- active not recruitingPHASE3
An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
This study is looking into a medicine called BMN 111 for children with Achondroplasia, a type of dwarfism. It's a follow-up to a previous study, checking how safe and effective the medicine is in the long run.
United States · Australia · Germany - recruitingPHASE2
A Study of TYRA-300 in Children With Achondroplasia: BEACH301
This study, called BEACH301, is looking at a new medicine called TYRA-300 for children aged 3 to 10 with achondroplasia. We want to find out if it's safe, how well children tolerate it, and what dose might work best to help with growth.
United States · Australia · Canada - active not recruitingPHASE2
A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)
This study looks at a medicine called BMN 111 for children with achondroplasia, a condition affecting bone growth. It aims to see if the medicine is safe and if it works well over a long time, building on previous research. It's for children who have already been taking BMN 111.
United States · Australia · France - recruitingPHASE2, PHASE3
Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia
This study is looking for children with achondroplasia, a type of dwarfism, who haven't had prior growth treatment. It compares a new medicine called BMN 333 with an existing one, Vosoritide, to see how well they help children grow and if they are safe.
United States · Australia · Canada - enrolling by invitationPHASE2, PHASE3
A Clinical Trial to Investigate Long-term Safety, Tolerability, and Efficacy of Weekly Subcutaneous Doses With TransCon CNP in Children and Adolescents With Achondroplasia
This study is for children and teenagers with achondroplasia who have already taken part in a trial for TransCon CNP. It aims to check the long-term safety and effects of this weekly medicine. Participants will continue treatment until their bones stop growing, with regular check-ups.
United States · Australia · Austria - enrolling by invitationPHASE2
Extension Study of Infigratinib in Children With Achondroplasia (ACH)
This study looks at a medicine called Infigratinib for children and teenagers aged 3-18 with achondroplasia, a type of dwarfism. It's an ongoing study to check the medicine's safety, how well it works, and its long-term effects on growth and quality of life.
United States · Argentina · Australia - authorisedPhase II and Phase III (Integrated)
A Multicenter, Randomized, Operationally Seamless Phase 2/3 Study to Evaluate the Efficacy and Safety of BMN 333 versus Vosoritide in Children with Achondroplasia
This study is looking at two medicines, BMN 333 and Voxzogo, for children with achondroplasia. It aims to see how well they help children grow taller and if they are safe to use. It's comparing a new treatment with one that's already approved.
Poland · Italy · Romania - recruiting
Prospective Longitudinal Monocentric Study to Measure Limb Movement in Patients With FGFR3-related Skeletal Dysplasia
This study is looking at how children and adults with achondroplasia or hypochondroplasia move their arms and legs. We want to see if a special digital tool called Syde® can accurately measure these movements. This will help us understand the conditions better.
France - ongoing, recruitingTherapeutic confirmatory (Phase III)
A Phase 3, Multicenter, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Infigratinib in Children 3 to <18 Years of Age with Achondroplasia: PROPEL 3
This study is testing a new medicine, Infigratinib, for children aged 3 to under 18 with achondroplasia. It aims to see if the medicine helps children grow taller and improves other health aspects, compared to a dummy pill.
Spain · France · Germany - active not recruitingPHASE2
An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia
This study is looking at an experimental medicine called BMN 111 for children with achondroplasia. It's an extension of a previous study, checking how safe and effective the medicine is long-term until children stop growing.
United States · Australia · Japan - ongoing, recruitingTherapeutic exploratory (Phase II)
A Phase 2, Open-Label, Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children with Achondroplasia
This study looks at the long-term effects of a medication called Voxzogo for children with achondroplasia. It checks how safe the medicine is and if it helps children grow. This is an ongoing study where researchers continue to monitor children already receiving the treatment.
France - recruitingPHASE2
A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Infants (0 to <2 Years of Age) With Achondroplasia
This study is testing a new medicine, Navepegritide, for babies and toddlers under two years old with achondroplasia. It aims to see if the medicine is safe and helps with their growth compared to a dummy medicine.
United States · Australia · Austria - active not recruitingPHASE2
A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia
This study is testing a medicine called vosoritide for babies up to one year old with achondroplasia. We want to see if it's safe and helps improve a tricky neck problem often seen in achondroplasia, especially in babies who might need surgery.
Australia · United Kingdom - active not recruitingPHASE2
A Phase 2 Clinical Trial to Evaluate Efficacy, Safety, and Tolerability of Navepegritide in Combination With Lonapegsomatropin in Children With Achondroplasia
This study is looking at a new way to treat achondroplasia in children aged 2-11. It combines two existing medicines, navepegritide and lonapegsomatropin, given as weekly injections, to see if they can work better together.
Denmark · Ireland · United Kingdom - recruitingPHASE2
Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH)
This study is testing a new medicine, infigratinib, for children under 3 years old with achondroplasia. It aims to find the right dose and see if it's safe and helps with growth. Some children will get the medicine, others a dummy pill.
United States · Australia · Canada - recruitingPHASE2
A Clinical Trial to Evaluate Efficacy and Safety of Navepegritide in Adolescents (12 - 18 Years of Age) With Achondroplasia.
This study is looking at a new medicine called navepegritide for teenagers aged 12 to 18 with achondroplasia. We want to see if it helps them grow and if it's safe. It's compared to a dummy medicine over 52 weeks.
Canada · Denmark · France