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

Phase 2

Terminated

Brief Summary: 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.

Recruitment & Eligibility

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.

Arm && Interventions

Primary Outcome Measures

Name	Time	Method
Number of Participants With a Positive Anti-BMN 701 Antibody	Baseline, Week 144	Status of Anti-BMN 701 antibody is corresponding to the test results of blood samples.
Number of Participants With a Positive Anti-BMN 701 Antibody Response	Baseline, Week 144	Status of Anti-IGF-II antibody is corresponding to the test results of blood samples

Secondary Outcome Measures

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

Locations (12): Univ of California San Diego School of Medicine
🇺🇸
San Diego, California, United States
University of Florida College of Medicine
🇺🇸
Gainesville, Florida, United States
Royal Brisbane and Women's Hospital
🇦🇺
Herston, Queensland, Australia
Tampa General Hospital
🇺🇸
Tampa, Florida, United States
Hôpital Pitié-Salpêtrière
🇫🇷
Paris, France
Auckland City and Starship Children's Hospital
🇳🇿
Auckland, New Zealand
Villa Metabolica, ZKJM MC University Mainz
🇩🇪
Mainz, Germany
Royal Free Hospital
🇬🇧
London, United Kingdom
Salford Royal NHS Foundation Trust
🇬🇧
Salford, United Kingdom
University Hospitals Birmingham NHS Foundation Trust
🇬🇧
Birmingham, United Kingdom
Royal Adelaide Hospital
🇦🇺
North Adelaide, South Australia, Australia
University of Kansas Medical Center
🇺🇸
Kansas City, Kansas, United States