Current Studies
Study 111-209
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
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Indication
Achondroplasia
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Investigational Therapeutic
BMN 111 (Analog of CNP for Achondroplasia) or Placebo
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Study Type
Phase 2
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Goal
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
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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 active, but not recruiting participants
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
Study 111-104
RecruitingA Phase 1, Randomized, Open-Label, Sponsor-Open, Two-Sequence, Two-Period Crossover Study to Investigate the Bioequivalence of a Single Subcutaneous Dose of BMN 111 Administered Using Either a Vial and Syringe or Injector Pen in Healthy Adult Participants
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Indication
Achondroplasia
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Investigational Therapeutic
Combination Product: BMN111 administration via Injector Pen
Drug: BMN111 administration via vial and syringe
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Study Type
Phase 1
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Goal
Investigate the Bioequivalence of a Single Subcutaneous Dose of BMN 111 Administered Using Either a Vial and Syringe or Injector Pen in Healthy Adult Participants
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Status
This study is currently recruiting participants
Completed 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 has been completed
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 has been completed
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