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

Official title

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

Overview

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

Interventions

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

Primary outcome measures

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

Secondary outcome measures (11)

• Evaluate the maintenance effect of vosoritide treatment on standing height [Time frame: Baseline to week 52]
• Evaluate the maintenance effect of vosoritide treatment on standing height Z-score [Time frame: Baseline to week 52]
• Evaluate long-term effect of vosoritide treatment on growth velocity [Time frame: 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 [Time frame: 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 [Time frame: 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 [Time frame: 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) [Time frame: 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 [Time frame: 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 [Time frame: 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 [Time frame: 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 [Time frame: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males]

Eligibility criteria

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation

N/A

Model

Single group

Masking

Open label

Primary purpose

Treatment

Study locations

United States · 4 centers

• 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

France · 4 centers

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

Japan · 4 centers

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

Canada · 3 centers

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

Italy · 3 centers

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

Germany · 2 centers

• Uniklinik Koln — Cologne
• Univeristatskinderklinik Magdeburg — Magdeburg

Australia · 1 center

• Murdoch Children's Research Institute — Parkville

Spain · 1 center

• Vithas Hospital San Jose — Vitoria-Gasteiz

United Kingdom · 1 center

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

Identifiers

NCT: NCT07073014 · 111-308

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗