Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia

Официальное название

A Phase 3, Open-Label, Long-Term Extension Study to Evaluate the Safety and Efficacy of Vosoritide in Children With Hypochondroplasia

Обзор

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

Вмешательства

• Препарат Vosoritide
  Open-label administration of vosoritide using weight-band dosing

Первичные конечные точки

• Evaluate the long-term efficacy of vosoritide treatment until final adult height (FAH) [Срок оценки: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males]

Вторичные конечные точки (11)

• Evaluate the maintenance effect of vosoritide treatment on standing height [Срок оценки: Baseline to week 52]
• Evaluate the maintenance effect of vosoritide treatment on standing height Z-score [Срок оценки: Baseline to week 52]
• Evaluate long-term effect of vosoritide treatment on growth velocity [Срок оценки: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males]
• Evaluate long-term effect of vosoritide treatment on body proportionality [Срок оценки: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males]
• Evaluate long-term effect of vosoritide treatment on bone age versus chronological age [Срок оценки: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males]
• Evaluate long-term effect of vosoritide treatment on bone metabolism biomarkers [Срок оценки: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males]
• Evaluate long-term changes in Health-Related Quality of Life (HRQoL) [Срок оценки: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males]
• Evaluate long-term effect of vosoritide treatment on bone quality [Срок оценки: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males]
• Evaluate long-term effect of vosoritide treatment on pubertal growth as measured by Tanner Staging [Срок оценки: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males]
• To monitor the incidence of otitis media [Срок оценки: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males]
• To monitor the frequency of seizures [Срок оценки: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males]

Критерии участия

Критерии включения

• 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

Критерии исключения

• 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

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

США · 4 центра

• Nemours Children's Hospital, Delaware (Alfred I. Dupont Hospital for Children) — Wilmington
• Children's National Medical Center — Washington D.C.
• Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago
• Medical College of Wisconsin — Milwaukee

Франция · 4 центра

• Hospices Civils de Lyon - Hopital Femme Mere Enfant — Lyon
• Hopital de la Timone — Marseille
• Hopital Necker-Enfants Malade — Paris
• CHU de Toulouse — Toulouse

Япония · 4 центра

• Osaka University Hosptial — Osaka
• Tokushima University Hospital — Tokushima
• Institute of Science Tokyo Hospital — Tokyo
• Tottori University Hospital — Tottori

Канада · 3 центра

• University of Alberta Stollery Children's Hospital — Edmonton
• SickKids - The Hospital for Sick Children — Toronto
• Centre Hospitalier Universitaire Sainte-Justine — Montreal

Италия · 3 центра

• Instituto Giannina Gaslini — Genoa
• Fondazione Policlinico Universitario Agostino Gemelli IRCCS — Rome
• Ospedale Pediatrico Bambino Gesu — Rome

Германия · 2 центра

• Uniklinik Koln — Cologne
• Univeristatskinderklinik Magdeburg — Magdeburg

Австралия · 1 центр

• Murdoch Children's Research Institute — Parkville

Испания · 1 центр

• Vithas Hospital San Jose — Vitoria-Gasteiz

Великобритания · 1 центр

• Great Ormond Street Hospital for Children NHS Foundation Trust — London

Идентификаторы

NCT: NCT07073014 · 111-308

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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