BMN 333 vs Vosoritide for Achondroplasia
(ASPEN Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
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.
Who Is on the Research Team?
Medical Director, PhD
Principal Investigator
BioMarin Pharmaceutical
Are You a Good Fit for This Trial?
Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive BMN 333 or vosoritide for growth promotion
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- BMN 333
How Is the Trial Designed?
6
Treatment groups
Experimental Treatment
Active Control
Participants will be randomized to receive different dose levels of BMN 333
Participants will be randomized to receive different dose levels of BMN 333
Participants will be randomized to receive different dose levels of BMN 333
weight band dosing, Modified recombinant human C-type natriuretic peptide Vosoritide
weight band dosing, Modified recombinant human C-type natriuretic peptide Vosoritide
Find a Clinic Near You
Who Is Running the Clinical Trial?
BioMarin Pharmaceutical
Lead Sponsor
Alexander Hardy
BioMarin Pharmaceutical
Chief Executive Officer since 2023
MBA from INSEAD
Greg Friberg
BioMarin Pharmaceutical
Chief Medical Officer
MD from New York Medical College
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