drugset
drugset / Trial / NCT04232085

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

NCT04232085 ↗

Phase 2 Recruiting 27 enrolled Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins Maryland Stem Cell Research Fund · collab
Non-randomizedParallel-groupOpen-labelTreatment

Summary

Phase II prospective trial to assess the rates of donor engraftment using reduced intensity conditioning (RIC) hematopoietic stem cell transplant (HSCT) and post-transplant cyclophosphamide (PTCy) for patients with primary immune deficiencies (PID), immune dysregulatory syndromes (IDS), inherited bone marrow failure syndromes (IBMFS), short telomere syndromes, Fanconi anemia, and non-Fanconi DNA double-strand break (DNA-dsb) repair disorder.

Timeline

Start
2020-02-12
Primary completion
2027-12-31
Completion
2028-12-31

Publications

Drugs

EvaluationDrugModalityDoseRoute
Subject Cyclophosphamide Other / unclassified 25 mg/kg Intravenous
Subject Cyclophosphamide Other / unclassified 50 mg/kg Intravenous
Subject Melphalan Small molecule 70 mg/m2 Intravenous
Subject Melphalan Small molecule 140 mg/m2 Intravenous
Background Alemtuzumab Monoclonal antibody 3 mg Intravenous
Background Alemtuzumab Monoclonal antibody 10 mg Intravenous
Background Alemtuzumab Monoclonal antibody 15 mg Intravenous
Background Alemtuzumab Monoclonal antibody 20 mg Intravenous
Background Fludarabine Small molecule 30 mg/m2 Intravenous
Background Mycophenolate Mofetil Small molecule 15 mg/kg Oral
Background Tacrolimus Small molecule 0.015 mg/kg Oral

Indications

Fanconi anemia Hoyeraal-Hreidarsson syndrome bone marrow failure syndrome dyskeratosis congenita and related telomere biology disorder immunodeficiency disease telomere syndrome