Condition category
Genetic Diseases
Date applied
09/02/2011
Date assigned
11/05/2011
Last edited
18/11/2016
Prospective/Retrospective
Prospectively registered
Overall trial status
Completed
Recruitment status
No longer recruiting

Plain English Summary

Not provided at time of registration

Trial website

Contact information

Type

Scientific

Primary contact

Dr Finbar O'Callaghan

ORCID ID

Contact details

Department of Paediatric Neurology
Level 6 UHB Education Centre
Upper Maudlin Street
Bristol
BS2 8AE
United Kingdom
+44 11 7342 0202
finbar.ocallaghan@bristol.ac.uk

Additional identifiers

EudraCT number

ClinicalTrials.gov number

Protocol/serial number

Version 1

Study information

Scientific title

A prospective, double-blind, randomised, parallel group, placebo-controlled trial of metformin treatment for renal angiomyolipomas in tuberous sclerosis complex

Acronym

MiTS

Study hypothesis

Metformin reduces the size of renal angiomyolipomas in people with tuberous sclerosis complex (TSC).

Ethics approval

Not provided at time of registration

Study design

Prospective double-blind randomised parallel group placebo-controlled trial

Primary study design

Interventional

Secondary study design

Randomised controlled trial

Trial setting

Hospitals

Trial type

Treatment

Patient information sheet

Not available in web format, please use the contact details below to request a patient information sheet

Condition

Tuberous sclerosis complex, renal angiomyolipoma

Intervention

Participants will be randomised to two groups: Placebo or Metformin

Metformin 500mg twice daily for 6 months. If this dose is tolerated then after 6 months the dose will be increased to metformin 500mg three times per day for a further 6 months. Renal angiomyolipoma volume will be measured by magnetic resonance imaging at day 0 (baseline) and 12 months.

Intervention type

Drug

Phase

Not Applicable

Drug names

Metformin

Primary outcome measures

Mean renal angiomyolipoma volume 12 months after initiation of intervention

Secondary outcome measures

1. Incidence of side effects
2. Mean subependymal giant cell astrocytoma volume 12 months after initiation of intervention
3. Health related quality of life 12 months after initiation of intervention
4. Cognitive ability, development and adaptive behaviour 12 months after initiation of
intervention
5. Epilepsy outcome
6. Facial angiofibroma appearance 12 months after initiation of intervention

Overall trial start date

01/09/2011

Overall trial end date

31/08/2014

Reason abandoned

Eligibility

Participant inclusion criteria

1. Clinically definite diagnosis of TSC (modified Gomez criteria)
2. Age between 10 to 65 years
3. One or more renal angiomyolipoma of at least one centimetre in largest diameter
4. Signed informed consent

Participant type

Patient

Age group

Adult

Gender

Both

Target number of participants

100

Participant exclusion criteria

1. Serious intercurrent illness or uncontrolled disease which could compromise
participation in the study
2. Impairment of renal function
3. Use of x-ray contrast medium containing iodine within the last 30 days
4. Multiple renal angiomyolipomas where individual lesions cannot be distinguished (and
as such cannot be accurately measured)
5. Renal haemorrhage within the preceding year
6. Known conservatively managed renal aneurysm(s) greater than 10mm
7. Liver insufficiency
8. Acute or chronic disease which may cause tissue hypoxia e.g.cardiac/respiratory failure,
recent myocardial infarction, shock
9. Diabetes
10. Treatment with any injected or oral hypoglycaemic drug
11. Use of an investigational drug within the last 30 days
12. Pregnant or intending to become pregnant during the study period
13. Breastfeeding

Recruitment start date

01/09/2011

Recruitment end date

31/08/2014

Locations

Countries of recruitment

United Kingdom

Trial participating centre

Department of Paediatric Neurology
Bristol
BS2 8AE
United Kingdom

Sponsor information

Organisation

University Hospitals Bristol NHS Foundation Trust (UK)

Sponsor details

University Hospitals Bristol NHS Foundation Trust
c/o Mrs Mary Perkins
Level 3 Education Centre
Upper Maudlin Street
Bristol
BS2 8AE
United Kingdom
+44 (0)11 7342 0233
mary.perkins@uhbristol.nhs.uk

Sponsor type

Government

Website

Funders

Funder type

Government

Funder name

National Institute for Health Research (NIHR) (UK) - Research for Patient Benefit (RfPB) programme (ref: PB-PG-0909-20131)

Alternative name(s)

Funding Body Type

Funding Body Subtype

Location

Results and Publications

Publication and dissemination plan

Not provided at time of registration

Intention to publish date

Participant level data

Not provided at time of registration

Results - basic reporting

Publication summary

Publication citations

Additional files

Editorial Notes

18/11/2016: No publications found in PubMed, verifying study status with principal investigator.