Clinical Trial: Extension Study for Patients Entered Into Study Infacort 003
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional
Official Title: Open-label, Long-term Follow-up of Safety and Biochemical Disease Control of Infacort® in Neonates, Infants and Children With Congenital Adrenal Hyperplasia and Adrenal Insu
Brief Summary: This is a Phase 3, open-label, single-group, non-randomised, observational study of the safety and biochemical disease control of Infacort® in neonates, infants and children with AI who have completed study Infacort 003. All subjects who have satisfactorily completed study Infacort 003 will be offered the opportunity to take part in Infacort 004.
Detailed Summary: This is a Phase 3, open-label, single-group, non-randomised, observational study of the safety and biochemical disease control of Infacort® in neonates, infants and children with AI who have completed study Infacort 003 (EudraCT number 2014-002265-30). All subjects who have satisfactorily completed study Infacort 003 will be offered the opportunity to participate in study Infacort 004 at or after their final visit of study Infacort 003. Subjects will receive the usual clinically-appropriate dose (since bioequivalence has been demonstrated with conventional hydrocortisone), as determined by the Investigator, which will be administered according to usual clinical practice - generally 3 or 4 times a day. Subjects can continue to be treated in this study until they meet the study withdrawal criteria, Infacort® is granted a marketing authorisation (and so is available commercially, which is expected within 2 years of initiation of this study), Infacort® is refused a marketing authorisation, or the Sponsor decides to discontinue the study.
Sponsor: Diurnal Limited
Current Primary Outcome: Incidence of serious adverse events (SAEs) and adverse events (AEs) [ Time Frame: 18 months ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Growth velocity [ Time Frame: 18 months ]Growth velocity standard deviation score (SDS)
- Cortisol levels [ Time Frame: 18 months ]Cortisol levels measured from dried blood spots
Original Secondary Outcome: Same as current
Information By: Diurnal Limited
Dates:
Date Received: March 16, 2016
Date Started: March 2016
Date Completion: August 2017
Last Updated: April 5, 2016
Last Verified: March 2016