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Vorinostat for GVHD Prevention in Young People with Non-Malignant Conditions

This study is testing a drug called vorinostat to help prevent graft-versus-host disease (GVHD) in young people (ages 1 to 26) who are having a bone marrow transplant for non-cancerous conditions. GVHD is a serious complication where the new immune cells from the donor attack the patient's body. Participants will receive vorinostat by mouth twice a day for about 40 days after their transplant, in addition to standard GVHD prevention medicines. The researchers want to see if adding vorinostat improves how many patients are free from GVHD and relapse one year after transplant, and if it helps prevent the new cells from failing. The study plans to enroll 55 participants, but its current status is unclear.

Study design
This is a single-arm, open-label study, meaning all participants will receive the same treatment and both patients and doctors will know what treatment is being given. It aims to enroll 55 participants.
What's involved
Participants will take vorinostat orally twice a day from 10 days before transplant to 30 days after transplant. The study does not specify other visits, procedures, or tests.
Compensation
Not stated in the trial record.
Follow-up
The study will measure outcomes at 1 year after transplant and will also track graft failure up to and beyond 42 days post-transplant.

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NCT06995521

Vorinostat for Graft-versus-host Disease (GVHD) Prevention in Non-Malignant Adolescent and Young Adults (AYA) Population

Not Yet Recruiting
PHASE2Ages 1–26InterventionalTreatment
Sung Won Choi
~55 participants
Updated 2026-06-30 on ClinicalTrials.gov
What's tested:Vorinostat

At a glance

Recruiting sites
0 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
GVHD-free relapse-free Survival
Measured over 1 year
+3 more outcomes measured

Conditions

GVHD

Explore related trials

Graft Versus Host DiseaseNon-malignant disorder

Where it's being run

1 sites across 1 states
Michigan1

Study leadership

  • Mark Vander Lugt, MD, MS · PRINCIPAL_INVESTIGATOR · University of Michigan

Who to contact

Tracey Churay
[email protected]
734-615-1307
Email the study team

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Eligibility criteria

Inclusion

Non-malignant condition amenable to transplantation, including but not limited to:
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:
Patients with transfusion-dependent anemias (per protocol) should have a liver MRI to document hepatic iron content (certain values will be excluded)
All patients of childbearing age must agree to practice 2 effective methods of contraception at the same time or agree to abstinence for 6 months after the last dose for females. Males with female sexual partners of reproductive potential should use contraception during treatment and for at least 3 months after the last dose.
Patients treated with other investigational therapies for underlying disorder must discontinue these therapies prior to enrollment on the study unless, in the opinion of the treating physician, discontinuing these therapies prior to transplant would place the patient at undue risk of morbidity or mortality. In this case, patients must discontinue investigational therapies prior to initiation of the conditioning regimen.

Exclusion

Diagnosis of idiopathic severe aplastic anemia
Diagnosis of severe combined immunodeficiency syndrome
Diagnosis of malignancy within the last 5 years.
Diagnosis of Epstein-Barr virus (EBV)-driven lymphoproliferative disorder within the last 5 years
Uncontrolled bacterial, viral, or fungal infection at the time of enrollment
Seropositive for HIV or HTLV
Active hepatitis B or C
Female patients that are pregnant or breast-feeding
Inability to take oral medications.
History of allergy to Vorinostat, related compounds, or any drugs used as part of the conditioning regimen or GVHD prophylaxis.
Patients with transfusion-dependent anemia (≥8 packed red blood cell (PRBC) transfusions/year or ≥20 lifetime transfusions) that have a liver iron content of \>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

What this trial measures

  • GVHD-free relapse-free Survival1 year

    Composite endpoint of 1-year GVHD-free, event free survival (GEFS) with the addition of vorinostat to standard serotherapy-free GVHD prophylaxis. Grade III-IV acute GVHD and chronic GVHD requiring immunosuppression will be considered in this assessment. Events contributing to this endpoint will include death due to any cause, primary or secondary graft failure/rejection, or second HSCT, whichever occurs first.

  • Primary graft failure/rejectionBy day+42 post-Hematopoietic stem cell transplant

    Defined as never achieving an absolute neutrophil count (ANC) ≥500/microliters (µL) or never achieving ≥5% donor myeloid chimerism assessed by peripheral blood chimerism assays by day +42 post-Hematopoietic stem cell transplant (HSCT). Second infusion of hematopoietic stem cells is also considered indicative of primary graft failure by day +42 post-HSCT.

  • Secondary graft failure/rejectionBeyond day +42 post-HSCT

    Defined as \<5% donor myeloid chimerism in peripheral blood beyond day +42 post-HSCT in patients with prior documentation of hematopoietic recovery with ≥5% donor cells by day +42 post-HSCT.

  • Secondary graft failure/rejectionBy day +42 post-HSCT

    Second infusion of hematopoietic stem cells is also considered indicative of primary graft failure by day +42 post-HSCT