Use of Vorinostat as GVHD Prophylaxis in Children, Adolescents, and Young Adults With Non-Malignant Disorders Undergoing Allogeneic Blood and Marrow Transplantation
Sung Won Choi
Summary
This is a single-arm, open label, phase 2 study to determine the safety and efficacy of vorinostat without serotherapy as GVHD prophylaxis when combined with either tacrolimus and methotrexate or post-transplant cyclophosphamide, tacrolimus, and mycophenolate in patients aged 1 to 26 years of age with non-malignant disorders undergoing bone marrow transplant following myeloablative conditioning.
Description
The Hypothesis of the trial: The addition of vorinostat to standard GVHD prophylaxis without serotherapy will lead to improved GVHD-free event-free survival (GEFS) at 1-year post-transplant compared to historical serotherapy-containing GVHD prophylaxis regimens in patients with non-malignant disorders (NMD) undergoing Hematopoietic stem cell transplant (HSCT).
Eligibility
- Age range
- 1–26 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: * Non-malignant condition amenable to transplantation, including but not limited to: 1. Primary Immunodeficiency/Primary Immune regulatory disorders 2. Inborn errors of metabolism 3. Red blood cell disorders including hemoglobinopathies per protocol. 4. Inherited bone marrow failure syndromes * Available donor per protocol (matched siblings and matched unrelated donors, haploidentical donors). The use of mismatched unrelated donors will not be allowed for this study. * Patient and/or legal guardian have signed the informed consent document * Adequate organ functio…
Interventions
- DrugVorinostat
For Matched sibling and matched unrelated donor transplant recipients: Vorinostat will be given orally at a dose of 60 milligrams per square meter two times a day (BID) (120 mg/m2/day) from day -10 to day 30 post-transplant. The maximum dose will be 100 mg BID. Haploidentical donor transplant recipients: Vorinostat will be given orally at a dose of 60 mg/m2 BID (120 mg/m2/day) from day +5 (at least 24 hours after completion of the day +4 cyclophosphamide) through day 30 post-transplant. The maximum dose will be 100 mg BID. Dosing may be rounded by +/- 10%. Patients that are able to take capsules and whose calculated dose is ≥91 mg may take 100 mg capsules.
Location
- University of MichiganAnn Arbor, Michigan