Vosoritide

(111-205 [Canopy ACH-2 EXT])

Trial Status Active, Not Recruiting

Condition

Achondroplasia

Technology

• Peptide

Title

A Study to Evaluate Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children With Achondroplasia (ACH)

Study Description

This Phase 2, open-label, extension study evaluates the long-term safety, tolerability, and efficacy of BMN 111 (vosoritide) in children with achondroplasia.

Primary Outcome Measure*

• Incidence of treatment-emergent adverse events

Select Inclusion Criteria*

• Have completed 24 months of vosoritide treatment in Study 111-202
• Parent/guardian provides informed written consent if required
• If sexually active, must be willing to use a highly effective method of contraception while participating in the study

Secondary Outcome Measures*

• Annualized growth velocity
• Height standard score

Select Exclusion Criteria*

• Requires any investigational agent prior to completion of study period
• Have a condition or circumstance that places the subject at high risk during this study

* Additional measures/criteria may apply.

For reference and comprehensive trial information, visit ClinicalTrials.gov NCT02724228

Pre-IND

Phase 1

Phase 2

Phase 3

Phase 4

Vosoritide

(111-208 [Canopy ACH-2I EXT])

Trial Status Active, Not Recruiting

Condition

Achondroplasia

Technology

• Peptide

Title

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

Study Description

This Phase 2, open-label, multicenter, long-term extension study, with approximately 70 subjects, evaluates the safety and efficacy of BMN 111 (vosoritide) in children with achondroplasia until subjects reach near-adult final height.

Primary Outcome Measures*

• Evaluate the incidence of treatment-emergent adverse events
• Evaluate change in height/length z-score in children with ACH treated with vosoritide

Select Inclusion Criteria*

• Must have completed Study 111-206 on investigational treatment
• Parent/guardian provides informed written consent if required

Secondary Outcome Measures*

• Evaluate the change from baseline in mean annualized growth velocity
• Characterize maximum concentration

Select Exclusion Criteria*

• Permanently discontinued vosoritide or placebo prior to completion of Study 111-206
• Have a clinically significant finding or arrhythmia on ECG that indicates abnormal cardiac function or conduction or QTc-F >450 msec

* Additional measures/criteria may apply.
ACH, achondroplasia; ECG, electrocardiogram; QTcF, corrected QT interval by Fridericia.

For reference and comprehensive trial information, visit ClinicalTrials.gov NCT03989947

Pre-IND

Phase 1

Phase 2

Phase 3

Phase 4

Vosoritide

(111-209 [Canopy ACH-2H])

Trial Status Active, Not Recruiting

Condition

Achondroplasia

Technology

• Peptide

Title

A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia

Study Description

This is a Phase 2 randomized, open-label clinical trial of BMN 111 (vosoritide) in infants and young children with a diagnosis of achondroplasia at a heightened risk of requiring cervicomedullary decompression surgery.

Primary Outcome Measure*

• Incidence of treatment-emergent adverse events

Select Inclusion Criteria*

• Parent/guardian provides informed written consent if required
• Have ACH, documented by genetic testing

Secondary Outcome Measure*

• Evaluate the effect of vosoritide on total foramen magnum volume

Select Exclusion Criteria*

• Have hypochondroplasia or short-stature condition other than achondroplasia
• Have CMC that either does not require surgical intervention or does require immediate surgical intervention

* Additional measures/criteria may apply.
ACH, achondroplasia; CMC, cervicomedullary compression.

For reference and comprehensive trial information, visit ClinicalTrials.gov NCT04554940

Pre-IND

Phase 1

Phase 2

Phase 3

Phase 4

Vosoritide

(111-302 [Canopy ACH-3 EXT])

Trial Status Active, Not Recruiting

Condition

Achondroplasia

Technology

• Peptide

Title

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

Study Description

This Phase 3, open-label, long-term extension study is intended and designed to assess BMN 111 (vosoritide) as a therapeutic option for the treatment of children with achondroplasia.

Primary Outcome Measure*

• Change from baseline in mean annualized growth velocity

Select Inclusion Criteria*

• Must have completed Study 111-301
• Females ≥10 years old, or those with menses, need negative pregnancy tests at baseline and during the study

Secondary Outcome Measures*

• Changes in health-related quality of life
• Potential changes in daily activity performance
• Characterize maximum concentration of vosoritide in plasma

Select Exclusion Criteria*

• Permanently discontinued vosoritide or placebo prior to completion of Study 111-301
• Have a clinically significant finding that indicates abnormal cardiac function
• Evidence of slowed growth of at least 6 months or closed growth plates in lower extremity X-rays

* Additional measures/criteria may apply.

For reference and comprehensive trial information, visit ClinicalTrials.gov NCT03424018

Pre-IND

Phase 1

Phase 2

Phase 3

Phase 4

Vosoritide

(111- 603 [Acorn])

Trial Status Recruiting

Condition

Achondroplasia

Technology

• Peptide

Title

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

Study Description

This post-authorization safety study will evaluate the long-term safety of treatment with vosoritide in patients with achondroplasia in a real-world setting.

Primary Outcome Measure*

• Exposure-adjusted incidence rate of all new bone-related safety events of interest which have not previously been observed in patients

* Additional measures/criteria may apply.

For reference and comprehensive trial information, visit EMA.Europa.eu EUPAS47514

Pre-IND

Phase 1

Phase 2

Phase 3

Phase 4

Vosoritide

(111-605 [VISTA])

Trial Status Recruiting

Condition

Achondroplasia

Technology

• Peptide

Title

A Participant-mediated Observational Virtual Registry of Children With Achondroplasia in the United States (VISTA).

Study Description

This is an observational study of a pediatric cohort with achondroplasia in the United States. This cohort consists of both individuals treated and untreated with vosoritide.

Primary Outcome Measure*

• Height, weight, body mass index, head circumference, annualized grown velocity, surgical procedures and/or medical interventions by age

Select Inclusion Criteria*

• Physician diagnosis of achondroplasia
• Age at time of enrollment
• Receiving medical care in the United States

Secondary Outcome Measure*

• Not applicable

Select Exclusion Criteria*

• Lack of any medical records

* Additional measures/criteria may apply.

For reference and comprehensive trial information, visit ClinicalTrials.gov NCT06168201

Pre-IND

Phase 1

Phase 2

Phase 3

Phase 4

BMN 333

Long-acting CNP

Trial Status Not started

Condition

Achondroplasia, Hypochondroplasia

Technology

• Peptide

Pre-IND

Phase 1

Phase 2

Phase 3

Phase 4