Current partner codePEPTIDESDE
NCT07126262·Phase 2·INTERVENTIONAL

A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months

Status

Recruiting

Phase

Phase 2

Enrollment

60

Locations

26

Results

Not posted

Publications

0

Study summary

What the protocol is testing.

The purpose of this study is to evaluate the safety and efficacy of daily administration of vosoritide in participants with HCH aged 0 to \< 36 months over a 52-week period.

Full detailed description

Study 111-212 is a Phase 2, randomized, double-blind, placebo-controlled, multicenter study to assess the safety and efficacy of vosoritide versus placebo in infants and young children with HCH. Eligible participants with documented HCH confirmed by genetic testing will be randomized in a 1:1 ratio to receive vosoritide or placebo. Participants will receive study treatment daily for 52 weeks by subcutaneous (SC) injection, followed by a 2-week safety follow-up visit. Vosoritide dosing will follow a weight-band regimen.

Interventions

Treatment arms and agents.

DRUG

Vosoritide

The vosoritide dose administered will be based on the participant's weight and will follow the weight-band dosing regimen approved for ACH

DRUG

Placebo

Subcutaneous injection of recommended dose of placebo

Timeline

From registration to results.

  1. First posted

    Aug 17, 2025

  2. Study start

    Jul 30, 2025

  3. Primary completion

    Jun 30, 2028

  4. Study completion

    Jun 30, 2028

  5. Results posted

    Not reported

  6. Registry updated

    Mar 31, 2026

Outcomes

What the study measures.

Primary outcomes

Incidence of treatment-emergent adverse events

Time frame · From baseline to end of treatment at 52 weeks

Incidence of serious adverse events versus placebo over the course of the study

Time frame · From baseline to end of treatment at 52 weeks

Changes from baseline in standard clinical laboratory values (hematology, urinalysis, and chemistry)

Time frame · At week 26, at week 52

Changes from baseline in heart rate

Time frame · At week 13, at week 26, at week 39, at week 52

Units of measure: bpm

Change from baseline in height Z-score

Time frame · At week 52

Changes from baseline in respiratory rate

Time frame · At week 13, at week 26, at week 39, at week 52

Units of measure: breaths/min

Changes from baseline in temperature

Time frame · At week 13, at week 26, at week 39, at week 52

Units of measure: celsius

Changes from baseline in blood pressure

Time frame · At week 13, at week 26, at week 39, at week 52

Units of measure: mmHg

Secondary outcomes

Change in height

Time frame · At week 52

Cumulative annualized growth velocity (AGV)

Time frame · At week 52

6-month interval AGV

Time frame · At week 26, at week 52

Change from baseline in upper to lower body segment ratio

Time frame · At week 52

Change from baseline in arm span

Time frame · At week 52

Change from baseline in total body (less head) bone mineral density (BMD) Z-score

Time frame · At week 52

Change from baseline in lumbar spine BMD Z-score

Time frame · At week 52

Change from baseline in total body (less head) bone mineral content (BMC) as measured by DXA

Time frame · At week 52

Change from baseline in lumbar spine BMC as measured by DXA

Time frame · At week 52

Area under the plasma vosoritide concentration time-curve from time 0 to infinity (AUC0-∞)

Time frame · At week 26, at week 52

Eligibility

Who can take part.

Minimum age
0 Months
Maximum age
36 Months
Sex
ALL
Healthy volunteers
No

Key Inclusion Criteria: 1. Participants must be 0 to \< 36 months of age at randomization. 2. Participants must have a confirmed genetic diagnosis of HCH (obtained via whole genome sequencing; presence of a FGFR3 pathogenic variant associated with HCH). 3. Participants aged 0 to \< 12 months must have a height Z-score of ≤ -1.0 SDS andparticipants aged ≥ 12 to \< 36 months must have a height Z-score of ≤ -2.0 SDS in reference to the average stature of the same sex and age, as calculated using the Center for Disease Control and Prevention (CDC) growth charts. 4. Participant's weight at the Day 1 visit (pre-treatment) must be ≥ 3 kg. Key Exclusion Criteria: 1. Short stature condition other than HCH (eg, ACH, trisomy 21, pseudoachondroplasia). 2. Have an unstable medical condition likely to require surgical intervention during the study period. 3. Taking any of the prohibited medications. 4. Have been treated with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroids in the 6 months prior to Screening, or long-term treatment (\> 3 months) at any time. 5. Require any investigational agent prior to completion of study period. 6. Have received another investigational product or investigational medical device within 30 days prior to the Screening visit. 7. Have used any other investigational product or investigational medical device for the treatment of HCH or short stature at any time. 8. Have current malignancy, history of malignancy, or currently under work-up for suspected malignancy. 9. Have known hypersensitivity to vosoritide or its excipients. 10. Have a condition or circumstance that, in the view of the investigator, places the participant at high risk for poor treatment compliance or for not completing the study. 11. Have any concurrent disease or condition that, in the view of the investigator, will interfere with study participation or safety evaluations, for any reason.

Study locations

26 registered sites.

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

Phoenix Children's Hospital - Thomas Campus (Main)

Phoenix, Arizona, United States

Cedars-Sinai Medical Center

Los Angeles, California, United States

Benioff Children's Hospital - Oakland

Oakland, California, 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

The Johns Hopkins University School of Medicine

Baltimore, Maryland, United States

University of Minneasota Masonic Children's Hospital

Minneapolis, Minnesota, United States

University of Missouri

Columbia, Missouri, United States

Cook Children's Endocrinology

Fort Worth, Texas, United States

Children's Wisconsin - Fox Valley Hospital

Neenah, Wisconsin, United States

Children's Health Queensland Hospital and Health Service

South Brisbane, Queensland, Australia

Royal Children's Hospital Melbourne

Parkville, Victoria, Australia

Hôpital Bicêtre

Le Kremlin-Bicêtre, Paris, France

Hospices Civils de Lyon - Hôpital Femme Mère Enfant

Bron, Rhone-Alps, France

Uniklinik Köln

Cologne, North Rhine-Westphalia, Germany

Universitätsklinikum des Saarlandes

Homburg, Saarland, Germany

Universitätskinderklinik Magdeburg

Magdeburg, Saxony-Anhalt, Germany

Fondazione Policlinico Universitario Agostino Gemelli IRCCS

Roma, Rome, Italy

IRCCS Istituto Giannina Gaslini

Genova, Italy

Kumamoto University Hospital

Kumamoto, Japan

Osaka Women's and Children's Hospital

Osaka, Japan

Institute of Science Tokyo Hospital

Tokyo, Japan

Nihon University Itabashi Hospital

Tokyo, Japan

Tottori University Hospital

Tottori, Japan

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.

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