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Trial record 35 of 64 for:    pompe

BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE 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: August 13, 2013
Last updated: October 14, 2014
Last verified: September 2014

Study 701-301 is a single-arm, open-label, switchover study in patients with late-onset Pompe disease who have been receiving treatment with recombinant human acid alpha-glucosidase (rhGAA) for 48 weeks or longer. Ambulatory patients who have mild to moderate respiratory impairment will switch directly to receive BMN 701 20 mg/kg by IV infusion every other week. All participants will receive active drug. No dose of existing therapy will be missed - experimental drug is started immediately.

Condition Intervention Phase
Late-onset Pompe Disease
Drug: BMN 701
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects With Late-onset Pompe Disease

Resource links provided by NLM:

Further study details as provided by BioMarin Pharmaceutical:

Primary Outcome Measures:
  • percent predicted MIP (Maximum Inspiratory Pressure) measured at the mouth by the Mueller maneuver [ Time Frame: 24 weeks ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • percent predicted MEP (Maximum Expiratory Pressure) and FVC (Forced Vital Capacity) Upright [ Time Frame: 24 weeks ] [ Designated as safety issue: No ]
  • 6 Minute Walk Test [ Time Frame: 24 weeks ] [ Designated as safety issue: No ]

Estimated Enrollment: 50
Study Start Date: March 2014
Estimated Primary Completion Date: June 2020 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: BMN 701
BMN 701 IV Infusion 20mg/kg every 2 weeks for 24 weeks followed by an optional extension of 240 weeks (total duration of therapy 264 weeks)
Drug: BMN 701


Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Willing and able to provide written informed consent, after the nature of the study has been explained, and prior to any study-related procedures.
  • Diagnosed with late-onset Pompe disease based on 2 currently or previously documented GAA gene mutations, and endogenous GAA activity <75% of the lower limit of the normal adult range reported by the testing laboratory, as assessed by dried blood spot or whole blood assay.
  • Has received prior treatment with commercial rhGAA as defined by ALL of the following:

    1. has received treatment with commercial rhGAA for ≥ 48 weeks (but no more than 20% of the study population can have received treatment for ≥ 6 years).
    2. has received > 80% of all scheduled treatments in the prior 48 weeks and ≥ 4 out of the prior 6 scheduled treatments.
    3. has received and completed the last two infusions without a drug-related adverse event resulting in dose interruption.
    4. has received last treatment of commercial rhGAA ≥ 10 and ≤ 31 days prior to anticipated initiation of treatment with BMN 701.
  • ≥ 18 years of age at the time of enrollment in the study.
  • Sexually active subjects must be willing to use two known effective methods of contraception while participating in the study and for at least 4 months following the last dose of BMN 701.
  • Females of childbearing potential must have a negative pregnancy test at Screening and Baseline visits and be willing to have additional pregnancy tests during the study. Females considered not of childbearing potential include those who have been in menopause at least 2 years, or had tubal ligation at least 1 year prior to Screening, or who have had total hysterectomy.
  • Has ≥ 30% predicted upright FVC and < 80% predicted upright FVC.
  • Has ≤60% predicted MIP.
  • Must meet ambulation criteria, as measured on two separate days conducted during the Screening and/or Baseline visit (use of assistive devices such as walker, cane, or crutches, is permitted with consistent use throughout the study).
  • Is willing and able to comply with all study procedures.

Exclusion Criteria:

  • Use of any investigational product or investigational medical device within 4 weeks prior to Screening, or requirement for any investigational agent other than BMN 701 prior to completion of at least the first 24 weeks of all scheduled study assessments.
  • Received any investigational medication for Pompe disease within the prior 12 months.
  • Has a diagnosis of diabetes and/or is currently being treated with or anticipated to require treatment with hypoglycemic agents during the course of the study.
  • Has been treated with any immunosuppressive medication other than glucocorticosteroids within the prior 12 months.
  • Requires noninvasive ventilatory support while awake and in the upright position.
  • Has previously been enrolled to this study.
  • Breastfeeding at Screening or planning to become pregnant (self or partner) at any time during the study.
  • Concurrent disease, medical condition, or extenuating circumstance that, in the opinion of the Investigator, might compromise subject safety, study treatment compliance and completion of the study, or the integrity of the data collected for the study.
  • Has known hypersensitivity to BMN 701 or its excipients.
  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: NCT01924845

Contact: Andrew Mazur

United States, Arizona
Neuromuscular Research Centre Recruiting
Phoenix, Arizona, United States, 85028
Principal Investigator: Kumaraswamy Sivakumar, MD         
United States, California
University of California, Irvine Recruiting
Orange, California, United States, 92868
Principal Investigator: Tahseet Mozaffar, MD         
United States, Florida
University of Florida Recruiting
Gainesville, Florida, United States, 32610
Contact: Terry Sexton    352-294-5757   
Principal Investigator: Barry Byrne, MD, PhD         
United States, Kansas
University of Kansas Medical Center Recruiting
Kansas City, Kansas, United States, 66160
Contact: Melissa Cooley    913-945-7697   
Principal Investigator: Richard Barohn, MD         
United States, Missouri
Washington University Recruiting
St. Louis, Missouri, United States, 36110
Principal Investigator: Alan Pestronk, MD         
United States, North Carolina
Not yet recruiting
Durham, North Carolina, United States
United States, Ohio
The Ohio State University - Wexner Medical Center Recruiting
Columbus, Ohio, United States, 43210
Contact: Julie Agriesti    614-293-4098   
Principal Investigator: John Kissel, MD         
United States, Utah
Not yet recruiting
Salt Lake City, Utah, United States
Not yet recruiting
Antwerp, Belgium
Not yet recruiting
Paris, France
Not yet recruiting
Mainz, Germany
Not yet recruiting
Muenster, Germany
Not yet recruiting
Munchen, Germany
Not yet recruiting
Messina, Italy
Not yet recruiting
Milan, Italy
Not yet recruiting
Rotterdam, Netherlands
Not yet recruiting
Porto, Portugal
United Kingdom
University Hospital Birmingham Recruiting
Birmingham, United Kingdom
Contact: Richard Martin    441213713178   
Principal Investigator: Tarekegn Hiwot, MD         
Royal Free Hospital Recruiting
London, United Kingdom, NW3 2QG
Contact: Allison Warwick    +44.207.7830.2814   
Principal Investigator: Derralynn Hughes, MD         
Salford Royal NHS Foundation Trust Recruiting
Salford, United Kingdom, M5 5AP
Contact: Nikki Harwood    441612067130   
Principal Investigator: Mark Roberts, MD         
Sponsors and Collaborators
BioMarin Pharmaceutical
Study Director: Ed Conner, MD BioMarin Pharmaceutical
  More Information

No publications provided

Responsible Party: BioMarin Pharmaceutical Identifier: NCT01924845     History of Changes
Other Study ID Numbers: 701-301, 2013-001768-48
Study First Received: August 13, 2013
Last Updated: October 14, 2014
Health Authority: United States: Food and Drug Administration
United Kingdom: Medicines and Healthcare Products Regulatory Agency
United Kingdom: Research Ethics Committee
France: Agence Nationale de Sécurité du Médicament et des produits de santé
Germany: Federal Institute for Drugs and Medical Devices

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 20, 2014