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Compares treatment options for Achondroplasia
Official title Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia
ClinicalTrials.gov ID: NCT07441876
What this study is testing
What is BMN 333?
BMN 333 is an investigational medicine, given as an once-weekly injection under the skin, being studied as a potential treatment for achondroplasia.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's 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.
- Phase 3: a large, late-stage study
- Which group you join is decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 2 to 17
You may be able to join if
- 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
- Participants must have ACH (confirmed by documented genetic testing) and open epiphyses
- Are Tanner Stage I (Phase 2) or any Tanner stage (Phase 3)
- Are ambulatory and able to stand without assistance
You likely can't join if
- Have any short stature condition other than ACH (eg, hypochondroplasia, trisomy 21, pseudoachondroplasia, GH deficiency)
- Have any of the following disorders: Hypothyroidism or hyperthyroidism, unless treated with evidence of normalized thyroid-stimulating hormone (TSH)...
- Have history of any renal insufficiency or cardiac/ cardiovascular disease that places the participant at increased risk of an adverse cardiac...
- Have had bone fractures of the long bones or spine within 6 months prior to screening.
- Have used vosoritide, any other approved product (except GH, as detailed below), investigational product, or investigational medical device for the...
- Have been treated with GH, insulin-like growth factor 1, or anabolic steroids in the 6 months prior to treatment start
The study team makes the final eligibility decision.
Where it's taking place
- Oakland, California, United States
- Wilmington, Delaware, United States
- Chicago, Illinois, United States
- Baltimore, Maryland, United States
- Cincinnati, Ohio, United States
- Philadelphia, Pennsylvania, United States
- Dallas, Texas, United States
- Houston, Texas, United States
- San Antonio, Texas, United States
- Parkville, Victoria, Australia
- Montreal, Quebec, Canada
- Genova, Italy
- Wroclaw, Poland
- Bucharest, Romania
- Craiova, Romania
- Seoul, South Korea
- Yangsan, South Korea
- Bristol, United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 2 years to 17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Oakland, California, United States; Wilmington, Delaware, United States; Chicago, Illinois, United States; Baltimore, Maryland, United States; Cincinnati, Ohio, United States; Philadelphia, Pennsylvania, United States and 12 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.