Treatment of Infantile and Juvenile Patients With Bardet-Biedl-Syndrome With Metformin. Evaluation of a Visual Improvement as a Side Effect of the Pediatric Treatment of Adipositas - a Prospective Pilot Study Without Control
Overview
- Phase
- Phase 2
- Intervention
- Metformin
- Conditions
- Bardet-Biedl Syndrome
- Sponsor
- University Hospital Tuebingen
- Primary Endpoint
- Visual acuity
- Status
- Withdrawn
- Last Updated
- 7 years ago
Overview
Brief Summary
In this prospective pilot study without control group children and young adults (10-25 years old) diagnosed with Bardet-Biedl syndrome and treated with Metformin for their adipositas will be evaluated for a possible additional effect of Metformin on visual acuity.
Detailed Description
Within experiments with animals a positive effect of Metformin concerning the photo receptors was shown. Several patients of the university hospital Tübingen with Bardet-Biedl-Syndrome and Metformin therapy due to their adipositas reported a subjective improvement of the visual acuity which was confirmed by opthalmologic tests. This will be checked in a prospective pilot study without control group including children and young adults (10-25 years old) diagnosed with Bardet-Biedl syndrome and treated with Metformin for their adipositas. Therefore a possible additional effect of Metformin on visual acuity will be evaluated under controlled conditions.
Investigators
Eligibility Criteria
Inclusion Criteria
- •clinically confirmed Bardet-Biedl-syndrome
- •Visual acuity between 0.05 and 0.8
- •Age \>=10 year to \<25 years
- •Visual field III4e or V4e with diameter \>=5°, if II4e not seen
- •informed consent of patient and/or legal representative
Exclusion Criteria
- •Hypoglycaemia (\<50mg/dl)
- •Therapy with Metformin within the last three months
- •Participation in another clinical trial
- •pregnancy, lactation
- •any contra indication concerning Metformin therapy
- •Renal failure (creatinine clearance \< 60ml/min)
- •any acute disorder accompanied by clouding of consciousness
- •acute or chronic disorders possible accompanied by tissue hypoxia
- •Liver insufficiency, alcohol abuse
- •not fluent in German language
Arms & Interventions
Metformin Therapy
Metformin therapy once daily for 24 weeks with a dose of 500, 850 or 1000 mg depending on body weight
Intervention: Metformin
Outcomes
Primary Outcomes
Visual acuity
Time Frame: Baseline to day 210
Course of visual acuity according to appropriate table
Secondary Outcomes
- Visual evoked potential (VEP) - latency time(Baseline to day 210)
- Electroretinogram (ERG) 30-Hz-Flicker(Baseline to day 210)
- FST(Baseline to day 210)
- Number of letters read(Baseline to day 210)
- Results of Kinetic perimetry(Baseline to day 210)
- Electroretinogram (ERG) standard flash(Baseline to day 210)
- Results of Static perimetry(Baseline to day 210)
- Electroretinogram (ERG) dim-flash(Baseline to day 210)
- Electroretinogram (ERG) cone-single-flash(Baseline to day 210)
- Visual evoked potential (VEP) - amplitude(Baseline to day 210)
- optical coherence tomography (OCT)(Baseline to day 210)