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