Adrenal FailurePhase 4RoSANCT01371526
Revival of Stem Cells in Addison's Study
A Phase 4 study testing depot tetracosactide in people with Adrenal Failure, run by Newcastle University at 1 site in United Kingdom.
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. Read more about this study
- Who is it for?
- Ages 16 to 66 years
- Conditions the study is for
- Adrenal Failure
- What is being tested?
- depot tetracosactide
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 13 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- Sep 2010to Sep 2012
- Phase
- Phase 4
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 16 to 66 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Established autoimmune adrenal failure for >1yr age 16 to 65
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
United Kingdom1 site
Newcastle University
Newcastle upon Tyne, Tyne and Wear, NE1 3BZ
About this study
Official title: Revival of Autochthonous Adrenocortical Stem Cells in Autoimmune Addison's Disease
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.
- Conditions
- Adrenal Failure
- Being tested
- depot tetracosactide
- Medical subject headings
- Addison Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
depot tetracosactideDrug
1mg, 3x weekly by sc injection
| Study group | Role | What they receive |
|---|---|---|
| Synacthen | Experimental | active treatment Receives: Drug: depot tetracosactide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Peak serum Cortisol following ACTH stimulation
Measured: Tested at 20 weeks
Secondary outcomes (1)
Change in QoL
Measured: 20 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Jun 2011 (estimated)
- Study startSep 2010
- Primary completionSep 2012
- Study completionSep 2012
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Adrenal FailureStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 13 participants (actual)
Sponsor and investigators
- Lead sponsor
- Newcastle University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, SHS Pearce, Newcastle University
- Investigators
- Simon H Pearce, MD, Principal investigator, Newcastle University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01371526
- Sponsor's protocol ID
- RoSAv1.2:04_10: R&D#5252
- Other IDs
- 2009-018074-56, 2009
- Acronym
- RoSA
- Status last verified
- Feb 2013
- First submitted
- 9 Mar 2011
- Last updated
- 6 Feb 2013
- Results posted
- No
- Data monitoring committee
- Yes
Publications
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.
PubMed 24170102
Study at a glance
From the registry record, last updated by the sponsor on 6 Feb 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Revival of Autochthonous Adrenocortical Stem Cells in Autoimmune Addison's Disease
- ClinicalTrials.gov ID
- NCT01371526
- Acronym / sponsor ID
- RoSA / RoSAv1.2:04_10: R&D#5252
- Conditions
- Adrenal Failure
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 13 participants (actual)
- Lead sponsor
- Newcastle UniversityUniversity, hospital or other
- Study start
- Sep 2010
- Primary completion
- Sep 2012 (actual)
- Study completion
- Sep 2012 (actual)
- First posted
- 13 Jun 2011 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Synacthen | Experimental | Drug: depot tetracosactideactive treatment |
Who may qualify
- Ages
- 16 to 66 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (1 listed)
- Established autoimmune adrenal failure for >1yr age 16 to 65
Key exclusion criteria (5 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| depot tetracosactide1mg, 3x weekly by sc injection | Drug | Synacthen |
Primary outcome
Peak serum Cortisol following ACTH stimulation
Time frame: Tested at 20 weeks
1 secondary outcome
Change in QoL
Time frame: 20 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Feb 2013.
- Derived from this study: PubMed 24170102Gan 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Simon H Pearce, MD principal investigator
Newcastle University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing depot tetracosactide in people with Adrenal Failure, run by Newcastle University at 1 site in United Kingdom.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 6 Feb 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Peak serum Cortisol following ACTH stimulation (time frame: Tested at 20 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01371526, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Feb 2013.