Achondroplasia Clinical Trials

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.

Vosoritide

Study 111-603 Acorn Recruiting

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

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Indication

Achondroplasia

Goal

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

Investigational therapeutic

Vosoritide

Status

This study is currently recruiting participants

Study Type

Observational

Study 111-604 Recruiting

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

Indication

Achondroplasia

Goal

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

Investigational therapeutic

Vosoritide

Status

This study is currently recruiting participants

Study Type

Observational

Study 111-605 VISTA Recruiting

A Virtual Study in Achondroplasia in the United States

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Indication

Achondroplasia

Goal

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

Investigational therapeutic

N/A

Status

This study is currently recruiting participants

Study Type

Observational

Study 111-209 Active, Not Recruiting

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

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

Investigational therapeutic

BMN 111 (Analog of CNP for Achondroplasia) or Placebo

Status

This study is active, but not recruiting participants

Study Type

Phase 2

Study 111-208 Active, Not Recruiting

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

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Indication

Achondroplasia

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

Investigational therapeutic

Vosoritide (BMN 111)

Status

This study is active, but not recruiting participants

Study Type

Phase 2

Study 111-302 Active, Not Recruiting

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

Goal

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

Investigational therapeutic

Vosoritide (BMN 111)

Status

This study is active, but not recruiting participants

Study Type

Phase 3

Study 111-205 Active, Not Recruiting

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

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

Investigational therapeutic

Vosoritide (BMN 111)

Status

This study is active, but not recruiting participants

Study Type

Phase 2

Study 111-206 Complete

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

Goal

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

Investigational therapeutic

Vosoritide (BMN 111)

Status

This study has been completed

Study Type

Phase 2

Study 111-502 Complete

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

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.

Investigational therapeutic

None

Status

This study has been completed

Study Type

Observational

Study 111-901 Complete

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

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Indication

Achondroplasia

Goal

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

Investigational therapeutic

None

Status

This study has been completed

Study Type

Observational

Study 111-501 Complete

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

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.

Investigational therapeutic

None

Status

This study has been completed

Study Type

Observational

Study 111-101 Complete

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

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.

Investigational therapeutic

Vosoritide (BMN 111)

Status

This study has been completed

Study Type

Phase 1

Study 111-202 Complete

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

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Indication

Achondroplasia

Goal

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

Investigational therapeutic

Vosoritide (BMN 111)

Status

This study has been completed

Study Type

Phase 2

Study 111-301 Complete

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

Goal

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

Investigational therapeutic

Vosoritide (BMN 111)

Status

This study has been completed

Study Type

Phase 3

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