Search | Clinical Trials Finder

These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.

ClinicalTrials.gov public records Last synced Nov 15, 2026, 7:35 AM EST

All trial information on this site comes from the official ClinicalTrials.gov public API (clinicaltrials.gov/api/v2), run by the U.S. National Library of Medicine. We only display that public information; we do not add opinions, endorsements, or any other intent. Always review the official ClinicalTrials.gov record for the latest information.

Showing 1177–1200 of 1,513 matching trials from the live ClinicalTrials.gov search.

Filters: Condition: Inborn Errors of Metabolism Disorders Clear all

Conditions: Iron Overload, Hereditary Hemochromatosis
Interventions: Deferasirox (ICL670); Drug
Lead sponsor: Novartis Pharmaceuticals; Industry
Eligibility: 18 Years and older
Enrollment: 49 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2006 – 2009
U.S. locations: 9
States / cities: Long Beach, California • Farmington, Connecticut • Detroit, Michigan + 6 more

Conditions: Achondroplasia
Interventions: Infigratinib 0.25 mg/kg/day, Placebo Comparator 0.25 mg/kg/day; Drug
Lead sponsor: QED Therapeutics, a BridgeBio company; Industry
Eligibility: 3 Years to 17 Years
Enrollment: 114 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2025
U.S. locations: 7
States / cities: San Francisco, California • Aurora, Colorado • Baltimore, Maryland + 4 more

Conditions: Congenital Adrenal Hyperplasia
Interventions: Questionnaires/Interviews; Behavioral
Lead sponsor: Penn State University; Other
Eligibility: 10 Years to 13 Years · Female only
Enrollment: 188 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2009 – 2013
U.S. locations: 1
States / cities: University Park, Pennsylvania

Conditions: Gut Health
Interventions: Butyrate + Polyphenol Formulation, Placebo; Dietary Supplement
Lead sponsor: Supplement Formulators, Inc.; Industry
Eligibility: 25 Years to 70 Years
Enrollment: 124 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2025 – 2026
U.S. locations: 1
States / cities: Fort Lauderdale, Florida

Conditions: Urea Cycle Disorders
Interventions: HPN-100, BUPHENYL®; Drug
Lead sponsor: Amgen; Industry
Eligibility: 18 Years and older
Enrollment: 14 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2008
U.S. locations: 2
States / cities: Minneapolis, Minnesota • Houston, Texas

Conditions: Hypercholesterolemia, Familial
Interventions: Torcetrapib/atorvastatin; Drug
Lead sponsor: Pfizer; Industry
Eligibility: 18 Years and older
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: G(M2) Ganglioside, Tay-Sachs Disease Ganglioside, Sandhoff Disease Ganglioside
Interventions: Pyrimethamine; Drug
Lead sponsor: Exsar Corporation; Industry
Eligibility: 18 Years and older
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2008
U.S. locations: 2
States / cities: New York, New York • Cleveland, Ohio

Conditions: Homozygous Familial Hypercholesterolemia
Interventions: ALN-PCSSC, Standard of Care; Drug
Lead sponsor: The Medicines Company; Industry
Eligibility: 12 Years and older
Enrollment: 9 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2018
U.S. locations: 1
States / cities: Los Angeles, California

Conditions: Endothelial Dysfunction
Interventions: Sapropterin Dihydrochloride, Sapropterin Dihydrochloride and Vitamin C; Drug
Lead sponsor: BioMarin Pharmaceutical; Industry
Eligibility: 18 Years to 75 Years
Enrollment: 52 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2009
U.S. locations: 1
States / cities: Hackensack, New Jersey

Conditions: Urea Cycle Disorders, Inborn
Interventions: Sodium Benzoate, Sodium Phenylbutyrate; Drug
Lead sponsor: Baylor College of Medicine; Other
Eligibility: 18 Years to 64 Years
Enrollment: 7 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2014 – 2015
U.S. locations: 1
States / cities: Houston, Texas

Conditions: Gaucher Disease, Type 1
Interventions: FLT201; Genetic
Lead sponsor: Spur Therapeutics; Industry
Eligibility: 18 Years and older
Enrollment: 75 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2029
U.S. locations: 1
States / cities: Fairfax, Virginia

Conditions: Mucopolysaccharidosis II, MPS II
Interventions: SB-913; Biological
Lead sponsor: Sangamo Therapeutics; Industry
Eligibility: 5 Years to 65 Years
Enrollment: 9 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2021
U.S. locations: 5
States / cities: Oakland, California • Chicago, Illinois • New York, New York + 2 more

Conditions: Transthyretin Amyloidosis, ATTR-CM, ATTRv-PN, ATTR, ATTR-Mixed, hATTR
Interventions: Treatment of transthyretin (ATTR) amyloidosis in observational study setting; Drug
Lead sponsor: AstraZeneca; Industry
Eligibility: 18 Years to 130 Years
Enrollment: 1,850 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2031
U.S. locations: 33
States / cities: La Jolla, California • Los Angeles, California • San Francisco, California + 24 more

Conditions: Hypophosphatasia
Interventions: Not listed
Lead sponsor: Duke University; Other
Eligibility: Not listed
Enrollment: 200 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2028
U.S. locations: 1
States / cities: Durham, North Carolina

Conditions: Pompe Disease
Interventions: Alglucosidase alfa; Drug
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 1 Year and older
Enrollment: 113 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2014
U.S. locations: 51
States / cities: Birmingham, Alabama • Phoenix, Arizona • Little Rock, Arkansas + 48 more

Conditions: Cerebral Adrenoleukodystrophy (CALD)
Interventions: Lenti-D; Genetic
Lead sponsor: Genetix Biotherapeutics Inc.; Industry
Eligibility: Up to 17 Years · Male only
Enrollment: 35 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2023
U.S. locations: 3
States / cities: Palo Alto, California • Boston, Massachusetts • Minneapolis, Minnesota

Conditions: Pompe Disease
Interventions: Albuterol; Drug
Lead sponsor: Duke University; Other
Eligibility: 18 Years and older
Enrollment: 8 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2013
U.S. locations: 1
States / cities: Durham, North Carolina

Conditions: Aspartylglucosaminuria, Aspartylglucosamidase (AGA) Deficiency
Interventions: scAAV9/AGA; Genetic
Lead sponsor: Rare Trait Hope; Other
Eligibility: 4 Years to 45 Years
Enrollment: 9 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2032
U.S. locations: 1
States / cities: Shreveport, Louisiana

Conditions: Hypophosphatasia (HPP)
Interventions: Asfotase Alfa; Biological
Lead sponsor: Alexion Pharmaceuticals, Inc.; Industry
Eligibility: 18 Years to 80 Years
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2009
U.S. locations: 2
States / cities: St Louis, Missouri • Durham, North Carolina

Conditions: Lysosomal Storage Diseases
Interventions: Enzyme assay and molecular sequencing; Diagnostic Test
Lead sponsor: Lysosomal and Rare Disorders Research and Treatment Center, Inc.; Other
Eligibility: Not listed
Enrollment: 100,000 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2016 – 2019
U.S. locations: 1
States / cities: Fairfax, Virginia

Conditions: Lysosomal Storage Diseases, Cystinosis
Interventions: CTNS-RD-04 (including CTNS-RD-04-LB manufactured with LentiBOOST); Genetic
Lead sponsor: University of California, San Diego; Other
Eligibility: 18 Years and older
Enrollment: 7 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2024
U.S. locations: 1
States / cities: La Jolla, California

Conditions: GM1 Gangliosidosis
Interventions: LYS-GM101; Genetic
Lead sponsor: LYSOGENE; Industry
Eligibility: Up to 3 Years
Enrollment: 5 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2023
U.S. locations: 1
States / cities: Orange, California

Conditions: Urea Cycle Disorders
Interventions: HPN-100; Drug
Lead sponsor: Amgen; Industry
Eligibility: 6 Years and older
Enrollment: 60 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2011
U.S. locations: 21
States / cities: Long Beach, California • Los Angeles, California • Stanford, California + 18 more

Conditions: Ornithine Transcarbamylase Deficiency, Wilson Disease, Glycogen Storage Disease Type IA
Interventions: Not listed
Lead sponsor: Ultragenyx Pharmaceutical Inc; Industry
Eligibility: 18 Years and older
Enrollment: 51 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2022
U.S. locations: 1
States / cities: Akron, Ohio

Search by objective public record fields.

Safety and Efficacy of Deferasirox (ICL670) in Patients With Iron Overload Resulting From Hereditary Hemochromatosis

A Study to Evaluate the Efficacy and Safety of Infigratinib in Children and Adolescents With Achondroplasia

Tweens to Teens Project at Penn State

A Study to Evaluate the Effects of a Butyrate-Polyphenol Formulation on Gut Health and Associated Symptoms

Dose-Escalation Safety Study of HPN-100 to Treat Urea Cycle Disorders

Study To Evaluate The Effect Of Torcetrapib/Atorvastatin In Patients With Genetic High Cholesterol Disorder

A Phase I Study of Pyrimethamine in Patients With GM2 Gangliosidosis

A Study of ALN-PCSSC in Participants With Homozygous Familial Hypercholesterolemia (HoFH)

A Phase 2, Pharmacokinetic (PK) Study of 6R-BH4 Alone or 6R-BH4 With Vitamin C in Subjects With Endothelial Dysfunction

Comparative Efficacy of Phenylbutyrate (PBA) vs. Benzoate in Urea Cycle Disorders

Long Term Follow-up Study of Type-1 Gaucher Subjects Post FLT201 Dose (GALILEO-2)

Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II

Non-interventional Study of Patients With Transthyretin (ATTR) Amyloidosis

Natural History Study of Patients With Hypophosphatasia (HPP)

A Study to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale for Pompe Disease

A Clinical Study to Assess the Efficacy and Safety of Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy (CALD)

Albuterol in Individuals With Late Onset Pompe Disease (LOPD)

Safety and Efficacy of scAAV9/AGA Gene Therapy in Participants With Aspartylglucosaminuria (AGU)

Safety Study of Human Recombinant Tissue Non-Specific Alkaline Phosphatase Fusion Protein Asfotase Alfa in Adults With Hypophosphatasia (HPP)

Screening of Lysosomal Storage Disorders Diseases in Minority Groups

Stem Cell Gene Therapy for Cystinosis

A Safety and Efficacy Study of LYS-GM101 Gene Therapy in Patients With GM1 Gangliosidosis

Study of the Safety of HPN (Hyperion)-100 for the Long-Term Treatment of Urea Cycle Disorders (Treat UCD)

Adeno-Associated Virus (AAV) Antibody Study in Subjects OTC Deficiency, GSDIa, and Wilson Disease