Metformin in Tuberous Sclerosis Complex

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

Status

Active, not recruiting

Phases

Unknown

Study type

Interventional

Source

ISRCTN

Registry ID

ISRCTN92545532

Enrollment

100

Registered

2011-05-11

Start date

2011-09-01

Completion date

Unknown

Last updated

2021-03-22

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Tuberous sclerosis complex, renal angiomyolipoma Genetic Diseases Tuberous sclerosis

Interventions

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

Eligibility

Sex/Gender

All

Inclusion criteria

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

Exclusion criteria

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

Design outcomes

Primary

Measure	Time frame
Mean renal angiomyolipoma volume 12 months after initiation of intervention	—

Secondary

Measure	Time frame
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	—

Countries

United Kingdom

Outcome results

None listed

Source: ISRCTN (via WHO ICTRP) · Data processed: Feb 4, 2026