Trial · NCT03197766

A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia

NCT03197766, Phase 3, Completed, BioMarin Pharmaceutical; results first posted 2022-03-02

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia. Trials, record NCT03197766. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03197766

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
NCT number: NCT03197766
Status: Completed
Phases: Phase 3
Enrollment: 121 participants, actual
Lead sponsor: BioMarin Pharmaceutical
Conditions: Achondroplasia
Interventions: BMN 111, Placebo
First posted: 2017-06-23
Last updated: 2022-03-02
Other registries' IDs: eudract:2015-003836-11
Results first posted: 2022-03-02

Source ClinicalTrials.gov

Registered outcomes4

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Change From Baseline in Annualized Growth Velocity (AGV) at Week 52	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
Secondary	Change From Baseline in Height Z-score at Week 52	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.
Secondary	Change From Baseline in Upper to Lower Segment Body Ratio at Week 52	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
Secondary	Summary of Subjects Experiencing Adverse Events (AEs) During Treatment	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)

Source ClinicalTrials.gov

Outcomes4

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Change From Baseline in Annualized Growth Velocity (AGV) at Week 52	At Baseline and Week 52	Analysis were performed on the Full Analysis Set (FAS) which included all randomized subjects with a signed informed consent	cm/year	LEAST_SQUARES_MEAN
SECONDARY	Change From Baseline in Height Z-score at Week 52	At baseline and Week 52	Analysis were performed on the Full Analysis Set (FAS) which included all randomized subjects with a signed informed consent.	Z-Score	LEAST_SQUARES_MEAN
SECONDARY	Change From Baseline in Upper to Lower Segment Body Ratio at Week 52	At baseline and Week 52	Analysis were performed on the Full Analysis Set (FAS) which included all randomized subjects with a signed informed consent	Ratio	LEAST_SQUARES_MEAN
SECONDARY	Summary of Subjects Experiencing Adverse Events (AEs) During Treatment	Up to Week 56	The Safety Population was defined as all subjects in the FAS who received at least one dose of double-blind BMN 111 or placebo in this study.	participants	NUMBER

Source ClinicalTrials.gov

Results by group14

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Spread	Units
Change From Baseline in Annualized Growth Velocity (AGV) at Week 52	BMN 111 - 15 μg/kg		1.71	95% Confidence Interval 1.40 to 2.01	cm/year
Change From Baseline in Annualized Growth Velocity (AGV) at Week 52	Placebo		0.13	95% Confidence Interval -0.18 to 0.45	cm/year
Change From Baseline in Height Z-score at Week 52	BMN 111 - 15 μg/kg		0.27	95% Confidence Interval 0.18 to 0.36	Z-Score
Change From Baseline in Height Z-score at Week 52	Placebo		-0.01	95% Confidence Interval -0.10 to 0.09	Z-Score
Change From Baseline in Upper to Lower Segment Body Ratio at Week 52	BMN 111 - 15 μg/kg		-0.03	95% Confidence Interval -0.06 to 0.00	Ratio
Change From Baseline in Upper to Lower Segment Body Ratio at Week 52	Placebo		-0.02	95% Confidence Interval -0.05 to 0.01	Ratio
Summary of Subjects Experiencing Adverse Events (AEs) During Treatment	BMN 111 - 15 μg/kg	Subjects with any AE	59		participants
Summary of Subjects Experiencing Adverse Events (AEs) During Treatment	Placebo	Subjects with any AE	60		participants
Summary of Subjects Experiencing Adverse Events (AEs) During Treatment	BMN 111 - 15 μg/kg	Subjects with any SAE	3		participants
Summary of Subjects Experiencing Adverse Events (AEs) During Treatment	Placebo	Subjects with any SAE	4		participants

1 to 10 of 14Source ClinicalTrials.gov

See all 14 results by group

Participant flow6

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	BMN 111 - 15 μg/kg	60
Overall Study	STARTED	Placebo	61
Overall Study	COMPLETED	BMN 111 - 15 μg/kg	58
Overall Study	COMPLETED	Placebo	61
Overall Study	NOT COMPLETED	BMN 111 - 15 μg/kg	2
Overall Study	NOT COMPLETED	Placebo	0

Source ClinicalTrials.gov

Adverse events370

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
other	Injection site reaction	General disorders	BMN 111 - 15 μg/kg	44	60
other	Injection site reaction	General disorders	Placebo	29	61
other	Injection site erythema	General disorders	BMN 111 - 15 μg/kg	41	60
other	Injection site erythema	General disorders	Placebo	40	61
other	Injection site swelling	General disorders	BMN 111 - 15 μg/kg	23	60
other	Injection site swelling	General disorders	Placebo	6	61
other	Pyrexia	General disorders	BMN 111 - 15 μg/kg	10	60
other	Pyrexia	General disorders	Placebo	13	61
other	Injection site urticaria	General disorders	BMN 111 - 15 μg/kg	8	60
other	Injection site urticaria	General disorders	Placebo	2	61

1 to 10 of 370Source ClinicalTrials.gov

See all 370 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	BMN 111 - 15 μg/kg	0	60
Total, all-cause mortality	Placebo	0	61
Total, other adverse events	BMN 111 - 15 μg/kg	59	60
Total, other adverse events	Placebo	60	61
Total, serious adverse events	BMN 111 - 15 μg/kg	3	60
Total, serious adverse events	Placebo	4	61

Source ClinicalTrials.gov

Papers4

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
34431071	DERIVED	PubMed
38649657	DERIVED	PubMed
39305160	DERIVED	PubMed
39740666	DERIVED	PubMed

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
BMN 111 - 15 μg/kg vs Placebo: deaths from any cause	Trial comparisons
BMN 111 - 15 μg/kg 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.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

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/NCT03197766

GET https://conductscience.org/api/trials/v1/NCT03197766/events?page=2

API reference For AI assistants