ASND0030 (reACHin)
Research type
Research Study
Full title
A Phase 2, Multicenter, Double-Blind, Randomized, Placebo-controlled Trial, evaluating Safety, Tolerability, and Efficacy of Subcutaneous Doses of TransCon CNP Administered Once Weekly for 52 Weeks in Infants (0 to <2 years of age) with Achondroplasia followed by an Open Label Extension (OLE) period.
IRAS ID
1008877
Contact name
Claus Strange
Contact email
Sponsor organisation
Ascendis Pharma Growth Disorders A/S
Clinicaltrials.gov Identifier
Research summary
Achondroplasia (ACH) is the most common form of skeletal growth disorder and the most frequent form disproportionately short stature, where the arms and legs are short in proportion to the body. 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 with an average size trunk, large head with a prominent forehead, and shortened limbs. Associated complications occur at various developmental stages. Newborns with ACH can be at high risk of problems related to growth of the skull and spine, such as a curvature of the spine or spinal cord compression causing weakness. Many infants with ACH may have sleep apnea and recurrent ear infections which may lead to hearing loss.
CNP is a chemical that plays a role in in controlling the movement of blood around the body and bone growth. There is an approved treatment using CNP, which requires daily injections under the skin to promote bone growth in children with ACH. However, daily injections can be burdensome. The TransConTM (Transient Conjugation) technology is designed to provide sustained release of CNP, decreasing injection frequency to once-weekly. TransCon CNP has been used in children aged 2-11 years in previous studies.
International clinical recommendations are to investigate children with ACH from birth, and to initiate treatment as early as possible. This study will therefore assess the safety and effectiveness of TransCon CNP compared to placebo in infants from birth to 2 years of age. Participants will be randomised (allocated by chance, like a flip of the coin) into either the placebo or active treatment group for 52 weeks, followed by an optional open-label treatment phase for a further 52 weeks. The study is funded by Ascendis Pharma Growth Disorders A/S and aims to recruit approximately 66 infants worldwide, including North America, Asia-Pacific, Europe and the UK.REC name
West Midlands - Edgbaston Research Ethics Committee
REC reference
24/WM/0006
Date of REC Opinion
20 Feb 2024
REC opinion
Further Information Favourable Opinion