Metformin in Tuberous Sclerosis Complex

Not Applicable

Completed

• Conditions: Tuberous sclerosis complex, renal angiomyolipoma; Genetic Diseases; Tuberous sclerosis

• Registration Number: ISRCTN92545532
• Lead Sponsor: niversity Hospitals Bristol NHS Foundation Trust (UK)

Brief Summary

2021 results in https://pubmed.ncbi.nlm.nih.gov/33681737/ (added 09/03/2021)

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2011-05-11
• Study Completion: 2014-08-31
• Last Update Posted: 22 March 2021

Recruitment & Eligibility

• Status: Completed
• Sex: All
• Target Recruitment: 55

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

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

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Mean renal angiomyolipoma volume 12 months after initiation of intervention

Secondary Outcome Measures

Name	Time	Method
<br> 1. Incidence of side effects<br> 2. Mean subependymal giant cell astrocytoma volume 12 months after initiation of intervention<br> 3. Health related quality of life 12 months after initiation of intervention<br> 4. Cognitive ability, development and adaptive behaviour 12 months after initiation of<br> intervention<br> 5. Epilepsy outcome<br> 6. Facial angiofibroma appearance 12 months after initiation of intervention<br>