Oral MK8669 in Children/Adolescents with Solid Tumours(6 - <18years)

• Research type

  Research Study

• Full title

  A Phase I Study of Ridaforolimus in Paediatric Patients with Advanced Solid Tumours

• IRAS ID

  87572

• Contact name

  Andrew David Jones Pearson

• Sponsor organisation

  Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc.

• Eudract number

  2011-000729-55

• Clinicaltrials.gov Identifier

  N/A

• Research summary

  BACKGROUNDRidaforolimus (MK-8669) is an investigational drug targeted and small-molecule inhibitor of the mammalian target rapamycinthe (mTOR), a protein that acts as a central regulator of protein synthesis, cell proliferation, cell cycle progression and cell survival, integrating signals from proteins, known to be important to malignancy. Blocking mTOR creates a starvation-like effect in cancer cells by interfering with cell growth, division, metabolism, and in the growth of new blood vessels from pre-existing vessels. The purpose of this study is to see how safe and well tolerated is ridaforolimus when given to paediatrics, between the ages of 6 to less than 18 years, with advanced solid tumours including lymphoma and tumours of the central nervous system. DESIGN: This is a multi-centre, open-label, early study (Phase 1) dose-escalation trial of ridaforolimus oral tablets to evaluate safety, tolerability and the amount of drug in the patient's blood (pharmacokinetics, PK). Ridaforolimus is administered as an oral formulation, and is supplied as 10 mg enteric-coated tablets. Dosing will be adjusted based on body surface area and rounded to the nearest 10 mg tablet. Patients will be monitored carefully for the development of adverse experiences and for clinical and/or radiographic evidence of disease progression according to usual standards of clinical practice. There will be no intra-patient dose escalation for patients enrolled on this study. Patients will be treated until disease progression, unacceptable toxicity, or the withdrawal of consent, and will be treated thereafter at the discretion of the doctor. It is anticipated that 18-30 patients will be enrolled into the study at 10-15 sites in North America and Europe, including two sites in the UK. The final number of patients will depend on empirical safety data and dose limited toxicity (DLTs) observed.

• REC name

  South Central - Oxford A Research Ethics Committee

• REC reference

  11/SC/0452

• Date of REC Opinion

  15 Dec 2011

• REC opinion

  Further Information Favourable Opinion