Trial · NCT01924845
BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study)
NCT01924845, Phase 3, Terminated, BioMarin Pharmaceutical; results first posted 2018-06-14
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study). Trials, record NCT01924845. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01924845
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study)
- NCT number
- NCT01924845
- Status
- Terminated
- Phases
- Phase 3
- Enrollment
- 24 participants, actual
- Lead sponsor
- BioMarin Pharmaceutical
- Conditions
- Late-onset Pompe Disease
- Interventions
- BMN 701
- First posted
- 2013-08-19
- Last updated
- 2018-06-14
- Other registries' IDs
- eudract:2013-001768-48
- Results first posted
- 2018-06-14
- Limitations and caveats
- Complete efficacy analyses were not able to be completed due to early study termination; however, efficacy results from interim analysis at week 24 are available, and thus have been reported.
Source ClinicalTrials.gov
Registered outcomes5
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percent Predicted Maximum Inspiratory Pressure (MIP) | Baseline, Week 24 | Pulmonary function test: Percent Predicted Maximum Inspiratory Pressure |
| Secondary | Percent Predicted Maximum Expiratory Pressure (MEP) | Baseline, Week 24 | Pulmonary function test: Percent Predicted Maximum Expiratory Pressure |
| Secondary | 6 Minute Walk Test (Meters) | Baseline, Week 24 | Distance walked within 6 minutes |
| Secondary | Percent Predicted Upright Forced Vital Capacity (FVC) | Baseline, Week 24 | Pulmonary function test: Percent Predicted Upright Forced Vital Capacity |
| Secondary | Number of Participants With Non-Serious AEs | Baseline through Week 24 +4 weeks follow-up | Number of participants with non-serious Adverse Events. Data is taken at final time point of Week 24, compared to baseline. For full AE data, please see AE section. |
Source ClinicalTrials.gov
Outcomes5
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percent Predicted Maximum Inspiratory Pressure (MIP) | Baseline, Week 24 | Full Analysis Set. Please note the Overall Number of Participants reflects the number of patients in the Analysis Population, while the Number Analyzed of patients at specific visit in the Outcome Measure Table reflects the participants who had data at that visit . | Percent Predicted | MEAN |
| SECONDARY | Percent Predicted Maximum Expiratory Pressure (MEP) | Baseline, Week 24 | Full Analysis Set. Please note the Overall Number of Participants reflects the number of patients in the Analysis Population, while the Number Analyzed of patients at specific visit in the Outcome Measure Table reflects the participants who had data at that visit . | Percent Predicted | MEAN |
| SECONDARY | 6 Minute Walk Test (Meters) | Baseline, Week 24 | Full Analysis Set. Please note the Overall Number of Participants reflects the number of patients in the Analysis Population, while the Number Analyzed of patients at specific visit in the Outcome Measure Table reflects the participants who had data at that visit . | Meter | MEAN |
| SECONDARY | Percent Predicted Upright Forced Vital Capacity (FVC) | Baseline, Week 24 | Full Analysis Set. Please note the Overall Number of Participants reflects the number of patients in the Analysis Population, while the Number Analyzed of patients at specific visit in the Outcome Measure Table reflects the participants who had data at that visit . | Percent Predicted | MEAN |
| SECONDARY | Number of Participants With Non-Serious AEs | Baseline through Week 24 +4 weeks follow-up | Full Analysis Set. | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group9
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percent Predicted Maximum Inspiratory Pressure (MIP) | BMN701 20 mg/kg | Baseline | 50.0 | Standard Deviation 17.5 | Percent Predicted |
| Percent Predicted Maximum Inspiratory Pressure (MIP) | BMN701 20 mg/kg | Change from Baseline to Week 24 | 2.2 | Standard Deviation 8.3 | Percent Predicted |
| Percent Predicted Maximum Expiratory Pressure (MEP) | BMN701 20 mg/kg | Baseline | 38.9 | Standard Deviation 12.3 | Percent Predicted |
| Percent Predicted Maximum Expiratory Pressure (MEP) | BMN701 20 mg/kg | Change from Baseline to Week 24 | 3.1 | Standard Deviation 8.7 | Percent Predicted |
| 6 Minute Walk Test (Meters) | BMN701 20 mg/kg | Baseline | 345.8 | Standard Deviation 95.3 | Meter |
| 6 Minute Walk Test (Meters) | BMN701 20 mg/kg | Change from Baseline to Week 24 | 26.1 | Standard Deviation 40.6 | Meter |
| Percent Predicted Upright Forced Vital Capacity (FVC) | BMN701 20 mg/kg | Baseline | 60.7 | Standard Deviation 15.1 | Percent Predicted |
| Percent Predicted Upright Forced Vital Capacity (FVC) | BMN701 20 mg/kg | Change from Baseline to Week 24 | -3.7 | Standard Deviation 4.4 | Percent Predicted |
| Number of Participants With Non-Serious AEs | BMN701 20 mg/kg | 23 | Participants |
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 | BMN 701 20 mg/kg | 24 | |
| Overall Study | COMPLETED | BMN 701 20 mg/kg | 18 | |
| Overall Study | NOT COMPLETED | BMN 701 20 mg/kg | 6 |
Source ClinicalTrials.gov
Adverse events51
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 | Palpitations | Cardiac disorders | BMN 701 20 mg/kg | 4 | 24 | 5 |
| other | Abdominal discomfort | Gastrointestinal disorders | BMN 701 20 mg/kg | 2 | 24 | 2 |
| other | Abdominal distension | Gastrointestinal disorders | BMN 701 20 mg/kg | 2 | 24 | 2 |
| other | Abdominal pain upper | Gastrointestinal disorders | BMN 701 20 mg/kg | 2 | 24 | 3 |
| other | Diarrhoea | Gastrointestinal disorders | BMN 701 20 mg/kg | 5 | 24 | 8 |
| other | Nausea | Gastrointestinal disorders | BMN 701 20 mg/kg | 8 | 24 | 22 |
| other | Vomiting | Gastrointestinal disorders | BMN 701 20 mg/kg | 4 | 24 | 7 |
| other | Chest discomfort | General disorders | BMN 701 20 mg/kg | 2 | 24 | 3 |
| other | Chills | General disorders | BMN 701 20 mg/kg | 4 | 24 | 6 |
| other | Fatigue | General disorders | BMN 701 20 mg/kg | 5 | 24 | 8 |
1 to 10 of 51Source ClinicalTrials.gov
See all 51 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | BMN 701 20 mg/kg | 0 | 24 |
| Total, other adverse events | BMN 701 20 mg/kg | 23 | 24 |
| Total, serious adverse events | BMN 701 20 mg/kg | 10 | 24 |
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/NCT01924845
GET https://conductscience.org/api/trials/v1/NCT01924845/events?page=2