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    Clinical Trial Results:
    An Open-label, Multicenter, Expanded Access Program For Asfotase Alfa (Human Recombinant Tissue-nonspecific Alkaline Phosphatase Fusion Protein) Treatment For Patients With Infantile - Or Juvenile-onset Hypophosphatasia (HPP)

    Summary
    EudraCT number
    2015-000809-39
    Trial protocol
    FR  
    Global end of trial date
    08 Mar 2018

    Results information
    Results version number
    v1(current)
    This version publication date
    23 Sep 2018
    First version publication date
    23 Sep 2018
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    AA-HPP-405
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT02496689
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    Alexion Pharmaceuticals, Inc.
    Sponsor organisation address
    100 College Street, New Haven, United States, 06510
    Public contact
    European Clinical Trial Information, Alexion Europe SAS, +33 147100606, clinicaltrials.eu@alexion.com
    Scientific contact
    European Clinical Trial Information, Alexion Europe SAS, +33 147100606, clinicaltrials.eu@alexion.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?
    Yes
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    08 Mar 2018
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    08 Mar 2018
    Global end of trial reached?
    Yes
    Global end of trial date
    08 Mar 2018
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To assess the safety and tolerability of subcutaneous (SC) injections of asfotase alfa administered 6 times weekly (at a dose of 1 milligram/kilogram [mg/kg] ) or 3 times weekly (at a dose of 2 mg/kg) at the discretion of the Investigator.
    Protection of trial subjects
    This study was conducted in accordance with International Conference on Harmonisation (ICH) Good Clinical Practice, and the principles of the Declaration of Helsinki, in addition to following the laws and regulations of the country or countries in which a study is conducted.
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    11 Sep 2015
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    France: 12
    Country: Number of subjects enrolled
    United States: 11
    Worldwide total number of subjects
    23
    EEA total number of subjects
    12
    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)
    1
    Children (2-11 years)
    9
    Adolescents (12-17 years)
    1
    Adults (18-64 years)
    11
    From 65 to 84 years
    1
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    This study was conducted at 5 investigational sites: 2 sites in the United States (US) and 3 sites in France

    Pre-assignment
    Screening details
    The study consisted of a screening period of up to 12 weeks prior to study initiation. The duration of treatment varied by participant and ranged from 56 to 588 days. The median (mean) duration of treatment was 470.0 (357.9) days.

    Period 1
    Period 1 title
    Overall Study (overall period)
    Is this the baseline period?
    Yes
    Allocation method
    Not applicable
    Blinding used
    Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    Perinatal/Infantile
    Arm description
    Participants received a total of 6 mg/kg of asfotase alfa each week (administered at a dosage regimen of 1 mg/kg 6 times per week or 2 mg/kg 3 times per week by SC injection, at the discretion of the Investigator). Participants were grouped based on age of onset of first symptoms of HPP (perinatal onset, in utero and at birth; infantile-onset, <6 months of age). Throughout the study, dose adjustments were made to account for changes in body weight.
    Arm type
    Experimental

    Investigational medicinal product name
    asfotase alfa
    Investigational medicinal product code
    Other name
    Strensiq®
    Pharmaceutical forms
    Solution for injection/infusion
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Asfotase alfa was administered subcutaneously. The dose used in this study is the approved dosage (6 mg/kg/week) used in the US, European Union (EU), and other countries. All participants received a total of 6 mg/kg of asfotase alfa each week (administered at a dosage regimen of 1 mg/kg 6 times per week or 2 mg/kg 3 times per week, at the discretion of the Investigator). Throughout the study, dose adjustments were made to account for changes in body weight. The maximum volume of medicinal product per injection was not to exceed 1.0 milliliter (mL). If more than 1.0 mL was required, multiple injections were administered at the same time. Throughout the study, injection sites were rotated among different body areas (the upper and lower thighs, buttocks, upper and lower abdomen, and upper arms) and were carefully monitored for sign(s) of potential reaction(s).

    Arm title
    Juvenile
    Arm description
    Participants received a total of 6 mg/kg of asfotase alfa each week (administered at a dosage regimen of 1 mg/kg 6 times per week or 2 mg/kg 3 times per week by SC injection, at the discretion of the Investigator). Participants were grouped based on age of onset of first symptoms of HPP (≥6 months to <18 years of age). Throughout the study, dose adjustments were made to account for changes in body weight.
    Arm type
    Experimental

    Investigational medicinal product name
    asfotase alfa
    Investigational medicinal product code
    Other name
    Strensiq®
    Pharmaceutical forms
    Solution for injection/infusion
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Asfotase alfa was administered subcutaneously. The dose used in this study is the approved dosage (6 mg/kg/week) used in the US, EU, and other countries. All participants received a total of 6 mg/kg of asfotase alfa each week (administered at a dosage regimen of 1 mg/kg 6 times per week or 2 mg/kg 3 times per week, at the discretion of the Investigator). Throughout the study, dose adjustments were made to account for changes in body weight. The maximum volume of medicinal product per injection was not to exceed 1.0 mL. If more than 1.0 mL was required, multiple injections were administered at the same time. Throughout the study, injection sites were rotated among different body areas (the upper and lower thighs, buttocks, upper and lower abdomen, and upper arms) and were carefully monitored for sign(s) of potential reaction(s).

    Number of subjects in period 1
    Perinatal/Infantile Juvenile
    Started
    12
    11
    Received at Least 1 Dose of Study Drug
    12
    11
    Completed
    12
    11

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Perinatal/Infantile
    Reporting group description
    Participants received a total of 6 mg/kg of asfotase alfa each week (administered at a dosage regimen of 1 mg/kg 6 times per week or 2 mg/kg 3 times per week by SC injection, at the discretion of the Investigator). Participants were grouped based on age of onset of first symptoms of HPP (perinatal onset, in utero and at birth; infantile-onset, <6 months of age). Throughout the study, dose adjustments were made to account for changes in body weight.

    Reporting group title
    Juvenile
    Reporting group description
    Participants received a total of 6 mg/kg of asfotase alfa each week (administered at a dosage regimen of 1 mg/kg 6 times per week or 2 mg/kg 3 times per week by SC injection, at the discretion of the Investigator). Participants were grouped based on age of onset of first symptoms of HPP (≥6 months to <18 years of age). Throughout the study, dose adjustments were made to account for changes in body weight.

    Reporting group values
    Perinatal/Infantile Juvenile Total
    Number of subjects
    12 11 23
    Age categorical
    Units: Subjects
        In utero
    0 0 0
        Preterm newborn infants (gestational age < 37 wks)
    0 0 0
        Newborns (0-27 days)
    0 0 0
        Infants and toddlers (28 days-23 months)
    1 0 1
        Children (2-11 years)
    5 4 9
        Adolescents (12-17 years)
    1 0 1
        Adults (18-64 years)
    5 6 11
        From 65-84 years
    0 1 1
        85 years and over
    0 0 0
    Gender categorical
    Units: Subjects
        Female
    8 7 15
        Male
    4 4 8
    Race
    Units: Subjects
        White
    4 7 11
        Not Reported
    8 4 12
    ethnicity
    Units: Subjects
        Hispanic or Latino
    1 0 1
        Not Hispanic or Latino
    7 10 17
        Not Reported
    4 1 5

    End points

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    End points reporting groups
    Reporting group title
    Perinatal/Infantile
    Reporting group description
    Participants received a total of 6 mg/kg of asfotase alfa each week (administered at a dosage regimen of 1 mg/kg 6 times per week or 2 mg/kg 3 times per week by SC injection, at the discretion of the Investigator). Participants were grouped based on age of onset of first symptoms of HPP (perinatal onset, in utero and at birth; infantile-onset, <6 months of age). Throughout the study, dose adjustments were made to account for changes in body weight.

    Reporting group title
    Juvenile
    Reporting group description
    Participants received a total of 6 mg/kg of asfotase alfa each week (administered at a dosage regimen of 1 mg/kg 6 times per week or 2 mg/kg 3 times per week by SC injection, at the discretion of the Investigator). Participants were grouped based on age of onset of first symptoms of HPP (≥6 months to <18 years of age). Throughout the study, dose adjustments were made to account for changes in body weight.

    Subject analysis set title
    Safety Set
    Subject analysis set type
    Safety analysis
    Subject analysis set description
    Safety Set: All participants who received ≥1 dose of study drug.

    Primary: Number of Participants Experiencing Serious Adverse Events (SAEs)

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    End point title
    Number of Participants Experiencing Serious Adverse Events (SAEs) [1]
    End point description
    The number of participants experiencing SAEs is presented for participants who received asfotase alfa in this open-label, expanded access study. The duration of treatment varied by participant, with a median duration of exposure of 502.5 days for participants in the perinatal/infantile group and 450.0 days for participants in the juvenile group. An adverse event (AE) was defined as any untoward medical occurrence in a participant administered a medicinal product that does not necessarily have a causal relationship with the treatment. AEs were classified as an SAE if the event met any one of the following criteria (at any dose): resulted in death, was life-threatening, resulted in hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect, was an important medical event. A summary of all SAEs and other non-serious AEs, regardless of causality, is located in the reported Adverse events module.
    End point type
    Primary
    End point timeframe
    Baseline, End of Program (EOP)
    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: Justification: Quantitative statistical analysis (for example, a p-value) was not performed for this end point. Only descriptive statistics were included.
    End point values
    Perinatal/Infantile Juvenile
    Number of subjects analysed
    12 [2]
    11 [3]
    Units: participants
    5
    0
    Notes
    [2] - Safety Set
    [3] - Safety Set
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Adverse event data were collected from baseline through the EOP visit.
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    20.1
    Reporting groups
    Reporting group title
    Perinatal/Infantile
    Reporting group description
    Participants were enrolled into this group based on age of onset of first symptoms of HPP (perinatal onset, in utero and at birth; infantile-onset, <6 months of age). Participants received a total of 6 mg/kg of asfotase alfa each week (administered at a dosage regimen of 1 mg/kg 6 times per week or 2 mg/kg 3 times per week by SC injection, at the discretion of the Investigator). Throughout the study, dose adjustments were made to account for changes in body weight.

    Reporting group title
    Juvenile
    Reporting group description
    Participants were enrolled into this group based on age of onset of first symptoms of HPP (≥6 months to <18 years of age). Participants received a total of 6 mg/kg of asfotase alfa each week (administered at a dosage regimen of 1 mg/kg 6 times per week or 2 mg/kg 3 times per week by SC injection, at the discretion of the Investigator). Throughout the study, dose adjustments were made to account for changes in body weight.

    Serious adverse events
    Perinatal/Infantile Juvenile
    Total subjects affected by serious adverse events
         subjects affected / exposed
    5 / 12 (41.67%)
    0 / 11 (0.00%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Injury, poisoning and procedural complications
    Ankle fracture
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 11 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Fibula fracture
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 11 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Asthma
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 11 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 11 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Haemorrhoids
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 11 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Musculoskeletal and connective tissue disorders
    Back pain
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 11 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Epiphysiolysis
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 11 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Hip deformity
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 11 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    Perinatal/Infantile Juvenile
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    12 / 12 (100.00%)
    10 / 11 (90.91%)
    Immune system disorders
    Hypersensitivity
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    General disorders and administration site conditions
    Injection site reaction
         subjects affected / exposed
    4 / 12 (33.33%)
    8 / 11 (72.73%)
         occurrences all number
    15
    50
    Injection site pain
         subjects affected / exposed
    2 / 12 (16.67%)
    3 / 11 (27.27%)
         occurrences all number
    6
    3
    Pyrexia
         subjects affected / exposed
    3 / 12 (25.00%)
    0 / 11 (0.00%)
         occurrences all number
    4
    0
    Fatigue
         subjects affected / exposed
    1 / 12 (8.33%)
    1 / 11 (9.09%)
         occurrences all number
    1
    1
    Injection site bruising
         subjects affected / exposed
    0 / 12 (0.00%)
    2 / 11 (18.18%)
         occurrences all number
    0
    8
    Injection site erythema
         subjects affected / exposed
    2 / 12 (16.67%)
    0 / 11 (0.00%)
         occurrences all number
    2
    0
    Chest pain
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    2
    Hypothermia
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    1
    Injection site haematoma
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 11 (0.00%)
         occurrences all number
    2
    0
    Pain
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    Psychiatric disorders
    Depressed mood
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    1
    Reproductive system and breast disorders
    Pelvic pain
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 11 (0.00%)
         occurrences all number
    2
    0
    Injury, poisoning and procedural complications
    Fall
         subjects affected / exposed
    0 / 12 (0.00%)
    2 / 11 (18.18%)
         occurrences all number
    0
    2
    Infusion related reaction
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    Investigations
    Crystal urine present
         subjects affected / exposed
    0 / 12 (0.00%)
    2 / 11 (18.18%)
         occurrences all number
    0
    2
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    1 / 12 (8.33%)
    1 / 11 (9.09%)
         occurrences all number
    2
    1
    Oropharyngeal pain
         subjects affected / exposed
    1 / 12 (8.33%)
    1 / 11 (9.09%)
         occurrences all number
    1
    1
    Asthma
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    Productive cough
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    Respiratory distress
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    1
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    Lymphadenopathy
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    1
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    1 / 12 (8.33%)
    1 / 11 (9.09%)
         occurrences all number
    1
    1
    Headache
         subjects affected / exposed
    3 / 12 (25.00%)
    0 / 11 (0.00%)
         occurrences all number
    3
    0
    Hypokinesia
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    Sensory loss
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    Eye disorders
    Cataract nuclear
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    Corneal deposits
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    1
    Diplopia
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    1
    Dry eye
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    Eyelid cyst
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    Photopsia
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    Ear and labyrinth disorders
    Deafness
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    Otorrhoea
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    Gastrointestinal disorders
    Constipation
         subjects affected / exposed
    3 / 12 (25.00%)
    0 / 11 (0.00%)
         occurrences all number
    3
    0
    Abdominal pain upper
         subjects affected / exposed
    1 / 12 (8.33%)
    1 / 11 (9.09%)
         occurrences all number
    1
    1
    Anal incontinence
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    1
    Defaecation urgency
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    1
    Diarrhoea
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 11 (0.00%)
         occurrences all number
    2
    0
    Gastrooesophageal reflux disease
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    Paraesthesia oral
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    Tooth loss
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    Toothache
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    1
    Vomiting
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    Hepatobiliary disorders
    Hepatic steatosis
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    1
    Renal and urinary disorders
    Nephrolithiasis
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    Renal mass
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    1
    Urinary incontinence
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    1
    Skin and subcutaneous tissue disorders
    Onychoclasis
         subjects affected / exposed
    0 / 12 (0.00%)
    2 / 11 (18.18%)
         occurrences all number
    0
    2
    Acne
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    Erythema
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    Hypertrichosis
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    Swelling face
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    1
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    1 / 12 (8.33%)
    2 / 11 (18.18%)
         occurrences all number
    5
    4
    Back pain
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    1
    Pain in extremity
         subjects affected / exposed
    1 / 12 (8.33%)
    1 / 11 (9.09%)
         occurrences all number
    2
    1
    Bone pain
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    1
    Epiphysiolysis
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    Hip deformity
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 11 (0.00%)
         occurrences all number
    3
    0
    Joint swelling
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    1
    Muscle contracture
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    2
    Muscle spasms
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    Musculoskeletal chest pain
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 11 (0.00%)
         occurrences all number
    4
    0
    Neck pain
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 11 (0.00%)
         occurrences all number
    3
    0
    Wrist deformity
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    Endocrine disorders
    Hyperparathyroidism
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    Hyperparathyroidism secondary
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    Metabolism and nutrition disorders
    Vitamin D deficiency
         subjects affected / exposed
    2 / 12 (16.67%)
    0 / 11 (0.00%)
         occurrences all number
    2
    0
    Infections and infestations
    Bronchitis
         subjects affected / exposed
    3 / 12 (25.00%)
    1 / 11 (9.09%)
         occurrences all number
    3
    1
    Nasopharyngitis
         subjects affected / exposed
    2 / 12 (16.67%)
    1 / 11 (9.09%)
         occurrences all number
    8
    1
    Rhinitis
         subjects affected / exposed
    3 / 12 (25.00%)
    0 / 11 (0.00%)
         occurrences all number
    4
    0
    Gastroenteritis
         subjects affected / exposed
    2 / 12 (16.67%)
    0 / 11 (0.00%)
         occurrences all number
    2
    0
    Sinusitis
         subjects affected / exposed
    1 / 12 (8.33%)
    1 / 11 (9.09%)
         occurrences all number
    2
    1
    Upper respiratory tract infection
         subjects affected / exposed
    1 / 12 (8.33%)
    1 / 11 (9.09%)
         occurrences all number
    1
    1
    Conjunctivitis
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    1
    Folliculitis
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 11 (0.00%)
         occurrences all number
    2
    0
    Influenza
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    1
    Pharyngitis streptococcal
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    Tracheitis
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    2
    Viral infection
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    03 Jun 2015
    The planned number of participants was changed from approximately 75 to 120 participants globally, to accurately reflect the number of participants that were planned for enrollment.

    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.
    The single-arm, open-label trial design, without any control group in this expanded access study, precluded any definitive conclusions about the safety or efficacy of asfotase alfa.
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