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Showing 1–24 of 98 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Autosomal Recessive Clear all
Recruiting No phase listed Observational Accepts healthy volunteers

Brain Metabolism Observed at 3 Tesla or 7 Tesla in Health and Metabolic Disease

NCT05085704
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
Weill Medical College of Cornell University
Other
Eligibility
16 Years to 80 Years
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2029
U.S. locations
2
States / cities
New York, New York • Dallas, Texas
Source: ClinicalTrials.gov public record
Updated May 31, 2026 · Synced Jul 21, 2026, 10:21 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 5, 2026 · Synced Jul 21, 2026, 10:21 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 27, 2017 · Synced Jul 21, 2026, 10:21 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 14, 2025 · Synced Jul 21, 2026, 10:21 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 23, 2016 · Synced Jul 21, 2026, 10:21 PM EDT
Completed Phase 3 Interventional Accepts healthy volunteers Results available

Brain Oxygenation and Hemodynamics During Sleep in Obstructive Sleep Apnea Sufferers

NCT00591591
View directory record Official record
Conditions
Obstructive Sleep Apnea Syndrome
Interventions
OxiplexTS
Device
Lead sponsor
ISS, Inc.
Industry
Eligibility
21 Years to 55 Years
Enrollment
309 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2007 – 2010
U.S. locations
4
States / cities
Orange, California • Stanford, California • Chicago, Illinois + 1 more
Source: ClinicalTrials.gov public record
Updated Mar 15, 2011 · Synced Jul 21, 2026, 10:21 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 Feb 22, 2026 · Synced Jul 21, 2026, 10:21 PM EDT
Terminated Phase 2 Interventional Results available

ATH-1017 Treatment in Subjects With Parkinson's Disease Dementia or Dementia With Lewy Bodies (SHAPE Trial)

NCT04831281
View directory record Official record
Conditions
Parkinson Disease Dementia, Dementia With Lewy Bodies
Interventions
ATH-1017, Placebo
Drug
Lead sponsor
LeonaBio
Industry
Eligibility
40 Years to 85 Years
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
10
States / cities
Boca Raton, Florida • West Palm Beach, Florida • Decatur, Georgia + 6 more
Source: ClinicalTrials.gov public record
Updated Mar 3, 2025 · Synced Jul 21, 2026, 10:21 PM EDT
Terminated Phase 2 Interventional Results available

Study to Evaluate the Safety and Activity of BB3 to Treat Heart Attack

NCT01539590
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 Jul 21, 2026, 10:21 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 14, 2026 · Synced Jul 21, 2026, 10:21 PM EDT
Recruiting Phase 1Phase 2 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
Akouos, Inc.
Industry
Eligibility
Not listed
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
6
States / cities
San Francisco, California • Iowa City, Iowa • Boston, Massachusetts + 3 more
Source: ClinicalTrials.gov public record
Updated Mar 3, 2026 · Synced Jul 21, 2026, 10:21 PM EDT
Completed No phase listed Observational

Gyrate Atrophy of the Choroid and Retina

NCT00001166
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced Jul 21, 2026, 10:21 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 7, 2025 · Synced Jul 21, 2026, 10:21 PM EDT
Recruiting No phase listed Observational

WiTNNess - TNNT1 Myopathy Natural History Study

NCT06374719
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 Jun 22, 2026 · Synced Jul 21, 2026, 10:21 PM EDT
Recruiting Phase 2 Interventional

EXACT Study: A Blinded Study in Patients With Alport Syndrome to Evaluate Exaluren Efficacy and Safety

NCT07523581
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jul 14, 2026 · Synced Jul 21, 2026, 10:21 PM EDT
Recruiting No phase listed Observational

Prevalence of Antibodies and Cytokines in Participants With Chronic Granulomatous Disease

NCT06605378
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 Jul 21, 2026, 10:21 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 25, 2019 · Synced Jul 21, 2026, 10:21 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 21, 2024 · Synced Jul 21, 2026, 10:21 PM EDT
Completed Phase 3 Interventional Results available

The De-novo Use of Eculizumab in Presensitized Patients Receiving Cardiac Transplantation

NCT02013037
View directory record Official record
Conditions
Antibody-mediated Rejection, Hyperacute Rejection of Cardiac Transplant, Left Ventricular Dysfunction, Cardiac Allograft Vasculopathy, Heart Graft Dysfunction
Interventions
Eculizumab
Drug
Lead sponsor
Cedars-Sinai Medical Center
Other
Eligibility
18 Years to 75 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2020
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated May 2, 2021 · Synced Jul 21, 2026, 10:21 PM EDT
Recruiting Phase 1Phase 2 Interventional

A Phase 1/2, First-in-Human Dose Escalation/Expansion Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of a Subretinal Injection of SB-007 in Subjects With Stargardt Disease (STGD1)

NCT06942572
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 Jul 21, 2026, 10:21 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 29, 2017 · Synced Jul 21, 2026, 10:21 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 5, 2023 · Synced Jul 21, 2026, 10:21 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 – 2026
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Jan 20, 2026 · Synced Jul 21, 2026, 10:21 PM EDT
Not yet recruiting Phase 1Phase 2 Interventional

Safety and Tolerability of Subretinal OPGx-RDH12-1001 for LCA-Associated Inherited Retinal Degeneration (LCA-IRD)

NCT07681778
View directory record Official record
Conditions
Leber Congenital Amaurosis, Leber Congenital Amaurosis (LCA)
Interventions
OPGx-RDH12
Drug
Lead sponsor
Opus Genetics, Inc
Industry
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2034
U.S. locations
3
States / cities
Phoenix, Arizona • Philadelphia, Pennsylvania • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jul 14, 2026 · Synced Jul 21, 2026, 10:21 PM EDT