A clinical trial to evaluate efficacy and safety of TransCon CNP in participants with achondroplasia
Research type
Research Study
Full title
ApproaCH: A Phase 2b, Multicenter, Double-Blind, Randomized, Placebo-controlled Trial evaluating Efficacy and Safety of Subcutaneous Doses of TransCon CNP Administered Once Weekly for 52 Weeks in Children with Achondroplasia followed by an Open Label Extension period
IRAS ID
1006817
Contact name
Claus Strange
Contact email
Sponsor organisation
Ascendis Pharma Growth Disorders A/S
Eudract number
2022-002954-25
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. ACH is caused by a gene mutation. 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. Existing interventions do not address the underlying cause of ACH and individuals with the condition undergo multiple surgeries and interventions throughout their lives.
CNP is a chemical that plays a role in in controlling the movement of blood around the body and bone growth. The TransCon (Transient Conjugation) technology is designed to provide sustained release of CNP, allowing maintenance of steady therapeutic levels between doses. Use of TransCon technology also allows for weekly injections, under the skin, thereby decreasing injection frequency, allowing for good compliance and convenience, and reducing burden compared to daily injection.
This study will assess the safety and effectiveness of TransCon CNP compared to placebo. Approximately 80 participants aged between 2 – 11 with ACH will be enrolled and will receive TransCon CNP or placebo for 52 weeks. Neither study doctor or participant will know if they are receiving TransCon CNP or placebo followed by a 52 week period where the participants and study doctor will know what medication they are receiving for a total of 104 weeks at a dose of 100 μg CNP/kg weekly, followed by a 5-week Follow-up period after EoT.
This study is funded by Ascendis Pharma Growth Disorders A/S and will take place at 1 Study center in the UK.
REC name
Yorkshire & The Humber - Leeds West Research Ethics Committee
REC reference
23/YH/0051
Date of REC Opinion
26 Jul 2023
REC opinion
Further Information Unfavourable Opinion