BMN 333 versus daily vosoritide for growth in children with achondroplasia

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This multicenter, multinational, randomized, active-controlled, operationally seamless Phase 2/3 study compares BMN 333 with vosoritide in treatment-naïve children with genetically confirmed achondroplasia. Phase 2 will identify the optimal BMN 333 dose and determine whether to proceed into Phase 3, and Phase 3 will compare the selected BMN 333 dose against daily vosoritide. Participants receive 52 weeks of treatment (BMN 333 once weekly; vosoritide once daily) and are followed for up to 61 weeks from screening to safety follow-up. Key eligibility includes specified age ranges, open epiphyses, ambulatory status, Tanner staging as required by phase, and no prior growth-promoting therapy.

Why it matters

Who can join: the full rules

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

Where it's running

• UCSF Benioff Children's Hospital Oakland
  Oakland, California, United States Recruiting
• Ann & Robert H. Lurie Children's Hospital of Chicago
  Chicago, Illinois, United States Recruiting
• Johns Hopkins Medicine
  Baltimore, Maryland, United States Recruiting
• Cincinnati Children's Hospital Medical Center
  Cincinnati, Ohio, United States Recruiting
• Children's Hospital of Philadelphia
  Philadelphia, Pennsylvania, United States Recruiting
• Texas Children's Hospital
  Dallas, Texas, United States Recruiting

Study contacts

• Study coordinator: Trial Specialist
• Email: medinfo@bmrn.com
• Phone: 1-800-983-4587

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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• Evaluating a new treatment for infants with achondroplasia

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.