their cells in order to develop new strategies of therapy and determine whether subjects could be candidates for treatment studies.

    Investigators

    • Marius Wernig
    • Anthony Oro, MD, PhD
    • M. Peter Marinkovich, MD
    • Jean Y. Tang MD PhD
    • Paul A. Khavari, MD, PhD
    Now accepting new patients View Details

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