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

BioNova Pharmaceuticals

Status and phase

Completed

Phase 2

Conditions

GVHD, Chronic

Treatments

Drug: BN101

Study type

Interventional

Funder types

Industry

Identifiers

NCT04930562

BN101-201

Details and patient eligibility

About

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.

Full 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)

Enrollment

30 patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

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.