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Conditions: Tuberous Sclerosis, Lymphangioleiomyomatosis
Interventions: RAD001; Drug
Lead sponsor: Children's Hospital Medical Center, Cincinnati; Other
Eligibility: 18 Years to 65 Years
Enrollment: 36 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2013
U.S. locations: 1
States / cities: Cincinnati, Ohio

Conditions: HSP, Hereditary Spastic Paraplegia, Hereditary Spastic Paraparesis, Hereditary Spastic Paraplegia Type 50, Hereditary Spastic Paraplegia Type 47, Hereditary Spastic Paraplegia Type 51, Hereditary Spastic Paraplegia Type 52, SPG47, AP4B1, Neurogenetic Disorders, Neurodevelopmental Conditions, Movement Disorders, Gene Therapy
Interventions: BFB-101 (AAV9-CBh-AP4B1); Biological
Lead sponsor: BlackfinBio Ltd; Industry
Eligibility: 12 Months to 60 Months
Enrollment: 5 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2032
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Breast Cancer
Interventions: Alpelisib, Fulvestrant, Metformin XR, Dapagliflozin + metformin XR, Dapagliflozin; Drug
Lead sponsor: Novartis Pharmaceuticals; Industry
Eligibility: 18 Years and older
Enrollment: 2 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2023
U.S. locations: 1
States / cities: St Louis, Missouri

Conditions: MPN (Myeloproliferative Neoplasms)
Interventions: Parsaclisib, Ruxolitinib; Drug
Lead sponsor: Incyte Corporation; Industry
Eligibility: 18 Years and older
Enrollment: 74 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2022
U.S. locations: 39
States / cities: Birmingham, Alabama • Scottsdale, Arizona • Berkeley, California + 33 more

Conditions: PIK3CA-Related Overgrowth Spectrum (PROS)/Proteus Syndrome (PS)
Interventions: Miransertib; Drug
Lead sponsor: Merck Sharp & Dohme LLC; Industry
Eligibility: 2 Years to 120 Years
Enrollment: 60 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2030
U.S. locations: 5
States / cities: Atlanta, Georgia • Boston, Massachusetts • Cincinnati, Ohio + 2 more

Conditions: Advanced Solid Tumor, Advanced Solid Malignancies
Interventions: PRJ1-3024; Drug
Lead sponsor: Zhuhai Yufan Biotechnologies Co., Ltd; Industry
Eligibility: 18 Years and older
Enrollment: 39 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2026
U.S. locations: 6
States / cities: Denver, Colorado • Orlando, Florida • Cincinnati, Ohio + 3 more

Conditions: Neonatal Brachial Plexus Palsy
Interventions: Standard NMES device, Sham NMES device; Device
Lead sponsor: University of Michigan; Other
Eligibility: 3 Months to 9 Months
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2018
U.S. locations: 1
States / cities: Ann Arbor, Michigan

Conditions: Renal Transplant
Interventions: Arm 1 Everolimus/Reduced dose tacrolimus; Drug
Lead sponsor: University of Toledo Health Science Campus; Other
Eligibility: 18 Years and older
Enrollment: 55 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2020
U.S. locations: 1
States / cities: Toledo, Ohio

Conditions: Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC), Amyloidosis, Hereditary, Amyloid Neuropathies, Familial, Amyloid Neuropathies, Amyloidosis, Hereditary, Transthyretin-Related, Familial Transthyretin Cardiac Amyloidosis
Interventions: Revusiran (ALN-TTRSC), Sterile Normal Saline (0.9% NaCl); Drug
Lead sponsor: Alnylam Pharmaceuticals; Industry
Eligibility: 18 Years to 90 Years
Enrollment: 206 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2017
U.S. locations: 41
States / cities: Bakersfield, California • Beverly Hills, California • La Mesa, California + 30 more

Conditions: Metastatic Castrate Resistant Prostate Cancer, BARD1, BRCA1, BRCA2, BRIP1, CHEK1, FANCL, PALB2, RAD51B, RAD51C, RAD51D, or RAD54L Mutations
Interventions: Carboplatin, Olaparib; Drug
Lead sponsor: VA Office of Research and Development; Federal
Eligibility: 18 Years and older · Male only
Enrollment: 100 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2027
U.S. locations: 18
States / cities: West Los Angeles, California • Aurora, Colorado • Washington D.C., District of Columbia + 15 more

Conditions: Non-Small Cell Lung Cancer
Interventions: M1774, Cemiplimab; Drug
Lead sponsor: EMD Serono Research & Development Institute, Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 61 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2026
U.S. locations: 6
States / cities: Santa Monica, California • Pittsburgh, Pennsylvania • Knoxville, Tennessee + 2 more

Conditions: Solid Tumors
Interventions: Cobimetinib, Pictilisib; Drug
Lead sponsor: Genentech, Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 178 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2014
U.S. locations: 6
States / cities: Baltimore, Maryland • Boston, Massachusetts • Detroit, Michigan + 2 more

Conditions: MELAS Syndrome, Lebers Hereditory Optic Neuropathy With Extra Ocular Symptoms (LHON-Plus)
Interventions: Glycerol Tributyrate; Drug
Lead sponsor: George Washington University; Other
Eligibility: 18 Years to 65 Years
Enrollment: 24 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2028
U.S. locations: 1
States / cities: Washington D.C., District of Columbia

Conditions: Charcot-Marie-Tooth Neuropathy Type 1A
Interventions: scAAV1.tMCK.NTF3; Drug
Lead sponsor: Nationwide Children's Hospital; Other
Eligibility: 18 Years to 35 Years
Enrollment: 3 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2027 – 2030
U.S. locations: 1
States / cities: Columbus, Ohio

Conditions: Glaucoma, Age Related Macular Degeneration, Optic Neuropathy, Retinal Degeneration
Interventions: nGoggle; Device
Lead sponsor: NGoggle; Industry
Eligibility: 18 Years to 90 Years
Enrollment: 200 participants
Timeline: 2019 – 2024
U.S. locations: 1
States / cities: Durham, North Carolina

Conditions: Solid Tumors, Lung Cancer
Interventions: BKM120, Everolimus; Drug
Lead sponsor: Emory University; Other
Eligibility: 18 Years and older
Enrollment: 43 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2017
U.S. locations: 1
States / cities: Atlanta, Georgia

Conditions: Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency, Carnitine Palmitoyltransferase Deficiencies (CPT1, CPT2), Mitochondrial Trifunctional Protein Deficiency, Long-chain Hydroxyacyl-CoA Dehydrogenase Deficiency, Glycogen Storage Disorders, Pyruvate Carboxylase Deficiency Disease, ACYL-CoA DEHYDROGENASE FAMILY, MEMBER 9, DEFICIENCY of, Barth Syndrome
Interventions: triheptanoin; Drug
Lead sponsor: Jerry Vockley, MD, PhD; Other
Eligibility: 1 Month and older
Healthy volunteers: Healthy volunteers not accepted
U.S. locations: 1
States / cities: Pittsburgh, Pennsylvania

Conditions: Alzheimer Disease, Neurodegenerative Diseases, Cancer
Interventions: Yogic Breathing, Attention Control; Behavioral
Lead sponsor: Medical University of South Carolina; Other
Eligibility: 18 Years and older
Enrollment: 20 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2013
U.S. locations: 1
States / cities: Charleston, South Carolina

Conditions: Charcot-Marie-Tooth Disease, Hereditary Motor and Sensory Neuropathy IV
Interventions: Not listed
Lead sponsor: Neurogene Inc.; Industry
Eligibility: Not listed
Enrollment: 21 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2022
U.S. locations: 2
States / cities: Iowa City, Iowa • Dallas, Texas

Conditions: Charcot-Marie-Tooth Disease, Type Ia
Interventions: Ascorbic acid (Vitamin C), placebo; Drug
Lead sponsor: Wayne State University; Other
Eligibility: 13 Years to 70 Years
Enrollment: 110 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2012
U.S. locations: 3
States / cities: Baltimore, Maryland • Detroit, Michigan • Rochester, New York

Conditions: Facioscapulohumeral Muscular Dystrophy, Charcot-Marie-Tooth Disease
Interventions: ACE-083; Drug
Lead sponsor: Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA; Industry
Eligibility: 18 Years and older
Enrollment: 62 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2020
U.S. locations: 19
States / cities: Orange, California • Sacramento, California • Aurora, Colorado + 16 more

Conditions: Nausea, Vomiting, Familial Dysautonomia
Interventions: Dronabinol, Placebo; Drug · Other
Lead sponsor: NYU Langone Health; Other
Eligibility: 18 Years to 60 Years
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2019
U.S. locations: 1
States / cities: New York, New York

Conditions: Mitochondrial Diseases
Interventions: Cysteamine Bitartrate; Drug
Lead sponsor: Amgen; Industry
Eligibility: 6 Years to 17 Years
Enrollment: 22 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2017
U.S. locations: 5
States / cities: San Diego, California • Stanford, California • Akron, Ohio + 2 more

Conditions: Optic Atrophy, Non-arteritic Anterior Ischemic Optic Neuropathy
Interventions: QPI-1007 at various doses; Drug
Lead sponsor: Quark Pharmaceuticals; Industry
Eligibility: 50 Years and older
Enrollment: 48 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2013
U.S. locations: 22
States / cities: Phoenix, Arizona • Beverly Hills, California • Los Angeles, California + 19 more

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RAD001 Therapy of Angiomyolipomata in Patients With TS Complex and Sporadic LAM

Safety and Efficacy of AAV9/AP4B1 (BFB-101) For Patients With AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47)

Study of Safety and Efficacy of Dapagliflozin + Metformin XR Versus Metformin XR in Participants With HR+, HER2-, Advanced Breast Cancer While on Treatment With Alpelisib and Fulvestrant

A Study of INCB050465 in Combination With Ruxolitinib in Subjects With Myelofibrosis

A Study of the Safety and Tolerability in Participants With PIK3CA-related Overgrowth Spectrum or Proteus Syndrome Who Are Being Treated With Miransertib (MK-7075) in Other Studies (MK-7075-006)

A Phase I Study, Evaluating the Safety, Pharmacokinetics and Efficacy of PRJ1-3024 in Subjects With Advanced Solid Tumors

NMES Efficacy on Patients With NBPP

Evaluation of Calcineurin-inhibitor Reduction With Conversion at 2 Months to Everolimus/Reduced Tacrolimus in Renal Transplant Recipients Following Campath® Induction

ENDEAVOUR: Phase 3 Multicenter Study of Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC)

Carboplatin or Olaparib for BRcA Deficient Prostate Cancer

Tuvusertib (M1774) in Combination With Cemiplimab in Participants With Non-Squamous NSCLC (DDRiver NSCLC 322)

Safety, Tolerability and Pharmacokinetics of Cobimetinib in Combination With Pictilisib in Patients With Locally Advanced or Metastatic Solid Tumors

A Basket Clinical Study to Assess Glycerol Tributyrate in Patients With Mitochondrial Encephalopathy, Lactic Acidosis, Stroke-like Episodes (MELAS) or Leber's Hereditary Optic Neuropathy-Plus (LHON-Plus)

Phase I/IIa Trial of scAAV1.tMCK.NTF3 for Treatment of CMT1A

Assessment of Visual Function With a Portable Brain-computer Interface

A Phase I Study of BKM120 and Everolimus in Advanced Solid Malignancies

Compassionate Use of Triheptanoin (C7) for Inherited Disorders of Energy Metabolism

Yogic Breathing Changes Salivary Components

A Natural History Study of Charcot-Marie-Tooth 4J (CMT4J)

High Dose Ascorbic Acid Treatment of CMT1A

Extension Study to Evaluate the Long-Term Effects of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD) and Charcot-Marie Tooth (CMT) Disease Types 1 and X (CMT1 and CMTX)

The Safety, Tolerability and Efficacy of Dronabinol, for the Treatment of Nausea and Vomiting in Familial Dysautonomia

A Long-term Extension of Study RP103-MITO-001 (NCT02023866) to Assess Cysteamine Bitartrate Delayed-release Capsules (RP103) in Children With Inherited Mitochondrial Disease

Safety Study of a Single IVT Injection of QPI-1007 in Chronic Optic Nerve Atrophy and Recent Onset NAION Patients