LONDON, UK: Diurnal Group plc announced the US Food and Drug Administration (FDA) has agreed a Special Protocol Assessment (SPA) for Chronocort (modified-release hydrocortisone) for the design, endpoints and statistical analysis approach of a pivotal study of Chronocort for the treatment for Congenital Adrenal Hyperplasia (CAH) in the US.
The SPA provides agreement that the Phase 3 trial design adequately addresses objectives that would support the regulatory submission for drug approval.
The pivotal study is entitled “A randomised, double-blind, active-controlled, Phase 3 study of Chronocort compared with immediate-release hydrocortisone replacement therapy in participants aged 16-years and over with Congenital Adrenal Hyperplasia (CAH)” – CONnECT – and is anticipated to start during H2 2021. Study start-up activities have already been commenced by the Company to enable a timely initiation and recruitment of up to 150 subjects with CAH who will be treated for a period of 52 weeks.
Chronocort is a preparation of hydrocortisone that has been specifically designed for patients with CAH, an orphan condition, caused by deficiency of adrenal enzymes, most commonly 21-hydroxylase. Diurnal holds the relevant Orphan Drug Designation for use of Chronocort in CAH in the US. Approximately two-thirds of CAH patients are estimated to have poor disease control, leading to elevated androgen levels. The condition is estimated to affect a total of approximately 16,000 patients in the US.
Martin Whitaker, Chief Executive Officer of Diurnal, commented: “We are pleased to have concluded a series of positive discussions with the FDA around the design of our pivotal Chronocort study to support a regulatory submission for marketing approval in the US. During the first half of 2021 the Diurnal team has been working hard to complete the required preparation activities for this trial and we look forward to starting this study as soon as possible.”
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12 Jul, 8:29 am GMT+1 ·Disclaimer
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