Current Studies
Study 111-206
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
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Indication
Achondroplasia
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Investigational Therapeutic
Vosoritide (BMN 111)
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Study Type
Phase 2
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Goal
Evaluate the safety and efficacy of BMN 111 in infants and young children
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Status
This study is active, but not recruiting participants
Study 111-502
Lifetime Impact Study for Achondroplasia (LISA) Patients from over 3 years old through to adults with Achondroplasia. Enrolling subjects in LatAm region only.
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Indication
Achondroplasia
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Investigational Therapeutic
None
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Study Type
Observational
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Goal
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.
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Status
This study is active, but not recruiting participants
Study 111-208
An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children with Achondroplasia
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Indication
Achondroplasia
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Investigational Therapeutic
Vosoritide (BMN 111)
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Study Type
Phase 2
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Goal
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
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Status
This study is enrolling participants by invitation only
Study 111-302
A Phase 3, Open-Label Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children with Achondroplasia
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Indication
Achondroplasia
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Investigational Therapeutic
Vosoritide (BMN 111)
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Study Type
Phase 3
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Goal
Assess BMN 111 as a therapeutic option for the treatment of children with achondroplasia
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Status
This study is active, but not recruiting participants
Study 111-205
A Phase 2 Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children With Achondroplasia
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Indication
Achondroplasia
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Investigational Therapeutic
Vosoritide (BMN 111)
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Study Type
Phase 2
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Goal
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
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Status
This study is active, but not recruiting participants
Completed Studies
Study 111-901
A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients with Achondroplasia
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Indication
Achondroplasia
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Investigational Therapeutic
None
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Study Type
Observational
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Goal
Collection of consistent baseline growth measurements on pediatric patients with achondroplasia being considered for subsequent enrollment in Study 111-301 or 111-206
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Status
This study has been completed
Study 111-501
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.
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Indication
Achondroplasia
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Investigational Therapeutic
None
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Study Type
Observational
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Goal
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.
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Status
This study has been completed
Study 111-101
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
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Indication
Achondroplasia
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Investigational Therapeutic
Vosoritide (BMN 111)
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Study Type
Phase 1
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Goal
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.
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Status
This study has been completed
Study 111-202
A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia (ACH)
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Indication
Achondroplasia
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Investigational Therapeutic
Vosoritide (BMN 111)
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Study Type
Phase 2
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Goal
Assess the safety and tolerability of daily BMN 111 administered to children with achondroplasia
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Status
This study has been completed
Study 111-301
A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of BMN 111 in Children with Achondroplasia
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Indication
Achondroplasia
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Investigational Therapeutic
Vosoritide (BMN 111)
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Study Type
Phase 3
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Goal
Evaluate the efficacy and safety of BMN 111 in children with achondroplasia
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Status
This study has been completed