Natural History Study of Children with Achondroplasia
Research type
Research Study
Full title
An International, Prospective Registry Investigating the Natural History of Children with Achondroplasia
IRAS ID
244565
Contact name
Christine/P Burren
Contact email
Sponsor organisation
Therachon SAS
Clinicaltrials.gov Identifier
NA, NA
Duration of Study in the UK
5 years, 0 months, 1 days
Research summary
The proposed study involves no drug and is called “natural history study”. Such natural history studies are necessary to gain information about rare diseases like achondroplasia and to track how they progress over time. This understanding will help in the clinical development of drugs and therapies to treat achondroplasia. \n\nIts purpose is to study the characteristics of achondroplasia, its symptoms, and markers of bone growth. The bone growth is detected by the length of the bones; it may also be detected by some special substances in children’s blood called biomarkers that may be related to how a child will grow.\n\nThe study consists of a Screening visit, a Baseline visit, 3-month interval visits, a yearly visit (±3 weeks) and a Final Visit. \n\nChildren’s information (about their diseases, symptoms, complaints, the way they grow), will be collected in a registry at baseline and at every 3-month interval visits, for a maximum of 5 years. \n\nIn addition to the collection of information the children will have a blood test for biomarkers assessments at the Baseline visit and then at each annual visit and measurements of their bodies (height, waist circumference, length of their arms, legs, etc.)\n
REC name
South West - Cornwall & Plymouth Research Ethics Committee
REC reference
18/SW/0159
Date of REC Opinion
23 Jul 2018
REC opinion
Further Information Favourable Opinion