Phase 3
N=91
A Study of the Efficacy, Safety and Tolerability of Chronocort in Treating CAH
Congenital Adrenal Hyperplasia
Bottom Line
View on ClinicalTrials.gov: NCT03062280 ↗Enrolled (actual)
91
Serious AEs
30.8%
Results posted
Oct 2024
Primary outcome: Primary: Safety and Tolerability - Number of Participants With Adrenal Insufficiency — 25; 41 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Hydrocortisone (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Neurocrine UK Limited
- Primary completion
- Jul 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Safety and Tolerability - Number of Participants With Adrenal Insufficiency |
25; 41 | — |
| PRIMARY Safety and Tolerability - Number of Participants Who Used Sick Day Medication |
79; 47 | — |
| PRIMARY Safety and Tolerability - Number of Participants Who Experienced at Least One Adrenal Crisis |
7 | — |
| PRIMARY Safety and Tolerability - Number of Participants With Adverse Events (AEs) |
90; 37; 80; 1; 7; 0 | — |
| PRIMARY Safety Laboratory Assessments - Number of Participants With a Shift in Laboratory Parameters Baseline Values to the Maximum Values |
6; 71; 5; 66; 5; 70 | — |
| PRIMARY Safety Laboratory Assessments - Number of Participants With a Shift in Laboratory Parameters Baseline Values to the Minimum Values |
5; 72; 13; 58; 12; 63 | — |
| PRIMARY Safety and Tolerability - Change From Pre-Chronocort Baseline to Month 30 in Vital Signs - Diastolic Blood Pressure |
2.7 | — |
| PRIMARY Safety and Tolerability - Change From Pre-Chronocort Baseline to Month 30 in Vital Signs - Systolic Blood Pressure |
-0.1 | — |
| PRIMARY Safety and Tolerability - Change From Pre-Chronocort Baseline to Month 30 in Vital Signs - Pulse Rate |
0.2 | — |
| PRIMARY Safety and Tolerability - Change From Pre-Chronocort Baseline to Month 30 in Vital Signs - Respiratory Rate |
-0.2 | — |
| PRIMARY Safety and Tolerability - Change From Pre-Chronocort Baseline to Month 30 in Vital Signs - Body Temperature |
0.03 | — |
| PRIMARY Safety and Tolerability - Change From Pre-Chronocort Baseline to Month 30 in Vital Signs - Body Weight |
0.14 | — |
| PRIMARY Safety and Tolerability - Change From Pre-Chronocort Baseline to Month 30 in Vital Signs - BMI |
0.086 | — |
| PRIMARY Safety and Tolerability - Change From Pre-Chronocort Baseline to Month 30 in Vital Signs - Waist Circumference |
1.95 | — |
| SECONDARY Total Daily Dose of Chronocort in Milligrams (mg)/Day of Hydrocortisone During the Time Interval Baseline to Week 4 and Month 18 to Month 24 |
30.00; 20.00 | — |
| SECONDARY Total Daily Dose of Chronocort in mg/Day/m^2 of Hydrocortisone by BSA During the Time Interval Baseline to Week 4 and Month 18 to Month 24 |
15.764; 11.766 | — |
| SECONDARY Number of Participants Achieving Disease Control at 09:00 Hours and 13:00 Hours for 17-hydroxyprogesterone (17-OHP) |
51; 39; 54; 33; 64; 27 | — |
| SECONDARY Number of Participants Achieving Disease Control at 09:00 Hours and 13.00 Hours for Androstenedione (A4) |
48; 42; 49; 38; 43; 48 | — |
| SECONDARY Change From Pre-Chronocort Baseline at Month 24 in Bone Turnover Markers - Osteocalcin |
19.486; 3.535 | — |
| SECONDARY Change From Pre-Chronocort Baseline at Month 24 in Bone Turnover Markers - C-Terminal Cross-linked Telopeptide (CTX) |
540.1; -43.6 | — |
| SECONDARY Change From Pre-Chronocort Baseline at Month 24 in Total Testosterone |
17.1490; 0.9535; -1.5850; -0.2892 | — |
| SECONDARY Change From Pre-Chronocort Baseline at Month 24 in Fasting Insulin |
77.8; -6.1 | — |
| SECONDARY Change From Pre-Chronocort Baseline at Month 24 in Fasting Glucose |
5.09; -0.02 | — |
| SECONDARY Change From Pre-Chronocort Baseline at Month 24 in Glycated Hemoglobin (HbA1c) |
5.17; 0.12 | — |
| SECONDARY Change From Pre-Chronocort Baseline at Month 24 in High Sensitivity C-reactive Protein (hsCRP) |
1.78; 0.56 | — |
| SECONDARY Change From Pre-Chronocort Baseline at Month 24 in Plasma Renin Activity (PRA) |
0.878; 0.010 | — |
| SECONDARY Change From Pre-Chronocort Baseline at Month 24 in Body Composition - Total Lean Mass |
45.819; -0.563 | — |
| SECONDARY Change From Pre-Chronocort Baseline at Month 24 in Body Composition - Bone Mineral Density |
1.0940; -0.0008 | — |
| SECONDARY Change From Pre-Chronocort Baseline at Month 24 in Body Composition - Total Fat Mass |
27.943; 0.811 | — |
| SECONDARY Change From Pre-Chronocort Baseline in Quality of Life - Medical Outcome Short Form Health Survey Form 36 (SF-36) Score at Months 12 and 18 |
0.362; 0.257; 0.496; 0.516 | — |
| SECONDARY Change From Pre-Chronocort Baseline in Quality of Life - Multidimensional Assessment of Fatigue (MAF) Global Fatigue Index (GFI) Score at Months 12 and 18 |
-1.76; -2.21 | — |
| SECONDARY Change From Pre-Chronocort Baseline in Quality of Life - Standardized Health Questionnaire 5-Dimension (EQ-5D) Index Score and Visual Analogue Scale (VAS) Score at Months 12 and 18 |
-0.022; -0.004; 1.529; 1.819 | — |
| SECONDARY Number of Participants With Dose Titrations |
0; 0; 0; 91; 2; 17 | — |
Summary
Subjects completing study DIUR-005 and those who have already completed study DIUR-003 will be offered the opportunity either to continue Chronocort® therapy or to switch from their current glucocorticoid therapy to Chronocort® in this open-label study.
Eligibility Criteria
Inclusion Criteria
- Subjects with CAH who have successfully completed a clinical trial with the current formulation of Chronocort®.
- Provision of signed written informed consent.
Exclusion Criteria
- Co-morbid condition requiring daily administration of a medication (or use of any medications/supplements) that interferes with the metabolism of glucocorticoids.
- Clinical or biochemical evidence of hepatic or renal disease. Creatinine over twice the ULN or elevated liver function tests (ALT or AST >2 times ULN]).
- Females who are pregnant or lactating.
- Subjects on regular daily inhaled, topical, nasal or oral steroids for any indication other than CAH.
- History of malignancy (other than basal cell carcinoma successfully treated >6 months prior to entry into the study).
- Subjects with a history of bilateral adrenalectomy.
- Participation in another clinical trial of an investigational or licensed drug or device within the 3 months prior to inclusion in this study, except for another clinical trial with the current formulation of Chronocort®.
- Subjects unable to comply with the requirements of the protocol.
- Subjects who routinely work night shifts and so do not sleep during the usual nighttime hours.
Data sourced from ClinicalTrials.gov (NCT03062280). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.