Vosoritide

(111- 603 [Acorn])

Trial Status Recruiting

Condition

Achondroplasia

Phase

• Phase 4

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

Vosoritide

(111-605 [VISTA])

Trial Status Recruiting

Condition

Achondroplasia

Phase

• Phase 4

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

BMN 333 Long-Acting CNP

(333-301 [ASPEN])

Trial Status Recruiting

Condition

Achondroplasia

Phase

• Phase 3

Title

A Phase 2/​3 Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia (ASPEN)

Study Description

To evaluate the effects of BMN 333 on growth compared with vosoritide in participants with achondroplasia who have not received any growth-promoting treatments. The study includes 2 parts: the Phase 2 part will select the optimal BMN 333 dose to be used in Phase 3 and determine study continuation into Phase 3; the Phase 3 part will compare the effects of the selected dose of BMN 333 with vosoritide.

Primary Outcome Measure*

• Predicted annualized growth velocity (AGV; Phase 2) and AGV (Phase 3)

Select Inclusion Criteria*

• Aged ≥ 2 to < 11 years (Phase 2) or ≥ 2 to < 18 years (Phase 3)
• Achondroplasia (confirmed by documented genetic testing) and open epiphyses
• Tanner Stage I (Phase 2) or any Tanner stage (Phase 3)
• Ambulatory and able to stand without assistance

Secondary Outcome Measures*

• Change from baseline in standing height, height Z-score, and upper to lower body segment ratio
• Incidence of adverse events (AEs), serious AEs, and events of interest

Select Exclusion Criteria*

• Any short stature condition other than achondroplasia
• History of any renal insufficiency or cardiac/ cardiovascular disease that places the participant at increased risk of an adverse cardiac outcome in the setting of hypotension
• Bone fractures of the long bones or spine within 6 months prior to screening
• Used vosoritide, any other approved product (except growth hormone [GH], as detailed below), investigational product, or investigational medical device for the treatment of achondroplasia or short stature at any time
• Been treated with GH, insulin-like growth factor 1, or anabolic steroids in the 6 months prior to treatment start

* Additional measures/criteria may apply.

For reference and comprehensive trial information, visit ClinicalTrials.gov NCT07441876

Vosoritide

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

Trial Status Active, Not Recruiting

Condition

Achondroplasia

Phase

• Phase 2

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

Vosoritide

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

Trial Status Active, Not Recruiting

Condition

Achondroplasia

Phase

• Phase 2

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

Vosoritide

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

Trial Status Active, Not Recruiting

Condition

Achondroplasia

Phase

• Phase 2

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

Vosoritide

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

Trial Status Active, Not Recruiting

Condition

Achondroplasia

Phase

• Phase 3

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