Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia
a study on Achondroplasia
Summary
- Eligibility
- for people ages 2-17 (full criteria)
- Location
- at UCSF
- Dates
- study startedstudy ends around
Description
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.
Official Title
A Multicenter, Randomized, Operationally Seamless Phase 2/3 Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia
Details
The main purpose of this study is to evaluate the effects of BMN 333 on growth compared with vosoritide in participants with achondroplasia who have not received any growth-promoting treatments. The study includes 2 parts: the Phase 2 part will select the optimal BMN 333 dose to be used in Phase 3 and determine study continuation into Phase 3; the Phase 3 part will compare the effects of the selected dose of BMN 333 with vosoritide. Study details for either Phase 2 or Phase 3 include the following:
- Study duration: up to 61 weeks (from screening to Safety Follow-up visit)
- Treatment duration: 52 weeks. Treatment frequency: BMN 333, once weekly; vosoritide, once daily
Keywords
Achondroplasia, ACH, Bone Diseases, Developmental Dwarfism, Bone Diseases, Genetic Diseases, Inborn, Musculoskeletal Diseases, Natriuretic Peptide, C-type, Osteochondrodysplasias, Physiological Effects of Drugs, Skeletal Dysplasias, Inborn Genetic Diseases, vosoritide, BMN 333, Vosoritide Injection [Voxzogo]
Eligibility
You can join if…
Open to people ages 2-17
- 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 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) 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.
- 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.
- 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 treatment of ACH or short stature at any time
- Have been treated with GH, insulin-like growth factor 1, or anabolic steroids in the 6 months prior to treatment start
Locations
- UCSF Benioff Children's Hospital Oakland
accepting new patients
Oakland California 94609 United States - Texas Children Hospital, Baylor College of Medicine, Houston TX
accepting new patients
Houston Texas 77030 United States
Details
- Status
- accepting new patients
- Start Date
- Completion Date
- (estimated)
- Sponsor
- BioMarin Pharmaceutical
- ID
- NCT07441876
- Phase
- Phase 2/3 Achondroplasia Research Study
- Study Type
- Interventional
- Participants
- Expecting 160 study participants
- Last Updated
Please contact me about this study
We will not share your information with anyone other than the team in charge of this study, which might include an external sponsor. Providing your contact details does not obligate you to participate in the research.
Thank you!
The study team should get back to you in a few business days.