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Showing 1–24 of 189 matching trials from the live ClinicalTrials.gov search.
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Completed Phase 1Phase 2 Interventional

Result of tDCS in ASD Children With Comorbidities Like PANDAS, Rare Genetic Diseases or Autoimmune Disorders

NCT06368726
View directory record Official record
Conditions
Attention, Visual Perceptual Weakness, Social Behavior, Fluency Disorder, EEG With Periodic Abnormalities
Interventions
tDCS or Transcranial Direct Current Stimulation, Risperidone, Sham tDCS
Device · Drug · Other
Lead sponsor
Spanish Foundation for Neurometrics Development
Other
Eligibility
6 Years to 11 Years
Enrollment
180 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Albuquerque, New Mexico
Source: ClinicalTrials.gov public record
Updated Sep 9, 2026 · Synced Sep 25, 2026, 11:11 AM EDT
Completed Phase 3 Interventional Results available

Efficacy and Safety of RAD001 in Patients Aged 18 and Over With Angiomyolipoma Associated With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM)

NCT00790400
View directory record Official record
Conditions
Tuberous Sclerosis Complex (TSC), Lymphangioleiomyomatosis (LAM)
Interventions
Everolimus (RAD001), Everolimus Placebo
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
118 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2015
U.S. locations
6
States / cities
Birmingham, Alabama • Phoenix, Arizona • Boston, Massachusetts + 3 more
Source: ClinicalTrials.gov public record
Updated Feb 16, 2017 · Synced Sep 25, 2026, 11:11 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Effect of Estrogen on Mental and Social Functioning in Girls With Turner's Syndrome

NCT00004274
View directory record Official record
Conditions
Turner's Syndrome
Interventions
Estrogen
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
8 Years to 12 Years · Female only
Enrollment
260 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 1990
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced Sep 25, 2026, 11:11 AM EDT
Active, not recruiting Phase 1 Interventional

Dose Escalation Study of CLR 131 in Pediatric Relapsed/Refractory Malignant Tumors Including Neuroblastoma and Sarcomas

NCT03478462
View directory record Official record
Conditions
Pediatric Solid Tumor, Pediatric Lymphoma, Pediatric Brain Tumor, DIPG, Neuroblastoma, Ewing Sarcoma, Rhabdomyosarcoma, Osteosarcoma
Interventions
CLR 131
Drug
Lead sponsor
Cellectar Biosciences, Inc.
Industry
Eligibility
2 Years to 25 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2027
U.S. locations
6
States / cities
Palo Alto, California • New York, New York • Chapel Hill, North Carolina + 3 more
Source: ClinicalTrials.gov public record
Updated Mar 17, 2026 · Synced Sep 25, 2026, 11:11 AM EDT
Completed Not applicable Interventional

Randomized Study of Folic Acid Therapy for Hyperhomocysteinemia in Patients With End Stage Renal Disease Receiving Hemodialysis

NCT00004495
View directory record Official record
Conditions
End Stage Renal Disease, Hyperhomocysteinemia
Interventions
cyanocobalamin, folic acid, pyridoxine
Drug
Lead sponsor
Georgetown University
Other
Eligibility
21 Years to 89 Years
Enrollment
84 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2000
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Mar 24, 2015 · Synced Sep 25, 2026, 11:11 AM EDT
Completed Phase 2 Interventional

Phase II Randomized Study of Alendronate Sodium for Osteopenia in Patients With Gaucher's Disease

NCT00004488
View directory record Official record
Conditions
Gaucher's Disease, Osteopenia
Interventions
alendronate sodium, calcium carbonate, cholecalciferol
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
18 Years to 50 Years
Enrollment
82 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2002
U.S. locations
2
States / cities
Cincinnati, Ohio • Dayton, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 24, 2015 · Synced Sep 25, 2026, 11:11 AM EDT
Recruiting Phase 1Phase 2 Interventional

A First-in-human Study of KK8123 in Adults With X-linked Hypophosphatemia

NCT06525636
View directory record Official record
Conditions
X-linked Hypophosphatemia
Interventions
KK8123
Drug
Lead sponsor
Kyowa Kirin Co., Ltd.
Industry
Eligibility
18 Years to 65 Years
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
5
States / cities
San Francisco, California • New Haven, Connecticut • Indianapolis, Indiana + 2 more
Source: ClinicalTrials.gov public record
Updated Sep 20, 2026 · Synced Sep 25, 2026, 11:11 AM EDT
Completed No phase listed Observational

Natural Disease Progression in Participants With Choroideremia

NCT04795206
View directory record Official record
Conditions
Choroideremia
Interventions
No Intervention
Other
Lead sponsor
Biogen
Industry
Eligibility
10 Years and older
Enrollment
1,178 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Sep 7, 2021 · Synced Sep 25, 2026, 11:11 AM EDT
Completed Not applicable Interventional

Study of Tauroursodeoxycholic Acid for Hepatobiliary Disease in Cystic Fibrosis

NCT00004441
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
tauroursodeoxycholic acid, ursodiol
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
Not listed
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1997 – 2001
U.S. locations
2
States / cities
Denver, Colorado • Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 24, 2015 · Synced Sep 25, 2026, 11:11 AM EDT
Completed No phase listed Observational

Williams Syndrome (WS) and Supravalvar Aortic Stenosis (SVAS) DNA and Tissue Bank

NCT02706639
View directory record Official record
Conditions
Williams Syndrome, Supravalvular Aortic Stenosis, Cardiovascular Disease
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
1 Day to 85 Years
Enrollment
305 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2025
U.S. locations
3
States / cities
Bethesda, Maryland • St Louis, Missouri • Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Aug 27, 2026 · Synced Sep 25, 2026, 11:11 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Study of Hepatic Glucose Production and De Novo Lipogenesis in Patients With Cystic Fibrosis

NCT00014781
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Not listed
Lead sponsor
University of Utah
Other
Eligibility
18 Years to 30 Years
Enrollment
39 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2001
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Mar 11, 2009 · Synced Sep 25, 2026, 11:11 AM EDT
Completed Phase 2 Interventional Results available

Hydroxychloroquine and Phlebotomy for Treating Porphyria Cutanea Tarda

NCT01573754
View directory record Official record
Conditions
Porphyria Cutanea Tarda
Interventions
Hydroxychloroquine, Phlebotomy
Drug · Procedure
Lead sponsor
The University of Texas Medical Branch, Galveston
Other
Eligibility
18 Years to 100 Years
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2021
U.S. locations
1
States / cities
Galveston, Texas
Source: ClinicalTrials.gov public record
Updated Feb 16, 2023 · Synced Sep 25, 2026, 11:11 AM EDT
Completed Not applicable Interventional

The VetSeq Study: a Pilot Study of Genome Sequencing in Veteran Care

NCT03380819
View directory record Official record
Conditions
Rare Diseases, Genetic Disease
Interventions
Genome sequencing
Diagnostic Test
Lead sponsor
VA Boston Healthcare System
Federal
Eligibility
Not listed
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 25, 2023 · Synced Sep 25, 2026, 11:11 AM EDT
Completed Not applicable Interventional

Study of Allogeneic Bone Marrow Transplantation Following Cyclophosphamide and Radiotherapy in Patients With Myelodysplastic Syndrome and Acute Leukemia Related to Fanconi's Anemia

NCT00005892
View directory record Official record
Conditions
Fanconi's Anemia, Myelodysplastic Syndromes, Leukemia, Nonlymphocytic, Acute, Leukemia, Lymphocytic, Acute
Interventions
cyclophosphamide, Allogeneic Bone Marrow Transplantation
Drug · Procedure
Lead sponsor
Fairview University Medical Center
Other
Eligibility
0 Years to 54 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced Sep 25, 2026, 11:11 AM EDT
Completed No phase listed Observational

Clinical and Molecular Correlations in Spinocerebellar Ataxia Type 10 (SCA10)

NCT00004306
View directory record Official record
Conditions
Hereditary Ataxia
Interventions
Not listed
Lead sponsor
Office of Rare Diseases (ORD)
NIH
Eligibility
3 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2009
U.S. locations
1
States / cities
Galveston, Texas
Source: ClinicalTrials.gov public record
Updated Mar 5, 2012 · Synced Sep 25, 2026, 11:11 AM EDT
Completed Phase 1 Interventional

Phase I Study of Retrovirally Mediated Transfer of the Human Glucocerebrosidase Gene Into Peripheral Blood Stem Cells for Autologous Transplantation in Patients With Type I Gaucher Disease

NCT00004294
View directory record Official record
Conditions
Gaucher's Disease
Interventions
human glucocerebrosidase gene into autologous peripheral blood stem cells
Genetic
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
18 Years to 65 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1999
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced Sep 25, 2026, 11:11 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Personalized Antisense Oligonucleotide Therapy for Rare Pediatric Genetic Disease: SCN2A

NCT06314490
View directory record Official record
Conditions
Genetic Disease
Interventions
nL-SCN2A-002
Drug
Lead sponsor
University of California, San Diego
Other
Eligibility
Not listed
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated Mar 31, 2025 · Synced Sep 25, 2026, 11:11 AM EDT
Recruiting Not applicable Interventional Accepts healthy volunteers

Genomic Uniformed-Screening Against Rare Disease In All Newborns

NCT05990179
View directory record Official record
Conditions
Early Onset Genetic Conditions With Near Complete Penetrance
Interventions
Genome sequencing-based newborn screening
Other
Lead sponsor
Columbia University
Other
Eligibility
1 Day to 1 Month
Enrollment
100,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2029
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 20, 2026 · Synced Sep 25, 2026, 11:11 AM EDT
Recruiting Not applicable Interventional Accepts healthy volunteers

Genetic Study to Determine the Cause of Birth Defects in Newborns in Texas

NCT07102966
View directory record Official record
Conditions
Rare Diseases
Interventions
Rapid whole genome sequencing
Genetic
Lead sponsor
Baylor College of Medicine
Other
Eligibility
1 Day to 90 Days
Enrollment
410 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2029
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jan 26, 2026 · Synced Sep 25, 2026, 11:11 AM EDT
Completed No phase listed Observational Accepts healthy volunteers Results available

PCORI Urea Cycle Disorder Study

NCT02740153
View directory record Official record
Conditions
Urea Cycle Disorders
Interventions
No Intervention Given
Other
Lead sponsor
Children's National Research Institute
Other
Eligibility
Up to 18 Years
Enrollment
187 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2020
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Jan 25, 2021 · Synced Sep 25, 2026, 11:11 AM EDT
Recruiting No phase listed Observational

Rare CFTR Mutation Cell Collection Protocol (RARE)

NCT03161808
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Not listed
Lead sponsor
George Solomon
Other
Eligibility
17 Years and older
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2028
U.S. locations
1
States / cities
Birmingham, Alabama
Source: ClinicalTrials.gov public record
Updated May 28, 2026 · Synced Sep 25, 2026, 11:11 AM EDT
Completed Not applicable Interventional

Study of Magnesium Sulfate in Children With Reduced Bone Density Secondary to Chronic Cholestatic Liver Disease

NCT00007033
View directory record Official record
Conditions
Alagille Syndrome, Cholestasis, Biliary Atresia
Interventions
magnesium gluconate, magnesium sulfate
Drug
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
3 Years to 18 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jan 21, 2009 · Synced Sep 25, 2026, 11:11 AM EDT
Recruiting No phase listed Observational

Rare Glycogen Storage Diseases Natural History Study

NCT06795152
View directory record Official record
Conditions
Glycogen Storage Disease, GSD Type 0A, GSD Type 0B, GSD VII, Tarui Disease, GSD X, GSD XII, GSD XIII, GSD XV, PGBM2, PRKAG2, Danon Disease, Polyglucosan Body Myopathy Type 1, Polyglucosan Body Myopathy Type 2, RBCK1 Deficiency
Interventions
No intervention
Other
Lead sponsor
Duke University
Other
Eligibility
0 Years to 90 Years
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2034
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jan 6, 2026 · Synced Sep 25, 2026, 11:11 AM EDT
Active, not recruiting Phase 2 Interventional

Talimogene Laherparepvec and Nivolumab in Treating Patients With Refractory Lymphomas or Advanced or Refractory Non-melanoma Skin Cancers

NCT02978625
View directory record Official record
Conditions
Anaplastic Large Cell Lymphoma, ALK-Negative, Anaplastic Large Cell Lymphoma, ALK-Positive, Apocrine Carcinoma, Carcinoma Arising From Cylindroma, Carcinoma Arising From Spiradenoma, Digital Papillary Adenocarcinoma, Endocrine Mucin-Producing Sweat Gland Carcinoma, Extramammary Paget Disease, Extraocular Sebaceous Carcinoma, Hidradenocarcinoma, Malignant Sweat Gland Neoplasm, Merkel Cell Carcinoma, Microcystic Adnexal Carcinoma, NK-Cell Lymphoma, Unclassifiable, Papillary Adenocarcinoma, Porocarcinoma, Primary Cutaneous Mucinous Carcinoma, Recurrent Mature T-Cell and NK-Cell Non-Hodgkin Lymphoma, Recurrent T-Cell Non-Hodgkin Lymphoma, Refractory Anaplastic Large Cell Lymphoma, Refractory Mature T-Cell and NK-Cell Non-Hodgkin Lymphoma, Refractory Merkel Cell Carcinoma, Refractory Mycosis Fungoides, Refractory Primary Cutaneous T-Cell Non-Hodgkin Lymphoma, Refractory Skin Squamous Cell Carcinoma, Refractory T-Cell Non-Hodgkin Lymphoma, Sezary Syndrome, Signet Ring Cell Adenocarcinoma, Skin Adenoid Cystic Carcinoma, Skin Adnexal Carcinoma, Skin Basal Cell Carcinoma, Skin Basosquamous Cell Carcinoma, Skin Keratoacanthoma, Skin Squamous Cell Carcinoma, Squamoid Eccrine Ductal Carcinoma, Squamous Cell Carcinoma of Unknown Primary, Sweat Gland Carcinoma, Trichilemmal Carcinoma, Vulvar Squamous Cell Carcinoma
Interventions
Biopsy Procedure, Biospecimen Collection, Computed Tomography, Laboratory Biomarker Analysis, Nivolumab, Positron Emission Mammography, Talimogene Laherparepvec
Procedure · Other · Biological
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
68 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2027
U.S. locations
36
States / cities
Los Angeles, California • Orange, California • Pasadena, California + 28 more
Source: ClinicalTrials.gov public record
Updated Aug 12, 2026 · Synced Sep 25, 2026, 11:11 AM EDT