Actively Recruiting
An Open-label Phase 1/2 Study of Belumosudil in Children Aged 1 to <18 Years with Moderate-to-Severe Chronic Graft Versus Host Disease Requiring Systemic Treatment
Led by Sanofi · Updated on 2026-06-05
37
Participants Needed
33
Research Sites
126 weeks
Total Duration
On this page
Sponsors
S
Sanofi
Lead Sponsor
M
Meiji Seika Pharma Co., Ltd.
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating belumosudil, an oral medication, in children aged 1 to less than 18 years who have active moderate to severe chronic graft versus host disease (cGVHD) that has not responded to or has returned after at least two prior systemic treatments. This open-label, single-group Phase 1/2 study aims first to understand how the drug behaves in children aged 1 to less than 12 years and to find the right dose, then to assess safety and effectiveness in children up to 18 years. Participants will take belumosudil by mouth, either as an oral suspension or tablet, once daily with a meal, with possible dose adjustments for those taking certain other medications. The study includes an initial screening period of up to 4 weeks, followed by treatment continuing until significant disease progression, relapse, unacceptable side effects, or end of study. After stopping treatment, participants will have 30 days of safety follow-up and then long-term monitoring until death or study end. During the study, participants will have regular assessments including blood tests and monitoring for side effects. Researchers will measure drug levels in the body, overall response to treatment within 24 weeks, survival outcomes, and duration of response. The total study duration for each participant may be up to 3 years after the last participant joins, ensuring ongoing safety and treatment effect monitoring.
CONDITIONS
Brief Title
A Study of Belumosudil in Children With Chronic Graft Versus Host Disease (schoolROCK)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participant must be 1 to <18 years old at consent/assent signing (Phase 1: 1 to <12 years)
- Must have undergone an allogeneic hematopoietic cell transplant (HCT)
- Has active moderate to severe chronic graft versus host disease (cGVHD) needing systemic therapy
- cGVHD is refractory or recurred after at least 2 prior systemic treatments
- Has received between 2 and 5 prior systemic therapies for cGVHD
- Stable corticosteroid dose for at least 2 weeks if used
- Lansky-Play score ≥60 if under 16 years or Karnofsky score ≥60 if 16 years or older
- Body weight of at least 8 kg
- Use of effective contraception if sexually active as per local regulations
- Capable of giving signed informed consent or has a legally authorized representative
- Life expectancy greater than 6 months
- Able to take study medication orally or via nasogastric tube
You will not qualify if you...
- Progressive underlying disease or post-transplant lymphoproliferative disease within 4 weeks before first dose
- Diagnosis of another malignancy within 3 years prior to first dose (except transplant indication)
- Severe illness or conditions making participation unsuitable (e.g., malabsorption, uncontrolled infections, psychiatric disease)
- Forced expiratory volume (FEV1) less than 39% or lung score of 3
- Pregnant or breastfeeding females
- Initiated new systemic GVHD treatment within 14 days prior to first dose
- Receiving certain systemic GVHD treatments without required dose stability or ability to stop within 14 days
- Use of other systemic GVHD treatments or investigational therapies without required washout
- Use of herbal or recreational drugs within 7 days before starting study drug
- Previous exposure to belumosudil
- Recent live or live-attenuated vaccines within 28 days before starting study drug
- Treatment with other investigational agents or devices within 28 days before first dose
- For Phase 1: use of strong CYP3A4 inducers or PPIs restricted around dosing
- Low blood counts (neutrophils <1.0 x10^9/L or platelets <25 x10^9/L)
- Elevated liver enzymes or bilirubin beyond specified limits
- Reduced kidney function (GFR <30 mL/min/1.73 m2)
- Active viral infections including hepatitis B, hepatitis C, CMV, EBV, or HIV
- Any condition or risk of noncompliance making participation unsuitable as judged by Investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 24 weeks or Cycle 7 Day 1, whichever comes first
Participants receive the study drug Belumosudil orally to treat moderate-to-severe chronic graft versus host disease. Dosage and frequency may vary depending on concomitant medications and participant weight.
Regular visits during treatment cycles
Duration - Up to 3 years after treatment completion
Participants are monitored for safety, adverse events, and long-term outcomes after completing treatment.
Periodic follow-up visits
Trial Site Locations
Total: 33 locations
1
Children's Hospital Los Angeles- Site Number : 8400009
Los Angeles, California, United States, 90027
Actively Recruiting
2
Children's National Medical Center - Washington- Site Number : 8400005
Washington D.C., District of Columbia, United States, 20010
Actively Recruiting
3
Memorial Sloan Kettering Cancer Center - New York - York Avenue- Site Number : 8400001
New York, New York, United States, 10065
Actively Recruiting
4
Texas Children's Hospital- Site Number : 8400008
Houston, Texas, United States, 77030
Actively Recruiting
5
Fred Hutchinson Cancer Research Center- Site Number : 8400002
Seattle, Washington, United States, 98109
Actively Recruiting
6
Investigational Site Number : 0560003
Ghent, Belgium, 9000
Actively Recruiting
7
Investigational Site Number : 0560001
Leuven, Belgium, 3000
Actively Recruiting
8
Investigational Site Number : 1240001
Vancouver, British Columbia, Canada, V6H 3N1
Actively Recruiting
9
Investigational Site Number : 1240002
Toronto, Ontario, Canada, M5G 1X8
Actively Recruiting
10
Investigational Site Number : 1560003
Beijing, China, 100045
Actively Recruiting
11
Investigational Site Number : 1560001
Shanghai, China, 200040
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12
Investigational Site Number : 1560004
Shenzhen, China, 518026
Actively Recruiting
13
Investigational Site Number : 1560002
Suzhou, China, 215025
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14
Investigational Site Number : 2500002
Marseille, France, 13885
Actively Recruiting
15
Investigational Site Number : 2500001
Paris, France, 75019
Actively Recruiting
16
Investigational Site Number : 2760001
Berlin, Germany, 13353
Actively Recruiting
17
Investigational Site Number : 3760005
Haifa, Israel, 3109601
Actively Recruiting
18
Investigational Site Number : 3760002
Jerusalem, Israel, 9112001
Actively Recruiting
19
Investigational Site Number : 3760004
Petah Tikva, Israel, 4920235
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20
Investigational Site Number : 3760003
Ramat Gan, Israel, 5262100
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21
Investigational Site Number : 3760001
Tel Aviv, Israel, 6423906
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22
Investigational Site Number : 3800002
Milan, Milano, Italy, 20122
Actively Recruiting
23
Investigational Site Number : 3800001
Rome, Roma, Italy, 00165
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24
Investigational Site Number : 3800003
Turin, Torino, Italy, 10126
Actively Recruiting
25
Investigational Site Number : 3920002
Tokyo, Japan, 157-8535
Actively Recruiting
26
Investigational Site Number : 5280001
Utrecht, Netherlands, 3584 CS
Actively Recruiting
27
Investigational Site Number : 7240003
Esplugues de Llobregat, Barcelona [Barcelona], Spain, 08950
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28
Investigational Site Number : 7240001
Barcelona, Spain, 08035
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29
Investigational Site Number : 7240002
Madrid, Spain, 28009
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30
Investigational Site Number : 7920003
Ankara, Turkey (Türkiye), 06800
Actively Recruiting
31
Investigational Site Number : 7920001
Izmir, Turkey (Türkiye), 35100
Actively Recruiting
32
Investigational Site Number : 8260002
Newcastle upon Tyne, England, United Kingdom, NE2 4HH
Actively Recruiting
33
Investigational Site Number : 8260001
London, London, City of, United Kingdom, WC1N 3JH
Actively Recruiting
Research Team
T
Trial Transparency email recommended (Toll free for US & Canada)
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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