Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia
Фаза 3 – широко изпитване преди одобрение
Заболявания:
Hypochondroplasia
Спонсор: BioMarin Pharmaceutical
Налично на:
БГ
Обобщение
The purpose of this study is to evaluate the long-term safety and efficacy of daily doses of vosoritide in participants with HCH
Кой може да участва
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
Места на провеждане
9
Австралия (1)
Murdoch Children's Research Institute
Parkville , Victoria
Канада (1)
University of Alberta Stollery Children's Hospital
Edmonton , Alberta
Франция (1)
Hospices Civils de Lyon - Hopital Femme Mere Enfant
Lyon
Германия (1)
Uniklinik Koln
Cologne
Италия (1)
Instituto Giannina Gaslini
Genoa
Япония (1)
Osaka University Hosptial
Osaka
Испания (1)
Vithas Hospital San Jose
Vitoria-Gasteiz
Великобритания (1)
Great Ormond Street Hospital for Children NHS Foundation Trust
London
САЩ (1)
Nemours Children's Hospital, Delaware (Alfred I. Dupont Hospital for Children)