Trial · NCT01230801
Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease
NCT01230801, Phase 1, Phase 2, Completed, BioMarin Pharmaceutical; results first posted 2018-06-11
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease. Trials, record NCT01230801. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01230801
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease
- NCT number
- NCT01230801
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 22 participants, actual
- Lead sponsor
- BioMarin Pharmaceutical
- Conditions
- Pompe Disease
- Interventions
- BMN 701
- First posted
- 2010-10-29
- Last updated
- 2018-06-11
- Other registries' IDs
- eudract:2010-023561-22
- Results first posted
- 2018-06-11
Source ClinicalTrials.gov
Registered outcomes7
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Adverse Events | 24 weeks | Number of Participants with Adverse Events as a Measure of Safety and Tolerability |
| Secondary | Change From Baseline in Six Minutes Walk Test | Baseline up to 24 weeks | Change from Baseline in Six Minutes Walk Test. The 6MWT measured the maximum distance the subject could walk on a flat, hard surface in a period of 6 minutes |
| Other | Change From Baseline in Percent Predicted Upright Forced Vital Capacity | Baseline up to 24 week | Change from Baseline in Percent Predicted Upright Forced Vital Capacity. Changes in respiratory function were assessed by measurement of MEP, MIP and MVV; and percent predicted upright and supine FVC. |
| Other | Change From Baseline in Percent Predicted Supine Forced Vital Capacity | Baseline up to 24 weeks | Change from Baseline in Percent Predicted Supine Forced Vital Capacity. Changes in respiratory function were assessed by measurement of MEP, MIP and MVV; and percent predicted upright and supine FVC. |
| Other | Change From Baseline in Percent Predicted Upright Maximum Expiratory Pressure | Baseline up to 24 weeks | Change from Baseline in Percent Predicted Upright Maximum Expiratory Pressure. Changes in respiratory function were assessed by measurement of MEP, MIP and MVV; and percent predicted upright and supine FVC. |
| Other | Change From Baseline in Percent Predicted Upright Maximum Inspiratory Pressure | Baseline up to 24 weeks | Change from Baseline in Percent Predicted Upright Maximum Inspiratory Pressure. Changes in respiratory function were assessed by measurement of MEP, MIP and MVV; and percent predicted upright and supine FVC. |
| Other | Change From Baseline in Upright Maximum Ventilatory Volume | Baseline up to 24 weeks | Change from Baseline in Upright Maximum Ventilatory Volume. Changes in respiratory function were assessed by measurement of MEP, MIP and MVV; and percent predicted upright and supine FVC. |
Source ClinicalTrials.gov
Outcomes7
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With Adverse Events | 24 weeks | Safety Population | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline in Six Minutes Walk Test | Baseline up to 24 weeks | ITT Population. As some patients did not attend or all tests were not completed at each visit, some time points had different numbers of patients analyzed. The total number of patients analyzed for each arm is listed as the maximum patients analyzed at any given time point. | meter | MEAN |
| OTHER_PRE_SPECIFIED | Change From Baseline in Percent Predicted Upright Forced Vital Capacity | Baseline up to 24 week | ITT Population. As some patients did not attend or all tests were not completed at each visit, some time points had different numbers of patients analyzed. The total number of patients analyzed for each arm is listed as the maximum patients analyzed at any given time point. | percentage of Predicted Upright FVC | MEAN |
| OTHER_PRE_SPECIFIED | Change From Baseline in Percent Predicted Supine Forced Vital Capacity | Baseline up to 24 weeks | ITT Population. As some patients did not attend or all tests were not completed at each visit, some time points had different numbers of patients analyzed. The total number of patients analyzed for each arm is listed as the maximum patients analyzed at any given time point. | percentage of Predicted Supine FVC | MEAN |
| OTHER_PRE_SPECIFIED | Change From Baseline in Percent Predicted Upright Maximum Expiratory Pressure | Baseline up to 24 weeks | ITT Population. As some patients did not attend or all tests were not completed at each visit, some time points had different numbers of patients analyzed. The total number of patients analyzed for each arm is listed as the maximum patients analyzed at any given time point. No patient had baseline assessment in group of BMN 701 5 mg/kg. | percentage of Predicted MEP | MEAN |
| OTHER_PRE_SPECIFIED | Change From Baseline in Percent Predicted Upright Maximum Inspiratory Pressure | Baseline up to 24 weeks | ITT Population. As some patients did not attend or all tests were not completed at each visit, some time points had different numbers of patients analyzed. The total number of patients analyzed for each arm is listed as the maximum patients analyzed at any given time point. No patient had baseline assessment in group of BMN 701 5 mg/kg. | percentage of Predicted MIP | MEAN |
| OTHER_PRE_SPECIFIED | Change From Baseline in Upright Maximum Ventilatory Volume | Baseline up to 24 weeks | ITT Population. As some patients did not attend or all tests were not completed at each visit, some time points had different numbers of patients analyzed. The total number of patients analyzed for each arm is listed as the maximum patients analyzed at any given time point. No patient had baseline assessment in group of BMN 701 5 mg/kg. | L/min | MEAN |
Source ClinicalTrials.gov
Results by group108
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Adverse Events | BMN 701 5 mg/kg | 3 | Participants | ||
| Number of Participants With Adverse Events | BMN 701 10 mg/kg | 3 | Participants | ||
| Number of Participants With Adverse Events | BMN 701 20 mg/kg | 15 | Participants | ||
| Change From Baseline in Six Minutes Walk Test | BMN 701 5 mg/kg | Baseline | 334 | Standard Deviation 227.12 | meter |
| Change From Baseline in Six Minutes Walk Test | BMN 701 10 mg/kg | Baseline | 360 | Standard Deviation 51.4 | meter |
| Change From Baseline in Six Minutes Walk Test | BMN 701 20 mg/kg | Baseline | 354.5 | Standard Deviation 156.94 | meter |
| Change From Baseline in Six Minutes Walk Test | Total | Baseline | 352.5 | Standard Deviation 151.05 | meter |
| Change From Baseline in Six Minutes Walk Test | BMN 701 5 mg/kg | Change from Baseline - Week 6 | -2.5 | Standard Deviation 23.33 | meter |
| Change From Baseline in Six Minutes Walk Test | BMN 701 10 mg/kg | Change from Baseline - Week 6 | -30.3 | Standard Deviation 56.50 | meter |
| Change From Baseline in Six Minutes Walk Test | BMN 701 20 mg/kg | Change from Baseline - Week 6 | 19.8 | Standard Deviation 35.11 | meter |
1 to 10 of 108Source ClinicalTrials.gov
See all 108 results by groupParticipant flow9
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | BMN 701 5 mg/kg | 3 | |
| Overall Study | STARTED | BMN 701 10 mg/kg | 3 | |
| Overall Study | STARTED | BMN 701 20 mg/kg | 16 | |
| Overall Study | COMPLETED | BMN 701 5 mg/kg | 3 | |
| Overall Study | COMPLETED | BMN 701 10 mg/kg | 3 | |
| Overall Study | COMPLETED | BMN 701 20 mg/kg | 15 | |
| Overall Study | NOT COMPLETED | BMN 701 5 mg/kg | 0 | |
| Overall Study | NOT COMPLETED | BMN 701 10 mg/kg | 0 | |
| Overall Study | NOT COMPLETED | BMN 701 20 mg/kg | 1 |
Source ClinicalTrials.gov
Adverse events508
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 | Bradycardia | Cardiac disorders | BMN 701 5 mg/kg | 0 | 3 | 0 |
| other | Bradycardia | Cardiac disorders | BMN 701 10 mg/kg | 0 | 3 | 0 |
| other | Bradycardia | Cardiac disorders | BMN 701 20 mg/kg | 1 | 16 | 1 |
| other | Bradycardia | Cardiac disorders | Total | 1 | 22 | 1 |
| other | Bundle branch block right | Cardiac disorders | BMN 701 5 mg/kg | 0 | 3 | 0 |
| other | Bundle branch block right | Cardiac disorders | BMN 701 10 mg/kg | 0 | 3 | 0 |
| other | Bundle branch block right | Cardiac disorders | BMN 701 20 mg/kg | 1 | 16 | 1 |
| other | Bundle branch block right | Cardiac disorders | Total | 1 | 22 | 1 |
| other | Palpitations | Cardiac disorders | BMN 701 5 mg/kg | 0 | 3 | 0 |
| other | Palpitations | Cardiac disorders | BMN 701 10 mg/kg | 1 | 3 | 1 |
1 to 10 of 508Source ClinicalTrials.gov
See all 508 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | BMN 701 5 mg/kg | 0 | 3 |
| Total, all-cause mortality | BMN 701 10 mg/kg | 0 | 3 |
| Total, all-cause mortality | BMN 701 20 mg/kg | 0 | 16 |
| Total, all-cause mortality | Total | 0 | 22 |
| Total, other adverse events | BMN 701 5 mg/kg | 3 | 3 |
| Total, other adverse events | BMN 701 10 mg/kg | 3 | 3 |
| Total, other adverse events | BMN 701 20 mg/kg | 15 | 16 |
| Total, other adverse events | Total | 21 | 22 |
| Total, serious adverse events | BMN 701 5 mg/kg | 0 | 3 |
| Total, serious adverse events | BMN 701 10 mg/kg | 0 | 3 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers0
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/NCT01230801
GET https://conductscience.org/api/trials/v1/NCT01230801/results?page=2