CRED Understanding Clinical Development 2024

Question 2 What data do you need to start Phase 2? Describe the design considerations for the first Phase 2 study for Devostatin. Would you consider one or more Phase 2 studies? What is the rationale for your recommendations to the project team?

Parameter

Answer

Rationale/Comments

Main study objectives Type of study Doses to be tested Control group(s) Study duration Age range Study population & Key inclusion/exclusion criteria Concomitant medicines (allowed or not allowed) Endpoints of interest (Primary, Secondary, other) Other clinical parameters of interest including safety monitoring

Size of study arms Number of studies

Part II (Phase 3) Phase 1 and Phase 2 studies have now been conducted on Devostatin. You have been asked to advise the Project Team on any regulatory guidelines, precedents and activities which could impact on the clinical development of this project and to assist your clinical colleagues in developing the phase 3 programme. Summary of the development programme to date A comprehensive preclinical development programme has been performed. There is no evidence of genotoxicity. The 6-month repeat dose studies and the reproductive toxicity studies are complete. The major target organs for toxicity are the GI tract and the kidney. Longer-term studies to support the long-term use of the product are ongoing. Human pharmacokinetics data confirm that Devostatin is readily absorbed from an oral dose. The Phase 1 and Phase 2 clinical studies are complete. In Phase 2, 3 doses were investigated. The studies showed a statistically significant difference compared to placebo for the highest dose (p=0.049). There was a dose-related trend for the other doses. No unexpected AEs were seen. A PIP has been filed in which a partial waiver for children younger than 6 years has been requested, on

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