COSCIENS Biopharma Inc T.CSCI

What could an additional trial, if needed, look like?

[prophetoffactz]

Perplexity Ai:

Based on the top-line results of the DETECT trial, an additional clinical trial to prove macimorelin's efficacy for the pediatric population could potentially look like this:

Proposed Follow-Up Efficacy Trial

Study Design:

• A shorter, focused efficacy trial comparing macimorelin to a single, well-established comparator test (e.g. insulin tolerance test)
• Open-label, randomized, two-way crossover design

Key Elements:

• Smaller sample size (e.g. 50-75 subjects) given confirmed safety profile
• Narrower age range (e.g. 5-15 years) to reduce variability
• Single comparator test to simplify analysis
• Refined cut-off point for macimorelin based on DETECT trial data
• Independent expert panel to adjudicate GHD status

Primary Endpoint:

• Agreement between macimorelin test and comparator test in diagnosing GHD

Secondary Endpoints:

• Sensitivity and specificity of macimorelin vs comparator
• Pharmacokinetic/pharmacodynamic analysis
• Patient/caregiver preference assessment

Duration:

• 6-9 months total (shorter screening and follow-up periods)

This streamlined trial could potentially be conducted more quickly and cost-effectively, leveraging the established safety profile from the DETECT study. The focus would be on demonstrating non-inferiority or superiority of macimorelin to a single established comparator test in accurately diagnosing childhood-onset GHD.The unexpected results from DETECT, particularly the high GH cut-off point for macimorelin, would need to be carefully analyzed to refine the protocol. Engaging with regulatory agencies to discuss the DETECT results and proposed follow-up trial design would be crucial before proceeding.