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

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ClinicalTrials.gov Identifier: NCT03490019
Recruitment Status : Not yet recruiting
First Posted : April 6, 2018
Last Update Posted : May 8, 2018
Sponsor:
Information provided by (Responsible Party):
University Hospital Tuebingen

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.

Condition or disease Intervention/treatment Phase
Bardet-Biedl Syndrome Visual Impairment Drug: Metformin Phase 2

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.


Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 16 participants
Intervention Model: Single Group Assignment
Intervention Model Description: Prospective pilot study without control group
Masking: None (Open Label)
Primary Purpose: Treatment
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
Estimated Study Start Date : July 2018
Estimated Primary Completion Date : October 2019
Estimated Study Completion Date : August 2020


Arm Intervention/treatment
Experimental: Metformin Therapy
Metformin therapy once daily for 24 weeks with a dose of 500, 850 or 1000 mg depending on body weight
Drug: Metformin
500, 850 or 1000 mg depending on body weight once daily for 24 weeks




Primary Outcome Measures :
  1. Visual acuity [ Time Frame: Baseline to day 210 ]
    Course of visual acuity according to appropriate table


Secondary Outcome Measures :
  1. Number of letters read [ Time Frame: Baseline to day 210 ]
    Course visual acuity defined as number of letters read

  2. Results of Static perimetry [ Time Frame: Baseline to day 210 ]
    Course of results of static perimetry given by measurement of light contrast sensitivity within at least 5 central points.

  3. Results of Kinetic perimetry [ Time Frame: Baseline to day 210 ]
    Course of results of kinetic perimetry given by mapping the visual field sensitivity boundaries using Goldmann perimetry

  4. Electroretinogram (ERG) dim-flash [ Time Frame: Baseline to day 210 ]
    Course of values of ERG dim-flash

  5. Electroretinogram (ERG) standard flash [ Time Frame: Baseline to day 210 ]
    Course of values of ERG standard flash at 3 ods/m2

  6. Electroretinogram (ERG) cone-single-flash [ Time Frame: Baseline to day 210 ]
    Course of values of ERG cone-single-flash at 30cd/m2

  7. Electroretinogram (ERG) 30-Hz-Flicker [ Time Frame: Baseline to day 210 ]
    Course of values of ERG measured by 30-Hz-Flicker in all patients

  8. Visual evoked potential (VEP) - amplitude [ Time Frame: Baseline to day 210 ]
    Course of results of measurement of VEP amplitude

  9. Visual evoked potential (VEP) - latency time [ Time Frame: Baseline to day 210 ]
    Course of results of measurement of VEP latency time

  10. FST [ Time Frame: Baseline to day 210 ]
    Full-Field-Stimulus Threshold

  11. optical coherence tomography (OCT) [ Time Frame: Baseline to day 210 ]
    Course of central thickness of retina measured by OCT



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Ages Eligible for Study:   10 Years to 25 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03490019


Contacts
Contact: Heiko Billing, PD Dr. med. 07071-2985480 heiko.billing@med.uni-tuebingen.de
Contact: Eberhart Zrenner, Prof. 07071-2984786 ez@uni-tuebingen.de

Sponsors and Collaborators
University Hospital Tuebingen
Investigators
Principal Investigator: Heiko Billing, PD Dr. med. Universtitätsklinikum Tübingen

Responsible Party: University Hospital Tuebingen
ClinicalTrials.gov Identifier: NCT03490019     History of Changes
Other Study ID Numbers: BBS V1.0
First Posted: April 6, 2018    Key Record Dates
Last Update Posted: May 8, 2018
Last Verified: February 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Vision, Low
Syndrome
Vision Disorders
Bardet-Biedl Syndrome
Laurence-Moon Syndrome
Disease
Pathologic Processes
Sensation Disorders
Neurologic Manifestations
Nervous System Diseases
Eye Diseases
Signs and Symptoms
Hypothalamic Diseases
Brain Diseases
Central Nervous System Diseases
Ciliopathies
Abnormalities, Multiple
Congenital Abnormalities
Genetic Diseases, Inborn
Metformin
Hypoglycemic Agents
Physiological Effects of Drugs