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 come from ClinicalTrials.gov. When the live registry is unavailable, a clearly labeled stored copy may be shown.

Clear filters

These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.

ClinicalTrials.gov public records Last synced Oct 20, 2026, 5:52 PM EDT

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

Showing 49–72 of 95 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Autosomal Recessive Disorder Clear all
Recruiting No phase listed Observational

Hereditary Spastic Paraplegia Genomic Sequencing Initiative (HSPseq)

NCT05354622
View directory record Official record
Conditions
Hereditary Spastic Paraplegia, Neurodegenerative Diseases, Pediatric Disorder, Spasticity, Muscle, Motor Neuron Disease, Movement Disorders
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
1 Month to 30 Years
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 18, 2026 · Synced Oct 20, 2026, 5:52 PM EDT
Withdrawn Phase 2 Interventional

Study of CAD-1883 for Spinocerebellar Ataxia

NCT04301284
View directory record Official record
Conditions
Spinocerebellar Ataxias, Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 2, Spinocerebellar Ataxia Type 3, Spinocerebellar Ataxia Type 6, Spinocerebellar Ataxia Type 7, Spinocerebellar Ataxia Type 8, Spinocerebellar Ataxia Type 10, Spinocerebellar Ataxia Type 17, ARCA1 - Autosomal Recessive Cerebellar Ataxia Type 1
Interventions
CAD-1883, Placebos
Drug
Lead sponsor
Cadent Therapeutics
Industry
Eligibility
18 Years to 75 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
6
States / cities
Long Beach, California • Los Angeles, California • Aurora, Colorado + 3 more
Source: ClinicalTrials.gov public record
Updated Apr 8, 2021 · Synced Oct 20, 2026, 5:52 PM EDT
Completed Not applicable Interventional

High Flow Nasal Cannula in Comparison With Nasal Continuous Positive Airway Pressure in the Management of Respiratory Distress Syndrome

NCT02587832
View directory record Official record
Conditions
Respiratory Distress Syndrome
Interventions
HHHFNC, NCPAP
Device
Lead sponsor
Sidra Medicine
Other
Eligibility
Not listed
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2012
U.S. locations
1
States / cities
Akron, Ohio
Source: ClinicalTrials.gov public record
Updated Oct 27, 2015 · Synced Oct 20, 2026, 5:52 PM EDT
Enrolling by invitation No phase listed Observational

SLC13A5 Deficiency Natural History Study - United States Only

NCT06144957
View directory record Official record
Conditions
Citrate Transporter Deficiency, Epilepsy, Rare Diseases, Movement Disorders, Genetic Disorder, SLC13A5 Deficiency, EIEE25, Kohlschutter-Tonz Syndrome (Non-ROGDI), Citrate Transporter Disorder, DEE25
Interventions
Not listed
Lead sponsor
TESS Research Foundation
Other
Eligibility
Not listed
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
3
States / cities
Palo Alto, California • Providence, Rhode Island • Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Aug 5, 2025 · Synced Oct 20, 2026, 5:52 PM EDT
Completed Phase 1 Interventional

Bone Marrow Transplant With Abatacept for Non-Malignant Diseases

NCT01917708
View directory record Official record
Conditions
Hurler Syndrome, Fanconi Anemia, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, Severe Congenital Neutropenia, Leukocyte Adhesion Deficiency, Shwachman-Diamond Syndrome, Diamond-Blackfan Anemia, Dyskeratosis-congenita, Chediak-Higashi Syndrome, Severe Aplastic Anemia, Thalassemia Major, Hemophagocytic Lymphohistiocytosis, Sickle Cell Disease
Interventions
Abatacept
Drug
Lead sponsor
Emory University
Other
Eligibility
Up to 21 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Dec 26, 2019 · Synced Oct 20, 2026, 5:52 PM EDT
Completed Phase 1Phase 2 Interventional

Ustekinumab (Anti-IL-12/23p40 Monoclonal Antibody) in Patients With Leukocyte Adhesion Deficiency Type 1 (LAD1) Who Have Inflammatory Pathology

NCT03366142
View directory record Official record
Conditions
LAD1
Interventions
Ustekinumab
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
12 Years to 65 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2024
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 22, 2024 · Synced Oct 20, 2026, 5:52 PM EDT
Completed Phase 2Phase 3 Interventional Results available

Stem Cell Transplant for Hemoglobinopathy

NCT00176852
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia, Severe Congenital Neutropenia, Diamond-Blackfan Anemia, Shwachman-Diamond Syndrome
Interventions
Busulfan, Fludarabine, ATG, TLI, Busulfan, Cyclophosphamide, ATG, GCSF, Campath, Fludarabine, Cyclophosphamide, Total Body Irradiation, Stem cell infusion
Drug · Radiation · Procedure
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 50 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2020
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Feb 27, 2020 · Synced Oct 20, 2026, 5:52 PM EDT
Terminated Not applicable Interventional Results available

Stem Cell Transplantation for Children Affected With Osteopetrosis

NCT00145587
View directory record Official record
Conditions
Osteopetrosis
Interventions
Stem Cell Transplantation, Miltenyi Biotec CliniMACS, Systemic chemotherapy and antibodies
Procedure · Device · Drug
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Not listed
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2009
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated May 30, 2017 · Synced Oct 20, 2026, 5:52 PM EDT
Completed Phase 2 Interventional

Do Endotypes Predict Response and Sequelae in OSA Patients

NCT04875364
View directory record Official record
Conditions
Obstructive Sleep Apnea, Sleep Apnea, Sleep Apnea, Obstructive, Sleep, Sleep Disorder
Interventions
Eszopiclone, Supplemental Oxygen, Continuous Positive Airway Pressure (CPAP)
Drug · Other · Device
Lead sponsor
University of California, San Diego
Other
Eligibility
21 Years to 65 Years
Enrollment
233 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
1
States / cities
La Jolla, California
Source: ClinicalTrials.gov public record
Updated Jun 18, 2026 · Synced Oct 20, 2026, 5:52 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

A Study of REGN5093 in Adult Patients With Mesenchymal Epithelial Transition Factor (MET)-Altered Advanced Non-Small Cell Lung Cancer

NCT04077099
View directory record Official record
Conditions
NSCLC
Interventions
REGN5093
Drug
Lead sponsor
Regeneron Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
95 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
8
States / cities
Orange, California • Washington D.C., District of Columbia • Tampa, Florida + 4 more
Source: ClinicalTrials.gov public record
Updated Sep 16, 2026 · Synced Oct 20, 2026, 5:52 PM EDT
Completed Not applicable Interventional

A Trial of Cognitive Behavioral Therapy in Familial Dysautonomia

NCT03013777
View directory record Official record
Conditions
Dysthymia, Anxiety Disorders, Familial Dysautonomia, Paroxysmal Hypertension, Autosomal Recessive Disease
Interventions
8-week CBT Program
Behavioral
Lead sponsor
NYU Langone Health
Other
Eligibility
18 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jan 29, 2019 · Synced Oct 20, 2026, 5:52 PM EDT
Recruiting Phase 3 Interventional

Study of ALK-001 on the Progression of Stargardt Disease

NCT07419334
View directory record Official record
Conditions
Stargardt Disease
Interventions
ALK-001, Placebo
Drug
Lead sponsor
Alkeus Pharmaceuticals, Inc.
Industry
Eligibility
8 Years to 45 Years
Enrollment
230 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
14
States / cities
Phoenix, Arizona • Aurora, Colorado • Gainesville, Florida + 11 more
Source: ClinicalTrials.gov public record
Updated Sep 18, 2026 · Synced Oct 20, 2026, 5:52 PM EDT
Recruiting No phase listed Observational

An Observational Study in Subjects to Follow the Progression of Stargardt Disease Type 1 (STGD1) Caused by Bi-Allelic Autosomal Recessive Mutations in the ABCA4 Gene

NCT06435000
View directory record Official record
Conditions
Stargardt, Stargardt's Disease, Stargardt Disease, STGD1
Interventions
Not listed
Lead sponsor
Splice Bio
Industry
Eligibility
12 Years to 65 Years
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
14
States / cities
San Diego, California • Aurora, Colorado • Gainesville, Florida + 10 more
Source: ClinicalTrials.gov public record
Updated Sep 19, 2025 · Synced Oct 20, 2026, 5:52 PM EDT
Recruiting No phase listed Observational

ARPKD Database Study

NCT01401998
View directory record Official record
Conditions
Hepato/Renal Fibrocystic Disease, Autosomal Recessive Polycystic Kidney Disease, Joubert Syndrome, Bardet Biedl Syndrome, Meckel-Gruber Syndrome, Congenital Hepatic Fibrosis, Caroli Syndrome, Oro-Facial-Digital Syndrome Type I, Nephronophthisis, Glomerulocystic Kidney Disease
Interventions
Not listed
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
Up to 18 Years
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2030
U.S. locations
6
States / cities
Washington D.C., District of Columbia • Atlanta, Georgia • Boston, Massachusetts + 3 more
Source: ClinicalTrials.gov public record
Updated Jun 15, 2026 · Synced Oct 20, 2026, 5:52 PM EDT
Active, not recruiting No phase listed Observational

Rate of Progression of PCDH15-Related Retinal Degeneration in Usher Syndrome 1F

NCT04765345
View directory record Official record
Conditions
Retinal Degeneration, Retinitis Pigmentosa, Eye Diseases, Hereditary
Interventions
Not listed
Lead sponsor
Jaeb Center for Health Research
Other
Eligibility
8 Years and older
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
3
States / cities
San Francisco, California • Baltimore, Maryland • Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Feb 4, 2026 · Synced Oct 20, 2026, 5:52 PM EDT
Completed Phase 2 Interventional Results available

Alemtuzumab, Fludarabine, and Busulfan Followed By Donor Stem Cell Transplant in Treating Young Patients With Hematologic Disorders

NCT00301834
View directory record Official record
Conditions
Congenital Amegakaryocytic Thrombocytopenia, Diamond-blackfan Anemia, Leukemia, Myelodysplastic Syndromes, Severe Congenital Neutropenia
Interventions
alemtuzumab, busulfan, cyclosporine, fludarabine phosphate, methotrexate, methylprednisolone, allogeneic bone marrow transplantation, allogeneic hematopoietic stem cell transplantation, peripheral blood stem cell transplantation, umbilical cord blood transplantation
Biological · Drug · Procedure
Lead sponsor
University of California, San Francisco
Other
Eligibility
Up to 21 Years
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2011
U.S. locations
2
States / cities
San Francisco, California • Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Sep 28, 2017 · Synced Oct 20, 2026, 5:52 PM EDT
Completed Phase 2 Interventional Results available

Diet Treatment Glucose Transporter Type 1 Deficiency (G1D)

NCT03181399
View directory record Official record
Conditions
GLUT1DS1, Epilepsy, Glut1 Deficiency Syndrome 1, Autosomal Recessive, Glucose Metabolism Disorders, Glucose Transport Defect, Glucose Transporter Type 1 Deficiency Syndrome, Glucose Transporter Protein Type 1 Deficiency Syndrome
Interventions
Triheptanoin
Drug
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
24 Months to 35 Years
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Aug 7, 2025 · Synced Oct 20, 2026, 5:52 PM EDT
Recruiting Phase 2 Interventional

A Phase 2 Study to Evaluate the Safety, PD, PK, and Clinical Activity of ADX-097 in Participants With IgAN, LN or C3G

NCT06419205
View directory record Official record
Conditions
IgA Nephropathy, Lupus Nephritis (LN), C3 (Complement Component 3) Glomerulopathy
Interventions
ADX-097
Drug
Lead sponsor
Akebia Therapeutics
Industry
Eligibility
16 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
3
States / cities
Hollywood, Florida • Lawrenceville, Georgia • Orangeburg, South Carolina
Source: ClinicalTrials.gov public record
Updated Oct 1, 2026 · Synced Oct 20, 2026, 5:52 PM EDT
Recruiting Phase 3 Interventional

Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss

NCT05821959
View directory record Official record
Conditions
Sensorineural Hearing Loss, Bilateral
Interventions
AAVAnc80-hOTOF via Akouos Delivery Device, AAVAnc80-hOTOF via Akouos Delivery Device and Precision Delivery Mechanism
Combination Product
Lead sponsor
Eli Lilly and Company
Industry
Eligibility
Not listed
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2029
U.S. locations
6
States / cities
San Francisco, California • Iowa City, Iowa • Boston, Massachusetts + 3 more
Source: ClinicalTrials.gov public record
Updated Sep 29, 2026 · Synced Oct 20, 2026, 5:52 PM EDT
Recruiting Early Phase 1 Interventional

Serial Blood Count Study

NCT07066085
View directory record Official record
Conditions
Cyclic Neutropenia, Congenital Neutropenia, Neutropenia
Interventions
Serial Blood Count Study, Athelas Home device
Device
Lead sponsor
University of Washington
Other
Eligibility
5 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Jul 15, 2025 · Synced Oct 20, 2026, 5:52 PM EDT
Recruiting Phase 3 Interventional

A Study to See if Tolvaptan is Safe in Infants and Children Who at Enrollment Are 28 Days to Less Than 18 Years Old With Autosomal Recessive Polycystic Kidney Disease (ARPKD)

NCT04782258
View directory record Official record
Conditions
Autosomal Recessive Polycystic Kidney (ARPKD)
Interventions
Tolvaptan Suspension, Tolvaptan Tablets
Drug
Lead sponsor
Otsuka Pharmaceutical Development & Commercialization, Inc.
Industry
Eligibility
28 Days to 18 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
12
States / cities
Washington D.C., District of Columbia • Atlanta, Georgia • Chicago, Illinois + 9 more
Source: ClinicalTrials.gov public record
Updated Aug 7, 2026 · Synced Oct 20, 2026, 5:52 PM EDT
Completed Phase 1 Interventional

A Safety, Pharmacokinetic, Single Ascending Dose Study of Tesevatinib in Pediatric Subjects With Autosomal Recessive Polycystic Kidney Disease (ARPKD)

NCT03096080
View directory record Official record
Conditions
Polycystic Kidney, Autosomal Recessive
Interventions
Tesevatinib
Drug
Lead sponsor
Kadmon Corporation, LLC
Industry
Eligibility
5 Years to 12 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
2
States / cities
Philadelphia, Pennsylvania • Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated May 11, 2022 · Synced Oct 20, 2026, 5:52 PM EDT
Completed No phase listed Observational

Natural History Study of ENPP1 Deficiency and the Early-onset Form of ABCC6 Deficiency

NCT05050669
View directory record Official record
Conditions
Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Generalized Arterial Calcification of Infancy, Autosomal Recessive Hypophosphatemic Rickets, Adenosine Triphosphate Binding Cassette Transporter Protein Subfamily C Member 6
Interventions
No Intervention for this observational study
Other
Lead sponsor
Inozyme Pharma
Industry
Eligibility
2 Years to 18 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
3
States / cities
Boston, Massachusetts • Philadelphia, Pennsylvania • Fort Worth, Texas
Source: ClinicalTrials.gov public record
Updated Jul 6, 2026 · Synced Oct 20, 2026, 5:52 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Language Development in Preterm Children

NCT01599780
View directory record Official record
Conditions
Language Development
Interventions
Not listed
Lead sponsor
Stanford University
Other
Eligibility
Up to 18 Months
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010 – 2018
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated Apr 18, 2018 · Synced Oct 20, 2026, 5:52 PM EDT