Metformin in Tuberous Sclerosis Complex
ISRCTN | ISRCTN92545532 |
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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
Scientific
Department of Paediatric Neurology
Level 6 UHB Education Centre
Upper Maudlin Street
Bristol
BS2 8AE
United Kingdom
Phone | +44 11 7342 0202 |
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finbar.ocallaghan@bristol.ac.uk |
Study information
Study design | Prospective double-blind randomised parallel group placebo-controlled trial |
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Primary study design | Interventional |
Secondary study design | Randomised controlled trial |
Study setting(s) | Hospital |
Study type | Treatment |
Participant information sheet | Not available in web format, please use the contact details below to request a patient 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 |
Pharmaceutical study type(s) | |
Phase | Not Applicable |
Drug / device / biological / vaccine name(s) | Metformin |
Primary outcome measure | 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 study start date | 01/09/2011 |
Completion date | 31/08/2014 |
Eligibility
Participant type(s) | Patient |
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Age group | Adult |
Sex | Both |
Target number of participants | 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
- England
- United Kingdom
Study participating centre
Department of Paediatric Neurology
Bristol
BS2 8AE
United Kingdom
BS2 8AE
United Kingdom
Sponsor information
University Hospitals Bristol NHS Foundation Trust (UK)
Hospital/treatment centre
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 |
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mary.perkins@uhbristol.nhs.uk | |
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 | |
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Individual participant data (IPD) Intention to share | No |
IPD sharing plan summary | Not provided at time of registration |
Publication and dissemination plan | Not provided at time of registration |
IPD sharing plan |
Study outputs
Output type | Details | Date created | Date added | Peer reviewed? | Patient-facing? |
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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.