ACcomplisH: Phase 2 TransCon CNP trial in children with achondroplasia
Research type
Research Study
Full title
ACcomplisH: A Phase 2, multicenter, double-blind, randomized, placebo-controlled, dose escalation trial evaluating safety, efficacy, and pharmacokinetics of subcutaneous doses of TransCon CNP administered once weekly for 52 weeks in prepubertal children with achondroplasia followed by an Open-Label Extension Period
IRAS ID
270767
Contact name
Melita Irving
Contact email
Sponsor organisation
Ascendis Pharma Growth Disorders A/S
Eudract number
2019-002754-22
Duration of Study in the UK
3 years, 2 months, 31 days
Research summary
Achondroplasia (ACH) is the most common form of abnormal skeletal growth and the most frequent form of short limb (or disproportionately short stature). It occurs with a frequency of 1 in 10,000 to 30,000 live births, affecting 250,000 men and women equally worldwide. ACH is caused by a gene mutation in a gene. Individuals with ACH have a distinct appearance, including short stature (but an average size trunk), large head with a prominent forehead, and shortened limbs. Associated complications occur at various developmental stages. No effective medicinal product is available for the treatment of ACH. Existing interventions do not address the underlying cause of ACH and individuals with the condition undergo multiple surgeries and interventions throughout their lives. TransCon CNP is injected subcutaneously (under the skin) once weekly. This is a dose escalation study to evaluate the safety, efficacy and pharmacokinetics of once weekly TransCon CNP administration in children aged 2-10 years old with ACH.The study will enrol approx. 60 male and female prepubertal children with ACH worldwide. After enrolment, the patients will be assigned to either TransCon CNP or placebo in a 3:1 ratio, at a dose of 6, 20, 50, 100 μg/kg/week, dependent on which dose level group to be enrolled into. If needed, a dose between greater than 100 μg/kg/week and up to 200 μg/kg/week will be tested based on the data received from the first 4 groups. The dose (volume) of study drug will be adjusted according to the participant’s weight at each visit. The total duration of the trial is up to approximately 165 weeks. Duration includes up to approximately 4weeks of screening, plus 156 weeks of treatment (52 weeks of treatment in the Randomized Period and additional 104 weeks of treatment in the Open-Label Extension Period) and a 5-week follow-up period.
REC name
London - City & East Research Ethics Committee
REC reference
22/LO/0005
Date of REC Opinion
24 Feb 2022
REC opinion
Further Information Favourable Opinion