Trial · NCT01697319
Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation
NCT01697319, Phase 2, Terminated, BioMarin Pharmaceutical; results first posted 2016-01-12
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation. Trials, record NCT01697319. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01697319
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation
- NCT number
- NCT01697319
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 16 participants, actual
- Lead sponsor
- BioMarin Pharmaceutical
- Conditions
- Mucopolysaccharidosis IVA, Morquio A Syndrome, MPS IVA
- Interventions
- BMN 110
- First posted
- 2012-10-02
- Last updated
- 2016-01-12
- Other registries' IDs
- eudract:2011-005703-33
- Results first posted
- 2016-01-12
Source ClinicalTrials.gov
Registered outcomes4
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percent Change From Baseline in Speed as Measured in Functional Dexterity Test (FDT) | Up to 96 weeks | FDT assesses the ability to use the hand in daily tasks. The test involves turning 16 wooden pegs over as quickly as possible on a hardwood pegboard with one hand requiring a three-jaw chuck prehension pattern between the fingers and thumb within a two-minute time limit. Hand function is evaluated by how fast a patient can turn over pegs in the given time limit, i.e. speed (number of pegs/minute). |
| Primary | Change From Baseline in Strength as Assessed by Grip and Pinch Test (GPT) | Up to 96 weeks | A grip-strength dynamometer and a pinch meter were used to measure grip strength and pinch strength. The results report change from baseline in strength for dominant and non-dominant hand in a forearm and wrist supported position. |
| Primary | Percent Change From Baseline in Speed as Measured in Timed 25-Foot Walk Test (25FWT) | Up to 96 weeks | The timed 25-Foot Walk Test (25FWT) is an assessment of mobility and performance of leg function. The patient was instructed to walk a marked 25-foot course as quickly as possible in a time limit of 3 minutes and immediately walk back the same distance when reaching one end.The patient is allowed to use any ambulation method to move. The outcome measures the speed (feet / min) of moving. |
| Secondary | Percent Change From Baseline in Normalized Urine Keratan Sulfate (uKS) | Up to 96 weeks | Urinary keratan sulfate and urinary creatinine were measured through quantitative analysis. uKS is normalized to creatinine. |
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 | Percent Change From Baseline in Speed as Measured in Functional Dexterity Test (FDT) | Up to 96 weeks | Modified ITT Population | % of change | MEAN |
| SECONDARY | Percent Change From Baseline in Normalized Urine Keratan Sulfate (uKS) | Up to 96 weeks | Modified ITT Population | % of change | MEAN |
| PRIMARY | Change From Baseline in Strength as Assessed by Grip and Pinch Test (GPT) | Up to 96 weeks | Modified ITT Population | kg | MEAN |
| PRIMARY | Percent Change From Baseline in Speed as Measured in Timed 25-Foot Walk Test (25FWT) | Up to 96 weeks | Modified ITT Population | % of change | MEAN |
Source ClinicalTrials.gov
Results by group32
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percent Change From Baseline in Speed as Measured in Functional Dexterity Test (FDT) | BMN 110 at 2.0 mg/kg/Week | Dominant Hand Wk 24 % Chg from Baseline | 1.8 | Standard Deviation 45.60 | % of change |
| Percent Change From Baseline in Speed as Measured in Functional Dexterity Test (FDT) | BMN 110 at 2.0 mg/kg/Week | Dominant Hand Wk 48 % Chg from Baseline | 23.1 | Standard Deviation 53.88 | % of change |
| Percent Change From Baseline in Speed as Measured in Functional Dexterity Test (FDT) | BMN 110 at 2.0 mg/kg/Week | Dominant Hand Wk 72 % Chg from Baseline | 37.2 | Standard Deviation 54.94 | % of change |
| Percent Change From Baseline in Speed as Measured in Functional Dexterity Test (FDT) | BMN 110 at 2.0 mg/kg/Week | Dominant Hand Wk 96 % Chg from Baseline | 22.5 | Standard Deviation 42.21 | % of change |
| Percent Change From Baseline in Speed as Measured in Functional Dexterity Test (FDT) | BMN 110 at 2.0 mg/kg/Week | Non-Dominant Hand Wk 24 % Chg from Baseline | 13.8 | Standard Deviation 32.54 | % of change |
| Percent Change From Baseline in Speed as Measured in Functional Dexterity Test (FDT) | BMN 110 at 2.0 mg/kg/Week | Non-Dominant Hand Wk 48 % Chg from Baseline | 6.0 | Standard Deviation 35.78 | % of change |
| Percent Change From Baseline in Speed as Measured in Functional Dexterity Test (FDT) | BMN 110 at 2.0 mg/kg/Week | Non-Dominant Hand Wk 72 % Chg from Baseline | 49.3 | Standard Deviation 126.26 | % of change |
| Percent Change From Baseline in Speed as Measured in Functional Dexterity Test (FDT) | BMN 110 at 2.0 mg/kg/Week | Non-Dominant Hand Wk 96 % Chg from Baseline | -2.0 | Standard Deviation 2.83 | % of change |
| Percent Change From Baseline in Normalized Urine Keratan Sulfate (uKS) | BMN 110 at 2.0 mg/kg/Week | Week 24 % Change from Baseline | -47.7 | Standard Deviation 15.40 | % of change |
| Percent Change From Baseline in Normalized Urine Keratan Sulfate (uKS) | BMN 110 at 2.0 mg/kg/Week | Week 48 % Change from Baseline | -43.4 | Standard Deviation 24.82 | % of change |
1 to 10 of 32Source ClinicalTrials.gov
See all 32 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Primary Phase | STARTED | BMN 110 at 2.0 mg/kg/Week | 16 | |
| Primary Phase | COMPLETED | BMN 110 at 2.0 mg/kg/Week | 15 | |
| Primary Phase | NOT COMPLETED | BMN 110 at 2.0 mg/kg/Week | 1 | |
| Extension Phase | STARTED | BMN 110 at 2.0 mg/kg/Week | 15 | |
| Extension Phase | COMPLETED | BMN 110 at 2.0 mg/kg/Week | 0 | |
| Extension Phase | NOT COMPLETED | BMN 110 at 2.0 mg/kg/Week | 15 |
Source ClinicalTrials.gov
Adverse events154
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 | Atrioventricular block first degree | Cardiac disorders | BMN 110 at 2.0 mg/kg/Week | 1 | 15 | 1 |
| other | Sinus tachycardia | Cardiac disorders | BMN 110 at 2.0 mg/kg/Week | 1 | 15 | 1 |
| other | Tachycardia | Cardiac disorders | BMN 110 at 2.0 mg/kg/Week | 2 | 15 | 2 |
| other | Ear discomfort | Ear and labyrinth disorders | BMN 110 at 2.0 mg/kg/Week | 2 | 15 | 2 |
| other | Ear disorder | Ear and labyrinth disorders | BMN 110 at 2.0 mg/kg/Week | 1 | 15 | 1 |
| other | Ear pain | Ear and labyrinth disorders | BMN 110 at 2.0 mg/kg/Week | 2 | 15 | 3 |
| other | Otorrhoea | Ear and labyrinth disorders | BMN 110 at 2.0 mg/kg/Week | 1 | 15 | 2 |
| other | Corneal opacity | Eye disorders | BMN 110 at 2.0 mg/kg/Week | 1 | 15 | 1 |
| other | Dry eye | Eye disorders | BMN 110 at 2.0 mg/kg/Week | 1 | 15 | 1 |
| other | Eye disorder | Eye disorders | BMN 110 at 2.0 mg/kg/Week | 1 | 15 | 1 |
1 to 10 of 154Source ClinicalTrials.gov
See all 154 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | BMN 110 at 2.0 mg/kg/Week | ||
| Total, other adverse events | BMN 110 at 2.0 mg/kg/Week | 15 | 15 |
| Total, serious adverse events | BMN 110 at 2.0 mg/kg/Week | 7 | 15 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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/NCT01697319
GET https://conductscience.org/api/trials/v1/NCT01697319/results?page=2