Phase 1/2 Dose Finding and Safety Study of Ibrutinib in Pediatric Subjects With Chronic Graft Versus Host Disease (cGVHD)
|
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
| ClinicalTrials.gov Identifier: NCT03790332 |
|
Recruitment Status :
Active, not recruiting
First Posted : December 31, 2018
Last Update Posted : February 8, 2022
|
Sponsor:
Pharmacyclics LLC.
Collaborator:
Janssen Research & Development, LLC
Information provided by (Responsible Party):
Pharmacyclics LLC.
- Study Details
- Tabular View
- No Results Posted
- Disclaimer
- How to Read a Study Record
Brief Summary:
Dose Finding and Safety Study of Ibrutinib in Pediatric Subjects with Chronic Graft Versus Host Disease (cGVHD)
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Chronic Graft Versus Host Disease | Drug: Ibrutinib | Phase 1 Phase 2 |
| Study Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 59 participants |
| Allocation: | N/A |
| Intervention Model: | Single Group Assignment |
| Masking: | None (Open Label) |
| Primary Purpose: | Treatment |
| Official Title: | Phase 1/2 Dose Finding and Safety Study of Ibrutinib in Pediatric Subjects With Chronic Graft Versus Host Disease (cGVHD) |
| Actual Study Start Date : | November 19, 2018 |
| Estimated Primary Completion Date : | January 4, 2026 |
| Estimated Study Completion Date : | January 4, 2026 |
Resource links provided by the National Library of Medicine
Drug Information available for:
Ibrutinib
| Arm | Intervention/treatment |
|---|---|
|
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. |
Drug: Ibrutinib
Ibrutinib capsule, tablet, or suspension administered orally once daily
Other Names:
|
Primary Outcome Measures :
- Part A- PK (measured by AUC) will be reported descriptively [ Time Frame: Approximately 24 months ]
- Part B- PK (measured by AUC) will be reported descriptively [ Time Frame: Approximately 7 years ]
- Number of patients with adverse events as a measure of safety and tolerability [ Time Frame: Approximately 7 years ]
Secondary Outcome Measures :
- Part A- Number of patients with adverse events as a measure of safety and tolerability [ Time Frame: Approximately 24 months ]
- Part A- Pharmacodynamic effects as measured by in vitro BTK occupancy will be reported descriptively [ Time Frame: Approximately 24 months ]
- Part A continuation cohort and Part B-Response rate at 24 weeks [ Time Frame: Approximately 6 months after last subject in enrolled ]
- Part A continuation cohort and Part B- Duration of response (DOR) [ Time Frame: Up to 48 weeks ]
- Part A continuation cohort and Part B-Overall survival (OS) [ Time Frame: Approximately 5 years after last subject enrolled ]
- Part A continuation cohort and Part B-Late Effects Surveillance [ Time Frame: Up to 5 years post enrollment ]
- Growth Parameter height in meters will be reported descriptively [ Time Frame: Up to 5 years post enrollment ]Subjects will be monitored for growth and development
- Growth Parameter weight in kilograms will be reported descriptively. [ Time Frame: Up to 5 years post enrollment ]Subjects will be monitored for growth and development
- Available immune reconstitution laboratory parameters will be reported descriptively [ Time Frame: 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 [ Time Frame: Up to 5 years post enrollment ]
Information from the National Library of Medicine
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
| Ages Eligible for Study: | 1 Year to 21 Years (Child, Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Criteria
Key Eligibility:
Inclusion Criteria:
- Part A: Subjects with moderate or severe cGVHD after failure of 1 or more lines of systemic therapy
- 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
- History of allogeneic stem cell transplantation
-
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
- 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
- Received an investigational agent within 28 days before enrollment.
- Received donor lymphocyte infusion (DLI) within 56 days before enrollment
- Progressive underlying malignant disease or active post-transplant lymphoproliferative disease
- Any uncontrolled infection or active infection requiring ongoing systemic treatment
- Known bleeding disorders
- Active hepatitis C virus (HCV) or hepatitis B virus (HBV)
Information from the National Library of Medicine
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03790332
Locations
Show 48 study locations
Sponsors and Collaborators
Pharmacyclics LLC.
Janssen Research & Development, LLC
Investigators
| Study Director: | Justin Wahlstrom, MD | Pharmacyclics LLC. |
| Responsible Party: | Pharmacyclics LLC. |
| ClinicalTrials.gov Identifier: | NCT03790332 |
| Other Study ID Numbers: |
PCYC-1146-IM 2017-004558-41 ( EudraCT Number ) |
| First Posted: | December 31, 2018 Key Record Dates |
| Last Update Posted: | February 8, 2022 |
| Last Verified: | February 2022 |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
Keywords provided by Pharmacyclics LLC.:
|
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 |
Additional relevant MeSH terms:
|
Graft vs Host Disease Immune System Diseases |