- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01924845
BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study)
June 12, 2018 updated by: BioMarin Pharmaceutical
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
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.
Study Overview
Study Type
Interventional
Enrollment (Actual)
24
Phase
- Phase 3
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Edegem, Belgium
- Antwerp University Hospital (UZA)
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Garches, France, 92380
- Hôpital Raymond Poincaré
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Marseille, France, 13005
- CHU de la Timone
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Mainz, Germany
- Villa Metabolica, ZKJM MC University Mainz
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München, Germany
- Klinikum der Universität München
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Münster, Germany, 48149
- Universitätsklinikum Münster
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Milano, Italy, 20133
- Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta
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ME
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Messina, ME, Italy, 98125
- Azienda Ospedaliera Universitaria Policlinico "G. Martino" - Messina
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Rotterdam, Netherlands, 3015 GJ
- Erasmus MC University Medical Center
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Porto, Portugal, 4200-319
- Centro Hospitalar de Sao Joao, EPE
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Birmingham, United Kingdom
- University Hospital Birmingham
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London, United Kingdom, NW3 2QG
- Royal Free Hospital
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London, United Kingdom
- National Hospital For Neurology and Neurosurgery
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Salford, United Kingdom, M5 5AP
- Salford Royal NHS Foundation Trust
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Arizona
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Phoenix, Arizona, United States, 85028
- Neuromuscular Research Centre
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California
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Orange, California, United States, 92868
- University of California, Irvine
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Florida
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Gainesville, Florida, United States, 32610
- University of Florida
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Kansas
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Kansas City, Kansas, United States, 66160
- University Of Kansas Medical Center
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Missouri
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Saint Louis, Missouri, United States, 36110
- Washington University
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North Carolina
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Durham, North Carolina, United States, 27710
- Duke University Medical Center
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Ohio
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Columbus, Ohio, United States, 43210
- The Ohio State University - Wexner Medical Center
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Utah
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Salt Lake City, Utah, United States, 84132
- University of Utah
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
18 years and older (ADULT, OLDER_ADULT)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
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:
- 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).
- has received > 80% of all scheduled treatments in the prior 48 weeks and ≥ 4 out of the prior 6 scheduled treatments.
- has received and completed the last two infusions without a drug-related adverse event resulting in dose interruption.
- 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.
- Is able to ambulate ≥75 meters and ≤500 meters on the 6MWT conducted during the Screening 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.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: TREATMENT
- Allocation: NA
- Interventional Model: SINGLE_GROUP
- Masking: NONE
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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EXPERIMENTAL: BMN 701 20 mg/kg
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)
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BMN 701 20 mg/kg for intravenous administration over approximately 4 hours every 2 weeks over a 24-week Treatment Period (total of 13 doses), and every 2 weeks over a 240-week Extension Period (up to 120 additional doses).
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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Percent Predicted Maximum Inspiratory Pressure (MIP)
Time Frame: Baseline, Week 24
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Pulmonary function test: Percent Predicted Maximum Inspiratory Pressure
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Baseline, Week 24
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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Percent Predicted Maximum Expiratory Pressure (MEP)
Time Frame: Baseline, Week 24
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Pulmonary function test: Percent Predicted Maximum Expiratory Pressure
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Baseline, Week 24
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6 Minute Walk Test (Meters)
Time Frame: Baseline, Week 24
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Distance walked within 6 minutes
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Baseline, Week 24
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Percent Predicted Upright Forced Vital Capacity (FVC)
Time Frame: Baseline, Week 24
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Pulmonary function test: Percent Predicted Upright Forced Vital Capacity
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Baseline, Week 24
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Number of Participants With Non-Serious AEs
Time Frame: Baseline through Week 24 +4 weeks follow-up
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Number of participants with non-serious Adverse Events.
Data is taken at final time point of Week 24, compared to baseline.
For full AE data, please see AE section.
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Baseline through Week 24 +4 weeks follow-up
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Study Director: Study Monitor, BioMarin Pharmaceutical
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (ACTUAL)
April 1, 2014
Primary Completion (ACTUAL)
September 12, 2016
Study Completion (ACTUAL)
September 12, 2016
Study Registration Dates
First Submitted
August 13, 2013
First Submitted That Met QC Criteria
August 15, 2013
First Posted (ESTIMATE)
August 19, 2013
Study Record Updates
Last Update Posted (ACTUAL)
June 14, 2018
Last Update Submitted That Met QC Criteria
June 12, 2018
Last Verified
June 1, 2018
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Metabolic Diseases
- Brain Diseases
- Central Nervous System Diseases
- Nervous System Diseases
- Genetic Diseases, Inborn
- Carbohydrate Metabolism, Inborn Errors
- Metabolism, Inborn Errors
- Lysosomal Storage Diseases
- Brain Diseases, Metabolic
- Brain Diseases, Metabolic, Inborn
- Lysosomal Storage Diseases, Nervous System
- Glycogen Storage Disease
- Glycogen Storage Disease Type II
Other Study ID Numbers
- 701-301
- 2013-001768-48 (EUDRACT_NUMBER)
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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