Ibrutinib for Graft-versus-Host Disease

Recruiting in Palo Alto (17 mi)

+47 other locations

Age: < 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Waitlist Available

Sponsor: Pharmacyclics LLC.

No Placebo Group

Breakthrough Therapy

Approved in 4 Jurisdictions

Trial Summary

What is the purpose of this trial?Dose Finding and Safety Study of Ibrutinib in Pediatric Subjects with Chronic Graft Versus Host Disease (cGVHD)

Eligibility Criteria

This trial is for children and young adults who have chronic Graft Versus Host Disease (cGVHD) after a stem cell transplant. It's open to those aged 1-12 for Part A, and 1-22 for Part B, who've tried at least one treatment without success or are newly diagnosed needing systemic therapy. Participants should be fairly active (performance status ≥60). Those with uncontrolled infections, hepatitis, only genito-urinary cGVHD, recent investigational drugs or donor lymphocyte infusions can't join.

Inclusion Criteria

I can care for myself but may need occasional help.

I have chronic GVHD that didn't improve after at least one treatment.

I have a new, severe immune reaction after a transplant needing strong medication.

+2 more

Exclusion Criteria

I have a known bleeding disorder.

My cancer is getting worse or I have a disease after a transplant.

Received an investigational agent within 28 days before enrollment

+4 more

Participant Groups

The study is testing the safety and appropriate dosing of Ibrutinib in pediatric patients with cGVHD. This phase involves finding the right dose (Phase 1) and then checking its safety profile more broadly (Phase 2).

1Treatment groups

Experimental Treatment

Group I: Phase 1/2Experimental Treatment1 Intervention

Part A: Subjects ≥1 to \<12 years of age with moderate or severe cGVHD after failure of 1 or more lines of systemic therapy, will receive oral ibrutinib once daily to determine Recommended Pediatric Equivalent Dose (RPED). Part A Continuation: Subjects participating in Part A may continue receiving daily ibrutinib until the RPED is determined, at which time their dose may be adjusted to the RPED. Part B: Subjects ≥1 to \<12 years of age( upper age limit is \< 22 years) with moderate or severe cGVHD after failure of 1 or more lines of systemic therapy or with newly diagnosed moderate or severe cGVHD will be dosed at the RPED. Subjects ≥12 will be given 420mg orally ibrutinib once daily.

Ibrutinib is already approved in European Union, United States, Canada, Japan for the following indications:

🇪🇺 Approved in European Union as Imbruvica for: