Revival of Stem Cells in Addison's Study

Adrenal Failure Clinical Trial

— RoSA  

Official title:

Revival of Autochthonous Adrenocortical Stem Cells in Autoimmune Addison's Disease

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01371526
• Other study ID #: RoSAv1.2:04_10: R&D#5252
• Secondary ID: 2009-018074-56
• Status: Completed
• Phase: Phase 4
• First received: March 9, 2011
• Last updated: February 5, 2013
• Start date: September 2010
• Est. completion date: September 2012

Study information

• Verified date: February 2013
• Source: Newcastle University
• Contact: n/a
• Is FDA regulated: No
• Health authority: United Kingdom: Medicines and Healthcare Products Regulatory Agency
• Study type: Interventional

Clinical Trial Summary

Autoimmune Addison's disease (AAD) is a rare and debilitating disease in which an autoimmune attack progressively destroys the adrenal cortex. Untreated it is universally fatal and treated people are absolutely dependent upon steroid medications lifelong, with a consequent excess in morbidity and mortality. A key feature of the adrenal cortex is that its cells are responsive to changes in circulating adrenocorticotrophic hormone (ACTH) concentration. This study aims to regenerate adrenocortical steroidogenic cell function in patients with established autoimmune Addison's disease (AAD) by stimulating proliferation and differentiation of their progenitor cells, the adrenocortical stem cells (ACSCs) (1,2). Using daily subcutaneous ACTH, administered according to two different regimens over 20 weeks, we will investigate whether regeneration of adrenal steroidogenic function through revival of ACSC activity is a realistic possibility.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 13
• Est. completion date: September 2012
• Est. primary completion date: September 2012
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 16 Years to 66 Years

Eligibility

Inclusion Criteria:

• Established autoimmune adrenal failure for >1yr age 16 to 65

Exclusion Criteria:

• Significant cardio-respiratory, chronic renal or non-autoimmune liver disease; malignancy

• Asthma, current infectious disease, recent live vaccination, acute psychosis, peptic ulcer disease

• 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

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Adrenal Failure
• Adrenal Insufficiency

Intervention

Drug:
• depot tetracosactide
1mg, 3x weekly by sc injection

Locations

Country	Name	City	State
United Kingdom	Newcastle University	Newcastle upon Tyne	Tyne and Wear

Sponsors (1)

Lead Sponsor	Collaborator
Newcastle University

Country where clinical trial is conducted

United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Peak serum Cortisol following ACTH stimulation		Tested at 20 weeks	No
Secondary	Change in QoL		20 weeks	No