Clinical trial NCT06455059: Interventional Study of Vosoritide for the Treatment of Children with Hypochondroplasia
Official title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Vosoritide in Children with Hypochondroplasia
Study type: INTERVENTIONAL
Overall status: ENROLLING_BY_INVITATION
Phase: PHASE3
Conditions: Hypochondroplasia
Keywords: Hypochondroplasia
Dwarfism
Bone Diseases
Bone Disease, Developmental
HCH
Natriuretic Peptide, C-Type
Musculoskeletal Diseases
Natriuretic Agents
Physiological Effects of Drugs
Skeletal Dysplasias
Genetic Diseases, Inborn
Osteochondrodysplasias
Vosoritide
Voxzogo
Lead sponsor: BioMarin Pharmaceutical
Sponsor class: INDUSTRY
First posted: 2024-06-12
Enrollment: 80
Sex: ALL
Minimum age (years): 3
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The intent and design of this Phase 3 study is to assess vosoritide as a therapeutic option for the treatment of children with hypochondroplasia (HCH).

Detailed description: This is a Phase 3 randomized, stratified, placebo-controlled, double-blind multicenter study to evaluate the effect of 52 weeks of daily vosoritide administration on annualized growth velocity (AGV) in participants with HCH. Eligible participants with documented HCH confirmed by genetic testing will roll over from Study 111-902 and enter the 111-303 study. Participants will be randomly assigned to one of two treatment groups: Placebo or Vosoritide. The route of administration is subcutaneous injection, and the frequency of administration is daily.

Eligibility criteria: Inclusion Criteria:

1. Participants must be ≥ 3 to \< 18 years of age at enrollment
2. A confirmed genetic diagnosis of HCH
3. A height Z score of ≤ - 2.0 standard deviations (SDs) in reference to the general population of the same age and sex, as calculated using the Center for Disease Control and Prevention (CDC) growth charts
4. Males and females are eligible to participate in this clinical study.
5. Females ≥ 10 years old or who have begun menses must have a negative pregnancy test at the Screening Visit and be willing to have additional pregnancy tests during the study.
6. If sexually active, participants must be willing to use a highly effective method of contraception while participating in the study.

Exclusion Criteria:

1. Short stature condition other than HCH
2. Have an unstable condition likely to require surgical intervention during the study.
3. Evidence of decreased growth velocity and/or growth plate closure
4. Taking any of the prohibited medications
5. Treated with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroids
6. Planned or expected to have limb-lengthening surgery during the study period.
7. Planned or expected bone-related surgery (ie, surgery involving disruption of bone cortex, excluding tooth extraction), during the study period
8. Require any investigational agent prior to completion of study period.
9. Received vosoritide or another investigational product or investigational medical device in the past
10. Have used any investigational product or investigational medical device for the treatment of HCH or short stature at any time, including vosoritide
11. Have current malignancy, history of malignancy, or currently under work-up for suspected malignancy.
12. Have known hypersensitivity to vosoritide or its excipients.
13. Concurrent disease or condition that, in the view of the investigator, would interfere with study participation or safety evaluations, for any reason.

Primary outcomes:
measure: Change from baseline in annualized growth velocity (AGV) at Week 52 versus placebo
timeFrame: At week 52
Secondary outcomes:
measure: Change from baseline in standing height at Week 52 versus placebo
timeFrame: At week 52
measure: Change from baseline in height Z-score at Week 52 versus placebo
timeFrame: At week 52
Other outcomes:
Locations:
city: Wilmington
country: United States
facility: Nemours Alfred I. DuPont Hospital for Children
state: Delaware
city: Washington
country: United States
facility: Children's National Medical Center
state: District of Columbia
city: Chicago
country: United States
facility: Ann & Robert H. Lurie Children's Hospital of Chicago
state: Illinois
city: Milwaukee
country: United States
facility: Medical College of Wisconsin
state: Wisconsin
city: Parkville
country: Australia
facility: Murdoch Children's Research Institute
state: Victoria
city: Edmonton
country: Canada
facility: University of Alberta Stollery Children's Hospital
state: Alberta
city: Toronto
country: Canada
facility: SickKids - The Hospital for Sick Children
state: Ontario
city: Montreal
country: Canada
facility: Centre Hospitalier Universitaire Sainte-Justine
state: Quebec
city: Lyon
country: France
facility: Hospices Civils de Lyon - Hopital Femme Mere Enfant
city: Marseille
country: France
facility: Hopital de la Timone
city: Paris
country: France
facility: Hopital Necker-Enfants Malade
city: Toulouse
country: France
facility: CHU de Toulouse
city: Koln
country: Germany
facility: Uniklinik Koln
city: Magdeburg
country: Germany
facility: Univeristatskinderklinik Magdeburg
city: Genoa
country: Italy
facility: Instituto Giannina Gaslini
city: Rome
country: Italy
facility: Fondazione Policlinico Universitario Agostino Gemelli IRCCS
city: Rome
country: Italy
facility: Ospedale Pediatrico Bambino Gesu
city: Osaka
country: Japan
facility: Osaka University Hosptial
city: Tokushima
country: Japan
facility: Tokushima University Hospital
city: Tokyo
country: Japan
facility: Institute of Science Tokyo Hospital
city: Tottori
country: Japan
facility: Tottori University Hospital
city: Vitoria-Gasteiz
country: Spain
facility: Vithas Hospital San Jose
city: London
country: United Kingdom
facility: Great Ormond Street Hospital for Children NHS Foundation Trust
Study officials:
affiliation: BioMarin Pharmaceutical
name: Medical Director, MD
role: STUDY_DIRECTOR
References: