A Study of Belumosudil in Children With Chronic Graft Versus Host Disease (schoolROCK)
Start Date
12/2/2025
Completion Date
2/28/2031
Summary
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. Upon completion and evaluation of Phase 1, Phase 2 will commence with the purpose of determining safety and efficacy (ORR by 24 weeks) of belumosudil in participants aged 1 to \<18 years. Study details include: The end of study is defined as 3 years after the last participant is recruited or all participants have discontinued treatment, or have died, whichever comes first. Minimum of 6 participants ages 1 to 6 years will be enrolled for each phase of study Individual participant duration on study will consist of: Up to 4 weeks for screening. Treatment until clinically significant progression of cGVHD, relapse/recurrence of the underlying disease, start of a new systemic treatment for cGVHD, experience of an unacceptable adverse event, request from participant or Investigator, or until the end of the study is reached, whichever comes first. 30 days of post treatment safety follow-up. Long-term follow-up until death or end of study, whichever occurs first.
Eligibility Criteria
Age Range: 1 years to 18 years
Interventions
Belumosudil
Belumosudil
Conditions
Locations
Children's Hospital Los Angeles- Site Number : 8400009
Los Angeles, California 90027
United States
Children's National Medical Center - Washington- Site Number : 8400005
Washington D.C., District of Columbia 20010
United States
Memorial Sloan Kettering Cancer Center - New York - York Avenue- Site Number : 8400001
New York, New York 10065
United States
Texas Children's Hospital- Site Number : 8400008
Houston, Texas 77030
United States
Fred Hutchinson Cancer Research Center- Site Number : 8400002
Seattle, Washington 98109
United States
Investigational Site Number : 0560003
Ghent, 9000
Belgium
Investigational Site Number : 0560001
Leuven, 3000
Belgium
Investigational Site Number : 1240001
Vancouver, British Columbia V6H 3N1
Canada
Investigational Site Number : 1240002
Toronto, Ontario M5G 1X8
Canada
Investigational Site Number : 1560003
Beijing, 100045
China
Investigational Site Number : 1560001
Shanghai, 200040
China
Investigational Site Number : 1560004
Shenzhen, 518026
China
Investigational Site Number : 1560002
Suzhou, 215025
China
Investigational Site Number : 2500002
Marseille, 13885
France
Investigational Site Number : 2500001
Paris, 75019
France
Investigational Site Number : 2760001
Berlin, 13353
Germany
Investigational Site Number : 3760005
Haifa, 3109601
Israel
Investigational Site Number : 3760002
Jerusalem, 9112001
Israel
Investigational Site Number : 3760004
Petah Tikva, 4920235
Israel
Investigational Site Number : 3760003
Ramat Gan, 5262100
Israel
Investigational Site Number : 3760001
Tel Aviv, 6423906
Israel
Investigational Site Number : 3800002
Milan, Milano 20122
Italy
Investigational Site Number : 3800001
Rome, Roma 00165
Italy
Investigational Site Number : 3800003
Turin, Torino 10126
Italy
Investigational Site Number : 3920002
Tokyo, 157-8535
Japan
Investigational Site Number : 5280001
Utrecht, 3584 CS
Netherlands
Investigational Site Number : 7240003
Esplugues de Llobregat, Barcelona [Barcelona] 08950
Spain
Investigational Site Number : 7240001
Barcelona, 08035
Spain
Investigational Site Number : 7240002
Madrid, 28009
Spain
Investigational Site Number : 7920003
Ankara, 06800
Turkey (Türkiye)
Investigational Site Number : 7920001
Izmir, 35100
Turkey (Türkiye)
Investigational Site Number : 8260002
Newcastle upon Tyne, England NE2 4HH
United Kingdom
Investigational Site Number : 8260001
London, London, City of WC1N 3JH
United Kingdom