Metformin in Tuberous Sclerosis Complex

ISRCTN	ISRCTN92545532
DOI	https://doi.org/10.1186/ISRCTN92545532
Clinical Trials Information System (CTIS)	2011-001319-30
Protocol serial number	Version 1
Sponsor	University Hospitals Bristol NHS Foundation Trust (UK)
Funder	National Institute for Health Research (NIHR) (UK) - Research for Patient Benefit (RfPB) programme (ref: PB-PG-0909-20131)

Submission date: 09/02/2011
Registration date: 11/05/2011
Last edited: 09/03/2021

Recruitment status: No longer recruiting
Overall study status: Completed
Condition category: Genetic Diseases

Prospectively registered

Protocol

Statistical analysis plan

Results

Individual participant data

Plain English summary of protocol

Not provided at time of registration

Contact information

Dr Finbar O'Callaghan
Scientific

Department of Paediatric Neurology
Level 6 UHB Education Centre
Upper Maudlin Street
Bristol
BS2 8AE
United Kingdom

Phone	+44 11 7342 0202
Email	finbar.ocallaghan@bristol.ac.uk

Study information

Primary study design	Interventional
Study design	Prospective double-blind randomised parallel group placebo-controlled trial
Secondary study design	Randomised controlled trial
Study type	Participant information sheet
Scientific title	A prospective, double-blind, randomised, parallel group, placebo-controlled trial of metformin treatment for renal angiomyolipomas in tuberous sclerosis complex
Study acronym	MiTS
Study objectives	Metformin reduces the size of renal angiomyolipomas in people with tuberous sclerosis complex (TSC).
Ethics approval(s)	Not provided at time of registration
Health condition(s) or problem(s) studied	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 / device / biological / vaccine name(s)	Metformin
Primary outcome measure(s)	Mean renal angiomyolipoma volume 12 months after initiation of intervention
Key secondary outcome measure(s)	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
Completion date	31/08/2014

Eligibility

Participant type(s)	Patient
Age group	Adult
Sex	All
Target sample size at registration	100
Total final enrolment	55
Key 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
Key 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
Date of first enrolment	01/09/2011
Date of final enrolment	31/08/2014

Locations

Countries of recruitment

United Kingdom
England

Study participating centre

Department of Paediatric Neurology

Bristol
BS2 8AE
United Kingdom

Results and Publications

Individual participant data (IPD) Intention to share	No
IPD sharing plan summary	Not provided at time of registration
IPD sharing plan

Study outputs

Output type	Details	Date created	Date added	Peer reviewed?	Patient-facing?
Results article	results	14/01/2021	09/03/2021	Yes	No
Participant information sheet	Participant information sheet	11/11/2025	11/11/2025	No	Yes

Editorial Notes

09/03/2021: Publication reference, total final enrolment and EudraCT number added.
18/11/2016: No publications found in PubMed, verifying study status with principal investigator.