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

NCT03490019 | Withdrawn Phase 2 DMC

• 1 other identifier: BBS V1.0
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Bardet-Biedl Syndrome; Visual Impairment

Outcome Measures

Primary Outcomes (1)

• Visual acuity

  Course of visual acuity according to appropriate table

  Baseline to day 210

Secondary Outcomes (11)

• Number of letters read

  Baseline to day 210

• Results of Static perimetry

  Baseline to day 210

• Results of Kinetic perimetry

  Baseline to day 210

• Electroretinogram (ERG) dim-flash

  Baseline to day 210

• Electroretinogram (ERG) standard flash

  Baseline to day 210

Study Arms (1)

Metformin Therapy

EXPERIMENTAL

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

Drug: Metformin

Interventions

Metformin DRUG

500, 850 or 1000 mg depending on body weight once daily for 24 weeks

Metformin Therapy

Eligibility Criteria

• Age: 10 Years - 25 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• University Hospital Tuebingen: lead

MeSH Terms

Conditions

Bardet-Biedl Syndrome; Vision Disorders

Interventions

Metformin

Condition Hierarchy (Ancestors)

Hypothalamic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Retinitis Pigmentosa; Eye Diseases, Hereditary; Eye Diseases; Ciliopathies; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn; Sensation Disorders; Neurologic Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Biguanides; Guanidines; Amidines; Organic Chemicals

Study Officials

• Heiko Billing, PD Dr. med.

  Universtitätsklinikum Tübingen

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: Prospective pilot study without control group

Study Record Dates

• First Submitted: March 19, 2018
• First Posted: April 6, 2018
• Study Start: April 1, 2018
• Primary Completion: October 1, 2019
• Study Completion: August 1, 2020
• Last Updated: April 18, 2019

Record last verified: 2018-02