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ClinicalTrials.gov public records Last synced May 21, 2026, 11:54 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 97 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Autosomal Recessive Clear all
Completed Phase 1Phase 2 Interventional
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 22, 2016 · Synced May 21, 2026, 11:54 PM EDT
Completed Not applicable Interventional
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 28, 2019 · Synced May 21, 2026, 11:54 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Glut1 Deficiency Syndrome 1, Glucose Metabolism Disorders, Epilepsy, Glut1 Deficiency Syndrome 1, Autosomal Recessive, Glucose Transporter Type 1 Deficiency Syndrome, Glucose Transporter Protein Type 1 Deficiency Syndrome, Glucose Transport Defect
Interventions
Magnetic resonance imaging
Device
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
16 Years to 65 Years
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2026
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Oct 24, 2023 · Synced May 21, 2026, 11:54 PM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Chronic Granulomatous Disease (CGD)
Interventions
Not listed
Lead sponsor
Ensoma
Industry
Eligibility
Not listed
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
50
States / cities
Montgomery, Alabama • Phoenix, Arizona • Little Rock, Arkansas + 47 more
Source: ClinicalTrials.gov public record
Updated Mar 2, 2026 · Synced May 21, 2026, 11:54 PM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
TNNT1-associated Myopathy, Infantile-onset Nemaline Rod Myopathy, Myopathies, Nemaline, Myopathy, Myopathy, Rod, Myopathy; Hereditary, Amish Nemaline Myopathy, Nemaline Myopathy 5, NEM5, Genetic Muscle Disease, Recessive Hereditary Disorder (Autosomal), ANM
Interventions
Not listed
Lead sponsor
Clinic for Special Children
Other
Eligibility
Not listed
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2027
U.S. locations
1
States / cities
Gordonville, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Feb 11, 2025 · Synced May 21, 2026, 11:54 PM EDT
Completed Phase 1 Interventional
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 11, 2012 · Synced May 21, 2026, 11:54 PM EDT
Completed Not applicable Interventional Results available
View directory record Official record
Conditions
Anemia, Aplastic, Amegakaryocytic Thrombocytopenia, Diamond-Blackfan Anemia, Kostmann Syndrome
Interventions
Allogeneic stem cell transplant
Device
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Up to 21 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2009
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated May 29, 2017 · Synced May 21, 2026, 11:54 PM EDT
Recruiting Not applicable Interventional
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 5, 2025 · Synced May 21, 2026, 11:54 PM EDT
Terminated Phase 2 Interventional Results available
View directory record Official record
Conditions
Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Leukemia in Remission, Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Acute Myeloid Leukemia With FLT3/ITD Mutation, Acute Myeloid Leukemia With Gene Mutations, Aplastic Anemia, B-Cell Non-Hodgkin Lymphoma, CD40 Ligand Deficiency, Chronic Granulomatous Disease, Chronic Leukemia in Remission, Chronic Lymphocytic Leukemia, Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Chronic Myelomonocytic Leukemia, Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Congenital Amegakaryocytic Thrombocytopenia, Congenital Neutropenia, Congenital Pure Red Cell Aplasia, Glanzmann Thrombasthenia, Immunodeficiency Syndrome, Myelodysplastic Syndrome, Myelofibrosis, Myeloproliferative Neoplasm, Paroxysmal Nocturnal Hemoglobinuria, Plasma Cell Myeloma, Polycythemia Vera, Recurrent Non-Hodgkin Lymphoma, Refractory Non-Hodgkin Lymphoma, Secondary Acute Myeloid Leukemia, Secondary Myelodysplastic Syndrome, Severe Aplastic Anemia, Shwachman-Diamond Syndrome, Sickle Cell Disease, T-Cell Non-Hodgkin Lymphoma, Thalassemia, Waldenstrom Macroglobulinemia, Wiskott-Aldrich Syndrome
Interventions
Cyclophosphamide, Fludarabine Phosphate, Laboratory Biomarker Analysis, Peripheral Blood Stem Cell Transplantation, Total-Body Irradiation
Drug · Other · Procedure + 1 more
Lead sponsor
Roswell Park Cancer Institute
Other
Eligibility
1 Year to 75 Years
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2023
U.S. locations
1
States / cities
Buffalo, New York
Source: ClinicalTrials.gov public record
Updated Jul 3, 2025 · Synced May 21, 2026, 11:54 PM EDT
Completed Phase 2 Interventional Results available
View directory record Official record
Conditions
Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(15;17)(q22;q12), Adult Acute Myeloid Leukemia With t(16;16)(p13;q22), Adult Acute Myeloid Leukemia With t(8;21)(q22;q22), Adult Grade III Lymphomatoid Granulomatosis, Adult Nasal Type Extranodal NK/T-cell Lymphoma, Anaplastic Large Cell Lymphoma, Angioimmunoblastic T-cell Lymphoma, Aplastic Anemia, Burkitt Lymphoma, Childhood Acute Lymphoblastic Leukemia in Remission, Childhood Acute Myeloid Leukemia in Remission, Childhood Chronic Myelogenous Leukemia, Childhood Diffuse Large Cell Lymphoma, Childhood Grade III Lymphomatoid Granulomatosis, Childhood Immunoblastic Large Cell Lymphoma, Childhood Myelodysplastic Syndromes, Childhood Nasal Type Extranodal NK/T-cell Lymphoma, Chronic Myelomonocytic Leukemia, Chronic Phase Chronic Myelogenous Leukemia, Congenital Amegakaryocytic Thrombocytopenia, Diamond-Blackfan Anemia, Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue, Hepatosplenic T-cell Lymphoma, Juvenile Myelomonocytic Leukemia, Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable, Nodal Marginal Zone B-cell Lymphoma, Paroxysmal Nocturnal Hemoglobinuria, Peripheral T-cell Lymphoma, Polycythemia Vera, Post-transplant Lymphoproliferative Disorder, Previously Treated Myelodysplastic Syndromes, Primary Myelofibrosis, Recurrent Adult Acute Lymphoblastic Leukemia, Recurrent Adult Acute Myeloid Leukemia, Recurrent Adult Burkitt Lymphoma, Recurrent Adult Diffuse Large Cell Lymphoma, Recurrent Adult Diffuse Mixed Cell Lymphoma, Recurrent Adult Diffuse Small Cleaved Cell Lymphoma, Recurrent Adult Grade III Lymphomatoid Granulomatosis, Recurrent Adult Hodgkin Lymphoma, Recurrent Adult Immunoblastic Large Cell Lymphoma, Recurrent Adult Lymphoblastic Lymphoma, Recurrent Adult T-cell Leukemia/Lymphoma, Recurrent Childhood Acute Lymphoblastic Leukemia, Recurrent Childhood Acute Myeloid Leukemia, Recurrent Childhood Anaplastic Large Cell Lymphoma, Recurrent Childhood Grade III Lymphomatoid Granulomatosis, Recurrent Childhood Large Cell Lymphoma, Recurrent Childhood Lymphoblastic Lymphoma, Recurrent Childhood Small Noncleaved Cell Lymphoma, Recurrent Cutaneous T-cell Non-Hodgkin Lymphoma, Recurrent Grade 1 Follicular Lymphoma, Recurrent Grade 2 Follicular Lymphoma, Recurrent Grade 3 Follicular Lymphoma, Recurrent Mantle Cell Lymphoma, Recurrent Marginal Zone Lymphoma, Recurrent Mycosis Fungoides/Sezary Syndrome, Recurrent Small Lymphocytic Lymphoma, Recurrent/Refractory Childhood Hodgkin Lymphoma, Refractory Chronic Lymphocytic Leukemia, Refractory Hairy Cell Leukemia, Refractory Multiple Myeloma, Secondary Acute Myeloid Leukemia, Secondary Myelodysplastic Syndromes, Secondary Myelofibrosis, Severe Combined Immunodeficiency, Severe Congenital Neutropenia, Shwachman-Diamond Syndrome, Splenic Marginal Zone Lymphoma, T-cell Large Granular Lymphocyte Leukemia, Waldenstrom Macroglobulinemia, Wiskott-Aldrich Syndrome
Interventions
fludarabine phosphate, melphalan, total-body irradiation, tacrolimus, mycophenolate mofetil, methotrexate, laboratory biomarker analysis, allogeneic hematopoietic stem cell transplantation, peripheral blood stem cell transplantation
Drug · Radiation · Other + 1 more
Lead sponsor
Roswell Park Cancer Institute
Other
Eligibility
3 Years to 75 Years
Enrollment
94 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2019
U.S. locations
1
States / cities
Buffalo, New York
Source: ClinicalTrials.gov public record
Updated Sep 23, 2019 · Synced May 21, 2026, 11:54 PM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Homozygous Familial Hypercholesterolemia
Interventions
Demographics, diagnosis type, genotype, lipid profile, treatment allocation, country of residence.
Other
Lead sponsor
University of Pennsylvania
Other
Eligibility
Not listed
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2026
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 17, 2025 · Synced May 21, 2026, 11:54 PM EDT
Completed No phase listed Observational
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
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 Sep 18, 2024 · Synced May 21, 2026, 11:54 PM EDT
Recruiting Not applicable Interventional
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 – 2026
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Jan 20, 2026 · Synced May 21, 2026, 11:54 PM EDT
Terminated Phase 2 Interventional Results available
View directory record Official record
Conditions
Myocardial Infarction
Interventions
BB3, Normal saline
Drug
Lead sponsor
Angion Biomedica Corp
Industry
Eligibility
21 Years to 80 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
2
States / cities
Minneapolis, Minnesota • New Haven, New York
Source: ClinicalTrials.gov public record
Updated Nov 20, 2014 · Synced May 21, 2026, 11:54 PM EDT
Withdrawn Not applicable Interventional
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 24, 2020 · Synced May 21, 2026, 11:54 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Glycogen Storage Disease
Interventions
mRNA-3745
Drug
Lead sponsor
ModernaTX, Inc.
Industry
Eligibility
6 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
8
States / cities
Farmington, Connecticut • Boston, Massachusetts • Durham, North Carolina + 4 more
Source: ClinicalTrials.gov public record
Updated Dec 14, 2025 · Synced May 21, 2026, 11:54 PM EDT
Recruiting Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Stargardt Disease, Stargardt Macular Degeneration, Stargardt Macular Dystrophy
Interventions
SB-007
Genetic
Lead sponsor
Splice Bio
Industry
Eligibility
12 Years to 65 Years
Enrollment
57 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
5
States / cities
Aurora, Colorado • Miami, Florida • Boston, Massachusetts + 2 more
Source: ClinicalTrials.gov public record
Updated Apr 28, 2025 · Synced May 21, 2026, 11:54 PM EDT
Enrolling by invitation No phase listed Observational
View directory record Official record
Conditions
Citrate Transporter Deficiency, Epilepsy, Rare Diseases, Movement Disorders, Genetic Disorder, SLC13A5 Deficiency, EIEE25, Kohlschutter-Tonz Syndrome (non-ROGDI), 17p13.1 Deletions Confined to SLC13A5 Gene, Citrate Transporter Disorder
Interventions
Not listed
Lead sponsor
TESS Research Foundation
Other
Eligibility
Not listed
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
1
States / cities
Palo Alto, California
Source: ClinicalTrials.gov public record
Updated Nov 21, 2024 · Synced May 21, 2026, 11:54 PM EDT
Enrolling by invitation Phase 1Phase 2 Interventional
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 13, 2025 · Synced May 21, 2026, 11:54 PM EDT
Not listed Phase 2 Interventional
View directory record Official record
Conditions
Autosomal Recessive Ichthyosis
Interventions
KB105, Placebo
Biological · Other
Lead sponsor
Krystal Biotech, Inc.
Industry
Eligibility
6 Months and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
Rancho Santa Margarita, California
Source: ClinicalTrials.gov public record
Updated Feb 22, 2023 · Synced May 21, 2026, 11:54 PM EDT
Recruiting Phase 1Phase 2 Interventional
View directory record Official record
Conditions
ARB, BVMD, Autosomal-Dominant Bestrophinopathy, Best Vitelliform Macular Dystrophy
Interventions
OPGx-BEST1
Genetic
Lead sponsor
Opus Genetics, Inc
Industry
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
4
States / cities
Los Angeles, California • Gainesville, Florida • Cincinnati, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Mar 23, 2026 · Synced May 21, 2026, 11:54 PM EDT
Recruiting No phase listed Observational
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 Nov 3, 2025 · Synced May 21, 2026, 11:54 PM EDT
Enrolling by invitation Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Chronic Granulomatous Disease, Granulomatous Disease, Chronic
Interventions
PM359
Biological
Lead sponsor
Prime Medicine, Inc.
Industry
Eligibility
6 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2030
U.S. locations
3
States / cities
Los Angeles, California • Bethesda, Maryland • Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Apr 5, 2026 · Synced May 21, 2026, 11:54 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers
View directory record Official record
Conditions
Molybdenum Cofactor Deficiency (MoCD), Rare Autosomal Recessive Disorder, Deficiency of Activity of Molybdenum-dependent Enzymes (Sulfite Oxidase [SOX], Xanthine Dehydrogenase, and Aldehyde Oxidase)
Interventions
ALXN1101, Placebo
Drug
Lead sponsor
Origin Biosciences
Industry
Eligibility
18 Years to 60 Years
Enrollment
24 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 18, 2019 · Synced May 21, 2026, 11:54 PM EDT