NCT07441876 Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia
| NCT ID | NCT07441876 |
| Status | Recruiting |
| Phase | Phase 2, Phase 3 |
| Sponsor | BioMarin Pharmaceutical |
| Condition | Achondroplasia |
| Study Type | INTERVENTIONAL |
| Enrollment | 160 participants |
| Start Date | 2026-04 |
| Primary Completion | 2029-06 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
In Phase 2, researchers evaluate early signs of effectiveness. You may be randomized to receive the active treatment or a comparator. Monitoring continues closely.
This trial targets 160 participants in total. It began in 2026-04 with a primary completion date of 2029-06.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
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.
Eligibility Criteria
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
Contact & Investigator
Medical Director, PhD
STUDY DIRECTOR
BioMarin Pharmaceutical
Frequently Asked Questions
Who can join the NCT07441876 clinical trial?
This trial is open to participants of all sexes, aged 2 Years or older, up to 17 Years, studying Achondroplasia. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT07441876 trial and what does that mean for participants?
Phase 2 trials evaluate whether the treatment shows signs of effectiveness while continuing to monitor safety. More participants are enrolled than in Phase 1 to help refine the treatment protocol.
Is NCT07441876 currently recruiting?
Yes, NCT07441876 is actively recruiting participants. Contact the research team at medinfo@bmrn.com for enrollment information.
Where is the NCT07441876 trial being conducted?
This trial is being conducted at Oakland, United States, Chicago, United States, Baltimore, United States, Houston, United States and 6 additional locations.
Who is sponsoring the NCT07441876 clinical trial?
NCT07441876 is sponsored by BioMarin Pharmaceutical. The principal investigator is Medical Director, PhD at BioMarin Pharmaceutical. The trial plans to enroll 160 participants.