Late-onset Pompe DiseasePhase 3NCT01924845
BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study)
A Phase 3 study testing BMN 701 in people with Late-onset Pompe Disease, run by BioMarin Pharmaceutical at 22 sites in 8 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Late-onset Pompe Disease
- What is being tested?
- BMN 701
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 22 sitesin 8 countries
- Study dates
- Apr 2014to 12 Sep 2016
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
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- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
22 sites in 8 countries.
22 sites in 8 countries
United States8 sites
The Ohio State University - Wexner Medical Center
Columbus, Ohio, 43210
Duke University Medical Center
Durham, North Carolina, 27710
University of Florida
Gainesville, Florida, 32610
University of Kansas Medical Center
Kansas City, Kansas, 66160
University of California, Irvine
Orange, California, 92868
Neuromuscular Research Centre
Phoenix, Arizona, 85028
University of Utah
Salt Lake City, Utah, 84132
Washington University
St Louis, Missouri, 36110
United Kingdom4 sites
University Hospital Birmingham
Birmingham
Royal Free Hospital
London, NW3 2QG
National Hospital for Neurology and Neurosurgery
London
Salford Royal NHS Foundation Trust
Salford, M5 5AP
Germany3 sites
Villa Metabolica, ZKJM MC University Mainz
Mainz
Klinikum der Universität München
München
Universitätsklinikum Münster
Münster, 48149
France2 sites
Hôpital Raymond Poincaré
Garches, 92380
CHU de la Timone
Marseille, 13005
Italy2 sites
Azienda Ospedaliera Universitaria Policlinico "G. Martino" - Messina
Messina, ME, 98125
Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta
Milan, 20133
About this study
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
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.
- Conditions
- Late-onset Pompe Disease
- Being tested
- BMN 701
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BMN 701Drug
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).
| Study group | Role | What they receive |
|---|---|---|
| BMN 701 20 mg/kg | Experimental | 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) Receives: Drug: BMN 701 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent Predicted Maximum Inspiratory Pressure (MIP)
Pulmonary function test: Percent Predicted Maximum Inspiratory Pressure
Measured: Baseline, Week 24
Secondary outcomes (4)
Percent Predicted Maximum Expiratory Pressure (MEP)
Pulmonary function test: Percent Predicted Maximum Expiratory Pressure
Measured: Baseline, Week 24
6 Minute Walk Test (Meters)
Distance walked within 6 minutes
Measured: Baseline, Week 24
Percent Predicted Upright Forced Vital Capacity (FVC)
Pulmonary function test: Percent Predicted Upright Forced Vital Capacity
Measured: Baseline, Week 24
Number of Participants With Non-Serious AEs
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.
Measured: Baseline through Week 24 +4 weeks follow-up
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Aug 2013 (estimated)
- Study startApr 2014
- Primary completion12 Sep 2016
- Study completion12 Sep 2016
Why it stopped: Sponsor decision
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Late-onset Pompe DiseaseStudies of the same condition, open and under way.
- More trials run by BioMarin PharmaceuticalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- BioMarin Pharmaceutical
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Monitor, Study director, BioMarin Pharmaceutical
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01924845
- Sponsor's protocol ID
- 701-301
- Other IDs
- 2013-001768-48
- Status last verified
- Jun 2018
- First submitted
- 13 Aug 2013
- Last updated
- 14 Jun 2018
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 14 Jun 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT01924845
- Acronym / sponsor ID
- 701-301
- Conditions
- Late-onset Pompe Disease
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Sponsor decision
- Enrolment
- 24 participants (actual)
- Lead sponsor
- BioMarin PharmaceuticalIndustry (pharma, biotech, devices)
- Study start
- Apr 2014 (actual)
- Primary completion
- 12 Sep 2016 (actual)
- Study completion
- 12 Sep 2016 (actual)
- First posted
- 19 Aug 2013 (estimated)
- Results
- Posted 14 Jun 2018
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| BMN 701 20 mg/kg | Experimental | Drug: BMN 701BMN 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) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (14 listed)
- 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:
- ≥ 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.
Key exclusion criteria (9 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BMN 701BMN 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). | Drug | BMN 701 20 mg/kg |
Primary outcome
Percent Predicted Maximum Inspiratory Pressure (MIP)
Time frame: Baseline, Week 24
Pulmonary function test: Percent Predicted Maximum Inspiratory Pressure
4 secondary outcomes
Percent Predicted Maximum Expiratory Pressure (MEP)
Time frame: Baseline, Week 24
6 Minute Walk Test (Meters)
Time frame: Baseline, Week 24
Percent Predicted Upright Forced Vital Capacity (FVC)
Time frame: Baseline, Week 24
Number of Participants With Non-Serious AEs
Time frame: Baseline through Week 24 +4 weeks follow-up
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Jun 2018.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Study Monitor study director
BioMarin Pharmaceutical
22 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing BMN 701 in people with Late-onset Pompe Disease, run by BioMarin Pharmaceutical at 22 sites in 8 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 14 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 14 Jun 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 22 sites in United States, United Kingdom, Germany, France, Italy and 3 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 14 Jun 2018). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percent Predicted Maximum Inspiratory Pressure (MIP) (time frame: Baseline, Week 24). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Jun 2018.