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NCT07441876PHASE2, PHASE3Recruiting

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

BioMarin Pharmaceutical

Start Date

4/20/2026

Completion Date

9/1/2029

Summary

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.

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

Eligibility Criteria

Age Range: 2 years to 17 years

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

Interventions

DRUG

BMN 333

DRUG

Vosoritide Injection [Voxzogo]

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Conditions

Achondroplasia

Locations

UCSF Benioff Children's Hospital Oakland

Oakland, California 94609

United States

Nemours Children's Health

Wilmington, Delaware 19803

United States

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

Chicago, Illinois 60611

United States

Johns Hopkins Medicine

Baltimore, Maryland 21205

United States

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio 45229

United States

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania 19104

United States

University of Texas Southwestern Medical Center

Dallas, Texas 75207

United States

Texas Children Hospital, Baylor College of Medicine

Houston, Texas 77030

United States

Consano Clinical Research, LLC

San Antonio, Texas 78231

United States

Murdoch Children's Research Institute

Parkville, Victoria 3052

Australia

Universite de Montreal - Centre Hospitalier Universitaire Sainte-Justine

Montreal, Quebec H3T 1C5

Canada

Irccs Ospedale Gaslini Di Genova

Genova, 16147

Italy

Osaka City General Hospital

Osaka, 534-0021

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, 200642

Romania

Seoul National University Hospital

Seoul,

South Korea

Pusan National University Yangsan Hospital

Yangsan, 50612

South Korea

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

Bristol,

United Kingdom