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| Name | Class |
|---|---|
| National Institutes of Health (NIH) | NIH |
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The purpose of this study is to gather safety and effectiveness information about a new formulation of Hydrocortisone (Chronocort®) used to treat patients with a disease called congenital adrenal hyperplasia (CAH). Hydrocortisone is the man-made version of the hormone cortisol, which is released in the body following a regular daily pattern. The objective of the study is to measure the levels of hydrocortisone that are absorbed into the bloodstream once Chronocort® is taken and what affects it has on other hormones in the body. Since Chronocort® is anticipated to mimic the same release pattern of cortisol in the body, it is hoped that patients with CAH will be treated more effectively to manage their disease.
This study is a Phase 2 pilot study to characterize and examine the pharmacokinetics and efficacy profile of Chronocort® in adults with congenital adrenal hyperplasia (CAH). It is designed as a two-part, single cohort, open label, multiple dose Phase 2 pilot study to: (Part A) characterize and examine the pharmacokinetics (PK) and disease bio-marker behavior following short-term dosing with Chronocort®; and to (Part B) examine the disease control after six months dose titration with Chronocort® in adults with CAH.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Hydrocortisone Modified Release Capsules | Experimental | Chronocort Modified Release Capsules, 5mg, 10mg and 20mg Dosing frequency twice-daily (mane and nocte) Dose setting by titration to achieve optimal biochemical and therapeutic response |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Hydrocortisone Modified Release Capsules | Drug | Patients with congenital adrenal hyperplasia standardised on conventional therapy is enrolled onto the study and treatment is switched to Chronocort, initially for pharmacokinetic assessment followed by longer-term biochemical and efficacy assessment |
| Measure | Description | Time Frame |
|---|---|---|
| Pharmacokinetic Profile (Cmax) Following Short-term Treatment With Chronocort® in Adult Patients With Congenital Adrenal Hyperplasia | The maximum plasma concentration (Cmax) of chronocort | 24 hours |
| Pharmacokinetic Profile (AUC0-24) Following Short-term Treatment With Chronocort® in Adult Patients With Congenital Adrenal Hyperplasia | Area under the curve (AUC) from 0 to 24 hours (sampling occurs at the following timepoints: 2300, 0100, 0300, 0500, 0600, 0700, 0800, 0900, 1000, 1100, 1200, 1300, 1400, 1500, 1600, 1700, 1900, 2100, 2300hrs) | 24 hours (at 2300, 0100, 0300, 0500, 0600, 0700, 0800, 0900, 1000, 1100, 1200, 1300, 1400, 1500, 1600, 1700, 1900, 2100, 2300hrs) |
| Pharmacokinetic Profile (Tmax) Following Short-term Treatment With Chronocort® in Adult Patients With Congenital Adrenal Hyperplasia | Time to maximum plasma concentration (tmax) | 24 hours |
| Measure | Description | Time Frame |
|---|---|---|
| The Percentage of Patients With 17-OHP and Androstenedione Levels at 0700h Within Proposed Optimal Ranges Whilst on Chronocort and Whilst on Standard Therapy (at Baseline) | Proposed optimal ranges of 17-OHP: 300-1200ng/dl Proposed optimal ranges of androstenedione: 40-150ng.dl for males and 30-200ng/dl for females | Specific time point (0700hrs) |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Deborah P Merke, BS, MS, MD | National Institutes of Health Clinical Center (CC) | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| National Institutes of Health Clinical Center | Bethesda | Maryland | 20892-1932 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 25494662 | Derived | Mallappa A, Sinaii N, Kumar P, Whitaker MJ, Daley LA, Digweed D, Eckland DJ, Van Ryzin C, Nieman LK, Arlt W, Ross RJ, Merke DP. A phase 2 study of Chronocort, a modified-release formulation of hydrocortisone, in the treatment of adults with classic congenital adrenal hyperplasia. J Clin Endocrinol Metab. 2015 Mar;100(3):1137-45. doi: 10.1210/jc.2014-3809. Epub 2014 Dec 11. |
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| ID | Title | Description |
|---|---|---|
| FG000 | Hydrocortisone Modified Release Capsules | Chronocort Modified Release Capsules, 5mg, 10mg and 20mg Dosing frequency twice-daily (mane and nocte) Dose setting by titration to achieve optimal biochemical and therapeutic response Hydrocortisone Modified Release Capsules: Patients with congenital adrenal hyperplasia standardised on conventional therapy is enrolled onto the study and treatment is switched to Chronocort, initially for pharmacokinetic assessment followed by longer-term biochemical and efficacy assessment |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
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| 17-OHP Levels at 0700h, 1700h and 2300h | 17-OHP levels at 0700h, 1700h and 2300h | Specified time points (0700h, 1700h and 2300h) |
| Androstenedione Levels at 0700h, 1700h and 2300h | Androstenedione levels at 0700h, 1700h and 2300h | Specified time points (0700h, 1700h and 2300h) |
| ACTH Levels at 0700h, 1700h and 2300h | ACTH levels at 0700h, 1700h and 2300h | Specified time points (0700h, 1700h and 2300h) |
| AUC Values (Nmol*h/L) for Androstenedione | AUC values (nmol*h/L) for Androstenedione for the following reporting periods: 24 hours (2300-2300h), 2300-0700h, 0700-1500h and 1500-2300h | Specific time points (2300-2300h, 2300-0700h, 0700-1500h and 1500-2300h) |
| AUC Values (Nmol*h/L) for 17-OHP | AUC values (nmol*h/L) for 17-OHP for the following reporting periods: 24 hours (2300-2300h), 2300-0700h, 0700-1500h and 1500-2300h | Specific time points (2300-2300h, 2300-0700h, 0700-1500h and 1500-2300h) |
| AUC Values (Pmol*h/L) for ACTH | AUC values (pmol*h/L) for ACTH for the following reporting periods: 24 hours (2300-2300h), 2300-0700h, 0700-1500h and 1500-2300h | Specific time points (2300-2300h, 2300-0700h, 0700-1500h and 1500-2300h) |
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| NOT COMPLETED |
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All treated subjects
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| ID | Title | Description |
|---|---|---|
| BG000 | Hydrocortisone Modified Release Capsules | Chronocort Modified Release Capsules, 5mg, 10mg and 20mg Dosing frequency twice-daily (mane and nocte) Dose setting by titration to achieve optimal biochemical and therapeutic response Hydrocortisone Modified Release Capsules: Patients with congenital adrenal hyperplasia standardised on conventional therapy is enrolled onto the study and treatment is switched to Chronocort, initially for pharmacokinetic assessment followed by longer-term biochemical and efficacy assessment |
| Units | Counts |
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| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Region of Enrollment | Number | participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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| Primary | Pharmacokinetic Profile (Cmax) Following Short-term Treatment With Chronocort® in Adult Patients With Congenital Adrenal Hyperplasia | The maximum plasma concentration (Cmax) of chronocort | All treated subjects | Posted | Mean | Standard Deviation | nmol/L | 24 hours |
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| Primary | Pharmacokinetic Profile (AUC0-24) Following Short-term Treatment With Chronocort® in Adult Patients With Congenital Adrenal Hyperplasia | Area under the curve (AUC) from 0 to 24 hours (sampling occurs at the following timepoints: 2300, 0100, 0300, 0500, 0600, 0700, 0800, 0900, 1000, 1100, 1200, 1300, 1400, 1500, 1600, 1700, 1900, 2100, 2300hrs) | All treated subjects | Posted | Mean | Standard Deviation | h*nmol/L | 24 hours (at 2300, 0100, 0300, 0500, 0600, 0700, 0800, 0900, 1000, 1100, 1200, 1300, 1400, 1500, 1600, 1700, 1900, 2100, 2300hrs) |
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| Primary | Pharmacokinetic Profile (Tmax) Following Short-term Treatment With Chronocort® in Adult Patients With Congenital Adrenal Hyperplasia | Time to maximum plasma concentration (tmax) | All treated subjects | Posted | Mean | Standard Deviation | Hours | 24 hours |
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| Secondary | The Percentage of Patients With 17-OHP and Androstenedione Levels at 0700h Within Proposed Optimal Ranges Whilst on Chronocort and Whilst on Standard Therapy (at Baseline) | Proposed optimal ranges of 17-OHP: 300-1200ng/dl Proposed optimal ranges of androstenedione: 40-150ng.dl for males and 30-200ng/dl for females | Posted | Number | percentage of participants | Specific time point (0700hrs) |
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| Secondary | 17-OHP Levels at 0700h, 1700h and 2300h | 17-OHP levels at 0700h, 1700h and 2300h | Posted | Mean | Standard Deviation | nmol/L | Specified time points (0700h, 1700h and 2300h) |
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| Secondary | Androstenedione Levels at 0700h, 1700h and 2300h | Androstenedione levels at 0700h, 1700h and 2300h | Posted | Mean | Standard Deviation | nmol/L | Specified time points (0700h, 1700h and 2300h) |
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| Secondary | ACTH Levels at 0700h, 1700h and 2300h | ACTH levels at 0700h, 1700h and 2300h | Posted | Mean | Standard Deviation | pmol/L | Specified time points (0700h, 1700h and 2300h) |
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| Secondary | AUC Values (Nmol*h/L) for Androstenedione | AUC values (nmol*h/L) for Androstenedione for the following reporting periods: 24 hours (2300-2300h), 2300-0700h, 0700-1500h and 1500-2300h | Posted | Mean | Standard Deviation | nmol*h/L | Specific time points (2300-2300h, 2300-0700h, 0700-1500h and 1500-2300h) |
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| Secondary | AUC Values (Nmol*h/L) for 17-OHP | AUC values (nmol*h/L) for 17-OHP for the following reporting periods: 24 hours (2300-2300h), 2300-0700h, 0700-1500h and 1500-2300h | Posted | Mean | Standard Deviation | nmol*h/L | Specific time points (2300-2300h, 2300-0700h, 0700-1500h and 1500-2300h) |
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| Secondary | AUC Values (Pmol*h/L) for ACTH | AUC values (pmol*h/L) for ACTH for the following reporting periods: 24 hours (2300-2300h), 2300-0700h, 0700-1500h and 1500-2300h | Posted | Mean | Standard Deviation | pmol*h/L | Specific time points (2300-2300h, 2300-0700h, 0700-1500h and 1500-2300h) |
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Approximately 7 months
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Hydrocortisone Modified Release Capsules | Chronocort Modified Release Capsules, 5mg, 10mg and 20mg Dosing frequency twice-daily (mane and nocte) Dose setting by titration to achieve optimal biochemical and therapeutic response Hydrocortisone Modified Release Capsules: Patients with congenital adrenal hyperplasia standardised on conventional therapy is enrolled onto the study and treatment is switched to Chronocort, initially for pharmacokinetic assessment followed by longer-term biochemical and efficacy assessment | 0 | 16 | 16 | 16 |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Headache | Nervous system disorders | MedDRA (16.1) | Non-systematic Assessment |
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| Fatigue | General disorders | MedDRA (16.1) | Non-systematic Assessment |
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| Anaemia | Blood and lymphatic system disorders | MedDRA (16.1) | Non-systematic Assessment |
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| Dizziness | Nervous system disorders | MedDRA (16.1) | Non-systematic Assessment |
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| Weight increase | Investigations | MedDRA (16.1) | Non-systematic Assessment |
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| Appetite decrease | Metabolism and nutrition disorders | MedDRA (16.1) | Non-systematic Assessment |
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| Appetite increase | Metabolism and nutrition disorders | MedDRA (16.1) | Non-systematic Assessment |
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| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (16.1) | Non-systematic Assessment |
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| Insomnia | Psychiatric disorders | MedDRA (16.1) | Non-systematic Assessment |
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| Acne | Skin and subcutaneous tissue disorders | MedDRA (16.1) | Non-systematic Assessment |
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| Asthenia | General disorders | MedDRA (16.1) | Non-systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| CEO | Diurnal Ltd. | +44 (0) 871 716 8848 | info@diurnal.co.uk |
| ID | Term |
|---|---|
| D004700 | Endocrine System Diseases |
| D000309 | Adrenal Insufficiency |
| D000312 | Adrenal Hyperplasia, Congenital |
| ID | Term |
|---|---|
| D000307 | Adrenal Gland Diseases |
| D047808 | Adrenogenital Syndrome |
| D012734 | Disorders of Sex Development |
| D014564 | Urogenital Abnormalities |
| D052776 | Female Urogenital Diseases |
| D005261 | Female Urogenital Diseases and Pregnancy Complications |
| D000091642 | Urogenital Diseases |
| D052801 | Male Urogenital Diseases |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D030342 | Genetic Diseases, Inborn |
| D043202 | Steroid Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D006058 | Gonadal Disorders |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
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| Unknown or Not Reported |
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