New hope for teens with chronic Graft-Versus-Host disease: belumosudil under study
NCT ID NCT06616415
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tested the drug belumosudil in Chinese teenagers aged 12 to 18 with chronic graft-versus-host disease (cGVHD), a complication after stem cell transplant. The goal was to see how well the drug works and how safe it is for those who didn't respond to steroids. Six participants took a tablet once daily, and researchers measured drug levels, response rates, and side effects.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- belumosudil (a drug taken as a tablet to treat chronic graft-versus-host disease)
- What this could lead to
- If it works, this could offer a new treatment option for teenagers with chronic graft-versus-host disease who haven't improved with steroids.
- What could go wrong
- This is a very small study (only 6 participants) and is already completed, so results may not apply to all patients. The drug may not work better than existing treatments or could cause side effects.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 4
Runs after approval, following long-term safety and how well the treatment works in everyday use.
- Participants
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6 people
The number who actually took part.
- Started
-
Dec 2024
- Finished
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May 2026
- Lead sponsor
-
A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
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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12 to 17 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: * Participant must be 12 to less than 18 years of age at the time of signing the informed consent. * Participant has undergone allogeneic hematopoietic stem cell transplantation (allo-HSCT). * Has active moderate to severe cGVHD. * Has received at least one line of prior systemic therapy for cGVHD. * Participant must receive a corticosteroid therapy for cGVHD with a stable dose for at least 2 weeks prior to the first dose of the IMP. * Has a Lansky-Play performance score of ≥60. * Participants should have an expected survival of longer than 6 months. * Body weight of 30 kg and above. * Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies. * The participant or their legally authorized representative (LAR) must be capable of giving signed informed consent. Exclusion Criteria: Participants are excluded from the study if any of the following criteria apply: * Recurrence of hematologic neoplasms (according to the corresponding criteria for recurrence of primary hematologic neoplasms) or post-transplant lymphoproliferative disease at screening. * Received investigational systemic therapy for cGVHD within 28 days prior to enrollment, unless the prior treatment had been washed out for at least 28 days or 5 half-lives prior to enrollment, whichever is shorter. * Absolute neutrophil count (ANC) \<1.0 × 10\^9/L. * Platelet count \<50 × 10\^9/L. * Alanine aminotransferase (ALT) \>3× the upper limit of normal (ULN), aspartate aminotransferase (AST) \>3 × ULN. * Total bilirubin (TBIL) \>1.5 × ULN (\>3 ULN if Gilbert's syndrome). * Estimated Glomerular Filtration Rate (eGFR) \<30 mL/min/1.73 m\^2 using the revised Bedside Schwartz formula . Revised Schwartz equation: CrCl (mL/min/1.73 m\^2) = 0.413 × (height \[in cm\])/Creatinine (in mg/dL) at screening visit. * Participant not suitable for participation, whatever the reason, as judged by the Investigator, including medical or clinical conditions, or participants potentially at risk of noncompliance to study procedures.
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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.
Contacts and locations
Locations
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Investigational Site Number : 1560001
Beijing, 100045, China
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Investigational Site Number : 1560002
Shanghai, 200127, China
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Stem cell infusions put to the test against a tough transplant complication
- Can adding rituximab improve remission in chronic GVHD?
- Can a new pill tame the immune System's attack after stem cell transplants?
- Can a new pill outperform standard care for a tough transplant complication?
- Can meditation tame the Long-Term side effects of a stem cell transplant?
- Could a liquid form of a Graft-Versus-Host disease drug be as effective as the pill?