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

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ClinicalTrials.gov Identifier: NCT01435772
Recruitment Status : Active, not recruiting
First Posted : September 19, 2011
Last Update Posted : April 29, 2016
Information provided by (Responsible Party):
BioMarin Pharmaceutical

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.

Condition or disease Intervention/treatment Phase
Pompe Disease Biological: BMN 701 Phase 2

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 45 participants
Intervention Model: Single Group Assignment
Masking: None (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
Study Start Date : August 2011
Estimated Primary Completion Date : August 2018
Estimated Study Completion Date : September 2018

Arm Intervention/treatment
Experimental: Experimental: BMN 701 Biological: BMN 701
GILT-tagged recombinant human GAA

Primary Outcome Measures :
  1. Number of Treatment-Emergent Adverse Events [ Time Frame: 264 weeks ]
  2. Anti-BMN 701 antibody titer [ Time Frame: 264 weeks ]
  3. Anti-Insulin-like-growth-factor antibody titer [ Time Frame: 264 weeks ]

Secondary Outcome Measures :
  1. Mean distance walked as measured by the Six-minute Walk Test (6MWT) [ Time Frame: 264 weeks ]

Information from the National Library of Medicine

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Ages Eligible for Study:   13 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): 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

Responsible Party: BioMarin Pharmaceutical
ClinicalTrials.gov Identifier: NCT01435772     History of Changes
Other Study ID Numbers: POM-002
2011-001805-28 ( EudraCT Number )
First Posted: September 19, 2011    Key Record Dates
Last Update Posted: April 29, 2016
Last Verified: April 2016

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