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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Terminated Not applicable Interventional

Sterol and Isoprenoid Disease Research Consortium: Smith-Lemli-Opitz Syndrome

NCT01356420
View directory record Official record
Conditions
Smith-Lemli-Opitz Syndrome
Interventions
Cholesterol supplementation
Dietary Supplement
Lead sponsor
Oregon Health and Science University
Other
Eligibility
Up to 85 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2015
U.S. locations
5
States / cities
Bethesda, Maryland • Omaha, Nebraska • Cincinnati, Ohio + 2 more
Source: ClinicalTrials.gov public record
Updated May 22, 2019 · Synced Jul 23, 2026, 10:18 AM EDT
Completed Phase 2 Interventional Results available

Early Administration of ATG Followed by Cyclophosphamide, Busulfan and Fludarabine Before a Donor Stem Cell Transplant in Patients With Hematological Cancer

NCT00787761
View directory record Official record
Conditions
Myeloproliferative Disorders, Kidney Cancer, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases
Interventions
anti-thymocyte globulin, busulfan, cyclophosphamide, fludarabine phosphate, methotrexate, tacrolimus, nonmyeloablative allogeneic HSCT
Biological · Drug · Procedure
Lead sponsor
Northside Hospital, Inc.
Other
Eligibility
Up to 75 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2012
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Sep 30, 2012 · Synced Jul 23, 2026, 10:18 AM EDT
Completed Phase 2 Interventional

Study to Evaluate Efficacy, Safety, and Tolerability of RGH-706 in Prader-Willi Syndrome

NCT05322096
View directory record Official record
Conditions
Prader-Willi Syndrome
Interventions
RGH-706, Placebo
Drug
Lead sponsor
Gedeon Richter Plc.
Industry
Eligibility
17 Years and older
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
6
States / cities
San Diego, California • Chicago, Illinois • Brooklyn, New York + 3 more
Source: ClinicalTrials.gov public record
Updated Jun 2, 2024 · Synced Jul 23, 2026, 10:18 AM EDT
Completed Phase 4 Interventional Results available

Curcumin Therapy to Treat Vascular Dysfunction in Children and Young Adults With ADPKD

NCT02494141
View directory record Official record
Conditions
Polycystic Kidney, Autosomal Dominant
Interventions
Curcumin, Placebo
Drug · Other
Lead sponsor
University of Colorado, Denver
Other
Eligibility
6 Years to 25 Years
Enrollment
68 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2021
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated Mar 2, 2022 · Synced Jul 23, 2026, 10:18 AM EDT
Completed Phase 2 Interventional Results available

A Study to Evaluate Multiple Doses of GLPG2222 in Adult Subjects With Cystic Fibrosis

NCT03119649
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
GLPG2222 50 mg, GLPG2222 100 mg, Placebo, GLPG2222 200 mg, GLPG2222 400 mg
Drug
Lead sponsor
Lakefront Biotherapeutics NV
Industry
Eligibility
18 Years to 99 Years
Enrollment
59 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017
U.S. locations
7
States / cities
Chatom, Alabama • Little Rock, Arkansas • Orlando, Florida + 4 more
Source: ClinicalTrials.gov public record
Updated Nov 15, 2018 · Synced Jul 23, 2026, 10:18 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

National Eye Institute Biorepository for Retinal Diseases

NCT01496625
View directory record Official record
Conditions
Age-Related Macular Degeneration, Diabetic Retinopathy, Von Hippel-Lindau Syndrome, Retinal Disease, Retinal Vein Occlusion
Interventions
Not listed
Lead sponsor
National Eye Institute (NEI)
NIH
Eligibility
2 Years to 120 Years
Enrollment
650 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2012
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 29, 2026 · Synced Jul 23, 2026, 10:18 AM EDT
Completed Phase 2 Interventional Results available

Efficacy and Safety Study of WTX101 (ALXN1840) in Adult Wilson Disease Patients

NCT02273596
View directory record Official record
Conditions
Wilson Disease
Interventions
ALXN1840
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
4
States / cities
Los Angeles, California • New Haven, Connecticut • Chicago, Illinois + 1 more
Source: ClinicalTrials.gov public record
Updated Sep 28, 2021 · Synced Jul 23, 2026, 10:18 AM EDT
Recruiting Phase 3 Interventional

A Study of Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Who Are Experiencing Recurrent and/or Serious Infections

NCT06056297
View directory record Official record
Conditions
Neutropenia
Interventions
Mavorixafor, Placebo
Drug
Lead sponsor
X4 Pharmaceuticals
Industry
Eligibility
12 Years and older
Enrollment
176 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
22
States / cities
Phoenix, Arizona • Irvine, California • La Jolla, California + 19 more
Source: ClinicalTrials.gov public record
Updated Jun 4, 2026 · Synced Jul 23, 2026, 10:18 AM EDT
Active, not recruiting Phase 1 Interventional Accepts healthy volunteers

A Study to Learn How Different Amounts of the Study Medicine Called PF-08049820 Are Tolerated and Act in the Body in Healthy Adults

NCT06686797
View directory record Official record
Conditions
Healthy
Interventions
PF-08049820, Placebo
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 65 Years
Enrollment
71 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2026
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Feb 2, 2026 · Synced Jul 23, 2026, 10:18 AM EDT
Completed Not applicable Interventional Results available

Minocycline in the Treatment of Angelman Syndrome

NCT01531582
View directory record Official record
Conditions
Angelman Syndrome
Interventions
minocycline
Drug
Lead sponsor
University of South Florida
Other
Eligibility
4 Years to 12 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2015
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Apr 17, 2023 · Synced Jul 23, 2026, 10:18 AM EDT
Terminated Phase 1Phase 2 Interventional

A Multiple Ascending Dose Study of ACN00177 (Pegtarviliase) in Subjects With CBS Deficiency

NCT05154890
View directory record Official record
Conditions
Homocystinuria Due to Cystathionine Beta-Synthase Deficiency
Interventions
Pegtarviliase IV, Pegtarviliase SC
Drug
Lead sponsor
Aeglea Biotherapeutics
Industry
Eligibility
12 Years and older
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Jul 26, 2023 · Synced Jul 23, 2026, 10:18 AM EDT
Recruiting No phase listed Observational

Molecular Genetics of Heterotaxy and Related Congenital Heart Defects

NCT02432079
View directory record Official record
Conditions
Heterotaxy Syndrome, Congenital Heart Defects
Interventions
Not listed
Lead sponsor
Indiana University
Other
Eligibility
Not listed
Enrollment
2,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2030
U.S. locations
1
States / cities
Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated Jun 21, 2026 · Synced Jul 23, 2026, 10:18 AM EDT
Recruiting No phase listed Observational

Clinical Registry Investigating Bardet-Biedl Syndrome

NCT02329210
View directory record Official record
Conditions
Bardet-Biedl Syndrome
Interventions
Not listed
Lead sponsor
Marshfield Clinic Research Foundation
Other
Eligibility
Not listed
Enrollment
1,200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2030
U.S. locations
1
States / cities
Marshfield, Wisconsin
Source: ClinicalTrials.gov public record
Updated Jul 18, 2022 · Synced Jul 23, 2026, 10:18 AM EDT
Completed Phase 1 Interventional

Motexafin Gadolinium and Doxorubicin in Treating Patients With Advanced Cancer

NCT00036790
View directory record Official record
Conditions
Breast Cancer, Chronic Myeloproliferative Disorders, Colorectal Cancer, Head and Neck Cancer, Leukemia, Lung Cancer, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic/Myeloproliferative Diseases, Prostate Cancer, Small Intestine Cancer, Unspecified Adult Solid Tumor, Protocol Specific
Interventions
doxorubicin hydrochloride, motexafin gadolinium
Drug
Lead sponsor
University of Wisconsin, Madison
Other
Eligibility
18 Years to 120 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2002
U.S. locations
2
States / cities
Pittsburgh, Pennsylvania • Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Dec 16, 2019 · Synced Jul 23, 2026, 10:18 AM EDT
Terminated Early Phase 1 Interventional

19F MRI of Ventilation in Subjects With Cystic Fibrosis Undergoing Treatment for Pulmonary Exacerbation

NCT03497117
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Lung Clearance Index, MRI with PFP
Device · Drug
Lead sponsor
University of North Carolina, Chapel Hill
Other
Eligibility
18 Years to 99 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
1
States / cities
Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Oct 8, 2020 · Synced Jul 23, 2026, 10:18 AM EDT
Terminated Phase 2 Interventional Results available

Safety, Efficacy and Pharmacokinetics of an Oral Iron Chelator Given for a Year to Pediatric Patients With Iron Overload

NCT01363908
View directory record Official record
Conditions
Transfusional Iron Overload, Beta-Thalassemia
Interventions
SPD602
Drug
Lead sponsor
Shire
Industry
Eligibility
6 Years to 17 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
2
States / cities
Boston, Massachusetts • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 13, 2021 · Synced Jul 23, 2026, 10:18 AM EDT
Completed Phase 3 Interventional Results available

An Open-label Extension Trial of Cannabidiol (GWP42003-P, CBD) for Seizures in Tuberous Sclerosis Complex (GWPCARE6)

NCT02544750
View directory record Official record
Conditions
Tuberous Sclerosis Complex, Seizures
Interventions
GWP42003-P
Drug
Lead sponsor
Jazz Pharmaceuticals
Industry
Eligibility
1 Year to 65 Years
Enrollment
199 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2021
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jul 13, 2022 · Synced Jul 23, 2026, 10:18 AM EDT
Completed Phase 1Phase 2 Interventional

A Phase 1-2 Study of PRT-201 Administered in Chronic Kidney Disease (CKD) Patients

NCT00679991
View directory record Official record
Conditions
Chronic Kidney Disease
Interventions
PRT-201
Drug
Lead sponsor
Proteon Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
66 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2011
U.S. locations
11
States / cities
Indianapolis, Indiana • Iowa City, Iowa • Portland, Maine + 8 more
Source: ClinicalTrials.gov public record
Updated Apr 29, 2015 · Synced Jul 23, 2026, 10:18 AM EDT
Terminated No phase listed Observational Accepts healthy volunteers

Studies of Disorders in Antibody Production and Related Primary Immunodeficiency States

NCT00266513
View directory record Official record
Conditions
Hyper-IgM Syndrome, Ectodermal Dysplasia
Interventions
Not listed
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
Not listed
Enrollment
119 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2005 – 2013
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 5, 2017 · Synced Jul 23, 2026, 10:18 AM EDT
Completed No phase listed Observational

Pulmonary Hypertension in Trisomy 21 Patients

NCT00478296
View directory record Official record
Conditions
Congenital Disorders
Interventions
Not listed
Lead sponsor
Children's Healthcare of Atlanta
Other
Eligibility
Up to 8 Days
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2008
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Mar 15, 2012 · Synced Jul 23, 2026, 10:18 AM EDT
Recruiting No phase listed Interventional

3'-Deoxy-3'-[18F] Fluorothymidine PET Imaging in Patients With Cancer

NCT00935090
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, Unspecified Adult Solid Tumor, Protocol Specific
Interventions
3'-deoxy-3'-[18F]fluorothymidine
Device
Lead sponsor
Barbara Ann Karmanos Cancer Institute
Other
Eligibility
18 Years to 120 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2028
U.S. locations
1
States / cities
Detroit, Michigan
Source: ClinicalTrials.gov public record
Updated Jul 7, 2025 · Synced Jul 23, 2026, 10:18 AM EDT
Terminated Phase 2 Interventional

Multiple-Dose Pharmacokinetics and Potential for Adrenal Suppression Following Treatment With Halobetasol Spray in Patients With Atopic Dermatitis

NCT03245385
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
Treatment with Halobetasol propionate 0.05% topical spray
Drug
Lead sponsor
Sun Pharmaceutical Industries Limited
Industry
Eligibility
12 Years to 70 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2018
U.S. locations
13
States / cities
North Hollywood, California • Pasadena, California • Hialeah, Florida + 6 more
Source: ClinicalTrials.gov public record
Updated Sep 24, 2019 · Synced Jul 23, 2026, 10:18 AM EDT
Completed Not applicable Interventional

A Study to Evaluate the Effects of a New Formula on the Growth, Safety and Tolerance of Infants With Growth Failure

NCT03563391
View directory record Official record
Conditions
Growth Failure, Congenital Heart Disease
Interventions
New Infant Formula
Other
Lead sponsor
Nutricia North America
Industry
Eligibility
1 Month to 8 Months
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
5
States / cities
Jacksonville, Florida • Atlanta, Georgia • Greenville, North Carolina + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 7, 2020 · Synced Jul 23, 2026, 10:18 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

An Online Health Tool About Down Syndrome: Down Syndrome Clinic to You

NCT04227197
View directory record Official record
Conditions
Down Syndrome
Interventions
Down Syndrome Clinic to You (DSC2U)
Other
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
1 Year and older
Enrollment
281 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2019
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 13, 2022 · Synced Jul 23, 2026, 10:18 AM EDT