Trial · NCT01609062
Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome
NCT01609062, Phase 2, Terminated, BioMarin Pharmaceutical; results first posted 2016-02-01
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome. Trials, record NCT01609062. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01609062
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome
- NCT number
- NCT01609062
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 25 participants, actual
- Lead sponsor
- BioMarin Pharmaceutical
- Conditions
- Mucopolysaccharidosis IVA, Morquio A Syndrome, MPS IVA
- Interventions
- BMN 110, BMN 110
- First posted
- 2012-05-31
- Last updated
- 2016-02-01
- Results first posted
- 2016-02-01
Source ClinicalTrials.gov
Registered outcomes13
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 192 weeks or ETV (early termination visit) | 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 | 6-minute Walk Test (6MWT) | Baseline, Week 12, 24, and 52 | Change from baseline to Week 12, 24, and 52 as measured in distance walked (meters) in 6MWT. |
| Secondary | 3-minute Stair Climb Test (3MSCT) | Baseline, Week 12, 24, and 52 | Change from baseline to Week 12, 24, and 52 as measured in speed (stairs/min) in 3MSCT. |
| Secondary | Respiratory Function Test (MVV and FVC) | Baseline, Week 12, 24, and 52 | Respiratory Function was assessed by spirometry in accordance with American Thoracic Society standards. Percent change from baseline to Week 12, 24, and 52 as measured by Maximum Voluntary Ventilation (MVV, L/min) Percent change from baseline to Week 12, 24, and 52 as measured by Forced Vital Capacity (FVC, L) |
| Secondary | Normalized Urine Keratan Sulfate (uKS) | Baseline, Week 12, 24, and 52 | Urinary KS was measured by a quantitative method and normalized using the sample urinary creatinine measurement. Percent change from baseline to Week 12, 24, and 52 in normalized urine keratan sulfate (ug/mg). |
| Secondary | Cardiopulmonary Exercise Testing (CPET) - Duration of Exercise | Baseline, Week 25 and 52 | Subjects performed maximal incremental exercise testing using an electronically braked upright cycle ergometer. Cycle ergometry is a method of CPET that may be feasible in subjects who have orthopedic, peripheral vascular, or neurological limitations that restrict weight bearing. Change from baseline to Week 25 and 52 as measured by the CPET Duration of Exercise (min) |
| Secondary | Cardiopulmonary Exercise Testing (CPET) - Peak Workload | Baseline, Week 25 and 52 | Subjects performed maximal incremental exercise testing using an electronically braked upright cycle ergometer. Cycle ergometry is a method of CPET that may be feasible in subjects who have orthopedic, peripheral vascular, or neurological limitations that restrict weight bearing. Percent change from baseline to Week 25 and 52 as measured by the CPET Peak workload (watt) |
| Secondary | Cardiopulmonary Exercise Testing (CPET) - O2 Pulse | Baseline, Week 25 and 52 | Subjects performed maximal incremental exercise testing using an electronically braked upright cycle ergometer. Cycle ergometry is a method of CPET that may be feasible in subjects who have orthopedic, peripheral vascular, or neurological limitations that restrict weight bearing. Percent change from baseline to Week 25 and 52 as measured by the CPET O2 pulse (ml/beat) |
| Secondary | Cardiopulmonary Exercise Testing (CPET) - Aerobic Efficiency | Baseline, Week 25 and 52 | Subjects performed maximal incremental exercise testing using an electronically braked upright cycle ergometer. Cycle ergometry is a method of CPET that may be feasible in subjects who have orthopedic, peripheral vascular, or neurological limitations that restrict weight bearing. Percent change from baseline to Week 25 and 52 as measured by the CPET Aerobic Efficiency (ml/watt). Note that decline in Aerobic Efficiency translate into an improvement |
| Secondary | Muscle Strength Testing (MST) - Knee Extension Test | Baseline, Week 25 and 52 | Change from baseline to Week 25 and 52 as measured by the peak force in MST knee extension test (newton meters). |
1 to 10 of 13Source ClinicalTrials.gov
See all 13 registered outcomesOutcomes13
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 192 weeks or ETV (early termination visit) | The primary outcome measure data is presented in more detail under the Adverse Events section. | participants | NUMBER |
| SECONDARY | 6-minute Walk Test (6MWT) | Baseline, Week 12, 24, and 52 | Modified Intent-to-Treat Analysis Set | meters | MEAN |
| SECONDARY | 3-minute Stair Climb Test (3MSCT) | Baseline, Week 12, 24, and 52 | Modified Intent-to-Treat Analysis Set | stairs/min | MEAN |
| SECONDARY | Respiratory Function Test (MVV and FVC) | Baseline, Week 12, 24, and 52 | Modified Intent-to-treat Analysis Set | Percent Change | MEAN |
| SECONDARY | Normalized Urine Keratan Sulfate (uKS) | Baseline, Week 12, 24, and 52 | Modified Intent-to-treat Analysis Set | Percent Change | MEAN |
| SECONDARY | Cardiopulmonary Exercise Testing (CPET) - Duration of Exercise | Baseline, Week 25 and 52 | A subset of mITT subjects who were scheduled to perform CPET | Percent Change | MEAN |
| SECONDARY | Cardiopulmonary Exercise Testing (CPET) - Peak Workload | Baseline, Week 25 and 52 | A subset of mITT subjects who were scheduled to perform CPET | Percent Change | MEAN |
| SECONDARY | Cardiopulmonary Exercise Testing (CPET) - O2 Pulse | Baseline, Week 25 and 52 | A subset of mITT subjects who were scheduled to perform CPET | Percent Change | MEAN |
| SECONDARY | Cardiopulmonary Exercise Testing (CPET) - Aerobic Efficiency | Baseline, Week 25 and 52 | A subset of mITT subjects who were scheduled to perform CPET | Percent Change | MEAN |
| SECONDARY | Muscle Strength Testing (MST) - Knee Extension Test | Baseline, Week 25 and 52 | Modified Intent-to-treat Analysis Set | Percent Change | MEAN |
1 to 10 of 13Source ClinicalTrials.gov
See all 13 outcomesResults by group102
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 Weekly at 2.0 mg/kg/Week | Serious Adverse Events | 3 | participants | |
| Safety Evaluation | BMN 110 Weekly at 4.0 mg/kg/Week | Serious Adverse Events | 2 | participants | |
| Safety Evaluation | All Subjects | Serious Adverse Events | 5 | participants | |
| Safety Evaluation | BMN 110 Weekly at 2.0 mg/kg/Week | Other (Not Including Serious) Adverse Events | 15 | participants | |
| Safety Evaluation | BMN 110 Weekly at 4.0 mg/kg/Week | Other (Not Including Serious) Adverse Events | 10 | participants | |
| Safety Evaluation | All Subjects | Other (Not Including Serious) Adverse Events | 25 | participants | |
| 6-minute Walk Test (6MWT) | BMN 110 Weekly at 2.0 mg/kg/Week | Week 12 | 14.7 | Standard Deviation 30.90 | meters |
| 6-minute Walk Test (6MWT) | BMN 110 Weekly at 4.0 mg/kg/Week | Week 12 | -8.9 | Standard Deviation 83.52 | meters |
| 6-minute Walk Test (6MWT) | All Subjects | Week 12 | 5.3 | Standard Deviation 57.55 | meters |
| 6-minute Walk Test (6MWT) | BMN 110 Weekly at 2.0 mg/kg/Week | Week 24 | -13.0 | Standard Deviation 54.12 | meters |
1 to 10 of 102Source ClinicalTrials.gov
See all 102 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Primary Phase | STARTED | BMN 110 Weekly at 2.0 mg/kg/Week | 15 | |
| Primary Phase | STARTED | BMN 110 Weekly at 4.0 mg/kg/Week | 10 | |
| Primary Phase | COMPLETED | BMN 110 Weekly at 2.0 mg/kg/Week | 15 | |
| Primary Phase | COMPLETED | BMN 110 Weekly at 4.0 mg/kg/Week | 10 | |
| Primary Phase | NOT COMPLETED | BMN 110 Weekly at 2.0 mg/kg/Week | 0 | |
| Primary Phase | NOT COMPLETED | BMN 110 Weekly at 4.0 mg/kg/Week | 0 | |
| Extension Phase | STARTED | BMN 110 Weekly at 2.0 mg/kg/Week | 15 | |
| Extension Phase | STARTED | BMN 110 Weekly at 4.0 mg/kg/Week | 10 | |
| Extension Phase | COMPLETED | BMN 110 Weekly at 2.0 mg/kg/Week | 0 | |
| Extension Phase | COMPLETED | BMN 110 Weekly at 4.0 mg/kg/Week | 0 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events354
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 | Lymph node pain | Blood and lymphatic system disorders | BMN 110 Weekly at 2.0 mg/kg/Week | 1 | 15 | 1 |
| other | Lymph node pain | Blood and lymphatic system disorders | BMN 110 Weekly at 4.0 mg/kg/Week | 0 | 10 | 0 |
| other | Bradycardia | Cardiac disorders | BMN 110 Weekly at 2.0 mg/kg/Week | 1 | 15 | 1 |
| other | Bradycardia | Cardiac disorders | BMN 110 Weekly at 4.0 mg/kg/Week | 0 | 10 | 0 |
| other | Sinus bradycardia | Cardiac disorders | BMN 110 Weekly at 2.0 mg/kg/Week | 0 | 15 | 0 |
| other | Sinus bradycardia | Cardiac disorders | BMN 110 Weekly at 4.0 mg/kg/Week | 1 | 10 | 1 |
| other | Tachycardia | Cardiac disorders | BMN 110 Weekly at 2.0 mg/kg/Week | 0 | 15 | 0 |
| other | Tachycardia | Cardiac disorders | BMN 110 Weekly at 4.0 mg/kg/Week | 1 | 10 | 2 |
| other | Pectus carinatum | Congenital, familial and genetic disorders | BMN 110 Weekly at 2.0 mg/kg/Week | 1 | 15 | 1 |
| other | Pectus carinatum | Congenital, familial and genetic disorders | BMN 110 Weekly at 4.0 mg/kg/Week | 0 | 10 | 0 |
1 to 10 of 354Source ClinicalTrials.gov
See all 354 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | BMN 110 Weekly at 2.0 mg/kg/Week | ||
| Total, all-cause mortality | BMN 110 Weekly at 4.0 mg/kg/Week | ||
| Total, other adverse events | BMN 110 Weekly at 2.0 mg/kg/Week | 15 | 15 |
| Total, other adverse events | BMN 110 Weekly at 4.0 mg/kg/Week | 10 | 10 |
| Total, serious adverse events | BMN 110 Weekly at 2.0 mg/kg/Week | 3 | 15 |
| Total, serious adverse events | BMN 110 Weekly at 4.0 mg/kg/Week | 2 | 10 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 29159458 | 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 comparisons1
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison |
|---|
| BMN 110 Weekly at 2.0 mg/kg/Week vs BMN 110 Weekly at 4.0 mg/kg/Week: serious adverse events |
Source 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.
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