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Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia

RecruitingPhase 2, Phase 3

Sponsor: BioMarin Pharmaceutical

Conditions: Achondroplasia

Interventions: BMN 333, Vosoritide Injection [Voxzogo]

Countries: United States, Australia, Canada, Italy, Japan, Poland, Romania, South Korea

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 overview

Sex: ALL

Age: 2 Years to 17 Years

Healthy volunteers: No

Study type: INTERVENTIONAL

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

Locations (19)

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
Osaka, Japan
Wroclaw, Poland
Bucharest, Romania
Craiova, Romania
Seoul, South Korea
Yangsan, South Korea
Bristol, United Kingdom