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Conditions: Type 1 Diabetes, Diabetes Insipidus, Diabetes Mellitus, Wolfram Syndrome
Interventions: Not listed
Lead sponsor: Washington University School of Medicine; Other
Eligibility: 1 Day and older
Enrollment: 101 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2012 – 2017
U.S. locations: 1
States / cities: St Louis, Missouri

Conditions: Mitochondrial Disorders, Mitochondrial Genetic Disorders, Mitochondrial Diseases, Disorder of Mitochondrial Respiratory Chain Complexes, Deletion and Duplication of Mitochondrial DNA
Interventions: Not listed
Lead sponsor: Columbia University; Other
Eligibility: Not listed
Enrollment: 1,000 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2011 – 2026
U.S. locations: 16
States / cities: San Diego, California • Stanford, California • Aurora, Colorado + 12 more

Conditions: Retinitis Pigmentosa, Usher Syndrome Type 2, Usher Syndrome Type 3
Interventions: NT-501, Sham; Drug · Procedure
Lead sponsor: Neurotech Pharmaceuticals; Industry
Eligibility: 18 Years to 55 Years
Enrollment: 22 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2019
U.S. locations: 1
States / cities: San Francisco, California

Conditions: Retinitis Pigmentosa
Interventions: N-acetylcysteine, Placebo; Drug
Lead sponsor: Johns Hopkins University; Other
Eligibility: 18 Years to 65 Years
Enrollment: 485 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2030
U.S. locations: 24
States / cities: Davis, California • Los Angeles, California • San Francisco, California + 21 more

Conditions: Retinitis Pigmentosa, Usher Syndrome Type 2, Deaf Blind, Retinal Disease, Eye Diseases, Eye Diseases, Hereditary, Eye Disorders Congenital, Vision Disorders
Interventions: Ultevursen, Sham-procedure; Drug · Other
Lead sponsor: Laboratoires Thea; Industry
Eligibility: 12 Years and older
Enrollment: 7 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2022
U.S. locations: 10
States / cities: San Diego, California • San Francisco, California • Miami, Florida + 7 more

Conditions: Charcot-Marie-Tooth Disease, Charcot-Marie-Tooth, Charcot-Marie-Tooth Disease, Type IA, Charcot-Marie-Tooth Disease Type 2A, Charcot-Marie-Tooth Disease Type 2, Charcot-Marie-Tooth Disease, Type 2C, Charcot-Marie-Tooth Disease Type 2A2B, Charcot-Marie-Tooth Disease Type 2B2, Charcot-Marie-Tooth Disease Type 2A1, Charcot-Marie-Tooth Disease Type 4B1, Charcot-Marie-Tooth Disease, Type IB, Charcot-Marie-Tooth Disease Type 2B1, Charcot-Marie-Tooth Disease Type 2U (Diagnosis), Charcot-Marie-Tooth Disease Type 4A, Charcot-Marie-Tooth Disease, Type 4A, Axonal Form, Charcot-Marie-Tooth Disease Type 2A2A, Charcot-Marie-Tooth Disease Type 2S (Disorder), Charcot-Marie-Tooth Disease and Deafness, Charcot-Marie-Tooth Disease Type 4B2, Charcot-Marie-Tooth Disease Type 4H, Charcot-Marie-Tooth Disease Type 1F, Charcot-Marie-Tooth Disease Type 4C, Charcot-Marie-Tooth Disease Type 4E, Charcot-Marie-Tooth Disease Type 1D, Charcot-Marie-Tooth Disease Type 2Q (Diagnosis), Charcot-Marie-Tooth Disease Type 2A2, Charcot-Marie-Tooth Disease Type 2N (Diagnosis), Charcot-Marie-Tooth Disease Type 2B5, Charcot-Marie-Tooth Disease Type 2D, Charcot-Marie-Tooth Disease Type 4D, Charcot-Marie-Tooth Disease Type 2K, Charcot-Marie-Tooth Disease Type 2L (Diagnosis), Charcot-Marie-Tooth Disease Type 2T, Charcot-Marie-Tooth Disease Type 2I, Charcot-Marie-Tooth Disease Type 2J, Charcot-Marie-Tooth Disease Type 2E, Charcot-Marie-Tooth Disease Type 2G, Charcot-Marie-Tooth Disease Type 1C, Charcot-Marie-Tooth Disease Type 2R, Charcot-Marie-Tooth Disease Type 2O (Diagnosis), Charcot-Marie-Tooth Disease Type 2M, Charcot-Marie-Tooth Disease Type 2P, Charcot-Marie-Tooth Disease Type 2Y, Charcot-Marie-Tooth Disease Type 4F (Diagnosis), Charcot-Marie-Tooth Disease Type 4B3, Charcot-Marie-Tooth Disease Type 2H, HNPP, X-Linked Charcot-Marie-Tooth Disease, Charcot Marie Tooth Disease (CMT)
Interventions: Not listed
Lead sponsor: Hereditary Neuropathy Foundation; Government
Eligibility: Not listed
Enrollment: 10,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2029
U.S. locations: 1
States / cities: New York, New York

Conditions: Hearing and Vision Loss
Interventions: Fitting algorithm; Other
Lead sponsor: University of Minnesota; Other
Eligibility: 18 Years to 80 Years
Enrollment: 41 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2023
U.S. locations: 1
States / cities: Minneapolis, Minnesota

Conditions: Deafness, Blindness
Interventions: DSL Workshop; Behavioral
Lead sponsor: US Department of Veterans Affairs; Federal
Eligibility: 55 Years and older
Enrollment: 13 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2013
U.S. locations: 2
States / cities: Decatur, Georgia • Portland, Oregon

Conditions: Deafness, Kidney Diseases, Kidney Neoplasms, Neoplasms, Retinal Diseases
Interventions: Not listed
Lead sponsor: National Institute of Neurological Disorders and Stroke (NINDS); NIH
Eligibility: Not listed
Enrollment: 75 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1997 – 2000
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Smith-Lemli-Opitz Syndrome, Cone-Rod Dystrophy, Hearing Loss
Interventions: Antioxidants, Cholesterol; Drug
Lead sponsor: University of Colorado, Denver; Other
Eligibility: Up to 65 Years
Enrollment: 100 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2025
U.S. locations: 1
States / cities: Aurora, Colorado

Conditions: Retinal Disease, Age-Related Macular Degeneration, Retinitis Pigmentosa, Stargardt Disease, Optic Neuropathy, Nonarteritic Ischemic Optic Neuropathy, Optic Atrophy, Optic Nerve Disease, Glaucoma, Leber Hereditary Optic Neuropathy, Blindness, Vision Loss Night, Vision Loss Partial, Vision, Low, Retinopathy, Maculopathy, Macular Degeneration, Retina Atrophy
Interventions: Arm 1; Procedure
Lead sponsor: MD Stem Cells; Industry
Eligibility: 18 Years and older
Enrollment: 500 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2028
U.S. locations: 2
States / cities: Westport, Connecticut • Coral Springs, Florida

Conditions: Retinal Degeneration, Retinitis Pigmentosa, Eye Diseases, Hereditary
Interventions: Not listed
Lead sponsor: Jaeb Center for Health Research; Other
Eligibility: 8 Years and older
Enrollment: 44 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2027
U.S. locations: 3
States / cities: San Francisco, California • Baltimore, Maryland • Durham, North Carolina

Conditions: Craniosynostosis, Muenke Syndrome
Interventions: Not listed
Lead sponsor: National Human Genome Research Institute (NHGRI); NIH
Eligibility: 1 Month and older
Enrollment: 137 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2020
U.S. locations: 2
States / cities: Washington D.C., District of Columbia • Bethesda, Maryland

Conditions: Usher Syndrome, Retinitis Pigmentosa
Interventions: SAR421869; Drug
Lead sponsor: Sanofi; Industry
Eligibility: 6 Years and older
Enrollment: 9 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2019
U.S. locations: 1
States / cities: Portland, Oregon

Conditions: Usher Syndrome, Cochlear Implantation
Interventions: Experimental manipulation of stimulation parameters; Other
Lead sponsor: Ohio State University; Other
Eligibility: 1 Year to 85 Years
Enrollment: 29 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2025
U.S. locations: 4
States / cities: Boston, Massachusetts • Columbus, Ohio • Nashville, Tennessee

Conditions: Wolfram Syndrome, Diabetes Mellitus, Optic Nerve Atrophy, Deafness, Diabetes Insipidus, Ataxia
Interventions: Not listed
Lead sponsor: Washington University School of Medicine; Other
Eligibility: 0 Years and older
Enrollment: 5,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2027
U.S. locations: 1
States / cities: St Louis, Missouri

Conditions: Primary Photoreceptor Disease, Retinitis Pigmentosa (RP), Usher Syndrome, Inherited Retinal Disease (IRD), Rod-Cone Dystrophy, Rod-Cone Disease, Retinal Degeneration, Cone-Rod Disease (C-RD), Cone-Rod Dystrophy
Interventions: OpCT-001; Biological
Lead sponsor: BlueRock Therapeutics; Industry
Eligibility: 18 Years and older
Enrollment: 54 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2030
U.S. locations: 5
States / cities: Los Angeles, California • Miami, Florida • Philadelphia, Pennsylvania + 2 more

Conditions: Metabolism, Inborn Errors, Hearing Loss, Hereditary Disease
Interventions: Well infant, whole exome sequencing, Diagnosed, whole exome sequencing; Genetic
Lead sponsor: University of North Carolina, Chapel Hill; Other
Eligibility: 1 Hour to 5 Years
Enrollment: 106 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2016 – 2019
U.S. locations: 1
States / cities: Chapel Hill, North Carolina

Conditions: Neonatal Hearing Loss
Interventions: Medial-Olivocochlear Reflex (MOCR) Strength Measurement; Diagnostic Test
Lead sponsor: Intelligent Hearing Systems; Industry
Eligibility: Not listed
Enrollment: 236 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2020 – 2023
U.S. locations: 1
States / cities: Nashville, Tennessee

Conditions: Retinal Degeneration, Age-Related Macular Degeneration, Retinitis Pigmentosa, Hydroxychloroquine Retinopathy, Usher Syndromes, Late-Onset Retinal Degeneration, Cone Dystrophy, Cone Rod Dystrophy, Rod Cone Dystrophy, Rod Dystrophy
Interventions: Adaptive optics imaging; Device
Lead sponsor: Food and Drug Administration (FDA); Federal
Eligibility: 21 Years and older
Enrollment: 100 participants
Timeline: 2021 – 2028
U.S. locations: 2
States / cities: Bethesda, Maryland • Silver Spring, Maryland

Conditions: Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia, Asparagine Synthetase Deficiency, CHARGE Syndrome, Early Infantile Epileptic Encephalopathy, FOXG1 Syndrome, KBG Syndrome, Noonan Syndrome, Severe Hemophilia A, Short Bowel Syndrome, Beta-Propeller Protein-Associated Neurodegeneration, Brain Injury of Prematurity With Periventricular Leukomalacia, Chromosome 17p13.3 Microdeletion Syndrome, Chromosome 1q43-1q44 Deletion, Cockayne Syndrome, Congenital Diaphragmatic Hernia, End-Stage Renal Disease With Cloacal Anomaly, Mitochondrial Depletion Disorder, Severe Factor VII Deficiency
Interventions: Family Centered pediatric palliative care for family caregivers of children with rare diseases.; Behavioral
Lead sponsor: Children's National Research Institute; Other
Eligibility: 12 Months to 99 Years
Enrollment: 480 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2025 – 2029
U.S. locations: 1
States / cities: Washington D.C., District of Columbia

Conditions: Behavior, Communication, Language
Interventions: TMW-Newborn Intervention video, Safe Sleep for your Baby (SIDS) educational video; Other
Lead sponsor: University of Chicago; Other
Eligibility: 18 Years and older · Female only
Enrollment: 694 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2015 – 2016
U.S. locations: 2
States / cities: Chicago, Illinois

Conditions: Wolfram Syndrome, Diabetes Mellitus, Optic Nerve Atrophy, Ataxia
Interventions: dantrolene sodium; Drug
Lead sponsor: Washington University School of Medicine; Other
Eligibility: 5 Years to 60 Years
Enrollment: 21 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2023
U.S. locations: 1
States / cities: St Louis, Missouri

Conditions: Retinitis Pigmentosa, Usher Syndrome Type 2, Deaf Blind, Retinal Disease, Eye Diseases, Eye Diseases, Hereditary, Eye Disorders Congenital, Vision Disorders
Interventions: QR-421a, Sham-procedure; Drug · Other
Lead sponsor: Laboratoires Thea; Industry
Eligibility: 12 Years and older
Enrollment: 5 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2022
U.S. locations: 2
States / cities: Dallas, Texas • Madison, Wisconsin

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Tracking Neurodegeneration in Early Wolfram Syndrome

North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC)

Retinal Imaging in CNTF -Releasing Encapsulated Cell Implant Treated Patients for Early-stage Retinitis Pigmentosa

Oral N-acetylcysteine for Retinitis Pigmentosa

Study to Evaluate the Efficacy Safety and Tolerability of Ultevursen in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene (Sirius)

Natural History Study for Charcot Marie Tooth Disease

Validating a Self-fitting Hearing Aid

Workshops for Veterans With Vision and Hearing Loss

Endolymphatic Sac Tumors in a Population of Patients With Von Hippel-Lindau Disease:The Natural History and Pathobiology, and a Prospective Non-Randomized Clinical Trial of Hearing Preservation Surgery in Patients With Early Stage Endolymphatic Sac Tumors

Cholesterol and Antioxidant Treatment in Patients With Smith-Lemli-Opitz Syndrome (SLOS)

Stem Cell Ophthalmology Treatment Study II

Rate of Progression of PCDH15-Related Retinal Degeneration in Usher Syndrome 1F

Clinical Study of Muenke Syndrome (FGFR3-Related Craniosynostosis)

Study of SAR421869 in Participants With Retinitis Pigmentosa Associated With Usher Syndrome Type 1B

Auditory Neural Function in Implanted Patients With Usher Syndrome

Wolfram Syndrome and WFS1-related Disorders International Registry and Clinical Study

A Study to Investigate the Safety of OpCT-001 in Adults Who Have Primary Photoreceptor Disease (CLARICO)

North Carolina Newborn Exome Sequencing for Universal Screening

Otoacoustic Emission Suppression Study

Adaptive Optics Imaging of Outer Retinal Diseases

Palliative Care Needs of Children With Rare Diseases and Their Families

Pilot Study of Novel Postpartum Educational Video Intervention

A Clinical Trial of Dantrolene Sodium in Pediatric and Adult Patients With Wolfram Syndrome

Study to Evaluate the Efficacy Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene With Early to Moderate Vision Loss (Celeste)