Comparison of Chronocort Versus Standard Hydrocortisone Replacement Therapy in Participants Aged 16 Years and Over With Congenital Adrenal Hyperplasia

• Status: Not yet recruiting
• Trial end date: 2023-12-01
• Target enrollment: 0
• Participant gender: All

Summary

This study is a randomized, double-blind, active-controlled, phase III study of Chronocort® compared with immediate-release hydrocortisone replacement therapy in participants aged 16 years and over with Congenital Adrenal Hyperplasia.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Diurnal Limited
• Treatments: Hydrocortisone; Hydrocortisone 17-butyrate 21-propionate; Hydrocortisone acetate; Hydrocortisone hemisuccinate

Criteria

Inclusion Criteria:

• Male or female participants must be aged 16 years or older at the time of signing the informed consent/assent.

• In participants aged <18 years, height velocity must be less than 2 cm/year in the last year and puberty must be completed (Tanner stage V).

• Participants with known classic CAH due to 21 hydroxylase deficiency diagnosed in childhood with documented (at any time) elevated 17-OHP and with or without elevated A4 and currently treated with hydrocortisone, prednisone, prednisolone or dexamethasone (or a combination of the aforementioned glucocorticoids) and on stable glucocorticoid therapy for a minimum of 3 months.

• Participants who are receiving fludrocortisone must be on a documented stable dose for a minimum of 3 months prior to enrollment and must have stable renin levels at baseline.

• Female participants of childbearing potential and all male participants must agree to the use of an accepted method of contraception during the study.

• A female participant is eligible to participate if she is not pregnant, not breastfeeding, and she is either not a woman of childbearing potential (WOCBP) or has a negative pregnancy test at entry into the study. Note: females presenting with oligomenorrhea or amenorrhea who are aged ≤55 years should be considered potentially fertile and therefore should undergo pregnancy testing like all other female participants.

• Capable of giving signed informed consent/assent which includes compliance with requirements and restrictions listed in the informed consent form (ICF) and in this protocol.

Exclusion Criteria:

• Clinical or biochemical evidence of hepatic or renal disease e.g. creatinine >2 times the upper limit of normal (ULN) or elevated liver function tests (alanine aminotransferase [ALT] or aspartate aminotransferase [AST] >2 times the ULN).

• History of bilateral adrenalectomy.

• History of malignancy (other than basal cell carcinoma successfully treated >26 weeks prior to entry into the study).

• Participants who have type 1 diabetes or receive regular insulin.

• Persistent signs of adrenal insufficiency or the participant does not tolerate treatment at the end of the 4-week run-in period.

• Participants with any other significant medical or psychiatric conditions that in the opinion of the Investigator would preclude participation in the study.

• Participants on regular daily inhaled, topical, nasal or oral steroids for any indication other than CAH.

• Co-morbid condition requiring daily administration of a medication or consumption of any material that interferes with the metabolism of glucocorticoids.

• Participants who are receiving <10 mg hydrocortisone dose at baseline or the hydrocortisone dose equivalent.

• Participants anticipating regular prophylactic use of additional steroids e.g. for strenuous exercise.

• Participation in another clinical study of an investigational or licensed drug or device within the 12 weeks prior to screening.

• Inclusion in any natural history or translational research study that would require evaluation of androgen levels during the study period outside of this protocol's assessments.

• Participants who have previously been exposed to Chronocort in any Diurnal study.

• Participants who routinely work night shifts and so do not sleep during the usual night-time hours.

• Participants, who in the opinion of the Investigator, will be unable to comply with the requirements of the protocol.