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

This study is currently recruiting participants. (see Contacts and Locations)
Verified September 2014 by BioMarin Pharmaceutical
Information provided by (Responsible Party):
BioMarin Pharmaceutical Identifier:
First received: September 8, 2011
Last updated: September 17, 2014
Last verified: September 2014

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: 24 weeks ] [ Designated as safety issue: Yes ]
  • Anti-BMN 701 antibody titer [ Time Frame: 24 weeks ] [ Designated as safety issue: Yes ]
  • Anti-Insulin-like-growth-factor antibody titer [ Time Frame: 24 weeks ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Mean distance walked as measured by the Six-minute Walk Test (6MWT) [ Time Frame: 24 weeks ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 45
Study Start Date: August 2011
Estimated Study Completion Date: September 2016
Estimated Primary Completion Date: August 2016 (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 identifier: NCT01435772

Contact: Carol Sichelmeier

United States, California
Univ of California San Diego School of Medicine Recruiting
La Jolla, California, United States
Contact: Jan Panyard-Davis    619-471-9585   
Principal Investigator: Bruce Barshop, MD, PhD         
United States, Florida
University of Florida College of Medicine Recruiting
Gainesville, Florida, United States
Contact: Lindsay Falk    352-273-9615   
Principal Investigator: Barry J Byrne, MD, PhD         
United States, Kansas
University of Kansas Medical Center Recruiting
Kansas City, Kansas, United States
Principal Investigator: Richard Barohn, MD         
Australia, Queensland
Royal Brisbane and Women's Hospital Recruiting
Herston, Queensland, Australia, 4029
Contact: Helen Woodhouse    +61736362513   
Principal Investigator: Robert Henderson, MD         
United Kingdom
University Hospitals Birmingham NHS Foundation Trust Recruiting
Birmingham, United Kingdom, B15 2TH
Contact: Katherine Peers    +44 (0) 121 3712000 ex 5926   
Principal Investigator: Tarekgen Hiwot, MD         
Royal Free Hospital Recruiting
London, United Kingdom, NW3 2QG
Contact: Alan Milligan    +4402074726409   
Principal Investigator: Derralynn Hughes, MD         
Sponsors and Collaborators
BioMarin Pharmaceutical
Study Director: William Lang, MD BioMarin Pharmaceutical
  More Information

No publications provided

Responsible Party: BioMarin Pharmaceutical Identifier: NCT01435772     History of Changes
Other Study ID Numbers: POM-002, 2011-001805-28
Study First Received: September 8, 2011
Last Updated: September 17, 2014
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 processed this record on November 25, 2014