Study of a new drug for children with achondroplasia
Part of Bones, joints & muscles, Genetic & congenital clinical trials.
This trial tests a new medicine for children with achondroplasia (a common form of dwarfism). It aims to see if the drug can improve growth. The study is for children aged 2.5 to under 11 years old who have not started puberty and can walk without help.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Your child has a confirmed diagnosis of achondroplasia with a specific FGFR3 gene mutation.
- Your child is between 2.5 and under 11 years old.
- Your child has not yet started puberty (Tanner stage 1).
- Your child can walk without assistance.
- Your child has not had any prior treatments that affect growth, like growth hormone or other experimental drugs for achondroplasia.
- Your child does not have other conditions that cause short stature or serious heart, bone, or sleep problems.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
Similar studies
Other trials that look related to this one.
This study tests an oral medicine (ABSK061) to see if it can safely improve growth in children with achondroplasia. It is a phase 1/2 trial, meaning it is early research to find the right dose and check for benefits.
This trial tests a new medicine, BMN 333, against an existing one (vosoritide) to see if it helps children with achondroplasia grow. It's for kids who haven't used these treatments before.
This trial tests a new weekly injection called TransCon CNP for infants with achondroplasia (the most common form of dwarfism). The goal is to see if it can help children grow better than a placebo (a shot with no medicine).
This trial tests an oral medication called infigratinib to see if it can help young children with achondroplasia. It is for children under 3 years old who have a confirmed genetic diagnosis.
This study tests an experimental oral medication (TYRA-300) for children with achondroplasia, a common form of dwarfism. The goal is to see if it can safely improve growth in children whose growth plates are still open.
This study is tracking how people with achondroplasia or hypochondroplasia move their limbs in daily life using a small wearable device. The information will help doctors better understand these conditions and may lead to better care.
Hear when a new Achondroplasia trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.