Current partner codePEPTIDESDE

Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia

Status

Recruiting

Phase

Phase 2 / Phase 3

Enrollment

160

Locations

19

Results

Not posted

Publications

0

Study summary

What the protocol is testing.

This is a multicenter, multinational, randomized, active-controlled, operationally seamless Phase 2/3 study of BMN 333 in treatment-naïve pediatric participants with achondroplasia (ACH). The study consists of a Phase 2 part and a Phase 3 part.

Full detailed description

The main purpose of this study is to evaluate the effects of BMN 333 on growth compared with vosoritide in participants with achondroplasia who have not received any growth-promoting treatments. The study includes 2 parts: the Phase 2 part will select the optimal BMN 333 dose to be used in Phase 3 and determine study continuation into Phase 3; the Phase 3 part will compare the effects of the selected dose of BMN 333 with vosoritide. Study details for either Phase 2 or Phase 3 include the following: * Study duration: up to 61 weeks (from screening to Safety Follow-up visit) * Treatment duration: 52 weeks. Treatment frequency: BMN 333, once weekly; vosoritide, once daily

Interventions

Treatment arms and agents.

DRUG

BMN 333

Administration: Weekly subcutaneous injection

DRUG

Vosoritide Injection [Voxzogo]

Administration: Daily subcutaneous injection

Timeline

From registration to results.

  1. First posted

    Mar 2, 2026

  2. Study start

    Apr 20, 2026

  3. Primary completion

    Jun 2029

  4. Study completion

    Sep 2029

  5. Results posted

    Not reported

  6. Registry updated

    Jul 24, 2026

Outcomes

What the study measures.

Primary outcomes

Phase 2: Predicted Annualized Growth Velocity (AGV) at Week 52 (based on AGV at Weeks 26, 39, and 52 [available cumulative data]

Time frame · 52 weeks

Phase 3: Annualized Growth Velocity (AGV) at Week 52

Time frame · 52 weeks

Secondary outcomes

Phase 2: AGV at Weeks 26 and 52

Time frame · 26 and 52 weeks

Phase 2: Change from Baseline in standing height

Time frame · 26 and 52 weeks

Measured in centimeters

Phase 2: Change from Baseline in height Z-score

Time frame · 26 and 52 weeks

Phase 2: Change from Baseline in upper to lower body segment ratio

Time frame · 26 and 52 weeks

Phase 2: Incidence of adverse events (AEs)

Time frame · 52 weeks

Phase 2: Incidence of serious adverse events (SAEs)

Time frame · 52 weeks

Phase 2: Incidence of events of interest (EOIs)

Time frame · 52 weeks

Phase 2: Maximum concentration (Cmax) of BMN 333 in plasma

Time frame · 52 weeks

Phase 2: Maximum concentration (Cmax) of released vosoritide in plasma

Time frame · 52 weeks

Phase 2: Time to reach maximum concentration (Tmax) for BMN 333

Time frame · 52 weeks

Eligibility

Who can take part.

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

Inclusion Criteria: 1. Participants must be aged ≥ 2 to \< 11 years (Phase 2) or ≥ 2 to \< 18 years (Phase 3), at the time of signing the informed consent 2. Participants must have ACH (confirmed by documented genetic testing) and open epiphyses 3. Are Tanner Stage I (Phase 2) or any Tanner stage (Phase 3) 4. Are ambulatory and able to stand without assistance Exclusion Criteria: 1. Have any short stature condition other than ACH (eg, hypochondroplasia, trisomy 21, pseudoachondroplasia, GH deficiency) 2. Have any of the following disorders: Hypothyroidism or hyperthyroidism, unless treated with evidence of normalized thyroid-stimulating hormone (TSH) levels, diabetes mellitus, unless considered well-controlled, autoimmune inflammatory disease, inflammatory bowel disease, autonomic neuropathy, anemia defined as hemoglobin \< 10 g/dL, vitamin D deficiency, significant hip pathology. 3. Have history of any renal insufficiency or cardiac/ cardiovascular disease that places the participant at increased risk of an adverse cardiac outcome in the setting of hypotension. 4. Have had bone fractures of the long bones or spine within 6 months prior to screening. 5. Have used vosoritide, any other approved product (except GH, as detailed below), investigational product, or investigational medical device for the treatment of ACH or short stature at any time 6. Have been treated with GH, insulin-like growth factor 1, or anabolic steroids in the 6 months prior to treatment start

Study locations

19 registered sites.

Australia · Canada · Italy · Japan · Poland · Romania · South Korea · United Kingdom · United States. Showing up to 24 locations stored in the fast local snapshot.

UCSF Benioff Children's Hospital Oakland

Oakland, California, United States

Nemours Children's Health

Wilmington, Delaware, United States

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

Chicago, Illinois, United States

Johns Hopkins Medicine

Baltimore, Maryland, United States

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

University of Texas Southwestern Medical Center

Dallas, Texas, United States

Texas Children Hospital, Baylor College of Medicine

Houston, Texas, United States

Consano Clinical Research, LLC

San Antonio, Texas, United States

Murdoch Children's Research Institute

Parkville, Victoria, Australia

Universite de Montreal - Centre Hospitalier Universitaire Sainte-Justine

Montreal, Quebec, Canada

Irccs Ospedale Gaslini Di Genova

Genova, Italy

Osaka City General Hospital

Osaka, Japan

Uniwersytecki Szpital Kliniczny im. J. Mikulicza-Radeckiego we Wroclawiu Klinika Pediatrii i Chorob Infekcyjnych

Wroclaw, Poland

Institutul National de Endocrinologie C.I.Parhon

Bucharest, Romania

Craiova Emergency Clinical County

Craiova, Romania

Seoul National University Hospital

Seoul, South Korea

Pusan National University Yangsan Hospital

Yangsan, South Korea

University Hospitals Bristol NHS Foundation Trust - Bristol Royal Hospital for Children

Bristol, 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.