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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

  • 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 is active, but not recruiting participants

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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 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 enrolling participants by invitation only

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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 111-901

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

Visit Study 111-301 Now