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ClinicalTrials.gov public records Last synced Oct 22, 2026, 10:37 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 793–816 of 8,007 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Genetics/Birth Defects Clear all
Not yet recruiting Phase 2 Interventional

Verapamil Effect in Cystic Fibrosis-related Dysglycemia

NCT07688070
View directory record Official record
Conditions
Cystic Fibrosis Related Diabetes, Cystic Fibrosis (CF), Abnormal Glucose Tolerance
Interventions
Verapamil Hydrochloride
Drug
Lead sponsor
Rhode Island Hospital
Other
Eligibility
14 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
2
States / cities
Boston, Massachusetts • Providence, Rhode Island
Source: ClinicalTrials.gov public record
Updated Jul 9, 2026 · Synced Oct 22, 2026, 10:37 PM EDT
Completed Phase 3 Interventional Results available

Efficacy and Safety of GWP42003-P for Seizures Associated With Lennox-Gastaut Syndrome in Children and Adults

NCT02224560
View directory record Official record
Conditions
Epilepsy, Lennox Gastaut Syndrome
Interventions
GWP42003-P, Placebo control
Drug
Lead sponsor
Jazz Pharmaceuticals
Industry
Eligibility
2 Years to 55 Years
Enrollment
225 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
20
States / cities
Birmingham, Alabama • Phoenix, Arizona • Los Angeles, California + 16 more
Source: ClinicalTrials.gov public record
Updated Sep 28, 2022 · Synced Oct 22, 2026, 10:37 PM EDT
Terminated Phase 2 Interventional Results available

Efficacy, Safety, and Pharmacokinetic Profiles of REGN3500 Administered to Adult Patients With Moderate-to-Severe Atopic Dermatitis

NCT03738423
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
REGN3500, REGN3500-Matching Placebo
Drug
Lead sponsor
Regeneron Pharmaceuticals
Industry
Eligibility
18 Years to 75 Years
Enrollment
129 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
31
States / cities
Birmingham, Alabama • Phoenix, Arizona • Scottsdale, Arizona + 24 more
Source: ClinicalTrials.gov public record
Updated Jun 10, 2022 · Synced Oct 22, 2026, 10:37 PM EDT
Suspended Phase 1Phase 2 Interventional

Study of ASN-002 to Treat Basal Cell Carcinomas (BCCs) in Individuals With Basal Cell Nevus Syndrome (BCNS)

NCT03208296
View directory record Official record
Conditions
Basal Cell Carcinoma in Basal Cell Nevus Syndrome
Interventions
ASN-002
Biological
Lead sponsor
Ascend Biopharmaceuticals Ltd
Industry
Eligibility
18 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2025
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Feb 6, 2023 · Synced Oct 22, 2026, 10:37 PM EDT
Completed Phase 3 Interventional Results available

Efficacy and Safety Study of DX-88 to Treat Acute Attacks of Hereditary Angioedema (HAE)

NCT00262080
View directory record Official record
Conditions
Hereditary Angioedema (HAE)
Interventions
ecallantide, Phosphate Buffer Saline (PBS),
Drug
Lead sponsor
Shire
Industry
Eligibility
10 Years and older
Enrollment
91 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2007
U.S. locations
1
States / cities
Wheaton, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 11, 2021 · Synced Oct 22, 2026, 10:37 PM EDT
Recruiting No phase listed Observational

The Myelin Disorders Biorepository Project

NCT03047369
View directory record Official record
Conditions
Leukodystrophy, White Matter Disease, Leukoencephalopathies, 4H Syndrome, Adrenoleukodystrophy, AMN, ALD, ALD Gene Mutation, ALD (Adrenoleukodystrophy), X-linked Adrenoleukodystrophy, X-ALD, Adrenomyeloneuropathy, Aicardi Goutieres Syndrome, AGS, Alexander Disease, Alexanders Leukodystrophy, AxD, ADLD, Canavan Disease, CTX, Cerebrotendinous Xanthomatoses, Krabbe Disease, GALC Deficiency, Globoid Leukodystrophy, TUBB4A-Related Leukodystrophy, H-ABC - Hypomyelination, Atrophy of Basal Ganglia and Cerebellum, HBSL, HBSL - Hypomyelination, Brain Stem, Spinal Cord, Leg Spasticity, LBSL, Leukoencephalopathy With Brain Stem and Spinal Cord Involvement and High Lactate Syndrome (Disorder), Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation, ALSP, CSF1R Gene Mutation, HCC - Hypomyelination and Congenital Cataract, MLC1, Megalencephalic Leukoencephalopathy With Subcortical Cysts, MLD, Metachromatic Leukodystrophy, PMD, Pelizaeus-Merzbacher Disease, PLP1 Null Syndrome, PLP1 Gene Duplication | Blood or Tissue | Mutations, Pelizaeus Merzbacher Like Disease, Peroxisomal Biogenesis Disorder, Zellweger Syndrome, Refsum Disease, Salla Disease, Sialic Storage Disease, Sjögren, Sjogren-Larsson Syndrome, Van Der Knapp Disease, Vanishing White Matter Disease, Charcot-Marie-Tooth, CMT, Mct8 (Slc16A2)-Specific Thyroid Hormone Cell Transporter Deficiency, Allan-Herndon-Dudley Syndrome, Cadasil, Cockayne Syndrome, Multiple Sulfatase Deficiency, Gangliosidoses, GM2 Gangliosidosis, BPAN, Labrune Syndrome, LCC, Mucopolysaccharidoses, TBCK-Related Intellectual Disability Syndrome
Interventions
Not listed
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
Not listed
Enrollment
12,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2030
U.S. locations
23
States / cities
Los Angeles, California • Orange, California • Palo Alto, California + 18 more
Source: ClinicalTrials.gov public record
Updated Oct 23, 2025 · Synced Oct 22, 2026, 10:37 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 8, 2017 · Synced Oct 22, 2026, 10:37 PM EDT
Recruiting Phase 3 Interventional

A 24-Month Trial of NPI-001 for the Preservation of Photoreceptors in Retinitis Pigmentosa Associated With Usher Syndrome

NCT07710196
View directory record Official record
Conditions
Retinitis Pigmentosa (RP), Usher Syndrome
Interventions
Placebo, NPI-001
Drug
Lead sponsor
Nacuity Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Oct 13, 2026 · Synced Oct 22, 2026, 10:37 PM EDT
Not listed Phase 1 Interventional

Phase I Study of Amifostine in Patients With Bone Marrow Failure Related to Fanconi's Anemia

NCT00006127
View directory record Official record
Conditions
Fanconi's Anemia
Interventions
amifostine
Drug
Lead sponsor
Dana-Farber Cancer Institute
Other
Eligibility
2 Years and older
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 22, 2026, 10:37 PM EDT
Completed Phase 2 Interventional Results available

Study to Evaluate the Safety and Efficacy of SPR001 in Subjects With Classic Congenital Adrenal Hyperplasia

NCT03687242
View directory record Official record
Conditions
Congenital Adrenal Hyperplasia, CAH - Congenital Adrenal Hyperplasia, CAH - 21-Hydroxylase Deficiency
Interventions
SPR001
Drug
Lead sponsor
Spruce Biosciences
Industry
Eligibility
18 Years and older
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
8
States / cities
Orange, California • San Diego, California • Atlanta, Georgia + 5 more
Source: ClinicalTrials.gov public record
Updated Apr 1, 2025 · Synced Oct 22, 2026, 10:37 PM EDT
Completed Phase 2 Interventional Results available

Treatment of Rett Syndrome With Recombinant Human IGF-1

NCT01777542
View directory record Official record
Conditions
Rett Syndrome
Interventions
Recombinant Human Insulin Growth Factor 1 (rhIGF-1), Placebo
Drug
Lead sponsor
Boston Children's Hospital
Other
Eligibility
2 Years to 10 Years · Female only
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 26, 2018 · Synced Oct 22, 2026, 10:37 PM EDT
Completed Phase 1Phase 2 Interventional Results available

An Open-label Extension Study of an Investigational Drug, Fitusiran, in Patients With Moderate or Severe Hemophilia A or B

NCT02554773
View directory record Official record
Conditions
Hemophilia A, Hemophilia B
Interventions
Fitusiran (SAR439774)
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years and older · Male only
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2023
U.S. locations
2
States / cities
Ann Arbor, Michigan • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 5, 2024 · Synced Oct 22, 2026, 10:37 PM EDT
Completed Phase 2 Interventional

Immunotherapy in Treating Patients Who Are Undergoing Bone Marrow or Peripheral Stem Cell Transplantation

NCT00002673
View directory record Official record
Conditions
Cancer
Interventions
infection prophylaxis and management
Procedure
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
12 Years to 60 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1995 – 2002
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Jun 17, 2010 · Synced Oct 22, 2026, 10:37 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Study of Total Energy Expenditure in Infants and Children With Moderate to Severe Cystic Fibrosis

NCT00006273
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Not listed
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years to 10 Years
Enrollment
60 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 1996
U.S. locations
1
States / cities
Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 22, 2026, 10:37 PM EDT
Completed No phase listed Observational

Variability of Ventricular Mass, Volume, & Ejection Fraction in Pediatric Cardiomyopathy Patients-Pediatric Heart Network

NCT00123071
View directory record Official record
Conditions
Cardiomyopathy, Dilated
Interventions
Not listed
Lead sponsor
Carelon Research
Other
Eligibility
Up to 22 Years
Enrollment
131 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2009
U.S. locations
7
States / cities
Boston, Massachusetts • St Louis, Missouri • New York, New York + 4 more
Source: ClinicalTrials.gov public record
Updated Jan 25, 2013 · Synced Oct 22, 2026, 10:37 PM EDT
Completed Phase 1Phase 2 Interventional

Tuvusertib (M1774) in Combination With Cemiplimab in Participants With Non-Squamous NSCLC (DDRiver NSCLC 322)

NCT05882734
View directory record Official record
Conditions
Non-Small Cell Lung Cancer
Interventions
M1774, Cemiplimab
Drug
Lead sponsor
EMD Serono Research & Development Institute, Inc.
Industry
Eligibility
18 Years and older
Enrollment
61 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
6
States / cities
Santa Monica, California • Pittsburgh, Pennsylvania • Knoxville, Tennessee + 2 more
Source: ClinicalTrials.gov public record
Updated Sep 14, 2026 · Synced Oct 22, 2026, 10:37 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Neurobiology and Treatment of Reading Disability in NF-1

NCT00624234
View directory record Official record
Conditions
Neurofibromatosis Type 1, Reading Disabilities
Interventions
Tutoring Program I, Tutoring Program II
Behavioral
Lead sponsor
Vanderbilt University
Other
Eligibility
8 Years to 17 Years
Enrollment
184 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2006 – 2017
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Jul 8, 2025 · Synced Oct 22, 2026, 10:37 PM EDT
Completed Phase 2 Interventional Results available

A Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)

NCT02760264
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Vamorolone 0.25 mg/kg/day, Vamorolone 0.75 mg/kg/day, Vamorolone 2.0 mg/kg/day, Vamorolone 6.0 mg/kg/day
Drug
Lead sponsor
ReveraGen BioPharma, Inc.
Industry
Eligibility
4 Years to 6 Years · Male only
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
6
States / cities
Davis, California • Gainesville, Florida • Orlando, Florida + 3 more
Source: ClinicalTrials.gov public record
Updated Jan 2, 2019 · Synced Oct 22, 2026, 10:37 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

Derazantinib Alone or in Combination With Paclitaxel, Ramucirumab or Atezolizumab in Gastric Adenocarcinoma

NCT04604132
View directory record Official record
Conditions
Gastric Adenocarcinoma
Interventions
Derazantinib, Derazantinib-paclitaxel-ramucirumab combination
Drug
Lead sponsor
Basilea Pharmaceutica
Industry
Eligibility
18 Years and older
Enrollment
47 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
2
States / cities
Orlando, Florida • New York, New York
Source: ClinicalTrials.gov public record
Updated Apr 4, 2024 · Synced Oct 22, 2026, 10:37 PM EDT
No Longer Available No phase listed Expanded access

A Second Intermediate-Size Expanded Access Protocol (EAP) for Pridopidine in People With Amyotrophic Lateral Sclerosis (Pridopidine EAP 2)

NCT06069934
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis
Interventions
Pridopidine
Drug
Lead sponsor
Prilenia
Industry
Eligibility
18 Years and older
U.S. locations
31
States / cities
Birmingham, Alabama • Phoenix, Arizona • La Jolla, California + 26 more
Source: ClinicalTrials.gov public record
Updated May 18, 2025 · Synced Oct 22, 2026, 10:37 PM EDT
Terminated Phase 4 Interventional

Imipenem/Cilastatin/Relebactam Pharmacokinetics, Safety, and Outcomes in Adults and Adolescents With Cystic Fibrosis

NCT05561764
View directory record Official record
Conditions
Cystic Fibrosis, Pneumonia, Bacterial
Interventions
Imipenem/Cilastatin/Relebactam
Drug
Lead sponsor
Hartford Hospital
Other
Eligibility
12 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
3
States / cities
Los Angeles, California • Hartford, Connecticut • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 17, 2024 · Synced Oct 22, 2026, 10:37 PM EDT
Completed No phase listed Observational

Consortium for Radiologic Imaging Studies of Polycystic Kidney Disease

NCT01039987
View directory record Official record
Conditions
Autosomal Dominant Polycystic Kidney Disease
Interventions
Not listed
Lead sponsor
University of Pittsburgh
Other
Eligibility
15 Years to 45 Years
Enrollment
211 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2011
U.S. locations
5
States / cities
Birmingham, Alabama • Atlanta, Georgia • Kansas City, Kansas + 2 more
Source: ClinicalTrials.gov public record
Updated Aug 17, 2017 · Synced Oct 22, 2026, 10:37 PM EDT
Terminated Phase 2 Interventional Results available

A Phase II Study of Pertuzumab and Erlotinib for Metastatic or Unresectable Neuroendocrine Tumors

NCT00947167
View directory record Official record
Conditions
Neuroendocrine Tumors, Carcinoid Tumors, Adrenal Gland Tumors, Neuroblastoma, Pancreatic Neuroendocrine Tumors, Multiple Endocrine Neoplasia
Interventions
pertuzumab, erlotinib
Drug
Lead sponsor
Pamela L. Kunz
Other
Eligibility
Not listed
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2010
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated Mar 3, 2017 · Synced Oct 22, 2026, 10:37 PM EDT
Recruiting Not applicable Interventional

Utility of PCD Diagnostics to Improve Clinical Care

NCT03704207
View directory record Official record
Conditions
Primary Ciliary Dyskinesia
Interventions
nNO testing
Other
Lead sponsor
Vanderbilt University Medical Center
Other
Eligibility
1 Year and older
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2028
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Jul 6, 2026 · Synced Oct 22, 2026, 10:37 PM EDT