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A Study of Belumosudil in Children With Chronic Graft Versus Host Disease (schoolROCK)

ConditionChronic Graft Versus Host Disease
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age1-18
SponsorSanofi

About this trial

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

This trial does not accept healthy volunteers

Qualifiers

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 to <18 years of age, at the time the consent/assent is signed.

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

Disqualifiers

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 infections, or poorly controlled psychiatric disease)

Has a forced expiratory volume (in the first second; FEV1) ≤39% or has lung score of 3

Trial design

Design model

Single group

Treatments tested in this trial

  • Belumosudil

    Drug

    Pharmaceutical form:Oral suspension -Route of administration:Oral or nasogastric tube

  • Belumosudil

    Drug

    Pharmaceutical form:Tablet formulation-Route of administration:Oral

Treatment groups

37 Participants
are divided into 1 treatment group
Group A: belumosudilExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Phase 1: AUC

PK parameter (AUC at steady state)

Time frame
Cycle 1 Day 15 after the last participant dosed in the phase 1 part.
2

Proportion of participants who achieve an overall response (partial response [PR] or complete response [CR]) by Week 25 or Cycle 7 Day 1 whichever is first

Proportion of participants who achieve an overall response (partial response \[PR\] or complete response \[CR\]) with up to 24 weeks of therapy (i.e. by the Week 25 or Cycle 7 Day 1 visit whichever is first), as defined by the National Institute of Health (NIH) Consensus response criteria

Time frame
Last participant completing 24 weeks (Week 25 visit or Cycle 7 Day 1 visit, whichever comes first) in the study

Secondary outcomes

1

Phase 1: Number of participants with treatment-emergent adverse events [TEAEs], serious TEAEs, and adverse events of special interest (AESIs)

Safety

Time frame
Up to 3 years after the last participant enrolled
2

Phase 1: Cmax

Steady-state belumosudil PK parameters

Time frame
Cycle 1 Day 15 after the last participant dosed in the phase 1 part
3

Phase 1: AUC0-6h

Steady-state belumosudil PK parameters

Time frame
Cycle 1 Day 15 after the last participant dosed in the phase 1 part
4

Phase 1: ORR

Proportion of participants who achieve an overall response (complete response \[CR\] or partial response \[PR\]) by 24 weeks, as defined by the 2014 National Institute of Health (NIH) Consensus response criteria

Time frame
last participant completing 24 weeks (Week 25 visit or Cycle 7 Day 1 visit, whichever comes first) in the study.

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Sanofi

Lead sponsor

Meiji Seika Pharma Co., Ltd.

Collaborator

Source ClinicalTrials.gov
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