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, 6:26 PM EDT

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

Showing 73–95 of 95 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Autosomal Recessive Disorder Clear all
Completed No phase listed Observational

Natural History Study of Patients With Canavan Disease (CANinform Study)

NCT04126005
View directory record Official record
Conditions
Canavan Disease
Interventions
Not listed
Lead sponsor
Aspa Therapeutics
Industry
Eligibility
Not listed
Enrollment
67 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
2
States / cities
Oakland, California • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 17, 2026 · Synced Oct 20, 2026, 6:26 PM EDT
Completed Phase 1 Interventional

Effect of DHA Supplements on Macular Function in Patients With Stargardt Macular Dystrophy and Stargardt-like Macular Dystrophy

NCT00060749
View directory record Official record
Conditions
Macular Degeneration
Interventions
Docosahexaenoic Acid (DHA) Dietary Supplement
Drug
Lead sponsor
National Eye Institute (NEI)
NIH
Eligibility
Not listed
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2007
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 20, 2026, 6:26 PM EDT
Recruiting No phase listed Observational

Investigation of the Genetics of Hematologic Diseases

NCT02720679
View directory record Official record
Conditions
Bone Marrow Failure Syndromes, Erythrocyte Disorder, Leukocyte Disorder, Hemostasis, Blood Coagulation Disorder, Sickle Cell Disease, Dyskeratosis Congenita, Diamond-Blackfan Anemia, Congenital Thrombocytopenia, Severe Congenital Neutropenia, Fanconi Anemia, Myelodysplastic Syndromes, Myeloproliferative Diseases
Interventions
Not listed
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Not listed
Enrollment
1,716 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2050
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Jun 9, 2026 · Synced Oct 20, 2026, 6:26 PM EDT
Terminated Phase 2 Interventional

ADAPT Study: Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency

NCT06462547
View directory record Official record
Conditions
Gene Mutations, Pseudoxanthoma Elasticum, Arterial Calcification, Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets Type 2
Interventions
INZ-701
Drug
Lead sponsor
Inozyme Pharma
Industry
Eligibility
1 Year and older
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
2
States / cities
Rochester, Minnesota • Eatontown, New Jersey
Source: ClinicalTrials.gov public record
Updated Oct 19, 2026 · Synced Oct 20, 2026, 6:26 PM EDT
Recruiting Not applicable Interventional

Efficacy of Low-intensity Shockwave vs Radial Wave for Treatment of Erectile Dysfunction and Pelvic Pain

NCT05868668
View directory record Official record
Conditions
Erectile Dysfunction Due to Arterial Insufficiency, Erectile Dysfunction, Erectile Dysfunction Due to Arterial Disease, Chronic Pelvic Pain Syndrome, Chronic Prostatitis, Erectile Dysfunction Following Radical Prostatectomy, Erectile Dysfunction Following Radiation Therapy
Interventions
Focused shockwave, Radial wave, Sham treatment
Device
Lead sponsor
The Cleveland Clinic
Other
Eligibility
40 Years and older · Male only
Enrollment
186 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Aug 19, 2026 · Synced Oct 20, 2026, 6:26 PM EDT
Completed Not applicable Interventional

Sleep Disordered Breathing and Its Impact on Neuro-cognitive Performance and Quality of Life in Parkinson Disease

NCT02034357
View directory record Official record
Conditions
Parkinson's Disease, Sleep Disordered Breathing, Sleep Obstructive Apnea
Interventions
Neurocognitive function, Sleep evaluations
Other
Lead sponsor
University of Miami
Other
Eligibility
18 Years and older
Enrollment
89 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
1
States / cities
Miami, Florida
Source: ClinicalTrials.gov public record
Updated Mar 24, 2016 · Synced Oct 20, 2026, 6:26 PM EDT
Enrolling by invitation No phase listed Observational

Long Term Follow-up Study of AAVAnc80-hOTOF Gene Therapy

NCT06696456
View directory record Official record
Conditions
Otoferlin Gene-mediated Hearing Loss
Interventions
Not listed
Lead sponsor
Akouos, Inc.
Industry
Eligibility
Not listed
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2033
U.S. locations
3
States / cities
Iowa City, Iowa • Boston, Massachusetts • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 6, 2026 · Synced Oct 20, 2026, 6:26 PM EDT
Completed Phase 1 Interventional

Donor Stem Cell Transplant in Treating Young Patients With Myelodysplastic Syndrome, Leukemia, Bone Marrow Failure Syndrome, or Severe Immunodeficiency Disease

NCT00295971
View directory record Official record
Conditions
Congenital Amegakaryocytic Thrombocytopenia, Leukemia, Myelodysplastic Syndromes, Severe Congenital Neutropenia
Interventions
anti-thymocyte globulin, therapeutic allogeneic lymphocytes, fludarabine phosphate, thiotepa, allogeneic bone marrow transplantation, allogeneic hematopoietic stem cell transplantation, in vitro-treated peripheral blood stem cell transplantation, total-body irradiation
Biological · Drug · Procedure + 1 more
Lead sponsor
University of California, San Francisco
Other
Eligibility
1 Year to 17 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2011
U.S. locations
2
States / cities
San Francisco, California • Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Nov 12, 2012 · Synced Oct 20, 2026, 6:26 PM EDT
Withdrawn Not applicable Interventional

CPAP Titration Using an Artificial Neural Network: A Randomized Controlled Study

NCT00497640
View directory record Official record
Conditions
Obstructive Sleep Apnea
Interventions
Artificial Neural Network
Procedure
Lead sponsor
State University of New York at Buffalo
Other
Eligibility
18 Years to 80 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2009
U.S. locations
1
States / cities
Buffalo, New York
Source: ClinicalTrials.gov public record
Updated Nov 25, 2020 · Synced Oct 20, 2026, 6:26 PM EDT
Terminated Not applicable Interventional

Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies

NCT02179359
View directory record Official record
Conditions
Sickle Cell Disease, Transfusion Dependent Alpha- or Beta- Thalassemia, Diamond Blackfan Anemia, Paroxysmal Nocturnal Hemoglobinuria, Glanzmann Thrombasthenia, Severe Congenital Neutropenia, Shwachman-Diamond Syndrome, Non-Malignant Hematologic Disorders
Interventions
Reduced Toxicity Ablative Regimen, Reduced Intensity Preparative Regimen, Myeloablative Preparative Regimen
Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 55 Years
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2024
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Mar 3, 2025 · Synced Oct 20, 2026, 6:26 PM EDT
Completed Phase 1Phase 2 Interventional

Busulfan, Antithymocyte Globulin, and Fludarabine Followed By a Donor Stem Cell Transplant in Treating Young Patients With Blood Disorders, Bone Marrow Disorders, Chronic Myelogenous Leukemia in First Chronic Phase, or Acute Myeloid Leukemia in First Remission

NCT00305708
View directory record Official record
Conditions
Congenital Amegakaryocytic Thrombocytopenia, Diamond-blackfan Anemia, Fanconi Anemia, Leukemia, Severe Congenital Neutropenia, Thrombocytopenia
Interventions
anti-thymocyte globulin, busulfan, fludarabine phosphate, allogeneic bone marrow transplantation, peripheral blood stem cell transplantation, umbilical cord blood transplantation, radiation therapy
Biological · Drug · Procedure + 1 more
Lead sponsor
University of California, San Francisco
Other
Eligibility
Up to 17 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2004
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Nov 12, 2012 · Synced Oct 20, 2026, 6:26 PM EDT
Completed Phase 1Phase 2 Interventional

Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells

NCT00730314
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia, Anemia, Granuloma, Wiskott-Aldrich Syndrome, Chediak Higashi Syndrome, Osteopetrosis, Neutropenia, Thrombocytopenia, Hurler Disease, Niemann-Pick Disease, Fucosidosis
Interventions
Hematopoietic stem cell transplantation
Procedure
Lead sponsor
Children's Hospital Los Angeles
Other
Eligibility
Up to 21 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2015
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jun 23, 2016 · Synced Oct 20, 2026, 6:26 PM EDT
Recruiting Not applicable Interventional

Treatment of Supine Hypertension in Autonomic Failure (CPAP)

NCT03312556
View directory record Official record
Conditions
Supine Hypertension, Autonomic Failure
Interventions
continuous positive airway pressure (CPAP), Placebo
Device · Drug
Lead sponsor
Vanderbilt University Medical Center
Other
Eligibility
18 Years to 85 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2026
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Nov 6, 2025 · Synced Oct 20, 2026, 6:26 PM EDT
Completed Phase 2 Interventional Results available

The Efficacy and Safety of Secukinumab in Patients With Ichthyoses

NCT03041038
View directory record Official record
Conditions
Ichthyosis, Autosomal Recessive Congenital Ichthyosis, Lamellar Ichthyosis, Congenital Ichthyosiform Erythroderma, Epidermolytic Ichthyosis, Netherton Syndrome
Interventions
Secukinumab, Placebo
Drug
Lead sponsor
Northwestern University
Other
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
2
States / cities
Chicago, Illinois • New York, New York
Source: ClinicalTrials.gov public record
Updated Aug 25, 2021 · Synced Oct 20, 2026, 6:26 PM EDT
Not listed Phase 1Phase 2 Interventional

CD34+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell Transplant

NCT02061800
View directory record Official record
Conditions
Chronic Myeloid Leukemia (CML), Acute Myelogenous Leukemia (AML), Myelodysplastic Syndrome (MDS), Juvenile Myelomonocytic Leukemia (JMML), Acute Lymphoblastic Leukemia (ALL), Lymphoma (Hodgkin's and Non-Hodgkin's)
Interventions
CliniMACS CD34+ Reagent System, Thiotepa, Cyclophosphamide, Alemtuzumab, Tacrolimus, Melphalan, Busulfan, Fludarabine, Methylprednisolone
Device · Drug
Lead sponsor
Diane George
Other
Eligibility
Up to 22 Years
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2026
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 4, 2024 · Synced Oct 20, 2026, 6:26 PM EDT
Enrolling by invitation Phase 1Phase 2 Interventional

rAAV-Olig001-ASPA Gene Therapy for Treatment of Children With Typical Canavan Disease

NCT04833907
View directory record Official record
Conditions
Canavan Disease
Interventions
rAAV-Olig001-ASPA, Levetiracetam, Prednisone
Drug
Lead sponsor
Myrtelle Inc.
Industry
Eligibility
3 Months to 60 Months
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
1
States / cities
Dayton, Ohio
Source: ClinicalTrials.gov public record
Updated Nov 14, 2025 · Synced Oct 20, 2026, 6:26 PM EDT
Recruiting Phase 1Phase 2 Interventional

A Study of DB-OTO, an Adeno-Associated Virus (AAV) Based Gene Therapy, in Children/Infants, Adolescents and Adults With Hearing Loss Due to Otoferlin Mutations

NCT05788536
View directory record Official record
Conditions
Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)
Interventions
DB-OTO
Genetic
Lead sponsor
Regeneron Pharmaceuticals
Industry
Eligibility
Not listed
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2032
U.S. locations
9
States / cities
Los Angeles, California • San Diego, California • Jacksonville, Florida + 6 more
Source: ClinicalTrials.gov public record
Updated Aug 5, 2026 · Synced Oct 20, 2026, 6:26 PM EDT
Enrolling by invitation Phase 1Phase 2 Interventional

pCCLCHIM-p47 (Lentiviral Vector Transduced CD34 Plus Cells) in Patients With p47 Autosomal Recessive Chronic Granulomatous Disease (AR-CGD)

NCT06253507
View directory record Official record
Conditions
p47, Autosomal Recessive, Chronic Granulomatous Disease
Interventions
Cryopreserved Autologous CD34+ cells transduced with pCCLCHIM-p47
Biological
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
3 Years to 65 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 15, 2026 · Synced Oct 20, 2026, 6:26 PM EDT
Withdrawn No phase listed Observational

Using CPAP to Improve Menstruation in Women With Polycystic Ovarian Syndrome and Obstructive Sleep Apnea

NCT03155828
View directory record Official record
Conditions
Sleep Apnea, Obstructive, Anovulation, Polycystic Ovarian Syndrome
Interventions
CPAP
Device
Lead sponsor
University of Louisville
Other
Eligibility
18 Years to 35 Years · Female only
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
1
States / cities
Louisville, Kentucky
Source: ClinicalTrials.gov public record
Updated Aug 3, 2018 · Synced Oct 20, 2026, 6:26 PM EDT
Completed No phase listed Observational

Natural History Study: ENPP1 Deficiency or the Early-Onset Form of ABCC6 Deficiency

NCT07745179
View directory record Official record
Conditions
Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, ATP-Binding Cassette Subfamily C Member 6 Deficiency, Generalized Arterial Calcification of Infancy, Autosomal Recessive Hypophosphatemic Rickets, PXE (Pseudoxanthoma Elasticum)
Interventions
Not listed
Lead sponsor
Inozyme Pharma
Industry
Eligibility
1 Day and older
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 4, 2026 · Synced Oct 20, 2026, 6:26 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Enlarged Vestibular Aqueduct Registry

NCT02798783
View directory record Official record
Conditions
Enlarged Vestibular Aqueduct, Sensorineural Hearing Loss
Interventions
Not listed
Lead sponsor
University Hospitals Cleveland Medical Center
Other
Eligibility
Up to 90 Years
Enrollment
165 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2023
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Dec 6, 2023 · Synced Oct 20, 2026, 6:26 PM EDT
Recruiting Phase 2 Interventional

AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion

NCT04099966
View directory record Official record
Conditions
Acute Leukemia, Severe Aplastic Anemia, Non-hodgkin Lymphoma, Hodgkin Lymphoma, Kostmann, Diamond Blackfan Anemia, Amegakaryocytic Thrombocytopenia, Sickle Cell Disease, Beta-Thalassemia
Interventions
alpha beta depletion
Drug
Lead sponsor
Mitchell Cairo
Other
Eligibility
1 Day to 30 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
1
States / cities
Valhalla, New York
Source: ClinicalTrials.gov public record
Updated Aug 8, 2025 · Synced Oct 20, 2026, 6:26 PM EDT
Completed Phase 2 Interventional Results available

Zimura Compared to Sham in Patients With Autosomal Recessive Stargardt Disease (STGD1)

NCT03364153
View directory record Official record
Conditions
Stargardt's Macular Dystrophy
Interventions
avacincaptad pegol, Sham
Drug
Lead sponsor
Astellas Pharma Global Development, Inc.
Industry
Eligibility
18 Years to 60 Years
Enrollment
121 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
17
States / cities
Phoenix, Arizona • Los Angeles, California • Gainesville, Florida + 14 more
Source: ClinicalTrials.gov public record
Updated Apr 15, 2026 · Synced Oct 20, 2026, 6:26 PM EDT