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

PHASE2RECRUITING

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

Study details:

Study 111-212 is a Phase 2, randomized, double-blind, placebo-controlled, multicenter study to assess the safety and efficacy of vosoritide versus placebo in infants and young children with HCH. Eligible participants with documented HCH confirmed by genetic testing will be randomized in a 1:1 ratio to receive vosoritide or placebo. Participants will receive study treatment daily for 52 weeks by subcutaneous (SC) injection, followed by a 2-week safety follow-up visit.

Vosoritide dosing will follow a weight-band regimen.

Eligibility criteria

Researchers look for people who fit a certain description, called eligibility criteria. See if you qualify.

Inclusion criteria

  1. Participants must be 0 to < 36 months of age at randomization.
  2. Participants must have a confirmed genetic diagnosis of HCH (obtained via whole genome sequencing; presence of a FGFR3 pathogenic variant associated with HCH).
  3. Participants aged 0 to < 12 months must have a height Z-score of ≤ -1.0 SDS and participants aged ≥ 12 to < 36 months must have a height Z-score of ≤ -2.0 SDS in reference to the average stature of the same sex and age, as calculated using the Center for Disease Control and Prevention (CDC) growth charts.
  4. Participant's weight at the Day 1 visit (pre-treatment) must be ≥ 3 kg.

Exclusion criteria

  1. Short stature condition other than HCH (eg, ACH, trisomy 21, pseudoachondroplasia).
  2. Have an unstable medical condition likely to require surgical intervention during the study period.
  3. Taking any of the prohibited medications.
  4. 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.
  5. Require any investigational agent prior to completion of study period.
  6. Have received another investigational product or investigational medical device within 30 days prior to the Screening visit.
  7. Have used any other investigational product or investigational medical device for the treatment of HCH or short stature at any time.
  8. Have current malignancy, history of malignancy, or currently under work-up for suspected malignancy.
  9. Have known hypersensitivity to vosoritide or its excipients.
  10. Have a condition or circumstance that, in the view of the investigator, places the participant at high risk for poor treatment compliance or for not completing the study.
  11. Have any concurrent disease or condition that, in the view of the investigator, will interfere with study participation or safety evaluations, for any reason.

Eligibility

Age eligible for study : NA

Healthy volunteers accepted : No

Gender eligible for study: ALL

Things to know

Study dates

Study start:

Primary completion:

Study completion finish:

study type

Study type

TREATMENT

phase

Phase

    PHASE2

trial

Study ID

NCT07126262

Intervention or treatment

DRUG: Vosoritide

DRUG: Placebo

Conditions

  • Hypochondroplasia

  • Condition: Hypochondroplasia

  • DRUG: Vosoritide and other drugs

  • Parkville, Victoria, Australia

  • Sponsor: BioMarin Pharmaceutical

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Are you running this trial?

If you're a clinic or sponsor, you can claim this study

Research sites nearby

Closest Location:

Royal Children's Hospital Melbourne

Select from list below to view details:

  • Royal Children's Hospital Melbourne

    Parkville, Victoria, Australia

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Who is running the clinical trial?

BP

BioMarin Pharmaceutical

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Participant Group/ArmIntervention/Treatment
EXPERIMENTAL: Vosoritide injection with vial and syringe
  • Subcutaneous injection of recommended dose of vosoritide based on weight-band dosing once daily.
Drug: Vosoritide
    PLACEBO_COMPARATOR: Placebo injection with vial and syringe
    • Subcutaneous injection of recommended dose of placebo
    Drug: Placebo

      What is the study measuring?

      Primary Outcome

      MeasureDescriptionTime Frame
      Incidence of treatment-emergent adverse eventsNot SpecifiedFrom baseline to end of treatment at 52 weeks
      Incidence of serious adverse events versus placebo over the course of the studyNot SpecifiedFrom baseline to end of treatment at 52 weeks
      Changes from baseline in standard clinical laboratory values (hematology, urinalysis, and chemistry)Not SpecifiedAt week 26, at week 52
      Changes from baseline in heart rateUnits of measure: bpmAt week 13, at week 26, at week 39, at week 52
      Change from baseline in height Z-scoreNot SpecifiedAt week 52
      Changes from baseline in respiratory rateUnits of measure: breaths/minAt week 13, at week 26, at week 39, at week 52
      Changes from baseline in temperatureUnits of measure: celsiusAt week 13, at week 26, at week 39, at week 52
      Changes from baseline in blood pressureUnits of measure: mmHgAt week 13, at week 26, at week 39, at week 52

      Secondary Outcome

      MeasureDescriptionTime Frame
      Change in heightNot SpecifiedAt week 52
      Cumulative annualized growth velocity (AGV)Not SpecifiedAt week 52
      6-month interval AGVNot SpecifiedAt week 26, at week 52
      Change from baseline in upper to lower body segment ratioNot SpecifiedAt week 52
      Change from baseline in arm spanNot SpecifiedAt week 52
      Change from baseline in total body (less head) bone mineral density (BMD) Z-scoreNot SpecifiedAt week 52
      Change from baseline in lumbar spine BMD Z-scoreNot SpecifiedAt week 52
      Change from baseline in total body (less head) bone mineral content (BMC) as measured by DXANot SpecifiedAt week 52
      Change from baseline in lumbar spine BMC as measured by DXANot SpecifiedAt week 52
      Area under the plasma vosoritide concentration time-curve from time 0 to infinity (AUC0-∞)Not SpecifiedAt week 26, at week 52
      Area under the plasma vosoritide concentration time-curve from time 0 to the last measurable concentration (AUC0-t)Not SpecifiedAt week 26, at week 52
      Elimination half-life of vosoritide (t½)Not SpecifiedAt week 26, at week 52
      Apparent clearance of vosoritideNot SpecifiedAt week 26, at week 52
      Apparent volume of distribution of vosoritide based upon the terminal phase (Vz/F)Not SpecifiedAt week 26, at week 52
      Time vosoritide is present at maximum concentration (Tmax)Not SpecifiedAt week 26, at week 52
      Maximum concentration (Cmax) of vosoritide in plasmaNot SpecifiedAt week 26, at week 52
      Change from pre-dose at pre-specified timepoints versus placebo in cyclic guanine monophosphate (cGMP)Not SpecifiedAt week 26 and week 52
      Incidence of otitis mediaNot SpecifiedFrom baseline to end of treatment at 52 weeks
      Seizure frequency over the course of the studyNot SpecifiedFrom baseline to end of treatment at 52 weeks
      Are you running this trial?If you're a clinic or sponsor, you can claim this study.
      Claim this trial

      References

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

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