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Showing 1–24 of 2,407 matching trials from the live ClinicalTrials.gov search.
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Completed Phase 2Phase 3 Interventional Results available

Stem Cell Transplant for Bone Marrow Failure Syndromes

NCT00176878
View directory record Official record
Conditions
Diamond-Blackfan Anemia, Kostmann's Neutropenia, Shwachman-Diamond Syndrome
Interventions
Stem cell transplant, Fludarabine monophosphate, Total lymphoid irradiation, Busulfan, anti-thymocyte globulin
Procedure · Drug · Biological
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 35 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2009
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 27, 2017 · Synced Sep 3, 2026, 10:45 PM EDT
Completed No phase listed Observational

Neonatal CMV-Ganciclovir Follow-up Study

NCT00031421
View directory record Official record
Conditions
Cytomegalovirus Infections
Interventions
Not listed
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
9 Years to 14 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2005
U.S. locations
1
States / cities
Birmingham, Alabama
Source: ClinicalTrials.gov public record
Updated Aug 11, 2011 · Synced Sep 3, 2026, 10:45 PM EDT
Completed Phase 3 Interventional Results available

ENVISION: A Study to Evaluate the Efficacy and Safety of Givosiran (ALN-AS1) in Patients With Acute Hepatic Porphyrias (AHP)

NCT03338816
View directory record Official record
Conditions
Acute Hepatic Porphyria, Acute Intermittent Porphyria, Porphyria, Acute Intermittent, Acute Porphyria, Hereditary Coproporphyria (HCP), Variegate Porphyria (VP), ALA Dehydratase Deficient Porphyria (ADP)
Interventions
Givosiran, Placebo
Drug
Lead sponsor
Alnylam Pharmaceuticals
Industry
Eligibility
12 Years and older
Enrollment
94 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
10
States / cities
Little Rock, Arkansas • San Francisco, California • Boston, Massachusetts + 7 more
Source: ClinicalTrials.gov public record
Updated Apr 21, 2024 · Synced Sep 3, 2026, 10:45 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC)

NCT01694940
View directory record Official record
Conditions
Mitochondrial Disorders, Mitochondrial Genetic Disorders, Mitochondrial Diseases, Disorder of Mitochondrial Respiratory Chain Complexes, Deletion and Duplication of Mitochondrial DNA
Interventions
Not listed
Lead sponsor
Columbia University
Other
Eligibility
Not listed
Enrollment
1,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2026
U.S. locations
16
States / cities
San Diego, California • Stanford, California • Aurora, Colorado + 12 more
Source: ClinicalTrials.gov public record
Updated Feb 3, 2026 · Synced Sep 3, 2026, 10:45 PM EDT
Terminated No phase listed Observational

Clinical Evaluation of a Non-Invasive Hypoglycemia Detector in a Glycogen Storage Disease Population

NCT02338817
View directory record Official record
Conditions
Glycogen Storage Disease
Interventions
Diabetes Sentry
Device
Lead sponsor
University of Florida
Other
Eligibility
Up to 70 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Oct 3, 2016 · Synced Sep 3, 2026, 10:45 PM EDT
Recruiting No phase listed Observational

Natural History of Sphingosine Phosphate Lyase Insufficiency Syndrome (SPLIS)

NCT06669949
View directory record Official record
Conditions
Sphingosine Phosphate Lyase Insufficiency Syndrome (SPLIS)
Interventions
no intervention
Other
Lead sponsor
University of California, San Francisco
Other
Eligibility
Not listed
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Aug 3, 2026 · Synced Sep 3, 2026, 10:45 PM EDT
Active, not recruiting No phase listed Observational

Long-Term Follow Up of CLN6 Batten Disease Subjects Following Gene Transfer

NCT04273243
View directory record Official record
Conditions
CLN6, Batten Disease
Interventions
AT-GTX-501
Genetic
Lead sponsor
Emily de los Reyes
Other
Eligibility
12 Months and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Oct 1, 2025 · Synced Sep 3, 2026, 10:45 PM EDT
Completed No phase listed Observational

The Role of Bacteria and Genetic Variations in Cystic Fibrosis

NCT00043225
View directory record Official record
Conditions
Psuedomas Infection, Cystic Fibrosis
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
9 Years to 80 Years
Enrollment
76 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2018
U.S. locations
4
States / cities
Bethesda, Maryland • Seattle, Washington • Madison, Wisconsin + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 28, 2021 · Synced Sep 3, 2026, 10:45 PM EDT
Completed No phase listed Observational

Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip Disease

NCT01707433
View directory record Official record
Conditions
Mucopolysaccharidosis IV A, Mucopolysaccharidosis VI
Interventions
Enzyme testing
Other
Lead sponsor
Children's Hospitals and Clinics of Minnesota
Other
Eligibility
Up to 21 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2015
U.S. locations
2
States / cities
Minneapolis, Minnesota • Saint Paul, Minnesota
Source: ClinicalTrials.gov public record
Updated Aug 3, 2015 · Synced Sep 3, 2026, 10:45 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of OP-101 After Intravenous Administration in Healthy Volunteers

NCT03500627
View directory record Official record
Conditions
Healthy
Interventions
OP-101
Drug
Lead sponsor
Orpheris, Inc.
Industry
Eligibility
18 Years to 65 Years
Enrollment
8 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 16, 2018 · Synced Sep 3, 2026, 10:45 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Androgenetic Alopecia in Fabry Disease

NCT01295008
View directory record Official record
Conditions
Fabry Disease
Interventions
Not listed
Lead sponsor
Baylor Research Institute
Other
Eligibility
18 Years to 64 Years · Male only
Enrollment
107 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010 – 2015
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Jan 12, 2016 · Synced Sep 3, 2026, 10:45 PM EDT
Completed Early Phase 1 Interventional

Pemetrexed Disodium in the Cerebrospinal Fluid of Patients With Leptomeningeal Metastases

NCT00424242
View directory record Official record
Conditions
Brain and Central Nervous System Tumors, Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Lymphoproliferative Disorder, Metastatic Cancer, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Precancerous Condition, Secondary Myelofibrosis, Unspecified Adult Solid Tumor, Protocol Specific
Interventions
Pemetrexed
Drug
Lead sponsor
Northwestern University
Other
Eligibility
18 Years to 120 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2011
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Sep 7, 2021 · Synced Sep 3, 2026, 10:45 PM EDT
Recruiting No phase listed Observational

Artificial Intelligence Guided Echocardiographic Screening of Rare Diseases (EchoNet-Screening)

NCT05139797
View directory record Official record
Conditions
Cardiac Amyloidosis
Interventions
EchoNet-LVH screening for cardiac amyloidosis
Other
Lead sponsor
Cedars-Sinai Medical Center
Other
Eligibility
18 Years and older
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jul 21, 2026 · Synced Sep 3, 2026, 10:45 PM EDT
No Longer Available No phase listed Expanded access

Canavan-Single Patient IND

NCT05317780
View directory record Official record
Conditions
Canavan Disease
Interventions
rAAV9-CB6-AspA
Drug
Lead sponsor
University of Florida
Other
Eligibility
18 Months to 24 Months · Male only
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Aug 22, 2023 · Synced Sep 3, 2026, 10:45 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency

NCT02292654
View directory record Official record
Conditions
Sphingomyelin Lipidosis
Interventions
Olipudase alfa
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Up to 17 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 22, 2022 · Synced Sep 3, 2026, 10:45 PM EDT
Completed No phase listed Observational

Genotype-Phenotype Correlations of Late Infantile Neuronal Ceroid Lipofuscinosis

NCT01035424
View directory record Official record
Conditions
Batten Disease, Late Infantile Neuronal Ceroid Lipofuscinosis
Interventions
Not listed
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
2 Years to 18 Years
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2016
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 28, 2020 · Synced Sep 3, 2026, 10:45 PM EDT
Completed No phase listed Observational

Observational Study for Subjects With Pompe Disease Undergoing Immune Modulation Therapies

NCT01451879
View directory record Official record
Conditions
Pompe Disease
Interventions
Rituximab, Miglustat
Drug
Lead sponsor
University of Florida
Other
Eligibility
Up to 65 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2017
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Dec 7, 2017 · Synced Sep 3, 2026, 10:45 PM EDT
Completed Phase 3 Interventional

Phase III Ganciclovir +/- rGM-CSF for AIDS-Related CMV Retinitis

NCT00002070
View directory record Official record
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Sargramostim, Ganciclovir
Drug
Lead sponsor
Schering-Plough
Industry
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
14
States / cities
Los Angeles, California • San Francisco, California • Sherman Oaks, California + 7 more
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced Sep 3, 2026, 10:45 PM EDT
Recruiting Phase 2 Interventional

Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH)

NCT07169279
View directory record Official record
Conditions
Achondroplasia
Interventions
Infigratinib is provided as a single dose of minitablets for oral administration, Infigratinib is provided as sprinkle capsules for daily oral administration, Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administration
Drug
Lead sponsor
QED Therapeutics, a BridgeBio company
Industry
Eligibility
0 Years to 32 Months
Enrollment
77 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
3
States / cities
Oakland, California • Baltimore, Maryland • Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Jul 12, 2026 · Synced Sep 3, 2026, 10:45 PM EDT
Completed Early Phase 1 Interventional

Ketones & Mitochondrial Heteroplasmy

NCT01252979
View directory record Official record
Conditions
MELAS Syndrome, Mitochondrial Diseases
Interventions
Medium-Chain Triglycerides
Dietary Supplement
Lead sponsor
The University of Texas Health Science Center, Houston
Other
Eligibility
Not listed
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2011
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Feb 5, 2012 · Synced Sep 3, 2026, 10:45 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers Results available

Phase 1 Study of ALXN1840 on the Metabolism of a CYP2C9 Substrate in Healthy Participants.

NCT04526197
View directory record Official record
Conditions
Wilson Disease
Interventions
ALXN1840, Celecoxib
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 50 Years
Enrollment
36 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020
U.S. locations
1
States / cities
Austin, Texas
Source: ClinicalTrials.gov public record
Updated Aug 20, 2023 · Synced Sep 3, 2026, 10:45 PM EDT
Terminated Phase 2 Interventional Results available

Severe Renal Disease Study in Fabry Patients Treated With Fabrazyme

NCT00837824
View directory record Official record
Conditions
Fabry Disease, Chronic Kidney Disease, Stage IV (Severe)
Interventions
Fabrazyme (agalsidase beta)
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
16 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2003
U.S. locations
9
States / cities
Farmington, Connecticut • Coral Springs, Florida • Kansas City, Kansas + 6 more
Source: ClinicalTrials.gov public record
Updated Apr 6, 2015 · Synced Sep 3, 2026, 10:45 PM EDT
Terminated No phase listed Observational

Behavioral Effects of Kuvan in Children With Mild Phenylketonuria

NCT00827762
View directory record Official record
Conditions
Phenylketonuria
Interventions
Kuvan
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
6 Years to 18 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2010
U.S. locations
4
States / cities
Chicago, Illinois • Columbia, Missouri • St Louis, Missouri + 1 more
Source: ClinicalTrials.gov public record
Updated Dec 4, 2013 · Synced Sep 3, 2026, 10:45 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Large-Scale Metabolomic Profiling for the Diagnosis of Inborn Errors of Metabolism

NCT04201067
View directory record Official record
Conditions
Congenital Disorders of Glycosylation
Interventions
Not listed
Lead sponsor
Mayo Clinic
Other
Eligibility
Not listed
Enrollment
240 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2025
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Oct 2, 2025 · Synced Sep 3, 2026, 10:45 PM EDT