PCYC-1146-IM Study of Ibrutinib in Paediatric Patients with cGVHD
Research type
Research Study
Full title
Phase 1/2 Dose Finding, Safety and Efficacy Study of Ibrutinib in Pediatric Subjects with Chronic Graft Versus Host Disease (cGVHD)
IRAS ID
244466
Contact name
Gauri Sunkersett
Contact email
Sponsor organisation
Pharmacyclics LLC, An AbbVie Company
Eudract number
2017-004558-41
Clinicaltrials.gov Identifier
102,688, IND
Duration of Study in the UK
7 years, 0 months, 0 days
Research summary
Chronic GVHD is a life threatening complication which can occur following stem cell transplantation (SCT). In the paediatric population, it is estimated to occur in 20 to 35% of SCT recipients. Overall survival for patients affected by cGVHD which is resistant to treatment is estimated to be around 20-25% 5 years post SCT. Beyond the use of steroid, and calcineurin inhibitors, there is currently no standard of care for cGVHD and there are no approved therapies for the treatment cGVHD in paediatric patients. The proposed study is being supported by recent FDA approval of ibrutinib for the treatment of cGVHD in adult patients.
This study will investigate the safety and efficacy of ibrutinib in combination with prednisone as a treatment for children and young adults with cGVHD.
The study consist of 2 parts. Part A will aim to enrol approximately 12 patients aged 1 to 11 years old to allow the identification of the safest Ibrutinib dose in the paediatric population. Part B will enrol between 10 and 32 patients aged 1 to 21 years to study the drug safety, efficacy and pharmacokinetics (how the drug is absorbed by the patients) of ibrutinib. Patients’ participation is expected to last about 5 years.
Both parts A and B of the study consist of 3 phases: screening, treatment and follow up. Study medicine, ibrutinib, will be taken orally, once a day. Patients with new-onset moderate / severe cGVHD will be required to take prednisone in combination with ibrutinib. During the study patients will undergo study assessments which will include physical examination, blood tests, electrocardiogram, lung function test and questionnaires. Patients will attend study visits and will be contacted by phone to discuss study treatment dosing changes. Patients who complete the study treatment may be eligible to take part in a long-term extension study. Patients who do not join the extension study will have the end of trial visit and will enter the long term follow up phase. Patients who complete the study will return to their standard of care treatment, which may be ibrutinib, if by this time, the study medicine receives regulatory approvals in UK.REC name
South Central - Hampshire A Research Ethics Committee
REC reference
19/SC/0125
Date of REC Opinion
29 May 2019
REC opinion
Further Information Favourable Opinion