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    Clinical Trial Results:
    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

    Summary
    EudraCT number
    2013-001768-48
    Trial protocol
    GB   BE   DE   IT   PT   AT  
    Global end of trial date
    12 Sep 2016

    Results information
    Results version number
    v1(current)
    This version publication date
    14 Oct 2017
    First version publication date
    14 Oct 2017
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    701-301
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT01924845
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    BioMarin Pharmaceutical Inc.
    Sponsor organisation address
    105 Digital Drive, Novato, United States, 94949
    Public contact
    BMN701 Clinical Program Management, BioMarin Europe Ltd., +44 0782455 2081, smccarthy@bmrn.com
    Scientific contact
    BMN701 Clinical Program Management, BioMarin Europe Ltd., +44 0782455 2081, smccarthy@bmrn.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    30 Jun 2017
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    12 Sep 2016
    Global end of trial reached?
    Yes
    Global end of trial date
    12 Sep 2016
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    The primary objective of the study is to determine if treatment with BMN 701 results in an increase in MIP measured at the mouth by the Mueller maneuver from Baseline to Week 24 in subjects with late-onset Pompe disease previously treated with rhGAA. The primary objective of the extension period of the study is to evaluate the long-term safety and efficacy of BMN 701 in subjects with late-onset Pompe disease previously treated with rhGAA.
    Protection of trial subjects
    An independent Data Monitoring Committee (DMC) acted in an advisory capacity to monitor safety in subjects who participated in Study 701-301. The DMC included at least one allergist/immunologist and may have also provided guidance on management of hypersensitivity reactions.
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    17 Mar 2014
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Belgium: 1
    Country: Number of subjects enrolled
    Germany: 9
    Country: Number of subjects enrolled
    United Kingdom: 7
    Country: Number of subjects enrolled
    United States: 7
    Worldwide total number of subjects
    24
    EEA total number of subjects
    17
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    0
    Children (2-11 years)
    0
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    22
    From 65 to 84 years
    2
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    -

    Pre-assignment
    Screening details
    Individuals who met any of the following exclusion criteria were not eligible to participate in the study: Use of any investigational product or investigational medical device within 4 weeks prior to Screening; Required noninvasive ventilatory support while awake and in the upright position; Had a diagnosis of diabetes.

    Period 1
    Period 1 title
    Treatment phase (overall period)
    Is this the baseline period?
    Yes
    Allocation method
    Non-randomised - controlled
    Blinding used
    Not blinded

    Arms
    Arm title
    BMN 701 20 mg/kg
    Arm description
    BMN 701 20 mg/kg
    Arm type
    Experimental

    Investigational medicinal product name
    BMN 701
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Concentrate for concentrate for solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    BMN 701 20 mg/kg for IV 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).

    Number of subjects in period 1
    BMN 701 20 mg/kg
    Started
    24
    Completed
    18
    Not completed
    6
         Consent withdrawn by subject
    2
         Adverse event, non-fatal
    3
         Protocol deviation
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    BMN 701 20 mg/kg
    Reporting group description
    BMN 701 20 mg/kg

    Reporting group values
    BMN 701 20 mg/kg Total
    Number of subjects
    24 24
    Age categorical
    Units: Subjects
        18-65
    22 22
        > 65
    2 2
    Age continuous
    Units: Years
        arithmetic mean (standard deviation)
    47.9 ± 13.27 -
    Gender categorical
    Units: Subjects
        Female
    12 12
        Male
    12 12

    End points

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    End points reporting groups
    Reporting group title
    BMN 701 20 mg/kg
    Reporting group description
    BMN 701 20 mg/kg

    Subject analysis set title
    Full Analysis Set
    Subject analysis set type
    Full analysis
    Subject analysis set description
    Full Analysis Set

    Primary: Baseline Percent Predicted MIP

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    End point title
    Baseline Percent Predicted MIP [1]
    End point description
    End point type
    Primary
    End point timeframe
    Baseline
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: The study was terminated because BioMarin decided to end the overall development program based on competing corporate priorities. The study was not terminated for efficacy or safety reasons. Patients
    End point values
    BMN 701 20 mg/kg Full Analysis Set
    Number of subjects analysed
    18
    18
    Units: percent
        arithmetic mean (standard deviation)
    50 ± 17.5
    50 ± 17.5
    No statistical analyses for this end point

    Primary: Change from Baseline to Week 24 - Percent Predicted MIP

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    End point title
    Change from Baseline to Week 24 - Percent Predicted MIP [2]
    End point description
    End point type
    Primary
    End point timeframe
    0-24 Weeks
    Notes
    [2] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: The study was terminated because BioMarin decided to end the overall development program based on competing corporate priorities. The study was not terminated for efficacy or safety reasons. Patients
    End point values
    BMN 701 20 mg/kg Full Analysis Set
    Number of subjects analysed
    18
    18
    Units: percent
        arithmetic mean (standard deviation)
    2.2 ± 8.3
    2.2 ± 8.3
    No statistical analyses for this end point

    Secondary: Baseline Percent Predicted MEP

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    End point title
    Baseline Percent Predicted MEP
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline
    End point values
    BMN 701 20 mg/kg Full Analysis Set
    Number of subjects analysed
    18
    18
    Units: percent
        arithmetic mean (standard deviation)
    38.9 ± 12.3
    38.9 ± 12.3
    No statistical analyses for this end point

    Secondary: Change from Baseline to Week 24 - Percent Predicted MEP

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    End point title
    Change from Baseline to Week 24 - Percent Predicted MEP
    End point description
    End point type
    Secondary
    End point timeframe
    0-24 Weeks
    End point values
    BMN 701 20 mg/kg Full Analysis Set
    Number of subjects analysed
    18
    18
    Units: percent
        arithmetic mean (standard deviation)
    3.1 ± 8.7
    3.1 ± 8.7
    No statistical analyses for this end point

    Secondary: Baseline Six Minutes Walk Test

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    End point title
    Baseline Six Minutes Walk Test
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline
    End point values
    BMN 701 20 mg/kg Full Analysis Set
    Number of subjects analysed
    17
    17
    Units: meter
        arithmetic mean (standard deviation)
    345.8 ± 95.3
    345.8 ± 95.3
    No statistical analyses for this end point

    Secondary: Change from Baseline to Week 24 - Six Minutes Walk Test

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    End point title
    Change from Baseline to Week 24 - Six Minutes Walk Test
    End point description
    End point type
    Secondary
    End point timeframe
    0-24 Weeks
    End point values
    BMN 701 20 mg/kg Full Analysis Set
    Number of subjects analysed
    17
    17
    Units: meter
        arithmetic mean (standard deviation)
    26.1 ± 40.6
    26.1 ± 40.6
    No statistical analyses for this end point

    Secondary: Baseline Percent Predicted FVC Upright

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    End point title
    Baseline Percent Predicted FVC Upright
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline
    End point values
    BMN 701 20 mg/kg Full Analysis Set
    Number of subjects analysed
    18
    18
    Units: percent
        arithmetic mean (standard deviation)
    60.7 ± 15.1
    60.7 ± 15.1
    No statistical analyses for this end point

    Secondary: Change from Baseline to Week 24 - Percent Predicted FVC Upright

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    End point title
    Change from Baseline to Week 24 - Percent Predicted FVC Upright
    End point description
    End point type
    Secondary
    End point timeframe
    0-24 Weeks
    End point values
    BMN 701 20 mg/kg Full Analysis Set
    Number of subjects analysed
    18
    18
    Units: percent
        arithmetic mean (standard deviation)
    -3.7 ± 4.4
    -3.7 ± 4.4
    No statistical analyses for this end point

    Secondary: Non-serious AEs

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    End point title
    Non-serious AEs
    End point description
    End point type
    Secondary
    End point timeframe
    0-24 weeks
    End point values
    BMN 701 20 mg/kg Full Analysis Set
    Number of subjects analysed
    24
    24
    Units: number
    23
    23
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Study Period
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    19.0
    Reporting groups
    Reporting group title
    BMN 701 20 mg/kg
    Reporting group description
    -

    Serious adverse events
    BMN 701 20 mg/kg
    Total subjects affected by serious adverse events
         subjects affected / exposed
    10 / 24 (41.67%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    Investigations
    Blood creatine phosphokinase increased
         subjects affected / exposed
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Injury, poisoning and procedural complications
    Fall
         subjects affected / exposed
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Vascular disorders
    Hypertension
         subjects affected / exposed
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Cardiac disorders
    Atrial fibrillation
         subjects affected / exposed
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Immune system disorders
    Anaphylactic reaction
         subjects affected / exposed
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Gastrointestinal disorders
    Abdominal pain upper
         subjects affected / exposed
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    2 / 2
         deaths causally related to treatment / all
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Pneumothorax
         subjects affected / exposed
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Respiratory failure
         subjects affected / exposed
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Skin and subcutaneous tissue disorders
    Erythema nodosum
         subjects affected / exposed
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Musculoskeletal and connective tissue disorders
    Pain in extremity
         subjects affected / exposed
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Metabolism and nutrition disorders
    Hypoglycaemia
         subjects affected / exposed
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    BMN 701 20 mg/kg
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    23 / 24 (95.83%)
    Injury, poisoning and procedural complications
    Fall
         subjects affected / exposed
    5 / 24 (20.83%)
         occurrences all number
    6
    Infusion related reaction
         subjects affected / exposed
    2 / 24 (8.33%)
         occurrences all number
    2
    Vascular disorders
    Hypotension
         subjects affected / exposed
    3 / 24 (12.50%)
         occurrences all number
    3
    Cardiac disorders
    Palpitations
         subjects affected / exposed
    4 / 24 (16.67%)
         occurrences all number
    5
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    8 / 24 (33.33%)
         occurrences all number
    12
    Headache
         subjects affected / exposed
    13 / 24 (54.17%)
         occurrences all number
    50
    Lethargy
         subjects affected / exposed
    2 / 24 (8.33%)
         occurrences all number
    2
    Tremor
         subjects affected / exposed
    4 / 24 (16.67%)
         occurrences all number
    5
    General disorders and administration site conditions
    Chest discomfort
         subjects affected / exposed
    2 / 24 (8.33%)
         occurrences all number
    3
    Chills
         subjects affected / exposed
    4 / 24 (16.67%)
         occurrences all number
    6
    Fatigue
         subjects affected / exposed
    5 / 24 (20.83%)
         occurrences all number
    8
    Pain
         subjects affected / exposed
    2 / 24 (8.33%)
         occurrences all number
    2
    Gastrointestinal disorders
    Abdominal discomfort
         subjects affected / exposed
    2 / 24 (8.33%)
         occurrences all number
    2
    Abdominal distension
         subjects affected / exposed
    2 / 24 (8.33%)
         occurrences all number
    2
    Abdominal pain upper
         subjects affected / exposed
    2 / 24 (8.33%)
         occurrences all number
    3
    Diarrhoea
         subjects affected / exposed
    5 / 24 (20.83%)
         occurrences all number
    8
    Nausea
         subjects affected / exposed
    8 / 24 (33.33%)
         occurrences all number
    22
    Vomiting
         subjects affected / exposed
    4 / 24 (16.67%)
         occurrences all number
    7
    Respiratory, thoracic and mediastinal disorders
    Dyspnoea
         subjects affected / exposed
    6 / 24 (25.00%)
         occurrences all number
    12
    Dyspnoea exertional
         subjects affected / exposed
    2 / 24 (8.33%)
         occurrences all number
    4
    Nasal congestion
         subjects affected / exposed
    4 / 24 (16.67%)
         occurrences all number
    4
    Nasal obstruction
         subjects affected / exposed
    2 / 24 (8.33%)
         occurrences all number
    3
    Oropharyngeal pain
         subjects affected / exposed
    5 / 24 (20.83%)
         occurrences all number
    5
    Skin and subcutaneous tissue disorders
    Cold sweat
         subjects affected / exposed
    2 / 24 (8.33%)
         occurrences all number
    3
    Hyperhidrosis
         subjects affected / exposed
    2 / 24 (8.33%)
         occurrences all number
    2
    Pruritus
         subjects affected / exposed
    2 / 24 (8.33%)
         occurrences all number
    4
    Rash
         subjects affected / exposed
    2 / 24 (8.33%)
         occurrences all number
    2
    Psychiatric disorders
    Insomnia
         subjects affected / exposed
    2 / 24 (8.33%)
         occurrences all number
    2
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    2 / 24 (8.33%)
         occurrences all number
    5
    Back pain
         subjects affected / exposed
    5 / 24 (20.83%)
         occurrences all number
    12
    Musculoskeletal pain
         subjects affected / exposed
    4 / 24 (16.67%)
         occurrences all number
    4
    Myalgia
         subjects affected / exposed
    3 / 24 (12.50%)
         occurrences all number
    5
    Neck pain
         subjects affected / exposed
    2 / 24 (8.33%)
         occurrences all number
    2
    Pain in extremity
         subjects affected / exposed
    5 / 24 (20.83%)
         occurrences all number
    6
    Infections and infestations
    Lower respiratory tract infection
         subjects affected / exposed
    3 / 24 (12.50%)
         occurrences all number
    4
    Nasopharyngitis
         subjects affected / exposed
    4 / 24 (16.67%)
         occurrences all number
    5
    Pharyngitis
         subjects affected / exposed
    2 / 24 (8.33%)
         occurrences all number
    2
    Upper respiratory tract infection
         subjects affected / exposed
    2 / 24 (8.33%)
         occurrences all number
    2
    Viral infection
         subjects affected / exposed
    3 / 24 (12.50%)
         occurrences all number
    3
    Metabolism and nutrition disorders
    Hypoglycaemia
         subjects affected / exposed
    16 / 24 (66.67%)
         occurrences all number
    165

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    18 Oct 2013
    Change in forced vital capacity (FVC) upright from Baseline to Week 24 has been changed from a tertiary endpoint to a secondary endpoint. The measurements of IGF-1, IGF-2, and IGF-BP3 will now be performed at the same visits as the immunogenicity assessments (Baseline and Weeks 2, 4, 8, 12, 16, 20, and 24) instead of at Baseline, Week 12 and Week 24 as previously scheduled. The repeated measures analysis of MIP (and likewise of the secondary efficacy endpoints) has been replaced with an analysis of covariance (ANCOVA) approach. Separate assessments of the 6MWT are now used for Screening (to determine eligibility) vs. Baseline (used for statistical analysis). It has been emphasized that Baseline, not Screening, values will be used to calculate the change values used in statistical analyses. The use of antihistamines in the desensitization protocol has been clarified as optional. The use of any form of ventilatory assistance will now be captured as part of the assessment of concomitant medications before study and at each study visit. The Borg scale, rather than the modified Borg scale, will now be used. The patient-reported outcome (PRO) instruments have been removed as Appendices to the protocol. The collection of exploratory blood and urine samples has been clarified as an optional assessment. Administrative updates have been made to improve consistency and clarity.
    03 Feb 2014
    An extension phase of up to 240 weeks has been added to the study. The neutralizing antibody for BMN 701 activity has been removed. Sleep assessments and analyses have been added to the study. References to a “light meal” to be eaten prior to BMN 701 infusions have been changed to a “meal.” The Week 12 magnetic resonance imaging (MRI) assessment has been removed. Chest X-rays taken within 12 months of study enrollment will now satisfy the requirement for a Screening chest X-ray (previously the X-ray must have been within 2 months of study enrollment). The requirement for both a posterior-anterior (PA) and lateral chest X-ray views has been removed. A PA view alone will suffice. The Baseline genomic sampling will be tested looking for angiotensin converting enzyme (ACE) polymorphisms. The collection of exploratory blood and urine samples has been clarified a required assessment; the genetic/genomic testing of these samples is optional. Language regarding assessments which may be necessary upon transition of the product to a new clinical lot has been added. Administrative updates have been made to improve consistency and clarity.
    16 Apr 2014
    The interval of assessment between the pulmonary function tests and the 6-minute walk test (6MWT) has been reduced from 2 hours to 30 minutes, the interval between consecutive 6MWTs has been reduced to a minimum of one hour, and the interval between the 6MWT and the Quick Motor Function Test (QMFT) has been reduced from 4 hours to 1 hour. The QMFT assessments have been removed from the Week 6 and Week 18 visits. The post-infusion observation time has been reduced from 3 hours to 2 hours. The Screening window may be extended, upon approval by the Medical Monitor. The window for last rhGAA administration prior to the start of treatment BMN 701 has been changed from 14-28 days to 10-31 days. The window for assessment of GAA activity following an rhGAA treatment has been reduced from ≥ 14 days to ≥ 12 days. The timing of pharmacokinetic (PK) assessments for subjects receiving the complete PK analysis has been changed to remove the 8 hour post-infusion collection timepoint and add a timepoint 5 hours post-infusion, with the final collection at 6 hours. Administrative updates have been made to improve consistency and clarity.
    23 Apr 2015
    The anticipated number of patients in the study has been increased to up to approximately 70. An additional pharmacokinetic (PK) and immunogenicity assessment visit has been added to the Transition Period (after the subject starts to receive study drug from the new clinical lot). Subjects in the complete PK cohort will also have complete PK testing at weeks 12 and 24 following the clinical lot transition. Blood glucose monitoring has been added to the transition period visits. The sleep study inclusion criteria have been modified. Visit windows ( ± 7 days) have been added to each of the visits during the Extension Period. Language was added to make it clear that all infusions should be administered with a minimum of 7 days between consecutive infusions. Language regarding PK sampling at the time of infusion end has been modified to change “prior to line flush” to “after all the drug has infused, including that within the tubing”. Language regarding “infusion-associated” reactions has been changed to reference “hypersensitivity” reactions. Clarified what was meant by the need to have ACLS-certified personnel and emergency equipment “readily available” during infusions. Clarified that the Appendix 1 guidance for medical management of subjects with hypoglycemia are intended as a suggested course of action, not a requirement. Added language to allow for discretionary home blood glucose monitoring. Reference to subjects transitioning to a new clinical lot prior to Week 24 of the study have been removed. Section 7 has been updated to reflect the latest information available in the Investigator’s Brochure. The Appendix 1 hypoglycemia management guidelines have been updated to provide more complete guidance. The adverse event reporting language has been updated to clarify that all AEs with severity of grade 3 or higher should be reported as serious AEs, and conform with BioMarin's internal processes. The identity of the medical monitor has been updated.
    15 Apr 2016
    Expanded inclusion criteria to permit enrollment of subjects aged 12-17. Added as an exclusion criterion the need for invasive ventilatory support to exclude subjects with invasive ventilator support based on complete respiratory muscle paralysis. Added as an exclusion criterion moderate to severe respiratory or cardiac disease not related to Pompe, including, but not limited to, symptomatic asthma, angina, congestive heart failure, or chronic obstructive pulmonary disease (COPD), causing respiratory and functional limitations requiring concurrent treatment. Added as an exclusion criterion a history of limb fracture which impairs ambulation at the time of consent. Added as an exclusion criterion the concurrent use of smoking tobacco within 6 months prior to study enrollment due to significant comorbid conditions not related to LOPD that would limit ability to adequately ascertain efficacy of reveglucosidase alfa on respiratory function and endurance. Subjects who have experienced reductions in blood glucose with CTCAE severity ≥ grade 2 or AEs of “hypoglycemia” or “blood glucose decreased” during the Treatment Period should have glucose monitoring during Extension Period infusions at the same intervals indicated for the initial 24-week Treatment Period (every 30 minutes) until 12 weeks have passed without hypoglycemia more severe than CTCAE grade 1. At that point, monitoring may return to every 1 hour (±10 minutes) after the start of the infusion, for at least 2 hours following completion of the infusion. Clarified that the subject’s blood glucose level must be stable and ≥72 mg/dL (≥4.0 mmol/L) on the last two measurements prior to discharge. Clarified that blood glucose must be within normal limits (≥ 72 mg/dL [≥ 4.0 mmol/L]) before the infusion may be started.

    Interruptions (globally)

    Were there any global interruptions to the trial? No

    Limitations and caveats

    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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