Extension Study for Patients Who Have Participated in a BMN 701 Study

Study Overview
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Brief Summary
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This is a Phase 2 open-label, multiple dose study of BMN 701 administered by IV infusion every 2 weeks (qow) to patients with late-onset Pompe disease.

Official Title
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A Long-Term Study for Extended BMN 701 Treatment of Patients With Pompe Disease Who Have Participated in a BMN 701 Study

Conditions
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Pompe Disease

Intervention / Treatment
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* Biological: BMN 701


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Have completed a prior BMN 701 clinical development study; Have provided written informed consent after the nature of the study has been explained prior to performance of any study-related procedures. Minors may participate as long as they provide written assent after the nature of the study has been explained to them and after their parent, or legal guardian has provided written informed consent, prior to the performance of any study-related procedures; Have been diagnosed with late-onset Pompe Disease, based on the entry criteria of a prior BMN 701 study; If sexually active, be willing to use 2 known effective methods of contraception from Screening until 4 months after the last dose of study-drug; If female, and not considered to be of childbearing potential, be at least 2 years post-menopausal, or have had tubal ligation at least 1 year prior to screening, or have had a total hysterectomy; If female, and of childbearing potential, have a negative pregnancy test during the Screening Period and at the Baseline visit, and be willing to have additional pregnancy tests during the study; Have the ability to comply with the protocol requirements, in the opinion of the Investigator. Exclusion Criteria: Have received any experimental or approved therapy for Pompe disease, other than BMN 701, subsequent to completion of a BMN 701 study and prior to entry into POM-002; Have received, or are anticipated to receive, any investigational medication, other than BMN 701, within 30 days prior to the first dose of study-drug; Are breastfeeding at screening or planning to become pregnant (self or partner) at any time during the study; Have a medical condition or extenuating circumstance that, in the opinion of the Investigator, might compromise the patient's ability to comply with the protocol requirements or compromise the patient's well being or safety.

Ages Eligible for Study
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Minimum Age: 13 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: BMN 701 20mg/kg<br>BMN 701 20mg/kg IV every other week | Biological: BMN 701<br>* GILT-tagged recombinant human GAA<br>|
| Experimental: BMN 701 10mg/kg<br>BMN 701 10mg/kg IV every other week | Biological: BMN 701<br>* GILT-tagged recombinant human GAA<br>|
| Experimental: BMN 701 5mg/kg<br>BMN 701 5mg/kg IV every other week | Biological: BMN 701<br>* GILT-tagged recombinant human GAA<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants With a Positive Anti-BMN 701 Antibody | Status of Anti-BMN 701 antibody is corresponding to the test results of blood samples. | Baseline, Week 144 | 
| Number of Participants With a Positive Anti-BMN 701 Antibody Response | Status of Anti-IGF-I antibody is corresponding to the test results of blood samples | Baseline, Week 144 | 
| Number of Participants With a Positive Anti-BMN 701 Antibody Response | Status of Anti-IGF-II antibody is corresponding to the test results of blood samples | Baseline, Week 144 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percent Predicted Maximal Inspiratory Pressure (MIP) | Pulmonary Function Test: Percent Predicted Maximal Inspiratory Pressure | Baseline, Week 144 | 
| Percent Predicted Maximum Expiratory Pressure (MEP) | Pulmonary Function Test: Percent Predicted Maximum Expiratory Pressure | Baseline, Week 144 | 
| 6 Minutes Walk Test (Meters) | Distance walked within 6 minutes | Baseline, Week 144 | 
| Maximum Voluntary Ventilation (MVV) | Pulmonary function test: Maximum Voluntary Ventilation (MVV) | Baseline, Week 144 | 
| Percent Predicted Upright Forced Vital Capacity (FVC) | Pulmonary function test: Percent Predicted Upright Forced Vital Capacity | Baseline, Week 144 | 
| Change From Baseline in Urine Tetrasaccharide Concentration at Week 144 | Change from Baseline in Urine Tetrasaccharide Concentration at Week 144 | Baseline, Week 144 | 
| Plasma IGF-I Concentration | Plasma IGF-I concentration from lab | Baseline, Week 144 | 
| Plasma IGF-II Concentration | Plasma IGF-II concentration from lab | Baseline, Week 144 | 
| Insulin-like Growth Factor Binding Protein 3 (IGFBP3) | insulin-like growth factor binding protein 3 from lab | Baseline, Week 144 |