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23 Sep 2019 - Ascendis Pharma is developing the TransCon CNP program at a swift pace and moving forward with phase 2 ACcomplisH Trial to evaluate the safety and efficacy of TransCon CNP in children with achondroplasia.  

accomplish
Ascendis has recently completed an Investigational New Drug (IND) application with the FDA, and the company expects to enroll the first children for the ACcomplisH trial in late 2019. Here is Ascendis press release. 

An Investigational New Drug (IND) application is the first step in the drug review process by the U.S. Food and Drug Administration (FDA). The application is submitted by the company responsible for developing the drug (the sponsor) to the FDA.

The ACcomplisH Trial is a global, phase 2, randomized, double-blind, placebo-controlled trial that will enroll approximately 60 children, ages 2 to 10 years, with achondroplasia.

The primary endpoint is annualized height velocity at 12 months. Key secondary endpoints include a change in body proportionality at 12 months and change in body mass index at 12 months. All children who complete the ACcomplisH Trial will have the opportunity to receive TransCon CNP in a long-term extension trial. 


TransCon CNP study arms
The 60 children in this trial will be distributed for 6 different groups, by random distribution, and each group will have a different dosage of TransCon CNP or placebo.

Arm 1. TransCon CNP 6 mcg CNP/kg or placebo mimicking TransCon CNP 6 mcg delivered once weekly by subcutaneous injection
Arm 2. TransCon CNP 20 mcg CNP/kg or placebo mimicking TransCon CNP 6 mcg delivered once weekly by subcutaneous injection
Arm 3. TransCon CNP 50 mcg CNP/kg or placebo mimicking TransCon CNP 50 mcg delivered once weekly by subcutaneous injection
Arm 4. TransCon CNP 100 mcg CNP/kg or placebo mimicking TransCon CNP 50 mcg delivered once weekly by subcutaneous injection

Arm 5. TransCon CNP >100 mcg CNP/kg delivered once weekly by subcutaneous injection (to be determined after completion of 100 mcg cohort)


This clinical trial is published at clinicaltrials.gov, yet not recruiting. The first group of children, called Cohort 1, will be recruited in a limited number of study sites. These sites are hospitals where the children are observed, have clinical evaluations and exams and get the first administration of the investigational drug and or placebo. 
Additionally, Ascendis continues to enroll in the ACHieve, a natural history study designed to gain insight into the experience of children with achondroplasia. ACHieve will study comorbidities, body proportionality, and height velocity over time of children with achondroplasia up to 8 years old. No study medication will be administered. 

To contact Ascendis team regarding ACComplisH, use This email address is being protected from spambots. You need JavaScript enabled to view it.

Resources
1. Ascendis Pharma

2. FDA
3. Clinicaltrial.gov

4. Investopedia
5. Achieve-study
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