Hypochondroplasia
Conditions
Interventions
Drug: Vosoritide
Subcutaneous injection of recommended dose of vosoritide based on weight-band dosing once daily for 52 weeks.
Other Names:
Modified recombinant human C-type natriuretic peptide
Drug:
Sponsors
Aoki Takuji
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1 Participants must be 0 to = 12 to = 3 kg.
Exclusion criteria
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| - Incidence of TEAEs versus placebo over the course of the study - Incidence of SAEs versus placebo over the course of the study - Changes in standard clinical laboratory values (urinalysis, chemistry, hematology) versus placebo over the course of the study - Changes in vital signs versus placebo over the course of the study - Change from baseline at Week 52 versus placebo in height Z-score | — |
Secondary
| Measure | Time frame |
|---|---|
| - Change from baseline at Week 52 versus placebo in height - Cumulative AGV at Week 52 versus placebo - 6-month interval AGV at Week 26 and Week 52, respectively (non-comparative) | — |
Countries
Australia, France, Germany, Italy, Japan, UK, United States
Contacts
Public ContactjRCT inquiry reception center EPS Corporation
EPS Corporation
Outcome results
None listed