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

Chronic Graft Versus Host Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT03790332
• Other study ID #: PCYC-1146-IM
• Secondary ID: 2017-004558-41
• Status: Active, not recruiting
• Phase: Phase 1/Phase 2
• Start date: November 19, 2018
• Est. completion date: January 4, 2026

Study information

• Verified date: November 2023
• Source: Pharmacyclics LLC.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 59
• Est. completion date: January 4, 2026
• Est. primary completion date: January 4, 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Year to 21 Years

Eligibility

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)

Related Conditions & MeSH terms

• Bronchiolitis Obliterans Syndrome
• Chronic Graft Versus Host Disease
• Graft vs Host Disease

Intervention

Drug:
• Ibrutinib
Ibrutinib capsule, tablet, or suspension administered orally once daily

Locations

Country	Name	City	State
Australia	The Royal Children's Hospital	Parkville	Victoria
Australia	Sydney Children's Hospital	Randwick
Australia	Queensland Children's Hospital	South Brisbane	Queensland
Australia	Cancer Center for Children. The Children's Hospital at Westmead	Westmead	New South Wales
Austria	St. Anna Kinderspital	Wien
Canada	CHU Sainte-Justine	Montréal	Quebec
Canada	children's and Women's Health Centre of British Columbia	Vancouver	British Columbia
France	CHU Nantes - Hopital Enfant Adolescent	Nantes
France	Hôpital Robert-Debré Ap-Hp	Paris
Germany	Charite-Universitaetsmedizin Berlin	Berlin
Israel	Hadassah Medical Centre	Jerusalem
Israel	Schneider Children's Medical Center in Israel	Petach Tikva
Israel	The Edmond and Lily Safra Children's Hospital	Ramat Gan
Italy	Fondazione MBBM-Clinica Pediatrica	Monza
Italy	U.O.C. Ematologia Oncoematologia Pediatrica	Pavia	PV
Italy	Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica	Roma
Italy	S.C. Farmacia Pediatrica - Presidio Ospedaliero Infantile Regina Margherita	Turin
Korea, Republic of	Asan Medical Center	Seoul
Korea, Republic of	Samsung Medical Center	Seoul
Korea, Republic of	Seoul National University Hospital	Seoul
Korea, Republic of	Seoul Saint Mary's Hospital	Seoul
Netherlands	Princess Maxima Center	Utrecht
Russian Federation	Federal State Budgetary Institution "Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology	Moscow
Russian Federation	Federal State Budgetary Educational Institution of Higher Professional Education "LP.Pavlov First Saint Petersburg State Medical	Saint Petersburg
Spain	Hospital Universitario Vall d'Hebron	Barcelona
Spain	Hospital Infantil Universitario Nino Jesus	Madrid
Spain	Hospital Universitario Universitario La Paz	Madrid
United Kingdom	Safari Day Care, Great Ormond Street Hospital	London	Greater London
United States	Children's Hospital	Aurora	Colorado
United States	Johns Hopkins University	Baltimore	Maryland
United States	Dana-Farber Cancer Institute	Boston	Massachusetts
United States	Ann & Robert H Lurie Children's Hospital of Chicago	Chicago	Illinois
United States	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio
United States	City of Hope	Duarte	California
United States	Hackensack University Medical Center	Hackensack	New Jersey
United States	St. Jude Children's Research Hospital	Memphis	Tennessee
United States	Medical College of Wisconsin	Milwaukee	Wisconsin
United States	University of Minnesota Masonic Children's Hospital	Minneapolis	Minnesota
United States	Memorial Sloan Kettering Cancer Center	New York	New York
United States	The Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
United States	Oregon Health and Science University	Portland	Oregon
United States	Washington University	Saint Louis	Missouri
United States	Johns Hopkins All Children's Hospital	Saint Petersburg	Florida
United States	Primary Children's Hospital	Salt Lake City	Utah
United States	Rady Children's Hospital	San Diego	California
United States	University of California	San Francisco	California
United States	Fred Hutchinson Cancer Research Center	Seattle	Washington
United States	Children's National Medical Center	Washington	District of Columbia

Sponsors (2)

Lead Sponsor	Collaborator
Pharmacyclics LLC.	Janssen Research & Development, LLC

Countries where clinical trial is conducted

United States,  Australia,  Austria,  Canada,  France,  Germany,  Israel,  Italy,  Korea, Republic of,  Netherlands,  Russian Federation,  Spain,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Part A- PK (measured by AUC) will be reported descriptively		Approximately 24 months
Primary	Part B- PK (measured by AUC) will be reported descriptively		Approximately 7 years
Primary	Number of patients with adverse events as a measure of safety and tolerability		Approximately 7 years
Secondary	Part A- Number of patients with adverse events as a measure of safety and tolerability		Approximately 24 months
Secondary	Part A- Pharmacodynamic effects as measured by in vitro BTK occupancy will be reported descriptively		Approximately 24 months
Secondary	Part A continuation cohort and Part B-Response rate at 24 weeks		Approximately 6 months after last subject in enrolled
Secondary	Part A continuation cohort and Part B- Duration of response (DOR)		Up to 48 weeks
Secondary	Part A continuation cohort and Part B-Overall survival (OS)		Approximately 5 years after last subject enrolled
Secondary	Part A continuation cohort and Part B-Late Effects Surveillance		Up to 5 years post enrollment
Secondary	Growth Parameter height in meters will be reported descriptively	Subjects will be monitored for growth and development	Up to 5 years post enrollment
Secondary	Growth Parameter weight in kilograms will be reported descriptively.	Subjects will be monitored for growth and development	Up to 5 years post enrollment
Secondary	Available immune reconstitution laboratory parameters will be reported descriptively	Subjects will be monitored for immune reconstitution	Up to 5 years post enrollment
Secondary	Late effects (Adverse events suspected to be related to treatment) will be quantified and reported descriptively		Up to 5 years post enrollment