Vorinostat for GVHD Prevention

Overseen By Tracey Churay

Age: < 65

Sex: Any

Trial Phase: Phase 2

Sponsor: Sung Won Choi

Must not be taking: Histone deacetylase inhibitors

Disqualifiers: Fanconi anemia, HIV, Hepatitis B, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 5-6 weeks

55 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

Research Team

Mark Vander Lugt, MD, MS

Principal Investigator

University of Michigan

Eligibility Criteria

This trial is for young individuals aged 1 to 26 with non-malignant disorders who are undergoing bone marrow transplant. It's designed to see if Vorinostat can prevent a common post-transplant complication called GVHD when combined with standard treatments.

Inclusion Criteria

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

I agree to use two forms of birth control or remain abstinent as required.

Patient and/or legal guardian have signed the informed consent document

Exclusion Criteria

I was diagnosed with cancer in the last 5 years.

Seropositive for HIV or HTLV

I have active hepatitis B or C.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Conditioning

Participants undergo myeloablative conditioning prior to transplant

1-2 weeks

Treatment

Vorinostat is administered as GVHD prophylaxis from day -10 to day 30 post-transplant for matched sibling and unrelated donors, and from day +5 to day 30 for haploidentical donors

5-6 weeks

Follow-up

Participants are monitored for safety and effectiveness, including overall survival and event-free survival at various time points post-transplant

1 year

Treatment Details

Interventions

• Vorinostat

Trial Overview The study tests the safety and effectiveness of Vorinostat, an anti-inflammatory drug, as a preventive treatment for GVHD in adolescents and young adults receiving bone marrow transplants. Participants will receive Vorinostat along with other standard medications.

Participant Groups

Group I: Vorinostat Experimental Treatment 1 Intervention

For matched sibling and matched unrelated donor transplant recipients Vorinostat will be given orally from day -10 to day 30 post-transplant. For Haploidentical donor transplant recipients Vorinostat will be given orally from day +5 (at least 24 hours after completion of the day +4 cyclophosphamide) through day 30 post-transplant. This will be given by liquid suspension or capsule by mouth.

Who Is Running the Clinical Trial?

Sung Won Choi

Lead Sponsor