Trial Outcomes & Findings for BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study) (NCT NCT01924845)
NCT ID: NCT01924845
Last Updated: 2018-06-14
Results Overview
Pulmonary function test: Percent Predicted Maximum Inspiratory Pressure
Recruitment status
TERMINATED
Study phase
PHASE3
Target enrollment
24 participants
Primary outcome timeframe
Baseline, Week 24
Results posted on
2018-06-14
Participant Flow
The study consisted of a 31-day Screening and Baseline Period (26-day Screening Period, a 5-day Baseline/Enrollment Period), a 24-week Treatment Period, and a 30-day Safety Follow-up Period. Following the Screening and Baseline assessments, qualified subjects were enrolled in the study.
Participant milestones
| Measure |
BMN 701 20 mg/kg
BMN 701 20 mg/kg
|
|---|---|
|
Overall Study
STARTED
|
24
|
|
Overall Study
COMPLETED
|
18
|
|
Overall Study
NOT COMPLETED
|
6
|
Reasons for withdrawal
| Measure |
BMN 701 20 mg/kg
BMN 701 20 mg/kg
|
|---|---|
|
Overall Study
Adverse Event
|
3
|
|
Overall Study
Withdrawal by Subject
|
2
|
|
Overall Study
Protocol Violation
|
1
|
Baseline Characteristics
BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study)
Baseline characteristics by cohort
| Measure |
BMN 701 20 mg/kg
n=24 Participants
BMN 701 20 mg/kg
|
|---|---|
|
Age, Continuous
|
47.9 years
STANDARD_DEVIATION 13.27 • n=5 Participants
|
|
Age, Customized
18-65
|
22 Participants
n=5 Participants
|
|
Age, Customized
> 65
|
2 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
12 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
12 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
24 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Asian
|
0 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Black or African American
|
0 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
White
|
24 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Other
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
Belgium
|
1 Participants
n=5 Participants
|
|
Region of Enrollment
Germany
|
9 Participants
n=5 Participants
|
|
Region of Enrollment
United Kingdom
|
7 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
7 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline, Week 24Population: 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 .
Pulmonary function test: Percent Predicted Maximum Inspiratory Pressure
Outcome measures
| Measure |
BMN701 20 mg/kg
n=18 Participants
BMN701 20 mg/kg
|
|---|---|
|
Percent Predicted Maximum Inspiratory Pressure (MIP)
Change from Baseline to Week 24
|
2.2 Percent Predicted
Standard Deviation 8.3
|
|
Percent Predicted Maximum Inspiratory Pressure (MIP)
Baseline
|
50.0 Percent Predicted
Standard Deviation 17.5
|
SECONDARY outcome
Timeframe: Baseline, Week 24Population: 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 .
Pulmonary function test: Percent Predicted Maximum Expiratory Pressure
Outcome measures
| Measure |
BMN701 20 mg/kg
n=18 Participants
BMN701 20 mg/kg
|
|---|---|
|
Percent Predicted Maximum Expiratory Pressure (MEP)
Baseline
|
38.9 Percent Predicted
Standard Deviation 12.3
|
|
Percent Predicted Maximum Expiratory Pressure (MEP)
Change from Baseline to Week 24
|
3.1 Percent Predicted
Standard Deviation 8.7
|
SECONDARY outcome
Timeframe: Baseline, Week 24Population: 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 .
Distance walked within 6 minutes
Outcome measures
| Measure |
BMN701 20 mg/kg
n=17 Participants
BMN701 20 mg/kg
|
|---|---|
|
6 Minute Walk Test (Meters)
Baseline
|
345.8 Meter
Standard Deviation 95.3
|
|
6 Minute Walk Test (Meters)
Change from Baseline to Week 24
|
26.1 Meter
Standard Deviation 40.6
|
SECONDARY outcome
Timeframe: Baseline, Week 24Population: 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 .
Pulmonary function test: Percent Predicted Upright Forced Vital Capacity
Outcome measures
| Measure |
BMN701 20 mg/kg
n=18 Participants
BMN701 20 mg/kg
|
|---|---|
|
Percent Predicted Upright Forced Vital Capacity (FVC)
Baseline
|
60.7 Percent Predicted
Standard Deviation 15.1
|
|
Percent Predicted Upright Forced Vital Capacity (FVC)
Change from Baseline to Week 24
|
-3.7 Percent Predicted
Standard Deviation 4.4
|
SECONDARY outcome
Timeframe: Baseline through Week 24 +4 weeks follow-upPopulation: Full Analysis Set.
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.
Outcome measures
| Measure |
BMN701 20 mg/kg
n=24 Participants
BMN701 20 mg/kg
|
|---|---|
|
Number of Participants With Non-Serious AEs
|
23 Participants
|
Adverse Events
BMN 701 20 mg/kg
Serious events: 10 serious events
Other events: 23 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
BMN 701 20 mg/kg
n=24 participants at risk
BMN 701 20 mg/kg
|
|---|---|
|
Cardiac disorders
Atrial fibrillation
|
4.2%
1/24 • Number of events 1 • 28 weeks (24 weeks trial + 4 weeks follow up)
|
|
Gastrointestinal disorders
Abdominal pain upper
|
4.2%
1/24 • Number of events 2 • 28 weeks (24 weeks trial + 4 weeks follow up)
|
|
Immune system disorders
Anaphylactic reaction
|
4.2%
1/24 • Number of events 1 • 28 weeks (24 weeks trial + 4 weeks follow up)
|
|
Injury, poisoning and procedural complications
Fall
|
4.2%
1/24 • Number of events 2 • 28 weeks (24 weeks trial + 4 weeks follow up)
|
|
Investigations
Blood creatine phosphokinase increased
|
4.2%
1/24 • Number of events 1 • 28 weeks (24 weeks trial + 4 weeks follow up)
|
|
Metabolism and nutrition disorders
Hypoglycaemia
|
4.2%
1/24 • Number of events 1 • 28 weeks (24 weeks trial + 4 weeks follow up)
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
4.2%
1/24 • Number of events 1 • 28 weeks (24 weeks trial + 4 weeks follow up)
|
|
Respiratory, thoracic and mediastinal disorders
Pneumothorax
|
4.2%
1/24 • Number of events 1 • 28 weeks (24 weeks trial + 4 weeks follow up)
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
4.2%
1/24 • Number of events 1 • 28 weeks (24 weeks trial + 4 weeks follow up)
|
|
Skin and subcutaneous tissue disorders
Erythema nodosum
|
4.2%
1/24 • Number of events 1 • 28 weeks (24 weeks trial + 4 weeks follow up)
|
|
Vascular disorders
Hypertension
|
4.2%
1/24 • Number of events 1 • 28 weeks (24 weeks trial + 4 weeks follow up)
|
Other adverse events
| Measure |
BMN 701 20 mg/kg
n=24 participants at risk
BMN 701 20 mg/kg
|
|---|---|
|
Cardiac disorders
Palpitations
|
16.7%
4/24 • Number of events 5 • 28 weeks (24 weeks trial + 4 weeks follow up)
|
|
Gastrointestinal disorders
Abdominal discomfort
|
8.3%
2/24 • Number of events 2 • 28 weeks (24 weeks trial + 4 weeks follow up)
|
|
Gastrointestinal disorders
Abdominal distension
|
8.3%
2/24 • Number of events 2 • 28 weeks (24 weeks trial + 4 weeks follow up)
|
|
Gastrointestinal disorders
Abdominal pain upper
|
8.3%
2/24 • Number of events 3 • 28 weeks (24 weeks trial + 4 weeks follow up)
|
|
Gastrointestinal disorders
Diarrhoea
|
20.8%
5/24 • Number of events 8 • 28 weeks (24 weeks trial + 4 weeks follow up)
|
|
Gastrointestinal disorders
Nausea
|
33.3%
8/24 • Number of events 22 • 28 weeks (24 weeks trial + 4 weeks follow up)
|
|
Gastrointestinal disorders
Vomiting
|
16.7%
4/24 • Number of events 7 • 28 weeks (24 weeks trial + 4 weeks follow up)
|
|
General disorders
Chest discomfort
|
8.3%
2/24 • Number of events 3 • 28 weeks (24 weeks trial + 4 weeks follow up)
|
|
General disorders
Chills
|
16.7%
4/24 • Number of events 6 • 28 weeks (24 weeks trial + 4 weeks follow up)
|
|
General disorders
Fatigue
|
20.8%
5/24 • Number of events 8 • 28 weeks (24 weeks trial + 4 weeks follow up)
|
|
General disorders
Pain
|
8.3%
2/24 • Number of events 2 • 28 weeks (24 weeks trial + 4 weeks follow up)
|
|
Infections and infestations
Lower respiratory tract infection
|
12.5%
3/24 • Number of events 4 • 28 weeks (24 weeks trial + 4 weeks follow up)
|
|
Infections and infestations
Nasopharyngitis
|
16.7%
4/24 • Number of events 5 • 28 weeks (24 weeks trial + 4 weeks follow up)
|
|
Infections and infestations
Pharyngitis
|
8.3%
2/24 • Number of events 2 • 28 weeks (24 weeks trial + 4 weeks follow up)
|
|
Infections and infestations
Upper respiratory tract infection
|
8.3%
2/24 • Number of events 2 • 28 weeks (24 weeks trial + 4 weeks follow up)
|
|
Infections and infestations
Viral infection
|
12.5%
3/24 • Number of events 3 • 28 weeks (24 weeks trial + 4 weeks follow up)
|
|
Injury, poisoning and procedural complications
Fall
|
20.8%
5/24 • Number of events 6 • 28 weeks (24 weeks trial + 4 weeks follow up)
|
|
Injury, poisoning and procedural complications
Infusion related reaction
|
8.3%
2/24 • Number of events 2 • 28 weeks (24 weeks trial + 4 weeks follow up)
|
|
Metabolism and nutrition disorders
Hypoglycaemia
|
66.7%
16/24 • Number of events 165 • 28 weeks (24 weeks trial + 4 weeks follow up)
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
8.3%
2/24 • Number of events 5 • 28 weeks (24 weeks trial + 4 weeks follow up)
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
20.8%
5/24 • Number of events 12 • 28 weeks (24 weeks trial + 4 weeks follow up)
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
16.7%
4/24 • Number of events 4 • 28 weeks (24 weeks trial + 4 weeks follow up)
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
12.5%
3/24 • Number of events 5 • 28 weeks (24 weeks trial + 4 weeks follow up)
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
8.3%
2/24 • Number of events 2 • 28 weeks (24 weeks trial + 4 weeks follow up)
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
20.8%
5/24 • Number of events 6 • 28 weeks (24 weeks trial + 4 weeks follow up)
|
|
Nervous system disorders
Dizziness
|
33.3%
8/24 • Number of events 12 • 28 weeks (24 weeks trial + 4 weeks follow up)
|
|
Nervous system disorders
Headache
|
54.2%
13/24 • Number of events 50 • 28 weeks (24 weeks trial + 4 weeks follow up)
|
|
Nervous system disorders
Lethargy
|
8.3%
2/24 • Number of events 2 • 28 weeks (24 weeks trial + 4 weeks follow up)
|
|
Nervous system disorders
Tremor
|
16.7%
4/24 • Number of events 5 • 28 weeks (24 weeks trial + 4 weeks follow up)
|
|
Psychiatric disorders
Insomnia
|
8.3%
2/24 • Number of events 2 • 28 weeks (24 weeks trial + 4 weeks follow up)
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
25.0%
6/24 • Number of events 12 • 28 weeks (24 weeks trial + 4 weeks follow up)
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea exertional
|
8.3%
2/24 • Number of events 4 • 28 weeks (24 weeks trial + 4 weeks follow up)
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
16.7%
4/24 • Number of events 4 • 28 weeks (24 weeks trial + 4 weeks follow up)
|
|
Respiratory, thoracic and mediastinal disorders
Nasal obstruction
|
8.3%
2/24 • Number of events 3 • 28 weeks (24 weeks trial + 4 weeks follow up)
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
20.8%
5/24 • Number of events 5 • 28 weeks (24 weeks trial + 4 weeks follow up)
|
|
Skin and subcutaneous tissue disorders
Cold sweat
|
8.3%
2/24 • Number of events 3 • 28 weeks (24 weeks trial + 4 weeks follow up)
|
|
Skin and subcutaneous tissue disorders
Hyperhidrosis
|
8.3%
2/24 • Number of events 2 • 28 weeks (24 weeks trial + 4 weeks follow up)
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
8.3%
2/24 • Number of events 4 • 28 weeks (24 weeks trial + 4 weeks follow up)
|
|
Skin and subcutaneous tissue disorders
Rash
|
8.3%
2/24 • Number of events 2 • 28 weeks (24 weeks trial + 4 weeks follow up)
|
|
Vascular disorders
Hypotension
|
12.5%
3/24 • Number of events 3 • 28 weeks (24 weeks trial + 4 weeks follow up)
|
Additional Information
Debra Lounsbury, Principal Scientist, Clinical Sciences
BioMarin Pharmaceuticals
Phone: 415-506-6348
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place