New hope for kids: drug may prevent transplant complications
NCT ID NCT06995521
First seen Jun 27, 2026 · Last updated Sep 01, 2026 · Updated 4 times
Summary
This study tests whether adding the drug vorinostat to standard care can prevent graft-versus-host disease (GVHD) in children and young adults (ages 1-26) with non-cancerous blood disorders who are getting a bone marrow transplant. GVHD is a serious complication where donor cells attack the patient's body. The trial will enroll 55 participants and measure how many are alive and free of GVHD one year after transplant.
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Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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About 55 people
The number the study aims to enrol. It can still change while the study runs.
- Expected to start
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Sep 2026
An estimate. Start dates often move.
- Expected to finish
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Sep 2028
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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1 year to 26 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
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) \<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. 4. Cardiac Function: Ejection fraction (EF) ≥50% or fractional shortening (FS) ≥26% 5. Performance Status: Karnofsky/Lansky score ≥70%(HIV) and Human T-lymphotropic virus type (HTLV) I/II negative 6. 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. Exclusion Criteria: \- 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 * 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
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
1 site. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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University of Michigan
RECRUITINGAnn Arbor, Michigan, 48109, United States
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