An Open-label Extension Study to Evaluate the Long-term Safety and Tolerability of Chronocort in the Treatment of Participants Aged 16 Years and Over with Congenital Adrenal Hyperplasia

A Phase 3 Open-label Extension Study to Evaluate the Long-term Safety and Tolerability of Chronocort in the Treatment of Participants Aged 16 Years and Over with Congenital Adrenal Hyperplasia

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2021-004467-26-FR

Enrollment

Registered

2021-12-09

Start date

2022-02-08

Completion date

Unknown

Last updated

2025-04-14

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Congenital Adrenal Hyperplasia due to 21-hydroxylase deficiency

Interventions

Trade Name: Efmody 5 mg modified release hard capsules Product Name: Chronocort 5mg Pharmaceutical Form: Modified-release capsule, hard INN or Proposed INN: HYDROCORTISONE Other descriptive name: HYD

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. Participants with CAH who have successfully completed Chronocort study DIUR-006 (sites in France and US only) or study DIUR-014. 2. Participants who are capable of giving signed informed consent/assent as described in Appendix 1 which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol. Are the trial subjects under 18? yes Number of subjects for this age range: 4 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 77 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range 0

Exclusion criteria

Exclusion criteria: Medical Conditions 1. Participants with 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). 2. Participants with a history of malignancy (other than basal cell carcinoma successfully treated >26 weeks prior to entry into the study). 3. Participants with a history of bilateral adrenalectomy. 4. Participants with any other significant medical or psychiatric conditions that in the opinion of the Investigator would preclude participation in the study. Prior/Concomitant Therapy 5. Participants with a co-morbid condition requiring daily administration of a medication or consumption of any material that interferes with the metabolism of glucocorticoids (examples at http://medicine.iupui.edu/clinpharm/ddis/clinical-table/). 6. Participants on regular daily inhaled, topical, nasal, or oral steroids for any indication other than CAH. 7. Participants anticipating regular prophylactic use of additional steroids e.g., for strenuous exercise. Prior/Concurrent Clinical Study Experience 8. Participation in another clinical study of an investigational or licensed drug or device within 3 months prior to inclusion in this study, except for another clinical study with the current formulation of Chronocort. Other Exclusions 9. Females who are pregnant or lactating. 10. Participants, who in the opinion of the Investigator, will be unable to comply with the requirements of the protocol. 11. Participants who routinely work night shifts and so do not sleep during the usual night-time hours. 12. Participants with a body weight of 50 kg or less. (Note: this exclusion criterion is only applicable for French sites).

Design outcomes

Secondary

Measure	Time frame
Secondary end point(s): 1. Change from pre-Chronocort baseline in total daily steroid dose in hydrocortisone equivalent dose at each visit. 2. Change in 17-OHP levels from pre-Chronocort baseline at each visit. 3. Change in A4 levels from pre-Chronocort baseline at each visit. 4. Change from pre-Chronocort baseline to each visit in menstrual regularity (only in pre-menopausal females without hysterectomy and not using hormonal contraceptives) 5. Change from pre-Chronocort baseline to each visit in luteinising hormone (LH) levels (only in men). 6. Change in testosterone from pre-Chronocort baseline at each study visit. 7. Change from pre-Chronocort baseline to each visit in waist circumference. 8. Change from pre-Chronocort baseline to each visit in body weight.;Timepoint(s) of evaluation of this end point: December 2024 (end of trial)	—

Measure

Time frame

Secondary end point(s): 1. Change from pre-Chronocort baseline in total daily steroid dose in hydrocortisone equivalent dose at each visit. 2. Change in 17-OHP levels from pre-Chronocort baseline at each visit. 3. Change in A4 levels from pre-Chronocort baseline at each visit. 4. Change from pre-Chronocort baseline to each visit in menstrual regularity (only in pre-menopausal females without hysterectomy and not using hormonal contraceptives) 5. Change from pre-Chronocort baseline to each visit in luteinising hormone (LH) levels (only in men). 6. Change in testosterone from pre-Chronocort baseline at each study visit. 7. Change from pre-Chronocort baseline to each visit in waist circumference. 8. Change from pre-Chronocort baseline to each visit in body weight.;Timepoint(s) of evaluation of this end point: December 2024 (end of trial)

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Primary

Measure	Time frame
Main Objective: To evaluate the long-term safety and tolerability of Chronocort in the treatment of participants with CAH.;Secondary Objective: 1. To assess the impact of treatment on dose of steroid required. 2. To assess the impact of treatment on 17-OHP levels. 3. To assess the impact of treatment on A4 levels. 4. To assess the impact of treatment on markers of fertility. 5. To assess the impact of treatment on testosterone by sex. 6. To assess the impact of treatment on waist circumference. 7. To assess the impact of treatment on body weight.;Primary end point(s): The safety of Chronocort over time, assessed using, but not limited to, the following outcome variables: • Signs and symptoms of adrenal insufficiency or over-treatment throughout the study. • Use of IRHC from the emergency packs for stress dosing or use of any additional glucocorticoid treatment during the study. • Occurrence of adrenal crises throughout the study. • Occurrence of AEs throughout the study. • Change from pre-Chronocort baseline in safety laboratory assessments at each visit throughout the study. • Change from pre-Chronocort baseline in vital signs at each visit throughout the study.;Timepoint(s) of evaluation of this end point: December 2024 (end of trial)	—

Measure

Time frame

Main Objective: To evaluate the long-term safety and tolerability of Chronocort in the treatment of participants with CAH.;Secondary Objective: 1. To assess the impact of treatment on dose of steroid required. 2. To assess the impact of treatment on 17-OHP levels. 3. To assess the impact of treatment on A4 levels. 4. To assess the impact of treatment on markers of fertility. 5. To assess the impact of treatment on testosterone by sex. 6. To assess the impact of treatment on waist circumference. 7. To assess the impact of treatment on body weight.;Primary end point(s): The safety of Chronocort over time, assessed using, but not limited to, the following outcome variables: • Signs and symptoms of adrenal insufficiency or over-treatment throughout the study. • Use of IRHC from the emergency packs for stress dosing or use of any additional glucocorticoid treatment during the study. • Occurrence of adrenal crises throughout the study. • Occurrence of AEs throughout the study. • Change from pre-Chronocort baseline in safety laboratory assessments at each visit throughout the study. • Change from pre-Chronocort baseline in vital signs at each visit throughout the study.;Timepoint(s) of evaluation of this end point: December 2024 (end of trial)

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Countries

France, Japan, United States

Contacts

Public ContactCardiff office contact information

Neurocrine UK Limited

infoEU@neurocrine.com442920682069

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026