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Revival of Stem Cells in Addison's Study (RoSA)

Primary Purpose

Adrenal Failure

Status

Completed

Phase

Phase 4

Locations

United Kingdom

Study Type

Interventional

Intervention

depot tetracosactide

About this trial

This is an interventional treatment trial for Adrenal Failure

Eligibility Criteria

16 Years - 66 Years (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Newcastle University

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Synacthen

Arm Description

active treatment

Outcomes

Primary Outcome Measures

Peak serum Cortisol following ACTH stimulation

Secondary Outcome Measures

Change in QoL

Full Information

NCT ID

NCT01371526

First Posted

March 9, 2011

Last Updated

February 5, 2013

Sponsor

Newcastle University

1. Study Identification

Unique Protocol Identification Number

NCT01371526

Brief Title

Revival of Stem Cells in Addison's Study

Acronym

RoSA

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

February 2013

Overall Recruitment Status

Completed

Study Start Date

September 2010 (undefined)

Primary Completion Date

September 2012 (Actual)

Study Completion Date

September 2012 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Newcastle University

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

Brief 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Adrenal Failure

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 4

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

13 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Synacthen

Arm Type

Experimental

Arm Description

active treatment

Intervention Type

Drug

Intervention Name(s)

depot tetracosactide

Intervention Description

1mg, 3x weekly by sc injection

Primary Outcome Measure Information:

Title

Peak serum Cortisol following ACTH stimulation

Time Frame

Tested at 20 weeks

Secondary Outcome Measure Information:

Title

Change in QoL

Time Frame

20 weeks

10. Eligibility

Sex

All

Minimum Age & Unit of Time

16 Years

Maximum Age & Unit of Time

66 Years

Accepts Healthy Volunteers

Eligibility Criteria

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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Simon H Pearce, MD

Organizational Affiliation

Newcastle University

Official's Role

Principal Investigator

Facility Information:

Facility Name

Newcastle University

City

Newcastle upon Tyne

State/Province

Tyne and Wear

ZIP/Postal Code

NE1 3BZ

Country

United Kingdom

12. IPD Sharing Statement

Citations:

PubMed Identifier

24170102

Citation

Gan EH, MacArthur K, Mitchell AL, Hughes BA, Perros P, Ball SG, James RA, Quinton R, Chen S, Furmaniak J, Arlt W, Pearce SH. Residual adrenal function in autoimmune Addison's disease: improvement after tetracosactide (ACTH1-24) treatment. J Clin Endocrinol Metab. 2014 Jan;99(1):111-8. doi: 10.1210/jc.2013-2449. Epub 2013 Dec 20.

Results Reference

derived

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Revival of Stem Cells in Addison's Study

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