Current partner codePEPTIDESDE
NCT06455059·Phase 3·INTERVENTIONAL

Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia

Status

Active, not recruiting

Phase

Phase 3

Enrollment

80

Locations

23

Results

Not posted

Publications

0

Study summary

What the protocol is testing.

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).

Full 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.

Interventions

Treatment arms and agents.

DRUG

Vosoritide

Subcutaneous injection of recommended dose of vosoritide based on weight-band dosing once daily.

DRUG

Placebo

Subcutaneous injection of recommended dose of placebo

Timeline

From registration to results.

  1. First posted

    Jun 12, 2024

  2. Study start

    Jun 17, 2024

  3. Primary completion

    Aug 1, 2026

  4. Study completion

    Aug 1, 2026

  5. Results posted

    Not reported

  6. Registry updated

    Jan 20, 2026

Outcomes

What the study measures.

Primary outcomes

Change from baseline in annualized growth velocity (AGV) at Week 52 versus placebo

Time frame · At week 52

Secondary outcomes

Change from baseline in standing height at Week 52 versus placebo

Time frame · At week 52

Change from baseline in height Z-score at Week 52 versus placebo

Time frame · At week 52

Eligibility

Who can take part.

Minimum age
3 Years
Maximum age
17 Years
Sex
ALL
Healthy volunteers
No

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.

Study locations

23 registered sites.

Australia · Canada · France · Germany · Italy · Japan · Spain · United Kingdom · United States. Showing up to 24 locations stored in the fast local snapshot.

Nemours Alfred I. DuPont Hospital for Children

Wilmington, Delaware, United States

Children's National Medical Center

Washington D.C., District of Columbia, United States

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, United States

Medical College of Wisconsin

Milwaukee, Wisconsin, United States

Murdoch Children's Research Institute

Parkville, Victoria, Australia

University of Alberta Stollery Children's Hospital

Edmonton, Alberta, Canada

SickKids - The Hospital for Sick Children

Toronto, Ontario, Canada

Centre Hospitalier Universitaire Sainte-Justine

Montreal, Quebec, Canada

Hospices Civils de Lyon - Hopital Femme Mere Enfant

Lyon, France

Hopital de la Timone

Marseille, France

Hopital Necker-Enfants Malade

Paris, France

CHU de Toulouse

Toulouse, France

Uniklinik Koln

Cologne, Germany

Univeristatskinderklinik Magdeburg

Magdeburg, Germany

Instituto Giannina Gaslini

Genoa, Italy

Fondazione Policlinico Universitario Agostino Gemelli IRCCS

Rome, Italy

Ospedale Pediatrico Bambino Gesu

Rome, Italy

Osaka University Hosptial

Osaka, Japan

Tokushima University Hospital

Tokushima, Japan

Institute of Science Tokyo Hospital

Tokyo, Japan

Tottori University Hospital

Tottori, Japan

Vithas Hospital San Jose

Vitoria-Gasteiz, Spain

Great Ormond Street Hospital for Children NHS Foundation Trust

London, United Kingdom

Publications

Results and literature.

No PMID-linked publications were present in this registry snapshot.

Primary links

Continue at the source.

Related trials

More studies on Vosoritide.

Related PeptideStat pages

Put the record in context.

Research pages describe evidence. Vendor pages, where available, describe independently tracked research-product listings and are not clinical recommendations.