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 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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
60
The vosoritide dose administered will be based on the participant's weight and will follow the weight-band dosing regimen approved for ACH
Subcutaneous injection of recommended dose of placebo
Phoenix Children's Hospital - Thomas Campus (Main)
Phoenix, Arizona, United States
Incidence of treatment-emergent adverse events
Time frame: From baseline to end of treatment at 52 weeks
Incidence of serious adverse events versus placebo over the course of the study
Time frame: From baseline to end of treatment at 52 weeks
Changes from baseline in standard clinical laboratory values (hematology, urinalysis, and chemistry)
Time frame: At week 26, at week 52
Changes from baseline in heart rate
Units of measure: bpm
Time frame: At week 13, at week 26, at week 39, at week 52
Change from baseline in height Z-score
Time frame: At week 52
Changes from baseline in respiratory rate
Units of measure: breaths/min
Time frame: At week 13, at week 26, at week 39, at week 52
Changes from baseline in temperature
Units of measure: celsius
Time frame: At week 13, at week 26, at week 39, at week 52
Changes from baseline in blood pressure
Units of measure: mmHg
Time frame: At week 13, at week 26, at week 39, at week 52
Change in height
Time frame: At week 52
Cumulative annualized growth velocity (AGV)
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Cedars-Sinai Medical Center
Los Angeles, California, United States
NOT_YET_RECRUITINGBenioff Children's Hospital - Oakland
Oakland, California, United States
NOT_YET_RECRUITINGChildren's National Medical Center
Washington D.C., District of Columbia, United States
RECRUITINGAnn & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
NOT_YET_RECRUITINGThe Johns Hopkins University School of Medicine
Baltimore, Maryland, United States
NOT_YET_RECRUITINGUniversity of Minneasota Masonic Children's Hospital
Minneapolis, Minnesota, United States
NOT_YET_RECRUITINGUniversity of Missouri
Columbia, Missouri, United States
NOT_YET_RECRUITINGChildren's Wisconsin - Fox Valley Hospital
Neenah, Wisconsin, United States
NOT_YET_RECRUITINGRoyal Children's Hospital Melbourne
Parkville, Victoria, Australia
NOT_YET_RECRUITING...and 9 more locations
Time frame: At week 52
6-month interval AGV
Time frame: At week 26, at week 52
Change from baseline in upper to lower body segment ratio
Time frame: At week 52
Change from baseline in arm span
Time frame: At week 52
Change from baseline in total body (less head) bone mineral density (BMD) Z-score
Time frame: At week 52
Change from baseline in lumbar spine BMD Z-score
Time frame: At week 52
Change from baseline in total body (less head) bone mineral content (BMC) as measured by DXA
Time frame: At week 52
Change from baseline in lumbar spine BMC as measured by DXA
Time frame: At week 52
Area under the plasma vosoritide concentration time-curve from time 0 to infinity (AUC0-∞)
Time frame: At week 26, at week 52
Area under the plasma vosoritide concentration time-curve from time 0 to the last measurable concentration (AUC0-t)
Time frame: At week 26, at week 52
Elimination half-life of vosoritide (t½)
Time frame: At week 26, at week 52
Apparent clearance of vosoritide
Time frame: At week 26, at week 52
Apparent volume of distribution of vosoritide based upon the terminal phase (Vz/F)
Time frame: At week 26, at week 52
Time vosoritide is present at maximum concentration (Tmax)
Time frame: At week 26, at week 52
Maximum concentration (Cmax) of vosoritide in plasma
Time frame: At week 26, at week 52
Change from pre-dose at pre-specified timepoints versus placebo in cyclic guanine monophosphate (cGMP)
Time frame: At week 26 and week 52
Incidence of otitis media
Time frame: From baseline to end of treatment at 52 weeks
Seizure frequency over the course of the study
Time frame: From baseline to end of treatment at 52 weeks