Vosoritide

(111-212 [Canopy HCH-2I])

Trial Status Recruiting

Condition

Hypochondroplasia

Technology

• Peptide

Title

A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months

Study Description

The purpose of this study is to evaluate the safety and efficacy of daily administration of vosoritide in participants with Hypochondroplasia aged 0 to < 36 months over a 52-week period.

Primary Outcome Measures*

• Incidence of treatment-emergent adverse events and serious adverse events
• Change from baseline in standard clinical laboratory values (hematology, urinalysis, and chemistry), heart rate, respiratory rate, temperature, blood pressure, and height Z-score

Select Inclusion Criteria*

• 0 to < 36 months of age
• Weight at Day 1 visit (pre-treatment) ≥3 kg
• Have a confirmed genetic diagnosis of HCH (obtained via whole genome sequencing; presence of a FGFR3 pathogenic variant associated with HCH)

Secondary Outcome Measures*

• Change in height
• Cumulative annualized growth velocity (AGV)
• 6-month interval AGV
• Change from baseline in upper to lower body segment ratio
• Change from baseline in arm span

Select Exclusion Criteria*

• Short stature condition other than HCH
• Have an unstable medical condition likely to require surgical intervention during the study period
• Have been treated with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroids in the 6 months prior to Screening, or long-term treatment (> 3 months) at any time
• Have used any other investigational product or investigational medical device for the treatment of HCH or short stature at any time

* Additional measures/criteria may apply.
HCH, Hypochondroplasia;

For reference and comprehensive trial information, visit ClinicalTrials.gov NCT07126262

Pre-IND

Phase 1

Phase 2

Phase 3

Phase 4

Vosoritide

(111-303 [Canopy HCH-3])

Trial Status Active, Not Recruiting

Condition

Hypochondroplasia

Technology

• Peptide

Title

Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia

Study Description

The intent and design of this Phase 3 study is to assess vosoritide as a therapeutic option for the treatment of children with hypochondroplasia (HCH).

Primary Outcome Measure*

• Change from baseline in annualized growth velocity (AGV)

Select Inclusion Criteria*

• Patients ≥ 3 to < 18 years of age
• Confirmed genetic diagnosis of HCH
• A height Z score of ≤-2 standard deviations in reference to the general population of the same age and sex, as calculated using the Center for Disease Control and Prevention (CDC) growth charts

Secondary Outcome Measure*

• Change from baseline in standing height and height Z-score

Select Exclusion Criteria*

• Have an unstable condition likely to require surgical intervention during the study
• Evidence of decreased growth velocity and/or growth plate closure
• Treated with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroids
• Planned or expected to have limb-lengthening surgery or bone-related surgery (ie, surgery involving disruption of bone cortex, excluding tooth extraction) during the study period

* Additional measures/criteria may apply.
HCH, hypochondroplasia.

For reference and comprehensive trial information, visit ClinicalTrials.gov NCT06455059

Pre-IND

Phase 1

Phase 2

Phase 3

Phase 4

Vosoritide

(111-308 [Canopy HCH-3 EXT])

Trial Status Enrolling by Invitation

Condition

Hypochondroplasia

Technology

• Peptide

Title

Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia (CANOPY-HCH-EXT)

Study Description

The purpose of this study is to evaluate the long-term safety and efficacy of daily doses of vosoritide in participants with HCH

Primary Outcome Measure*

• Change from baseline in height Z-score

Select Inclusion Criteria*

• Completed the Week 52 visit for Study 111-303 or 111-212 and have open epiphyses as assessed by left hand antero-posterior (AP) X-rays
• Females ≥ 10 years old or who have begun menses must have a negative pregnancy test at the Baseline visit and be willing to have additional pregnancy tests during the study
• If sexually active, participants must be willing to use a highly effective method of contraception while participating in the study

Secondary Outcome Measures*

• Change from baseline in height
• Absolute 6- or 12-month interval AGV
• Change from baseline in upper to lower body segment ratio
• Change from baseline in Quality of Life In Short-Statured Youth (QoLISSY) total score

Select Exclusion Criteria*

• Permanently discontinued study treatment in Study 111-303 or 111-212
• Evidence of decreased growth velocity (AGV <1.5 cm/year) as assessed over a period of at least 6 months and growth plate closure assessed as per standard of care
• Planned or expected to have limb-lengthening surgery during the study period
• Planned or expected bone-related surgery during the study period

* Additional measures/criteria may apply.

For reference and comprehensive trial information, visit ClinicalTrials.gov NCT07073014

Pre-IND

Phase 1

Phase 2

Phase 3

Phase 4

Hypochondroplasia

(111-809)

Trial Status Not started

Condition

Hypochondroplasia

Technology

• Observational

Title

Generating Growth Curves for Standing and Sitting Height in Hypochondroplasia: A Multi-Center Retrospective Natural History Study

Study Description

This study will be a retrospective chart review of height measurements in patients ≤18 years old with a clinically or genetically confirmed diagnosis of hypochondroplasia (HCH)

Primary Outcome Measure*

• To develop sex- and age-specific growth curves for height (standing and sitting, as well as lower-to-upper body segment) for patients with HCH

Secondary Outcome Measure*

• To calculate sex- and age-specific Z-scores (referenced to Centers for Disease Control and Prevention [CDC] average stature) for height (standing and sitting) in patients with HCH

* Additional measures/criteria may apply.¹²

Pre-IND

Phase 1

Phase 2

Phase 3

Phase 4

Hypochondroplasia

(111-902 [Canopy HCH-OS])

Trial Status Recruiting

Condition

Hypochondroplasia

Technology

• Observational

Title

A Multicenter Multinational Observational Study of Children With Hypochondroplasia

Study Description

This study will collect growth measurements and other variables of interest to assess growth and the clinical course of hypochondroplasia over time.

Primary Outcome Measures*

• Change in growth (annualized growth velocity, height Z-score, height)
• Change in anthropometric measures (body mass index [BMI], upper and lower length ratio, arms spam to standing height ratio)

Select Inclusion Criteria*

• Patients ≤ 15 years of age
• Genetic confirmation of hypochondroplasia

Secondary Outcome Measures*

• Change in quality of life and patient and caregiver assessments of disease severity
• Rates of medical events of interest

Select Exclusion Criteria*

• Diagnosis of genetic short stature condition other than hypochondroplasia or a genetic variant known to cause another genetic syndrome associated with short stature
• Received an investigational product or medical device within the last 6 months

* Additional measures/criteria may apply.

For reference and comprehensive trial information, visit ClinicalTrials.gov NCT06212947

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