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Compares treatment options for Chronic Graft Versus Host Disease
Official title A Study of Belumosudil in Children With Chronic Graft Versus Host Disease (schoolROCK)
ClinicalTrials.gov ID: NCT07116031
What this study is testing
What is Belumosudil?
Belumosudil is an investigational medicine, given as a pill taken by mouth, being studied as a potential treatment for chronic graft versus host disease.
Also referred to as SAR445761, REZUROCK.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This is an open-label, single group, Phase 1/2, 1-arm study for treatment of children aged 1 to \<18 years with active moderate-to-severe cGVHD that is refractory to or recurred after at least 2 prior lines of systemic therapy for cGVHD. The purpose of Phase 1 is to determine the PK profiles and to establish the Recommended Pediatric Equivalent Dose (RPED) of belumosudil in participants aged 1 to \<12 years with active moderate to severe cGVHD.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 1 to 18
You may be able to join if
- Participant must be 1 to \<18 years of age, at the time the consent/assent is signed. For Phase 1: participant must be 1 to \<12 years of age, at the...
- Participant has undergone an allogeneic HCT
- Has active moderate to severe cGVHD, defined using the NIH Consensus diagnosis and staging criteria for which systemic therapy is required
- cGVHD is refractory to or has recurred after at least 2 prior lines of systemic treatment
- Has received at least two lines of prior systemic therapy for cGVHD, but no more than 5 lines.
You likely can't join if
- Progressive underlying disease or post-transplant lymphoproliferative disease within 4 weeks prior to the first dose of the IMP.
- Diagnosed with another malignancy (other than malignancy for which transplant was performed) within 3 years prior to the first dose of the IMP
- History or other evidence of severe illness or any other conditions that would make the participant, in the opinion of the Investigator, unsuitable...
- Has a forced expiratory volume (in the first second; FEV1) ≤39% or has lung score of 3
- Female participants who are pregnant or breastfeeding
- Participants who meet any of the following criteria regarding systemic GVHD treatments:
See the full eligibility criteria
Who can join
- Participant must be 1 to \<18 years of age, at the time the consent/assent is signed. For Phase 1: participant must be 1 to \<12 years of age, at the time the consent/assent is signed. For Phase 2: participant must be 1...
- Participant has undergone an allogeneic HCT
- Has active moderate to severe cGVHD, defined using the NIH Consensus diagnosis and staging criteria for which systemic therapy is required
- cGVHD is refractory to or has recurred after at least 2 prior lines of systemic treatment
- Has received at least two lines of prior systemic therapy for cGVHD, but no more than 5 lines.
- If participant receives corticosteroid therapy for cGVHD, the dose must be stable for at least 2 weeks prior to the first dose of the IMP
- Has a Lansky-Play (if aged \<16 years) or Karnofsky (if aged ≥16 years) performance scale of ≥60
- Body weight of 8 kg and above
- Contraceptive use by sexually active male and female 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
- Life expectancy of \>6 months
- Participants can take the IMP orally or via a nasogastric tube
What rules you out
- Progressive underlying disease or post-transplant lymphoproliferative disease within 4 weeks prior to the first dose of the IMP.
- Diagnosed with another malignancy (other than malignancy for which transplant was performed) within 3 years prior to the first dose of the IMP
- History or other evidence of severe illness or any other conditions that would make the participant, in the opinion of the Investigator, unsuitable for the study (such as malabsorption syndromes, active, uncontrolled...
- Has a forced expiratory volume (in the first second; FEV1) ≤39% or has lung score of 3
- Female participants who are pregnant or breastfeeding
- Participants who meet any of the following criteria regarding systemic GVHD treatments:
- Participants who newly initiated any systemic GVHD treatment within 14 days prior to the first dose of belumosudil.
- Participants receiving systemic GVHD treatments ibrutinib, ruxolitinib, mycophenolate (MMF), methotrexate, rituximab, axatilimab, or imatinib who are unable to meet the following requirements:
- No dose increases from 14 days prior to belumosudil initiation and continuing for the first 14 days of belumosudil treatment (dose reductions and discontinuations are permitted during this period)
- Ability to discontinue these therapies within 14 days after initiating belumosudil (allowing for a maximum overlap period of up to 14 days with belumosudil treatment)
- Participants receiving other systemic GVHD treatments (apart from corticosteroids and calcineurin inhibitors) including investigational treatments who have not completed a washout period of at least 28 days or 5...
- The use of herbal and recreational drugs within 7 days before the start of study intervention
- Participant has had previous exposure to belumosudil
- Administration of live or live-attenuated vaccines is prohibited within 28 days or 5 elimination half-lives of the respective vaccine, whichever is longer, prior to IMP administration and until study intervention...
- Treatment with any non-GVHD investigational agent, or any investigational device or procedure, within 28 days (or 5 half-lives, whichever is longer) of enrollment, prior to the first dose of the IMP
- For Phase 1 only: Administration with strong CYP3A4 inducers is not allowed within 14 days or 5 half-lives (whichever is longer) of the first dose of IMP until the study intervention discontinuation.
- For Phase 1 only: PPIs are not allowed within 1 day or 5 half-lives (whichever is longer) of the first dose of IMP and Day 15 of Cycle 1. They can be restarted on Cycle 1 Day 16.
- Absolute neutrophil count \<1.0 × 109/L. The use of granulocyte-colony stimulating factor (G-CSF) is not allowed within 7 days prior to the ANC test to reach this level during screening
- Platelet count \<25 × 109/L. Platelet transfusions are not allowed within 72 hours before hematology screening test. Participants with platelet transfusion refractoriness will be excluded. (Participants who have...
- Alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST) \>3× upper limit of normal (ULN) (\> 5x ULN if abnormalities are due to cGVHD)
- Total bilirubin \>1.5 × ULN (\>3 x ULN if Gilbert's syndrome or if abnormalities are due to cGVHD)
- Glomerular filtration rate (GFR) \<30 mL/min/1.73 m2 using the revised Bedside Schwartz calculator
- Participants with an active viral disease including hepatitis B virus (HBV) and hepatitis C virus (HCV)
- Active uncontrolled Cytomegalovirus (CMV) or Epstein-Barr virus (EBV) infection
- Known history of human immunodeficiency virus (HIV)
- 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 The above...
The study team makes the final eligibility decision.
Where it's taking place
- Los Angeles, California, United States
- Washington D.C., District of Columbia, United States
- New York, New York, United States
- Houston, Texas, United States
- Seattle, Washington, United States
- Ghent, Belgium
- Leuven, Belgium
- Vancouver, British Columbia, Canada
- Toronto, Ontario, Canada
- Beijing, China
- Shanghai, China
- Shenzhen, China
- Suzhou, China
- Marseille, France
- Paris, France
- Berlin, Germany
- Haifa, Israel
- Jerusalem, Israel
- Petah Tikva, Israel
- Ramat Gan, Israel
+ 13 more site(s).
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 1 year to 18 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Los Angeles, California, United States; Washington D.C., District of Columbia, United States; New York, New York, United States; Houston, Texas, United States; Seattle, Washington, United States; Ghent, Belgium and 27 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.