Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia (CANOPY-HCH-EXT)

BioMarin Pharmaceutical

Status and phase

Invitation-only

Phase 3

Conditions

Hypochondroplasia

Treatments

Drug: Vosoritide

Study type

Interventional

Funder types

Industry

Identifiers

111-308

Details and patient eligibility

About

The purpose of this study is to evaluate the long-term safety and efficacy of daily doses of vosoritide in participants with HCH

Enrollment

140 estimated patients

Sex

All

Volunteers

No Healthy Volunteers

Inclusion criteria

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
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
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
If sexually active, participants must be willing to use a highly effective method of contraception while participating in the study
Participants are willing and able to perform all study procedures as physically possible
Parent(s) or caregiver(s) are willing to administer daily injections to the participants and willing to complete the required training

Exclusion criteria

Permanently discontinued study treatment in the studies 111-303 or 111-212
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
Taking or planning to take any prohibited medications
Planned or expected to have limb-lengthening surgery during the study period
Planned or expected bone-related surgery during the study period
Require any investigational agent prior to completion of study period
Have current malignancy, history of malignancy, or currently under work-up for suspected malignancy
Have known hypersensitivity to vosoritide or its excipients
Is pregnant or breastfeeding at Baseline or planning to become pregnant (self or partner) at any time during the study
Concurrent disease or condition that, in the view of the investigator, would interfere with study participation or safety evaluations, for any reason