Achondroplasia is a rare genetic bone growth condition characterized by disproportionate short stature, curvature of the spine and an enlarged head (macrocephaly).
Learn more about our achondroplasia clinical trials below.
A multicentre, non-interventional study to evaluate long-term safety in patients with achondroplasia treated with vosoritide
Achondroplasia
To evaluate the long-term impact of treatment with vosoritide on safety and skeletal effects on patients with Achondroplasia in a real-world setting in Europe
Vosoritide
This study is currently recruiting participants
Observational
Drug Use Survey of vosoritide Subcutaneous Injection 0.4 mg/0.56 mg/1.2 mg Long-Term Safety and Effectiveness in Patients with Achondroplasia in Japan
Achondroplasia
To evaluate long-term safety and efficacy of vosoritide in Japan
Vosoritide
This study is currently recruiting participants
Observational
A Virtual Study in Achondroplasia in the United States
Achondroplasia
A patient centered online real world study to understand the lived experience of children with Achondroplasia in the United States
N/A
This study is currently recruiting participants
Observational
Phase 2 randomized, open-label clinical trial of BMN 111 in infants and young children with a diagnosis of Achondroplasia at a heightened risk of requiring cervicomedullary decompression surgery
Achondroplasia
This randomized, open label study is to evaluate is to evaluate the safety and effect of BMN 111 in infants and young children with Achondroplasia at risk of requiring cervicomedullary decompression surgery
BMN 111 (Analog of CNP for Achondroplasia) or Placebo
This study is active, but not recruiting participants
Phase 2
An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children with Achondroplasia
Achondroplasia
This is a Phase 2, open-label, long-term extension study to evaluate the safety and efficacy of BMN111 in children with achondroplasia until subjects reach near-adult final height
Vosoritide (BMN 111)
This study is active, but not recruiting participants
Phase 2
A Phase 3, Open-Label Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children with Achondroplasia
Achondroplasia
Assess BMN 111 as a therapeutic option for the treatment of children with achondroplasia
Vosoritide (BMN 111)
This study is active, but not recruiting participants
Phase 3
A Phase 2 Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children With Achondroplasia
Achondroplasia
Evaluate the long-term safety and tolerability of daily SC injections of BMN 111 in children with ACH who have completed two years of treatment in the 111-202 study
Vosoritide (BMN 111)
This study is active, but not recruiting participants
Phase 2
A Phase 2 Randomized, Double-Blind, Placebo-Controlled Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia, Age 0 to < 60 Months
Achondroplasia
Evaluate the safety and efficacy of BMN 111 in infants and young children
Vosoritide (BMN 111)
This study has been completed
Phase 2
Lifetime Impact Study for Achondroplasia (LISA) Patients from over 3 years old through to adults with Achondroplasia. Enrolling subjects in LatAm region only.
Achondroplasia
Describe the impact on individuals with achondroplasia based on Quality of life (QoL), Clinical burden (functional impact, comorbidities, complications, medical and surgical care), Healthcare resource use, Socio-economic burden (educational, personal, employment and financial impact) and Psychosocial burden (psychological and socialization impact) Data will be collected over a minimum of the three years prior to the date of enrollment. Clinical and healthcare resource use data will be collected from medical records. Data about QoL, mobility, psychosocial burden, socio-economic burden and healthcare resource use will be collected via validated and structured questionnaires.
None
This study has been completed
Observational
A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients with Achondroplasia
Achondroplasia
Collection of consistent baseline growth measurements on pediatric patients with achondroplasia being considered for subsequent enrollment in Study 111-301 or 111-206
None
This study has been completed
Observational
The Impact of Achondroplasia on Quality of Life, Healthcare Resource Use, Clinical, Socio-economic and Psychosocial state of the Individual (LIAISE). Patients from over 5 years old through to adults with Achondroplasia. Enrolling subjects in EU region only.
Achondroplasia
Describe the impact on individuals with achondroplasia based on quality of life (QoL), clinical burden (functional impact, comorbidities, complications, medical and surgical care), healthcare resource use, socio-economic burden (educational, personal, employment and financial impact) and psychosocial burden (psychological and socialization impact) Data will be collected over a minimum of the five years prior to the date of enrollment. Clinical and healthcare resource use data will be collected from medical records. Data about QoL, mobility, psychosocial burden, socio-economic burden and healthcare resource use will be collected via a booklet of validated and structured questionnaires.
None
This study has been completed
Observational
Phase 1, two-part, double-blind, placebo-controlled study to evaluate safety, tolerability, and pharmacokinetics of single and multiple doses of BMN 111 administered to healthy adult volunteers
Achondroplasia
Measure how much of the study drug gets into the bloodstream and how long it takes the body to get rid of it when given as a single dose. Information about any side effects that may occur will also be collected.
Vosoritide (BMN 111)
This study has been completed
Phase 1
A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia (ACH)
Achondroplasia
Assess the safety and tolerability of daily BMN 111 administered to children with achondroplasia
Vosoritide (BMN 111)
This study has been completed
Phase 2
A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of BMN 111 in Children with Achondroplasia
Achondroplasia
Evaluate the efficacy and safety of BMN 111 in children with achondroplasia
Vosoritide (BMN 111)
This study has been completed
Phase 3