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NCT06995521
Vorinostat for Graft-versus-host Disease (GVHD) Prevention in Non-Malignant Adolescent and Young Adults (AYA) Population
Conditions: GVHD
Sex: All
Ages: 1 Year – 26 Years
Healthy volunteers: No
Phase: PHASE2
Enrollment: 55
Sponsor: Sung Won Choi
Location: University of Michigan Ann Arbor Michigan
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.
Eligibility Criteria
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 function and performance status for allogeneic hematopoietic stem cell transplantation as defined by institutional practice:
1. Pulmonary Function: diffusing capacity of the lungs for carbon monoxide (DLCO), forced expiratory volume in the first second (FEV1), Forced vital capacity (FVC) ≥50% predicted.
2. Renal Function: Estimated or actual glomerular filtration rate (GFR) of ≥50 milliliters per minute (mL/min)/1.72 square meter (m2)
3. Liver Function: aspartate aminotransferase (AST), alanine aminotransferase (ALT) \8 milligram (mg) Iron(Fe)/Gram dry weight or evidence of bridging fibrosis or cirrhosis on biopsy.
* Patients enrolled on another GVHD-prevention clinical trial.
* Patients receiving an ex-vivo T cell-depleted or cluster of differentiation (CD34)-selected stem cell graft.
* Subjects that have had prior treatment with a histone deacetylase inhibitor (e.g. vorinostat, valproic acid) within the last 30 days.
* History of prolonged corrected QT interval (QTc) syndrome.
* Uncontrolled intercurrent illness including, but not limited to, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements
Source: ClinicalTrials.gov (NCT06995521). StuddyBuddy aggregates publicly available trial information.