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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

No trials match your search ""

Psoriasis Clinical Trials

  • Fluorescent Probe VGT-309 to ID Cancerous Colorectal Lesions During Augmented Colonoscopy

    The purpose of this study is to determine the safety and feasibility of VGT-309 for the visualization of colorectal tumors in real-time using near-infrared (NIR) fluorescence endoscopy. In addition, signatures of 50+ biomarkers will be evaluated in biopsies using CODEX multi-plexing.

    Investigators

    • Erqi Pollom
    • George A. Fisher Jr.
    • Shai Friedland
    • Stephan Rogalla, M.D. Ph.D.
    Now accepting new patients View Details
  • International Consortium for Multimodality Phenotyping in Adults With Non-compaction

    Non-compaction cardiomyopathy (NCCM) is a heterogeneous, poorly understood disorder characterized by a prominent inner layer of loose myocardial tissue, and associated with heart failure, stroke, severe rhythm irregularities and death. For a growing population diagnosed with NCCM there is a need for better risk stratification to appropriately allocate (or safely withhold) these impactful preventive measures. The goal of this international consortium is to improve care of patients with non-compaction cardiomyopathy. We hypothesize that comprehensive analysis of clinical, genetic, structural and functional information will improve risk stratification. In addition, we hypothesize that detailed structural analysis will allow for differentiation of pathological and benign patterns of non-compaction. In a large cohort of adult patients with suspected NCCM we will perform in-depth phenotyping, including clinical information, pedigree data, genetics, echocardiography and MRI, and follow patients for up to 3 years. We will apply machine-learning based analytics to develop predictive models and compare their performance to currently used models and treatment criteria. Secondly, in a subset of patients we will perform high-resolution cardiac CT for detailed structural characterization of the myocardial wall. We will investigate associations between myocardial structure and regional contractile function, as assessed by echo and MRI. The aim of this proposal is to identify a structural signature associated with pathological non-compaction and improve developed risk prediction models. Discovery of pathological structural signatures through innovative imaging techniques, in relation to myocardial contractility, will advance our understanding of NCCM.

    Investigators

    • Koen Nieman
    • Matthew Wheeler
    Now accepting new patients View Details
  • Neutrophil and Monocyte Deactivation Via the SeLective CytopheretIc Device - a Randomized Clinical Trial in Acute Kidney Injury

    This randomized, controlled, pivotal study is intended to determine whether up to ten sequential 24-hour treatments with the Selective Cytopheretic Device (SCD) will improve survival in patients with Acute Kidney Injury (AKI) requiring continuous kidney replacement therapy (CKRT) when compared to CKRT alone (standard of care). This study is further intended to determine whether SCD therapy will reduce the duration of maintenance dialysis secondary to AKI. This study will enroll approximately 200 subjects across 30 US sites. Participants will be patients in an intensive care unit (ICU) setting with a diagnosis of AKI requiring CKRT.

    Investigators

    • Glenn M. Chertow
    • Vikram Fielding-Singh
    Now accepting new patients View Details
  • Neoadjuvant ADI-PEG 20 + Ifosfamide + Radiotherapy in Soft Tissue Sarcoma

    In this study, patients with soft tissue sarcoma (STS) will receive ADI-PEG 20 and ifosfamide in combination with radiation as neoadjuvant therapy. In phase I of the study, up to 5 dose levels will be tested to find the recommended phase II dose (RP2D), after which patients enrolling to phase II will be treated at that dose level to assess efficacy.

    Investigator

    • Nam Quoc Bui
    Now accepting new patients View Details
  • Personalized Therapeutic Neuromodulation for Anhedonic Depression

    This study will investigate the anti-anhedonic efficacy of a novel neurostimulation strategy termed accelerated intermittent theta burst stimulation (aiTBS) in participants with treatment resistant depression (TRD).

    Investigator

    • David Spiegel
    Now accepting new patients View Details
  • Long-Term Follow-Up of Patients Who Have Participated in Children's Oncology Group Studies

    This clinical trial keeps track of and collects follow-up information from patients who are currently enrolled on or have participated in a Children's Oncology Group study. Developing a way to keep track of patients who have participated in Children's Oncology Group studies may allow doctors learn more about the long-term effects of cancer treatment and help them reduce problems related to treatment and improve patient quality of life.

    Investigators

    • Neyssa Marina
    • Jay Michael S. Balagtas
    Now accepting new patients View Details
  • PRT for Adolescents With High Functioning Autism

    The purpose of this study is to identify improvement in behavioral and social function and changes in the brain following Pivotal Response Treatment (PRT) for Adolescents in highly verbal adolescents with autism spectrum disorder (ASD).

    Investigator

    • Daniel A. Abrams
    Now accepting new patients View Details
  • Neural Circuit Biomarkers of Transcranial Magnetic Stimulation Study

    This study is currently recruiting Veterans only. The objective of this observational study is to test whether neuroimaging biomarkers of repetitive transcranial magnetic stimulation (TMS) can be prospectively replicated in a large ecologically valid sample. We focus on cognitive network connectivity as a predictive biomarker of the clinical effect of TMS, and as a response biomarker of change with TMS. We address this objective through a pragmatic approach in which we recruit patients undergoing routine clinical care and program evaluation in a Veterans Administration multi-site clinical TMS program.

    Investigator

    • Leanne Williams
    Now accepting new patients View Details
  • Delayed or Upfront Brain RAdiotherapy in Treatment naïve Lung Cancer Patients With Asymptomatic or Minimally Symptomatic Brain Metastases and ALK rEarrangements

    This study will consist of a Phase 1b and Phase 2 portion. The Phase 1b portion will enroll first followed by the Phase 2 portion. Each cycle of treatment = 28 days.

    Subjects will receive alectinib twice daily. Those in the Phase 1b portion will receive alectinib alone. Those in Phase 2 Arm A will receive alectinib alone. Those in Phase 2, Arm B will receive SRS + alectinib.

    A maximum of 25 cycles (2 years) of alectinib may be administered on study.

    Investigator

    • Nathaniel Myall
    Now accepting new patients View Details
  • Sotagliflozin to Slow Kidney Function Decline in Persons with Type 1 Diabetes and Diabetic Kidney Disease

    Powerful new drugs that can prevent or delay end stage kidney disease (ESKD) - so called sodium-glucose cotransporter-2 inhibitors (SGLT2i) - are now available for patients with type 2 diabetes. Whether these drugs have similar effects in patients with type 1 diabetes (T1D) remains unknown because of the few studies in this population, due to concerns about the increase in risk of diabetic ketoacidosis (DKA, a serious, potentially fatal acute complication of diabetes due to the accumulation of substances called ketone bodies) observed with SGLT2i therapy in T1D. One of the few T1D studies conducted to date showed that implementing an enhanced DKA prevention plan can reduce the risk of DKA associated with the SGLT2i sotagliflozin (SOTA) to very low levels. In the present study, a similar DKA prevention program will be used to carry-out a 3-year trial to test the kidney benefit of SOTA in 150 persons with T1D and moderate to advanced DKD. After a 2-month period, during which diabetes care will be standardized and education on monitoring and minimizing DKA implemented, eligible study subjects will be randomly assigned (50/50) to take one tablet of SOTA (200 mg) or a similarly looking inactive tablet (placebo) every day for 3 years followed by 2-months without treatment. Neither the participants nor the study staff will know whether a person was assigned to taking SOTA or the inactive tablet. Kidney function at the end of the study will be compared between the two treatment groups to see whether SOTA prevented kidney function loss in those treated with this drug as compared to those who took the inactive tablet. The DKA prevention program will include participant education, close follow-up with study staff, continuous glucose monitoring, and systematic ketone body self-monitoring with a meter provided by the study. If successful, this study will provide efficacy and safety data that could be used to seek FDA approval of SOTA for the prevention of kidney function decline in patients with T1D and DKD.

    Investigator

    • David Maahs
    Now accepting new patients View Details
  • Dosing of Brentuximab Vedotin for Mycosis Fungoides, Sezary Syndrome Patients

    The purpose of this study is to test any good and bad effects of the study drug called brentuximab vedotin at a lower dose than is FDA-approved.

    Investigator

    • Youn H Kim, MD
    Now accepting new patients View Details
  • Re-Energize Fontan

    Survival of children with single ventricles ("half a heart") beyond the neonatal period has increased dramatically with the staged Fontan palliation. Yet, long-term morbidity remains high. By the age of 40, 50% of Fontan patients will have died or undergone heart transplantation. With \>1,000 Fontan palliations performed in the US annually, there is a burgeoning population of Fontan patients at risk for progressive heart failure and death. Factors that contribute to onset and progression of heart failure in Fontan patients remain incompletely understood. However, it is established that Fontan patients have poor exercise capacity, associated with a greater risk of morbidity and mortality, in addition to decreased muscle mass, abnormal muscle function, and endothelial dysfunction contributing to disease progression. In adult patients with two ventricles and heart failure, reduced exercise capacity, muscle mass, and muscle strength are powerful predictors of poor outcomes, and exercise interventions can not only improve exercise capacity and muscle mass, but also reverse endothelial dysfunction. Limited exercise interventions in children with congenital heart disease have demonstrated that exercise is safe and effective; however, these studies have been conducted in small, heterogeneous groups, and most had few Fontan patients. Furthermore, none of these interventions have studied the impact of exercise on muscle mass or mitochondrial function, or endothelial function. The investigators propose a milestone-driven, randomized controlled trial in pediatric Fontan patients to test the hypothesis that a live-video-supervised exercise (aerobic + resistance) intervention will improve cardiac and physical capacity; muscle mass, strength and function; and endothelial function. The investigators' ultimate goal is the translation of this model to clinical application as an "exercise prescription" to intervene early in pediatric Fontan patients and decrease long-term morbidity and mortality.

    Investigator

    • Seda Tierney
    Now accepting new patients View Details
  • Investigation to Evaluate the Safety and Effectiveness of Cochlear Implantation in Children and Adults With Unilateral Hearing Loss/Single-sided Deafness

    The aim of the study is to assess the continued efficacy and safety of cochlear implantation in participants aged 5 years and above with Unilateral Hearing Loss (UHL)/Single Sided Deafness (SSD) supporting a change indication for use.

    Investigator

    • Jennifer Alyono, MD, MS
    Now accepting new patients View Details
  • Modulating Inhibitory Control Networks in Gambling Disorder With Theta Burst Stimulation

    In this project the investigators propose a randomized double-blind placebo-controlled design in which 40 patients with GD will receive active or sham cTBS to the pre-SMA for 2 weeks. The investigators will combine TMS, multimodal structural and functional MRI and behavioral measures in order to identify circuit-level mechanisms of action and therapeutic targets (connectivity changes that explain clinical improvement) and assess the efficacy of TMS in modulating inhibitory control and symptom severity in this population.

    Now accepting new patients View Details
  • Multiomic Signatures of Microbial Metabolites Following Prebiotic Fiber Supplementation

    The investigators propose a comprehensive, multiomic study that will integrate longitudinal data associating changes in specific gut bacteria and host in response to prebiotic fiber supplementation. These data will guide the development of an integrative biological signature relating bacterial-derived metabolites with biological outcome in the host. The open sharing of data generated by the proposed research represents a significant public resource that will support and accelerate future novel studies.

    Investigator

    • Michael Snyder, Ph.D.
    Now accepting new patients View Details
  • Electrical Stimulation for the Treatment of Glaucoma

    The overall aim of this study is to see whether long-term electrical stimulation with a home-stimulation device works well and is safe for the treatment of open-angle glaucoma. Open-Angle Glaucoma is a disease where the nerves in the back of your eye die off faster than expected regardless of your eye pressure.

    Investigator

    • Jeffrey Goldberg, MD, PhD
    Now accepting new patients View Details
  • Can Neural Network Instability in Schizophrenia Be Improved with a Very Low Carbohydrate Ketogenic Diet?

    Wide ranging cognitive deficits are major drivers of functional decline and poor outcomes in people with schizophrenia (SZ) and bipolar disorder (BD). Medications do not target pathophysiological mechanisms thought to underlie these deficits. In the search for interventions targeting underlying cognitive impairment in SZ and BD, we look comprehensively beyond just the brain and to the potential role of dysfunctional systemic metabolism.

    Disrupted insulin and glucose metabolism are seen in medication-naïve first-episode SZ, suggesting that SZ itself, and not just the medications used to treat it, is associated with risk of Type 2 diabetes, cardiovascular morbidity and mortality, and more generally, accelerated aging. Even young people with SZ have increased risk of metabolic disease and cognitive deficits. Sadly, their life span is shortened by 15-20 years. BD is associated with similar but less severe disruptions in glucose and insulin metabolism and life expectancy.

    Although the human brain is 2% of the body's volume, it consumes over 20% of its energy, and accordingly, the brain is particularly vulnerable to the dysregulation of glucose metabolism seen in SZ and BD. While glucose is considered to be the brain's default fuel, ketones provide 27% more free energy and are a major source of energy for the brain. Ketones prevent or improve various age-associated diseases, and a ketogenic diet (70% fat, 20% protein, 10% carbohydrates) has been posited as an anti-aging and dementia antidote.

    The premise of the work is based on recent evidence that ketogenic diets improve dynamic neural network instability, related to cognitive deficits, aging, and Type 2 diabetes (Mujica-Parodi et al., Proc Natl Acad Sci U S A. 2020;117(11):6170-7.). The rigor of the work rests on findings of (1) poor cerebral glucose homeostasis in SZ and BD, (2) neural network instability in SZ and BD, and (3) direct effects of ketosis on network instability. Unknown is whether ketogenic diets can improve network instability in people with SZ and BD.

    Investigator

    • Shebani Sethi MD, ABOM
    Now accepting new patients View Details
  • Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease

    This study is a first-in-human, single-arm, open-label Phase I/II study of nula-cel in approximately 15 participants, diagnosed with severe Sickle Cell Disease. The primary objective is to evaluate safety of the treatment in this patient population, as well as preliminary efficacy and pharmacodynamic data.

    Investigator

    • Matthew Porteus
    Now accepting new patients View Details
  • HLA-Mismatched Unrelated Donor Peripheral Blood Stem Cell Transplantation with Reduced Dose Post Transplantation Cyclophosphamide GvHD Prophylaxis

    The goal of this clinical trial is to determine the effectiveness of Reduced Dose Post-Transplant Cyclophosphamide (PTCy) in patients with hematologic malignancies after receiving an HLA-Mismatched Unrelated Donor (MMUD) . The main question\[s\] it aims to answer are:

    * Does a reduced dose of PTCy reduce the occurrence of infections in the first 100 days after transplant?* Does a reduced dose of PTCy maintain the same level of protection against Graft Versus Host Disease (GvHD) as the standard dose of PTCy?

    Now accepting new patients View Details
  • Copper Cu 64 PSMA I&T PET Imaging in Men With Newly Diagnosed Prostate Cancer

    This is a prospective, open-label Phase 3 study to evaluate copper Cu 64 PSMA I\&T injection for PET/CT imaging in patients with newly diagnosed unfavorable intermediate high-risk, high-risk or very high-risk prostate cancer.

    Investigator

    • Farshad Moradi
    Now accepting new patients View Details
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Dermatology Clinical Trials

  • Fluorescent Probe VGT-309 to ID Cancerous Colorectal Lesions During Augmented Colonoscopy

    The purpose of this study is to determine the safety and feasibility of VGT-309 for the visualization of colorectal tumors in real-time using near-infrared (NIR) fluorescence endoscopy. In addition, signatures of 50+ biomarkers will be evaluated in biopsies using CODEX multi-plexing.

    Investigators

    • Erqi Pollom
    • George A. Fisher Jr.
    • Shai Friedland
    • Stephan Rogalla, M.D. Ph.D.
    Now accepting new patients View Details
  • International Consortium for Multimodality Phenotyping in Adults With Non-compaction

    Non-compaction cardiomyopathy (NCCM) is a heterogeneous, poorly understood disorder characterized by a prominent inner layer of loose myocardial tissue, and associated with heart failure, stroke, severe rhythm irregularities and death. For a growing population diagnosed with NCCM there is a need for better risk stratification to appropriately allocate (or safely withhold) these impactful preventive measures. The goal of this international consortium is to improve care of patients with non-compaction cardiomyopathy. We hypothesize that comprehensive analysis of clinical, genetic, structural and functional information will improve risk stratification. In addition, we hypothesize that detailed structural analysis will allow for differentiation of pathological and benign patterns of non-compaction. In a large cohort of adult patients with suspected NCCM we will perform in-depth phenotyping, including clinical information, pedigree data, genetics, echocardiography and MRI, and follow patients for up to 3 years. We will apply machine-learning based analytics to develop predictive models and compare their performance to currently used models and treatment criteria. Secondly, in a subset of patients we will perform high-resolution cardiac CT for detailed structural characterization of the myocardial wall. We will investigate associations between myocardial structure and regional contractile function, as assessed by echo and MRI. The aim of this proposal is to identify a structural signature associated with pathological non-compaction and improve developed risk prediction models. Discovery of pathological structural signatures through innovative imaging techniques, in relation to myocardial contractility, will advance our understanding of NCCM.

    Investigators

    • Koen Nieman
    • Matthew Wheeler
    Now accepting new patients View Details
  • Neutrophil and Monocyte Deactivation Via the SeLective CytopheretIc Device - a Randomized Clinical Trial in Acute Kidney Injury

    This randomized, controlled, pivotal study is intended to determine whether up to ten sequential 24-hour treatments with the Selective Cytopheretic Device (SCD) will improve survival in patients with Acute Kidney Injury (AKI) requiring continuous kidney replacement therapy (CKRT) when compared to CKRT alone (standard of care). This study is further intended to determine whether SCD therapy will reduce the duration of maintenance dialysis secondary to AKI. This study will enroll approximately 200 subjects across 30 US sites. Participants will be patients in an intensive care unit (ICU) setting with a diagnosis of AKI requiring CKRT.

    Investigators

    • Glenn M. Chertow
    • Vikram Fielding-Singh
    Now accepting new patients View Details
  • Neoadjuvant ADI-PEG 20 + Ifosfamide + Radiotherapy in Soft Tissue Sarcoma

    In this study, patients with soft tissue sarcoma (STS) will receive ADI-PEG 20 and ifosfamide in combination with radiation as neoadjuvant therapy. In phase I of the study, up to 5 dose levels will be tested to find the recommended phase II dose (RP2D), after which patients enrolling to phase II will be treated at that dose level to assess efficacy.

    Investigator

    • Nam Quoc Bui
    Now accepting new patients View Details
  • Personalized Therapeutic Neuromodulation for Anhedonic Depression

    This study will investigate the anti-anhedonic efficacy of a novel neurostimulation strategy termed accelerated intermittent theta burst stimulation (aiTBS) in participants with treatment resistant depression (TRD).

    Investigator

    • David Spiegel
    Now accepting new patients View Details
  • Long-Term Follow-Up of Patients Who Have Participated in Children's Oncology Group Studies

    This clinical trial keeps track of and collects follow-up information from patients who are currently enrolled on or have participated in a Children's Oncology Group study. Developing a way to keep track of patients who have participated in Children's Oncology Group studies may allow doctors learn more about the long-term effects of cancer treatment and help them reduce problems related to treatment and improve patient quality of life.

    Investigators

    • Neyssa Marina
    • Jay Michael S. Balagtas
    Now accepting new patients View Details
  • PRT for Adolescents With High Functioning Autism

    The purpose of this study is to identify improvement in behavioral and social function and changes in the brain following Pivotal Response Treatment (PRT) for Adolescents in highly verbal adolescents with autism spectrum disorder (ASD).

    Investigator

    • Daniel A. Abrams
    Now accepting new patients View Details
  • Neural Circuit Biomarkers of Transcranial Magnetic Stimulation Study

    This study is currently recruiting Veterans only. The objective of this observational study is to test whether neuroimaging biomarkers of repetitive transcranial magnetic stimulation (TMS) can be prospectively replicated in a large ecologically valid sample. We focus on cognitive network connectivity as a predictive biomarker of the clinical effect of TMS, and as a response biomarker of change with TMS. We address this objective through a pragmatic approach in which we recruit patients undergoing routine clinical care and program evaluation in a Veterans Administration multi-site clinical TMS program.

    Investigator

    • Leanne Williams
    Now accepting new patients View Details
  • Delayed or Upfront Brain RAdiotherapy in Treatment naïve Lung Cancer Patients With Asymptomatic or Minimally Symptomatic Brain Metastases and ALK rEarrangements

    This study will consist of a Phase 1b and Phase 2 portion. The Phase 1b portion will enroll first followed by the Phase 2 portion. Each cycle of treatment = 28 days.

    Subjects will receive alectinib twice daily. Those in the Phase 1b portion will receive alectinib alone. Those in Phase 2 Arm A will receive alectinib alone. Those in Phase 2, Arm B will receive SRS + alectinib.

    A maximum of 25 cycles (2 years) of alectinib may be administered on study.

    Investigator

    • Nathaniel Myall
    Now accepting new patients View Details
  • Sotagliflozin to Slow Kidney Function Decline in Persons with Type 1 Diabetes and Diabetic Kidney Disease

    Powerful new drugs that can prevent or delay end stage kidney disease (ESKD) - so called sodium-glucose cotransporter-2 inhibitors (SGLT2i) - are now available for patients with type 2 diabetes. Whether these drugs have similar effects in patients with type 1 diabetes (T1D) remains unknown because of the few studies in this population, due to concerns about the increase in risk of diabetic ketoacidosis (DKA, a serious, potentially fatal acute complication of diabetes due to the accumulation of substances called ketone bodies) observed with SGLT2i therapy in T1D. One of the few T1D studies conducted to date showed that implementing an enhanced DKA prevention plan can reduce the risk of DKA associated with the SGLT2i sotagliflozin (SOTA) to very low levels. In the present study, a similar DKA prevention program will be used to carry-out a 3-year trial to test the kidney benefit of SOTA in 150 persons with T1D and moderate to advanced DKD. After a 2-month period, during which diabetes care will be standardized and education on monitoring and minimizing DKA implemented, eligible study subjects will be randomly assigned (50/50) to take one tablet of SOTA (200 mg) or a similarly looking inactive tablet (placebo) every day for 3 years followed by 2-months without treatment. Neither the participants nor the study staff will know whether a person was assigned to taking SOTA or the inactive tablet. Kidney function at the end of the study will be compared between the two treatment groups to see whether SOTA prevented kidney function loss in those treated with this drug as compared to those who took the inactive tablet. The DKA prevention program will include participant education, close follow-up with study staff, continuous glucose monitoring, and systematic ketone body self-monitoring with a meter provided by the study. If successful, this study will provide efficacy and safety data that could be used to seek FDA approval of SOTA for the prevention of kidney function decline in patients with T1D and DKD.

    Investigator

    • David Maahs
    Now accepting new patients View Details
  • Dosing of Brentuximab Vedotin for Mycosis Fungoides, Sezary Syndrome Patients

    The purpose of this study is to test any good and bad effects of the study drug called brentuximab vedotin at a lower dose than is FDA-approved.

    Investigator

    • Youn H Kim, MD
    Now accepting new patients View Details
  • Re-Energize Fontan

    Survival of children with single ventricles ("half a heart") beyond the neonatal period has increased dramatically with the staged Fontan palliation. Yet, long-term morbidity remains high. By the age of 40, 50% of Fontan patients will have died or undergone heart transplantation. With \>1,000 Fontan palliations performed in the US annually, there is a burgeoning population of Fontan patients at risk for progressive heart failure and death. Factors that contribute to onset and progression of heart failure in Fontan patients remain incompletely understood. However, it is established that Fontan patients have poor exercise capacity, associated with a greater risk of morbidity and mortality, in addition to decreased muscle mass, abnormal muscle function, and endothelial dysfunction contributing to disease progression. In adult patients with two ventricles and heart failure, reduced exercise capacity, muscle mass, and muscle strength are powerful predictors of poor outcomes, and exercise interventions can not only improve exercise capacity and muscle mass, but also reverse endothelial dysfunction. Limited exercise interventions in children with congenital heart disease have demonstrated that exercise is safe and effective; however, these studies have been conducted in small, heterogeneous groups, and most had few Fontan patients. Furthermore, none of these interventions have studied the impact of exercise on muscle mass or mitochondrial function, or endothelial function. The investigators propose a milestone-driven, randomized controlled trial in pediatric Fontan patients to test the hypothesis that a live-video-supervised exercise (aerobic + resistance) intervention will improve cardiac and physical capacity; muscle mass, strength and function; and endothelial function. The investigators' ultimate goal is the translation of this model to clinical application as an "exercise prescription" to intervene early in pediatric Fontan patients and decrease long-term morbidity and mortality.

    Investigator

    • Seda Tierney
    Now accepting new patients View Details
  • Investigation to Evaluate the Safety and Effectiveness of Cochlear Implantation in Children and Adults With Unilateral Hearing Loss/Single-sided Deafness

    The aim of the study is to assess the continued efficacy and safety of cochlear implantation in participants aged 5 years and above with Unilateral Hearing Loss (UHL)/Single Sided Deafness (SSD) supporting a change indication for use.

    Investigator

    • Jennifer Alyono, MD, MS
    Now accepting new patients View Details
  • Modulating Inhibitory Control Networks in Gambling Disorder With Theta Burst Stimulation

    In this project the investigators propose a randomized double-blind placebo-controlled design in which 40 patients with GD will receive active or sham cTBS to the pre-SMA for 2 weeks. The investigators will combine TMS, multimodal structural and functional MRI and behavioral measures in order to identify circuit-level mechanisms of action and therapeutic targets (connectivity changes that explain clinical improvement) and assess the efficacy of TMS in modulating inhibitory control and symptom severity in this population.

    Now accepting new patients View Details
  • Multiomic Signatures of Microbial Metabolites Following Prebiotic Fiber Supplementation

    The investigators propose a comprehensive, multiomic study that will integrate longitudinal data associating changes in specific gut bacteria and host in response to prebiotic fiber supplementation. These data will guide the development of an integrative biological signature relating bacterial-derived metabolites with biological outcome in the host. The open sharing of data generated by the proposed research represents a significant public resource that will support and accelerate future novel studies.

    Investigator

    • Michael Snyder, Ph.D.
    Now accepting new patients View Details
  • Electrical Stimulation for the Treatment of Glaucoma

    The overall aim of this study is to see whether long-term electrical stimulation with a home-stimulation device works well and is safe for the treatment of open-angle glaucoma. Open-Angle Glaucoma is a disease where the nerves in the back of your eye die off faster than expected regardless of your eye pressure.

    Investigator

    • Jeffrey Goldberg, MD, PhD
    Now accepting new patients View Details
  • Can Neural Network Instability in Schizophrenia Be Improved with a Very Low Carbohydrate Ketogenic Diet?

    Wide ranging cognitive deficits are major drivers of functional decline and poor outcomes in people with schizophrenia (SZ) and bipolar disorder (BD). Medications do not target pathophysiological mechanisms thought to underlie these deficits. In the search for interventions targeting underlying cognitive impairment in SZ and BD, we look comprehensively beyond just the brain and to the potential role of dysfunctional systemic metabolism.

    Disrupted insulin and glucose metabolism are seen in medication-naïve first-episode SZ, suggesting that SZ itself, and not just the medications used to treat it, is associated with risk of Type 2 diabetes, cardiovascular morbidity and mortality, and more generally, accelerated aging. Even young people with SZ have increased risk of metabolic disease and cognitive deficits. Sadly, their life span is shortened by 15-20 years. BD is associated with similar but less severe disruptions in glucose and insulin metabolism and life expectancy.

    Although the human brain is 2% of the body's volume, it consumes over 20% of its energy, and accordingly, the brain is particularly vulnerable to the dysregulation of glucose metabolism seen in SZ and BD. While glucose is considered to be the brain's default fuel, ketones provide 27% more free energy and are a major source of energy for the brain. Ketones prevent or improve various age-associated diseases, and a ketogenic diet (70% fat, 20% protein, 10% carbohydrates) has been posited as an anti-aging and dementia antidote.

    The premise of the work is based on recent evidence that ketogenic diets improve dynamic neural network instability, related to cognitive deficits, aging, and Type 2 diabetes (Mujica-Parodi et al., Proc Natl Acad Sci U S A. 2020;117(11):6170-7.). The rigor of the work rests on findings of (1) poor cerebral glucose homeostasis in SZ and BD, (2) neural network instability in SZ and BD, and (3) direct effects of ketosis on network instability. Unknown is whether ketogenic diets can improve network instability in people with SZ and BD.

    Investigator

    • Shebani Sethi MD, ABOM
    Now accepting new patients View Details
  • Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease

    This study is a first-in-human, single-arm, open-label Phase I/II study of nula-cel in approximately 15 participants, diagnosed with severe Sickle Cell Disease. The primary objective is to evaluate safety of the treatment in this patient population, as well as preliminary efficacy and pharmacodynamic data.

    Investigator

    • Matthew Porteus
    Now accepting new patients View Details
  • HLA-Mismatched Unrelated Donor Peripheral Blood Stem Cell Transplantation with Reduced Dose Post Transplantation Cyclophosphamide GvHD Prophylaxis

    The goal of this clinical trial is to determine the effectiveness of Reduced Dose Post-Transplant Cyclophosphamide (PTCy) in patients with hematologic malignancies after receiving an HLA-Mismatched Unrelated Donor (MMUD) . The main question\[s\] it aims to answer are:

    * Does a reduced dose of PTCy reduce the occurrence of infections in the first 100 days after transplant?* Does a reduced dose of PTCy maintain the same level of protection against Graft Versus Host Disease (GvHD) as the standard dose of PTCy?

    Now accepting new patients View Details
  • Copper Cu 64 PSMA I&T PET Imaging in Men With Newly Diagnosed Prostate Cancer

    This is a prospective, open-label Phase 3 study to evaluate copper Cu 64 PSMA I\&T injection for PET/CT imaging in patients with newly diagnosed unfavorable intermediate high-risk, high-risk or very high-risk prostate cancer.

    Investigator

    • Farshad Moradi
    Now accepting new patients View Details
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