Education and Training

  • Prospective, Longitudinal Study of the Natural History and Functional Status of Patients With Myotubular Myopathy (MTM)

    This is a prospective, non-interventional, longitudinal study of the natural history and function of approximately 60 patients with MTM from the United States, Canada and Europe. The duration of the study, including the enrollment period, will be 36 months. Data from the study will be used to characterize the disease course of MTM and determine which outcome measures will be the best to assess the efficacy of potential therapies.

    Investigator

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  • Pediatric ACL: Understanding Treatment Options

    Pediatric ACL: Understanding Treatment Outcomes (PLUTO) is a multi-center, prospective cohort study. Specific aims of PLUTO are to evaluate the safety and comparative effectiveness of non-operative treatment, as well as four operative treatments including (1) transphyseal ACL reconstruction (2) partial transphyseal ACL reconstruction, (3) physeal-sparing epiphyseal ACL reconstruction using the Anderson technique, and (4) physeal-sparing ACL reconstruction using the Micheli/Kocher technique in prepubescent and pubescent skeletally immature patients.

    Investigator

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  • Study of Tisagenlecleucel in Combination With Ibrutinib in r/r Diffuse Large B-cell Lymphoma Patients

    A multi-center, open-label, phase Ib study to evaluate the safety and tolerability of the administration of tisagenlecleucel in combination with ibrutinib in patients with r/r DLBCL who have received two or more lines of systemic therapy, including an anti-CD20 and anthracycline based chemotherapy, and who have progressed after or are not candidates for ASCT.

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  • Pazopanib in Imatinib Refractory or Intolerant Gastrointestinal Stromal Tumors (GIST)

    This study is being done to gather information about the safety (any harmful effects) and effectiveness (usefulness) of Pazopanib in the treatment of Gastrointestinal Stroma Tumors (GIST) that cannot be treated by surgery or has spread to other organs. The Food and Drug Administration (FDA) have approved Pazopanib for the treatment of advanced kidney cancer but it is not approved for the treatment of GIST. The investigators hope to learn about the safety and usefulness (effectiveness) of Pazopanib for patients with GIST.

    Primary Objective:

    Non-progression rate based on RECIST criteria (CR+PR+SD)

    Secondary Objectives:

    - Response per Choi criteria

    - 6 month progression-free survival

    - Safety and tolerability

    Investigator

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  • Pilot LDE225 in Locally Advanced or Metastatic BCC + Previously Tx Non-LDE225 Smoothened Inhibitors

    This is a prospective single-center, open label, pilot study to investigate the safety and efficacy of LDE225 in patients with locally advanced or metastatic basal cell carcinoma.

    Primary Objectives:

    • To explore the effects of oral LDE225 on the Progression Free Survival (PFS) of individuals with locally advanced or metastatic BCC who have been previously treated with a non-LDE225 Smo inhibitor.

    Secondary Objectives:

    - To evaluate the effect of oral LDE225 on tumor tissue biomarkers of BCC activation (Gii 1, 2, Patched 1,2 and Ki67) in individuals which are non-na"ive to Smo inhibitors other than LDE225, at baseline and at end-of-treatment

    - To describe adverse effects of oral LDE225 in individuals with a history of non-LDE225 Smo inhibitor usage

    - To assess the overall survival rates of individuals with locally advanced BCC or metastatic BCC who have previously taken a non-LDE225 Smo inhibitor after treatment with LDE225

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  • Safety and Efficacy Study of Treatment of Pathological Fractures in Humerus

    This study will collect safety and performance data of the Photodynamic Bone Stabilization System (PBSS) when used for the treatment of fractures of the humerus secondary to metastatic cancer.

    Investigator

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  • Phase 3 Study of Pexidartinib for Pigmented Villonodular Synovitis (PVNS) or Giant Cell Tumor of the Tendon Sheath (GCT-TS)

    This is a Phase 3 clinical study, which aims to evaluate the effectiveness of an investigational drug called pexidartinib for the treatment of certain tumors for which surgical removal could cause more harm than good.

    The main purpose of this study is to gather information about the investigational drug pexidartinib, which may help to treat tumors of pigmented villonodular synovitis (PVNS) or giant cell tumor of the tendon sheath (GCT-TS).

    The study consists of two parts with a follow-up period. In Part 1, eligible study participants will be assigned to receive either pexidartinib or matching placebo for 24 weeks. A number of assessments will be carried out during the course of the study, including physical examinations, blood tests, imaging studies, electrocardiograms, and questionnaires. MRI scans will be used to evaluate the response of the tumors to the treatment. Some subjects, assigned to placebo in Part 1 transitioned to pexidartinib for Part 2.

    Then a protocol amendment was written to allow only pexidartinib patients to continue into Part 2. Part 2 is a long-term treatment phase in which all participants receive open-label pexidartinib. There was also a follow-up period added to Part 2.

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  • Phase 1/2a Study of SQ3370 in Patients With Advanced Solid Tumors

    The purpose of this study is to evaluate the safety, tolerability, and preliminary activity of SQ3370 in patients with advanced solid tumors.

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  • Stanford Letter or Traditional Advance Directive in Advance Care Planning in Patients Undergoing Bone Marrow Transplant

    The purpose of the proposed research study is to evaluate whether bone marrow transplant patients prefer the Stanford letter advance care planning tool to the standard Advance directive.

    Completion of advance care planning prior to BMT is very important, but not often done. The investigators believe that the Stanford Letter will be preferred by patients and will allow them to feel more comfortable and share more of their wishes with family members and the medical team.

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  • Phase 1/2 Dose Escalation Study in Patients With Relapsed or Refractory Waldenstrom's Macroglobulinemia

    Recent reports have identified a specific oncogenic mutation L265P of the MYD88 gene in approximately 90% of the patients with Waldenström's macroglobulinemia. MYD88 is a key linker protein in the signaling pathway of Toll Like Receptors (TLRs) 7, 8, and 9, and IMO-8400 is an oligonucleotide specifically designed to inhibit TLRs 7,8, and 9. The scientific hypothesis for use of IMO-8400 to treat patients with Waldenström's macroglobulinemia depends on the inhibition of mutant MYD88 signaling in the TLR pathway, thereby interrupting the proliferation of cell populations responsible for the propagation of the disease.

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  • Phase I Rindopepimut After Conventional Radiation in Children w/ Diffuse Intrinsic Pontine Gliomas

    This is a research study of patients with diffuse intrinsic pontine gliomas. We hope to learn about the safety and efficacy of treating pediatric diffuse intrinsic pontine glioma patients with the EGFRvIII peptide vaccine after conventional radiation.

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  • Teplizumab for Prevention of Type 1 Diabetes In Relatives "At-Risk"

    The study will determine whether the anti-CD3 monoclonal antibody, teplizumab, can help to prevent or delay the onset of type 1 diabetes (T1D) in relatives determined to be at very high risk for developing the disease. Teplizumab has been studied in new onset type 1 diabetes for testing of efficacy and safety in previous studies; other studies are currently in progress. The results of previous studies indicate that teplizumab reduces the loss of insulin production during the first year after diagnosis in individuals with type 1 diabetes. The purpose of this study is to determine if teplizumab can interdict the immune process that causes the destruction of insulin secreting beta cells in the pancreas during the "pre-diabetic" state and thereby prevent or delay the onset of type 1 diabetes.

    Investigator

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  • Personalized Integrated Alcohol and Sexual Assault Prevention Among College Students

    Heavy episodic drinking and sexual assault are problematic on college campuses. This study includes a randomized controlled trial of Positive Change (+Change), an integrated alcohol and sexual assault prevention program, compared to an attention-matched control condition across two universities in reducing alcohol use, sexual assault victimization, sexual assault perpetration, and increasing sexual assault bystander intervention. This study will also test the efficacy of +Change plus Booster session, an identical version of +Change delivered 6 months after the baseline, compared to +Change alone in long-term reductions in alcohol use, sexual assault victimization, sexual assault perpetration, and increases in sexual assault bystander intervention. This research is the next step of a NIAAA-funded planning grant (R34AA025691).

    Investigator

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  • Phase II MEDI4736 in Combination With Chemotherapy for First-Line Treatment of Unresectable Mesothelioma

    Patients with pleural mesothelioma that can not be surgically removed will receive durvalumab, in combination with standard chemotherapy of pemetrexed and cisplatin as first-line treatment.

    Durvalumab is a type of drug called a monoclonal antibody (a type of protein). Laboratory tests show that it works by allowing the immune system to detect your cancer and reactivates the immune response. This may help to slow down the growth of cancer or may cause cancer cells to die.

    The purpose of this study is to see whether adding durvalumab to standard chemotherapy will improve overall survival (OS).

    Investigator

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  • Phase 2/3 Study Evaluating the Safety and Efficacy of PTX-022 in Treatment of Adults With Pachyonychia Congenita

    This study evaluates the safety and efficacy of PTX-022, topical rapamycin, in the treatment of adults with moderate to severe Pachyonychia Congenita.

    This study includes four-parts, and if a participant completes all parts, the participant will have received at least 3-months of PTX-022 treatment.

    Investigator

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  • Safety, PK and Biodistribution of 18F-OP-801 in Patients With ALS, AD, MS, PD and Healthy Volunteers

    This is a Phase 1/2 study to evaluate the safety and tolerability of 18F-OP-801 in subjects with ALS, AD, MS, PD and age-matched HVs. 18F-OP-801 is intended as a biomarker for PET imaging of activated microglia and macrophages in regions of neuroinflammation.

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  • Pilot Study in Young Adults to Examine the Kinetics of Changes in the B-cell Repertoire Following TIV Immunization

    The purpose is to investigate B-cell response to the trivalent Influenza Vaccine (TIV) in healthy young adults by vaccinating participants and obtaining blood samples at designated time points before and after vaccination.

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  • Phase I Study of APX005M in Pediatric CNS Tumors

    This phase I trial studies the side effects and best dose of APX005M in treating younger patients with primary malignant central nervous system tumor that is growing, spreading, or getting worse (progressive), or newly diagnosed diffuse intrinsic pontine glioma. APX005M can trigger activation of B cells, monocytes, and dendritic cells and stimulat cytokine release from lymphocytes and monocytes. APX005M can mediate a direct cytotoxic effect on CD40+ tumor cells.

    Investigator

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  • Sarilumab in Patients With Glucocorticoid-Dependent Sarcoidosis

    The purpose of this study is to compare the effectiveness and the safety of sarilumab in patients with glucocorticoid-dependent sarcoidosis.

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  • Phase 1 Nilotinib in Steroid Dependent/Refractory Chronic Graft Versus Host Disease

    PRIMARY OBJECTIVES:

    Determine the safety and tolerability of nilotinib in steroid dependent / refractory cGVHD.

    SECONDARY OBJECTIVES:

    Determine the clinical efficacy of nilotinib in steroid dependent / refractory cGVHD.

    Investigators

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