- Conditions
- Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis, Inherited Bone Marrow Failure Syndrome, Hemoglobinopathies, Metabolic Disorders
- Interventions
- BPX-501 and Rimiducid
- Biological
- Lead sponsor
- Bellicum Pharmaceuticals
- Industry
- Eligibility
- 4 Months to 55 Years
- Enrollment
- 1 participant
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2018
- U.S. locations
- 1
- States / cities
- Seattle, Washington
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Showing 25–32
of 8
matching trials from the live ClinicalTrials.gov search.
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- 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
- Conditions
- Severe Combined Immunodeficiency
- Interventions
- Gene transfer
- Biological
- Lead sponsor
- David Williams
- Other
- Eligibility
- Male only
- Enrollment
- 8 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2023
- U.S. locations
- 3
- States / cities
- Los Angeles, California • Boston, Massachusetts • Cincinnati, Ohio
- 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
- Severe Combined Immunodeficiency
- Interventions
- Gene-Transduced Autologous CD34+ Stem Cells
- Drug
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
- NIH
- Eligibility
- 18 Months to 20 Years
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2001 – 2011
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- X-linked Severe Combined Immunodeficiency, X-SCID, XSCID
- Interventions
- Plerixafor, Filgrastim, Palifermin, Busulfan, Base-edited hematopoietic stem and progenitor cells
- Genetic · Drug · Biological
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
- NIH
- Eligibility
- 3 Years to 99 Years · Male only
- Enrollment
- 18 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2034
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Hematologic Malignancies
- Interventions
- UCB and HPDSC
- Procedure
- Lead sponsor
- Celgene Corporation
- Industry
- Eligibility
- Up to 55 Years
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2013
- U.S. locations
- 1
- States / cities
- New Orleans, Louisiana
- Conditions
- X-Linked Combined Immunodeficiency Diseases
- Interventions
- CD34+ HSCs transduced with the lentivirus vector, VSV-G pseudotyped CL20-4i-EF1α-hγc-OPT, Palifermin, Busulfan
- Biological · Drug
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
- NIH
- Eligibility
- 2 Years to 40 Years
- Enrollment
- 13 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2022
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland