Treatment of Bardet-Biedl-Syndrome With Metformin for Evaluation of a Possible Visual Improvement (BBS)

University Hospital Tuebingen

Status and phase: Withdrawn; Phase 2

Conditions: Bardet-Biedl Syndrome; Visual Impairment

Treatments: Drug: Metformin

Study type: Interventional

Funder types: Other

Identifiers

NCT03490019

BBS V1.0

Details and patient eligibility

About

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.

Full 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.

Sex: All

Ages: 10 to 25 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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0 participants in 1 patient group

Metformin Therapy

Experimental group

Description:

Metformin therapy once daily for 24 weeks with a dose of 500, 850 or 1000 mg depending on body weight

Treatment:

Drug: Metformin