Change From Baseline in Annualized Growth Velocity (AGV) at Week 52
| Group | Value (cm/year) |
|---|---|
| BMN 111 - 15 μg/kg | 1.71 |
| Placebo | 0.13 |
Trial · Interventional
CompletedPhase 3Results postedRegistry dataUnited States, Australia, Germany, Japan, Spain, Turkey (Türkiye), United Kingdom
1 trial · 1 completed · 1 with posted results
A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
NCT0319776612 Dec 2016 to 30 Oct 2019
Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.
The intent and design of this Phase 3 study is to assess BMN 111 as a therapeutic option for the treatment of children with Achondroplasia.
Experimental
Active BMN 111
Daily subcutaneous injection of 15 micrograms per kilogram BMN111
Placebo comparator
Placebo
Daily subcutaneous injection of placebo
Outcomes the sponsor listed as primary.
Change From Baseline in Annualized Growth Velocity (AGV) at Week 52
Time frame · At Baseline and Week 52
AGV at a Post-baseline Visit is defined as \[(Height at Post-baseline Visit - Height at Baseline)/(Date of Post-baseline Visit - Date of Baseline Assessment)\] x 365.25 AGV at Baseline is defined as \[(Height at Baseline - last height measurement in Study 111-901 at least 6 months prior to Baseline)/(Date of Baseline Assessment - Date of last height measurement in Study 111-901 at least 6 months prior to Baseline)\] x 365.25
Change From Baseline in Height Z-score at Week 52
Time frame · At baseline and Week 52
Z-Scores were derived using age-sex specific reference data (means and SDS) for average stature children per the Centers for Disease Control and Prevention. A height Z score of 0 would indicate that the subject's height is equal to the mean height for the average stature population of the same sex and age. A positive height Z score indicates that the subjects height is above the mean height for the average stature population of the same sex and age, whilst a negative height Z score indicates that the subjects height is below the mean height for the average stature population of the same sex and age. To conclude if the height Z score increases then this means the height deficit has decreased.
Change From Baseline in Upper to Lower Segment Body Ratio at Week 52
Time frame · At baseline and Week 52
Evaluate change from baseline in mean upper:lower segment body ratio in subjects treated with BMN 111 compared with control subjects in the placebo group at 52 weeks
Summary of Subjects Experiencing Adverse Events (AEs) During Treatment
Time frame · Up to Week 56
AEs with onset or worsening after the initiation of study drug and up to 30 days after study drug discontinuation were included. serious adverse event (SAE)
Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.
| Group | Value (cm/year) |
|---|---|
| BMN 111 - 15 μg/kg | 1.71 |
| Placebo | 0.13 |
| Group | Value (Z-Score) |
|---|---|
| BMN 111 - 15 μg/kg | 0.27 |
| Placebo | -0.01 |
| Group | Value (Ratio) |
|---|---|
| BMN 111 - 15 μg/kg | -0.03 |
| Placebo | -0.02 |
| Group | Value (participants) |
|---|---|
| BMN 111 - 15 μg/kg | 59 |
| Placebo | 60 |
| Group | Deaths | Serious events | Other events |
|---|---|---|---|
| BMN 111 - 15 μg/kgBMN 111: Subcutaneous injection of 15 μg/kg of BMN 111 daily | 0 / 60 | 3 / 60 | 59 / 60 |
| PlaceboPlacebo: Subcutaneous injection of placebo daily | 0 / 61 | 4 / 61 | 60 / 61 |
Each cell reads participants affected / participants at risk, as the registry posted them.
Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works
Studies that cite this trial.
3 studies on this page
Indexing hint: Randomized controlled trial · 2025
Savarirayan et al. 2025Med (New York, N.Y.)cited by 15
PMID 39740666DOI 10.1016/j.medj.2024.11.019PUB-39740666
PubMed tag: none · 2024
Qi et al. 2024Clinical pharmacokinetics
PMID 38649657PMC11106139DOI 10.1007/s40262-024-01371-6PUB-38649657
Indexing hint: Randomized controlled trial · 2024
Savarirayan et al. 2024Genetics in medicine : official journal of the American College of Medical Geneticscited by 15
PMID 39305160DOI 10.1016/j.gim.2024.101274PUB-39305160
Indexing hint: Randomized controlled trial · 2022
Chan et al. 2022Clinical pharmacokineticscited by 30
PMID 34431071PMC8813707DOI 10.1007/s40262-021-01059-1PUB-34431071
Who could join, as posted.
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