Independent directory Public ClinicalTrials.gov records United States
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ClinicalTrials.gov public records Last synced Sep 8, 2026, 6:54 AM EDT

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Showing 25–28 of 4 matching trials from the live ClinicalTrials.gov search.
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Conditions
Inherited Metabolic Diseases, Lysosomal Storage Disorders, Peroxisomal Storage Diseases, Inborn Errors of Metabolism, Mucopolysaccharidosis
Interventions
ALD-101
Biological
Lead sponsor
Aldagen
Industry
Eligibility
Up to 16 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2011
U.S. locations
3
States / cities
Los Angeles, California • New York, New York • Durham, North Carolina
Conditions
Primary Mitochondrial Myopathy
Interventions
elamipretide, placebo comparator, elamipretide open label treatment
Combination Product
Lead sponsor
Stealth BioTherapeutics Inc.
Industry
Eligibility
16 Years to 80 Years
Enrollment
218 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
13
States / cities
La Jolla, California • Palo Alto, California • Aurora, Colorado + 9 more
Conditions
Pellucid Marginal Corneal Degeneration, Keratoconus, Keratectasia
Interventions
Brillouin Ocular Scanner
Device
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
15 Years to 75 Years
Enrollment
85 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2020
U.S. locations
1
States / cities
Boston, Massachusetts
Conditions
Pelizaeus-Merzbacher Disease
Interventions
Not listed
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
6 Months to 17 Years · Male only
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2029
U.S. locations
2
States / cities
Atlanta, Georgia • Philadelphia, Pennsylvania