Comparing 6 approaches for achondroplasia
Official title: Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia
A Multicenter, Randomized, Operationally Seamless Phase 2/3 Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia
- Phase 2
- 6 groups
- One site, in Montreal
- Recruiting
Interventions
- Medication
BMN 333
Administration: Weekly subcutaneous injection
- Medication
Vosoritide Injection [Voxzogo]
Administration: Daily subcutaneous injection
Canadian Sites (1)
1 listed, none recruiting
- Not yet recruiting
Universite de Montreal - Centre Hospitalier Universitaire Sainte-Justine
Montreal, Quebec
Eligibility Criteria
See who this study is looking for10 criteria
The trial’s own eligibility text, word for word from the registry. Only the trial site can say who takes part.
Inclusion
- +Participants must be aged ≥ 2 to \< 11 years (Phase 2Phase 2A middle-stage study looking at what a treatment does and watching for side effects.Read more →) or ≥ 2 to \< 18 years (Phase 3Phase 3A large study comparing a treatment against the current standard.Read more →), at the time of signing the informed consentInformed consentThe process of being told what taking part involves, then choosing freely.Read more →
- +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
Exclusion
- −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 screeningScreeningThe checks done before joining, to see whether a study fits.Read more →.
- −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
In plain language
Assembled directly from this trial’s registry record. Every sentence traces to a field below — nothing here is generated or interpreted.
What this study is
This study is testing a treatment for a condition.
Phase 2Phase 2A middle-stage study looking at what a treatment does and watching for side effects.Read more →/3 — a combined study that runs the middle stage and the large comparison stage together.
From the trial registry
Built from these fields:
- designModule.designInfo.primaryPurpose
- designModule.phases
Who receives what
There are 6 groups in this study.
Groups A, B, C and E receive BMN 333.
Registry label: A: Phase 2: Low Dose · B: Phase 2: Medium Dose · C: Phase 2: High Dose · E: Phase 3: BMN 333 at selected dose after Phase 2
Groups D and F, the comparison group, receive Vosoritide Injection [Voxzogo].
Registry label: D: Phase 2: Vosoritide · F: Phase 3: Vosoritide
From the trial registry
Built from these fields:
- armsInterventionsModule.armGroups[].label
- armsInterventionsModule.armGroups[].type
- armsInterventionsModule.armGroups[].interventionNames
How the study is run
Which group you would be placed in is decided by chance, like a coin flip — not by you and not by your doctor.
One group of people involved — usually the participants — is not told which treatment is which.
From the trial registry
Built from these fields:
- designModule.designInfo.allocation
- designModule.designInfo.maskingInfo.masking
Is there a placebo?
This study does not list a placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → group.
One group receives an existing treatment, so the two can be compared.
From the trial registry
Built from these fields:
- armsInterventionsModule.armGroups[].type
- armsInterventionsModule.armGroups[].interventionNames
Who the study is looking for
The study lists an age range of 2 years to 17 years.
The study is open to people of any sex.
The study does not accept healthy volunteersHealthy volunteerSomeone without the condition being studied who takes part anyway.Read more →.
These are the criteria the study lists. Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can decide whether someone can take part.
From the trial registry
Built from these fields:
- eligibilityModule.minimumAge
- eligibilityModule.maximumAge
- eligibilityModule.sex
- eligibilityModule.healthyVolunteers
How big and how long
The study aims to enrol about 160 people.
The study is currently expected to finish around September 2029.
The main measurement is taken over: 52 weeks.
From the trial registry
Built from these fields:
- designModule.enrollmentInfo.count
- statusModule.completionDateStruct.date
- outcomesModule.primaryOutcomes[].timeFrame
What the study measures
Phase 2Phase 2A middle-stage study looking at what a treatment does and watching for side effects.Read more →: Predicted Annualized Growth Velocity (AGV) at Week 52 (based on AGV at Weeks 26, 39, and 52 [available cumulative data] — measured over 52 weeks.
Phase 3Phase 3A large study comparing a treatment against the current standard.Read more →: Annualized Growth Velocity (AGV) at Week 52 — measured over 52 weeks.
From the trial registry
Built from these fields:
- outcomesModule.primaryOutcomes[].measure
- outcomesModule.primaryOutcomes[].timeFrame
Source: NCT07441876 on ClinicalTrials.gov. The full registry text is further down this page — this summary never replaces it.
Not medical advice. What do these terms mean?
What is being tested — in plain terms
About BMN 333Drug
Administration: Weekly subcutaneous injection
From the trial registry — its own words, unedited.
What a drug is here: A medicine — a chemical or small-molecule treatment — given as the thing being studied.
Read the full explanation → · in clinical review
About Vosoritide Injection [Voxzogo]Drug
Administration: Daily subcutaneous injection
From the trial registry — its own words, unedited.
What a drug is here: A medicine — a chemical or small-molecule treatment — given as the thing being studied.
Read the full explanation → · in clinical review
Common questions
Answered from this trial’s registry record. Where the record doesn’t say, these answers say so rather than filling the gap.
Am I eligible for this trial?
Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can decide whether someone can take part. Concord cannot make that determination, and neither can any tool that has not examined you.
What the study lists: an age range of 2 years to 17 years.
The full inclusionInclusion criteriaThe things you must have or be for a study to consider you.Read more → and exclusion criteriaExclusion criteriaThe things that would prevent someone from taking part.Read more → are published on this page, exactly as the study team wrote them.
The useful next step is to bring this trial to your doctor. Answering a few questions first gives them something concrete to review.
From the trial registry
- eligibilityModule.minimumAge
- eligibilityModule.maximumAge
- eligibilityModule.eligibilityCriteria
Is there a placebo?
This study does not list a placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → group.
One group receives an existing treatment so the two can be compared.
From the trial registry
- armsInterventionsModule.armGroups[].type
Would I know which treatment I am getting?
One group of people involved — usually the participants — is not told which treatment is which.
Which group you would be placed in is decided by chance, like a coin flip — not by you and not by your doctor.
From the trial registry
- designModule.designInfo.maskingInfo.masking
- designModule.designInfo.allocation
Who can join?
The study lists an age range of 2 years to 17 years.
It is open to people of any sex.
It does not accept healthy volunteersHealthy volunteerSomeone without the condition being studied who takes part anyway.Read more →.
Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can confirm whether a particular person can take part.
From the trial registry
- eligibilityModule.minimumAge
- eligibilityModule.maximumAge
- eligibilityModule.sex
- eligibilityModule.healthyVolunteers
How long would this take?
The study's main measurement is taken over: 52 weeks.
The study as a whole is currently expected to finish around 2029-09.
How long any one person takes part can differ from the study length. The study team can tell you what the schedule looks like in practice.
From the trial registry
- outcomesModule.primaryOutcomes[].timeFrame
- statusModule.completionDateStruct.date
How many people are taking part?
The study aims to enrol about 160 people.
From the trial registry
- designModule.enrollmentInfo.count
Where is this happening?
This study lists one location: Montreal, Quebec, Canada.
Sites can open and close during a study, so confirm with the team before travelling.
From the trial registry
- trial_locations
About 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.
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See if this trial could fit youThis page is for informational purposes only and does not constitute medical advice. Clinical trial eligibility can only be determined by the trial site after proper screening. Trial information is sourced from ClinicalTrials.gov and may not reflect the most current status. Always consult your healthcare provider before making decisions about clinical trial participation.