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
Status
Recruiting
Phase
Phase 2
Enrollment
60
Locations
26
Results
Not posted
Publications
0
Study summary
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.
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
DRUG
The vosoritide dose administered will be based on the participant's weight and will follow the weight-band dosing regimen approved for ACH
DRUG
Subcutaneous injection of recommended dose of placebo
Timeline
First posted
Aug 17, 2025
Study start
Jul 30, 2025
Primary completion
Jun 30, 2028
Study completion
Jun 30, 2028
Results posted
Not reported
Registry updated
Mar 31, 2026
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
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
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
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
No PMID-linked publications were present in this registry snapshot.
Related trials
BioMarin · Achondroplasia
Phase 2 / Phase 3
Recruiting
160
2026-07
BioMarin · Idiopathic Short Stature
Phase 2
Recruiting
100
2026-05
BioMarin · Noonan Syndrome
Phase 2
Recruiting
30
2026-05
Andrew Dauber · Short Stature
Phase 2
Active, not recruiting
56
2026-03
BioMarin · Achondroplasia
Phase 2
Active, not recruiting
30
2026-03
BioMarin · Achondroplasia
Phase 3
Active, not recruiting
119
2026-03
BioMarin · Achondroplasia
Phase 2
Active, not recruiting
73
2026-03
BioMarin · Achondroplasia
Phase 2
Active, not recruiting
20
2026-03
Related PeptideStat pages
Research pages describe evidence. Vendor pages, where available, describe independently tracked research-product listings and are not clinical recommendations.