- 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
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- Conditions
- Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions
- Interventions
- data collection
- Drug
- Lead sponsor
- Paul Szabolcs
- Other
- Eligibility
- 2 Months to 60 Years
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2028
- U.S. locations
- 1
- States / cities
- Pittsburgh, Pennsylvania
- Conditions
- Primary Immune Deficiency Disease
- Interventions
- Flebogamma 5% DIF
- Biological
- Lead sponsor
- Instituto Grifols, S.A.
- Industry
- Eligibility
- 2 Years to 16 Years
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2011
- U.S. locations
- 7
- States / cities
- St. Petersburg, Florida • Atlanta, Georgia • Chicago, Illinois + 4 more
- 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
- Conditions
- Wiskott-Aldrich Syndrome
- Interventions
- Patient Interview guide, Caregiver interview guide, Sociodemographic questionnaire, Pediatric quality of life (PedsQL) questionnaire, Clinical questionnaire
- Other
- Lead sponsor
- GlaxoSmithKline
- Industry
- Eligibility
- 12 Years to 30 Years · Male only
- Enrollment
- 19 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018
- U.S. locations
- 1
- States / cities
- Collegeville, Pennsylvania
- Conditions
- Primary Immunodeficiencies, APECED, CGD, Wiskott-Aldrich Syndrome, SCID
- Interventions
- Not listed
- Lead sponsor
- National Human Genome Research Institute (NHGRI)
- NIH
- Eligibility
- 1 Month and older
- Enrollment
- 716 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2023
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland