ASND0045 (teACH)
Research type
Research Study
Full title
A Phase 2b, Multicenter, Double-Blind, Randomized, Placebo-controlled Trial evaluating Efficacy and Safety of Subcutaneous Doses of Navepegritide Administered Once Weekly for 52 Weeks in Adolescents (12-<18 years of age) with Achondroplasia.
IRAS ID
1010463
Contact name
Claus Strange
Contact email
Sponsor organisation
Ascendis Pharma Growth Disorders A/S
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.
Navepegritide is a chemical that plays a role in controlling the movement of blood around the body and bone growth. There is an approved
treatment using navepegritide, which requires daily injections under the skin to promote bone growth in children with ACH. However, daily
injections can be burdensome. The TransCon (Transient Conjugation) technology is designed to provide sustained release of
navepegritide, decreasing injection frequency to once-weekly. Navepegritide 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 assess the safety and effectiveness of navepegritide compared to placebo in adolescents age 12 - <18 years old. Participants will be randomised (allocated by chance, like a flip of the coin) into either the placebo or active treatment group for up to 52 weeks or until they hare judged to have reached adult height. The study is funded by Ascendis Pharma Growth Disorders A/S and aims to recruit approximately 24 participants in UK and EU.REC name
North East - Newcastle & North Tyneside 1 Research Ethics Committee
REC reference
25/NE/0027
Date of REC Opinion
12 Mar 2025
REC opinion
Further Information Favourable Opinion