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ClinicalTrials.gov public records Last synced Oct 20, 2026, 10:16 PM EDT

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Showing 145–168 of 10,330 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Congenital, Hereditary, and Neonatal Diseases and Abnormalities 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 20, 2026, 10:16 PM EDT
Completed Phase 2 Interventional

PTC124 for Cystic Fibrosis

NCT00234663
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
PTC124
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2006
U.S. locations
5
States / cities
Birmingham, Alabama • Palo Alto, California • Denver, Colorado + 2 more
Source: ClinicalTrials.gov public record
Updated Jan 13, 2009 · Synced Oct 20, 2026, 10:16 PM EDT
Completed Phase 4 Interventional Accepts healthy volunteers

The Role of 5-alpha Reductase in Mediating Testosterone Actions

NCT00493987
View directory record Official record
Conditions
Healthy
Interventions
Testosterone Enanthate, Dutasteride
Drug
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
18 Years to 50 Years · Male only
Enrollment
184 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2002 – 2010
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated May 13, 2010 · Synced Oct 20, 2026, 10:16 PM EDT
Completed Not applicable Interventional Results available

Closure of Muscular Ventricular Septal Defects (VSDs) With the AMPLATZER Muscular VSD (MuVSD) Occluder - Post Approval Study

NCT00647387
View directory record Official record
Conditions
Ventricular Septal Defects
Interventions
Muscular VSD Occluder device implantation (AMPLATZER Muscular VSD Occluder)
Device
Lead sponsor
Abbott Medical Devices
Industry
Eligibility
Not listed
Enrollment
92 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2024
U.S. locations
48
States / cities
Phoenix, Arizona • Los Angeles, California • Madera, California + 40 more
Source: ClinicalTrials.gov public record
Updated Feb 11, 2025 · Synced Oct 20, 2026, 10:16 PM EDT
Completed Phase 3 Interventional Results available

Phase III Study of Idebenone in Duchenne Muscular Dystrophy (DMD)

NCT01027884
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne, Ambulatory Care
Interventions
Placebo, Idebenone
Drug
Lead sponsor
Santhera Pharmaceuticals
Industry
Eligibility
10 Years to 18 Years · Male only
Enrollment
65 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2014
U.S. locations
9
States / cities
Sacramento, California • Aurora, Colorado • Gainesville, Florida + 6 more
Source: ClinicalTrials.gov public record
Updated Oct 19, 2015 · Synced Oct 20, 2026, 10:16 PM EDT
Completed No phase listed Observational

A Non-Drug Study of The Suitability of Neurocognitive Tests And Functioning Scales For The Measurement of Cognitive And Functioning Changes in Individuals With Down Syndrome

NCT01580384
View directory record Official record
Conditions
Down Syndrome
Interventions
Not listed
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
12 Years to 30 Years
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2014
U.S. locations
2
States / cities
Tucson, Arizona • Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Mar 3, 2015 · Synced Oct 20, 2026, 10:16 PM EDT
Completed Phase 1 Interventional

RLY-1971 in Subjects With Advanced or Metastatic Solid Tumors

NCT04252339
View directory record Official record
Conditions
Solid Tumor, Unspecified, Adult
Interventions
RLY-1971
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
18 Years and older
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
6
States / cities
Lake Mary, Florida • Sarasota, Florida • Boston, Massachusetts + 1 more
Source: ClinicalTrials.gov public record
Updated Jan 31, 2023 · Synced Oct 20, 2026, 10:16 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex (TSC) and Neurofibromatosis I (NF1)

NCT01031901
View directory record Official record
Conditions
Tuberous Sclerosis, Neurofibromatoses, Angiofibroma, Neurofibroma
Interventions
Skincerity, Skincerity plus sirolimus/rapamycin, Skinercity plus sirolimus/rapamycin
Drug
Lead sponsor
The University of Texas Health Science Center, Houston
Other
Eligibility
13 Years and older
Enrollment
52 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2011
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Feb 27, 2012 · Synced Oct 20, 2026, 10:16 PM EDT
Completed Phase 2 Interventional

A Study With RO4917523 in Patients With Fragile X Syndrome

NCT01015430
View directory record Official record
Conditions
Fragile X Syndrome
Interventions
Placebo (for RO4917523 ascending doses), Placebo (for RO4917523 fixed dose), RO4917523
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
18 Years to 50 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2011
U.S. locations
5
States / cities
Sacramento, California • Decatur, Georgia • Chicago, Illinois + 2 more
Source: ClinicalTrials.gov public record
Updated Nov 2, 2016 · Synced Oct 20, 2026, 10:16 PM EDT
Terminated Phase 1 Interventional

A Study to Investigate the Safety, Tolerability, and Pharmacokinetics of Oral GSK4172239D Compared With Placebo in Sickle Cell Disease Participants Aged 18 to 50 Years

NCT05660265
View directory record Official record
Conditions
Hematologic Diseases, Anaemia, Sickle Cell
Interventions
GSK4172239D, Placebo
Drug · Other
Lead sponsor
GlaxoSmithKline
Industry
Eligibility
18 Years to 50 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
6
States / cities
Miami, Florida • Tamarac, Florida • Atlanta, Georgia + 3 more
Source: ClinicalTrials.gov public record
Updated Jan 22, 2026 · Synced Oct 20, 2026, 10:16 PM EDT
Completed Phase 1 Interventional

Combination Chemotherapy Followed By Umbilical Cord Blood Transplantation in Treating Patients With Hematologic Cancer or Severe Aplastic Anemia

NCT00054236
View directory record Official record
Conditions
Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases
Interventions
anti-thymocyte globulin, filgrastim, cyclophosphamide, fludarabine phosphate, umbilical cord blood transplantation, methylprednisolone
Biological · Drug · Procedure
Lead sponsor
Case Comprehensive Cancer Center
Other
Eligibility
Up to 120 Years
Enrollment
55 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2011
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Jul 27, 2020 · Synced Oct 20, 2026, 10:16 PM EDT
Terminated Phase 2 Interventional Results available

Open-Label Study of Perhexiline in Patients With Hypertrophic Cardiomyopathy and Moderate to Severe Heart Failure

NCT02862600
View directory record Official record
Conditions
Cardiomyopathy, Hypertrophic, Cardiomyopathy, Hypertrophic, Familial
Interventions
Perhexiline, Use of bioanalytical assay to monitor plasma levels of perhexiline
Drug · Device
Lead sponsor
Heart Metabolics Limited
Industry
Eligibility
18 Years and older
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
11
States / cities
Stanford, California • Indianapolis, Indiana • Baltimore, Maryland + 7 more
Source: ClinicalTrials.gov public record
Updated Aug 31, 2017 · Synced Oct 20, 2026, 10:16 PM EDT
Not listed No phase listed Observational

Cure Cystinosis International Registry

NCT01327807
View directory record Official record
Conditions
Cystinosis, Nephropathic Cystinosis, Renal Fanconi Syndrome
Interventions
Not listed
Lead sponsor
Cystinosis Research Foundation
Other
Eligibility
Not listed
Enrollment
750 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2022
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated Mar 12, 2014 · Synced Oct 20, 2026, 10:16 PM EDT
Active, not recruiting No phase listed Observational

Longitudinal Assessment of Atypical Tripeptidyl Peptidase 1 Enzyme Deficiency Patients

NCT04098211
View directory record Official record
Conditions
Neuronal Ceroid-Lipofuscinoses, Neuronal Ceroid Lipofuscinosis CLN2, Spinocerebellar Ataxia, Autosomal Recessive 7
Interventions
Not listed
Lead sponsor
Children's Hospital of Orange County
Other
Eligibility
4 Years and older
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
1
States / cities
Orange, California
Source: ClinicalTrials.gov public record
Updated Apr 8, 2026 · Synced Oct 20, 2026, 10:16 PM EDT
Recruiting No phase listed Observational

Trial-Ready Cohort-Down Syndrome (TRC-DS)

NCT04165109
View directory record Official record
Conditions
Down Syndrome, Alzheimer Disease, Dementia
Interventions
Not listed
Lead sponsor
University of Southern California
Other
Eligibility
25 Years to 55 Years
Enrollment
550 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
18
States / cities
Phoenix, Arizona • Orange, California • Aurora, Colorado + 15 more
Source: ClinicalTrials.gov public record
Updated May 18, 2026 · Synced Oct 20, 2026, 10:16 PM EDT
Terminated Phase 1 Interventional

Gene Expression During Chemotherapy in Patients With Newly Diagnosed Acute Myeloid Leukemia Treated With Choline Magnesium Trisalicylate

NCT00156299
View directory record Official record
Conditions
Leukemia
Interventions
choline magnesium trisalicylate, Dexamethasone
Drug
Lead sponsor
Rutgers, The State University of New Jersey
Other
Eligibility
18 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2008
U.S. locations
1
States / cities
New Brunswick, New Jersey
Source: ClinicalTrials.gov public record
Updated Nov 19, 2013 · Synced Oct 20, 2026, 10:16 PM EDT
Completed No phase listed Observational

Microarray Analysis for Human Genetic Disease

NCT00001898
View directory record Official record
Conditions
Breast Neoplasm, Hereditary Neoplastic Syndrome, Melanoma, Ovarian Neoplasm
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
Not listed
Enrollment
1,500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2008
U.S. locations
5
States / cities
Tucson, Arizona • Baltimore, Maryland • Ann Arbor, Michigan + 2 more
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 20, 2026, 10:16 PM EDT
Completed Phase 3 Interventional Results available

Phase III Confirmatory Study in Erythropoietic Protoporphyria

NCT01605136
View directory record Official record
Conditions
Erythropoietic Protoporphyria
Interventions
Afamelanotide, Placebo
Drug
Lead sponsor
Clinuvel Pharmaceuticals Limited
Industry
Eligibility
18 Years and older
Enrollment
93 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
7
States / cities
Birmingham, Alabama • San Francisco, California • Detroit, Michigan + 4 more
Source: ClinicalTrials.gov public record
Updated Sep 19, 2019 · Synced Oct 20, 2026, 10:16 PM EDT
Recruiting Phase 1Phase 2 Interventional

Study to Evaluate ACDN-01 in ABCA4-related Stargardt Retinopathy (STELLAR)

NCT06467344
View directory record Official record
Conditions
Stargardt Disease, Cone Rod Dystrophy, Juvenile Macular Degeneration, Stargardt Disease 1
Interventions
ACDN-01
Drug
Lead sponsor
Ascidian Therapeutics, Inc
Industry
Eligibility
12 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2030
U.S. locations
11
States / cities
San Francisco, California • Gainesville, Florida • Miami, Florida + 8 more
Source: ClinicalTrials.gov public record
Updated Oct 7, 2026 · Synced Oct 20, 2026, 10:16 PM EDT
Completed Not applicable Interventional

Skin and Serum Carotenoids in Preterm Infants Fed on a Formula Supplemented With Carotenoids

NCT00872664
View directory record Official record
Conditions
Retinopathy of Prematurity
Interventions
carotenoids
Dietary Supplement
Lead sponsor
University of Utah
Other
Eligibility
Up to 21 Days
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2013
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Dec 4, 2013 · Synced Oct 20, 2026, 10:16 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Congenital Heart Initiative-Redefining Outcomes and Navigation to Adult Centered Care

NCT05185232
View directory record Official record
Conditions
Congenital Heart Disease, Comorbidities and Coexisting Conditions
Interventions
Impact of gaps in care/loss to healthcare follow-up
Other
Lead sponsor
Children's National Research Institute
Other
Eligibility
18 Years and older
Enrollment
3,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2024
U.S. locations
16
States / cities
San Francisco, California • Aurora, Colorado • Washington D.C., District of Columbia + 9 more
Source: ClinicalTrials.gov public record
Updated Jan 11, 2022 · Synced Oct 20, 2026, 10:16 PM EDT
Completed Phase 2Phase 3 Interventional Results available

Trial of Leptin Replacement Therapy in Patients With Lipodystrophy

NCT00896298
View directory record Official record
Conditions
Hypoleptinemia, Generalized Lipodystrophy, Partial Lipodystrophy, Insulin Resistance
Interventions
Leptin, Placebo
Drug
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
6 Years to 70 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2014
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Mar 27, 2024 · Synced Oct 20, 2026, 10:16 PM EDT
Completed No phase listed Observational

Comparing Expectorated and Induced Sputum and Pharyngeal Swabs for Cultures, AFB Smears, and Cytokines in Pulmonary Nontuberculous Mycobacterial Infection

NCT01150721
View directory record Official record
Conditions
Mycobacterium Infections, Non-tuberculous
Interventions
Not listed
Lead sponsor
National Institutes of Health Clinical Center (CC)
NIH
Eligibility
18 Years to 80 Years
Enrollment
177 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2010
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 13, 2026 · Synced Oct 20, 2026, 10:16 PM EDT
Recruiting Phase 2 Interventional

Phase 2 Study to Assess the Safety and Efficacy of ANG003

NCT07450547
View directory record Official record
Conditions
Exocrine Pancreatic Insufficiency (EPI), Cystic Fibrosis (CF)
Interventions
ANG003 Dose A, ANG003 Dose B, Creon
Drug
Lead sponsor
Anagram Therapeutics, Inc.
Industry
Eligibility
12 Years and older
Enrollment
113 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
27
States / cities
Long Beach, California • Los Angeles, California • Denver, Colorado + 23 more
Source: ClinicalTrials.gov public record
Updated Oct 6, 2026 · Synced Oct 20, 2026, 10:16 PM EDT