Clinical trial NCT03790332: Phase 1/2 Dose Finding and Safety Study of Ibrutinib in Pediatric Subjects With Chronic Graft Versus Host Disease (cGVHD)
Official title: Phase 1/2 Dose Finding and Safety Study of Ibrutinib in Pediatric Subjects With Chronic Graft Versus Host Disease (cGVHD)
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE1
PHASE2
Conditions: Chronic Graft Versus Host Disease
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
moderate cGVHD
severe cGVHD
moderate chronic graft versus host disease
severe chronic graft versus host disease
Lead sponsor: Pharmacyclics LLC.
Sponsor class: INDUSTRY
Collaborators: name: Janssen Research & Development, LLC
First posted: 2018-12-31
Enrollment: 59
Sex: ALL
Minimum age (years): 1
Maximum age (years): 21
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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


Eligibility criteria: 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)

Primary outcomes:
measure: Part A- PK (measured by AUC) will be reported descriptively
timeFrame: Approximately 24 months
measure: Part B- PK (measured by AUC) will be reported descriptively
timeFrame: Approximately 7 years
measure: Number of patients with adverse events as a measure of safety and tolerability
timeFrame: Approximately 7 years
Secondary outcomes:
measure: Part A- Number of patients with adverse events as a measure of safety and tolerability
timeFrame: Approximately 24 months
measure: Part A- Pharmacodynamic effects as measured by in vitro BTK occupancy will be reported descriptively
timeFrame: Approximately 24 months
measure: Part A continuation cohort and Part B-Response rate at 24 weeks
timeFrame: Approximately 6 months after last subject in enrolled
measure: Part A continuation cohort and Part B- Duration of response (DOR)
timeFrame: Up to 48 weeks
measure: Part A continuation cohort and Part B-Overall survival (OS)
timeFrame: Approximately 5 years after last subject enrolled
measure: Part A continuation cohort and Part B-Late Effects Surveillance
timeFrame: Up to 5 years post enrollment
measure: Growth Parameter height in meters will be reported descriptively
timeFrame: Up to 5 years post enrollment
measure: Growth Parameter weight in kilograms will be reported descriptively.
timeFrame: Up to 5 years post enrollment
measure: Available immune reconstitution laboratory parameters will be reported descriptively
timeFrame: Up to 5 years post enrollment
measure: Late effects (Adverse events suspected to be related to treatment) will be quantified and reported descriptively
timeFrame: Up to 5 years post enrollment
Other outcomes:
Locations:
city: Duarte
country: United States
facility: City of Hope
state: California
city: San Diego
country: United States
facility: Rady Children's Hospital
state: California
city: San Francisco
country: United States
facility: University of California
state: California
city: Aurora
country: United States
facility: Children's Hospital
state: Colorado
city: Washington
country: United States
facility: Children's National Medical Center
state: District of Columbia
city: Saint Petersburg
country: United States
facility: Johns Hopkins All Children's Hospital
state: Florida
city: Chicago
country: United States
facility: Ann & Robert H Lurie Children's Hospital of Chicago
state: Illinois
city: Baltimore
country: United States
facility: Johns Hopkins University
state: Maryland
city: Boston
country: United States
facility: Dana-Farber Cancer Institute
state: Massachusetts
city: Minneapolis
country: United States
facility: University of Minnesota Masonic Children's Hospital
state: Minnesota
city: Saint Louis
country: United States
facility: Washington University
state: Missouri
city: Hackensack
country: United States
facility: Hackensack University Medical Center
state: New Jersey
city: New York
country: United States
facility: Memorial Sloan Kettering Cancer Center
state: New York
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Portland
country: United States
facility: Oregon Health and Science University
state: Oregon
city: Philadelphia
country: United States
facility: The Children's Hospital of Philadelphia
state: Pennsylvania
city: Memphis
country: United States
facility: St. Jude Children's Research Hospital
state: Tennessee
city: Salt Lake City
country: United States
facility: Primary Children's Hospital
state: Utah
city: Seattle
country: United States
facility: Fred Hutchinson Cancer Research Center
state: Washington
city: Milwaukee
country: United States
facility: Medical College of Wisconsin
state: Wisconsin
city: Westmead
country: Australia
facility: Cancer Center for Children. The Children's Hospital at Westmead
state: New South Wales
city: South Brisbane
country: Australia
facility: Queensland Children's Hospital
state: Queensland
city: Parkville
country: Australia
facility: The Royal Children's Hospital
state: Victoria
city: Randwick
country: Australia
facility: Sydney Children's Hospital
city: Wien
country: Austria
facility: St. Anna Kinderspital
city: Vancouver
country: Canada
facility: children's and Women's Health Centre of British Columbia
state: British Columbia
city: Montréal
country: Canada
facility: CHU Sainte-Justine
state: Quebec
city: Nantes
country: France
facility: CHU Nantes - Hopital Enfant Adolescent
city: Paris
country: France
facility: Hôpital Robert-Debré Ap-Hp
city: Berlin
country: Germany
facility: Charite-Universitaetsmedizin Berlin
city: Jerusalem
country: Israel
facility: Hadassah Medical Centre
city: Petach Tikva
country: Israel
facility: Schneider Children's Medical Center in Israel
city: Ramat Gan
country: Israel
facility: The Edmond and Lily Safra Children's Hospital
city: Pavia
country: Italy
facility: U.O.C. Ematologia Oncoematologia Pediatrica
state: PV
city: Monza
country: Italy
facility: Fondazione MBBM-Clinica Pediatrica
city: Roma
country: Italy
facility: Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica
city: Turin
country: Italy
facility: S.C. Farmacia Pediatrica - Presidio Ospedaliero Infantile Regina Margherita
city: Seoul
country: Korea, Republic of
facility: Seoul National University Hospital
city: Seoul
country: Korea, Republic of
facility: Asan Medical Center
city: Seoul
country: Korea, Republic of
facility: Samsung Medical Center
city: Seoul
country: Korea, Republic of
facility: Seoul Saint Mary's Hospital
city: Utrecht
country: Netherlands
facility: Princess Maxima Center
city: Moscow
country: Russian Federation
facility: Federal State Budgetary Institution "Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology
city: Saint Petersburg
country: Russian Federation
facility: Federal State Budgetary Educational Institution of Higher Professional Education "LP.Pavlov First Saint Petersburg State Medical
city: Barcelona
country: Spain
facility: Hospital Universitario Vall d'Hebron
city: Madrid
country: Spain
facility: Hospital Infantil Universitario Nino Jesus
city: Madrid
country: Spain
facility: Hospital Universitario Universitario La Paz
city: London
country: United Kingdom
facility: Safari Day Care, Great Ormond Street Hospital
state: Greater London
Study officials:
affiliation: Pharmacyclics LLC.
name: Gauri Sunkersett
role: STUDY_DIRECTOR
References: