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Showing 1–2 of 2 matching trials from the live ClinicalTrials.gov search.

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Filters: Condition: Congenital Marrow Failures Clear all

Recruiting Phase 2 Interventional

Regenerative Medicine to Restore Hematopoiesis and Immune Function in Immunodeficiencies and Inherited Bone Marrow Failures

NCT04232085

View directory record Official record

Conditions: Primary Immune Deficiency Disorder, Immune Deficiency Disease, Bone Marrow Failure, Short Telomere Length, Fanconi Anemia, Non Fanconi DNA-DSB Repair Disorder, Hoyeraal-Hreidarsson Syndrome, Dyskeratosis Congenita, Telomere Biology Disorder, Short Telomere Syndrome
Interventions: Alemtuzumab, Fludarabine, Melphalan, Low Dose Total Body Irradiation, Cyclophosphamide, Tacrolimus, Mycophenolate Mofetil; Drug · Radiation
Lead sponsor: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins; Other
Eligibility: 4 Months to 50 Years

Enrollment: 27 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2028
U.S. locations: 1
States / cities: Baltimore, Maryland

Source: ClinicalTrials.gov public record

Updated Nov 27, 2025 · Synced May 21, 2026, 7:22 PM EDT

Completed Phase 1 Interventional Results available

Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders

NCT00744692

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Conditions: Non Malignant Disorders, Immunodeficiencies, Congenital Marrow Failures, Hemoglobinopathies, Inborn Errors of Metabolism, Sickle Cell, Thalassemia, Lysosomal Storage Disease
Interventions: Unrelated Umbilical Cord Blood Transplant, Reduced Intensity Conditioning; Biological · Drug
Lead sponsor: Duke University; Other
Eligibility: Up to 21 Years

Enrollment: 22 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2014
U.S. locations: 1
States / cities: Durham, North Carolina

Source: ClinicalTrials.gov public record

Updated Aug 12, 2014 · Synced May 21, 2026, 7:22 PM EDT