- Conditions
- Gyrate Atrophy
- Interventions
- Not listed
- Lead sponsor
- National Eye Institute (NEI)
- NIH
- Eligibility
- Not listed
- Enrollment
- 65 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1978 – 2004
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
Search Results
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Showing 25–48
of 24
matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
- 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
- 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
- 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
- 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
- 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
- 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
- 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
- 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
- Conditions
- Bone Marrow Failure Syndrome, Severe Aplastic Anemia, Severe Congenital Neutropenia, Amegakaryocytic Thrombocytopenia, Diamond-Blackfan Anemia, Schwachman Diamond Syndrome, Primary Immunodeficiency Syndromes, Acquired Immunodeficiency Syndromes, Histiocytic Syndrome, Familial Hemophagocytic Lymphocytosis, Lymphohistiocytosis, Macrophage Activation Syndrome, Langerhans Cell Histiocytosis (LCH), Hemoglobinopathies, Sickle Cell Disease, Sickle Cell-beta-thalassemia
- Interventions
- CD34 Stem Cell Selection Therapy
- Biological
- Lead sponsor
- Diane George
- Other
- Eligibility
- Up to 40 Years
- Enrollment
- 37 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2030
- U.S. locations
- 1
- States / cities
- New York, New York
- 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
- Conditions
- Stargardt Disease, Stargardt Macular Degeneration, Stargardt Macular Dystrophy, Autosomal Recessive Stargardt Disease 1 (ABCA4-related)
- Interventions
- ALK-001, Placebo
- Drug
- Lead sponsor
- Alkeus Pharmaceuticals, Inc.
- Industry
- Eligibility
- 8 Years to 70 Years
- Enrollment
- 160 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2026
- U.S. locations
- 14
- States / cities
- Phoenix, Arizona • Los Angeles, California • Aurora, Colorado + 11 more
- Conditions
- Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets, Generalized Arterial Calcification of Infancy, ATP-Binding Cassette Subfamily C Member 6 Deficiency, Pseudoxanthoma Elasticum
- Interventions
- INZ-701
- Drug
- Lead sponsor
- Inozyme Pharma
- Industry
- Eligibility
- Up to 1 Year
- Enrollment
- 16 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2027
- U.S. locations
- 5
- States / cities
- San Diego, California • Boston, Massachusetts • Columbus, Ohio + 2 more
- Conditions
- Aortopathies, Thoracic Aortic Aneurysm, Aortic Valve Disease, Thoracic Aortic Disease, Thoracic Aortic Dissection, Thoracic Aortic Rupture, Ascending Aortic Disease, Descending Aortic Disease, Ascending Aortic Aneurysm, Descending Aortic Aneurysm, Marfan Syndrome, Loeys-Dietz Syndrome, Vascular Ehlers-Danlos Syndrome, Shprintzen-Goldberg Syndrome, Turner Syndrome, PHACE Syndrome, Autosomal Recessive Cutis Laxa, Congenital Contractural Arachnodactyly, Arterial Tortuosity Syndrome, Bicuspid Aortic Valve-Associated Aortopathy, Bicuspid Aortic Valve, Familial Thoracic Aortic Aneurysm and Aortic Dissection
- Interventions
- Not listed
- Lead sponsor
- Yale University
- Other
- Eligibility
- Not listed
- Enrollment
- 3,000 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2030
- U.S. locations
- 2
- States / cities
- Atlanta, Georgia • Indianapolis, Indiana
- Conditions
- Ciliopathy
- Interventions
- Not listed
- Lead sponsor
- National Human Genome Research Institute (NHGRI)
- NIH
- Eligibility
- 5 Months to 80 Years
- Enrollment
- 374 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2003 – 2021
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- 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
- 5
- States / cities
- Phoenix, Arizona • Gainesville, Florida • Erie, Pennsylvania + 2 more
- Conditions
- Alport Syndrome, X-Linked, Alport Syndrome, Autosomal Recessive
- Interventions
- Exaluren
- Drug
- Lead sponsor
- Eloxx Pharmaceuticals, Inc.
- Industry
- Eligibility
- 12 Years and older
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2027
- U.S. locations
- 8
- States / cities
- Los Angeles, California • Stanford, California • Denver, Colorado + 5 more
- 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
- 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
- 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
- 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
- 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
- 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
- Conditions
- Neuronal Ceroid-Lipofuscinoses, Neuronal Ceroid Lipofuscinosis CLN2, Spinocerebellar Ataxia, Autosomal Recessive 7
- Interventions
- Not listed
- Lead sponsor
- Children's Hospital of Orange County
- Other
- Eligibility
- 4 Years and older
- Enrollment
- 5 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2026
- U.S. locations
- 1
- States / cities
- Orange, California