A trial comparing continuous subcutaneous hydrocortisone infusion (CSHI) therapy with conventional oral glucocorticoid therapy in patients with Congenital Andrenal Hyperplasia (CAH)

Phase 1

• Conditions: Congenital Adrenal Hyperplasia; MedDRA version: 14.1Level: LLTClassification code 10010323Term: Congenital adrenal hyperplasiaSystem Organ Class: 100000004850; Therapeutic area: Diseases [C] - Hormonal diseases [C19]

• Registration Number: EUCTR2011-005822-23-SE
• Lead Sponsor: Haukeland University Hospital

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2013-09-20
• Last Update Posted: 30 October 2017

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 20

Inclusion Criteria

- Patients with verified salt-wasting CAH and simple virilising CAH
- Age 18-60
- On single prednisone, or hydrocortisone treatment
- Written informed consent
- In case of concomitant endocrine/autoimmune diseases, should be on stable treatment during the study period

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 10
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

- Patients with diabetes mellitus
- Cardiovascular disease (Myocardial infarction or angina pectoris within the last 2 years, moderate to severe heart failure)
- Active malignant disease
- Pregnancy or breast feeding
- Pharmacological treatment with glucocorticoids or drugs that induces liver enzymes (antiepileptics, rifampicin, St. Johns wart)
- Intake of grapefruit juice (that inhibits CYP3A4) the last two weeks before or during the study period

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To evaluate the effects of CSHI (physiological) replacement as compared to conventional (unphysiological) glucocorticoid therapy on steroid metabolism, cardiovascular risk markers, bone, clinical status and subjective health;Secondary Objective: Follow-up of Adverse Events;Primary end point(s): Normalised androgen levels (in particular androstenedione and androstenediol-glucoronide) as parameters of adequate suppression of androgen production;Timepoint(s) of evaluation of this end point: 0, 2, 3, 5, 7, 8, 14 months

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): The effect of glucocorticoid replacement on <br>- Steroid and bone metabolism, i.e ACTH, cortisol, bone markers<br>- Cardiovascular risk markers such as fasting glucose/insulin, HbA1c, lipid levels, CRP<br>- Clinical status (BMI, waist circumference and blood pressure)<br>- DXA (body composition, bone mineral density) <br>- Subjective health status (SF-36, AddiQoL) <br>;Timepoint(s) of evaluation of this end point: Steriod and bone metabolism - 0, 2, 3, 5, 7, 8, 14 months<br>Cardiovasdular risk markers - 0, 2, 3, 5, 7, 8, 14 months<br>Clinical status - 0, 2, 3, 5, 7, 8, 14 months<br>DXA - 0, 14 months<br>Subjective health status - 0, 2, 3, 5, 7, 8, 14 months