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Achondroplasia

Current Clinical Trials: Achondroplasia

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: The objectives of the study are to 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 socialisation 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: Enrolling

Additional information: View on Clinicaltrials.gov


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: The objectives of the study are to 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 socialisation impact)

Data will be collected over a minimum of the three years prior to the date of enrolment. 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: Enrolling

Additional information: View on Clinicaltrials.gov


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 is currently recruiting participants

Additional information: View on Clinicaltrials.gov


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: BMN 111 (Analog of CNP for Achondroplasia) or Placebo

Study Type: Phase 2

Goal: This randomized, double-blind, placebo-controlled study is to evaluate is to evaluate the safety and efficacy of BMN 111 in infants and young children

Status: Enrolling by invitation only

Additional information: View on Clinicaltrials.gov


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: BMN 111 (Analog of CNP for Achondroplasia) or Placebo

Study Type: Phase 3

Goal: A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia

Status: This study is enrolling participants by invitation only

Additional information: View on Clinicaltrials.gov


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: BMN 111 (Analog of CNP for Achondroplasia)

Study Type: Phase 3

Goal: The intent and design of this Phase 3 study is to assess BMN 111 as a therapeutic option for the treatment of children with Achondroplasia

Status: Enrolling participants by invitation only

Additional information: View on Clinicaltrials.gov


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: BMN 111 (Analog of CNP for Achondroplasia)

Study Type: Phase 2

Goal: This is a Phase 2, Open-Label, Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children with Achondroplasia. The primary objective is to 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: Active, not recruiting.

Additional information: View on Clinicaltrials.gov

Completed Clinical Trials: Achondroplasia

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: BMN 111

Study Type: Phase 1

Goal: The purpose of this study is to measure how much of the study drug gets into the blood- stream 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

Additional information: View on Clinicaltrials.gov


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: BMN111 (Analog of CNP for Achondroplasia)

Study Type: Phase 2

Goal: To assess the safety and tolerability of daily BMN 111 administered to children with achondroplasia

Status: Completed

Additional information: View on Clinicaltrials.gov

Roadmap to a New Therapy

Roadmap to a new therapy

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Clinical Trial

Study 111-501: Study 111-501: The Impact of Achondroplasia on Quality of Life, Healthcare Resource Use, Clinical, Socio-economic and Psychosocial state of the Individual (LIAISE).
Read more »

Clinical Trial

Study 111-502: Lifetime Impact Study for Achondroplasia (LISA).
Read more »

Clinical Trial

Study 111-901: A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients with Achondroplasia
Read more »

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
Read more »

Clinical Trial

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
Read more »

Clinical Trial

Study 111-202: A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia (ACH)
Read more »

Clinical Trial

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
Read more »

Clinical Trial

Study 111-205: A Phase 2 Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children With Achondroplasia
Read more »

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Lysosomal Storage Disorders (MPS I, MPS IVA, MPS VI, CLN2 disease) PKU, LEMS

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