Congenital Disorders

MCDS - In Follow-up

An open label phase I/IIa trial repurposing carbamazepine (CBZ) for the treatment of skeletal dysplasia in children

• Study stage: In Follow-up
• Sponsor: The Newcastle upon Tyne Hospitals NHS Foundation Trust
• Funder: European Commission: Horizon 2020
• Therapeutic area: Congenital Disorder
• Type of study: CTIMP (Rare Disease)

Aim: To assess the safety and tolerability of carbamazepine (CBZ) in the treatment of children with MCDS who are ambulant but have not yet reached skeletal maturity (open epiphyses)

Primary outcome:

Stage 1:

• Laboratory safety assessments, adverse events and physical examinations collected post IMP administration.
• Outcome of dose-titration safety review at 6 months post IMP treatment initiation.

Stage 2:

• Alteration from baseline in growth velocity over 12 months
• Growth velocity follow-up data at 12 months post treatment initiation

• Population: Paediatric
• Phase: I/IIa
• Design: Two-stage, open label
• Setting: Secondary care
• Planned Sample Size: 40 (11 participants Stage 1 dose titration; 29 participants Stage 2 treatment)

Website: http://www.mcds-therapy.eu