ISRCTN ISRCTN92545532
DOI https://doi.org/10.1186/ISRCTN92545532
EudraCT/CTIS number 2011-001319-30
Secondary identifying numbers Version 1
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

Study designProspective double-blind randomised parallel group placebo-controlled trial
Primary study designInterventional
Secondary study designRandomised controlled trial
Study setting(s)Hospital
Study typeTreatment
Participant information sheet Not available in web format, please use the contact details below to request a patient information sheet
Scientific titleA prospective, double-blind, randomised, parallel group, placebo-controlled trial of metformin treatment for renal angiomyolipomas in tuberous sclerosis complex
Study acronymMiTS
Study objectivesMetformin 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) studiedTuberous sclerosis complex, renal angiomyolipoma
InterventionParticipants 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 typeDrug
Pharmaceutical study type(s)
PhaseNot Applicable
Drug / device / biological / vaccine name(s)Metformin
Primary outcome measureMean renal angiomyolipoma volume 12 months after initiation of intervention
Secondary outcome measures1. 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 study start date01/09/2011
Completion date31/08/2014

Eligibility

Participant type(s)Patient
Age groupAdult
SexBoth
Target number of participants100
Total final enrolment55
Key inclusion criteria1. 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 criteria1. 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 enrolment01/09/2011
Date of final enrolment31/08/2014

Locations

Countries of recruitment

  • England
  • United Kingdom

Study participating centre

Department of Paediatric Neurology
Bristol
BS2 8AE
United Kingdom

Sponsor information

University Hospitals Bristol NHS Foundation Trust (UK)
Hospital/treatment centre

University Hospitals Bristol NHS Foundation Trust
c/o Mrs Mary Perkins
Level 3 Education Centre
Upper Maudlin Street
Bristol
BS2 8AE
England
United Kingdom

Phone +44 (0)11 7342 0233
Email mary.perkins@uhbristol.nhs.uk
ROR logo "ROR" https://ror.org/04nm1cv11

Funders

Funder type

Government

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

No information available

Results and Publications

Intention to publish date
Individual participant data (IPD) Intention to shareNo
IPD sharing plan summaryNot provided at time of registration
Publication and dissemination planNot 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

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.