ASND0042 (COACH)
Research type
Research Study
Full title
A Phase 2, Open-Label, Single-Arm, 156-week Trial to Investigate the Efficacy, Safety and Tolerability of Combined Once Weekly Navepegritide and Lonapegsomatropin in Children with Achondroplasia
IRAS ID
1009262
Contact name
Claus Strange
Contact email
Sponsor organisation
Ascendis Pharma Growth Disorders A/S
Research summary
Achondroplasia (ACH) is the most common form of abnormal skeletal growth and is caused by a gene mutation. Individuals with ACH have short stature (but an average size trunk), large head with 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 during their lives. CNP is a chemical that plays a role in in controlling bone growth. The TransConTM (Transient Conjugation) technology is designed to provide sustained release of CNP. TransCon CNP (called navepregritide) may help with bone growth in children with ACH. Growth hormone (GH) is a key regulator of growth of the long bones during infant development. A sustained-release form of human GH using TransCon technology (called lonapegsomatropin,) has been approved in the EU, US and the UK (as “Skytrofa”) for use in children with growth failure due to growth hormone deficiency. Both treatments are administered as injections under the skin once a week. Compared to treatment with CNP or GH alone, treatment with both CNP and GH is expected to provide additional benefit the. growth of long bones/limbs and associated clinical outcomes, due to the two medications targeting different parts of the underlying cause of ACH. The primary aims of this clinical study are to investigate the effect of navepegritide and lonapegsomatropin on linear growth in children with ACH and to see how safe treatment with a combination of the two medications is. Quality of life and body composition and how the two drugs work within the body when given together, will be assessed. 18 patients aged 2-11 years are planned to be included in the study, which involves a 5-week screening period, followed by a 3 year treatment period, where participants will receive both nagepregritide and lonapegsomatropin once weekly, and a follow up visit 5 weeks later.
REC name
East of Scotland Research Ethics Service REC 2
REC reference
24/ES/0021
Date of REC Opinion
23 Apr 2024
REC opinion
Further Information Favourable Opinion