GVHDPhase 2NCT06995521
Vorinostat for Graft-versus-host Disease (GVHD) Prevention in Non-Malignant Adolescent and Young Adults (AYA) Population
A Phase 2 study testing Vorinostat in people with GVHD, run by Sung Won Choi at 1 site in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages 1 year to 26 years
- Conditions the study is for
- GVHD
- What is being tested?
- Vorinostat
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 55 participants
- Where
- 1 siteUnited States
- Study dates
- Dec 2026to Sep 2028
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year to 26 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Non-malignant condition amenable to transplantation, including but not limited to:
- Primary Immunodeficiency/Primary Immune regulatory disorders
- Inborn errors of metabolism
- Red blood cell disorders including hemoglobinopathies per protocol.
- 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.
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- 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:
- 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.
- Renal Function: Estimated or actual glomerular filtration rate (GFR) of ≥50 milliliters per minute (mL/min)/1.72 square meter (m2)
- Liver Function: aspartate aminotransferase (AST), alanine aminotransferase (ALT) <3x upper limit of normal; total bilirubin ≤2.5 milligrams per deciliter (mg/dL) unless related to disease or Gilbert syndrome. There is no upper limit for bilirubin in patients with confirmed Gilbert syndrome.
- Cardiac Function: Ejection fraction (EF) ≥50% or fractional shortening (FS) ≥26%
- Performance Status: Karnofsky/Lansky score ≥70%(HIV) and Human T-lymphotropic virus type (HTLV) I/II negative
- Infectious Disease testing: human immunodeficiency virus
- 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.
Who cannot join
- Previous diagnosis of Fanconi anemia, dyskeratosis congenita or other telomere biology disorders, inherited genetic conditions known to adversely affect DNA-repair, or other disorders with known chemo- or radiosensitivity.
- Additional testing may be conducted per investigator discretion but is not required for enrollment.
- 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
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- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
University of Michigan
Ann Arbor, Michigan, 48109
Tracey Churay, 734-936-4000, tchuray@med.umich.edu
Mark Vander Lugt, MD, MS
About this study
Official title: Use of Vorinostat as GVHD Prophylaxis in Children, Adolescents, and Young Adults With Non-Malignant Disorders Undergoing Allogeneic Blood and Marrow Transplantation
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.
Read the detailed 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).
- Conditions
- GVHD
- Being tested
- Vorinostat
- Keywords
- Non-malignant condition, Medication Vorinostat
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VorinostatDrug
Also called Zolinza
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.
| Study group | Role | What they receive |
|---|---|---|
| Vorinostat | Experimental | 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. Receives: Drug: Vorinostat |
What is being measured
The primary outcome is the main question the study is designed to answer.
GVHD-free relapse-free Survival
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.
Measured: 1 year
Primary graft failure/rejection
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.
Measured: By day+42 post-Hematopoietic stem cell transplant
Secondary graft failure/rejection
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.
Measured: Beyond day +42 post-HSCT
Secondary graft failure/rejection
Second infusion of hematopoietic stem cells is also considered indicative of primary graft failure by day +42 post-HSCT
Measured: By day +42 post-HSCT
Secondary outcomes (11)
Overall survival (OS) at day 100, 6-months, and 1-year post-HSCT
This will be measured after HSCT.
Measured: Day 100, 6-months, and 1-year post-HSCT
Event Free Survival (EFS) at 1-year post-HSCT
An event will be defined as death due to any cause, graft rejection/failure, or second transplant, whichever occurs first.
Measured: 1 year post-HSCT
Neutrophil engraftment at day 42
The time to engraftment of neutrophils >500/microliter (μl) was defined as per Center for International Blood and Marrow Transplant Research (CIBMTR) standards, requiring donor chimerism for neutrophil engraftment.
Measured: Day 42 post-HSCT
Platelet engraftment at day 100 post-HSCT
Platelet engraftment is defined as independence from platelet transfusion for at least 3 days with a platelet count of more than ≥20 × 10^9/L.
Measured: Day 100 post-HSCT
Donor chimerism at day 28, 100, and 1-year post-HSCT
Number of participants with full (95-100%), mixed (5-94%), and no (0-4%) donor cells. Chimerism is reported for unsorted whole blood and T cells.
Measured: Day 28, 100, and 1-year post-HSCT
Primary graft failure at day 42
Measured: Day 42 post-HSCT
Secondary graft failure post-HSCT
Measured: Day 42 post-HSCT
Day 100 grade II-IV and grade III-IV GVHD
Measured: Day 100 post-HSCT
Chronic GVHD requiring immunosuppressive therapy (IST) at 1-year post-HSCT
Measured: 1-year post-HSCT
Incidence of grade 4 systemic infections (septicemia) at 1-year post-HSCT
Measured: 1-year post-HSCT
Incidence of viral reactivation by 1-year post-HSCT
Measured: 1 year post-HSCT
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 May 2025
- Study startDec 2026 (estimated)
- Primary completionSep 2028 (estimated)
- Study completionSep 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for GVHDStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 55 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Sung Won Choi
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Sung Won Choi, University of Michigan
- Investigators
- Mark Vander Lugt, MD, MS, Principal investigator, University of Michigan
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06995521
- Sponsor's protocol ID
- HUM00254327
- Status last verified
- Sep 2026
- First submitted
- 12 May 2025
- Last updated
- 16 Oct 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Use of Vorinostat as GVHD Prophylaxis in Children, Adolescents, and Young Adults With Non-Malignant Disorders Undergoing Allogeneic Blood and Marrow Transplantation
- ClinicalTrials.gov ID
- NCT06995521
- Acronym / sponsor ID
- HUM00254327
- Conditions
- GVHD
- Keywords
- Non-malignant condition, Medication Vorinostat
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 55 participants (estimated)
- Lead sponsor
- Sung Won ChoiUniversity, hospital or other
- Study start
- Dec 2026 (estimated)
- Primary completion
- Sep 2028 (estimated)
- Study completion
- Sep 2028 (estimated)
- First posted
- 29 May 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed 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).
Study design
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Vorinostat | Experimental | Drug: VorinostatFor 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 may qualify
- Ages
- 1 year to 26 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (17 listed)
- 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)
Key exclusion criteria (18 listed)
- Previous diagnosis of Fanconi anemia, dyskeratosis congenita or other telomere biology disorders, inherited genetic conditions known to adversely affect DNA-repair, or other disorders with known chemo- or radiosensitivity.
- Additional testing may be conducted per investigator discretion but is not required for enrollment.
- Diagnosis of idiopathic severe aplastic anemia
- Diagnosis of severe combined immunodeficiency syndrome
- Diagnosis of malignancy within the last 5 years.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| VorinostatAlso: ZolinzaFor 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. | Drug | Vorinostat |
Primary outcome
GVHD-free relapse-free Survival
Time frame: 1 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/rejection
Time frame: By 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/rejection
Time frame: Beyond 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/rejection
Time frame: By day +42 post-HSCT
Second infusion of hematopoietic stem cells is also considered indicative of primary graft failure by day +42 post-HSCT
11 secondary outcomes
Overall survival (OS) at day 100, 6-months, and 1-year post-HSCT
Time frame: Day 100, 6-months, and 1-year post-HSCT
Event Free Survival (EFS) at 1-year post-HSCT
Time frame: 1 year post-HSCT
Neutrophil engraftment at day 42
Time frame: Day 42 post-HSCT
Platelet engraftment at day 100 post-HSCT
Time frame: Day 100 post-HSCT
Donor chimerism at day 28, 100, and 1-year post-HSCT
Time frame: Day 28, 100, and 1-year post-HSCT
Primary graft failure at day 42
Time frame: Day 42 post-HSCT
Secondary graft failure post-HSCT
Time frame: Day 42 post-HSCT
Day 100 grade II-IV and grade III-IV GVHD
Time frame: Day 100 post-HSCT
Chronic GVHD requiring immunosuppressive therapy (IST) at 1-year post-HSCT
Time frame: 1-year post-HSCT
Incidence of grade 4 systemic infections (septicemia) at 1-year post-HSCT
Time frame: 1-year post-HSCT
Incidence of viral reactivation by 1-year post-HSCT
Time frame: 1 year post-HSCT
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Oct 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Mark Vander Lugt, MD, MS principal investigator
University of Michigan
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Vorinostat in people with GVHD, run by Sung Won Choi at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 17 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 16 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Tracey Churay (734-615-1307, tchuray@med.umich.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
GVHD-free relapse-free Survival (time frame: 1 year). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Oct 2026.