Clinical trial NCT03490019: Treatment of Bardet-Biedl-Syndrome With Metformin for Evaluation of a Possible Visual Improvement
Official title: 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
Study type: INTERVENTIONAL
Overall status: WITHDRAWN
Phase: PHASE2
Conditions: Bardet-Biedl Syndrome
Visual Impairment
Lead sponsor: University Hospital Tuebingen
Sponsor class: OTHER
First posted: 2018-04-06
Enrollment: 0
Sex: ALL
Minimum age (years): 10
Maximum age (years): 25
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Prospective pilot study without control group
Primary purpose: TREATMENT

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.

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

Primary outcomes:
measure: Visual acuity
timeFrame: Baseline to day 210
Secondary outcomes:
measure: Number of letters read
timeFrame: Baseline to day 210
measure: Results of Static perimetry
timeFrame: Baseline to day 210
measure: Results of Kinetic perimetry
timeFrame: Baseline to day 210
measure: Electroretinogram (ERG) dim-flash
timeFrame: Baseline to day 210
measure: Electroretinogram (ERG) standard flash
timeFrame: Baseline to day 210
measure: Electroretinogram (ERG) cone-single-flash
timeFrame: Baseline to day 210
measure: Electroretinogram (ERG) 30-Hz-Flicker
timeFrame: Baseline to day 210
measure: Visual evoked potential (VEP) - amplitude
timeFrame: Baseline to day 210
measure: Visual evoked potential (VEP) - latency time
timeFrame: Baseline to day 210
measure: FST
timeFrame: Baseline to day 210
measure: optical coherence tomography (OCT)
timeFrame: Baseline to day 210
Other outcomes:
Locations:
Study officials:
affiliation: Universtitätsklinikum Tübingen
name: Heiko Billing, PD Dr. med.
role: PRINCIPAL_INVESTIGATOR
References: