Bardet-Biedl SyndromePhase 2BBSNCT03490019
Treatment of Bardet-Biedl-Syndrome With Metformin for Evaluation of a Possible Visual Improvement
A Phase 2 study testing Metformin in people with Bardet-Biedl Syndrome and Visual Impairment, run by University Hospital Tuebingen.
Trial 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. Read more about this study
- Who is it for?
- Ages 10 to 25 years
- Conditions the study is for
- Bardet-Biedl Syndrome, Visual Impairment
- What is being tested?
- Metformin
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- 1 Apr 2018to Aug 2020
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 10 to 25 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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
Who cannot join
- 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)
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- 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
About this study
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
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.
Read the 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.
- Conditions
- Bardet-Biedl Syndrome, Visual Impairment
- Being tested
- Metformin
- Medical subject headings
- Bardet-Biedl Syndrome, Vision Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MetforminDrug
500, 850 or 1000 mg depending on body weight once daily for 24 weeks
| Study group | Role | What they receive |
|---|---|---|
| Metformin Therapy | Experimental | Metformin therapy once daily for 24 weeks with a dose of 500, 850 or 1000 mg depending on body weight Receives: Drug: Metformin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Visual acuity
Course of visual acuity according to appropriate table
Measured: Baseline to day 210
Secondary outcomes (11)
Number of letters read
Course visual acuity defined as number of letters read
Measured: Baseline to day 210
Results of Static perimetry
Course of results of static perimetry given by measurement of light contrast sensitivity within at least 5 central points.
Measured: Baseline to day 210
Results of Kinetic perimetry
Course of results of kinetic perimetry given by mapping the visual field sensitivity boundaries using Goldmann perimetry
Measured: Baseline to day 210
Electroretinogram (ERG) dim-flash
Course of values of ERG dim-flash
Measured: Baseline to day 210
Electroretinogram (ERG) standard flash
Course of values of ERG standard flash at 3 ods/m2
Measured: Baseline to day 210
Electroretinogram (ERG) cone-single-flash
Course of values of ERG cone-single-flash at 30cd/m2
Measured: Baseline to day 210
Electroretinogram (ERG) 30-Hz-Flicker
Course of values of ERG measured by 30-Hz-Flicker in all patients
Measured: Baseline to day 210
Visual evoked potential (VEP) - amplitude
Course of results of measurement of VEP amplitude
Measured: Baseline to day 210
Visual evoked potential (VEP) - latency time
Course of results of measurement of VEP latency time
Measured: Baseline to day 210
FST
Full-Field-Stimulus Threshold
Measured: Baseline to day 210
optical coherence tomography (OCT)
Course of central thickness of retina measured by OCT
Measured: Baseline to day 210
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Apr 2018
- Study start1 Apr 2018 (estimated)
- Primary completionOct 2019 (estimated)
- Study completionAug 2020 (estimated)
Why it stopped: unrealizable wishes of national authorities
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Bardet-Biedl SyndromeStudies of the same condition, open and under way.
- More trials run by University Hospital TuebingenThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Prospective pilot study without control group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Hospital Tuebingen
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Heiko Billing, PD Dr. med., Principal investigator, Universtitätsklinikum Tübingen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03490019
- Sponsor's protocol ID
- BBS V1.0
- Acronym
- BBS
- Status last verified
- Feb 2018
- First submitted
- 19 Mar 2018
- Last updated
- 18 Apr 2019
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 18 Apr 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03490019
- Acronym / sponsor ID
- BBS / BBS V1.0
- Conditions
- Bardet-Biedl Syndrome, Visual Impairment
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: unrealizable wishes of national authorities
- Enrolment
- 0 participants (actual)
- Lead sponsor
- University Hospital TuebingenUniversity, hospital or other
- Study start
- 1 Apr 2018 (estimated)
- Primary completion
- Oct 2019 (estimated)
- Study completion
- Aug 2020 (estimated)
- First posted
- 6 Apr 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Prospective pilot study without control group
| Arm | Type | Receives |
|---|---|---|
| Metformin Therapy | Experimental | Drug: MetforminMetformin therapy once daily for 24 weeks with a dose of 500, 850 or 1000 mg depending on body weight |
Who may qualify
- Ages
- 10 to 25 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (10 listed)
- Hypoglycaemia (<50mg/dl)
- Therapy with Metformin within the last three months
- Participation in another clinical trial
- pregnancy, lactation
- any contra indication concerning Metformin therapy
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Metformin500, 850 or 1000 mg depending on body weight once daily for 24 weeks | Drug | Metformin Therapy |
Primary outcome
Visual acuity
Time frame: Baseline to day 210
Course of visual acuity according to appropriate table
11 secondary outcomes
Number of letters read
Time frame: Baseline to day 210
Results of Static perimetry
Time frame: Baseline to day 210
Results of Kinetic perimetry
Time frame: Baseline to day 210
Electroretinogram (ERG) dim-flash
Time frame: Baseline to day 210
Electroretinogram (ERG) standard flash
Time frame: Baseline to day 210
Electroretinogram (ERG) cone-single-flash
Time frame: Baseline to day 210
Electroretinogram (ERG) 30-Hz-Flicker
Time frame: Baseline to day 210
Visual evoked potential (VEP) - amplitude
Time frame: Baseline to day 210
Visual evoked potential (VEP) - latency time
Time frame: Baseline to day 210
FST
Time frame: Baseline to day 210
optical coherence tomography (OCT)
Time frame: Baseline to day 210
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Apr 2019.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Heiko Billing, PD Dr. med. principal investigator
Universtitätsklinikum Tübingen
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Metformin in people with Bardet-Biedl Syndrome and Visual Impairment, run by University Hospital Tuebingen.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 18 Apr 2019. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Visual acuity (time frame: Baseline to day 210). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Apr 2019.