Chronic Graft Versus Host Disease
Conditions
Keywords
cGVHD, chronic graft versus host disease, PCYC1146IM, Pediatric, GVHD, chronic, 1146, Pharmacyclics, PCYC, Imbruvica, Ibrutinib, Immunology, graft versus host disease, corticosteroids, prednisone, PCI32765, PCYC1146, refractory, new onset graft versus host disease, refractory graft versus host disease, moderate cGVHD, severe cGVHD, moderate chronic graft versus host disease, severe chronic graft versus host disease
Brief summary
Dose Finding and Safety Study of Ibrutinib in Pediatric Subjects with Chronic Graft Versus Host Disease (cGVHD)
Interventions
DRUGIbrutinib
Ibrutinib capsule, tablet, or suspension administered orally once daily
Sponsors
Janssen Research & Development, LLC
Pharmacyclics LLC.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
1 Years to 21 Years
Healthy volunteers
No
Inclusion 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)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Part A- PK (measured by AUC) will be reported descriptively | Approximately 24 months |
| Part B- PK (measured by AUC) will be reported descriptively | Approximately 7 years |
| Number of patients with adverse events as a measure of safety and tolerability | Approximately 7 years |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Part A- Number of patients with adverse events as a measure of safety and tolerability | Approximately 24 months | — |
| Part A- Pharmacodynamic effects as measured by in vitro BTK occupancy will be reported descriptively | Approximately 24 months | — |
| Part A continuation cohort and Part B-Response rate at 24 weeks | Approximately 6 months after last subject in enrolled | — |
| Part A continuation cohort and Part B-Late Effects Surveillance | Up to 5 years post enrollment | — |
| Growth Parameter weight in kilograms will be reported descriptively. | Up to 5 years post enrollment | Subjects will be monitored for growth and development |
| Available immune reconstitution laboratory parameters will be reported descriptively | Up to 5 years post enrollment | Subjects will be monitored for immune reconstitution |
| Late effects (Adverse events suspected to be related to treatment) will be quantified and reported descriptively | Up to 5 years post enrollment | — |
| Part A continuation cohort and Part B- Duration of response (DOR) | Up to 48 weeks | — |
| Growth Parameter height in meters will be reported descriptively | Up to 5 years post enrollment | Subjects will be monitored for growth and development |
| Part A continuation cohort and Part B-Overall survival (OS) | Approximately 5 years after last subject enrolled | — |
Countries
Australia, Austria, Canada, France, Germany, Israel, Italy, Netherlands, Russia, South Korea, Spain, United Kingdom, United States
Outcome results
None listed