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ClinicalTrials.gov public records Last synced Oct 1, 2026, 5:22 PM EDT

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Showing 25–48 of 2,403 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Inherited Metabolic Disease Clear all
Enrolling by invitation Phase 3 Interventional

Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA

NCT04360265
View directory record Official record
Conditions
Mucopolysaccharidosis IIIA, MPS IIIA, Sanfilippo Syndrome, Sanfilippo A
Interventions
No Investigational Product, Adjuvant Immunomodulatory (IM) Therapy
Other · Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
Not listed
Enrollment
41 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Jul 17, 2026 · Synced Oct 1, 2026, 5:22 PM EDT
Recruiting Phase 1Phase 2 Interventional

Campath/Fludarabine/Melphalan Transplant Conditioning for Non-Malignant Diseases

NCT00920972
View directory record Official record
Conditions
Metabolic Disorders, Hematologic, Immune, or Bone Marrow Disorders, Hemoglobinopathies, Non-malignant Disorders
Interventions
Treatment Plan 1: Stratum 1, Treatment Plan 2: Strata 2, 3, or 4, GVHD Regimen A: UCB Recipients, GVHD Regimen B: BM Recipients
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
Up to 20 Years
Enrollment
220 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2031
U.S. locations
25
States / cities
Phoenix, Arizona • Orange, California • San Diego, California + 20 more
Source: ClinicalTrials.gov public record
Updated Sep 16, 2026 · Synced Oct 1, 2026, 5:22 PM EDT
Completed No phase listed Observational

Sjogren-Larsson Syndrome: Natural History, Clinical Variation and Evaluation of Biochemical Markers

NCT01971957
View directory record Official record
Conditions
Sjogren-Larsson Syndrome (SLS)
Interventions
Not listed
Lead sponsor
University of Nebraska
Other
Eligibility
Not listed
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2023
U.S. locations
2
States / cities
Omaha, Nebraska • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 8, 2024 · Synced Oct 1, 2026, 5:22 PM EDT
Completed Phase 2 Interventional Accepts healthy volunteers

A Study to Evaluate OPTI-BIOME™ Bacillus Subtilis MB40 on Abdominal Discomfort, Gas and Bloating in a Healthy Population

NCT02950012
View directory record Official record
Conditions
Bloating
Interventions
OPTI-BIOME™ Bacillus subtilis MB40, Placebo
Dietary Supplement · Other
Lead sponsor
BIO-CAT Microbials, LLC
Industry
Eligibility
18 Years to 75 Years
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2017
U.S. locations
2
States / cities
Foothill Ranch, California • Orlando, Florida
Source: ClinicalTrials.gov public record
Updated Oct 29, 2021 · Synced Oct 1, 2026, 5:22 PM EDT
No Longer Available No phase listed Expanded access

Emergency Administration of EPI-743 to a Single Patient With Leber's Hereditary Optic Neuropathy [LHON]

NCT02300753
View directory record Official record
Conditions
Leber's Hereditary Optic Neuropathy
Interventions
EPI-743
Drug
Lead sponsor
Edison Pharmaceuticals Inc
Industry
Eligibility
8 Years to 65 Years
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Nov 25, 2014 · Synced Oct 1, 2026, 5:22 PM EDT
Recruiting Not applicable Interventional

Regulating Together for Prader-Willi Syndrome: A Group Behavioral Therapy for Emotion Dysregulation

NCT07607730
View directory record Official record
Conditions
Prader-Willi Syndrome
Interventions
Regulating Together group therapy
Behavioral
Lead sponsor
Children's Mercy Hospital Kansas City
Other
Eligibility
13 Years to 17 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2027
U.S. locations
1
States / cities
Kansas City, Missouri
Source: ClinicalTrials.gov public record
Updated Sep 2, 2026 · Synced Oct 1, 2026, 5:22 PM EDT
Active, not recruiting No phase listed Observational Accepts healthy volunteers

Diffusion Tensor Imaging (DTI) in Infants With Krabbe Disease

NCT00787865
View directory record Official record
Conditions
Krabbe Disease
Interventions
Not listed
Lead sponsor
University of Pittsburgh
Other
Eligibility
Up to 17 Years
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2008 – 2027
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 6, 2026 · Synced Oct 1, 2026, 5:22 PM EDT
Terminated Phase 4 Interventional Results available

Long-term Safety and Efficacy of Ferriprox® in Iron Overloaded Patients With Sickle Cell Disease or Other Anemias

NCT02443545
View directory record Official record
Conditions
Iron Overload, Sickle Cell Disease, Other Anemias
Interventions
Deferiprone
Drug
Lead sponsor
ApoPharma
Industry
Eligibility
3 Years and older
Enrollment
134 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
5
States / cities
Oakland, California • Ann Arbor, Michigan • Detroit, Michigan + 2 more
Source: ClinicalTrials.gov public record
Updated Jan 10, 2024 · Synced Oct 1, 2026, 5:22 PM EDT
Terminated Not applicable Interventional Results available

Methadone, Morphine, or Oxycodone in Treating Pain in Patients With Cancer

NCT00726830
View directory record Official record
Conditions
Brain and Central Nervous System Tumors, Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Lymphoproliferative Disorder, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms, Pain, Precancerous Condition, Unspecified Adult Solid Tumor, Protocol Specific
Interventions
methadone hydrochloride, morphine sulfate, oxycodone hydrochloride
Drug
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
18 Years and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2010
U.S. locations
2
States / cities
Spartanburg, South Carolina • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Sep 24, 2020 · Synced Oct 1, 2026, 5:22 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Bioavailability Study of Ciprofloxacin 1000 mg ER Tablets of Dr. Reddy's Under Non-Fasting Conditions

NCT01372358
View directory record Official record
Conditions
Fed
Interventions
CIPRO®XR
Drug
Lead sponsor
Dr. Reddy's Laboratories Limited
Industry
Eligibility
18 Years to 45 Years
Enrollment
32 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2005
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jun 13, 2011 · Synced Oct 1, 2026, 5:22 PM EDT
Terminated Phase 1 Interventional

12-O-Tetradecanoylphorbol-13-acetate in Treating Patients With Hematologic Cancer or Bone Marrow Disorder

NCT00004058
View directory record Official record
Conditions
Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases, Precancerous/Nonmalignant Condition
Interventions
tetradecanoylphorbol acetate
Drug
Lead sponsor
University of Medicine and Dentistry of New Jersey
Other
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2004
U.S. locations
1
States / cities
New Brunswick, New Jersey
Source: ClinicalTrials.gov public record
Updated Jan 26, 2010 · Synced Oct 1, 2026, 5:22 PM EDT
Completed Phase 2 Interventional

A Single-Dose Open-Label Study of XOMA 358 in Subjects With Congenital Hyperinsulinism

NCT02604485
View directory record Official record
Conditions
Congenital Hyperinsulinism
Interventions
Cohort 1, Cohort 2, Cohort 3, Cohort 4
Drug
Lead sponsor
XOMA (US) LLC
Industry
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Feb 6, 2017 · Synced Oct 1, 2026, 5:22 PM EDT
Completed Phase 3 Interventional Results available

Safety and Efficacy of Tafamidis in Patients With Transthyretin Cardiomyopathy

NCT01994889
View directory record Official record
Conditions
Transthyretin (TTR) Amyloid Cardiomyopathy
Interventions
Tafamidis, Placebo
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 90 Years
Enrollment
441 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2018
U.S. locations
50
States / cities
Birmingham, Alabama • Phoenix, Arizona • Scottsdale, Arizona + 24 more
Source: ClinicalTrials.gov public record
Updated Apr 24, 2019 · Synced Oct 1, 2026, 5:22 PM EDT
Completed Phase 3 Interventional Results available

Safety/Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Cysteamine Treatment Naive Patients With Cystinosis

NCT01744782
View directory record Official record
Conditions
Cystinosis
Interventions
RP103
Drug
Lead sponsor
Amgen
Industry
Eligibility
Up to 6 Years
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Dec 27, 2024 · Synced Oct 1, 2026, 5:22 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Study of the Absorption, Metabolism, and Excretion of GBT440 in Healthy Male Subjects

NCT02497924
View directory record Official record
Conditions
Anemia, Sickle Cell
Interventions
GBT440
Drug
Lead sponsor
Global Blood Therapeutics
Industry
Eligibility
18 Years to 55 Years · Male only
Enrollment
7 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2016
U.S. locations
1
States / cities
Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Apr 12, 2017 · Synced Oct 1, 2026, 5:22 PM EDT
Completed Phase 2 Interventional Results available

Controlled Trial of Panhematin in Treatment of Acute Attacks of Porphyria

NCT02180412
View directory record Official record
Conditions
Acute Porphyrias
Interventions
Panhematin, Glucose
Biological · Other
Lead sponsor
The University of Texas Medical Branch, Galveston
Other
Eligibility
18 Years to 100 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2022
U.S. locations
1
States / cities
Galveston, Texas
Source: ClinicalTrials.gov public record
Updated Apr 11, 2025 · Synced Oct 1, 2026, 5:22 PM EDT
Recruiting Phase 3 Interventional

Study to Evaluate Efficacy and Safety of Inclisiran in Children With Heterozygous Familial Hypercholesterolemia

NCT06597019
View directory record Official record
Conditions
Familial Hypercholesterolemia - Heterozygous
Interventions
Inclisiran, Placebo
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
6 Years to 11 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
9
States / cities
San Francisco, California • Washington D.C., District of Columbia • Boca Raton, Florida + 4 more
Source: ClinicalTrials.gov public record
Updated May 15, 2026 · Synced Oct 1, 2026, 5:22 PM EDT
Recruiting Phase 1Phase 2 Interventional

NORTHERA (DROXIDOPA) for Dysautonomia in Pediatric Survivors of Menkes Disease

NCT07398508
View directory record Official record
Conditions
Menkes Disease
Interventions
Droxidopa Oral Product, Placebo Control
Drug · Other
Lead sponsor
Stephen G. Kaler
Other
Eligibility
7 Years to 17 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 20, 2026 · Synced Oct 1, 2026, 5:22 PM EDT
Withdrawn Not applicable Interventional

Iron Prophylaxis for Anemia in Infants With Cyanotic Congenital Heart Disease

NCT00459225
View directory record Official record
Conditions
Anemia
Interventions
Iron (ferrous sulfate)
Drug
Lead sponsor
Emory University
Other
Eligibility
Up to 2 Months
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2013
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Dec 9, 2014 · Synced Oct 1, 2026, 5:22 PM EDT
Completed Phase 2 Interventional Results available

A Study to Evaluate Efficacy and Safety of Anakinra in the Treatment of Acute Gouty Arthritis

NCT03002974
View directory record Official record
Conditions
Acute Gouty Arthritis
Interventions
Anakinra 100 mg, Triamcinolone Acetonide 40 mg, Placebo to Anakinra 100 mg, Placebo to Triamcinolone Acetonide 40 mg
Drug
Lead sponsor
Swedish Orphan Biovitrum
Industry
Eligibility
18 Years and older
Enrollment
165 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
37
States / cities
Birmingham, Alabama • Gulf Shores, Alabama • Mobile, Alabama + 32 more
Source: ClinicalTrials.gov public record
Updated Jul 15, 2020 · Synced Oct 1, 2026, 5:22 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Intranasal Oxytocin for Infants With Prader-Willi Syndrome

NCT03245762
View directory record Official record
Conditions
Prader-Willi Syndrome
Interventions
Oxytocin, Placebo
Drug
Lead sponsor
University of Florida
Other
Eligibility
1 Week to 6 Months
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2018
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Mar 26, 2020 · Synced Oct 1, 2026, 5:22 PM EDT
Completed Phase 1Phase 2 Interventional

Cholesterol in ASD: Characterization and Treatment

NCT00965068
View directory record Official record
Conditions
Autism, Asperger Disorder, PDD-NOS
Interventions
Cholesterol
Other
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
4 Years to 12 Years
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2013
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 5, 2018 · Synced Oct 1, 2026, 5:22 PM EDT
Terminated No phase listed Observational Accepts healthy volunteers

Gene Function in Bone Marrow Cells From Patients With Fanconi Anemia and From Healthy Participants

NCT00896740
View directory record Official record
Conditions
Fanconi Anemia
Interventions
microarray analysis, biopsy
Genetic · Procedure
Lead sponsor
OHSU Knight Cancer Institute
Other
Eligibility
1 Year to 55 Years
Enrollment
90 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2002 – 2007
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Dec 4, 2017 · Synced Oct 1, 2026, 5:22 PM EDT
Completed Phase 1 Interventional

Study to Evaluate Safety, Tolerability, PK and PD of DCR-PHXC in PH Type 3 Patients

NCT04555486
View directory record Official record
Conditions
Primary Hyperoxaluria Type 3
Interventions
DCR-PHXC, Sterile Normal Saline (0.9% NaCl)
Drug
Lead sponsor
Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
Industry
Eligibility
6 Years and older
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
3
States / cities
Boston, Massachusetts • Rochester, Minnesota • New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 19, 2024 · Synced Oct 1, 2026, 5:22 PM EDT