A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature

Phase 2

Not yet recruiting

• Conditions: Idiopathic Short Stature (ISS)

• Registration Number: 2024-520137-74-00
• Lead Sponsor: Biomarin Pharmaceutical Inc.

Brief Summary

Dose-Finding Phase: To evaluate the effect of 3 vosoritide doses versus placebo on AGV after 6 months of treatment

Long-Term Phase: To evaluate the effect of the therapeutic dose of vosoritide versus hGH on height and height Z-score after long-term treatment

Detailed Description

Following a minimum 6 month observational period in which baseline growth is assessed, participants in the vosoritide and placebo groups will complete a minimum of 6 months of randomized treatment (maximum of 6 months of placebo treatment), followed by open-label treatment with vosoritide until they reach near-final adult height, or at least 16 years of age for females or 18 years of age for males, whichever comes later. Participants randomized to the hGH group will receive open-label hGH for a minimum of 4 years.

Study Timeline

• Study First Posted: 2025-06-24
• Last Update Posted: 2025-06-24

Recruitment & Eligibility

• Status: Authorised, recruitment pending
• Sex: Not specified
• Target Recruitment: 20

Inclusion Criteria

1. Height assessment corresponding to a height Z-score of ≤ -2.25 SDs in reference to the general population of the same age and sex, as calculated using the Centers for Disease Control and Prevention (CDC) growth charts

2. Tanner Stage 1, at time of signing the ICF (unless too young to stage).

3. Participants must be ≥ 3 years old, and < 10 years old (female) or < 11 years old (males) at the time of signing the informed consent form (ICF).

Exclusion Criteria

1. Known chromosomal imbalance or genetic variant causing short stature syndrome, including but not limited to Laron syndrome, Prader-Willi syndrome, Russell-Silver Syndrome, Turner syndrome, disproportionate skeletal dysplasias, abnormal SHOX gene analysis, or Rasopathy (including Noonan syndrome)

2. Previous treatment with a growth promoting agent

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Dose-Finding Phase: Change from baseline in AGV at 6 months Long-Term Phase: Change from baseline in height and height Z-score at 4 years		Dose-Finding Phase: Change from baseline in AGV at 6 months Long-Term Phase: Change from baseline in height and height Z-score at 4 years

Secondary Outcome Measures

Name	Time	Method
1. Incidence of treatment-emergent adverse events over the course of the study		1. Incidence of treatment-emergent adverse events over the course of the study
2. Change from baseline in height Z-score at 6 months		2. Change from baseline in height Z-score at 6 months
3. Change from baseline in height at each visit and at FAH		3. Change from baseline in height at each visit and at FAH
4. Change from baseline in height Z-score at each visit and at FAH		4. Change from baseline in height Z-score at each visit and at FAH
5. PK parameters (eg, Tmax, Cmax, AUC0-t, AUC0-inf, t1/2, CL/F, Vz/F)		5. PK parameters (eg, Tmax, Cmax, AUC0-t, AUC0-inf, t1/2, CL/F, Vz/F)
6. Change from baseline at prespecified timepoints in: • urine cGMP • serum CXM		6. Change from baseline at prespecified timepoints in: • urine cGMP • serum CXM
7. Change from baseline in bone age/chronological age at prespecified timepoints		7. Change from baseline in bone age/chronological age at prespecified timepoints
8. Change from baseline at each visit in the following, as measured by DXA: • total body (less head) BMD Z-score • lumbar spine BMD Z-score • total body (less head) BMC • lumbar spine BMC		8. Change from baseline at each visit in the following, as measured by DXA: • total body (less head) BMD Z-score • lumbar spine BMD Z-score • total body (less head) BMC • lumbar spine BMC

Trial Locations

Locations (5)

Centre Hospitalier Universitaire De Montpellier; 🇫🇷 Montpellier, France

Centre Hospitalier Regional De Marseille; 🇫🇷 Marseille, France

Centre Hospitalier Universitaire D'Angers; 🇫🇷 Angers, France

Bicetre Hospital; 🇫🇷 Le Kremlin Bicetre Cedex, France

Hopital Des Enfants; 🇫🇷 Toulouse Cedex 9, France

Centre Hospitalier Universitaire De Montpellier

🇫🇷Montpellier, France

Cyril AMOUROUX

Site contact

+33467336506

cyril-amouroux@chu-montpellier.fr