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RecruitingNCT06382155Updated May 22, 2026

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

A Phase 2 interventional study of Vosoritide Injection and Human Growth Hormone in Idiopathic Short Stature, sponsored by BioMarin Pharmaceutical. Recruiting at 47 sites in 6 countries. Open to participants aged 3 Years to 11 Years. Per ClinicalTrials.gov, last updated 2026-05-22.

Sponsored by BioMarin Pharmaceutical · Phase 2, Interventional, and Treatment

From the registry’s dates

  • Primary completion was expected by Jun 2026, 5 months ago, but the record still lists the study as recruiting.
  • Started Oct 2024; still recruiting 2 years later.
Phase
Phase 2
Study type
Interventional
Enrollment
100
Allocation
Randomized
Ages
3 Years to 11 Years
Sex
All
01

Study summary

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).

Read the 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

02

Conditions studied

  • Idiopathic Short Stature
03

In context

Lead sponsor

BioMarin Pharmaceutical is the lead sponsor of 112 studies on the registry; 13 are open to participants now.

Of its 17 completed or terminated interventional studies of FDA-regulated products, 12 (71%) have results posted.

Browse BioMarin Pharmaceutical studies →

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
3 Years to 11 Years
Sexes eligible
All
Accepts healthy volunteers
No

Eligibility criteria

Key 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. If participant is ≥ 5 years at Screening,must be Tanner Stage I to be eligible for enrollment and randomization3. Historic stimulation test result with serum or plasma GH level greater than 10 μg/L or serum IGF-1 in the normal range for age (≥ -1.00 SDs and ≤+2.00 SDs).

Key Exclusions:

  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), ACAN deficiency.
  2. Previous treatment with a growth promoting agent
05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment
100 participants (estimated)

Study arms

  • Experimental
    Vosoritide Dose 1 - Low Dose

    Vosoritide Dose 1 daily injection

    Drug: Vosoritide Injection

  • Experimental
    Vosoritide Dose 2 - Medium Dose

    Vosoritide Dose 2 daily injection

    Drug: Vosoritide Injection

  • Experimental
    Vosoritide Dose 3 - High Dose

    Vosoritide Dose 3 daily injection

    Drug: Vosoritide Injection

  • Placebo comparator
    Placebo

    Placebo daily injection

    Drug: Placebo

  • Active comparator
    Human Growth Hormone

    hGH daily injection (US only)

    Drug: Human Growth Hormone

Interventions

06

What researchers measure

Primary outcomes

  1. Change from baseline in Annualized Growth Velocity (AGV)

    Time frame: At 6 months

  2. Change from baseline in height

    Time frame: At 4 years

  3. Change from baseline in height Z-score

    Time frame: At 4 years

Secondary outcomes

  1. Incidence of treatment-emergent adverse events

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

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

    Time frame: At 6 months

  3. Change from baseline in height

    Time frame: Every 6 months through the end of study, up to 15 years

  4. Change from baseline in height Z score

    Time frame: Every 6 months through the end of study, up to 15 years

  5. Change from baseline at prespecified timepoints in urine cyclic guanine monophosphate (cGMP)

    Time frame: Every 6 months through the end of study, up to 15 years

  6. Change from baseline at pre-specified timepoints in serum collagen X marker (CXM)

    Time frame: Every 6 months through the end of study, up to 15 years

  7. Change from baseline in bone age minus chronological age at pre-specified timepoints

    Time frame: Every 6 months through the end of study, up to 15 years

  8. Change from baseline in whole body (less head) bone mineral density (BMD) Z-score

    Time frame: Every 6 months through the end of study, up to 15 years

  9. Change from baseline in lumbar spine BMD Z-score

    Time frame: Every 6 months through the end of study, up to 15 years

  10. Change from baseline in total body (less head) bone mineral content (BMC)

    Time frame: Every 6 months through the end of study, up to 15 years

  11. Change from baseline in lumbar spine BMC

    Time frame: Every 6 months through the end of study, up to 15 years

  12. Maximum concentration (Cmax) of vosoritide in plasma

    Time frame: Every 6 months through the end of study, up to 15 years

  13. Area under the plasma vosoritide concentration time-curve from time 0 to infinity (AUC0-∞)

    Time frame: Every 6 months through the end of study, up to 15 years

  14. Area under the plasma vosoritide concentration time-curve from time 0 to the last measurable concentration (AUC0-t)

    Time frame: Every 6 months through the end of study, up to 15 years

  15. Elimination half-life of vosoritide (t½)

    Time frame: Every 6 months through the end of study, up to 15 years

  16. Apparent clearance of vosoritide

    Time frame: Every 6 months through the end of study, up to 15 years

  17. Apparent volume of distribution of vosoritide based upon the terminal phase (Vz/F)

    Time frame: Every 6 months through the end of study, up to 15 years

  18. Time vosoritide is present at maximum concentration (Tmax)

    Time frame: Every 6 months through the end of study, up to 15 years

07

Study locations

43 of 47 sites recruiting
08

References and documents

Individual participant data

Plan to share: Undecided

No publications or documents are linked to this record.

09

Timeline

Tracking since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reached

Started Oct 2024 (actual). Primary completion is estimated for Jun 2026, 20 months after the start.

How these figures are worked out, and which studies are left out of them.

Registered
Apr 19, 2024
First posted
Apr 24, 2024
StartActual
Oct 21, 2024
Primary completionEst.
Jun 2026
CompletionEst.
Dec 2036
Last update
May 22, 2026
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on May 22, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT06382155
Lead sponsor
BioMarin Pharmaceutical
Responsible party
Sponsor
First posted
Apr 24, 2024
Start date
Oct 21, 2024
Primary completion
Jun 2026 (estimated)
Completion
Dec 2036 (estimated)
Last update
May 22, 2026

Study contacts

Trial Specialist
Contact
medinfo@bmrn.com
+1 415.475.5852
Medical Director MD
study director · BioMarin Pharmaceutical

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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