Revival of autochthonous adrenocortical stem cells in autoimmune Addison’s disease (RoSA) - Revival of Stem cells in Addison's disease (RoSA)

Conditions: Autoimmune Addison's disease
MedDRA version: 9.1Level: HLTClassification code 10001343Term: Adrenal cortical hypofunction

Registration Number: EUCTR2009-018074-56-GB

Lead Sponsor: ewcastle upon Tyne Hospitals NHS Foundation Trust

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 12

Inclusion Criteria

- Subjects between 16 and 65 years of age
- Adrenocortical failure, as judged by a basal cortisol of less than 100nmol/l or peak ACTH-stimulated cortisol response of less than 300nmol/l that was established a minimum of 1 year previously and persists at run-in assessments
- Elevation of ACTH at diagnosis or during testing in the run-in phase of the study
- Positive adrenal antibodies at baseline or recorded during illness
- Normal or atrophic adrenal glands on CT scan
- Willingness to travel to Newcastle CRF for study
- Willingness to attend education sessions about indications for parenteral glucocorticoid administration and technique of administration
- Female participants of child-bearing age will be willing to take acceptable contraception during study, and for 3 months following last synacthen dose

Are the trial subjects under 18? yes
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
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

- Significant cardio-respiratory, chronic renal or non-autoimmune liver disease; malignancy
- Pregnancy, breast feeding or plan for pregnancy within 9 months
- Known non-autoimmune cause for adrenal failure (haemorrhage, adrenoleukodystrophy etc.)
- Known hypersensitivity or allergy to Synacthen, synacthen depot or zinc salts

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: We wish to find out whether repeated stimulation of adrenocortical stem cells with synacthen (synthetic ACTH peptide) can regenerate adrenal function in established autoimmune Addison's disease (adrenal failure). This might be possible because stem cells are probably not destroyed by the autoimmune attack in Addison's disease, they likely remain dormant because ACTH levels drop following treatment with replacement steroids.;Secondary Objective: Is this a safe regimen of treatment;Primary end point(s): • Restoration of adrenocortical synthetic function, as judged by either: basal cortisol >400nmol/l (rise in 300nmol/l over pre-treatment), or synacthen-stimulated cortisol >550nmol/l (rise in 250nmol/l over pre-treatment) at 30 or 60 min after iv synacthen 250µg.

Secondary Outcome Measures