search
Back to results

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

Primary Purpose

Bardet-Biedl Syndrome, Visual Impairment

Status
Withdrawn
Phase
Phase 2
Locations
Study Type
Interventional
Intervention
Metformin
Sponsored by
University Hospital Tuebingen
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Bardet-Biedl Syndrome

Eligibility Criteria

10 Years - 25 Years (Child, Adult)All SexesDoes not accept 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

Sites / Locations

    Arms of the Study

    Arm 1

    Arm Type

    Experimental

    Arm Label

    Metformin Therapy

    Arm Description

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

    Outcomes

    Primary Outcome Measures

    Visual acuity
    Course of visual acuity according to appropriate table

    Secondary Outcome Measures

    Number of letters read
    Course visual acuity defined as number of letters read
    Results of Static perimetry
    Course of results of static perimetry given by measurement of light contrast sensitivity within at least 5 central points.
    Results of Kinetic perimetry
    Course of results of kinetic perimetry given by mapping the visual field sensitivity boundaries using Goldmann perimetry
    Electroretinogram (ERG) dim-flash
    Course of values of ERG dim-flash
    Electroretinogram (ERG) standard flash
    Course of values of ERG standard flash at 3 ods/m2
    Electroretinogram (ERG) cone-single-flash
    Course of values of ERG cone-single-flash at 30cd/m2
    Electroretinogram (ERG) 30-Hz-Flicker
    Course of values of ERG measured by 30-Hz-Flicker in all patients
    Visual evoked potential (VEP) - amplitude
    Course of results of measurement of VEP amplitude
    Visual evoked potential (VEP) - latency time
    Course of results of measurement of VEP latency time
    FST
    Full-Field-Stimulus Threshold
    optical coherence tomography (OCT)
    Course of central thickness of retina measured by OCT

    Full Information

    First Posted
    March 19, 2018
    Last Updated
    April 16, 2019
    Sponsor
    University Hospital Tuebingen
    search

    1. Study Identification

    Unique Protocol Identification Number
    NCT03490019
    Brief Title
    Treatment of Bardet-Biedl-Syndrome With Metformin for Evaluation of a Possible Visual Improvement
    Acronym
    BBS
    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

    2. Study Status

    Record Verification Date
    February 2018
    Overall Recruitment Status
    Withdrawn
    Why Stopped
    unrealizable wishes of national authorities
    Study Start Date
    April 1, 2018 (Anticipated)
    Primary Completion Date
    October 2019 (Anticipated)
    Study Completion Date
    August 2020 (Anticipated)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    University Hospital Tuebingen

    4. Oversight

    Studies a U.S. FDA-regulated Drug Product
    No
    Studies a U.S. FDA-regulated Device Product
    No
    Data Monitoring Committee
    Yes

    5. Study Description

    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.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Bardet-Biedl Syndrome, Visual Impairment

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 2
    Interventional Study Model
    Single Group Assignment
    Model Description
    Prospective pilot study without control group
    Masking
    None (Open Label)
    Allocation
    N/A
    Enrollment
    0 (Actual)

    8. Arms, Groups, and Interventions

    Arm Title
    Metformin Therapy
    Arm Type
    Experimental
    Arm Description
    Metformin therapy once daily for 24 weeks with a dose of 500, 850 or 1000 mg depending on body weight
    Intervention Type
    Drug
    Intervention Name(s)
    Metformin
    Intervention Description
    500, 850 or 1000 mg depending on body weight once daily for 24 weeks
    Primary Outcome Measure Information:
    Title
    Visual acuity
    Description
    Course of visual acuity according to appropriate table
    Time Frame
    Baseline to day 210
    Secondary Outcome Measure Information:
    Title
    Number of letters read
    Description
    Course visual acuity defined as number of letters read
    Time Frame
    Baseline to day 210
    Title
    Results of Static perimetry
    Description
    Course of results of static perimetry given by measurement of light contrast sensitivity within at least 5 central points.
    Time Frame
    Baseline to day 210
    Title
    Results of Kinetic perimetry
    Description
    Course of results of kinetic perimetry given by mapping the visual field sensitivity boundaries using Goldmann perimetry
    Time Frame
    Baseline to day 210
    Title
    Electroretinogram (ERG) dim-flash
    Description
    Course of values of ERG dim-flash
    Time Frame
    Baseline to day 210
    Title
    Electroretinogram (ERG) standard flash
    Description
    Course of values of ERG standard flash at 3 ods/m2
    Time Frame
    Baseline to day 210
    Title
    Electroretinogram (ERG) cone-single-flash
    Description
    Course of values of ERG cone-single-flash at 30cd/m2
    Time Frame
    Baseline to day 210
    Title
    Electroretinogram (ERG) 30-Hz-Flicker
    Description
    Course of values of ERG measured by 30-Hz-Flicker in all patients
    Time Frame
    Baseline to day 210
    Title
    Visual evoked potential (VEP) - amplitude
    Description
    Course of results of measurement of VEP amplitude
    Time Frame
    Baseline to day 210
    Title
    Visual evoked potential (VEP) - latency time
    Description
    Course of results of measurement of VEP latency time
    Time Frame
    Baseline to day 210
    Title
    FST
    Description
    Full-Field-Stimulus Threshold
    Time Frame
    Baseline to day 210
    Title
    optical coherence tomography (OCT)
    Description
    Course of central thickness of retina measured by OCT
    Time Frame
    Baseline to day 210

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    10 Years
    Maximum Age & Unit of Time
    25 Years
    Accepts Healthy Volunteers
    No
    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
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    Heiko Billing, PD Dr. med.
    Organizational Affiliation
    Universtitätsklinikum Tübingen
    Official's Role
    Principal Investigator

    12. IPD Sharing Statement

    Plan to Share IPD
    Undecided

    Learn more about this trial

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

    We'll reach out to this number within 24 hrs