Chronic Graft Versus Host DiseasePhase 1/2NCT03790332
Phase 1/2 Dose Finding and Safety Study of Ibrutinib in Pediatric Subjects With Chronic Graft Versus Host Disease (cGVHD)
A Phase 1/2 study testing Ibrutinib in people with Chronic Graft Versus Host Disease, run by Pharmacyclics LLC. at 48 sites in 13 countries.
Trial summary
Dose Finding and Safety Study of Ibrutinib in Pediatric Subjects with Chronic Graft Versus Host Disease (cGVHD) Read more about this study
- Who is it for?
- Ages 1 year to 21 years
- Conditions the study is for
- Chronic Graft Versus Host Disease
- What is being tested?
- Ibrutinib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 59 participants
- Where
- 48 sitesin 13 countries
- Study dates
- 19 Nov 2018to 9 Oct 2025
- Phase
- Phase 1/2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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:
Who cannot join
- 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)
Trial locations and contacts
48 sites in 13 countries.
48 sites in 13 countries
United States20 sites
Children's Hospital
Aurora, Colorado, 80045
Johns Hopkins University
Baltimore, Maryland, 21287
Dana-Farber Cancer Institute
Boston, Massachusetts, 02215
Ann & Robert H Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
City of Hope
Duarte, California, 91010
Hackensack University Medical Center
Hackensack, New Jersey, 07601
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105-2729
Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
University of Minnesota Masonic Children's Hospital
Minneapolis, Minnesota, 55454
Memorial Sloan Kettering Cancer Center
New York, New York, 10065
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Oregon Health and Science University
Portland, Oregon, 97239
Primary Children's Hospital
Salt Lake City, Utah, 84113
Rady Children's Hospital
San Diego, California, 92123
University of California
San Francisco, California, 94158
Fred Hutchinson Cancer Research Center
Seattle, Washington, 98109
Washington University
St Louis, Missouri, 63110
Johns Hopkins All Children's Hospital
St. Petersburg, Florida, 33701
Children's National Medical Center
Washington D.C., District of Columbia, 20010
Australia4 sites
The Royal Children's Hospital
Parkville, Victoria, 3052
Sydney Children's Hospital
Randwick, 2031
Queensland Children's Hospital
South Brisbane, Queensland, 4101
Cancer Center for Children. The Children's Hospital at Westmead
Westmead, New South Wales, 2145
Italy4 sites
Fondazione MBBM-Clinica Pediatrica
Monza, 20900
U.O.C. Ematologia Oncoematologia Pediatrica
Pavia, PV, 27100
Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica
Roma, 00165
S.C. Farmacia Pediatrica - Presidio Ospedaliero Infantile Regina Margherita
Turin, 10126
South Korea4 sites
Seoul National University Hospital
Seoul, 03080
Asan Medical Center
Seoul, 05505
Samsung Medical Center
Seoul, 06351
Seoul Saint Mary's Hospital
Seoul, 06591
Israel3 sites
Hadassah Medical Centre
Jerusalem, 9112001
Schneider Children's Medical Center in Israel
Petah Tikva, 4920235
The Edmond and Lily Safra Children's Hospital
Ramat Gan, 5262100
Spain3 sites
Hospital Universitario Vall d'Hebron
Barcelona, 08035
Hospital Infantil Universitario Nino Jesus
Madrid, 28009
Hospital Universitario Universitario La Paz
Madrid, 28046
Canada2 sites
CHU Sainte-Justine
Montreal, Quebec, H3T1C5
children's and Women's Health Centre of British Columbia
Vancouver, British Columbia, V6H 3V4
France2 sites
CHU Nantes - Hopital Enfant Adolescent
Nantes, 44093
Hôpital Robert-Debré Ap-Hp
Paris, 75019
Russia2 sites
Federal State Budgetary Institution "Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology
Moscow, 117997
Federal State Budgetary Educational Institution of Higher Professional Education "LP.Pavlov First Saint Petersburg State Medical
Saint Petersburg, 197022
United Kingdom1 site
Safari Day Care, Great Ormond Street Hospital
London, Greater London, WC1N 3JH
About this study
Dose Finding and Safety Study of Ibrutinib in Pediatric Subjects with Chronic Graft Versus Host Disease (cGVHD)
- Conditions
- Chronic Graft Versus Host Disease
- Being tested
- Ibrutinib
- Keywords
- 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
- Medical subject headings
- Bronchiolitis Obliterans Syndrome, Graft vs Host Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IbrutinibDrug
Also called IMBRUVICA®, PCI-32765
Ibrutinib capsule, tablet, or suspension administered orally once daily
| Study group | Role | What they receive |
|---|---|---|
| Phase 1/2 | Experimental | 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. Receives: Drug: Ibrutinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Part A- PK (measured by AUC) will be reported descriptively
Measured: Approximately 24 months
Part B- PK (measured by AUC) will be reported descriptively
Measured: Approximately 7 years
Number of patients with adverse events as a measure of safety and tolerability
Measured: Approximately 7 years
Secondary outcomes (10)
Part A- Number of patients with adverse events as a measure of safety and tolerability
Measured: Approximately 24 months
Part A- Pharmacodynamic effects as measured by in vitro BTK occupancy will be reported descriptively
Measured: Approximately 24 months
Part A continuation cohort and Part B-Response rate at 24 weeks
Measured: Approximately 6 months after last subject in enrolled
Part A continuation cohort and Part B- Duration of response (DOR)
Measured: Up to 48 weeks
Part A continuation cohort and Part B-Overall survival (OS)
Measured: Approximately 5 years after last subject enrolled
Part A continuation cohort and Part B-Late Effects Surveillance
Measured: Up to 5 years post enrollment
Growth Parameter height in meters will be reported descriptively
Subjects will be monitored for growth and development
Measured: Up to 5 years post enrollment
Growth Parameter weight in kilograms will be reported descriptively.
Subjects will be monitored for growth and development
Measured: Up to 5 years post enrollment
Available immune reconstitution laboratory parameters will be reported descriptively
Subjects will be monitored for immune reconstitution
Measured: Up to 5 years post enrollment
Late effects (Adverse events suspected to be related to treatment) will be quantified and reported descriptively
Measured: Up to 5 years post enrollment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Dec 2018
- Study start19 Nov 2018
- Primary completion9 Oct 2025
- Study completion9 Oct 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Chronic Graft Versus Host DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 59 participants (actual)
Sponsor and investigators
- Lead sponsor
- Pharmacyclics LLC.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Janssen Research & Development, LLC
- Responsible party
- Sponsor
- Investigators
- Gauri Sunkersett, Study director, Pharmacyclics LLC.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03790332
- Sponsor's protocol ID
- PCYC-1146-IM
- Other IDs
- 2017-004558-41, 2017
- Status last verified
- May 2025
- First submitted
- 12 Dec 2018
- Last updated
- 21 Oct 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No