Trial · NCT01515956
Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)
NCT01515956, Phase 2, Completed, BioMarin Pharmaceutical; results first posted 2017-07-06
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome). Trials, record NCT01515956. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01515956
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)
- NCT number
- NCT01515956
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 15 participants, actual
- Lead sponsor
- BioMarin Pharmaceutical
- Conditions
- Mucopolysaccharidosis IVA, Morquio A Syndrome, MPS IVA
- Interventions
- BMN 110
- First posted
- 2012-01-24
- Last updated
- 2017-08-10
- Results first posted
- 2017-07-06
Source ClinicalTrials.gov
Registered outcomes3
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | To Evaluate Safety and Tolerability of Infusions of BMN 110 at a Dose of 2.0 mg/kg/Week Over a 52-week Period in MPS IVA Subjects Less Than 5 Years of Age at Time of First Study Drug Infusion | 52 weeks | Number of Participants Experiencing Adverse Events |
| Secondary | Percent Change From Baseline to Week 52 in Urinary Keratan Sulfate Measures | Baseline to Week 52 | Percent Change from Baseline to Week 52 for Urinary Keratan Sulfate measures. |
| Secondary | Change From Baseline in Normalized Growth Rate Z-Scores | Baseline to Week 52 | Changes in growth over time will be assessed using anthropometric measurements and radiographs of lower extremities. Z-scores are the normalized scores derived from the reference population mean and standard deviation (A positive change from baseline indicates that the population has moved closer to the reference population and represents a positive outcome). |
Source ClinicalTrials.gov
Outcomes3
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | To Evaluate Safety and Tolerability of Infusions of BMN 110 at a Dose of 2.0 mg/kg/Week Over a 52-week Period in MPS IVA Subjects Less Than 5 Years of Age at Time of First Study Drug Infusion | 52 weeks | Safety Population | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Percent Change From Baseline to Week 52 in Urinary Keratan Sulfate Measures | Baseline to Week 52 | Efficacy Analysis Set - All enrolled patients with at least 1 post treatment efficacy measurement will be included in efficacy analyses. | percentage change | MEAN |
| SECONDARY | Change From Baseline in Normalized Growth Rate Z-Scores | Baseline to Week 52 | Efficacy analysis set - All enrolled patients with at least 1 post treatment efficacy measurement will be included in efficacy analyses. | z-score | MEAN |
Source ClinicalTrials.gov
Results by group4
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| To Evaluate Safety and Tolerability of Infusions of BMN 110 at a Dose of 2.0 mg/kg/Week Over a 52-week Period in MPS IVA Subjects Less Than 5 Years of Age at Time of First Study Drug Infusion | BMN110 2.0 mg/kg/Week | 15 | Participants | ||
| Percent Change From Baseline to Week 52 in Urinary Keratan Sulfate Measures | BMN110 2.0 mg/kg/Week | -44.3 | Standard Deviation 21.15 | percentage change | |
| Change From Baseline in Normalized Growth Rate Z-Scores | BMN110 2.0 mg/kg/Week | Baseline | -0.6 | Standard Deviation 0.64 | z-score |
| Change From Baseline in Normalized Growth Rate Z-Scores | BMN110 2.0 mg/kg/Week | Change from Baseline to Week 52 | 0.2 | Standard Deviation 1.04 | z-score |
Source ClinicalTrials.gov
Participant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | BMN110 2.0 mg/kg/Week | 15 | |
| Overall Study | COMPLETED | BMN110 2.0 mg/kg/Week | 15 | |
| Overall Study | NOT COMPLETED | BMN110 2.0 mg/kg/Week | 0 |
Source ClinicalTrials.gov
Adverse events211
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 | Lymphadenopathy | Blood and lymphatic system disorders | BMN110 2.0 mg/kg/Week | 1 | 15 | 1 |
| other | Tachycardia | Cardiac disorders | BMN110 2.0 mg/kg/Week | 4 | 15 | 9 |
| other | Developmental hip dysplasia | Congenital, familial and genetic disorders | BMN110 2.0 mg/kg/Week | 1 | 15 | 1 |
| other | Aural polyp | Ear and labyrinth disorders | BMN110 2.0 mg/kg/Week | 1 | 15 | 1 |
| other | Cerumen impaction | Ear and labyrinth disorders | BMN110 2.0 mg/kg/Week | 1 | 15 | 1 |
| other | Conductive deafness | Ear and labyrinth disorders | BMN110 2.0 mg/kg/Week | 1 | 15 | 2 |
| other | Deafness | Ear and labyrinth disorders | BMN110 2.0 mg/kg/Week | 2 | 15 | 2 |
| other | Deafness bilateral | Ear and labyrinth disorders | BMN110 2.0 mg/kg/Week | 1 | 15 | 1 |
| other | Ear haemorrhage | Ear and labyrinth disorders | BMN110 2.0 mg/kg/Week | 1 | 15 | 1 |
| other | Ear pain | Ear and labyrinth disorders | BMN110 2.0 mg/kg/Week | 3 | 15 | 17 |
1 to 10 of 211Source ClinicalTrials.gov
See all 211 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | BMN110 2.0 mg/kg/Week | ||
| Total, other adverse events | BMN110 2.0 mg/kg/Week | 15 | 15 |
| Total, serious adverse events | BMN110 2.0 mg/kg/Week | 8 | 15 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 26331768 | DERIVED |
Source 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 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/NCT01515956
GET https://conductscience.org/api/trials/v1/NCT01515956/events?page=2