- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03790332
Phase 1/2 Dose Finding and Safety Study of Ibrutinib in Pediatric Subjects With Chronic Graft Versus Host Disease (cGVHD)
Study Overview
Status
Conditions
Intervention / Treatment
Study Type
Enrollment (Actual)
Phase
- Phase 2
- Phase 1
Contacts and Locations
Study Locations
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Randwick, Australia, 2031
- Sydney Children's Hospital
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New South Wales
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Westmead, New South Wales, Australia, 2145
- Cancer Center for Children. The Children's Hospital at Westmead
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Queensland
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South Brisbane, Queensland, Australia, 4101
- Queensland Children's Hospital
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Victoria
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Parkville, Victoria, Australia, 3052
- The Royal Children's Hospital
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Wien, Austria, 1090
- St. Anna Kinderspital
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British Columbia
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Vancouver, British Columbia, Canada, V6H 3V4
- Children's and Women's Health Centre of British Columbia
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Quebec
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Montréal, Quebec, Canada, H3T1C5
- CHU Sainte-Justine
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Nantes, France, 44093
- CHU Nantes - Hopital Enfant Adolescent
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Paris, France, 75019
- Hôpital Robert-Debré Ap-Hp
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Berlin, Germany, 13353
- Charite-Universitaetsmedizin Berlin
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Jerusalem, Israel, 9112001
- Hadassah Medical Centre
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Petach Tikva, Israel, 4920235
- Schneider Children's Medical Center in Israel
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Ramat Gan, Israel, 5262100
- The Edmond and Lily Safra Children's Hospital
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Monza, Italy, 20900
- Fondazione MBBM-Clinica Pediatrica
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Roma, Italy, 00165
- Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica
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Turin, Italy, 10126
- S.C. Farmacia Pediatrica - Presidio Ospedaliero Infantile Regina Margherita
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PV
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Pavia, PV, Italy, 27100
- U.O.C. Ematologia Oncoematologia Pediatrica
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Seoul, Korea, Republic of, 03080
- Seoul National University Hospital
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Seoul, Korea, Republic of, 05505
- Asan Medical Center
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Seoul, Korea, Republic of, 06351
- Samsung Medical Center
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Seoul, Korea, Republic of, 06591
- Seoul Saint Mary's Hospital
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Utrecht, Netherlands, 3584 CS
- Princess Máxima Center
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Moscow, Russian Federation, 117997
- Federal State Budgetary Institution "Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology
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Saint Petersburg, Russian Federation, 197022
- Federal State Budgetary Educational Institution of Higher Professional Education "LP.Pavlov First Saint Petersburg State Medical
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Barcelona, Spain, 08035
- Hospital Universitario Vall d'Hebron
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Madrid, Spain, 28009
- Hospital Infantil Universitario Niño Jesús
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Madrid, Spain, 28046
- Hospital Universitario Universitario La Paz
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Greater London
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London, Greater London, United Kingdom, WC1N 3JH
- Safari Day Care, Great Ormond Street Hospital
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California
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Duarte, California, United States, 91010
- City of Hope
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San Diego, California, United States, 92123
- Rady Children's Hospital
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San Francisco, California, United States, 94158
- University of California
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Colorado
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Aurora, Colorado, United States, 80045
- Children's Hospital
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District of Columbia
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Washington, District of Columbia, United States, 20010
- Children's National Medical Center
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Florida
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Saint Petersburg, Florida, United States, 33701
- Johns Hopkins All Children's Hospital
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Illinois
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Chicago, Illinois, United States, 60611
- Ann & Robert H Lurie Children's Hospital of Chicago
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Maryland
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Baltimore, Maryland, United States, 21287
- Johns Hopkins University
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Massachusetts
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Boston, Massachusetts, United States, 02215
- Dana-Farber Cancer Institute
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Minnesota
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Minneapolis, Minnesota, United States, 55454
- University of Minnesota Masonic Children's Hospital
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Missouri
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Saint Louis, Missouri, United States, 63110
- Washington University
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New Jersey
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Hackensack, New Jersey, United States, 07601
- Hackensack University Medical Center
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New York
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New York, New York, United States, 10065
- Memorial Sloan Kettering Cancer Center
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Ohio
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Cincinnati, Ohio, United States, 45229
- Cincinnati Children's Hospital Medical Center
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Oregon
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Portland, Oregon, United States, 97239
- Oregon Health and Science University
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Pennsylvania
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Philadelphia, Pennsylvania, United States, 19104
- The Children's Hospital of Philadelphia
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Tennessee
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Memphis, Tennessee, United States, 38105-2729
- St. Jude Children's Research Hospital
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Utah
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Salt Lake City, Utah, United States, 84113
- Primary Children's Hospital
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Washington
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Seattle, Washington, United States, 98109
- Fred Hutchinson Cancer Research Center
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Wisconsin
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Milwaukee, Wisconsin, United States, 53226
- Medical College Of Wisconsin
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Description
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)
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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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. |
Ibrutinib capsule, tablet, or suspension administered orally once daily
Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
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Part A- PK (measured by AUC) will be reported descriptively
Time Frame: Approximately 24 months
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Approximately 24 months
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Part B- PK (measured by AUC) will be reported descriptively
Time Frame: Approximately 7 years
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Approximately 7 years
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Number of patients with adverse events as a measure of safety and tolerability
Time Frame: Approximately 7 years
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Approximately 7 years
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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Part A- Number of patients with adverse events as a measure of safety and tolerability
Time Frame: Approximately 24 months
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Approximately 24 months
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Part A- Pharmacodynamic effects as measured by in vitro BTK occupancy will be reported descriptively
Time Frame: Approximately 24 months
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Approximately 24 months
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Part A continuation cohort and Part B-Response rate at 24 weeks
Time Frame: Approximately 6 months after last subject in enrolled
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Approximately 6 months after last subject in enrolled
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Part A continuation cohort and Part B- Duration of response (DOR)
Time Frame: Up to 48 weeks
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Up to 48 weeks
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Part A continuation cohort and Part B-Overall survival (OS)
Time Frame: Approximately 5 years after last subject enrolled
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Approximately 5 years after last subject enrolled
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Part A continuation cohort and Part B-Late Effects Surveillance
Time Frame: Up to 5 years post enrollment
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Up to 5 years post enrollment
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Growth Parameter height in meters will be reported descriptively
Time Frame: Up to 5 years post enrollment
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Subjects will be monitored for growth and development
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Up to 5 years post enrollment
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Growth Parameter weight in kilograms will be reported descriptively.
Time Frame: Up to 5 years post enrollment
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Subjects will be monitored for growth and development
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Up to 5 years post enrollment
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Available immune reconstitution laboratory parameters will be reported descriptively
Time Frame: Up to 5 years post enrollment
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Subjects will be monitored for immune reconstitution
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Up to 5 years post enrollment
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Late effects (Adverse events suspected to be related to treatment) will be quantified and reported descriptively
Time Frame: Up to 5 years post enrollment
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Up to 5 years post enrollment
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Collaborators and Investigators
Sponsor
Collaborators
Investigators
- Study Director: Gauri Sunkersett, Pharmacyclics LLC.
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Estimated)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
- Immunology
- Pediatric
- chronic
- Pharmacyclics
- PCYC
- Ibrutinib
- refractory
- GVHD
- cGVHD
- corticosteroids
- prednisone
- graft versus host disease
- Imbruvica
- chronic graft versus host disease
- PCI32765
- new onset graft versus host disease
- PCYC1146IM
- 1146
- PCYC1146
- 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
Other Study ID Numbers
- PCYC-1146-IM
- 2017-004558-41 (EudraCT Number)
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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