COhort for Bardet-Bield Syndrome and Alström Syndrome for Translational Research Monocentric Interventional Study

Recruiting N/A Interventional Study
Bardet-Biedl Syndrome Alström Syndrome
No Placebo Group Every participant receives an active treatment — no one gets a placebo.
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At a Glance
Age
4 Months and older
Sex
Any
Study type
Interventional
Purpose
Supportive Care
Participants needed
350 (estimated)
Sponsor
University Hospital, Strasbourg, France · Other
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About This Trial
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. the investigators aim to establish a French cohort for these diseases to improve patient care and assess the effect of actual therapies on qua…
Trial Locations
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Eligibility Criteria
Inclusion 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 …
Contacts

Hélène DOLFFUS

+33.3.69.55.19.55

helene.dollfus@chru-strasbourg.fr

CONTACT