Hypochondroplasia
Conditions
Interventions
Drug: Vosoritide
Subcutaneous injection of recommended dose of vosoritide based on weight-band dosing once daily until final adult height (FAH).
Other Names:
Modified recombinant human C-type natriure
Sponsors
Koichi Sasaki
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Participants must have completed the Week 52 visit for 111-303 or 111-212 and have open epiphyses as assessed by left hand antero-posterior (AP) X-rays. 2. Parent(s) or guardian(s) must be willing and able to provide written, signed informed consent after the nature of the study has been explained and prior to performance of any study-related procedure. 3. Females >- 10 years old or who have begun menses must have a negative pregnancy test at the Baseline visit and be willing to have additional pregnancy tests during the study. 4. If sexually active, participants must be willing to use a highly effective method of contraception while participating in the study. 5. Participants are willing and able to perform all study procedures as physically possible. 6. Parent(s) or caregiver(s) are willing to administer daily injections to the participants and willing to complete the required training.
Exclusion criteria
Exclusion criteria: 1. Permanently discontinued study treatment in the studies 111-303 or 111-212. 2. Evidence of decreased growth velocity (AGV < 1.5 cm/year) as assessed over a period of at least 6 months and growth plate closure assessed as per standard of care. Prior/Concomitant Therapies: 3. Taking or planning to take any of the prohibited medications listed in Section 6.9. 4. Planned or expected to have limb-lengthening surgery during the study period. 5. Planned or expected bone-related surgery (ie, surgery involving disruption of bone cortex, excluding tooth extraction), during the study period. 6. Require any investigational agent prior to completion of study period. Diagnostic Assessments 7. Have current malignancy, history of malignancy, or currently under work-up for suspected malignancy. 8. Have known hypersensitivity to vosoritide or its excipients. 9. Is pregnant or breastfeeding at Baseline or planning to become pregnant (self or partner) at any time during the study. 10. Concurrent disease or condition that, in the view of the investigator, would interfere with study participation or safety evaluations, for any reason.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| - Incidence of treatment emergent adverse events over time until FAH - Change from baseline in height Z-score yearly until FAH - Change from baseline in height yearly until FAH | — |
Secondary
| Measure | Time frame |
|---|---|
| - Change from baseline in height at Week 52 of Study 111-308 - Change from baseline in height Z-score at Week 52 of Study 111-308 Baseline is based on first dose of study drug (vosoritide or placebo) in the parent study (111-303 or 111-212). | — |
Countries
Australia, Canada, France, Germany, Italy, Japan, Spain, UK, United States
Contacts
Public ContactEPS Corporation jRCT inquiry reception center
EPS Corporation
Outcome results
None listed