PVNS
Research type
Research Study
Full title
Phase II study of nilotinib efficacy in Pigmented Villo-Nodular Synovitis/Tenosynovial Giant Cell Tumour (PVNS/TGCT)
IRAS ID
70750
Contact name
Bass Hassan
Sponsor organisation
Centre Leon-Berard
Eudract number
2010-018869-29
Clinicaltrials.gov Identifier
Research summary
Pigmented villo-nodular synovitis(PVNS), also known as Tenosynovial Giant Cell Tumour (TGCT),is a rare pathological entity affecting the synovium in young adults. Imatinib is a treatment indicated for chronic myeloid leukaemia and gastro intestinal stromal tumour which blocks M-CSF receptor activation at therapeutic concentration.PVNS/TGCT is usually treated by surgery alone. However, relapses may occur, and re-excision may be needed, sometimes with possible impairment. Blay et al. recently reported the case of a patient with recurrent and symptomatic PVNS/TGCT following surgery, in which surgical re-excision would have had important functional consequences. In the case report the patient was treated with imatinib providing a rapid tumour response. A relapse was observed at discontinuation and a secondary response obtained at reintroduction. Imatinib is a good candidate to induce complete responses in relapsing PVNS and may offer anoption in patients in whom surgery is not feasible. Nilotinib has inhibitory properties similar to imatinib on the M-CSF pathway. The reason for selecting nilotinib over imatinib was becasue of the limited experience of imatinib and PVNS so far the toxicity was substantial and nilotinib has a more favourable toxicity profile in particular with soft tissue and facial oedema. This study is designed to explore the efficacy of nilotinib as treatment of patients with inoperable PVNS/TGCT. The disease being rare, this clinical trial is a non-randomised open-label and international study. Nilotinib will be administered in patients with progressive or relapsing PVNS. The main benefit anticipated for patients will be the tumour reduction and the consequent functional improvements. The main risk will be the non-response to the treatment and the known adverse effects of the nilotinib.
REC name
South Central - Oxford C Research Ethics Committee
REC reference
11/SC/0471
Date of REC Opinion
16 Feb 2012
REC opinion
Further Information Favourable Opinion