Trial · NCT03583697
A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia
NCT03583697, Phase 2, Completed, BioMarin Pharmaceutical; results first posted 2024-06-13
3 sourcestrials-2026-11-12-0.5.0
Download JSON
Cite this record
ConductScience Library. A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia. Trials, record NCT03583697. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03583697
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia
- NCT number
- NCT03583697
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 75 participants, actual
- Lead sponsor
- BioMarin Pharmaceutical
- Conditions
- Achondroplasia
- Interventions
- BMN 111, Placebo
- First posted
- 2018-07-11
- Last updated
- 2024-06-13
- Other registries' IDs
- eudract:2016-003826-18
- Results first posted
- 2024-06-13
Source ClinicalTrials.gov
Registered outcomes55
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Adverse Events (AEs) by Severity Grade and Study Drug Treatment-emergent Adverse Events (TEAEs) | Up to Week 56 (Safety Follow-Up +/-7d) | A treatment-emergent Adverse Events (TEAE) is any Adverse Events that newly appeared, increased in frequency or worsened in severity following initiation of study drug administration. A severity grade was defined by the Common Terminology Criteria for Adverse Events (CTCAE) Version 4.03. As per CTCAE, Grade 1 scales as Mild; Grade 2 scales as Moderate; Grade 3 scales as severe or medically significant but not immediately life threatening; Grade 4 scales as life-threatening consequences; and Grade 5 scales as death related to AE. Safety Population includes all sentinel and randomized participants in the FAS who received at least one dose of vosoritide or placebo in this study. Serious adverse event (SAE) |
| Primary | Change From Baseline in Height Z-score at Week 52. | Baseline to 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. standard deviation score (SDS). The primary efficacy analysis population was the subset of randomized participants in the FAS. |
| Secondary | Change From Baseline in Height at Week 52 | Baseline to Week 52 | As a general rule, standing height/sitting height was used if participants were aged \>= 24 months at baseline. Body length/crown to rump was used if participants were aged \< 24 months at baseline. If body length was not measured and standing height was available, standing height was used. |
| Secondary | Change From Baseline in Annualized Growth Velocity (AGV) at Week 52 | Baseline to Week 52 | AGV was derived over 12-month intervals starting from the baseline visit. Annualized growth velocity (AGV) = Standing Height at Date 2 - Standing Height at Date 1/Interval Length (Days) x 365.25. Difference in LS means were obtained from an analysis of covariance model. |
| Secondary | Change From Baseline in Upper to Lower Body Segment Ratio at Week 52 | Baseline to Week 52 | Upper to Lower Body ratio=Sitting Height / (Standing Height - Sitting Height). |
| Secondary | Change From Baseline in Other Growth Measures (Upper Body Length, Head Circumference, Arm Span, Upper Arm Length, Lower Arm Length, Lower Body Length, Upper Leg Length, Knee to Heel Length, and Tibial Length) at Week 52 | Baseline to Week 52 | Change from baseline in other growth measures (upper body length, head circumference, arm span, upper arm length, lower arm (Forearm) length, Lower Body Length, Upper Leg Length (Thigh), knee to heel length, and tibial length) at Week 52. Participants aged \<24 months, body length and crown to rump length take precedence over standing height and sitting height. Participants aged \<24 months at baseline and \>=24 months at Week 52, body length and crown to rump length take precedence. |
| Secondary | Change From Baseline in Other Body Proportion Ratios (Arm Span to Height Ratio, Upper Arm Length to Lower Arm [Forearm] Length Ratio, Upper Leg Length [Thigh] to Knee to Heel Length Ratio, and Upper Leg Length (Thigh) to Tibial Length Ratio) at Week 52 | Baseline to Week 52 | For Height: subjects aged \< 24 months, body length takes precedence over standing height. Subjects aged \< 24 months at baseline and \>= 24 months at Week 52, body length takes precedence. |
| Secondary | Change From Baseline in Body Mass Index (BMI) at Week 52 | Baseline to Week 52 | Body Mass Index (BMI): For height used for BMI calculation, participants aged \< 24 months, body length takes precedence over standing height. Participants aged \< 24 months at baseline and \>= 24 months at Week 52, body length takes precedence. |
| Secondary | Change From Baseline in BMI Z-score at Week 52 | Baseline to Week 52 | The body mass index (BMI) Z-score represents the participant's BMI converted to an age and sex appropriate standard deviation score (SDS) by comparison with an average stature reference population. The weight Z-score of 0 represents the mean BMI for age and sex of the reference population. A positive BMI Z-score means BMI is above the mean BMI for the average stature population of the same sex and age and a negative BMI Z-Score means BMI is below the mean BMI for the average stature population of the same sex and age. BMI Z-scores are derived only for participants aged 24 months or older, the change from baseline to Week 52 in BMI Z-score is summarized for Cohort 1 only as no participants in Cohort 2 or Cohort 3 were 24 months at baseline. |
| Secondary | Change From Baseline in Weight Z-score at Week 52 | Baseline to Week 52 | The weight Z-score represents the participant's weight converted to an age and sex appropriate standard deviation score (SDS) by comparison with an average stature reference population. The weight Z-score of 0 represents the mean weight for age and sex of the reference population. A positive weight Z-score means weight is above the mean weight for the average stature population of the same sex and age and a negative weight Z-Score means weight is below the mean weight for the average stature population of the same sex and age. |
1 to 10 of 55Source ClinicalTrials.gov
See all 55 registered outcomesOutcomes55
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With Adverse Events (AEs) by Severity Grade and Study Drug Treatment-emergent Adverse Events (TEAEs) | Up to Week 56 (Safety Follow-Up +/-7d) | Safety Population: The Safety Population was a subset of the FAS who received at least one dose of vosoritide or placebo in the study. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Change From Baseline in Height Z-score at Week 52. | Baseline to Week 52 | FAS \& FAS (randomized) population:The FAS (randomized) was a subset of the FAS, which included 64 participants who were randomized to study treatment (32 participants randomized to vosoritide \& 32 participants randomized to placebo).~This is model-based analysis, it is true that the participants analyzed for placebo FAS \& FAS (randomized) are the same, but participants analyzed for vosoritide arm FAS \& FAS(randomized) are different, that's why the model-based results for placebo are different | Z-score | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in Height at Week 52 | Baseline to Week 52 | FAS (randomized) Population and FAS population.~This is model-based analysis, it is true that the participants analyzed for placebo FAS \& FAS (randomized) are the same, but participants analyzed for vosoritide arm FAS \& FAS (randomized) are different, that's why the model-based results for placebo are different. | cm | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in Annualized Growth Velocity (AGV) at Week 52 | Baseline to Week 52 | FAS (randomized) Population and FAS population.~This is model-based analysis, it is true that the participants analyzed for placebo FAS \& FAS (randomized) are the same, but participants analyzed for vosoritide arm FAS \& FAS (randomized) are different, that's why the model-based results for placebo are different. | cm/year | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in Upper to Lower Body Segment Ratio at Week 52 | Baseline to Week 52 | FAS (randomized) Population and FAS population.~This is model-based analysis, it is true that the participants analyzed for placebo FAS \& FAS (randomized) are the same, but participants analyzed for vosoritide arm FAS \& FAS (randomized) are different, that's why the model-based results for placebo are different. | Ratio | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in Other Growth Measures (Upper Body Length, Head Circumference, Arm Span, Upper Arm Length, Lower Arm Length, Lower Body Length, Upper Leg Length, Knee to Heel Length, and Tibial Length) at Week 52 | Baseline to Week 52 | FAS population.~Change from baseline was based on the participants with available measurements at both time points. Baseline is defined as Day 1 or screening if a Day 1 measurement is not available. | cm | MEAN |
| SECONDARY | Change From Baseline in Other Body Proportion Ratios (Arm Span to Height Ratio, Upper Arm Length to Lower Arm [Forearm] Length Ratio, Upper Leg Length [Thigh] to Knee to Heel Length Ratio, and Upper Leg Length (Thigh) to Tibial Length Ratio) at Week 52 | Baseline to Week 52 | FAS population.~Change from baseline was based on the subjects with available measurements at both time points. Baseline is defined as Day 1 or screening if a Day 1 measurement is not available | Ratio | MEAN |
| SECONDARY | Change From Baseline in Body Mass Index (BMI) at Week 52 | Baseline to Week 52 | FAS population.~Change from baseline was based on the subjects with available measurements at both time points. Baseline is defined as Day 1 or screening if a Day 1 measurement is not available. | kg/m^2 | MEAN |
| SECONDARY | Change From Baseline in BMI Z-score at Week 52 | Baseline to Week 52 | FAS population. | Z-Score | MEAN |
| SECONDARY | Change From Baseline in Weight Z-score at Week 52 | Baseline to Week 52 | FAS population~Change from baseline was based on the subjects with available measurements at both time points. Baseline is defined as Day 1 or screening if a Day 1 measurement is not available | Z-score | MEAN |
1 to 10 of 55Source ClinicalTrials.gov
See all 55 outcomesResults by group765
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Adverse Events (AEs) by Severity Grade and Study Drug Treatment-emergent Adverse Events (TEAEs) | Sentinel | Participants with any AE | 11 | Participants | |
| Number of Participants With Adverse Events (AEs) by Severity Grade and Study Drug Treatment-emergent Adverse Events (TEAEs) | Randomized Vosoritide | Participants with any AE | 32 | Participants | |
| Number of Participants With Adverse Events (AEs) by Severity Grade and Study Drug Treatment-emergent Adverse Events (TEAEs) | Randomized Placebo | Participants with any AE | 32 | Participants | |
| Number of Participants With Adverse Events (AEs) by Severity Grade and Study Drug Treatment-emergent Adverse Events (TEAEs) | Sentinel | Participants with any SAE | 0 | Participants | |
| Number of Participants With Adverse Events (AEs) by Severity Grade and Study Drug Treatment-emergent Adverse Events (TEAEs) | Randomized Vosoritide | Participants with any SAE | 3 | Participants | |
| Number of Participants With Adverse Events (AEs) by Severity Grade and Study Drug Treatment-emergent Adverse Events (TEAEs) | Randomized Placebo | Participants with any SAE | 6 | Participants | |
| Number of Participants With Adverse Events (AEs) by Severity Grade and Study Drug Treatment-emergent Adverse Events (TEAEs) | Sentinel | Participants with any treatment-related AE | 8 | Participants | |
| Number of Participants With Adverse Events (AEs) by Severity Grade and Study Drug Treatment-emergent Adverse Events (TEAEs) | Randomized Vosoritide | Participants with any treatment-related AE | 29 | Participants | |
| Number of Participants With Adverse Events (AEs) by Severity Grade and Study Drug Treatment-emergent Adverse Events (TEAEs) | Randomized Placebo | Participants with any treatment-related AE | 17 | Participants | |
| Number of Participants With Adverse Events (AEs) by Severity Grade and Study Drug Treatment-emergent Adverse Events (TEAEs) | Sentinel | Participants with any treatment-related SAEs | 0 | Participants |
1 to 10 of 765Source ClinicalTrials.gov
See all 765 results by groupParticipant flow18
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Sentinel | 11 | |
| Overall Study | STARTED | Randomized Vosoritide | 32 | |
| Overall Study | STARTED | Randomized Placebo | 32 | |
| Overall Study | Cohort 1: Age >= 24 to < 60 Months | Sentinel | 4 | |
| Overall Study | Cohort 1: Age >= 24 to < 60 Months | Randomized Vosoritide | 15 | |
| Overall Study | Cohort 1: Age >= 24 to < 60 Months | Randomized Placebo | 16 | |
| Overall Study | Cohort 2: Age >= 6 to < 24 Months | Sentinel | 4 | |
| Overall Study | Cohort 2: Age >= 6 to < 24 Months | Randomized Vosoritide | 8 | |
| Overall Study | Cohort 2: Age >= 6 to < 24 Months | Randomized Placebo | 8 | |
| Overall Study | Cohort 3: Age 0 to < 6 Months | Sentinel | 3 |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 participant flowAdverse events54
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Rhinitis | Infections and infestations | Placebo | 0 | 32 | 0 |
| other | Rhinitis | Infections and infestations | Vosoritide | 4 | 43 | 8 |
| other | Viral upper respiratory tract infection | Infections and infestations | Placebo | 0 | 32 | 0 |
| other | Viral upper respiratory tract infection | Infections and infestations | Vosoritide | 3 | 43 | 3 |
| other | Lower respiratory tract infection | Infections and infestations | Placebo | 1 | 32 | 3 |
| other | Lower respiratory tract infection | Infections and infestations | Vosoritide | 4 | 43 | 4 |
| other | Viral infection | Infections and infestations | Placebo | 4 | 32 | 8 |
| other | Viral infection | Infections and infestations | Vosoritide | 8 | 43 | 28 |
| other | Injection site reaction | General disorders | Placebo | 13 | 32 | 154 |
| other | Injection site reaction | General disorders | Vosoritide | 34 | 43 | 3,057 |
1 to 10 of 54Source ClinicalTrials.gov
See all 54 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Placebo | 0 | 32 |
| Total, all-cause mortality | Vosoritide | 1 | 43 |
| Total, other adverse events | Placebo | 32 | 32 |
| Total, other adverse events | Vosoritide | 43 | 43 |
| Total, serious adverse events | Placebo | 6 | 32 |
| Total, serious adverse events | Vosoritide | 3 | 43 |
Source ClinicalTrials.gov
Papers3
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Vosoritide vs Placebo: deaths from any cause | Trial comparisons |
| Vosoritide vs Placebo: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT03583697
GET https://conductscience.org/api/trials/v1/NCT03583697/registered?page=2