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

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ClinicalTrials.gov Identifier: NCT03790332
Recruitment Status : Active, not recruiting
First Posted : December 31, 2018
Last Update Posted : August 13, 2021
Sponsor:
Collaborator:
Janssen Research & Development, LLC
Information provided by (Responsible Party):
Pharmacyclics LLC.

Tracking Information
First Submitted Date  ICMJE December 12, 2018
First Posted Date  ICMJE December 31, 2018
Last Update Posted Date August 13, 2021
Actual Study Start Date  ICMJE November 19, 2018
Estimated Primary Completion Date January 4, 2026   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: December 27, 2018)
  • 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 ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: December 27, 2018)
  • 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 ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Phase 1/2 Dose Finding and Safety Study of Ibrutinib in Pediatric Subjects With Chronic Graft Versus Host Disease (cGVHD)
Official Title  ICMJE Phase 1/2 Dose Finding and Safety Study of Ibrutinib in Pediatric Subjects With Chronic Graft Versus Host Disease (cGVHD)
Brief Summary Dose Finding and Safety Study of Ibrutinib in Pediatric Subjects with Chronic Graft Versus Host Disease (cGVHD)
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Chronic Graft Versus Host Disease
Intervention  ICMJE Drug: Ibrutinib
Ibrutinib capsule, tablet, or suspension administered orally once daily
Other Names:
  • IMBRUVICA®
  • PCI-32765
Study Arms  ICMJE 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.

Intervention: Drug: Ibrutinib
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Actual Enrollment  ICMJE
 (submitted: August 12, 2021)
58
Original Estimated Enrollment  ICMJE
 (submitted: December 27, 2018)
44
Estimated Study Completion Date  ICMJE January 4, 2026
Estimated Primary Completion Date January 4, 2026   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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)
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 1 Year to 21 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Australia,   Austria,   Canada,   France,   Germany,   Israel,   Italy,   Korea, Republic of,   Netherlands,   Russian Federation,   Spain,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03790332
Other Study ID Numbers  ICMJE PCYC-1146-IM
2017-004558-41 ( EudraCT Number )
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Pharmacyclics LLC.
Study Sponsor  ICMJE Pharmacyclics LLC.
Collaborators  ICMJE Janssen Research & Development, LLC
Investigators  ICMJE
Study Director: Justin Wahlstrom, MD Pharmacyclics LLC.
PRS Account Pharmacyclics LLC.
Verification Date August 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP