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ClinicalTrials.gov public records Last synced Jul 22, 2026, 4:52 AM EDT

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Showing 1–7 of 7 matching trials from the live ClinicalTrials.gov search.
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Conditions
Common Variable Immunodeficiency (CVID), Primary Immune Regulatory Disorder, Immune Dysregulation, DiGeorge Syndrome, STAT 1 Gain of Function, STAT 3 Gain of Function, Hypomorphic RAG1 Deficiency, CD40 Ligand Deficiency, Mendelian Susceptibility to Mycobacterial Disease, GATA2 Associated Immunodeficiency, CD40 Deficiency, Hypomorphic RAG2 Deficiency, Immune Dysregulation Polyendocrinopathy Enteropathy X-Linked Syndrome, Omenn Syndrome, Chronic Granulomatous Disease
Interventions
Hematopoietic stem cell transplant (HSCT)
Biological
Lead sponsor
Paul Szabolcs
Other
Eligibility
5 Years to 40 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Terminated Not applicable Interventional Accepts healthy volunteers

Mendelian Reverse Cholesterol Transport Study

NCT01782027
Conditions
Cholesterol, HDL, Lipid Metabolism, Inborn Errors, Tangier Disease, LCAT Deficiency, Cholesteryl Ester Transfer Protein (CETP) Deficiency
Interventions
3H-cholesterol bound to albumin
Drug
Lead sponsor
University of Pennsylvania
Other
Eligibility
18 Years to 75 Years
Enrollment
17 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2024
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Conditions
Enchondromatosis
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
2 Years to 100 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Hidradenitis Suppurativa
Interventions
Not listed
Lead sponsor
University of Chicago
Other
Eligibility
7 Years and older
Enrollment
97 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
1
States / cities
Chicago, Illinois
Conditions
Severe Combined Immunodeficiency (SCID), Immunodeficiency With Predominant T-cell Defect, Unspecified, Severe Chronic Neutropenia, Chronic Granulomatous Disease (CGD), Hyper IgE Syndromes, Hyper IgM Deficiencies, Wiskott-Aldrich Syndrome, Mendelian Susceptibility to Mycobacterial Disease, Common Variable Immune Deficiency (CVID)
Interventions
CD3/CD19 negative allogeneic hematopoietic stem cells
Biological
Lead sponsor
Paul Szabolcs
Other
Eligibility
5 Years to 45 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2027
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Conditions
Anxiety Disorder, Depression, Genetic Disorder, Hematopoietic and Lymphoid Cell Neoplasm, Malignant Solid Neoplasm
Interventions
Electronic Health Record Review
Other
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
Not listed
Enrollment
1 participant
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2028
U.S. locations
1
States / cities
Houston, Texas
Not listed Not applicable Interventional Results available

Perinatal Precision Medicine

NCT03211039
Conditions
Genetic Diseases, Genetic Syndrome, Mendelian Disorders
Interventions
Genomic sequencing and molecular diagnostic results, if any.
Genetic
Lead sponsor
Rady Pediatric Genomics & Systems Medicine Institute
Other
Eligibility
Up to 4 Months
Enrollment
213 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2024
U.S. locations
1
States / cities
San Diego, California