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New treatment option for Bardet-Biedl Syndrome
Official title COhort for Bardet-Bield Syndrome and Alström Syndrome for Translational Research Monocentric Interventional Study
ClinicalTrials.gov ID: NCT04461444
What this study is testing
What is Skin biopsy?
Skin biopsy is an investigational medicine, being studied as a potential treatment for bardet-biedl syndrome.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- ALMS and BBS syndromes are rare diseases with overlapping features of multiple sensory and metabolic impairments, including diabetes mellitus. There are to date no specific treatments available and limited information on the natural history of the diseases.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 4 and older
You may be able to join if
- Patients of both sex
- Age minimum\
- patients with social protection
- Written informed consent form signed prior initiating any trial related procedure:
- by \> 18-year old patients
You likely can't join if
- Serious active intercurrent pathology that may impact the collected data
- Patient under judicial protection
- Participation in another treatment clinical trial which includes an exclusion period
- Non protected adult with difficulty of comprehension, or inability to understand the delivered information (emergency situation ...).
See the full eligibility criteria
- Patients of both sex
- Age minimum\
- patients with social protection
- Written informed consent form signed prior initiating any trial related procedure:
- by \> 18-year old patients
- by both parents for minor patients \> 4 months or legal representative for protected adults, and by minor and protected adults patients if able to understand and/or give their assent.
- For foreign patients, a third party will translate, if required, the information prior to the consent.
- a diagnosis of BBS or ALMS based on molecular assessment or clinical evaluation/or patient with mutation and none of the diagnosis criteria
- and/or an identified mutation in BBS genes or ALMS1 gene
- The inclusion of children is essential to a cohort study that is attempting an early identification of visual, metabolic and renal abnormalities. Many of the age-dependent manifestations of BBS develop during childhood...
- Serious active intercurrent pathology that may impact the collected data
- Patient under judicial protection
- Participation in another treatment clinical trial which includes an exclusion period
- Non protected adult with difficulty of comprehension, or inability to understand the delivered information (emergency situation ...).
The study team makes the final eligibility decision.
Where it's taking place
- Strasbourg, France
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 4 months and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Strasbourg, France. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.