Trial · NCT02055157
A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia
NCT02055157, Phase 2, Completed, BioMarin Pharmaceutical; results first posted 2021-01-15
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia. Trials, record NCT02055157. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02055157
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia
- NCT number
- NCT02055157
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 35 participants, actual
- Lead sponsor
- BioMarin Pharmaceutical
- Conditions
- Achondroplasia
- Interventions
- BMN 111
- First posted
- 2014-02-05
- Last updated
- 2021-01-15
- Other registries' IDs
- eudract:2013-004137-32
- Results first posted
- 2021-01-15
Source ClinicalTrials.gov
Registered outcomes23
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Overall Summary of Adverse Events During Initial 6-Month Period | Up to Month 6 ± 7 Days | 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. Serious adverse event (SAE). |
| Primary | Overall Summary of Adverse Events During Entire Study Period | Up to Month 25 ± 7 Days | 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. TEAE - Treatment-emergent adverse event. SAE - Serious adverse event. |
| Secondary | Change From Baseline in Annualized Growth Velocity (AGV) During Initial 6-Month | At 6 month (Day 183) | Annualized Growth Velocity at Day 183 is assessed on standing height as ((Height at Day 183 Visit - Height at Baseline Visit)/(Date at Day 183 Visit - Baseline Visit Date)) x 365.25. |
| Secondary | Change From Baseline in Annualized Growth Velocity (AGV) During Entire Study Period - Cohort 3 and 4 | At month 24 | Annualized Growth Velocity at Day 183 visit is assessed on standing height as ((Height at Day 183 Visit - Height at Baseline Visit)/(Date at Day 183 Visit - Baseline Visit Date)) x 365.25. |
| Secondary | Change From Baseline in Annualized Growth Velocity (AGV) During Entire Study Period - Cohort 1 and 2 Switchers | At month 24 | Annualized Growth Velocity at 1st visit with \>= 12 months on 15ug/kg is assessed on standing height as ((Height at 1st Visit with \>= 12 Months on 15 μg/kg - Height at 1st Visit on 15 μg/kg)/(Date of the 1st Visit with \>= 12 months on 15 μg/kg - Date of at 1st Visit on 15 μg/kg)) x 365.25. |
| Secondary | Change From Baseline in Height Z-Scores Using Centers for Disease Control and Prevention (CDC) Reference Standard During Initial 6-Months | At month 6 (Day 183) | Height Z scores indicates how far a particular child is from the average height for children of the same sex and age. A positive height Z score indicates the child's height is above average whilst a negative Z score indicates the child's height is below average. Height Z scores below -2 Standard Deviation Scores (SDs) indicate a child's height is no longer within normal height range for average stature children of the same sex and age. Z-scores are derived using non-ACH age-sex-specific reference data (means and standard deviations) per Centers for Disease Control and Prevention (CDC). |
| Secondary | Change From Baseline in Height Z-Scores Using CDC Reference Standard During Entire Study Period - Cohort 3 and 4 | At month 24 | Height Z scores indicates how far a particular child is from the average height for children of the same sex and age. A positive height Z score indicates the child's height is above average whilst a negative Z score indicates the child's height is below average. Height Z scores below -2 SDs indicate a child's height is no longer within normal height range for average stature children of the same sex and age. Z-scores are derived using non-ACH age-sex-specific reference data (means and standard deviations) per Centers for Disease Control and Prevention (CDC). |
| Secondary | Change From Baseline in Height Z-Scores Using CDC Reference Standard During Entire Study Period - Cohort 1 and 2 Switchers | At month 24 | Height Z scores indicates how far a particular child is from the average height for children of the same sex and age. A positive height Z score indicates the child's height is above average whilst a negative Z score indicates the child's height is below average. Height Z scores below -2 SDs indicate a child's height is no longer within normal height range for average stature children of the same sex and age. Z-scores are derived using non-ACH age-sex-specific reference data (means and standard deviations) per Centers for Disease Control and Prevention (CDC). |
| Secondary | Change From Baseline in Upper to Lower Body Ratios During Initial 6-Months | At month 6 (Day 183) | The Upper to Lower Body ratio prior to treatment, at baseline, and through 6 months is assessed on Sitting Height / (Standing Height - Sitting Height) |
| Secondary | Change From Baseline in Upper to Lower Body Ratios During Entire Study Period - Cohort 3 and 4 | At month 24 | The Upper to Lower Body ratio prior to treatment, at baseline, and through 24 months is assessed on Sitting Height / (Standing Height - Sitting Height) |
1 to 10 of 23Source ClinicalTrials.gov
See all 23 registered outcomesOutcomes23
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Overall Summary of Adverse Events During Initial 6-Month Period | Up to Month 6 ± 7 Days | Safety Analysis Population includes all subjects who received at least one dose of study treatment and were used for safety analysis in the initial 6-month period and entire study period. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Overall Summary of Adverse Events During Entire Study Period | Up to Month 25 ± 7 Days | Safety Analysis Population. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline in Annualized Growth Velocity (AGV) During Initial 6-Month | At 6 month (Day 183) | Efficacy Analysis Population includes all subjects who received at least one dose of study treatment and who had post treatment data for any efficacy endpoint in the corresponding period were included in the Efficacy Analysis and Extension Efficacy Analysis population. | cm/year | MEAN |
| SECONDARY | Change From Baseline in Annualized Growth Velocity (AGV) During Entire Study Period - Cohort 3 and 4 | At month 24 | Efficacy Analysis Population | cm/year | MEAN |
| SECONDARY | Change From Baseline in Annualized Growth Velocity (AGV) During Entire Study Period - Cohort 1 and 2 Switchers | At month 24 | Efficacy Analysis Population | cm/year | MEAN |
| SECONDARY | Change From Baseline in Height Z-Scores Using Centers for Disease Control and Prevention (CDC) Reference Standard During Initial 6-Months | At month 6 (Day 183) | Efficacy Analysis Population | z score | MEAN |
| SECONDARY | Change From Baseline in Height Z-Scores Using CDC Reference Standard During Entire Study Period - Cohort 3 and 4 | At month 24 | Efficacy Analysis Population | z score | MEAN |
| SECONDARY | Change From Baseline in Height Z-Scores Using CDC Reference Standard During Entire Study Period - Cohort 1 and 2 Switchers | At month 24 | Efficacy Analysis Population | z score | MEAN |
| SECONDARY | Change From Baseline in Upper to Lower Body Ratios During Initial 6-Months | At month 6 (Day 183) | Efficacy Analysis Population | Ratio | MEAN |
| SECONDARY | Change From Baseline in Upper to Lower Body Ratios During Entire Study Period - Cohort 3 and 4 | At month 24 | Efficacy Analysis Population | ratio | MEAN |
1 to 10 of 23Source ClinicalTrials.gov
See all 23 outcomesResults by group150
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Overall Summary of Adverse Events During Initial 6-Month Period | Cohort 1 | Subjects with at Least 1 Reported TEAE | 8 | Participants | |
| Overall Summary of Adverse Events During Initial 6-Month Period | Cohort 2 | Subjects with at Least 1 Reported TEAE | 8 | Participants | |
| Overall Summary of Adverse Events During Initial 6-Month Period | Cohort 3 | Subjects with at Least 1 Reported TEAE | 10 | Participants | |
| Overall Summary of Adverse Events During Initial 6-Month Period | Cohort 4 | Subjects with at Least 1 Reported TEAE | 9 | Participants | |
| Overall Summary of Adverse Events During Initial 6-Month Period | Cohort 1 | Subjects Least Reported Study Drug-Related TEAE | 7 | Participants | |
| Overall Summary of Adverse Events During Initial 6-Month Period | Cohort 2 | Subjects Least Reported Study Drug-Related TEAE | 8 | Participants | |
| Overall Summary of Adverse Events During Initial 6-Month Period | Cohort 3 | Subjects Least Reported Study Drug-Related TEAE | 9 | Participants | |
| Overall Summary of Adverse Events During Initial 6-Month Period | Cohort 4 | Subjects Least Reported Study Drug-Related TEAE | 9 | Participants | |
| Overall Summary of Adverse Events During Initial 6-Month Period | Cohort 1 | Subjects with at Least 1 Reported SAE | 0 | Participants | |
| Overall Summary of Adverse Events During Initial 6-Month Period | Cohort 2 | Subjects with at Least 1 Reported SAE | 0 | Participants |
1 to 10 of 150Source ClinicalTrials.gov
See all 150 results by groupParticipant flow24
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Initial 6 Months | STARTED | Cohort 1 | 8 | |
| Initial 6 Months | STARTED | Cohort 2 | 8 | |
| Initial 6 Months | STARTED | Cohort 3 | 10 | |
| Initial 6 Months | STARTED | Cohort 4 | 9 | |
| Initial 6 Months | COMPLETED | Cohort 1 | 7 | |
| Initial 6 Months | COMPLETED | Cohort 2 | 7 | |
| Initial 6 Months | COMPLETED | Cohort 3 | 10 | |
| Initial 6 Months | COMPLETED | Cohort 4 | 8 | |
| Initial 6 Months | NOT COMPLETED | Cohort 1 | 1 | |
| Initial 6 Months | NOT COMPLETED | Cohort 2 | 1 |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 participant flowAdverse events1,992
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 | Injection site reaction | General disorders | Initial 6-Months Period 2.5 µg/kg. | 2 | 8 | |
| other | Injection site reaction | General disorders | Initial 6-Months Period 7.5 µg/kg. | 6 | 8 | |
| other | Injection site reaction | General disorders | Initial 6-Months Period 15 µg/kg. | 8 | 10 | |
| other | Injection site reaction | General disorders | Initial 6-Months Period 30 µg/kg. | 9 | 9 | |
| other | Injection site reaction | General disorders | Extension Period 2.5 µg/kg. | 2 | 7 | |
| other | Injection site reaction | General disorders | Extension Period 7.5 µg/kg. | 5 | 14 | |
| other | Injection site reaction | General disorders | Extension Period 15 µg/kg. | 15 | 22 | |
| other | Injection site reaction | General disorders | Extension Period 30 µg/kg. | 6 | 8 | |
| other | Injection site reaction | General disorders | Entire Study Period 2.5 µg/kg. | 2 | 8 | |
| other | Injection site reaction | General disorders | Entire Study Period 7.5 µg/kg. | 8 | 15 |
1 to 10 of 1,992Source ClinicalTrials.gov
See all 1,992 adverse eventsAdverse event totals36
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Initial 6-Months Period 2.5 µg/kg. | 0 | 8 |
| Total, all-cause mortality | Initial 6-Months Period 7.5 µg/kg. | 0 | 8 |
| Total, all-cause mortality | Initial 6-Months Period 15 µg/kg. | 0 | 10 |
| Total, all-cause mortality | Initial 6-Months Period 30 µg/kg. | 0 | 9 |
| Total, all-cause mortality | Extension Period 2.5 µg/kg. | 0 | 7 |
| Total, all-cause mortality | Extension Period 7.5 µg/kg. | 0 | 14 |
| Total, all-cause mortality | Extension Period 15 µg/kg. | 0 | 22 |
| Total, all-cause mortality | Extension Period 30 µg/kg. | 0 | 8 |
| Total, all-cause mortality | Entire Study Period 2.5 µg/kg. | 0 | 8 |
| Total, all-cause mortality | Entire Study Period 7.5 µg/kg. | 0 | 15 |
1 to 10 of 36Source ClinicalTrials.gov
See all 36 adverse event totalsPapers3
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 comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
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/NCT02055157
GET https://conductscience.org/api/trials/v1/NCT02055157/registered?page=2