Recruiting
Phase 2
Phase 3

BMN 333

Sponsor:

BioMarin Pharmaceutical

Code:

NCT07441876

Conditions

Achondroplasia

Eligibility Criteria

Sex: All

Age: 2 - 17

Healthy Volunteers: Not accepted

Interventions

BMN 333

Vosoritide Injection [Voxzogo]

Study Details

Brief 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.

Conditions

Achondroplasia

Study ID

NCT07441876

Start date

Apr 20, 2026

Status verified date

Aug, 2026

Completion date

Sep, 2029

Anticipated

Primary completion date

Jun, 2029

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 2 - 17

Healthy Volunteers: Not accepted

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 Design

Enrollment

160 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Phase 2: Low Dose

Participants will be randomized to receive different dose levels of BMN 333

experimental: Phase 2: Medium Dose

Participants will be randomized to receive different dose levels of BMN 333

experimental: Phase 2: High Dose

Participants will be randomized to receive different dose levels of BMN 333

active comparator: Phase 2: Vosoritide

weight band dosing, Modified recombinant human C-type natriuretic peptide Vosoritide

experimental: Phase 3: BMN 333 at selected dose after Phase 2

active comparator: Phase 3: Vosoritide

weight band dosing, Modified recombinant human C-type natriuretic peptide Vosoritide

Interventions

BMN 333

Administration: Weekly subcutaneous injection

Vosoritide Injection [Voxzogo]

Administration: Daily subcutaneous injection

Primary outcome measure

  • 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 ]

Central Contacts and Locations

Central contacts

Locations

UCSF Benioff Children's Hospital Oakland

Recruiting

Oakland, California, United States, 94609

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

Recruiting

Chicago, Illinois, United States, 60611

Johns Hopkins Medicine

Recruiting

Baltimore, Maryland, United States, 21205

Cincinnati Children's Hospital Medical Center

Recruiting

Cincinnati, Ohio, United States, 45229

Texas Children Hospital, Baylor College of Medicine

Recruiting

Houston, Texas, United States, 77030

Consano Clinical Research, LLC

Recruiting

San Antonio, Texas, United States, 78231

More Information

Sponsor

BioMarin Pharmaceutical

Last update posted

Aug 21, 2026

Last verified

Aug, 2026

Keywords

  • Achondroplasia
  • ACH
  • Bone Diseases, Developmental Dwarfism
  • Bone Diseases
  • Genetic Diseases, Inborn
  • Musculoskeletal Diseases
  • Natriuretic Peptide, C-type
  • Osteochondrodysplasias
  • Physiological Effects of Drugs
  • Skeletal Dysplasias

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by BioMarin Pharmaceutical on 2026-08-21.