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

November 10, 2023 updated by: Pharmacyclics LLC.
Dose Finding and Safety Study of Ibrutinib in Pediatric Subjects with Chronic Graft Versus Host Disease (cGVHD)

Study Overview

Status

Active, not recruiting

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

59

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

      • Randwick, Australia, 2031
        • Sydney Children's Hospital
    • New South Wales
      • Westmead, New South Wales, Australia, 2145
        • Cancer Center for Children. The Children's Hospital at Westmead
    • Queensland
      • South Brisbane, Queensland, Australia, 4101
        • Queensland Children's Hospital
    • Victoria
      • Parkville, Victoria, Australia, 3052
        • The Royal Children's Hospital
      • Wien, Austria, 1090
        • St. Anna Kinderspital
    • British Columbia
      • Vancouver, British Columbia, Canada, V6H 3V4
        • Children's and Women's Health Centre of British Columbia
    • Quebec
      • Montréal, Quebec, Canada, H3T1C5
        • CHU Sainte-Justine
      • Nantes, France, 44093
        • CHU Nantes - Hopital Enfant Adolescent
      • Paris, France, 75019
        • Hôpital Robert-Debré Ap-Hp
      • Berlin, Germany, 13353
        • Charite-Universitaetsmedizin Berlin
      • Jerusalem, Israel, 9112001
        • Hadassah Medical Centre
      • Petach Tikva, Israel, 4920235
        • Schneider Children's Medical Center in Israel
      • Ramat Gan, Israel, 5262100
        • The Edmond and Lily Safra Children's Hospital
      • Monza, Italy, 20900
        • Fondazione MBBM-Clinica Pediatrica
      • Roma, Italy, 00165
        • Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica
      • Turin, Italy, 10126
        • S.C. Farmacia Pediatrica - Presidio Ospedaliero Infantile Regina Margherita
    • PV
      • Pavia, PV, Italy, 27100
        • U.O.C. Ematologia Oncoematologia Pediatrica
      • Seoul, Korea, Republic of, 03080
        • Seoul National University Hospital
      • Seoul, Korea, Republic of, 05505
        • Asan Medical Center
      • Seoul, Korea, Republic of, 06351
        • Samsung Medical Center
      • Seoul, Korea, Republic of, 06591
        • Seoul Saint Mary's Hospital
      • Utrecht, Netherlands, 3584 CS
        • Princess Máxima Center
      • Moscow, Russian Federation, 117997
        • Federal State Budgetary Institution "Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology
      • Saint Petersburg, Russian Federation, 197022
        • Federal State Budgetary Educational Institution of Higher Professional Education "LP.Pavlov First Saint Petersburg State Medical
      • Barcelona, Spain, 08035
        • Hospital Universitario Vall d'Hebron
      • Madrid, Spain, 28009
        • Hospital Infantil Universitario Niño Jesús
      • Madrid, Spain, 28046
        • Hospital Universitario Universitario La Paz
    • Greater London
      • London, Greater London, United Kingdom, WC1N 3JH
        • Safari Day Care, Great Ormond Street Hospital
    • California
      • Duarte, California, United States, 91010
        • City of Hope
      • San Diego, California, United States, 92123
        • Rady Children's Hospital
      • San Francisco, California, United States, 94158
        • University of California
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Children's Hospital
    • District of Columbia
      • Washington, District of Columbia, United States, 20010
        • Children's National Medical Center
    • Florida
      • Saint Petersburg, Florida, United States, 33701
        • Johns Hopkins All Children's Hospital
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Ann & Robert H Lurie Children's Hospital of Chicago
    • Maryland
      • Baltimore, Maryland, United States, 21287
        • Johns Hopkins University
    • Massachusetts
      • Boston, Massachusetts, United States, 02215
        • Dana-Farber Cancer Institute
    • Minnesota
      • Minneapolis, Minnesota, United States, 55454
        • University of Minnesota Masonic Children's Hospital
    • Missouri
      • Saint Louis, Missouri, United States, 63110
        • Washington University
    • New Jersey
      • Hackensack, New Jersey, United States, 07601
        • Hackensack University Medical Center
    • New York
      • New York, New York, United States, 10065
        • Memorial Sloan Kettering Cancer Center
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Cincinnati Children's Hospital Medical Center
    • Oregon
      • Portland, Oregon, United States, 97239
        • Oregon Health and Science University
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • The Children's Hospital of Philadelphia
    • Tennessee
      • Memphis, Tennessee, United States, 38105-2729
        • St. Jude Children's Research Hospital
    • Utah
      • Salt Lake City, Utah, United States, 84113
        • Primary Children's Hospital
    • Washington
      • Seattle, Washington, United States, 98109
        • Fred Hutchinson Cancer Research Center
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53226
        • Medical College Of Wisconsin

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 year to 21 years (Child, Adult)

Accepts Healthy Volunteers

No

Description

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)

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

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
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:
  • PCI-32765
  • IMBRUVICA®

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Part A- PK (measured by AUC) will be reported descriptively
Time Frame: Approximately 24 months
Approximately 24 months
Part B- PK (measured by AUC) will be reported descriptively
Time Frame: Approximately 7 years
Approximately 7 years
Number of patients with adverse events as a measure of safety and tolerability
Time Frame: Approximately 7 years
Approximately 7 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Part A- Number of patients with adverse events as a measure of safety and tolerability
Time Frame: Approximately 24 months
Approximately 24 months
Part A- Pharmacodynamic effects as measured by in vitro BTK occupancy will be reported descriptively
Time Frame: Approximately 24 months
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
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
Up to 48 weeks
Part A continuation cohort and Part B-Overall survival (OS)
Time Frame: Approximately 5 years after last subject enrolled
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
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
Up to 5 years post enrollment
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
Up to 5 years post enrollment
Available immune reconstitution laboratory parameters will be reported descriptively
Time Frame: Up to 5 years post enrollment
Subjects will be monitored for immune reconstitution
Up to 5 years post enrollment
Late effects (Adverse events suspected to be related to treatment) will be quantified and reported descriptively
Time Frame: Up to 5 years post enrollment
Up to 5 years post enrollment

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Investigators

  • Study Director: Gauri Sunkersett, Pharmacyclics LLC.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

November 19, 2018

Primary Completion (Estimated)

January 4, 2026

Study Completion (Estimated)

January 4, 2026

Study Registration Dates

First Submitted

December 12, 2018

First Submitted That Met QC Criteria

December 27, 2018

First Posted (Actual)

December 31, 2018

Study Record Updates

Last Update Posted (Actual)

November 14, 2023

Last Update Submitted That Met QC Criteria

November 10, 2023

Last Verified

November 1, 2023

More Information

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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