Afatinib in Chordoma
Research type
Research Study
Full title
A phase 2, single arm, European multi-center trial evaluating the efficacy of afatinib as first line or later line treatment in advanced chordoma
IRAS ID
222545
Contact name
Sandra Strauss
Contact email
Sponsor organisation
Leiden University Medical Center
Eudract number
2016-002766-31
Clinicaltrials.gov Identifier
Duration of Study in the UK
4 years, 2 months, 2 days
Research summary
Chordomas are rare (incidence of 1 in 1,000,000), slow-growing bone tumours that arise from bones in the spine, most commonly the sacrum but can affect the base of skull. They are locally aggressive and although they rarely metastasize (spread to other parts of the body), they negatively impact on the quality of life and lead to premature death.
Currently, complete surgical resection (removal) followed by radiotherapy is the best option for patients with localised disease. However, the decision whether a complete resection can be performed depends on the anatomical location of the tumour and its proximity to important parts of the central nervous system. This also affects the possibility of delivery of high doses of radiation, as chordomas are relatively resistant to radiotherapy.
No cytotoxic chemotherapy or systemic therapies are currently available or recommended in any line of treatment. There is, therefore, a huge unmet medical need for new systemic treatments in chordoma patients.
This is a phase 2, multinational, single arm study of afatinib in adult patients. The primary aim of the study is to test safety, tolerability and efficacy of afatinib. Afatinib is a licenced drug that blocks signalling from the EGFR pathway in order to inhibit cell growth in tumours that express EGF receptors. Studies on chordoma tumour samples have shown a high level of EGFR expression, thus making the EGFR pathway an interesting therapeutic target. No clinical studies with afatinib have been performed in chordoma before.
Afatinib will be investigated in 2 cohorts of patients: 20 first-line patients, and 20 second-, or further, line patients. It will be given daily in a dose of 40mg orally in a 4-week cycles until disease progression or patient withdrawal. Three European centres will participate in the study (UK, Netherlands, and Italy); UCLH will be the only site in the UK.REC name
London - Harrow Research Ethics Committee
REC reference
18/LO/1440
Date of REC Opinion
23 Nov 2018
REC opinion
Further Information Favourable Opinion