GO42286 - Alectinib in Paediatric Patients

• Research type

  Research Study

• Full title

  A PHASE I/II, OPEN-LABEL, MULTI-CENTRE STUDY EVALUATING THE SAFETY, PHARMACOKINETICS, AND EFFICACY OF ALECTINIB IN PAEDIATRIC PATIENTS WITH ALK FUSION POSITIVE SOLID OR CNS TUMOURS FOR WHOM PRIOR TREATMENT HAS PROVEN TO BE INEFFECTIVE OR FOR WHOM THERE IS NO SATISFACTORY TREATMENT AVAILABLE

• IRAS ID

  1003535

• Contact name

• Sponsor organisation

  F. Hoffmann-La Roche Ltd

• Eudract number

  2020-004239-25

• Research summary

  Although overall survival(OS) in children with cancer has improved tremendously over the past 40 years, the outcome for paediatric patients with relapsed/refractory tumours or those considered incurable at diagnosis remains dismal. Recent studies have demonstrated a variety of paediatric solid tumours harbour Anaplastic Lymphoma Kinase(ALK) fusions. For most of patients with those tumour types, the clinical outcome after conventional primary treatment remains poor, especially in primary CNS tumours for which there is often no satisfactory first-line treatment.
  Alectinib is approved in Japan in children with recurrent/refractory ALK-positive Anaplastic Large Cell Lymphoma. There are several other small studies in literature that have demonstrated the efficacy of anti-ALK therapies, when there were no other medical options, in a variety of paediatric malignancies, with very poor outcomes.
  Given the mechanism of action of alectinib, the extensive clinical experience, and high response rate in adult patients with ALK-fusion positive NSCLC(including CNS disease), very promising clinical outcome with other ALK inhibitors in ALK-fusion-positive tumours and the poor long-term survival of children/adolescents with ALK positive relapsed/refractory solid tumours/CNS tumours, there is a strong rationale and expected benefit to develop alectinib in paediatric patients with solid/CNS ALK fusion-positive disease.

  This Phase I/II study will evaluate the safety, pharmacokinetics, and efficacy of alectinib in children with ALK fusion-positive solid/CNS tumours for whom prior treatment has proven to be ineffective or for whom there is no satisfactory standard treatment available.
  The study will recruit a minimum of 30 children globally with approx. 8 from the UK across 4 sites.
  The study will last approx. 8 years from screening of the first patient to LPLV.
  The study is sponsored by F. Hoffman La Roche
  Research Summary; Version 1 dated 07-Jan-2021

• REC name

  South Central - Berkshire B Research Ethics Committee

• REC reference

  21/SC/0110

• Date of REC Opinion

  10 Jun 2021

• REC opinion

  Further Information Favourable Opinion