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

NCT ID: NCT03790332

Last Updated: 2025-10-21

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

59 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-11-19

Study Completion Date

2025-10-09

Brief Summary

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Dose Finding and Safety Study of Ibrutinib in Pediatric Subjects with Chronic Graft Versus Host Disease (cGVHD)

Detailed Description

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Conditions

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Chronic Graft Versus Host Disease

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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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.

Group Type EXPERIMENTAL

Ibrutinib

Intervention Type DRUG

Ibrutinib capsule, tablet, or suspension administered orally once daily

Interventions

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Ibrutinib

Ibrutinib capsule, tablet, or suspension administered orally once daily

Intervention Type DRUG

Other Intervention Names

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IMBRUVICA® PCI-32765

Eligibility Criteria

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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)
Minimum Eligible Age

1 Year

Maximum Eligible Age

21 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Janssen Research & Development, LLC

INDUSTRY

Sponsor Role collaborator

Pharmacyclics LLC.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Gauri Sunkersett

Role: STUDY_DIRECTOR

Pharmacyclics LLC.

Locations

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City of Hope

Duarte, California, United States

Site Status

Rady Children's Hospital

San Diego, California, United States

Site Status

University of California

San Francisco, California, United States

Site Status

Children's Hospital

Aurora, Colorado, United States

Site Status

Children's National Medical Center

Washington D.C., District of Columbia, United States

Site Status

Johns Hopkins All Children's Hospital

St. Petersburg, Florida, United States

Site Status

Ann & Robert H Lurie Children's Hospital of Chicago

Chicago, Illinois, United States

Site Status

Johns Hopkins University

Baltimore, Maryland, United States

Site Status

Dana-Farber Cancer Institute

Boston, Massachusetts, United States

Site Status

University of Minnesota Masonic Children's Hospital

Minneapolis, Minnesota, United States

Site Status

Washington University

St Louis, Missouri, United States

Site Status

Hackensack University Medical Center

Hackensack, New Jersey, United States

Site Status

Memorial Sloan Kettering Cancer Center

New York, New York, United States

Site Status

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Site Status

Oregon Health and Science University

Portland, Oregon, United States

Site Status

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status

St. Jude Children's Research Hospital

Memphis, Tennessee, United States

Site Status

Primary Children's Hospital

Salt Lake City, Utah, United States

Site Status

Fred Hutchinson Cancer Research Center

Seattle, Washington, United States

Site Status

Medical College of Wisconsin

Milwaukee, Wisconsin, United States

Site Status

Cancer Center for Children. The Children's Hospital at Westmead

Westmead, New South Wales, Australia

Site Status

Queensland Children's Hospital

South Brisbane, Queensland, Australia

Site Status

The Royal Children's Hospital

Parkville, Victoria, Australia

Site Status

Sydney Children's Hospital

Randwick, , Australia

Site Status

St. Anna Kinderspital

Vienna, , Austria

Site Status

children's and Women's Health Centre of British Columbia

Vancouver, British Columbia, Canada

Site Status

CHU Sainte-Justine

Montreal, Quebec, Canada

Site Status

CHU Nantes - Hopital Enfant Adolescent

Nantes, , France

Site Status

Hôpital Robert-Debré Ap-Hp

Paris, , France

Site Status

Charite-Universitaetsmedizin Berlin

Berlin, , Germany

Site Status

Hadassah Medical Centre

Jerusalem, , Israel

Site Status

Schneider Children's Medical Center in Israel

Petah Tikva, , Israel

Site Status

The Edmond and Lily Safra Children's Hospital

Ramat Gan, , Israel

Site Status

U.O.C. Ematologia Oncoematologia Pediatrica

Pavia, PV, Italy

Site Status

Fondazione MBBM-Clinica Pediatrica

Monza, , Italy

Site Status

Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica

Roma, , Italy

Site Status

S.C. Farmacia Pediatrica - Presidio Ospedaliero Infantile Regina Margherita

Turin, , Italy

Site Status

Princess Maxima Center

Utrecht, , Netherlands

Site Status

Federal State Budgetary Institution "Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology

Moscow, , Russia

Site Status

Federal State Budgetary Educational Institution of Higher Professional Education "LP.Pavlov First Saint Petersburg State Medical

Saint Petersburg, , Russia

Site Status

Seoul National University Hospital

Seoul, , South Korea

Site Status

Asan Medical Center

Seoul, , South Korea

Site Status

Samsung Medical Center

Seoul, , South Korea

Site Status

Seoul Saint Mary's Hospital

Seoul, , South Korea

Site Status

Hospital Universitario Vall d'Hebron

Barcelona, , Spain

Site Status

Hospital Infantil Universitario Nino Jesus

Madrid, , Spain

Site Status

Hospital Universitario Universitario La Paz

Madrid, , Spain

Site Status

Safari Day Care, Great Ormond Street Hospital

London, Greater London, United Kingdom

Site Status

Countries

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United States Australia Austria Canada France Germany Israel Italy Netherlands Russia South Korea Spain United Kingdom

Other Identifiers

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2017-004558-41

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

PCYC-1146-IM

Identifier Type: -

Identifier Source: org_study_id

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