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ClinicalTrials.gov public records Last synced Oct 26, 2026, 4:49 AM EDT

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Showing 361–384 of 2,421 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Metabolic Abnormalities Clear all
Active, not recruiting Not applicable Interventional

Weight Management and Health Behavior Intervention in Lowering Cancer Risk for BRCA Positive and Lynch Syndrome Families

NCT04125914
View directory record Official record
Conditions
Hereditary Breast Carcinoma, Hereditary Ovarian Carcinoma, Lynch Syndrome
Interventions
Behavioral Dietary Intervention, E-mail, Exercise Intervention, Health Promotion and Education, Internet-Based Intervention, Questionnaire Administration, Telephone-Based Intervention
Behavioral · Other
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
18 Years and older
Enrollment
384 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2028
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Oct 6, 2026 · Synced Oct 26, 2026, 4:49 AM EDT
Enrolling by invitation Phase 1 Interventional

PET Imaging With [18F]Fluselenamyl in People With Cardiac Amyloidosis

NCT07771621
View directory record Official record
Conditions
Cardiac Amyloidosis
Interventions
[18F]Fluselenamyl (FSA)
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
18 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Aug 18, 2026 · Synced Oct 26, 2026, 4:49 AM EDT
Completed No phase listed Observational

Real World Evidence Study in Subjects With Wilson's Disease

NCT05783687
View directory record Official record
Conditions
Wilson Disease
Interventions
NCC-Sp Assay
Diagnostic Test
Lead sponsor
Orphalan
Industry
Eligibility
18 Years and older
Enrollment
50 participants
Timeline
2023 – 2025
U.S. locations
9
States / cities
Sacramento, California • New Haven, Connecticut • Miami, Florida + 6 more
Source: ClinicalTrials.gov public record
Updated Apr 6, 2026 · Synced Oct 26, 2026, 4:49 AM EDT
Recruiting No phase listed Observational

Avalglucosidase Alfa Pregnancy Study

NCT05734521
View directory record Official record
Conditions
Pompe Disease, Pregnancy
Interventions
avalglucosidase alfa-NGPT (GZ402666) IV, avalglucosidase alfa-NGPT (GZ402666)
Biological
Lead sponsor
Sanofi
Industry
Eligibility
Female only
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2032
U.S. locations
1
States / cities
Bridgewater, New Jersey
Source: ClinicalTrials.gov public record
Updated Jan 16, 2026 · Synced Oct 26, 2026, 4:49 AM EDT
Withdrawn Phase 4 Interventional

A Study in Patients With Fabry Disease Who Are on Chronic Hemodialysis Therapy for Treatment of End-stage Renal Insufficiency.

NCT00312767
View directory record Official record
Conditions
Fabry Disease
Interventions
Fabrazyme (agalsidase beta)
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years to 65 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2006
U.S. locations
1
States / cities
North Charleston, South Carolina
Source: ClinicalTrials.gov public record
Updated Feb 5, 2014 · Synced Oct 26, 2026, 4:49 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Hematopoietic Stem Cell Transplantation for Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using Total Body Irradiation, Cyclophosphamide and Fludarabine

NCT00595127
View directory record Official record
Conditions
Fanconi Anemia
Interventions
Cyclophosphamide, Fludarabine, Total body irradiation (TBI)
Drug · Radiation
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
Not listed
Enrollment
21 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2001 – 2013
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Aug 10, 2016 · Synced Oct 26, 2026, 4:49 AM EDT
Completed Phase 2 Interventional Results available

A Phase 2 Study to Evaluate the Safety and Efficacy of Pitolisant in Patients With Prader-Willi Syndrome, Followed by an Open Label Extension

NCT04257929
View directory record Official record
Conditions
Prader-Willi Syndrome
Interventions
Pitolisant oral tablets, Placebo oral tablet
Drug
Lead sponsor
Harmony Biosciences Management, Inc.
Industry
Eligibility
6 Years to 65 Years
Enrollment
65 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
13
States / cities
San Diego, California • San Ramon, California • Santa Monica, California + 10 more
Source: ClinicalTrials.gov public record
Updated May 12, 2026 · Synced Oct 26, 2026, 4:49 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Comparative Evaluation of the FINDER Instrument and FINDER G6PD Cartridge in Adults and Neonates

NCT04146246
View directory record Official record
Conditions
G6PD, G6PD Deficiency
Interventions
G6PD assay
Diagnostic Test
Lead sponsor
Baebies, Inc.
Industry
Eligibility
35 Weeks to 100 Years
Enrollment
119 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2020
U.S. locations
3
States / cities
Durham, North Carolina • Raleigh, North Carolina • Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Nov 23, 2020 · Synced Oct 26, 2026, 4:49 AM EDT
Active, not recruiting No phase listed Observational

Long-Term Follow-Up of Subjects Treated With AXO-AAV-GM2 for Tay-Sachs or Sandhoff Disease

NCT06614569
View directory record Official record
Conditions
GM2 Gangliosidosis, Tay Sachs Disease, Sandhoff Disease
Interventions
AXO-AAV-GM2
Drug
Lead sponsor
Terence Flotte
Other
Eligibility
Not listed
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
1
States / cities
Worcester, Massachusetts
Source: ClinicalTrials.gov public record
Updated Oct 16, 2025 · Synced Oct 26, 2026, 4:49 AM EDT
Active, not recruiting Not applicable Interventional

Impact of Bright Light Therapy on Prader-Willi Syndrome

NCT05939453
View directory record Official record
Conditions
Prader-Willi Syndrome, Excessive Daytime Sleepiness, Hyperphagia, Body Weight, Mood, Behavior
Interventions
Sham Light, Bright Light Therapy
Other
Lead sponsor
Maimonides Medical Center
Other
Eligibility
6 Years to 88 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
1
States / cities
Brooklyn, New York
Source: ClinicalTrials.gov public record
Updated Oct 6, 2026 · Synced Oct 26, 2026, 4:49 AM EDT
Terminated Phase 2 Interventional

Human Heterologous Liver Cells for Infusion in Children With Urea Cycle Disorders

NCT01195753
View directory record Official record
Conditions
Urea Cycle Disorders
Interventions
HHLivC
Biological
Lead sponsor
Cytonet GmbH & Co. KG
Industry
Eligibility
1 Day to 5 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2015
U.S. locations
4
States / cities
Palo Alto, California • San Diego, California • New Haven, Connecticut + 1 more
Source: ClinicalTrials.gov public record
Updated Feb 8, 2016 · Synced Oct 26, 2026, 4:49 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)

NCT04411654
View directory record Official record
Conditions
Gaucher Disease, Type 2
Interventions
LY3884961, Methylprednisolone, Sirolimus, Prednisone
Genetic · Drug
Lead sponsor
Prevail Therapeutics
Industry
Eligibility
0 Months to 24 Months
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
5
States / cities
Oakland, California • Minneapolis, Minnesota • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Oct 22, 2026 · Synced Oct 26, 2026, 4:49 AM EDT
Enrolling by invitation No phase listed Observational Accepts healthy volunteers

Advanced Ultrasound Signal Processing of Suspicious Breast Images

NCT07206888
View directory record Official record
Conditions
Breast Cancer, Breast Cyst, Microcalcification
Interventions
Imaging
Diagnostic Test
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years and older · Female only
Enrollment
252 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2037
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 14, 2025 · Synced Oct 26, 2026, 4:49 AM EDT
Recruiting No phase listed Observational

LCAT (Lecithin Cholesterol Acyl Transferase) Natural History Study

NCT06217588
View directory record Official record
Conditions
LCAT Deficiency
Interventions
Demographics, diagnosis type, genotype, lipid profile, renal function profile, treatment allocation, ophthalmology exam, country of residence.
Other
Lead sponsor
University of Pennsylvania
Other
Eligibility
Not listed
Enrollment
40 participants
Timeline
2022 – 2028
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated May 6, 2026 · Synced Oct 26, 2026, 4:49 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Feasibility, Acceptability and Directional Signal Effect on Blood Folate Levels of Iodized Salt Fortified With Folic Acid: Clinical Study

NCT05935631
View directory record Official record
Conditions
Neural Tube Defects, Folic Acid Deficiency, Fortification
Interventions
Salt with folic acid and iodine
Dietary Supplement
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
18 Years to 40 Years · Female only
Enrollment
32 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023
U.S. locations
1
States / cities
Birmingham, Alabama
Source: ClinicalTrials.gov public record
Updated Aug 1, 2023 · Synced Oct 26, 2026, 4:49 AM EDT
Completed Phase 2 Interventional Results available

Open Label Extension of ISIS 301012 (Mipomersen) to Treat Familial Hypercholesterolemia

NCT00477594
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
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2011
U.S. locations
5
States / cities
Chicago, Illinois • Auburn, Maine • Biddeford, Maine + 2 more
Source: ClinicalTrials.gov public record
Updated Sep 9, 2016 · Synced Oct 26, 2026, 4:49 AM EDT
Completed Phase 1Phase 2 Interventional

Phase I/II Study of Retroviral-Mediated Transfer of Iduronate-2-Sulfatase Gene Into Lymphocytes of Patients With Mucopolysaccharidosis II (Mild Hunter Syndrome)

NCT00004454
View directory record Official record
Conditions
Mucopolysaccharidosis II
Interventions
lymphocyte gene therapy
Genetic
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
18 Years and older
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1996
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 26, 2026, 4:49 AM EDT
Completed Phase 2 Interventional Results available

Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase Deficiency

NCT02112994
View directory record Official record
Conditions
Lysosomal Acid Lipase Deficiency
Interventions
Sebelipase Alfa
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
8 Months and older
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
3
States / cities
Chicago, Illinois • Shreveport, Louisiana • Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Dec 4, 2019 · Synced Oct 26, 2026, 4:49 AM EDT
Not listed Phase 3 Interventional

AT-007 in Adult Subjects With Classic Galactosemia (CG)

NCT05418829
View directory record Official record
Conditions
Classic Galactosemia
Interventions
AT-007
Drug
Lead sponsor
Applied Therapeutics, Inc.
Industry
Eligibility
18 Years to 65 Years
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
1
States / cities
Berlin, New Jersey
Source: ClinicalTrials.gov public record
Updated Jun 14, 2022 · Synced Oct 26, 2026, 4:49 AM EDT
Active, not recruiting Phase 3 Interventional

A Study to Evaluate the Safety and Efficacy of Zilganersen (ION373) in Patients With Alexander Disease (AxD)

NCT04849741
View directory record Official record
Conditions
Alexander Disease
Interventions
zilganersen, Placebo
Drug
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
2 Years to 65 Years
Enrollment
54 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2034
U.S. locations
4
States / cities
Palo Alto, California • Atlanta, Georgia • Boston, Massachusetts + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 21, 2026 · Synced Oct 26, 2026, 4:49 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH)

NCT04783181
View directory record Official record
Conditions
Congenital Adrenal Hyperplasia
Interventions
AAV BBP-631
Biological
Lead sponsor
Adrenas Therapeutics Inc
Industry
Eligibility
18 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2029
U.S. locations
5
States / cities
Chicago, Illinois • Bethesda, Maryland • Minneapolis, Minnesota + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 23, 2025 · Synced Oct 26, 2026, 4:49 AM EDT
Terminated No phase listed Observational

Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX

NCT04628871
View directory record Official record
Conditions
Hemophilia B, Mucopolysaccharidosis I, Mucopolysaccharidosis II
Interventions
SB-318, SB-913, SB-FIX
Biological
Lead sponsor
Sangamo Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
5
States / cities
Oakland, California • Chicago, Illinois • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Aug 13, 2026 · Synced Oct 26, 2026, 4:49 AM EDT
Completed Not applicable Interventional Results available

Vitamin D in HIV-Infected Patients on HAART

NCT01250899
View directory record Official record
Conditions
Vitamin D Deficiency, HIV
Interventions
Vitamin D
Dietary Supplement
Lead sponsor
University of California, Los Angeles
Other
Eligibility
18 Years to 90 Years
Enrollment
122 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2012
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Nov 25, 2014 · Synced Oct 26, 2026, 4:49 AM EDT
Recruiting Phase 2 Interventional

Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders

NCT03579875
View directory record Official record
Conditions
Fanconi Anemia, Severe Aplastic Anemia, Myelodysplastic Syndromes, T Cell Receptor Alpha/Beta Depletion, Telomere Biology Disorder, Bone Marrow Failure, Dyskeratosis Congenita, Telomere Biology Disorders
Interventions
Total Body Irradiation (TBI) (Plan 1), Cyclophosphamide (CY) (Plan 1), Fludarabine (FLU), Methylprednisolone (MP), Donor mobilized PBSC infusion, G-CSF, Cyclophosphamide (CY) (Plan 2), Rituximab, Busulfan, Alemtuzumab, Melphalan
Drug · Device
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 65 Years
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2029
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jan 30, 2026 · Synced Oct 26, 2026, 4:49 AM EDT