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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03790332




Registration number
NCT03790332
Ethics application status
Date submitted
12/12/2018
Date registered
31/12/2018
Date last updated
14/11/2023

Titles & IDs
Public title
Phase 1/2 Dose Finding and Safety Study of Ibrutinib in Pediatric Subjects With Chronic Graft Versus Host Disease (cGVHD)
Scientific title
Phase 1/2 Dose Finding and Safety Study of Ibrutinib in Pediatric Subjects With Chronic Graft Versus Host Disease (cGVHD)
Secondary ID [1] 0 0
2017-004558-41
Secondary ID [2] 0 0
PCYC-1146-IM
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Chronic Graft Versus Host Disease 0 0
Condition category
Condition code
Respiratory 0 0 0 0
Other respiratory disorders / diseases
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Ibrutinib

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.


Treatment: Drugs: Ibrutinib
Ibrutinib capsule, tablet, or suspension administered orally once daily
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Part A- PK (measured by AUC) will be reported descriptively
Timepoint [1] 0 0
Approximately 24 months
Primary outcome [2] 0 0
Part B- PK (measured by AUC) will be reported descriptively
Timepoint [2] 0 0
Approximately 7 years
Primary outcome [3] 0 0
Number of patients with adverse events as a measure of safety and tolerability
Timepoint [3] 0 0
Approximately 7 years
Secondary outcome [1] 0 0
Part A- Number of patients with adverse events as a measure of safety and tolerability
Timepoint [1] 0 0
Approximately 24 months
Secondary outcome [2] 0 0
Part A- Pharmacodynamic effects as measured by in vitro BTK occupancy will be reported descriptively
Timepoint [2] 0 0
Approximately 24 months
Secondary outcome [3] 0 0
Part A continuation cohort and Part B-Response rate at 24 weeks
Timepoint [3] 0 0
Approximately 6 months after last subject in enrolled
Secondary outcome [4] 0 0
Part A continuation cohort and Part B- Duration of response (DOR)
Timepoint [4] 0 0
Up to 48 weeks
Secondary outcome [5] 0 0
Part A continuation cohort and Part B-Overall survival (OS)
Timepoint [5] 0 0
Approximately 5 years after last subject enrolled
Secondary outcome [6] 0 0
Part A continuation cohort and Part B-Late Effects Surveillance
Timepoint [6] 0 0
Up to 5 years post enrollment
Secondary outcome [7] 0 0
Growth Parameter height in meters will be reported descriptively
Timepoint [7] 0 0
Up to 5 years post enrollment
Secondary outcome [8] 0 0
Growth Parameter weight in kilograms will be reported descriptively.
Timepoint [8] 0 0
Up to 5 years post enrollment
Secondary outcome [9] 0 0
Available immune reconstitution laboratory parameters will be reported descriptively
Timepoint [9] 0 0
Up to 5 years post enrollment
Secondary outcome [10] 0 0
Late effects (Adverse events suspected to be related to treatment) will be quantified and reported descriptively
Timepoint [10] 0 0
Up to 5 years post enrollment

Eligibility
Key inclusion criteria
Key Eligibility:



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:
Minimum age
1 Year
Maximum age
21 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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 design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
19/11/2018
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
4/01/2026
Actual
Sample size
Target
Accrual to date
Final
59
Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC
Recruitment hospital [1] 0 0
Cancer Center for Children. The Children's Hospital at Westmead - Westmead
Recruitment hospital [2] 0 0
Queensland Children's Hospital - South Brisbane
Recruitment hospital [3] 0 0
The Royal Children's Hospital - Parkville
Recruitment hospital [4] 0 0
Sydney Children's Hospital - Randwick
Recruitment postcode(s) [1] 0 0
2145 - Westmead
Recruitment postcode(s) [2] 0 0
4101 - South Brisbane
Recruitment postcode(s) [3] 0 0
3052 - Parkville
Recruitment postcode(s) [4] 0 0
2031 - Randwick
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Colorado
Country [3] 0 0
United States of America
State/province [3] 0 0
District of Columbia
Country [4] 0 0
United States of America
State/province [4] 0 0
Florida
Country [5] 0 0
United States of America
State/province [5] 0 0
Illinois
Country [6] 0 0
United States of America
State/province [6] 0 0
Maryland
Country [7] 0 0
United States of America
State/province [7] 0 0
Massachusetts
Country [8] 0 0
United States of America
State/province [8] 0 0
Minnesota
Country [9] 0 0
United States of America
State/province [9] 0 0
Missouri
Country [10] 0 0
United States of America
State/province [10] 0 0
New Jersey
Country [11] 0 0
United States of America
State/province [11] 0 0
New York
Country [12] 0 0
United States of America
State/province [12] 0 0
Ohio
Country [13] 0 0
United States of America
State/province [13] 0 0
Oregon
Country [14] 0 0
United States of America
State/province [14] 0 0
Pennsylvania
Country [15] 0 0
United States of America
State/province [15] 0 0
Tennessee
Country [16] 0 0
United States of America
State/province [16] 0 0
Utah
Country [17] 0 0
United States of America
State/province [17] 0 0
Washington
Country [18] 0 0
United States of America
State/province [18] 0 0
Wisconsin
Country [19] 0 0
Austria
State/province [19] 0 0
Wien
Country [20] 0 0
Canada
State/province [20] 0 0
British Columbia
Country [21] 0 0
Canada
State/province [21] 0 0
Quebec
Country [22] 0 0
France
State/province [22] 0 0
Nantes
Country [23] 0 0
France
State/province [23] 0 0
Paris
Country [24] 0 0
Germany
State/province [24] 0 0
Berlin
Country [25] 0 0
Israel
State/province [25] 0 0
Jerusalem
Country [26] 0 0
Israel
State/province [26] 0 0
Petach Tikva
Country [27] 0 0
Israel
State/province [27] 0 0
Ramat Gan
Country [28] 0 0
Italy
State/province [28] 0 0
PV
Country [29] 0 0
Italy
State/province [29] 0 0
Monza
Country [30] 0 0
Italy
State/province [30] 0 0
Roma
Country [31] 0 0
Italy
State/province [31] 0 0
Turin
Country [32] 0 0
Korea, Republic of
State/province [32] 0 0
Seoul
Country [33] 0 0
Netherlands
State/province [33] 0 0
Utrecht
Country [34] 0 0
Russian Federation
State/province [34] 0 0
Moscow
Country [35] 0 0
Russian Federation
State/province [35] 0 0
Saint Petersburg
Country [36] 0 0
Spain
State/province [36] 0 0
Barcelona
Country [37] 0 0
Spain
State/province [37] 0 0
Madrid
Country [38] 0 0
United Kingdom
State/province [38] 0 0
Greater London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Pharmacyclics LLC.
Address
Country
Other collaborator category [1] 0 0
Commercial sector/industry
Name [1] 0 0
Janssen Research & Development, LLC
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Dose Finding and Safety Study of Ibrutinib in Pediatric Subjects with Chronic Graft Versus Host Disease (cGVHD)
Trial website
https://clinicaltrials.gov/study/NCT03790332
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Gauri Sunkersett
Address 0 0
Pharmacyclics LLC.
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT03790332