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Phase 2 N=16 Treatment

Pilot Study to Characterize and Examine the Pharmacokinetics and Efficacy of Chronocort® in Adults With CAH

Endocrine Disease · Adrenal Insufficiency · Congenital Adrenal Hyperplasia

Enrolled (actual)
16
Serious AEs
0.0%
Results posted
May 2017
Primary outcome: Primary: Pharmacokinetic Profile (Cmax) Following Short-term Treatment With Chronocort® in Adult Patients With Congenital Adrenal Hyperplasia — 601.213 nmol/L

Study Design & Population

Study type
Interventional
Phase
Phase 2
Interventions
Hydrocortisone Modified Release Capsules (Drug)
Age
Adult, Older Adult · 18+ yrs
Sex
All
Sponsor
Neurocrine UK Limited
Primary completion
Dec 2013

Outcome Measures

OutcomeResultp-value
PRIMARY
Pharmacokinetic Profile (Cmax) Following Short-term Treatment With Chronocort® in Adult Patients With Congenital Adrenal Hyperplasia
601.213
PRIMARY
Pharmacokinetic Profile (AUC0-24) Following Short-term Treatment With Chronocort® in Adult Patients With Congenital Adrenal Hyperplasia
5027.641
PRIMARY
Pharmacokinetic Profile (Tmax) Following Short-term Treatment With Chronocort® in Adult Patients With Congenital Adrenal Hyperplasia
7.9
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)
0; 12.5; 56.3; 81.3
SECONDARY
17-OHP Levels at 0700h, 1700h and 2300h
76.97; 70.99; 55.35; 7.72; 23.55; 27.27
SECONDARY
Androstenedione Levels at 0700h, 1700h and 2300h
7.92; 7.54; 7.43; 3.11; 4.62; 5.37
SECONDARY
ACTH Levels at 0700h, 1700h and 2300h
21.06; 20.93; 7.75; 4.16; 4.71; 7.37
SECONDARY
AUC Values (Nmol*h/L) for Androstenedione
166.19; 41.42; 65.33; 59.44; 104.37; 36.02
SECONDARY
AUC Values (Nmol*h/L) for 17-OHP
1407.83; 243.99; 607.74; 556.11; 446.90; 169.83
SECONDARY
AUC Values (Pmol*h/L) for ACTH
356.25; 54.72; 166.18; 135.35; 120.00; 52.22

Summary

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.

Eligibility Criteria

Inclusion Criteria

  • Known CAH due to 21-hydroxylase deficiency (classic CAH) based on hormonal and genetic testing currently treated with hydrocortisone, prednisone, prednisolone or dexamethasone on a stable dosage for a minimum of 3 months.
  • Male or female patients aged 18 and above.
  • Provision of signed written informed consent.
  • Good general health.
  • Females of childbearing potential must have a negative pregnancy test initially and at all visits. Females who are engaging in sexual intercourse must be using a medically acceptable method of contraception (as defined in the protocol, section 10.5).
  • Plasma renin activity must be within the clinically acceptable range at screening (less than 1.5 times upper normal range).

Exclusion Criteria

  • Co-morbid condition requiring daily administration of a medication that induces hepatic enzymes or interferes with the metabolism of glucocorticoids.
  • Clinical or biochemical evidence of hepatic or renal disease. Creatinine above the normal range or elevated liver function tests (ALT or AST) > 2 times the upper limits of normal.
  • Females who are pregnant or lactating.
  • Women taking an estrogen-containing oral contraceptive pill and who have taken it within 6 weeks of recruitment.
  • Patients taking spironolactone.
  • Patients on inhaled or oral steroids apart from treatment for CAH.
  • Patients with any other significant medical or psychiatric conditions that in the opinion of the Investigator would preclude participation in the trial.
  • Participation in another clinical trial of an investigational or licensed drug or device within the 3 months prior to inclusion in this study.
  • Patients with history of bilateral adrenalectomy.
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01735617). 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.

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