Clinical trial NCT04930562: Efficacy and Safety of BN101 in Subjects With Chronic Graft Versus Host Disease (cGVHD)
Official title: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: GVHD, Chronic
Lead sponsor: BioNova Pharmaceuticals (Shanghai) LTD.
Sponsor class: INDUSTRY
First posted: 2021-06-18
Enrollment: 30
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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: 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)

Eligibility criteria: 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.

Primary outcomes:
measure: Overall Response Rate (ORR)
timeFrame: 12 Months
Secondary outcomes:
measure: Duration of Response (DOR)
timeFrame: 12 Months
measure: Time-to-Response (TTR)
timeFrame: 12 months
measure: Number of Participants With Best Response in Each Individual Organ
timeFrame: 12 months
measure: Number of Participants With Change From Baseline in Overall Score on Lee cGvHD Symptom Scale at Specified Time Points
timeFrame: Baseline and 12 months
measure: Failure-free Survival (FFS)
timeFrame: 12 months
measure: Time to Next Therapy (TTNT)
timeFrame: 12months
measure: Overall Survival (OS)
timeFrame: 12months
measure: Change From Baseline in Corticosteroids Dose
timeFrame: 12months
measure: Number of Participants With Change From Baseline in Calcineurin Inhibitor (CNI) Usage.
timeFrame: 12months
measure: Change From Baseline in in Global Severity Rating (GSR) Score by Clinician-reported cGVHD Assessment
timeFrame: 12months
measure: Change From Baseline in Symptom Activity by cGVHD Activity Assessment Participant Self-Report
timeFrame: 12months
Other outcomes:
Locations:
city: Beijing
country: China
facility: Peking University People's Hospital
city: Chongqing
country: China
facility: Xinqiao Hospital, Army Medical University
city: Guangzhou
country: China
facility: Guangzhou First People's Hospital
city: Guangzhou
country: China
facility: Nanfang Hospital
city: Guangzhou
country: China
facility: Zhujiang Hospital of Southern Medical University
city: Suzhou
country: China
facility: The First Affiliated Hospital of Soochow University
city: Xuzhou
country: China
facility: The Affiliated Hospital of Xuzhou Medical University
Study officials:
affiliation: The First Affiliated Hospital of Soochow University
name: Depei Wu, Prof
role: PRINCIPAL_INVESTIGATOR
References: