US-based biopharmaceutical company Neuren has completed enrolment for its Phase II clinical trial of trofinetide in pediatric Rett syndrome.
Trofinetide has been developed as a synthetic analogue of a naturally occurring neurotrophic peptide derived from IGF-1, a growth factor produced by brain cells.
Rett Syndrome is a rarely occurring, severe brain disorder affecting girls. It slows down head growth and leads to loss of muscle tone accompanied with uncoordinated breathing and seizures.
The Phase II trial has been designed as a randomised, double-blind, placebo-controlled study, which has enrolled 82 girls aged five to 15 with Rett syndrome.
In the trial, 62 patients were previously divided into one of four treatment groups receiving 50mg/kg, 100mg/kg, 200mg/kg of trofinetide and placebo.
The additional 20 patients have been randomised into one of two treatment groups: 200mg/kg and placebo.
The trial is primarily focused on determining the safety and tolerability of trofinetide compared with placebo.
Additional endpoints of the study include determining the efficacy of trofinetide in children.
The efficacy analysis will be based on the Motor Behaviour Assessment Change Index, in which the clinician rates the patient’s current level of function.
Efficacy analysis will be based on Domain Specific Concerns Visual Analog Scale and Caregiver Top 3 Concerns Visual Analog scale, which are the assessment of severity concerns identified for each subject on an individual basis.
The Clinical Global Impression of Improvement determines the baseline improvement of the patient’s overall illness.
The other factor that will help in the analysis of trofinetide’s efficacy is the Rett Syndrome Behaviour Questionnaire, a rating scale on which the patient’s caregiver rates the frequency of symptoms.