Phase 1/2 Dose Finding and Safety Study of Ibrutinib in Pediatric Subjects With Chronic Graft Versus Host Disease (cGVHD)
Purpose
Dose Finding and Safety Study of Ibrutinib in Pediatric Subjects with Chronic Graft Versus Host Disease (cGVHD)
Condition
- Chronic Graft Versus Host Disease
Eligibility
- Eligible Ages
- Between 1 Year and 21 Years
- Eligible Genders
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- 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
- 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)
Study Design
- Phase
- Phase 1/Phase 2
- Study Type
- Interventional
- Allocation
- N/A
- Intervention Model
- Single Group Assignment
- Primary Purpose
- Treatment
- Masking
- None (Open Label)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental Phase 1/2 |
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. |
|
Recruiting Locations
More Details
- NCT ID
- NCT03790332
- Status
- Active, not recruiting
- Sponsor
- Pharmacyclics LLC.