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Trial record 38 of 106 for:    Pompe Disease

BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study)

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ClinicalTrials.gov Identifier: NCT01924845
Recruitment Status : Terminated (Sponsor decision)
First Posted : August 19, 2013
Results First Posted : June 14, 2018
Last Update Posted : June 14, 2018
Sponsor:
Information provided by (Responsible Party):
BioMarin Pharmaceutical

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Late-onset Pompe Disease
Intervention Drug: BMN 701
Enrollment 24
Recruitment Details  
Pre-assignment Details 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.
Arm/Group Title BMN 701 20 mg/kg
Hide Arm/Group Description BMN 701 20 mg/kg
Period Title: Overall Study
Started 24
Completed 18
Not Completed 6
Reason Not Completed
Adverse Event             3
Withdrawal by Subject             2
Protocol Violation             1
Arm/Group Title BMN 701 20 mg/kg
Hide Arm/Group Description BMN 701 20 mg/kg
Overall Number of Baseline Participants 24
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[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 24 participants
47.9  (13.27)
Age, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 24 participants
18-65
22
  91.7%
> 65
2
   8.3%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 24 participants
Female
12
  50.0%
Male
12
  50.0%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 24 participants
Hispanic or Latino
0
   0.0%
Not Hispanic or Latino
24
 100.0%
Unknown or Not Reported
0
   0.0%
Race/Ethnicity, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 24 participants
American Indian or Alaska Native
0
   0.0%
Asian
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
0
   0.0%
White
24
 100.0%
Other
0
   0.0%
Region of Enrollment  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 24 participants
Belgium
1
   4.2%
Germany
9
  37.5%
United Kingdom
7
  29.2%
United States
7
  29.2%
1.Primary Outcome
Title Percent Predicted Maximum Inspiratory Pressure (MIP)
Hide Description Pulmonary function test: Percent Predicted Maximum Inspiratory Pressure
Time Frame Baseline, Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
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 .
Arm/Group Title BMN701 20 mg/kg
Hide Arm/Group Description:
BMN701 20 mg/kg
Overall Number of Participants Analyzed 18
Mean (Standard Deviation)
Unit of Measure: Percent Predicted
Baseline 50.0  (17.5)
Change from Baseline to Week 24 2.2  (8.3)
2.Secondary Outcome
Title Percent Predicted Maximum Expiratory Pressure (MEP)
Hide Description Pulmonary function test: Percent Predicted Maximum Expiratory Pressure
Time Frame Baseline, Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
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 .
Arm/Group Title BMN701 20 mg/kg
Hide Arm/Group Description:
BMN701 20 mg/kg
Overall Number of Participants Analyzed 18
Mean (Standard Deviation)
Unit of Measure: Percent Predicted
Baseline 38.9  (12.3)
Change from Baseline to Week 24 3.1  (8.7)
3.Secondary Outcome
Title 6 Minute Walk Test (Meters)
Hide Description Distance walked within 6 minutes
Time Frame Baseline, Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
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 .
Arm/Group Title BMN701 20 mg/kg
Hide Arm/Group Description:
BMN701 20 mg/kg
Overall Number of Participants Analyzed 17
Mean (Standard Deviation)
Unit of Measure: Meter
Baseline 345.8  (95.3)
Change from Baseline to Week 24 26.1  (40.6)
4.Secondary Outcome
Title Percent Predicted Upright Forced Vital Capacity (FVC)
Hide Description Pulmonary function test: Percent Predicted Upright Forced Vital Capacity
Time Frame Baseline, Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
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 .
Arm/Group Title BMN701 20 mg/kg
Hide Arm/Group Description:
BMN701 20 mg/kg
Overall Number of Participants Analyzed 18
Mean (Standard Deviation)
Unit of Measure: Percent Predicted
Baseline 60.7  (15.1)
Change from Baseline to Week 24 -3.7  (4.4)
5.Secondary Outcome
Title Number of Participants With Non-Serious AEs
Hide Description 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.
Time Frame Baseline through Week 24 +4 weeks follow-up
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set.
Arm/Group Title BMN701 20 mg/kg
Hide Arm/Group Description:
BMN701 20 mg/kg
Overall Number of Participants Analyzed 24
Measure Type: Count of Participants
Unit of Measure: Participants
23
  95.8%
Time Frame 28 weeks (24 weeks trial + 4 weeks follow up)
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title BMN 701 20 mg/kg
Hide Arm/Group Description BMN 701 20 mg/kg
All-Cause Mortality
BMN 701 20 mg/kg
Affected / at Risk (%)
Total   0/24 (0.00%)    
Show Serious Adverse Events Hide Serious Adverse Events
BMN 701 20 mg/kg
Affected / at Risk (%) # Events
Total   10/24 (41.67%)    
Cardiac disorders   
Atrial fibrillation  1  1/24 (4.17%)  1
Gastrointestinal disorders   
Abdominal pain upper  1  1/24 (4.17%)  2
Immune system disorders   
Anaphylactic reaction  1  1/24 (4.17%)  1
Injury, poisoning and procedural complications   
Fall  1  1/24 (4.17%)  2
Investigations   
Blood creatine phosphokinase increased  1  1/24 (4.17%)  1
Metabolism and nutrition disorders   
Hypoglycaemia  1  1/24 (4.17%)  1
Musculoskeletal and connective tissue disorders   
Pain in extremity  1  1/24 (4.17%)  1
Respiratory, thoracic and mediastinal disorders   
Pneumothorax  1  1/24 (4.17%)  1
Respiratory failure  1  1/24 (4.17%)  1
Skin and subcutaneous tissue disorders   
Erythema nodosum  1  1/24 (4.17%)  1
Vascular disorders   
Hypertension  1  1/24 (4.17%)  1
1
Term from vocabulary, MedDRA 19.0
Indicates events were collected by systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
BMN 701 20 mg/kg
Affected / at Risk (%) # Events
Total   23/24 (95.83%)    
Cardiac disorders   
Palpitations  1  4/24 (16.67%)  5
Gastrointestinal disorders   
Abdominal discomfort  1  2/24 (8.33%)  2
Abdominal distension  1  2/24 (8.33%)  2
Abdominal pain upper  1  2/24 (8.33%)  3
Diarrhoea  1  5/24 (20.83%)  8
Nausea  1  8/24 (33.33%)  22
Vomiting  1  4/24 (16.67%)  7
General disorders   
Chest discomfort  1  2/24 (8.33%)  3
Chills  1  4/24 (16.67%)  6
Fatigue  1  5/24 (20.83%)  8
Pain  1  2/24 (8.33%)  2
Infections and infestations   
Lower respiratory tract infection  1  3/24 (12.50%)  4
Nasopharyngitis  1  4/24 (16.67%)  5
Pharyngitis  1  2/24 (8.33%)  2
Upper respiratory tract infection  1  2/24 (8.33%)  2
Viral infection  1  3/24 (12.50%)  3
Injury, poisoning and procedural complications   
Fall  1  5/24 (20.83%)  6
Infusion related reaction  1  2/24 (8.33%)  2
Metabolism and nutrition disorders   
Hypoglycaemia  1  16/24 (66.67%)  165
Musculoskeletal and connective tissue disorders   
Arthralgia  1  2/24 (8.33%)  5
Back pain  1  5/24 (20.83%)  12
Musculoskeletal pain  1  4/24 (16.67%)  4
Myalgia  1  3/24 (12.50%)  5
Neck pain  1  2/24 (8.33%)  2
Pain in extremity  1  5/24 (20.83%)  6
Nervous system disorders   
Dizziness  1  8/24 (33.33%)  12
Headache  1  13/24 (54.17%)  50
Lethargy  1  2/24 (8.33%)  2
Tremor  1  4/24 (16.67%)  5
Psychiatric disorders   
Insomnia  1  2/24 (8.33%)  2
Respiratory, thoracic and mediastinal disorders   
Dyspnoea  1  6/24 (25.00%)  12
Dyspnoea exertional  1  2/24 (8.33%)  4
Nasal congestion  1  4/24 (16.67%)  4
Nasal obstruction  1  2/24 (8.33%)  3
Oropharyngeal pain  1  5/24 (20.83%)  5
Skin and subcutaneous tissue disorders   
Cold sweat  1  2/24 (8.33%)  3
Hyperhidrosis  1  2/24 (8.33%)  2
Pruritus  1  2/24 (8.33%)  4
Rash  1  2/24 (8.33%)  2
Vascular disorders   
Hypotension  1  3/24 (12.50%)  3
1
Term from vocabulary, MedDRA 19.0
Indicates events were collected by systematic assessment
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.
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Debra Lounsbury, Principal Scientist, Clinical Sciences
Organization: BioMarin Pharmaceuticals
Phone: 415-506-6348
EMail: DLounsbury@bmrn.com
Layout table for additonal information
Responsible Party: BioMarin Pharmaceutical
ClinicalTrials.gov Identifier: NCT01924845     History of Changes
Other Study ID Numbers: 701-301
2013-001768-48 ( EudraCT Number )
First Submitted: August 13, 2013
First Posted: August 19, 2013
Results First Submitted: September 19, 2017
Results First Posted: June 14, 2018
Last Update Posted: June 14, 2018