Overview

This trial is active, not recruiting.

Condition graft versus host disease
Treatment ibrutinib
Phase phase 1/phase 2
Target BTK
Sponsor Pharmacyclics
Start date July 2014
End date December 2017
Trial size 45 participants
Trial identifier NCT02195869, PCYC-1129-CA

Summary

The purpose of this study is to assess the safety and clinical efficacy of ibrutinib in subjects with steroid dependent or refractory Chronic Graft Versus Host Disease.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation non-randomized
Endpoint classification safety/efficacy study
Intervention model parallel assignment
Masking open label
Primary purpose treatment
Arm
(Experimental)
Ibrutinib PO administered daily
ibrutinib PCI32765
(Experimental)
Ibrutinib (PO) administered daily
ibrutinib PCI32765
(Experimental)
Ibrutinib (PO) administered daily
ibrutinib PCI32765
(Experimental)
Ibrutinib (PO) administered daily for 18 months
ibrutinib PCI32765

Primary Outcomes

Measure
Phase 1b: Number of dose-limiting toxicities as a measure of safety profile to determine recommended dose of ibrutinib
time frame: 28 treatment days after last subject enrolled in Phase 1 dose level(s).
Phase 2 (Efficacy): Overall cGVHD response rate defined as the proportion of evaluable subjects who achieve a [NIH-defined Complete Response (CR) and Partial Response (PR)] over all subjects who were treated with RP2D
time frame: When the last subject completes 6 months of treatment.

Secondary Outcomes

Measure
Failure Free Survival (FFS)
time frame: When the last subject completes 6 and 12 months of treatment.
Change in symptom burden by the Lee cGVHD Symptom Scale
time frame: Approximately 18 months after last subject is enrolled or up to disease progression, whichever occurs first.
Corticosteroid requirement changes over Time
time frame: Approximately 18 months after last subject is enrolled or up to disease progression, whichever occurs first.
Number of subjects with Adverse Events (AE) as a measure of safety and tolerability of ibrutinib
time frame: Approximately 18 months after last subject is enrolled.
Determine the plasma pharmacokinetics of ibrutinib and the metabolite, PCI-45227
time frame: Samples will be collected during the first two weeks of subject receiving study drug and will be assessed approximately 1 month after completion of the PK sample collections of the last subject enrolled at each dose level.

Eligibility Criteria

Male or female participants at least 18 years old.

Inclusion Criteria: - Steroid dependent or refractory classic chronic GVHD disease. - No more than 3 previous treatments for cGVHD. - Receiving baseline systemic glucocorticoid therapy (at stable dose) for cGVHD at study entry. - Men and women ≥18 years old. - Karnofsky performance status ≥60. Exclusion Criteria: - Known or suspected active acute GVHD. - Current treatment with sirolimus AND either cyclosporine or tacrolimus. - History of treatment with a tyrosine kinase inhibitor (eg, imatinib), purine analogs or other cancer chemotherapy in the 4 weeks prior to starting study drug. - Currently active, clinically significant cardiovascular disease. - Uncontrolled infections not responsive to antibiotics, antiviral medicines, or antifungal medicines or a recent infection requiring systemic treatment that was completed ≤14 days before the first dose of study drug. - Progressive underlying malignant disease including post-transplant lymphoproliferative disease. - History of other malignancy (not including the underlying malignancy that was the indication for transplant) - Concomitant use of warfarin or other Vitamin K antagonists - Known bleeding disorders or hemophilia. - History of stroke or intracranial hemorrhage within 6 months prior to enrollment. - Known history of human immunodeficiency virus (HIV) or active with hepatitis C virus (HCV) or hepatitis B virus (HBV). - Concurrent use of a strong cytochrome P450(CYP) 3A inhibitor.

Additional Information

Official title A Multicenter Open-Label Phase 1b/2 Study of Ibrutinib in Steroid Dependent or Refractory Chronic Graft Versus Host Disease
Trial information was received from ClinicalTrials.gov and was last updated in April 2016.
Information provided to ClinicalTrials.gov by Pharmacyclics.
Location data was received from the National Cancer Institute and was last updated in April 2016.