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ClinicalTrials.gov public records Last synced Oct 23, 2026, 6:15 AM EDT

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Showing 385–408 of 1,510 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Inborn Errors of Metabolism Disorders Clear all
Completed Phase 2 Interventional

Study of Deferasirox in Iron Overload From Beta-thalassemia Unable to be Treated With Deferoxamine or Chronic Anemias

NCT00061763
View directory record Official record
Conditions
Beta-thalassemia, Myelodysplastic Syndromes, Fanconi Syndrome, Anemia, Diamond-Blackfan, Anemia, Aplastic
Interventions
Deferasirox
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years and older
Enrollment
175 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2003
U.S. locations
6
States / cities
Oakland, California • Stanford, California • Arlington Heights, Illinois + 3 more
Source: ClinicalTrials.gov public record
Updated Aug 22, 2017 · Synced Oct 23, 2026, 6:15 AM EDT
Completed Phase 2 Interventional

Phase II Pilot Randomized Study of Sodium Dichloroacetate in Patients With Congenital Lactic Acidemia

NCT00004493
View directory record Official record
Conditions
Lactic Acidosis
Interventions
sodium dichloroacetate
Drug
Lead sponsor
University of California, San Diego
Other
Eligibility
3 Months and older
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2003
U.S. locations
1
States / cities
La Jolla, California
Source: ClinicalTrials.gov public record
Updated Mar 25, 2015 · Synced Oct 23, 2026, 6:15 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)

NCT04093349
View directory record Official record
Conditions
Pompe Disease, Pompe Disease (Late-onset), Glycogen Storage Disease Type 2, Glycogen Storage Disease Type II, LOPD, Lysosomal Storage Diseases, Acid Maltase Deficiency
Interventions
SPK-3006
Genetic
Lead sponsor
Spark Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2032
U.S. locations
10
States / cities
Phoenix, Arizona • Orange, California • Atlanta, Georgia + 7 more
Source: ClinicalTrials.gov public record
Updated Nov 27, 2024 · Synced Oct 23, 2026, 6:15 AM EDT
Terminated Not applicable Interventional Results available

Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome

NCT02156674
View directory record Official record
Conditions
Maroteaux-Lamy Syndrome
Interventions
Naglazyme®
Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
3 Years and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 23, 2020 · Synced Oct 23, 2026, 6:15 AM EDT
Completed No phase listed Observational

Monitoring of Early Disease Progression in Hereditary Transthyretin Amyloidosis

NCT03431896
View directory record Official record
Conditions
Amyloidosis, Amyloid, Amyloid Neuropathies, Familial, Amyloid Cardiomyopathy, Amyloid - Primary, Transthyretin Amyloidosis, AL Amyloidosis
Interventions
Not listed
Lead sponsor
The Cleveland Clinic
Other
Eligibility
Not listed
Enrollment
37 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2026
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 12, 2026 · Synced Oct 23, 2026, 6:15 AM EDT
Completed Phase 2 Interventional

OGT 918-006: A Phase I/II Randomized, Controlled Study of OGT 918 in Patients With Neuronopathic Gaucher Disease

NCT00041535
View directory record Official record
Conditions
Gaucher Disease
Interventions
OGT 918
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
4 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2007
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 23, 2026, 6:15 AM EDT
Active, not recruiting Phase 3 Interventional

A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT)

NCT04573023
View directory record Official record
Conditions
Mucopolysaccharidosis II
Interventions
JR-141, Idursulfase, JR-141 or Idursulfase
Drug
Lead sponsor
JCR Pharmaceuticals Co., Ltd.
Industry
Eligibility
Not listed
Enrollment
86 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
7
States / cities
Phoenix, Arizona • Oakland, California • Chicago, Illinois + 4 more
Source: ClinicalTrials.gov public record
Updated Oct 2, 2026 · Synced Oct 23, 2026, 6:15 AM EDT
Completed Not applicable Interventional

Stem Cell Transplantation (SCT) for Genetic Diseases

NCT00004378
View directory record Official record
Conditions
Thrombocytopenia, Metachromatic Leukodystrophy, Fanconi's Anemia, Thalassemia Major, Pure Red-Cell Aplasia, Inborn Errors of Metabolism
Interventions
Stem Cell Transplantation
Procedure
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years to 17 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1995
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 23, 2026, 6:15 AM EDT
Completed Phase 2 Interventional Results available

Long-Term Extension of Previous rAvPAL-PEG Protocols in Subjects With PKU (PAL-003)

NCT00924703
View directory record Official record
Conditions
Phenylketonuria
Interventions
rAvPAL-PEG
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
16 Years to 55 Years
Enrollment
68 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2019
U.S. locations
14
States / cities
Aurora, Colorado • Gainesville, Florida • Chicago, Illinois + 11 more
Source: ClinicalTrials.gov public record
Updated Oct 12, 2021 · Synced Oct 23, 2026, 6:15 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Diagnosis and Treatment of Patients With Inborn Errors of Metabolism

NCT00369421
View directory record Official record
Conditions
Arterial Calcification Due to Deficiency of CD73
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
1 Month to 115 Years
Enrollment
4,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 1978
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 13, 2026 · Synced Oct 23, 2026, 6:15 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Study to Evaluate Pharmacokinetic Parameters and Safety of Eliglustat Absorption Through the Mouth

NCT06193304
View directory record Official record
Conditions
Gaucher's Disease
Interventions
Eliglustat
Drug
Lead sponsor
Sanofi
Industry
Eligibility
18 Years to 55 Years
Enrollment
6 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014
U.S. locations
1
States / cities
Evansville, Indiana
Source: ClinicalTrials.gov public record
Updated Jan 5, 2024 · Synced Oct 23, 2026, 6:15 AM EDT
Completed No phase listed Observational

"The MaP Study": Mapping the Patient Journey in MMA and PA

NCT03484767
View directory record Official record
Conditions
Methylmalonic Acidemia, Propionic Acidemia
Interventions
Not listed
Lead sponsor
ModernaTX, Inc.
Industry
Eligibility
Not listed
Enrollment
97 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
11
States / cities
Stanford, California • Atlanta, Georgia • Chicago, Illinois + 8 more
Source: ClinicalTrials.gov public record
Updated Aug 2, 2021 · Synced Oct 23, 2026, 6:15 AM EDT
Completed Phase 1 Interventional

A Phase I Study of the Safety of Proleukin (Aldesleukin) in Combination With Ganciclovir and Antiretroviral Therapy in HIV Seropositive Patients With Cytomegalovirus (CMV) Retinitis

NCT00002321
View directory record Official record
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Aldesleukin
Drug
Lead sponsor
Chiron Corporation
Industry
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Toledo, Ohio
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 23, 2026, 6:15 AM EDT
Recruiting Phase 3 Interventional

[64Cu]FBP8 PET for Early Detection of Intracardiac Thrombus in Amyloid Cardiomyopathy

NCT07560306
View directory record Official record
Conditions
Cardiac Amyloidosis
Interventions
[64Cu]FBP8 PET/MR
Drug
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jul 21, 2026 · Synced Oct 23, 2026, 6:15 AM EDT
Completed Phase 2 Interventional

Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI

NCT00048711
View directory record Official record
Conditions
Mucopolysaccharidosis VI
Interventions
N-acetylgalactosamine 4-sulfatase
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
5 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2006
U.S. locations
1
States / cities
Novato, California
Source: ClinicalTrials.gov public record
Updated Aug 25, 2009 · Synced Oct 23, 2026, 6:15 AM EDT
Completed Phase 3 Interventional Results available

Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia

NCT03409744
View directory record Official record
Conditions
Homozygous Familial Hypercholesterolemia
Interventions
evinacumab
Drug
Lead sponsor
Regeneron Pharmaceuticals
Industry
Eligibility
12 Years and older
Enrollment
116 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
9
States / cities
Los Angeles, California • Boca Raton, Florida • Boston, Massachusetts + 5 more
Source: ClinicalTrials.gov public record
Updated Apr 8, 2025 · Synced Oct 23, 2026, 6:15 AM EDT
Recruiting No phase listed Observational

A Global Prospective Observational Registry of Patients With Pompe Disease

NCT06121011
View directory record Official record
Conditions
Pompe Disease
Interventions
Cipaglucosidase alfa, Miglustat, Alglucosidase alfa or Avalglucosidase alfa, Untreated
Biological · Drug · Other
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
Not listed
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2034
U.S. locations
15
States / cities
Little Rock, Arkansas • Irvine, California • Jacksonville, Florida + 10 more
Source: ClinicalTrials.gov public record
Updated Mar 10, 2026 · Synced Oct 23, 2026, 6:15 AM EDT
Recruiting Phase 1Phase 2 Interventional

A First-in-human Study of KK8123 in Adults With X-linked Hypophosphatemia

NCT06525636
View directory record Official record
Conditions
X-linked Hypophosphatemia
Interventions
KK8123
Drug
Lead sponsor
Kyowa Kirin Co., Ltd.
Industry
Eligibility
18 Years to 65 Years
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
5
States / cities
San Francisco, California • New Haven, Connecticut • Indianapolis, Indiana + 2 more
Source: ClinicalTrials.gov public record
Updated Sep 21, 2026 · Synced Oct 23, 2026, 6:15 AM EDT
Completed Phase 3 Interventional Results available

Study to Evaluate Efficacy and Safety of Inclisiran in Adolescents With Homozygous Familial Hypercholesterolemia

NCT04659863
View directory record Official record
Conditions
Familial Hypercholesterolemia - Homozygous
Interventions
Inclisiran, Placebo
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
12 Years to 17 Years
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
1
States / cities
Boca Raton, Florida
Source: ClinicalTrials.gov public record
Updated Jan 13, 2026 · Synced Oct 23, 2026, 6:15 AM 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 23, 2026, 6:15 AM EDT
Completed Phase 1 Interventional

Effect of S-adenosylmethionine (SAMe) on Blood Levels of Homocysteine

NCT00473200
View directory record Official record
Conditions
Hyperhomocysteinemia
Interventions
S-adenosylmethionine
Dietary Supplement
Lead sponsor
Baylor Research Institute
Other
Eligibility
18 Years and older
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2013
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Mar 14, 2018 · Synced Oct 23, 2026, 6:15 AM EDT
Completed Phase 2 Interventional Results available

Safety, Tolerability and Efficacy of Microsomal Triglyceride Protein (MTP) Inhibitor

NCT01556906
View directory record Official record
Conditions
Homozygous Familial Hypercholesterolemia
Interventions
Lomitapide
Drug
Lead sponsor
Aegerion Pharmaceuticals, Inc.
Industry
Eligibility
13 Years and older
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2004
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 10, 2013 · Synced Oct 23, 2026, 6:15 AM EDT
Completed Phase 2Phase 3 Interventional Results available

Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Idursulfase

NCT00630747
View directory record Official record
Conditions
Hunter Syndrome, Mucopolysaccharidosis II (MPS II)
Interventions
Idursulfase
Biological
Lead sponsor
Shire
Industry
Eligibility
5 Years and older · Male only
Enrollment
94 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2008
U.S. locations
16
States / cities
Phoenix, Arizona • Oakland, California • Denver, Colorado + 13 more
Source: ClinicalTrials.gov public record
Updated Jun 10, 2021 · Synced Oct 23, 2026, 6:15 AM EDT
Completed Phase 3 Interventional Results available

An Open-label Extension Study to Assess the Long-term Safety and Efficacy of ISIS 301012 (Mipomersen) in Patients With Familial Hypercholesterolemia or Severe-Hypercholesterolemia

NCT00694109
View directory record Official record
Conditions
Lipid Metabolism, Inborn Errors, Hypercholesterolemia, Autosomal Dominant, Hyperlipidemias, Metabolic Diseases, Hyperlipoproteinemia Type II, Metabolism, Inborn Errors, Genetic Diseases, Inborn, Infant, Newborn, Diseases, Metabolic Disorder, Congenital Abnormalities, Hypercholesterolemia, Hyperlipoproteinemias, Dyslipidemias, Lipid Metabolism Disorders
Interventions
Mipomersen Sodium
Drug
Lead sponsor
Kastle Therapeutics, LLC
Industry
Eligibility
12 Years and older
Enrollment
144 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2014
U.S. locations
20
States / cities
Mission Viejo, California • Newport Beach, California • Bridgeport, Connecticut + 17 more
Source: ClinicalTrials.gov public record
Updated Sep 9, 2016 · Synced Oct 23, 2026, 6:15 AM EDT