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Current Studies

 

Study 111-104

Recruiting

A 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

  • Indication

    Achondroplasia

  • Investigational Therapeutic

    Combination Product: BMN111 administration via Injector Pen

    Drug: BMN111 administration via vial and syringe

  • Study Type

    Phase 1

  • 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

  • Status

    This study is currently recruiting participants

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Study 111-603 Acorn

Recruiting

A multicentre, non-interventional study to evaluate long-term safety in patients with achondroplasia treated with VOXZOGO® (vosoritide)

  • Indication

    Achondroplasia

  • Investigational Therapeutic

    VOXZOGO® (vosoritide)

  • Study Type

    Observational

  • Goal

    To evaluate the long-term impact of treatment with VOXZOGO on safety and skeletal effects on patients with Achondroplasia in a real-world setting in Europe

  • Status

    This study is currently recruiting participants

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Study 111-604

Recruiting

Drug Use Survey of VOXZOGO® Subcutaneous Injection 0.4 mg/0.56 mg/1.2 mg Long-Term Safety and Effectiveness in Patients with Achondroplasia in Japan

  • Indication

    Achondroplasia

  • Investigational Therapeutic

    VOXZOGO® (vosoritide)

  • Study Type

    Observational

  • Goal

    To evaluate long-term safety and efficacy of VOXZOGO in Japan

  • Status

    This study is currently recruiting participants

 

Study 111-605 VISTA

Recruiting

A Virtual Study in Achondroplasia in the United States

  • Indication

    Achondroplasia

  • Investigational Therapeutic

    N/A

  • Study Type

    Observational

  • Goal

    A patient centered online real world study to understand the lived experience of children with Achondroplasia in the United States

  • Status

    This study is currently recruiting participants

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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

  • Indication

    Achondroplasia

  • Investigational Therapeutic

    BMN 111 (Analog of CNP for Achondroplasia) or Placebo

  • Study Type

    Phase 2

  • 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

  • Status

    This study is active, but not recruiting participants

     

     

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Study 111-208

An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children with Achondroplasia

  • Indication

    Achondroplasia

  • Investigational Therapeutic

    Vosoritide (BMN 111)

  • Study Type

    Phase 2

  • 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

  • Status

    This study is active, but not recruiting participants

     

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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

  • Indication

    Achondroplasia

  • Investigational Therapeutic

    Vosoritide (BMN 111)

  • Study Type

    Phase 3

  • Goal

    Assess BMN 111 as a therapeutic option for the treatment of children with achondroplasia

  • Status

    This study is active, but not recruiting participants

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Study 111-205

A Phase 2 Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children With Achondroplasia

  • Indication

    Achondroplasia

  • Investigational Therapeutic

    Vosoritide (BMN 111)

  • Study Type

    Phase 2

  • 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

  • Status

    This study is active, but not recruiting participants

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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

  • Indication

    Achondroplasia

  • Investigational Therapeutic

    Vosoritide (BMN 111)

  • Study Type

    Phase 2

  • Goal

    Evaluate the safety and efficacy of BMN 111 in infants and young children

  • Status

    This study has been completed

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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.

  • Indication

    Achondroplasia

  • Investigational Therapeutic

    None

  • Study Type

    Observational

  • 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.

  • Status

    This study has been completed

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Study 111-901

A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients with Achondroplasia

  • Indication

    Achondroplasia

  • Investigational Therapeutic

    None

  • Study Type

    Observational

  • Goal

    Collection of consistent baseline growth measurements on pediatric patients with achondroplasia being considered for subsequent enrollment in Study 111-301 or 111-206

  • Status

    This study has been completed

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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.

  • Indication

    Achondroplasia

  • Investigational Therapeutic

    None

  • Study Type

    Observational

  • 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.

  • Status

    This study has been completed

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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

  • Indication

    Achondroplasia

  • Investigational Therapeutic

    Vosoritide (BMN 111)

  • Study Type

    Phase 1

  • 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.

  • Status

    This study has been completed

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Study 111-202

A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia (ACH)

  • Indication

    Achondroplasia

  • Investigational Therapeutic

    Vosoritide (BMN 111)

  • Study Type

    Phase 2

  • Goal

    Assess the safety and tolerability of daily BMN 111 administered to children with achondroplasia

  • Status

    This study has been completed

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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

  • Indication

    Achondroplasia

  • Investigational Therapeutic

    Vosoritide (BMN 111)

  • Study Type

    Phase 3

  • Goal

    Evaluate the efficacy and safety of BMN 111 in children with achondroplasia

  • Status

    This study has been completed

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