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ClinicalTrials.gov public records Last synced Oct 31, 2026, 2:56 PM EDT

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Showing 793–816 of 1,511 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Inborn Errors of Metabolism Disorders Clear all
Completed Phase 3 Interventional Results available

A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE)

NCT00943111
View directory record Official record
Conditions
Gaucher Disease, Type 1
Interventions
Eliglustat tartrate, Imiglucerase
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years and older
Enrollment
160 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2015
U.S. locations
17
States / cities
Beverly Hills, California • San Francisco, California • Aurora, Colorado + 13 more
Source: ClinicalTrials.gov public record
Updated Nov 25, 2016 · Synced Oct 31, 2026, 2:56 PM EDT
Completed No phase listed Observational

A Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)

NCT02493998
View directory record Official record
Conditions
Mucopolysaccharidosis Type IIIB, Mucopolysaccharidosis Type 3 B, MPS III B, MPS 3 B
Interventions
Not listed
Lead sponsor
Allievex Corporation
Industry
Eligibility
1 Year to 10 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Mar 6, 2020 · Synced Oct 31, 2026, 2:56 PM EDT
Recruiting Not applicable Interventional

Development of Novel Psychological Assessment Tools and Anxiety Intervention for Phenylketonuria

NCT06560736
View directory record Official record
Conditions
Phenylketonurias
Interventions
Show Me FIRST coping skills intervention
Behavioral
Lead sponsor
University of Missouri-Columbia
Other
Eligibility
10 Years to 17 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Columbia, Missouri
Source: ClinicalTrials.gov public record
Updated Dec 23, 2025 · Synced Oct 31, 2026, 2:56 PM EDT
Completed Phase 2 Interventional

Alternative Dosing and Regimen of Replagal to Treat Fabry Disease

NCT00075244
View directory record Official record
Conditions
Fabry Disease
Interventions
Replagal
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Male only
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2005
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 31, 2026, 2:56 PM EDT
Completed No phase listed Observational

Estimation of the Carrier Frequency and Incidence of Smith-Lemli-Opitz Syndrome in African Americans

NCT00017732
View directory record Official record
Conditions
Smith-Lemli-Opitz Syndrome
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
Not listed
Enrollment
2,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2003
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 31, 2026, 2:56 PM EDT
Suspended Phase 1Phase 2 Interventional

Study of Safety, Tolerability and Efficacy of PBKR03 in Pediatric Subjects With Early Infantile Krabbe Disease

NCT04771416
View directory record Official record
Conditions
Leukodystrophy, Globoid Cell
Interventions
PBKR03
Biological
Lead sponsor
Gemma Biotherapeutics
Industry
Eligibility
1 Month to 9 Months
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2030
U.S. locations
4
States / cities
Chicago, Illinois • New York, New York • Philadelphia, Pennsylvania + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 30, 2026 · Synced Oct 31, 2026, 2:56 PM EDT
Completed No phase listed Observational

A Retrospective Natural History Study of Patients With Lysosomal Acid Lipase Deficiency/Wolman Phenotype

NCT01358370
View directory record Official record
Conditions
Lysosomal Acid Lipase Deficiency, Wolman Disease
Interventions
Not listed
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
Not listed
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
7
States / cities
Los Angeles, California • Palo Alto, California • Minneapolis, Minnesota + 2 more
Source: ClinicalTrials.gov public record
Updated Jun 27, 2016 · Synced Oct 31, 2026, 2:56 PM EDT
Withdrawn No phase listed Observational

Mucopolysaccharidosis Type II Observational

NCT04591834
View directory record Official record
Conditions
Mucopolysaccharidosis II
Interventions
Observational
Other
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
1 Month to 8 Years · Male only
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
2
States / cities
Oakland, California • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 10, 2022 · Synced Oct 31, 2026, 2:56 PM EDT
Completed Phase 2 Interventional Results available

Extension Study of Protocol ENB-006-09 - Study of Asfotase Alfa in Children With Hypophosphatasia (HPP)

NCT01203826
View directory record Official record
Conditions
Hypophosphatasia (HPP)
Interventions
Asfotase Alfa
Biological
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
5 Years to 12 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2016
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Mar 13, 2019 · Synced Oct 31, 2026, 2:56 PM EDT
Completed No phase listed Observational

Mitochondrial Video Assessment Source Material Study

NCT06180421
View directory record Official record
Conditions
PolG, Primary Mitochondrial Myopathy
Interventions
Not listed
Lead sponsor
The Emmes Company, LLC
Industry
Eligibility
16 Years and older
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
Kingston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Nov 14, 2024 · Synced Oct 31, 2026, 2:56 PM EDT
Completed Phase 4 Interventional Results available

A Study of the Safety and Efficacy of Fabrazyme (Agalsidase Beta) as Compared to Placebo in Patients With Advanced Fabry Disease

NCT00074984
View directory record Official record
Conditions
Fabry Disease
Interventions
Fabrazyme (agalsidase beta), Placebo
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
16 Years and older
Enrollment
82 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2004
U.S. locations
19
States / cities
Birmingham, Alabama • Los Angeles, California • San Francisco, California + 16 more
Source: ClinicalTrials.gov public record
Updated Dec 27, 2013 · Synced Oct 31, 2026, 2:56 PM EDT
Completed Early Phase 1 Interventional

Anaplerotic Therapy Using Triheptanoin for Patients With Glycogen Storage Disease Type I

NCT03665636
View directory record Official record
Conditions
Glycogen Storage Disease Type I
Interventions
Triheptanoin
Drug
Lead sponsor
Areeg El-Gharbawy
Other
Eligibility
1 Month to 65 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Dec 15, 2021 · Synced Oct 31, 2026, 2:56 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

Open Label, Study Of Efficacy and Safety Of AVR-RD-01 for Treatment-Naive Subjects With Classic Fabry Disease

NCT03454893
View directory record Official record
Conditions
Fabry Disease
Interventions
AVR-RD-01
Drug
Lead sponsor
AVROBIO
Industry
Eligibility
16 Years to 50 Years · Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
2
States / cities
Hackensack, New Jersey • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 5, 2024 · Synced Oct 31, 2026, 2:56 PM EDT
Completed Phase 2 Interventional

Effect of CER-001 on Plaque Volume in Homozygous Familial Hypercholesterolemia (HoFH) Subjects

NCT01412034
View directory record Official record
Conditions
Homozygous Familial Hypercholesterolemia
Interventions
CER-001
Drug
Lead sponsor
Cerenis Therapeutics, SA
Industry
Eligibility
12 Years and older
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
2
States / cities
Hartford, Connecticut • North Massapequa, New York
Source: ClinicalTrials.gov public record
Updated Aug 18, 2015 · Synced Oct 31, 2026, 2:56 PM EDT
Completed Phase 2Phase 3 Interventional Results available

Study of Long Term Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Participants Who Have Completed Study HGT-HIT-094

NCT02412787
View directory record Official record
Conditions
Hunter Syndrome
Interventions
Idursulfase-IT, Elaprase
Drug
Lead sponsor
Shire
Industry
Eligibility
Up to 18 Years · Male only
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2024
U.S. locations
3
States / cities
Oakland, California • Chicago, Illinois • Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Jun 19, 2025 · Synced Oct 31, 2026, 2:56 PM EDT
Completed Phase 1Phase 2 Interventional Accepts healthy volunteers

Safety and Tolerability of SYNB1618 in Healthy Adult Volunteers and Adult Subjects With Phenylketonuria

NCT03516487
View directory record Official record
Conditions
Phenylketonuria, Healthy
Interventions
SYNB1618, Placebo
Drug
Lead sponsor
Synlogic
Industry
Eligibility
18 Years to 64 Years
Enrollment
70 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2019
U.S. locations
4
States / cities
Boston, Massachusetts • Pittsburgh, Pennsylvania • Nashville, Tennessee + 1 more
Source: ClinicalTrials.gov public record
Updated May 13, 2021 · Synced Oct 31, 2026, 2:56 PM EDT
Recruiting Phase 3 Interventional

A Study to Evaluate Atumelnant in Adults With Congenital Adrenal Hyperplasia

NCT07144163
View directory record Official record
Conditions
Congenital Adrenal Hyperplasia, Classic Congenital Adrenal Hyperplasia
Interventions
Atumelnant, Placebo
Drug
Lead sponsor
Crinetics Pharmaceuticals Inc.
Industry
Eligibility
18 Years to 74 Years
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
13
States / cities
Los Angeles, California • Aurora, Colorado • Gainesville, Florida + 10 more
Source: ClinicalTrials.gov public record
Updated Oct 16, 2026 · Synced Oct 31, 2026, 2:56 PM EDT
Completed Phase 4 Interventional

A Multicenter Study of the Efficacy of Cerezyme in Testing Skeletal Disease in Patients With Type I Gaucher Disease.

NCT00365131
View directory record Official record
Conditions
Gaucher Disease Type I, Cerebroside Lipidosis Syndrome, Clucocerebrosidase Deficiency Disease, Glucosylceramide Beta-Glucosidase Deficiency Disease, Gaucher Disease, Non-Neuronopathic Form
Interventions
Cerezyme (imiglucerase for injection)
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
10 Years to 65 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1997 – 2004
U.S. locations
1
States / cities
Coral Springs, Florida
Source: ClinicalTrials.gov public record
Updated Mar 5, 2015 · Synced Oct 31, 2026, 2:56 PM EDT
Recruiting No phase listed Observational

Use of Cysteamine in the Treatment of Cystinosis

NCT00359684
View directory record Official record
Conditions
Cystinosis
Interventions
Cysteamine
Drug
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
1 Week to 115 Years
Enrollment
330 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1979
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 16, 2026 · Synced Oct 31, 2026, 2:56 PM EDT
Active, not recruiting No phase listed Observational

Prescreening Study to Identify Potential Wilson Disease Participants for Gene-Editing Clinical Trial

NCT07226622
View directory record Official record
Conditions
Wilson Disease
Interventions
Not listed
Lead sponsor
Prime Medicine, Inc.
Industry
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
7
States / cities
Los Angeles, California • Sacramento, California • New Haven, Connecticut + 4 more
Source: ClinicalTrials.gov public record
Updated May 12, 2026 · Synced Oct 31, 2026, 2:56 PM EDT
Completed Phase 1Phase 2 Interventional Results available

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

NCT00395629
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated May 30, 2011 · Synced Oct 31, 2026, 2:56 PM EDT
Completed Phase 3 Interventional Results available

Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) ERT Compared With Imiglucerase in Type I Gaucher Disease

NCT00553631
View directory record Official record
Conditions
Gaucher Disease, Type 1
Interventions
velaglucerase alfa, imiglucerase
Biological
Lead sponsor
Shire
Industry
Eligibility
2 Years and older
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jun 8, 2021 · Synced Oct 31, 2026, 2:56 PM EDT
Not yet recruiting Phase 1 Interventional

High-Dose Vitamin C in G6PDA and Pyruvate Kinase Deficiency: A Safety Study

NCT07612345
View directory record Official record
Conditions
Pyruvate Kinase Deficiency, Glucose 6 Phosphate Dehydrogenase Deficiency
Interventions
Vitamin C
Drug
Lead sponsor
University of Utah
Other
Eligibility
18 Years and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated May 28, 2026 · Synced Oct 31, 2026, 2:56 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Effect of G6PD Deficiency on Red Blood Cell Storage

NCT04081272
View directory record Official record
Conditions
G6PD Deficiency
Interventions
Sodium Chromate Cr51
Drug
Lead sponsor
Columbia University
Other
Eligibility
18 Years to 70 Years · Male only
Enrollment
40 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2017
U.S. locations
2
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 17, 2019 · Synced Oct 31, 2026, 2:56 PM EDT