A Single Arm Phase II Trial of BMN 673 for Inoperable, Advanced Endometrial Cancer With Retrospective PTEN, MSI and MRE11 Analysis PTEN= Phosphatase and tensin homolog MSI= Microsatellite instability MRE11= Double-strand break repair protein MRE11A This trial will investigate whether the drug BMN 673 has therapeutic benefit in the treatment of advanced endometrial cancer. Nearly 8,000 patients are diagnosed with endometrial cancer in the UK every year. A significant proportion are either diagnosed with advanced disease which may be inoperable and/or metastatic (i.e spread to other organs outside the endometrium), or curable disease which relapses following first line treatment. There is no established standard of care for these patients as both chemo and hormone therapy has limited effectiveness and survival benefit. Survival rates have not improved in the past 20 years. Furthermore there are no so called 'targeted' drugs licensed for its treatment i.e. drugs that block the growth and spread of cancer by interfering with specific molecules involved in tumor growth and progression. This leaves an unmet need for effective systemic treatments for advanced, inoperable and metastatic endometrial cancer. BMN 673 has been shown to be potentially effective in treating cancers known to behave similarly to endometrial disease, both in the laboratory and in Phase I studies involving patients with advanced cancers. Similarly the drug appears to be relatively tolerable. A Phase II trial such as the one proposed by this application could demonstrate activity that might lead to a new effective treatment for patients with inoperable, advanced, recurrent or metastatic endometrial cancer, while the proposed substudy also presents the possibility of discovering a subset of patients more likely to derive benefit from BMN 673. This trial is for adult women (18 and above) with advanced, inoperable or metastatic endometrial cancer. Patients will be recruited from approximately 15 National Health Service (NHS) Trusts based in the United Kingdom (UK). The study is expected to last approximately 18-24 months in terms of recruitment time, and a maximum of 100 eligible women will be registered. All patients will receive BMN 673 until their disease worsens or their doctor decides they should stop treatment.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Starting oral dose of 1.0 mg once daily to be taken until progression, death, unacceptable toxicity, withdrawal consent or any other criterion felt by the Investigator to preclude continuation of treatment.
Royal Sussex County Hospital
Brighton, East Sussex, United Kingdom
The Beatson West of Scotland Cancer Centre
Glasgow, Greater Glasgow, United Kingdom
St Bartholomew's Hospital
London, Greater London, United Kingdom
University College Hospital
London, Greater London, United Kingdom
The Christie Hospital
Manchester, Greater Manchester, United Kingdom
Western General Hospital
Edinburgh, Lothian, United Kingdom
The Churchill Hospital
Oxford, Oxfordshire, United Kingdom
Velindre Cancer Centre
Cardiff, South Glamorgan, United Kingdom
St James's University Hospital
Leeds, South Yorkshire, United Kingdom
Royal Marsden Hospital (Sutton)
Sutton, Surrey, United Kingdom
...and 6 more locations
Progression free survival (PFS) rate
Measured from date of first BMN 673 dose to first progression (defined using Response Evaluation Criteria in Solid Tumours (RECIST) v1.1) or death, whichever is the sooner.
Time frame: 6 months
Best Response
Measured from the date of first BMN 673 dose.
Time frame: Up to 30 months
Overall survival (OS)
Time frame: Up to 30 months
Response at each radiological assessment
Time frame: Up to 30 months
Duration of Response (DoR)
Time frame: Up to 30 months
Median PFS
Time frame: Up to 30 months
Safety and toxicity
Common Terminology Criteria for Adverse Events (CTCAE) v4.03 grade 3-4 toxicity; dose reductions, omissions, delays; exposure; compliance.
Time frame: Up to 30 months
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.