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Conditions: Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Sensation Disorders, Vision Disorder, Neurological Manifestations, Eye Diseases, Hereditary, Eye Diseases, Eye Disorders Congenital, Retinal Disease
Interventions: sepofarsen, Placebo IVT; Drug · Other
Lead sponsor: Laboratoires Thea; Industry
Eligibility: 6 Years and older
Enrollment: 32 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2028
U.S. locations: 5
States / cities: San Francisco, California • Miami, Florida • Iowa City, Iowa + 2 more

Conditions: Friedreich Ataxia
Interventions: Omaveloxolone Capsules, 2.5 mg, Omaveloxolone Capsules, 5 mg, Omaveloxolone Capsules, 10 mg, Placebo, Omaveloxolone Capsules, 20 mg, Omaveloxolone Capsules, 40 mg, Omaveloxolone Capsules, 80 mg, Omaveloxolone Capsules, 160 mg, Omaveloxolone Capsules, 300 mg, Omaveloxolone Capsules, 150 mg; Drug
Lead sponsor: Biogen; Industry
Eligibility: 16 Years to 40 Years
Enrollment: 172 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2025
U.S. locations: 7
States / cities: Los Angeles, California • Gainesville, Florida • Tampa, Florida + 4 more

Conditions: Colitis, HIV Infections
Interventions: Ganciclovir; Drug
Lead sponsor: Hoffmann-La Roche; Industry
Eligibility: 21 Years and older · Male only
Healthy volunteers: Healthy volunteers not accepted
U.S. locations: 1
States / cities: Palo Alto, California

Conditions: Cytomegalovirus Retinitis, HIV Infections
Interventions: Fomivirsen sodium, Ganciclovir; Drug
Lead sponsor: Ionis Pharmaceuticals, Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 194 participants
Healthy volunteers: Healthy volunteers not accepted
U.S. locations: 18
States / cities: Los Angeles, California • Pasadena, California • San Diego, California + 14 more

Conditions: Mitochondrial Diseases, Mitochondrial Myopathies, Mitochondrial Encephalomyopathies, MELAS, MERRF
Interventions: Promoting Resilience in Stress Management (PRISM), Clinical-focused narrative (CFN); Behavioral
Lead sponsor: Children's Hospital of Philadelphia; Other
Eligibility: 18 Years and older
Enrollment: 16 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2024
U.S. locations: 1
States / cities: Philadelphia, Pennsylvania

Conditions: Friedreich Ataxia
Interventions: DT-216, Placebo; Drug
Lead sponsor: Design Therapeutics, Inc.; Industry
Eligibility: 18 Years to 55 Years
Enrollment: 32 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2023
U.S. locations: 1
States / cities: Eatontown, New Jersey

Conditions: Cytomegalovirus Retinitis, HIV Infections
Interventions: Globulin, Immune, Ganciclovir; Drug
Lead sponsor: American National Red Cross; Other
Eligibility: 18 Years and older
Healthy volunteers: Healthy volunteers not accepted
U.S. locations: 1
States / cities: Washington D.C., District of Columbia

Conditions: Colitis, HIV Infections
Interventions: Glutamic acid hydrochloride, Ganciclovir; Drug
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 13 Years and older
Enrollment: 24 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Ends 1998
U.S. locations: 5
States / cities: Birmingham, Alabama • San Francisco, California • St Louis, Missouri + 2 more

Conditions: Eye Diseases Hereditary, Retinal Disease, Achromatopsia, Bardet-Biedl Syndrome, Bassen-Kornzweig Syndrome, Batten Disease, Best Disease, Choroidal Dystrophy, Choroideremia, Cone Dystrophy, Cone-Rod Dystrophy, Congenital Stationary Night Blindness, Enhanced S-Cone Syndrome, Fundus Albipunctatus, Goldmann-Favre Syndrome, Gyrate Atrophy, Juvenile Macular Degeneration, Kearns-Sayre Syndrome, Leber Congenital Amaurosis, Refsum Syndrome, Retinitis Pigmentosa, Retinitis Punctata Albescens, Retinoschisis, Rod-Cone Dystrophy, Rod Dystrophy, Rod Monochromacy, Stargardt Disease, Usher Syndrome
Interventions: Not listed
Lead sponsor: Foundation Fighting Blindness; Other
Eligibility: Not listed
Enrollment: 20,000 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2014 – 2037
U.S. locations: 1
States / cities: Columbia, Maryland

Conditions: Urea Cycle Disorder, Organic Acidemia, Maple Syrup Urine Disease, Glutaric Acidemia I, Fatty Acid Oxidation Disorder, Hypoxic-Ischemic Encephalopathy
Interventions: Not listed
Lead sponsor: Children's National Research Institute; Other
Eligibility: 7 Years to 18 Years
Enrollment: 24 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2027
U.S. locations: 1
States / cities: Washington D.C., District of Columbia

Conditions: Friedreich Ataxia
Interventions: Methylprednisolone; Drug
Lead sponsor: Children's Hospital of Philadelphia; Other
Eligibility: 5 Years and older
Enrollment: 11 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2018
U.S. locations: 1
States / cities: Philadelphia, Pennsylvania

Conditions: Leigh Syndrome, Leigh Disease, Leigh's Necrotizing Encephalopathy, Subacute Necrotizing Encephalomyopathy, Subacute Necrotizing Encephalomyelopathy
Interventions: Not listed
Lead sponsor: The University of Texas Health Science Center, Houston; Other
Eligibility: 0 Days to 100 Years
Enrollment: 200 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2030
U.S. locations: 1
States / cities: Houston, Texas

Conditions: Friedreich Ataxia
Interventions: Rosuvastatin; Drug
Lead sponsor: Children's Hospital of Philadelphia; Other
Eligibility: 18 Years to 65 Years
Enrollment: 12 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2017
U.S. locations: 1
States / cities: Philadelphia, Pennsylvania

Conditions: Friedreich Ataxia
Interventions: MR; Device
Lead sponsor: University of Minnesota; Other
Eligibility: 18 Years and older
Enrollment: 20 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2017 – 2023
U.S. locations: 1
States / cities: Minneapolis, Minnesota

Conditions: Mucopolysaccharidosis Disorders, Hurler Syndrome, Hunter Syndrome, Maroteaux Lamy Syndrome, Sly Syndrome, Alpha-Mannosidosis, Fucosidosis, Aspartylglucosaminuria, Glycoprotein Metabolic Disorders, Sphingolipidoses, Recessive Leukodystrophies, Globoid Cell Leukodystrophy, Metachromatic Leukodystrophy, Niemann-Pick B, Niemann-Pick C Subtype 2, Sphingomyelin Deficiency, Peroxisomal Disorders, Adrenoleukodystrophy With Cerebral Involvement, Zellweger Syndrome, Neonatal Adrenoleukodystrophy, Infantile Refsum Disease, Acyl-CoA Oxidase Deficiency, D-Bifunctional Enzyme Deficiency, Multifunctional Enzyme Deficiency, Alpha-methylacyl-CoA Racmase Deficiency, Mitochondrial Neurogastrointestingal Encephalopathy, Severe Osteopetrosis, Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation), Inherited Metabolic Disorders
Interventions: Stem Cell Transplantation, IMD Preparative Regimen, Osteopetrosis Only Preparative Regimen, Osteopetrosis Haploidentical Only Preparative Regimen, cALD SR-A (Standard-Risk, Regimen A), cALD SR-B (Standard-Risk, Regimen B), cALD HR-D (High-Risk, Regimen C), cALD HR-D (High-Risk, Regimen D); Biological · Drug
Lead sponsor: Masonic Cancer Center, University of Minnesota; Other
Eligibility: Up to 55 Years
Enrollment: 149 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2029
U.S. locations: 1
States / cities: Minneapolis, Minnesota

Conditions: Barth Syndrome
Interventions: Resistance exercise and protein supplementation; Behavioral
Lead sponsor: Washington University School of Medicine; Other
Eligibility: 15 Years to 35 Years · Male only
Enrollment: 12 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2012 – 2019
U.S. locations: 1
States / cities: St Louis, Missouri

Conditions: Ischemic Optic Neuropathy, Branch Retinal Artery Occlusion, Hemianopia, Leber Hereditary Optic Neuropathy, Acute Zonal Occult Outer Retinopathy
Interventions: LSFG-NAVI; Device
Lead sponsor: Randy Kardon; Other
Eligibility: 18 Years to 99 Years
Enrollment: 500 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2019 – 2028
U.S. locations: 1
States / cities: Iowa City, Iowa

Conditions: MELAS Syndrome
Interventions: Dichloroacetate; Drug
Lead sponsor: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD); NIH
Eligibility: 6 Years and older
Enrollment: 35 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2000
U.S. locations: 1
States / cities: New York, New York

Conditions: Friedreich's Ataxia
Interventions: Low dose cohort, High dose cohort; Drug
Lead sponsor: Biojiva LLC; Industry
Eligibility: 18 Years to 50 Years
Enrollment: 19 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2016
U.S. locations: 2
States / cities: Long Beach, California • Tampa, Florida

Conditions: Cytomegalovirus Retinitis, HIV Infections
Interventions: Ganciclovir; Drug
Lead sponsor: Hoffmann-La Roche; Industry
Eligibility: 13 Years and older
Enrollment: 150 participants
Healthy volunteers: Healthy volunteers not accepted
U.S. locations: 12
States / cities: Berkeley, California • San Francisco, California • Washington D.C., District of Columbia + 6 more

Conditions: Cytomegalovirus Retinitis, HIV Infections
Interventions: Ganciclovir; Drug
Lead sponsor: Hoffmann-La Roche; Industry
Eligibility: 13 Years and older
Healthy volunteers: Healthy volunteers not accepted
U.S. locations: 1
States / cities: Palo Alto, California

Conditions: Metabolic Diseases
Interventions: human hepatocyte transplantation, Preparative Radiation Therapy; Drug · Radiation
Lead sponsor: Ira Fox; Other
Eligibility: Up to 21 Years
Enrollment: 5 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2022
U.S. locations: 1
States / cities: Pittsburgh, Pennsylvania

Conditions: Cytomegalovirus Infections, HIV Infections
Interventions: Ganciclovir; Drug
Lead sponsor: Hoffmann-La Roche; Industry
Eligibility: 13 Years and older
Enrollment: 700 participants
Healthy volunteers: Healthy volunteers not accepted
U.S. locations: 19
States / cities: Berkeley, California • Los Angeles, California • San Diego, California + 12 more

Conditions: Multiple Acyl-CoA Dehydrogenase Deficiency
Interventions: Not listed
Lead sponsor: Icahn School of Medicine at Mount Sinai; Other
Eligibility: Not listed
Enrollment: 50 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2031
U.S. locations: 2
States / cities: Aurora, Colorado • New York, New York

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Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)

A Study to Learn About the Effects and Safety of RTA 408 (Omaveloxolone) in People Aged 16 to 40 With Friedreich's Ataxia

A Study of Ganciclovir in the Treatment of Cytomegalovirus of the Large Intestine in Patients With AIDS

A Randomized Comparison of Intravitreal ISIS 2922 Plus Ganciclovir Versus Ganciclovir as Treatment for Patients With Cytomegalovirus Retinitis ( CMVR )

Promoting Resilience in Stress Management (PRISM) and Clinical-focused Narrative (CFN) Pilot in Adults With Primary Mitochondrial Disease (PMD).

Study to Evaluate Multiple Ascending Dose and Multi-Dose of DT-216 in Adult Patients With Friedreich Ataxia

A Pilot Study to Obtain Preliminary Information Regarding the Efficacy and Safety of the Combination of Immune Globulin and Ganciclovir as Compared to Ganciclovir Alone in the Treatment of Sight-Threatening CMV Retinitis in Patients With AIDS

A Randomized Comparative Pharmacokinetic Study of Oral Ganciclovir After Treatment With Intravenous Ganciclovir for Cytomegalovirus Gastrointestinal Disease in AIDS Patients

Inherited Retinal Degenerative Disease Registry

Systemic Biomarkers of Brain Injury From Hyperammonemia

Methylprednisolone Treatment of Friedreich Ataxia

The International Registry for Leigh Syndrome

Rosuvastatin (Crestor) in Friedreich Ataxia

TCA Cycle in the Dentate in Friedreich's Ataxia

MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis

Resistance Exercise in Barth Syndrome

New Non-invasive Modalities for Assessing Retinal Structure and Function

Evaluating the Effectiveness of a Dichloroacetate in MELAS Syndrome

A First in Human Study of RT001 in Patients With Friedreich's Ataxia

A Randomized Controlled Study of the Efficacy and Safety of Maintenance Treatment With Oral Ganciclovir for Newly Diagnosed Cytomegalovirus Retinitis in People With AIDS

An Open-Label Safety Study of Oral Ganciclovir Maintenance Treatment of CMV Retinitis in People With Limited Venous Access

Hepatocyte Transplantation for Liver Based Metabolic Disorders

A Randomized, Double-Blind Study of the Efficacy and Safety of Oral Ganciclovir for the Prevention of CMV Disease in People Infected With the Human Immunodeficiency Virus

Natural History of MADD