Independent directory Public ClinicalTrials.gov records United States
Clinical Trials Finder
Objective public record navigation
Search Results

Search by objective public record fields.

Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results are retrieved from ClinicalTrials.gov and synchronized into the directory. Search pages remain noindex by default.

Clear filters
ClinicalTrials.gov public records Last synced Sep 22, 2026, 5:46 PM EDT

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

Showing 1–24 of 7,885 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Genetic Diseases, Inborn Clear all
Completed Phase 1 Interventional

PK Study of N91115 in Cystic Fibrosis Patients

NCT02227888
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
N91115
Drug
Lead sponsor
Nivalis Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
3
States / cities
Birmingham, Alabama • Aurora, Colorado • Denver, Colorado
Source: ClinicalTrials.gov public record
Updated Nov 6, 2016 · Synced Sep 22, 2026, 5:46 PM EDT
Completed No phase listed Observational

Needs Assessment for Individuals and Families Affected by Dyskeratosis Congenita (DC) and Related Telomere Biology Disorders (TBD)

NCT04959188
View directory record Official record
Conditions
Dyskeratosis Congenita, Telomere Biology Disorders
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years to 100 Years
Enrollment
53 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 10, 2024 · Synced Sep 22, 2026, 5:46 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Study to Evaluate ASN008 Topical Gel (TG)

NCT03798561
View directory record Official record
Conditions
Dermatitis, Atopic, Pruritus, Dermatitis Eczema
Interventions
ASN008 TG, Placebo TG
Drug
Lead sponsor
Asana BioSciences
Industry
Eligibility
18 Years to 65 Years
Enrollment
24 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2020
U.S. locations
4
States / cities
Cortland, New York • High Point, North Carolina • San Antonio, Texas + 1 more
Source: ClinicalTrials.gov public record
Updated May 8, 2023 · Synced Sep 22, 2026, 5:46 PM EDT
Completed Phase 2 Interventional

A Study to Investigate the Pharmacokinetics (PK) and Safety and to Provide Proof of Mechanism of Alogabat in Children and Adolescents Aged 5-17 Years With Angelman Syndrome (AS) With Deletion Genotype.

NCT05630066
View directory record Official record
Conditions
Angelman Syndrome
Interventions
Alogabat
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
5 Years to 17 Years
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
6
States / cities
Chicago, Illinois • Boston, Massachusetts • New York, New York + 3 more
Source: ClinicalTrials.gov public record
Updated Dec 3, 2025 · Synced Sep 22, 2026, 5:46 PM EDT
Completed Not applicable Interventional

Caspofungin Acetate in Treating Children With Fever and Neutropenia

NCT00020527
View directory record Official record
Conditions
Fever, Sweats, and Hot Flashes, Infection, Kidney Cancer, Leukemia, Lymphoma, Neuroblastoma, Neutropenia, Sarcoma
Interventions
caspofungin acetate
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
2 Years to 17 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2003
U.S. locations
5
States / cities
Washington D.C., District of Columbia • Baltimore, Maryland • Bethesda, Maryland + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 18, 2013 · Synced Sep 22, 2026, 5:46 PM EDT
Completed Phase 3 Interventional Results available

A Study to Assess the Efficacy and Safety of Ruxolitinib Cream in Children With Atopic Dermatitis (TRuE-AD3)

NCT04921969
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
Ruxolitinib, Vehicle Cream
Drug
Lead sponsor
Incyte Corporation
Industry
Eligibility
2 Years to 11 Years
Enrollment
330 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
64
States / cities
Birmingham, Alabama • Hoover, Alabama • Gilbert, Arizona + 49 more
Source: ClinicalTrials.gov public record
Updated Mar 27, 2025 · Synced Sep 22, 2026, 5:46 PM EDT
Terminated No phase listed Observational

Natural History of Autosomal Dominant Optic Atrophy (ADOA), Caused by OPA1 Mutation

NCT06140329
View directory record Official record
Conditions
Autosomal Dominant Optic Atrophy, Optic Atrophy, Autosomal Dominant, Optic Atrophies, Hereditary, Kjer Optic Atrophy
Interventions
Not listed
Lead sponsor
PYC Therapeutics
Industry
Eligibility
8 Years and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
2
States / cities
Miami, Florida • Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Mar 13, 2025 · Synced Sep 22, 2026, 5:46 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Comparative Bioavailability Study of Lisinopril Tablets, 40 mg - Effect of Food Study

NCT01380431
View directory record Official record
Conditions
To Determine Bioequivalence Under Fed Conditions
Interventions
Lisinopril
Drug
Lead sponsor
Par Pharmaceutical, Inc.
Industry
Eligibility
18 Years to 50 Years · Male only
Enrollment
18 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1999
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 26, 2011 · Synced Sep 22, 2026, 5:46 PM EDT
Recruiting Phase 2 Interventional

Adenovirus-specific Cytotoxic T-lymphocytes for Refractory Adenovirus Infection

NCT03266627
View directory record Official record
Conditions
Adenovirus, Primary Immune Deficiency Disorder
Interventions
cytotoxic t-lymphocytes
Drug
Lead sponsor
New York Medical College
Other
Eligibility
1 Month to 79 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2027
U.S. locations
10
States / cities
Los Angeles, California • San Francisco, California • Aurora, Colorado + 6 more
Source: ClinicalTrials.gov public record
Updated Aug 7, 2025 · Synced Sep 22, 2026, 5:46 PM EDT
Completed Phase 4 Interventional Results available

Long Term Safety of Tobramycin Inhalation Powder in Patients With Cystic Fibrosis

NCT01519661
View directory record Official record
Conditions
Pulmonary Infections, Pseudomonas Aeruginosa in Cystic Fibrosis
Interventions
TBM100
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
6 Years and older
Enrollment
157 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2014
U.S. locations
20
States / cities
Little Rock, Arkansas • Denver, Colorado • Jacksonville, Florida + 16 more
Source: ClinicalTrials.gov public record
Updated Feb 9, 2015 · Synced Sep 22, 2026, 5:46 PM EDT
Enrolling by invitation Phase 2 Interventional

Open-Label, Long-Term, Extension Study of Infigratinib in Children With Hypochondroplasia

NCT07393373
View directory record Official record
Conditions
Hypochondroplasia
Interventions
Infigratinib
Drug
Lead sponsor
QED Therapeutics, a BridgeBio company
Industry
Eligibility
3 Years to 18 Years
Enrollment
135 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2036
U.S. locations
8
States / cities
Oakland, California • Aurora, Colorado • Washington D.C., District of Columbia + 5 more
Source: ClinicalTrials.gov public record
Updated Aug 25, 2026 · Synced Sep 22, 2026, 5:46 PM EDT
Completed Not applicable Interventional

Investigating Distress Tolerance in Adults With Tourette Syndrome

NCT05047705
View directory record Official record
Conditions
Tourette Syndrome, Tic Disorders, Tics
Interventions
Distress Tolerance Skills Training
Behavioral
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 6, 2024 · Synced Sep 22, 2026, 5:46 PM EDT
Active, not recruiting No phase listed Observational Accepts healthy volunteers

Pediatric Radio Frequency Coils Generic

NCT01633866
View directory record Official record
Conditions
Duchenne Muscular Dystrophy, Musculoskeletal Abnormalities
Interventions
Not listed
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
Not listed
Enrollment
75 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2031
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jun 11, 2026 · Synced Sep 22, 2026, 5:46 PM EDT
Active, not recruiting No phase listed Observational

Long - Term Follow Up of Sickle Cell Disease and Beta-thalassemia Subjects Previously Exposed to BIVV003 or ST-400.

NCT05145062
View directory record Official record
Conditions
Blood and Lymphatic Diseases
Interventions
BIVV003, ST-400
Drug
Lead sponsor
Sangamo Therapeutics
Industry
Eligibility
18 Years to 45 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2038
U.S. locations
7
States / cities
Oakland, California • Sacramento, California • Atlanta, Georgia + 3 more
Source: ClinicalTrials.gov public record
Updated Nov 11, 2025 · Synced Sep 22, 2026, 5:46 PM EDT
Terminated Phase 2 Interventional Results available

Continuation Study of Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Recifercept in Achondroplasia

NCT05116046
View directory record Official record
Conditions
Achondroplasia
Interventions
Recifercept
Biological
Lead sponsor
Pfizer
Industry
Eligibility
15 Months to 12 Years
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
6
States / cities
Irvine, California • Long Beach, California • Torrance, California + 2 more
Source: ClinicalTrials.gov public record
Updated Feb 7, 2024 · Synced Sep 22, 2026, 5:46 PM EDT
Completed Early Phase 1 Interventional

Ranibizumab for Neurofibromas Associated With Neurofibromatosis 1

NCT00657202
View directory record Official record
Conditions
Neurofibromatosis Type 1, Cutaneous Neurofibromas
Interventions
Ranibizumab
Drug
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years and older
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2013
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jul 6, 2016 · Synced Sep 22, 2026, 5:46 PM EDT
Completed No phase listed Observational

Antiproteinuric Agents and Fabry Disease

NCT00343577
View directory record Official record
Conditions
Fabry Disease, Proteinuria
Interventions
Not listed
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
14 Years to 95 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2006
U.S. locations
1
States / cities
Birmingham, Alabama
Source: ClinicalTrials.gov public record
Updated Nov 18, 2013 · Synced Sep 22, 2026, 5:46 PM EDT
Completed Phase 3 Interventional Results available

A Study to Investigate the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride) Oral Solution in Children and Adults With Epileptic Encephalopathy Including Dravet Syndrome and Lennox-Gastaut Syndrome

NCT03936777
View directory record Official record
Conditions
Dravet Syndrome, Lennox Gastaut Syndrome, Epileptic Encephalopathy
Interventions
ZX008 (Fenfluramine Hydrochloride)
Drug
Lead sponsor
Zogenix, Inc.
Industry
Eligibility
2 Years and older
Enrollment
412 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
26
States / cities
Tucson, Arizona • Los Angeles, California • San Diego, California + 23 more
Source: ClinicalTrials.gov public record
Updated Nov 16, 2025 · Synced Sep 22, 2026, 5:46 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Pilot Phase I/II Study of Amino Acid Leucine in Treatment of Patients With Transfusion-Dependent Diamond Blackfan Anemia

NCT01362595
View directory record Official record
Conditions
Diamond Blackfan Anemia, Blackfan Diamond Syndrome, DBA, Congenital Hypoplastic Anemia, Pure Red Cell Aplasia
Interventions
leucine
Drug
Lead sponsor
Northwell Health
Other
Eligibility
2 Years and older
Enrollment
55 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2020
U.S. locations
11
States / cities
Phoenix, Arizona • Palo Alto, California • Indianapolis, Indiana + 8 more
Source: ClinicalTrials.gov public record
Updated Dec 1, 2022 · Synced Sep 22, 2026, 5:46 PM EDT
Completed Not applicable Interventional

Melatonin for Huntington's Disease (HD) Gene Carriers With HD Related Sleep Disturbance - a Pilot Study

NCT04421339
View directory record Official record
Conditions
Huntington Disease
Interventions
Melatonin, Placebo
Dietary Supplement · Other
Lead sponsor
The University of Texas Health Science Center, Houston
Other
Eligibility
18 Years to 75 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated May 14, 2023 · Synced Sep 22, 2026, 5:46 PM EDT
Completed Not applicable Interventional Results available

MN4000 for Treatment of CF and MND Patients in the Home Setting

NCT03797677
View directory record Official record
Conditions
Cystic Fibrosis, Motor Neuron Disease, Airway Clearance Impairment
Interventions
MN 4000
Device
Lead sponsor
Hill-Rom
Industry
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Aug 6, 2024 · Synced Sep 22, 2026, 5:46 PM EDT
Recruiting Phase 3 Interventional

A Study Evaluating the Safety and Pharmacokinetics of QTORIN Rapamycin 3.9% Anhydrous Gel in the Treatment of Adults With Pachyonychia Congenita

NCT05643872
View directory record Official record
Conditions
Pachyonychia Congenita
Interventions
PTX-022
Drug
Lead sponsor
Palvella Therapeutics, Inc.
Industry
Eligibility
16 Years and older
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
1
States / cities
New Brighton, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 8, 2022 · Synced Sep 22, 2026, 5:46 PM EDT
Completed Phase 3 Interventional Results available

Study Investigating a Delayed-Release Pancrelipase in Patients With Exocrine Pancreatic Insufficiency Due to Cystic Fibrosis

NCT00510484
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Pancrelipase Delayed Release, Placebo Comparator
Drug
Lead sponsor
Solvay Pharmaceuticals
Industry
Eligibility
12 Years and older
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2008
U.S. locations
19
States / cities
Long Beach, California • Los Angeles, California • San Francisco, California + 16 more
Source: ClinicalTrials.gov public record
Updated Jun 2, 2010 · Synced Sep 22, 2026, 5:46 PM EDT
Completed Phase 3 Interventional Results available

Mineralocorticoid Antagonism and Endothelial Dysfunction in Autosomal Dominant Polycystic Kidney Disease (ADPKD)

NCT01853553
View directory record Official record
Conditions
ADPKD
Interventions
Spironolactone, Sugar pill
Drug
Lead sponsor
University of Colorado, Denver
Other
Eligibility
20 Years to 55 Years
Enrollment
61 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated Aug 19, 2019 · Synced Sep 22, 2026, 5:46 PM EDT