NCT06382155·Phase 2·INTERVENTIONAL

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

Vosoritide Other Recruiting

Status

Recruiting

Phase

Phase 2

Enrollment

100

Locations

Results

Not posted

Publications

Study summary

What the protocol is testing.

The purpose of this study is to evaluate i) the effect of multiple doses of vosoritide and ii) the effect of the therapeutic dose of vosoritide compared to human growth hormone (hGH)(hGH; only in the United States), in children with idiopathic short stature (ISS).

Full 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. A Treatment Completion visit will occur approximately 4 weeks after the last administration of investigational product. Participants who discontinue study treatment after ≥13 weeks of exposure may remain in the study and complete follow-up assessments per the protocol schedule (including annual and periodic assessments through End of Study). If a participant discontinues treatment and declines further participation, they will be asked to return for a final Treatment Completion and End of Study visit approximately 4 weeks after the last injection. Safety monitoring includes regular clinical and imaging assessments, including hip and lower-extremity evaluations, monitoring for hypotension, fractures, and slipped capital femoral epiphysis, with oversight by an independent Data Monitoring Committee An independent Data Monitoring Committee will periodically review safety data and may recommend treatment discontinuation based on predefined safety signals

Interventions

Treatment arms and agents.

DRUG

Vosoritide Injection

Experimental Drug Lyophilized powder for reconstitution

DRUG

Human Growth Hormone

Commercial product containing somatotropin

DRUG

Placebo

Lyophilized powder for reconstitution

Timeline

From registration to results.

First posted
Apr 24, 2024
Study start
Oct 21, 2024
Primary completion
Jun 2026
Study completion
Dec 2036
Results posted
Not reported
Registry updated
May 22, 2026

Outcomes

What the study measures.

Primary outcomes

Change from baseline in Annualized Growth Velocity (AGV)

Time frame · At 6 months

Change from baseline in height

Time frame · At 4 years

Change from baseline in height Z-score

Time frame · At 4 years

Secondary outcomes

Incidence of treatment-emergent adverse events

Time frame · Until the end of the study, up to 15 years

Change from baseline in Height Z-score (average stature reference)

Time frame · At 6 months

Change from baseline in height