- Conditions
- X-Linked Adrenoleukodystrophy (X-ALD), Cerebral Adrenoleukodystrophy (CALD), Adrenoleukodystrophy (ALD)
- Interventions
- Allo-HSCT
- Genetic
- Lead sponsor
- Genetix Biotherapeutics Inc.
- Industry
- Eligibility
- Up to 17 Years · Male only
- Enrollment
- 59 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2019
- U.S. locations
- 6
- States / cities
- Los Angeles, California • Palo Alto, California • Boston, Massachusetts + 3 more
Search Results
Search by objective public record fields.
Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results are retrieved from ClinicalTrials.gov and synchronized into the directory. Search pages remain noindex by default.
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Showing 25–48
of 24
matching trials from the live ClinicalTrials.gov search.
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- Conditions
- Ataxia Telangiectasia
- Interventions
- Baclofen, Placebo
- Drug
- Lead sponsor
- Johns Hopkins University
- Other
- Eligibility
- 12 Years and older
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2011
- U.S. locations
- 2
- States / cities
- Baltimore, Maryland
- Conditions
- Pancreatic Ductal Adenocarcinoma, Pancreatic Cyst, Chronic Pancreatitis, Fatty Pancreas, Genetic Pancreatic Cancer, Genetic Pancreatitis, BRCA Mutation, Lynch Syndrome, FAP, Familial Atypical Multiple Mole-Melanoma, PALB2 Gene Mutation, Peutz-Jeghers Syndrome, Ataxia Telangiectasia
- Interventions
- Data collection
- Other
- Lead sponsor
- H. Lee Moffitt Cancer Center and Research Institute
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 317 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2022 – 2026
- U.S. locations
- 1
- States / cities
- Tampa, Florida
- Conditions
- Fabry Disease
- Interventions
- agalsidase alfa
- Biological
- Lead sponsor
- Shire
- Industry
- Eligibility
- Not listed
- U.S. locations
- 25
- States / cities
- Tucson, Arizona • La Jolla, California • Los Angeles, California + 21 more
- Conditions
- Ataxia-Telangiectasia (A-T)
- Interventions
- A-T iPS cell line, Carrier patients iPS cell line
- Other
- Lead sponsor
- Johns Hopkins University
- Other
- Eligibility
- 3 Years to 100 Years
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2018
- U.S. locations
- 1
- States / cities
- Baltimore, Maryland
- Conditions
- Friedreich Ataxia
- Interventions
- plethysmography
- Diagnostic Test
- Lead sponsor
- University of Pittsburgh
- Other
- Eligibility
- 18 Years to 79 Years
- Enrollment
- 7 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2023
- U.S. locations
- 1
- States / cities
- Pittsburgh, Pennsylvania
- Conditions
- Niemann-Pick Diseases
- Interventions
- Not listed
- Lead sponsor
- National Niemann-Pick Disease Foundation
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 76 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Arginase I Deficiency, Hyperargininemia
- Interventions
- Pegzilarginase, Placebo
- Drug
- Lead sponsor
- Aeglea Biotherapeutics
- Industry
- Eligibility
- 2 Years and older
- Enrollment
- 32 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2023
- U.S. locations
- 17
- States / cities
- Rogers, Arkansas • Orange, California • Stanford, California + 14 more
- Conditions
- Gaucher Disease
- Interventions
- Standard of Care
- Other
- Lead sponsor
- Takeda
- Industry
- Eligibility
- Up to 5 Years
- Enrollment
- 11 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2023
- U.S. locations
- 1
- States / cities
- Fairfax, Virginia
- Conditions
- ARG1 Deficiency
- Interventions
- Loargys Arginine Test System
- Device
- Lead sponsor
- Immedica Pharma US Inc
- Industry
- Eligibility
- 0 Years to 100 Years
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2027
- U.S. locations
- 1
- States / cities
- Atlanta, Georgia
- Conditions
- Pompe Disease, Glycogen Storage Disease Type II (GSD-II), Glycogenesis 2 Acid Maltase Deficiency
- Interventions
- alglucosidase alfa
- Biological
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- 6 Months and older
- Enrollment
- 13 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2010
- U.S. locations
- 9
- States / cities
- Birmingham, Alabama • Stanford, California • Washington D.C., District of Columbia + 6 more
- Conditions
- Gaucher Disease, Type 1
- Interventions
- Not listed
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- 16 Years to 65 Years
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2009
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Pompe Disease, Glycogen Storage Disease Type II (GSD II)
- Interventions
- alglucosidase alfa
- Biological
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- Not listed
- Enrollment
- 21 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2020
- U.S. locations
- 4
- States / cities
- Valhalla, New York • Cincinnati, Ohio • Salt Lake City, Utah + 1 more
- 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
- MELAS Syndrome
- Interventions
- Fluzone®
- Biological
- Lead sponsor
- Stanford University
- Other
- Eligibility
- 13 Years to 65 Years
- Enrollment
- 22 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2010 – 2012
- U.S. locations
- 1
- States / cities
- Stanford, California
- Conditions
- Friedreich Ataxia
- Interventions
- CTI-1601, Placebo
- Biological
- Lead sponsor
- Larimar Therapeutics, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 27 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2021
- U.S. locations
- 1
- States / cities
- Eatontown, New Jersey
- Conditions
- Fabry Disease
- Interventions
- Fabrazyme (agalsidase beta)
- Biological
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- 7 Years to 15 Years
- Enrollment
- 16 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2002 – 2005
- U.S. locations
- 1
- States / cities
- Tucson, Arizona
- Conditions
- Hurler Syndrome, Inherited Metabolic Disorder, Lysosomal Storage Disorder, Metachromatic Leukodystrophy, Inborn Errors of Metabolism
- Interventions
- rivogenlecleucel, rimiducid
- Biological · Drug
- Lead sponsor
- Bellicum Pharmaceuticals
- Industry
- Eligibility
- 3 Months to 21 Years
- U.S. locations
- 2
- States / cities
- Los Angeles, California • Palo Alto, California
- Conditions
- Phenylketonuria
- Interventions
- rAvPAL-PEG
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 16 Years to 70 Years
- Enrollment
- 16 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2015
- U.S. locations
- 9
- States / cities
- Aurora, Colorado • Gainesville, Florida • Louisville, Kentucky + 6 more
- Conditions
- Fabry Disease
- Interventions
- migalastat HCl
- Drug
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- 18 Years to 65 Years · Female only
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2008
- U.S. locations
- 1
- States / cities
- Decatur, Georgia
- Conditions
- COPD, Tetrahydrobiopterin Deficiency, Oxidative Stress
- Interventions
- Tetrahydrobiopterin, Placebo oral tablet
- Drug
- Lead sponsor
- University of Massachusetts, Amherst
- Other
- Eligibility
- 18 Years to 85 Years
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2021 – 2022
- U.S. locations
- 1
- States / cities
- Amherst, Massachusetts
- Conditions
- Fabry Disease
- Interventions
- migalastat HCl
- Drug
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- 18 Years to 55 Years · Male only
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2008
- U.S. locations
- 5
- States / cities
- Los Angeles, California • Decatur, Georgia • Bethesda, Maryland + 2 more
- Conditions
- Aspartylglucosaminuria, Fucosidosis, Galactosialidosis, Alpha Mannosidosis, Beta Mannosidosis, Mucolipidosis II, Mucolipidosis III, Schindler Disease, Sialidosis
- Interventions
- Non-interventional.
- Other
- Lead sponsor
- Greenwood Genetic Center
- Other
- Eligibility
- Not listed
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2020
- U.S. locations
- 1
- States / cities
- North Charleston, South Carolina
- Conditions
- GM1 Gangliosidosis, GM1 Gangliosidosis, Type I, GM1 Gangliosidosis, Type 2, Beta-Galactosidase-1 (GLB1) Deficiency
- Interventions
- PBGM01
- Biological
- Lead sponsor
- Gemma Biotherapeutics
- Industry
- Eligibility
- 1 Month to 24 Months
- Enrollment
- 26 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2029
- U.S. locations
- 4
- States / cities
- Oakland, California • Minneapolis, Minnesota • New Brunswick, New Jersey + 1 more