Trial · NCT01242111
A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
NCT01242111, Phase 1, Phase 2, Terminated, BioMarin Pharmaceutical; results first posted 2015-09-30
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome). Trials, record NCT01242111. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01242111
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
- NCT number
- NCT01242111
- Status
- Terminated
- Phases
- Phase 1, Phase 2
- Enrollment
- 20 participants, actual
- Lead sponsor
- BioMarin Pharmaceutical
- Conditions
- MPS IV A, Mucopolysaccharidosis IVA, Morquio A Syndrome
- Interventions
- BMN 110
- First posted
- 2010-11-16
- Last updated
- 2015-09-30
- Results first posted
- 2015-09-30
Source ClinicalTrials.gov
Registered outcomes6
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Safety Evaluation | Entire Study Period, up to 240 weeks | The primary objective of the study is to evaluate the safety of weekly infusions of BMN 110; the safety variables included Adverse Events (AEs). The primary outcome measure data is presented in more detail under the Adverse Events section. |
| Secondary | Change From Baseline in Endurance as Measured by the 6-minute Walk Test During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | Baseline and every 12 weeks for up to 72 weeks during the MOR-002 pilot trial and every 24 weeks for up to 192 weeks during the MOR-100 extension trial | Change from baseline in meters in 6-minute Walk Test. As a measure of endurance, a 6-minute walk test (6MWT) was performed according to the American Thoracic Society Guidelines. Patients were instructed to walk as far as possible in 6 minutes. |
| Secondary | Change in Baseline in Endurance as Measured by the 3 Minute Stair Climb During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | Baseline and every 12 weeks for up to 72 weeks during the MOR-002 pilot trial and every 24 weeks for up to 192 weeks during the MOR-100 extension trial | Change from baseline in the 3-minute Stair Climb Test (3MSCT). Patients walked up stairs that have a railing, which could be used for support, for 3 minutes, with the number of stairs climbed recorded. The test result was the number of steps climbed per minute. |
| Secondary | Percent Change From Baseline in Urine Keratan Sulfate (uKS) Levels During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | Baseline and every 12 weeks for up to 72 weeks during the MOR-002 pilot trial and every 24 weeks for up to 168 weeks during the MOR-100 extension trial | Percent Change from baseline in Normalized Urine KS. The percent change was calculated (Week X value - baseline value)/baseline value \*100% |
| Secondary | Percent Change From Baseline in Respiratory Function Test MVV During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | Baseline and every 12 weeks for up to 72 weeks during the MOR-002 pilot trial and every 24 weeks for up to 192 weeks during the MOR-100 extension trial | Percent Change from baseline in Maximum Voluntary Ventilation. |
| Secondary | Percent Change From Baseline in Respiratory Function Test FVC During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | Baseline and every 12 weeks for up to 72 weeks during the MOR-002 pilot trial and every 24 weeks for up to 192 weeks during the MOR-100 extension trial | Percent Change from baseline in Forced Vital Capacity. |
Source ClinicalTrials.gov
Outcomes6
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Safety Evaluation | Entire Study Period, up to 240 weeks | The primary outcome measure data is presented in more detail under the Adverse Events section. | participants | NUMBER |
| SECONDARY | Change From Baseline in Endurance as Measured by the 6-minute Walk Test During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | Baseline and every 12 weeks for up to 72 weeks during the MOR-002 pilot trial and every 24 weeks for up to 192 weeks during the MOR-100 extension trial | Intent-to-Treat population (all subjects who enrolled in the study). Two patients were either physically (score was designated as 0 m) or developmentally (score was set to missing) unable to perform the 6MWT. The analysis was based on observed cases. | meters | MEAN |
| SECONDARY | Change in Baseline in Endurance as Measured by the 3 Minute Stair Climb During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | Baseline and every 12 weeks for up to 72 weeks during the MOR-002 pilot trial and every 24 weeks for up to 192 weeks during the MOR-100 extension trial | Intent-to-Treat population (all subjects who enrolled in the study). One patient was developmentally unable to perform the 3MSCT and the test scores were set to missing. The analysis was based on observed cases. | steps/min | MEAN |
| SECONDARY | Percent Change From Baseline in Urine Keratan Sulfate (uKS) Levels During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | Baseline and every 12 weeks for up to 72 weeks during the MOR-002 pilot trial and every 24 weeks for up to 168 weeks during the MOR-100 extension trial | Intent-to-Treat population (all subjects who enrolled in the study). The analysis was based on observed cases. | percentage change | MEAN |
| SECONDARY | Percent Change From Baseline in Respiratory Function Test MVV During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | Baseline and every 12 weeks for up to 72 weeks during the MOR-002 pilot trial and every 24 weeks for up to 192 weeks during the MOR-100 extension trial | Intent-to-Treat population (all subjects who enrolled in the study). The analysis was based on observed cases. | percentage change | MEAN |
| SECONDARY | Percent Change From Baseline in Respiratory Function Test FVC During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | Baseline and every 12 weeks for up to 72 weeks during the MOR-002 pilot trial and every 24 weeks for up to 192 weeks during the MOR-100 extension trial | Intent-to-Treat population (all subjects who enrolled in the study). The analysis was based on observed cases. | percentage change | MEAN |
Source ClinicalTrials.gov
Results by group64
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Safety Evaluation | BMN 110 | Serious Adverse Events | 19 | participants | |
| Safety Evaluation | BMN 110 | Other (Not Including Serious) Adverse Events | 20 | participants | |
| Change From Baseline in Endurance as Measured by the 6-minute Walk Test During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | BMN 110 | MOR-002 Week 12 Change from Baseline (n=19) | -20.7 | Standard Deviation 85.95 | meters |
| Change From Baseline in Endurance as Measured by the 6-minute Walk Test During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | BMN 110 | MOR-002 Week 24 Change from Baseline (n=17) | 16.3 | Standard Deviation 71.74 | meters |
| Change From Baseline in Endurance as Measured by the 6-minute Walk Test During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | BMN 110 | MOR-002 Week 36 Change from Baseline (n=17) | 13.8 | Standard Deviation 63.25 | meters |
| Change From Baseline in Endurance as Measured by the 6-minute Walk Test During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | BMN 110 | MOR-002 Week 48 Change from Baseline (n=17) | -4.8 | Standard Deviation 64.70 | meters |
| Change From Baseline in Endurance as Measured by the 6-minute Walk Test During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | BMN 110 | MOR-002 Week 72 Change from Baseline (n=17) | 4.0 | Standard Deviation 87.24 | meters |
| Change From Baseline in Endurance as Measured by the 6-minute Walk Test During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | BMN 110 | MOR-100 Week 0 Change from Baseline (n=16) | 15.7 | Standard Deviation 89.00 | meters |
| Change From Baseline in Endurance as Measured by the 6-minute Walk Test During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | BMN 110 | MOR-100 Week 24 Change from Baseline (n=16) | 24.5 | Standard Deviation 101.23 | meters |
| Change From Baseline in Endurance as Measured by the 6-minute Walk Test During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | BMN 110 | MOR-100 Week 48 Change from Baseline (n=16) | 6.8 | Standard Deviation 98.66 | meters |
1 to 10 of 64Source ClinicalTrials.gov
See all 64 results by groupParticipant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | BMN 110 | 20 | |
| Overall Study | COMPLETED | BMN 110 | 17 | |
| Overall Study | NOT COMPLETED | BMN 110 | 3 |
Source ClinicalTrials.gov
Adverse events344
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 | Eosinophilia | Blood and lymphatic system disorders | BMN 110 | 1 | 20 | 1 |
| other | Lymphadenopathy | Blood and lymphatic system disorders | BMN 110 | 4 | 20 | 5 |
| other | Aortic valve incompetence | Cardiac disorders | BMN 110 | 3 | 20 | 3 |
| other | Diastolic dysfunction | Cardiac disorders | BMN 110 | 1 | 20 | 1 |
| other | Mitral valve disease | Cardiac disorders | BMN 110 | 2 | 20 | 2 |
| other | Mitral valve incompetence | Cardiac disorders | BMN 110 | 3 | 20 | 3 |
| other | Pulmonary valve incompetence | Cardiac disorders | BMN 110 | 3 | 20 | 3 |
| other | Tachycardia | Cardiac disorders | BMN 110 | 3 | 20 | 6 |
| other | Tricuspid valve incompetence | Cardiac disorders | BMN 110 | 3 | 20 | 3 |
| other | Congenital heart valve disorder | Congenital, familial and genetic disorders | BMN 110 | 1 | 20 | 1 |
1 to 10 of 344Source ClinicalTrials.gov
See all 344 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | BMN 110 | ||
| Total, other adverse events | BMN 110 | 20 | 20 |
| Total, serious adverse events | BMN 110 | 19 | 20 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 29526614 | 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/NCT01242111
GET https://conductscience.org/api/trials/v1/NCT01242111/results?page=2