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

This study is ongoing, but not recruiting participants.
Information provided by (Responsible Party):
BioMarin Pharmaceutical
ClinicalTrials.gov Identifier:
First received: September 8, 2011
Last updated: April 27, 2016
Last verified: April 2016
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.

Condition Intervention Phase
Pompe Disease
Biological: BMN 701
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Long-Term Study for Extended BMN 701 Treatment of Patients With Pompe Disease Who Have Participated in a BMN 701 Study

Resource links provided by NLM:

Further study details as provided by BioMarin Pharmaceutical:

Primary Outcome Measures:
  • Number of Treatment-Emergent Adverse Events [ Time Frame: 264 weeks ] [ Designated as safety issue: Yes ]
  • Anti-BMN 701 antibody titer [ Time Frame: 264 weeks ] [ Designated as safety issue: Yes ]
  • Anti-Insulin-like-growth-factor antibody titer [ Time Frame: 264 weeks ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Mean distance walked as measured by the Six-minute Walk Test (6MWT) [ Time Frame: 264 weeks ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 45
Study Start Date: August 2011
Estimated Study Completion Date: September 2018
Estimated Primary Completion Date: August 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Experimental: BMN 701 Biological: BMN 701
GILT-tagged recombinant human GAA


Ages Eligible for Study:   13 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01435772

United States, California
Univ of California San Diego School of Medicine
San Diego, California, United States, 92103
United States, Florida
University of Florida College of Medicine
Gainesville, Florida, United States, 32610
Tampa General Hospital
Tampa, Florida, United States, 33606
United States, Kansas
University of Kansas Medical Center
Kansas City, Kansas, United States, 66160
Australia, Queensland
Royal Brisbane and Women's Hospital
Herston, Queensland, Australia, 4029
Australia, South Australia
Royal Adelaide Hospital
North Adelaide, South Australia, Australia, 5006
Hôpital Pitié-Salpêtrière
Paris, France, 75013
Villa Metabolica, ZKJM MC University Mainz
Mainz, Germany, 55131
New Zealand
Auckland City and Starship Children's Hospital
Auckland, New Zealand, 1142
United Kingdom
University Hospitals Birmingham NHS Foundation Trust
Birmingham, United Kingdom, B15 2TH
Royal Free Hospital
London, United Kingdom, NW3 2QG
Salford Royal NHS Foundation Trust
Salford, United Kingdom, M5 5AP
Sponsors and Collaborators
BioMarin Pharmaceutical
Study Director: Medical Monitor BioMarin Pharmaceutical
  More Information

Responsible Party: BioMarin Pharmaceutical
ClinicalTrials.gov Identifier: NCT01435772     History of Changes
Other Study ID Numbers: POM-002  2011-001805-28 
Study First Received: September 8, 2011
Last Updated: April 27, 2016
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Glycogen Storage Disease Type II
Brain Diseases
Brain Diseases, Metabolic
Brain Diseases, Metabolic, Inborn
Carbohydrate Metabolism, Inborn Errors
Central Nervous System Diseases
Genetic Diseases, Inborn
Glycogen Storage Disease
Lysosomal Storage Diseases
Lysosomal Storage Diseases, Nervous System
Metabolic Diseases
Metabolism, Inborn Errors
Nervous System Diseases

ClinicalTrials.gov processed this record on May 26, 2016