A multi-center, observational study of children with achondroplasia.
Research type
Research Study
Full title
ACHieve: A multi-center, longitudinal, observational study of children with achondroplasia.
IRAS ID
262598
Contact name
Melita Irving
Contact email
Sponsor organisation
Ascendis Pharma Growth Disorders A/S
Clinicaltrials.gov Identifier
Duration of Study in the UK
4 years, 10 months, 20 days
Research summary
Achondroplasia (ACH) is the most frequent form of short-limb (or disproportionately short stature). It occurs with a frequency of 1 in 20,000 to 30,000 live births, affecting 250,000 men and women equally worldwide. Eighty percent of cases are sporadic, occurring in children of parents with normal stature.
Individuals with ACH have a distinct appearance, including short stature (but an average size trunk), large head with a prominent forehead, and shortened proximal limbs. Associated complications occur at various developmental stages.
No effective medicinal product is available for the indication of ACH. Most available treatments are surgical interventions intended to alleviate specific complications associated with ACH . As none of the available therapies address the underlying pathology of ACH, individuals with ACH undergo multiple surgeries and other interventions throughout the lives. Furthermore, individuals with ACH suffer numerous clinically significant comorbidities, some of which have no effective therapeutic option.
Ascendis Pharma Growth Disorders A/S is assessing the changes in growth velocity, body proportionality, and comorbidities over time in children with achondroplasia.
This study will enroll worldwide approximately 200 children with achondroplasia from birth to 8 years of age. After enrollment, the child will be evaluated every six months for body measurement parameters and complications of achondroplasia. Children will be followed for up to 60 months.
REC name
South Central - Berkshire Research Ethics Committee
REC reference
19/SC/0300
Date of REC Opinion
30 May 2019
REC opinion
Favourable Opinion