Long-term Safety Study of Chronocort in the Treatment of Participants With Congenital Adrenal Hyperplasia

NCT05299554 | Completed Phase 3 FDA Drug

• 2 other identifiers: DIUR-0152021-004467-26
• Study Type: interventional
• Target: 76
• Locations: 3 countries
• Sites: 21

Brief Summary

This phase III study is an open-label extension study to be conducted at approximately 21 investigational sites across 3 countries. The study will evaluate the long-term safety and tolerability of Chronocort in participants aged 16 years and over when used as treatment for Congenital Adrenal Hyperplasia (CAH).

Conditions

Congenital Adrenal Hyperplasia

Keywords

Extension study

Outcome Measures

Primary Outcomes (8)

• Signs and symptoms of over-treatment [Safety and Tolerability] throughout the study.

  Over-replacement normally presents with chronic effects such as weight gain, increased appetite, sleeping difficulties, increased acne and Cushingoid syndrome.

  Up to 32 months

• Signs and symptoms of under-treatment [Safety and Tolerability] throughout the study.

  Under-replacement normally presents with acute effects such as sudden weight loss, lack of appetite, nausea, vomiting, headache, blurred vision, fatigue, weakness, dizziness, light-headedness and syncope.

  Up to 32 months.

• To measure signs or symptoms of under treatment [Safety and Tolerability] in terms of use of additional glucocorticoid treatment throughout the study.

  Use of Immediate Release Hydrocortisone (IRHC) from the emergency packs for stress dosing or use of any additional glucocorticoid treatment.

  Up to 32 months

• To measure signs or symptoms of under treatment [Safety and Tolerability] in terms of incidence of adrenal crises throughout the study.

  Occurrence of adrenal crises throughout the study.

  Up to 32 months

• To measure the incidence of Treatment-Emergent Adverse Events [Safety and Tolerability].

  The incidence, nature, severity, relatedness, duration, outcome, seriousness, and expectedness of treatment-emergent adverse events (TEAEs) throughout the study.

  Up to 32 months

• To measure the change from pre-Chronocort baseline in terms of hematology safety laboratory assessments [Safety and Tolerability].

  Lab parameters will be summarized and compared throughout the study as follows: Platelet count, Red blood cell count (RBC), Haemoglobin, Haematocrit, RBC Indices: Mean corpuscular volume (MCV), Mean cell haemoglobin (MCH). Mean cell haemoglobin concentration (MCHC), Red cell distribution width (RDW). White blood cell (WBC) count with differential (absolute and %): Neutrophils, Lymphocytes, Monocytes, Eosinophils, Basophils.

  Up to 32 months

• To measure the change from pre-Chronocort baseline in terms of clinical chemistry safety laboratory assessments [Safety and Tolerability].

  To measure the change from pre-Chronocort baseline in terms of clinical chemistry safety laboratory assessments \[Safety and Tolerability\]. Blood urea nitrogen (BUN), Creatinine, Chloride, Total magnesium, Potassium, Sodium, Calcium, Total carbon dioxide (CO2), Inorganic phosphorus, AST/serum glutamic-oxaloacetic transaminase (SGOT), ALT/serum glutamic-pyruvic transaminase (SGPT), Alkaline phosphatase, Albumin, Total and direct bilirubin, Total protein, Lactate dehydrogenase (LDH), Total creatine kinase (CK), Uric acid.

  Up to 32 months

• To measure the change from pre-Chronocort baseline in terms of vital signs assessments.

  Blood pressure measurements throughout the study will be summarised and compared.

  Up to 32 months

Secondary Outcomes (8)

• To assess the impact of treatment on dose of steroid required - Change from pre-Chronocort baseline

  Up to 32 months

• To assess the impact of treatment on 17-Hydroxyprogesterone (17-OHP) levels - Change from pre-Chronocort baseline

  Up to 32 months

• To assess the impact of treatment on Androstenedione (A4) levels - Change from pre-Chronocort baseline

  Up to 32 months

• To assess the impact of treatment on menstrual regularity (recorded in a participant diary) as a marker of fertility - Change from pre-Chronocort baseline

  Up to 32 months

• To assess the impact of treatment on luteinising hormone (LH) levels in men as a marker of fertility - Change from pre-Chronocort baseline

  Up to 32 months

Other Outcomes (10)

• To assess the impact of treatment on the dose of steroid - Change from initial study baseline daily dose

  Up to 32 months

• To assess the impact of treatment on 17-Hydroxyprogesterone (17-OHP) levels - Change from initial study baseline

  Up to 32 months

• To assess the impact of treatment on Androstenedione (A4) levels - Change from initial study baseline

  Up to 32 months

Study Arms (1)

Chronocort (hydrocortisone modified-release capsule)

EXPERIMENTAL

Chronocort (hydrocortisone modified-release capsules) supplied as 5 mg and 10 mg per capsule for oral administration.

Drug: Chronocort

Interventions

Chronocort DRUG

Hydrocortisone modified-release capsule 5 mg and 10 mg

Also known as: Hydrocortisone modified-release hard capsule

Chronocort (hydrocortisone modified-release capsule)

Eligibility Criteria

• Age: 16 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Participants with Congenital Adrenal Hyperplasia (CAH) who have successfully completed Chronocort study DIUR-006 (sites in France and US only) or study DIUR-014.
• Participants who are capable of giving signed informed consent/assent, which includes compliance with the requirements and restrictions listed in the study's informed consent form (ICF) and in the protocol.

You may not qualify if:

• 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).
• Participants with a history of malignancy (other than basal cell carcinoma successfully treated \>26 weeks prior to entry into the study).
• Participants with a history of bilateral adrenalectomy.
• Participants with any other significant medical or psychiatric conditions that in the opinion of the Investigator would preclude participation in the study.
• Participants with a co-morbid condition requiring daily administration of a medication or consumption of any material that interferes with the metabolism of glucocorticoids.
• Participants on regular daily inhaled, topical, nasal, or oral steroids for any indication other than CAH.
• Participants anticipating regular prophylactic use of additional steroids e.g., for strenuous exercise.
• Females who are pregnant or lactating.
• Participants, who in the opinion of the Investigator, will be unable to comply with the requirements of the protocol.
• Participants who routinely work night shifts and so do not sleep during the usual night-time hours.

Sponsors & Collaborators

• Neurocrine UK Limited: lead

Study Sites (21)

Neurocrine Investigational Site in California

Los Angeles, California, 90027, United States

Location

Neurocrine Investigational Site in California

Orange, California, 92868, United States

Location

Neurocrine Investigational Site in Florida

Jacksonville, Florida, 32207, United States

Location

Neurocrine Investigational Site in Iowa

Iowa City, Iowa, 52242, United States

Location

National Institutes of Health Center

Bethesda, Maryland, 20892-1932, United States

Location

Neurocrine Investigational Site in Michigan

Ann Arbor, Michigan, 48114, United States

Location

Neurocrine Investigational Site in Minnesota

Rochester, Minnesota, 55901, United States

Location

Neurocrine Investigational Site in Nevada

Las Vegas, Nevada, 89148, United States

Location

Neurocrine Investigational Site in Texas

Dallas, Texas, 75235, United States

Location

Neurocrine Investigational Site in Washington

Seattle, Washington, 98105, United States

Location

Neurocrine Investigational Site in Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

Neurocrine Investigational Site in Caen

Caen, 14033, France

Location

Neurocrine investigational Site in Lyon

Lyon, 69677, France

Location

Neurocrine Investigational Site in Paris

Paris, 75651, France

Location

Neurocrine Investigational Site in Pessac

Pessac, 33604, France

Location

Neurocrine Investigational Site in Toulouse (Children's hospital)

Toulouse, 31059, France

Location

Neurocrine Investigational Site in Toulouse

Toulouse, 31059, France

Location

Neurocrine Investigational Site in Asahi-ku

Yokohama, Kanagawa, 241-0811, Japan

Location

Neurocrine Investigational Site in Yushima

Bunkyō-Ku, Tokyo, 113-8519, Japan

Location

Neurocrine Investigational Site in Okura

Setagaya-Ku, Tokyo, 157-8535, Japan

Location

Neurocrine Investigational Site in Toyama

Shinjuku-Ku, Tokyo, 162-8655, Japan

Location

MeSH Terms

Conditions

Adrenal Hyperplasia, Congenital

Condition Hierarchy (Ancestors)

Adrenogenital Syndrome; Disorders of Sex Development; Urogenital Abnormalities; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn; Steroid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Metabolic Diseases; Nutritional and Metabolic Diseases; Adrenal Gland Diseases; Endocrine System Diseases; Gonadal Disorders

Study Officials

• D Merke

  National Instiututes of Health Clinical Centre, Bethesda, Maryland, United States, 20892-1932

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Single arm extension study

Study Record Dates

• First Submitted: January 5, 2022
• First Posted: March 29, 2022
• Study Start: April 1, 2022
• Primary Completion: January 15, 2026
• Study Completion: January 15, 2026
• Last Updated: February 4, 2026

Record last verified: 2026-02

Locations

JP (4); US (11); FR (6)