BP42992 A Phase 2 study to evaluate treatment with Basmisanil in children with DUP15Q syndrome

• Research type

  Research Study

• Full title

  A PHASE II, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL GROUP STUDY TO EVALUATE THE SAFETY, EFFICACY, AND PHARMACODYNAMICS OF 52 WEEKS OF TREATMENT WITH BASMISANIL IN CHILDREN WITH DUP15Q SYNDROME.

• IRAS ID

  1004246

• Contact name

  Rebecca Shields

• Contact email

  welwyn.uk_ethics@roche.com

• Sponsor organisation

  F. Hoffmann La-Roche Ltd

• Eudract number

  2021-003791-13

• Clinicaltrials.gov Identifier

  NCT05307679

• Research summary

  The purpose of this study is to evaluate the safety and efficacy of 52 weeks of basmisanil treatment in children with Dup 15q syndrome.
  Dup15q syndrome is a rare and severe neurodevelopmental disorder (NDD) caused by copy number gains of the q11.2-q13.1 region on chromosome 15 that contains both UBE3A and GABR genes. Basmisanil is a clinically characterised, brain penetrant, and highly selective negative allosteric modulator (NAM) of the GABAA α5R.
  Clinical evidence indicates that treatment with basmisanil has the potential to affect disease pathophysiology and core neurodevelopmental symptoms by normalising the excessive GABAA α5R activity in Dup15q syndrome. The safety monitoring plan in place to continuously assess risks in this population (as compared to the established risk profile of basmisanil), are in place to safeguard study participants. The benefit/risk balance is thus considered to be favourable for paediatric participants in this study, who are affected by a rare disease with no treatment options.

  Approximately 90 children will take part in the study globally, recruitment will be staggered by age and the presence or absence of epilepsy. Overall the study targets to enrol approximately one-third of the children in the 2 to 5 year age category. Study participants will be randomised 2:1 to receive oral treatment with either basmisanil or placebo for 52 weeks

  The participants in this study will be children aged 2 to 11 years with genetically confirmed maternal duplications (3 copies) or triplications (4 copies) of chromosome 15q11.2􀀀q13.1 (Dup15q syndrome)
  The duration of the study for each participant is approximately 61 weeks.

  There will be approximately 8 UK patients recruited at 3 UK sites

  The study is sponsored by F. Hoffman La Roche

  Research Summary; Version 1 dated 28/9/21

• REC name

  West Midlands - Coventry & Warwickshire Research Ethics Committee

• REC reference

  21/WM/0270

• Date of REC Opinion

  5 Jan 2022

• REC opinion

  Further Information Favourable Opinion