Clinical trial NCT06616415: A Clinical Study to Evaluate the Pharmacokinetics, Efficacy, and Safety of Belumosudil in Chinese Adolescents With cGVHD Who Have Had an Inadequate Response to Glucocorticoids or Other Systemic Therapies
Official title: A Multicenter, Open-label, Single-arm, Phase 4 Clinical Study to Evaluate the Pharmacokinetics, Efficacy, and Safety of Belumosudil Mesylate Tablets in Chinese Adolescents (Aged From 12 to Less Than 18 Years) With Chronic Graft-versus-host Disease (cGVHD) Who Have Had an Inadequate Response to Glucocorticoids or Other Systemic Therapies
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE4
Conditions: Chronic Graft Versus Host Disease
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
First posted: 2024-09-27
Enrollment: 6
Sex: ALL
Minimum age (years): 12
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This is a single group, Phase 4, single-arm post-marketing study for treatment.

The purpose of this study is to verify the pharmacokinetics, efficacy, and safety of belumosudil mesylate tablets in Chinese adolescent participants (aged from 12 to less than 18) with cGVHD who have had an inadequate response to glucocorticoids or other systemic therapies.

Participants will receive treatment with belumosudil tablets 200 mg once daily in 28-day cycles during the study.


Eligibility criteria: 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.

Primary outcomes:
measure: Plasma concentrations of belumosudil at specified time points
timeFrame: At Day 1 and Day 29(±3)
Secondary outcomes:
measure: Overall response rate (ORR)
timeFrame: Up to 18 month
measure: Duration of response (DoR)
timeFrame: Up to 18 month
measure: System organ response rate
timeFrame: Up to 18 month
measure: Number and proportion of participants with dose reduction in corticosteroid during the treatment period
timeFrame: Up to 18 month
measure: Failure-free survival (FFS)
timeFrame: Up to 18 month
measure: Overall survival (OS)
timeFrame: Up to 18 month
measure: Change in cGVHD severity based on physician-reported overall cGVHD activity
timeFrame: Up to 18 month
measure: Change in symptom activity assessed based on patient-reported cGVHD activity
timeFrame: Up to 18 month
measure: Modified Lee cGVHD Symptom Scale change
timeFrame: Up to 18 month
measure: Number and proportion of participants with calcineurin inhibitor (CNI) dose reduction or discontinuation during the treatment period
timeFrame: Up to 18 month
measure: Number of participants with treatment-emergent adverse events (TEAEs), serious TEAEs, and adverse events of special interest (AESIs)
timeFrame: Up to 18 month
Other outcomes:
Locations:
city: Beijing
country: China
facility: Investigational Site Number: 1560001
city: Shanghai
country: China
facility: Investigational Site Number: 1560002
Study officials:
affiliation: Sanofi
name: Clinical Sciences & Operations
role: STUDY_DIRECTOR
References: