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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Terminated Phase 2 Interventional Accepts healthy volunteers Results available

Thrombocytopenia and Bleeding in Wiskott-Aldrich Syndrome (WAS) Patients

NCT00909363
View directory record Official record
Conditions
Wiskott-Aldrich Syndrome, Thrombocytopenia, Bleeding
Interventions
Promacta, blood drawing in patients with WAS, blood drawing in healthy controls
Drug · Diagnostic Test
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
3 Months to 80 Years · Male only
Enrollment
24 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2017
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 17, 2019 · Synced Jul 21, 2026, 4:52 PM EDT
Not yet recruiting Phase 1 Interventional

Prophylactic and Therapeutic DLI-X for Leukemia Relapse After HCT

NCT07254793
View directory record Official record
Conditions
Acute Lymphoid Leukemia, Acute Myeloid Leukemia, Acute Undifferentiated Leukemia (AUL), Myelodysplastic Syndrome, Chronic Myeloid Leukemia, Non-Hodgkin Lymphoma
Interventions
Exercise Mobilized Prophylactic DLI, Standard Prophylactic DLI, Exercise Mobilized Therapeutic DLI, Standard Therapeutic DLI
Biological
Lead sponsor
University of Arizona
Other
Eligibility
Up to 65 Years
Enrollment
94 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
1
States / cities
Tucson, Arizona
Source: ClinicalTrials.gov public record
Updated Jul 12, 2026 · Synced Jul 21, 2026, 4:52 PM EDT
Completed Phase 4 Interventional Results available

A Study to Find Out How Safe and Effective Gammaplex® is in Young People With Primary Immunodeficiency

NCT01289847
View directory record Official record
Conditions
Primary Immune Deficiency Disorders, Common Variable Immunodeficiency, X-linked Agammaglobulinemia, Hyper-IgM Syndrome, Wiskott-Aldrich Syndrome
Interventions
Gammaplex
Biological
Lead sponsor
Bio Products Laboratory
Other
Eligibility
2 Years to 16 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
8
States / cities
Irvine, California • Los Angeles, California • Centennial, Colorado + 5 more
Source: ClinicalTrials.gov public record
Updated Dec 22, 2014 · Synced Jul 21, 2026, 4:52 PM EDT
Active, not recruiting Phase 2Phase 3 Interventional

CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome)

NCT03566043
View directory record Official record
Conditions
Mucopolysaccharidosis Type II (MPS II)
Interventions
RGX-121
Genetic
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
4 Months to 5 Years · Male only
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
4
States / cities
Oakland, California • New Brunswick, New Jersey • Philadelphia, Pennsylvania + 1 more
Source: ClinicalTrials.gov public record
Updated Jan 27, 2025 · Synced Jul 21, 2026, 4:52 PM EDT
Not listed No phase listed Observational

Immune Response Following COVID-19 Vaccination

NCT04736524
View directory record Official record
Conditions
IgM and IgG in Blood
Interventions
Rapid antibody test
Other
Lead sponsor
Texas Cardiac Arrhythmia Research Foundation
Other
Eligibility
16 Years to 85 Years
Enrollment
500 participants
Timeline
2021 – 2022
U.S. locations
1
States / cities
Austin, Texas
Source: ClinicalTrials.gov public record
Updated Feb 2, 2021 · Synced Jul 21, 2026, 4:52 PM EDT
Completed No phase listed Observational

The Clinical Study of Sex Chromosome Variants

NCT01661010
View directory record Official record
Conditions
Male Factor Infertility, Female Factor Infertility
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years and older
Enrollment
112 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2012
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 10, 2026 · Synced Jul 21, 2026, 4:52 PM EDT
Completed Phase 2 Interventional

Modified Stem Cell Transplantation Procedure for Treating Chronic Granulomatous Disease

NCT00006417
View directory record Official record
Conditions
Chronic Granulomatous Disease
Interventions
Stem cell transplantation
Procedure
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
Not listed
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2004
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced Jul 21, 2026, 4:52 PM EDT
Terminated Phase 2 Interventional Results available

Phase 2 Study of a New Ophthalmic Formulation of Cyclosporine (Restasis® X) in Patients With Dry Eye Disease

NCT02013791
View directory record Official record
Conditions
Dry Eye Syndromes
Interventions
Vehicle, Sham, Cyclosporine New Ophthalmic Formulation
Drug · Other
Lead sponsor
Allergan
Industry
Eligibility
18 Years and older
Enrollment
55 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
8
States / cities
Artesia, California • Glendale, California • Irvine, California + 5 more
Source: ClinicalTrials.gov public record
Updated May 14, 2018 · Synced Jul 21, 2026, 4:52 PM EDT
Enrolling by invitation No phase listed Observational

Natural History Study of SCID Disorders

NCT01186913
View directory record Official record
Conditions
Severe Combined Immunodeficiency (SCID), Leaky SCID, Omenn Syndrome, Reticular Dysgenesis, ADA SCID, XSCID
Interventions
Not listed
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
Not listed
Enrollment
690 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2028
U.S. locations
39
States / cities
Birmingham, Alabama • Phoenix, Arizona • Los Angeles, California + 34 more
Source: ClinicalTrials.gov public record
Updated Nov 9, 2020 · Synced Jul 21, 2026, 4:52 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, 4:52 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Study to Evaluate the Effect of GSK1265744 on Cardiac Conduction

NCT02027454
View directory record Official record
Conditions
Infection, Human Immunodeficiency Virus
Interventions
GSK1265744, GSK1265744 matching placebo, Moxifloxacin
Drug
Lead sponsor
ViiV Healthcare
Industry
Eligibility
18 Years to 55 Years
Enrollment
42 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014
U.S. locations
1
States / cities
Overland Park, Kansas
Source: ClinicalTrials.gov public record
Updated Jun 15, 2014 · Synced Jul 21, 2026, 4:52 PM EDT
Suspended Phase 3 Interventional

Efficacy of Topiramate for Weight Loss in Subjects With Metabolic Syndrome

NCT00243984
View directory record Official record
Conditions
Metabolic Syndrome X, Diabetes Mellitus, Type 2
Interventions
topiramate
Drug
Lead sponsor
Northeastern Ohio Universities College of Medicine
Other
Eligibility
18 Years to 75 Years
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2007
U.S. locations
5
States / cities
Akron, Ohio • Canton, Ohio • Youngstown, Ohio
Source: ClinicalTrials.gov public record
Updated Dec 9, 2007 · Synced Jul 21, 2026, 4:52 PM EDT
Completed Phase 2 Interventional

A Safety Study of NNZ-2566 in Patients With Fragile X Syndrome

NCT01894958
View directory record Official record
Conditions
Fragile X Syndrome
Interventions
NNZ-2566, Placebo
Drug
Lead sponsor
Neuren Pharmaceuticals Limited
Industry
Eligibility
12 Years to 45 Years · Male only
Enrollment
72 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
17
States / cities
Sacramento, California • Denver, Colorado • Washington D.C., District of Columbia + 14 more
Source: ClinicalTrials.gov public record
Updated Feb 4, 2018 · Synced Jul 21, 2026, 4:52 PM EDT
Completed Phase 3 Interventional Results available

Efficacy, Safety and Pharmacokinetics of Gammaplex in Primary Immunodeficiency Diseases.

NCT00278954
View directory record Official record
Conditions
Primary Immunodeficiency, Common Variable Hypogammaglobulinemia, X-linked Hypogammaglobulinemia, Hypogammaglobulinemia, Immunodeficiency With Hyper-IgM, Wiskott-Aldrich Syndrome
Interventions
Gammaplex (Intravenous immunoglobulin)
Biological
Lead sponsor
Bio Products Laboratory
Other
Eligibility
3 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2007
U.S. locations
7
States / cities
North Palm Beach, Florida • Atlanta, Georgia • Chicago, Illinois + 3 more
Source: ClinicalTrials.gov public record
Updated Jan 23, 2013 · Synced Jul 21, 2026, 4:52 PM EDT
Not listed No phase listed Observational

Brain Development Research Program

NCT00305305
View directory record Official record
Conditions
Brain Disorders, Aicardi Syndrome
Interventions
Not listed
Lead sponsor
University of California, San Francisco
Other
Eligibility
Not listed
Enrollment
2,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2025
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Oct 9, 2023 · Synced Jul 21, 2026, 4:52 PM EDT
Completed Not applicable Interventional

Treatment of Disruptive Behaviors in Fragile X Syndrome

NCT03510156
View directory record Official record
Conditions
Fragile X Syndrome, Disruptive Behavior
Interventions
Behavior analytic treatment
Behavioral
Lead sponsor
Stanford University
Other
Eligibility
3 Years to 10 Years · Male only
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2022
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated May 3, 2022 · Synced Jul 21, 2026, 4:52 PM EDT
Enrolling by invitation Phase 1 Interventional

Sensitivity of the NIH Toolbox to Stimulant Treatment in Intellectual Disabilities

NCT05301361
View directory record Official record
Conditions
Intellectual Disability, Fragile X Syndrome, Down Syndrome, Attention Deficit Hyperactivity Disorder
Interventions
Methylphenidate Oral Solution
Drug
Lead sponsor
University of California, Davis
Other
Eligibility
6 Years to 24 Years
Enrollment
68 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
3
States / cities
Sacramento, California • Chicago, Illinois • Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Dec 14, 2025 · Synced Jul 21, 2026, 4:52 PM EDT
Completed Not applicable Interventional

Soy Nut Study on Markers of Health

NCT01173380
View directory record Official record
Conditions
Metabolic Syndrome X
Interventions
Soy nuts, Matched food
Other
Lead sponsor
University of California, Davis
Other
Eligibility
21 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2012
U.S. locations
1
States / cities
Davis, California
Source: ClinicalTrials.gov public record
Updated May 29, 2017 · Synced Jul 21, 2026, 4:52 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Inflammation and the Metabolic Syndrome in Humans

NCT00954824
View directory record Official record
Conditions
Metabolic Syndrome X
Interventions
Endotoxin (LPS)
Biological
Lead sponsor
University of Pennsylvania
Other
Eligibility
18 Years to 40 Years
Enrollment
50 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2003 – 2007
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 29, 2017 · Synced Jul 21, 2026, 4:52 PM EDT
Completed Phase 2 Interventional Results available

Treosulfan and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Nonmalignant Inherited Disorders

NCT00919503
View directory record Official record
Conditions
Non-Neoplastic Hematologic and Lymphocytic Disorder
Interventions
Allogeneic Bone Marrow Transplantation, Anti-Thymocyte Globulin, Cyclosporine, Fludarabine Phosphate, Laboratory Biomarker Analysis, Methotrexate, Mycophenolate Mofetil, Peripheral Blood Stem Cell Transplantation, Tacrolimus, Total-Body Irradiation, Treosulfan, Umbilical Cord Blood Transplantation
Procedure · Biological · Drug + 2 more
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
Up to 49 Years
Enrollment
98 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2020
U.S. locations
6
States / cities
Aurora, Colorado • Portland, Oregon • Nashville, Tennessee + 2 more
Source: ClinicalTrials.gov public record
Updated Aug 12, 2021 · Synced Jul 21, 2026, 4:52 PM EDT
Completed Phase 3 Interventional

Efficacy and Safety Study of STX209 (Arbaclofen) for the Treatment of Social Withdrawal in Children With Fragile X Syndrome

NCT01325220
View directory record Official record
Conditions
Fragile X Syndrome
Interventions
arbaclofen, Placebo
Drug
Lead sponsor
Seaside Therapeutics, Inc.
Industry
Eligibility
5 Years to 11 Years
Enrollment
172 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2011
U.S. locations
28
States / cities
Phoenix, Arizona • Long Beach, California • Sacramento, California + 24 more
Source: ClinicalTrials.gov public record
Updated Jul 30, 2013 · Synced Jul 21, 2026, 4:52 PM EDT
Completed Phase 2 Interventional

Safety and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome

NCT01253629
View directory record Official record
Conditions
Fragile X Syndrome
Interventions
AFQ056, Placebo
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years to 45 Years
Enrollment
175 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
12
States / cities
Phoenix, Arizona • Sacramento, California • Decatur, Georgia + 9 more
Source: ClinicalTrials.gov public record
Updated Dec 22, 2020 · Synced Jul 21, 2026, 4:52 PM EDT
Terminated Phase 2Phase 3 Interventional Results available

T-Cell Depletion and Stem Cell Transplant for Immune Deficiencies and Histiocytic Disorders

NCT00176826
View directory record Official record
Conditions
Hemophagocytic Lymphohistiocytosis, X-Linked Lymphoproliferative Disorders, Chediak-Higashi Syndrome, Griscelli Syndrome, Immunologic Diseases, Langerhans-Cell Histiocytosis, Hematologic Diseases
Interventions
Stem Cell Transplant, Myeloablative conditioning regimen
Procedure · Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 55 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2015
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jan 22, 2018 · Synced Jul 21, 2026, 4:52 PM EDT
Completed Phase 4 Interventional Results available

Riluzole in Fragile X Syndrome

NCT00895752
View directory record Official record
Conditions
Fragile X Syndrome
Interventions
Riluzole
Drug
Lead sponsor
Indiana University
Other
Eligibility
18 Years and older
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2010
U.S. locations
1
States / cities
Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated Apr 17, 2017 · Synced Jul 21, 2026, 4:52 PM EDT