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

ConditionIdiopathic Short Stature
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age3-11
SponsorBioMarin Pharmaceutical

About this trial

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).

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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

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).

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.

Previous treatment with a growth promoting agent

Disqualifiers

None

Trial design

Design model

Parallel

Treatments tested in this trial

  • Vosoritide Injection

    Drug

    Experimental Drug Lyophilized powder for reconstitution

  • Human Growth Hormone

    Drug

    Commercial product containing somatotropin

  • Placebo

    Drug

    Lyophilized powder for reconstitution

Treatment groups

100 Participants
are divided into 5 treatment groups
Group A: Vosoritide Dose 1 - Low DoseExperimental treatment 1 intervention
Group B: Vosoritide Dose 2 - Medium DoseExperimental treatment 1 intervention
Group C: Vosoritide Dose 3 - High DoseExperimental treatment 1 intervention
Group D: PlaceboPlacebo comparator 1 intervention
Group E: Human Growth HormoneActive comparator 1 intervention

Trial outcomes

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

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

BioMarin Pharmaceutical

Lead sponsor

Source ClinicalTrials.gov
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