Efficacy and Safety of BN101 in Subjects With Chronic Graft Versus Host Disease (cGVHD)

Study Overview
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Brief Summary
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This is a phase 2, open-label, multicenter trial to evaluate the efficacy and safety of BN101 in subjects with Chronic Graft Versus Host Disease (cGVHD) after at least First Line of systemic therapy.

Detailed Description
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Approximately 30 subjects will be enrolled to receive orally administered BN101 200 mg QD (once daily) Study drug will be administered in 28-day cycles until disease progression or unacceptable toxicity. Subjects may receive study drug in the inpatient or outpatient setting. Curative Effect analysis The efficacy was analyzed based on MITT The point estimate and 95%CI of ORR were calculated based on the exact probability method of binomial distribution.If applicable, a logistic regression model will be used for multivariate analysis. Descriptive statistical analyses were provided for all secondary efficacy endpoints. The following subgroups will be analysed: Severe cGVHD (Yes/No) Number of organs involved (<4 vs. ≥4) Number of previous systemic cGVHD treatment (1 vs. ≥2) Duration of cGVHD before inclusion (i.e., from the time of cGVHD diagnosis to the time of inclusion) Lung Involvement (Yes/No)

Official Title
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A Phase 2, Multicenter Study to Evaluate the Efficacy and Safety of BN101 in Subject With Chronic Graft Versus Host Disease (cGVHD) After at Least Fist Line of Systemic Therapy

Conditions
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GVHD, Chronic

Intervention / Treatment
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* Drug: BN101


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Male and female subjects at least 18 years of age who have had allogenic hematopoietic cell transplant (HCT). Previously received at least 1 and not more than 5 lines of systemic therapy for cGVHD Receiving glucocorticoid therapy with a stable dose over the 2 weeks prior to screening; Have persistent cGVHD manifestations and systemic therapy is indicated Exclusion Criteria: Subject has not been on a stable dose / regimen of systemic cGVHD treatments for at least 2 weeks prior to screening. (Note: Concomitant corticosteroids, calcineurin inhibitors, sirolimus, MMF, methotrexate, rituximab, and extracorporeal photophoresis (ECP) are acceptable. Systemic investigational GVHD treatments are not permitted). Histological relapse of the underlying cancer or post-transplant lymphoproliferative disease at the time of screening. Current treatment with ibrutinib. Prior treatment with ibrutinib is allowed with a washout of at least 28 days prior to treatment.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: 200mg qd<br>200mg qd po. | Drug: BN101<br>* BN101 is an orally ROCK2 selective inhibitor<br>* Other names: KD025;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Overall Response Rate (ORR) | The primary endpoint is the ORR with responses as defined by the 2014 National Institute of Health (NIH) Consensus Development Project on clinical trials in cGVHD. | 12 Months |