Phase 1/2 Dose Finding and Safety Study of Ibrutinib in Pediatric Subjects With Chronic Graft Versus Host Disease (cGVHD)

Pharmacyclics

Status and phase: Completed; Phase 2; Phase 1

Conditions: Chronic Graft Versus Host Disease

Treatments: Drug: Ibrutinib

Study type: Interventional

Funder types: Industry

Identifiers

NCT03790332

PCYC-1146-IM

2017-004558-41 (EudraCT Number)

Details and patient eligibility

About

Dose Finding and Safety Study of Ibrutinib in Pediatric Subjects with Chronic Graft Versus Host Disease (cGVHD)

Enrollment: 59 patients

Sex: All

Ages: 1 to 21 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Eligibility:

Inclusion Criteria:

1. Part A: Subjects with moderate or severe cGVHD after failure of 1 or more lines of systemic therapy

2. Part B: Subjects with moderate or severe cGVHD after failure of 1 or more lines of systemic therapy, or subjects with new onset moderate or severe cGVHD and in need of systemic immunosuppression

3. History of allogeneic stem cell transplantation

4. Age

   • Part A: ≥1 to <12 years of age at the time of enrollment
   • Part B: ≥1 to <22 years of age at the time of enrollment

5. Karnofsky or Lansky (subjects <16 years of age) performance status ≥60

Key Eligibility:

Exclusion Criteria:

1. Presence of single organ genito-urinary involvement as the only manifestation of cGVHD
2. Received an investigational agent within 28 days before enrollment.
3. Received donor lymphocyte infusion (DLI) within 56 days before enrollment
4. Progressive underlying malignant disease or active post-transplant lymphoproliferative disease
5. Any uncontrolled infection or active infection requiring ongoing systemic treatment
6. Known bleeding disorders
7. Active hepatitis C virus (HCV) or hepatitis B virus (HBV)

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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59 participants in 1 patient group

Phase 1/2

Experimental group

Description:

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.

Treatment:

Drug: Ibrutinib