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    Clinical Trial Results:
    A Phase 1/2, randomised, placebo-controlled, double-blind, multi-centre study to evaluate the efficacy and safety of OC5 to reduce urinary oxalate in subjects with primary hyperoxaluria

    Summary
    EudraCT number
    		 2012-005606-22
    Trial protocol
    		 DE   GB  
    Global end of trial date
    23 Jan 2015

    Results information
    Results version number
    		 v1(current)
    This version publication date
    19 May 2017
    First version publication date
    19 May 2017
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    OC5-DB-01
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT02012985
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    OxThera IP AB
    Sponsor organisation address
    Sturegatan 56, Stockholm, Sweden, 11436
    Public contact
    Director of Clinical Operations, OxThera AB, +46 86600223, anna.sjogren@oxthera.com
    Scientific contact
    Director of Clinical Operations, OxThera AB, +46 86600223, anna.sjogren@oxthera.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
    23 Mar 2015
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    23 Jan 2015
    Global end of trial reached?
    Yes
    Global end of trial date
    23 Jan 2015
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate the efficacy of OC5 to reduce urinary oxalate levels during 8 weeks in subjects with Primary Hyperoxaluria (PH).
    Protection of trial subjects
    Patients were observed in the clinics during the study visits. Physical exam and vital signs were taken at screening and at last study visit to the clinic. Local laboratory safety tests included haematology, chemistry and urinalysis were assessed at each study visit (week 0, 5, 10, 14).
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    06 Dec 2013
    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
    United Kingdom: 3
    Country: Number of subjects enrolled
    France: 12
    Country: Number of subjects enrolled
    Germany: 13
    Worldwide total number of subjects
    28
    EEA total number of subjects
    28
    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)
    9
    Adolescents (12-17 years)
    10
    Adults (18-64 years)
    9
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 This was a randomised, placebo-controlled, double-blind, multi-centre study conducted at 8 sites in Germany, France and the UK. First patient enrolled 06 Dec 2013, and last patient completed 23 Jan 2015. In total 44 patients were screened of which 28 patients were randomised.

    Pre-assignment
    Screening details
    		 Male and females aged 2 year or above (at least 5 years old in the UK) with a diagnosis of primary hyperoxaluria type I, II or III were to be screened for the study. Patients were screened and followed during a 4-week baseline period before randomised 1:1 to receive 8 to 10 weeks of treatment with OC5 or placebo.

    Period 1
    Period 1 title
    Randomisation
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Monitor, Data analyst, Carer, Assessor
    Blinding implementation details
    Placebo capsules had the same appearance and mode of administration as the active product and were filled with microcrystalline cellulose.

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Baseline OC5
    Arm description
    		 Patients randomised to receive treatment with OC5.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Oxabact
    Investigational medicinal product code
    OC5
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    OC5 in enteric-coated size-4 capsule at a dose of minimum 10^9 colony-forming units (CFU) per capsule. Administration orally with breakfast and dinner as one capsule twice daily, during 8-10 weeks.

    Arm title
    		 Baseline Placebo
    Arm description
    		 Patients randomised to placebo treatment.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Placebo
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    Placebo in enteric-coated size-4 capsule filled with microcrystalline cellulose. Administration orally with breakfast and dinner as one capsule twice daily, during 8-10 weeks.

    Number of subjects in period 1
    		Baseline OC5 Baseline Placebo
    Started
    14
    14
    Completed
    14
    14
    Period 2
    Period 2 title
    Treatment
    Is this the baseline period?
    		 No
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Monitor, Data analyst, Carer, Assessor

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 OC5 group
    Arm description
    		 Patients randomised to receive treatment with OC5.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Oxabact
    Investigational medicinal product code
    OC5
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    OC5 in enteric-coated size-4 capsule at a dose of minimum 10^9 colony-forming units (CFU) per capsule. Administration orally with breakfast and dinner as one capsule twice daily, during 8-10 weeks.

    Arm title
    		 Placebo
    Arm description
    		 Patients randomised to placebo treatment.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Placebo
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    Placebo in enteric-coated size-4 capsule filled with microcrystalline cellulose. Administration orally with breakfast and dinner as one capsule twice daily, during 8-10 weeks.

    Number of subjects in period 2
    		OC5 group Placebo
    Started
    14
    14
    Completed
    14
    14

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Baseline OC5
    Reporting group description
    		 Patients randomised to receive treatment with OC5.

    Reporting group title
    Baseline Placebo
    Reporting group description
    		 Patients randomised to placebo treatment.

    Reporting group values
    		 Baseline OC5 Baseline Placebo Total
    Number of subjects
    		 14 14 28
    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)
    		 0 0 0
        Children (2-11 years)
    		 4 5 9
        Adolescents (12-17 years)
    		 5 5 10
        Adults (18-64 years)
    		 5 4 9
        From 65-84 years
    		 0 0 0
        85 years and over
    		 0 0 0
    Gender categorical
    Units: Subjects
        Female
    		 4 9 13
        Male
    		 10 5 15
    Primary hyperoxaluria medical history
    Units: Subjects
        Type I
    		 13 13 26
        Type II
    		 0 1 1
        Type III
    		 1 0 1
    PH Diagnosis confirmed by
    Units: Subjects
        DNA screening
    		 11 12 23
        Enxyme deficiency
    		 3 1 4
        Elevated urine glycolate
    		 0 1 1
    Time since diagnosis
    Units: Days
        arithmetic mean (standard deviation)
    		 3193.3 ± 2038.3 3358.5 ± 1921.4 -
    Baseline plasma oxalate
    Units: micromole(s)/litre
        arithmetic mean (standard deviation)
    		 14.755 ± 8.935 14.664 ± 11.2 -
    Baseline estimated Glomerular Filtration Rate
    Units: mL/min/1.73 m2
        arithmetic mean (standard deviation)
    		 97.471 ± 38.666 123.111 ± 45.425 -
    Baseline urinary oxalate excretion (without centrifuging)
    Centrifuging the urine removed larger calcium oxalate crystals and thus reduced spontaneous variability due to random sampling of larger crystals.
    Units: mmol/24 h/1.73 m2
        arithmetic mean (standard deviation)
    		 1.733 ± 0.488 1.737 ± 0.696 -
    Baseline urinary oxalate excretion (with centrifuging)
    Centrifuging the urine removed larger calcium oxalate crystals and thus reduced spontaneous variability due to random sampling of larger crystals.
    Units: mmol/24 h/1.73 m2
        arithmetic mean (standard deviation)
    		 1.396 ± 0.521 1.456 ± 0.776 -

    End points

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    End points reporting groups
    Reporting group title
    Baseline OC5
    Reporting group description
    		 Patients randomised to receive treatment with OC5.

    Reporting group title
    Baseline Placebo
    Reporting group description
    		 Patients randomised to placebo treatment.
    Reporting group title
    OC5 group
    Reporting group description
    		 Patients randomised to receive treatment with OC5.

    Reporting group title
    Placebo
    Reporting group description
    		 Patients randomised to placebo treatment.

    Primary: Efficacy of OC5 in reducing urinary oxalate excretion levels during 8 weeks

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    End point title
    		 Efficacy of OC5 in reducing urinary oxalate excretion levels during 8 weeks
    End point description
    The primary endpoint was defined as absolute change in urinary oxalate excretion (mmol/24 h/1.73 m2) after 8 weeks treatment from baseline. Patients had to have daily urinary excretion of minimum 1 mmol/24 h/1.73 m2 to meet the inclusion criteria for the protocol. Patients were stratified for daily urinary oxalate excretion above and below 1.5 mmol/24 h/1.73 m2.
    End point type
    Primary
    End point timeframe
    Change after 8 weeks of treatment compared to baseline
    End point values
    		 OC5 group Placebo
    Number of subjects analysed
    14
    14
    Units: mmol/24 h/1.73 m2
        least squares mean (confidence interval 95%)
    0.042 (-0.162 to 0.245)
    -0.14 (-0.355 to 0.076)
    Statistical analysis title
    		 OC5 difference to placebo
    Statistical analysis description
    Based on a mixed-model repeated measures analysis of variance including treatment, visit and visit-by-treatment interaction and baseline crystal-bound oxalate as covariate
    Comparison groups
    OC5 group v Placebo
    Number of subjects included in analysis
    28
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.2174
    Method
    		 Mixed models analysis
    Confidence interval

    Secondary: Change in number of O. formigenes in faeces after 8 weeks of treatment

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    End point title
    		 Change in number of O. formigenes in faeces after 8 weeks of treatment
    End point description
    Measurements provided the number of O. formigenes bacteria (genotypes 1 and 2) and OC5 consists of O. formigenes derived from the human strain HC-1 of O. formigenes genotype 1. One subject in the study had detectable O. formigenes type 1 at the start of the study, albeit very low (SCR03-0005, in the active group). Three subjects in the active group and three subjects in the placebo group had detectable levels of O. formigenes type 2 during baseline. All subjects but one in the active group increased in O. formigenes type 1 count; the subject with no increase in O. formigenes type 1 had undetectable levels of any Oxalobacter. No O. formigenes type 1 were detectable in the placebo group during any time of the study. In terms of absolute change in number of O. formigenes in faeces, the LS mean difference from baseline after 8 weeks of treatment was 12,723,497 greater in the OC5 group than in the Placebo group (95% CI: 4,788,219–20,658,775; p=0.00023).
    End point type
    Secondary
    End point timeframe
    Change in faeces after 8 weeks of treatment compared to baseline
    End point values
    		 OC5 group Placebo
    Number of subjects analysed
    14
    14
    Units: amount
        number (confidence interval 95%)
    13814394 (7992437 to 19636352)
    1090897 (-4293285 to 6475081)
    Statistical analysis title
    		 OC5 difference to placebo
    Comparison groups
    OC5 group v Placebo
    Number of subjects included in analysis
    28
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority [1]
    P-value
    		 = 0.00023
    Method
    		 Mixed models analysis
    Confidence interval
    Notes
    [1] - Based on a mixed-model repeated measures analysis of variance including treatment, visit and visit-by-treatment interaction and baseline number of O.formigenes as a covariate.

    Secondary: Change in plasma oxalate concentration after 8 weeks of treatment

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    End point title
    		 Change in plasma oxalate concentration after 8 weeks of treatment
    End point description
    The LS mean difference between plasma oxalate concentration at baseline and after 8 weeks of treatment was 0.894 μmol/L (95% CI: -0.636, 2.423) in the OC5 group and 0.053 μmol/L (95% CI: -1.534, 1.640) in the Placebo group. The difference between the groups did not achieve statistical significance (0.841 μmol/L; 95% CI: -1.363, 3.045; p=0.4383).
    End point type
    Secondary
    End point timeframe
    Change after 8 weeks of treatment
    End point values
    		 OC5 group Placebo
    Number of subjects analysed
    14
    14
    Units: micromole(s)/litre
        least squares mean (confidence interval 95%)
    0.894 (-0.636 to 2.423)
    0.053 (-1.534 to 1.64)
    Statistical analysis title
    		 OC5 difference to placebo
    Statistical analysis description
    Based on a mixed-model repeated measures analysis of variance including treatment, visit and visit-by-treatment interaction and baseline plasma oxalate as covariate. Baseline level of plasma oxalate is defined as last non-missing and valid assessment before first dose of study drug.
    Comparison groups
    OC5 group v Placebo
    Number of subjects included in analysis
    28
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.4383
    Method
    		 Mixed models analysis
    Confidence interval

    Secondary: Change in urinary oxalate excretion after 4 weeks of treatment compared with baseline

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    End point title
    		 Change in urinary oxalate excretion after 4 weeks of treatment compared with baseline
    End point description
    The LS mean difference between urinary oxalate excretion at baseline and after 4 weeks of treatment was 0.007 mmol/24 h/1.73 m2 (95% CI: -0.186, 0.201) in the OC5 group and -0.177 (95% CI: -0.358, 0.005) in the Placebo group. The difference of 0.184 mmol/24 h/1.73 m2 (95% CI: -0.079, 0.448) failed to achieve statistical significance (p=0.1617). Similar results were obtained when the samples were centrifuged.
    End point type
    Secondary
    End point timeframe
    Change after 4 weeks of treatment compared with baseline
    End point values
    		 OC5 group Placebo
    Number of subjects analysed
    14
    14
    Units: mmol/24 h/1.73 m2
        least squares mean (confidence interval 95%)
    0.007 (-0.186 to 0.201)
    -0.177 (-0.358 to 0.005)
    Statistical analysis title
    		 OC5 difference to placebo
    Statistical analysis description
    Based on a mixed-model repeated measures analysis of variance including treatment, stratification variable, visit and visit-by-treatment interaction. Baseline level of urinary oxalate is the mean from eligible 24-h urine collections taken before first dose of study drug.
    Comparison groups
    OC5 group v Placebo
    Number of subjects included in analysis
    28
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.1617
    Method
    		 Mixed models analysis
    Confidence interval

    Secondary: Estimated glomerular filtration rate

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    End point title
    		 Estimated glomerular filtration rate
    End point description
    Estimated glomerular filtration rate reported as absolut change after 8 weeks of treatment compared to baseline value.
    End point type
    Secondary
    End point timeframe
    Change after 8 weeks of treatment compared to baseline
    End point values
    		 OC5 group Placebo
    Number of subjects analysed
    14
    14
    Units: mL/min/1.73 m2
        arithmetic mean (standard deviation)
    -3.199 ± 13.594
    -6.529 ± 15.135
    No statistical analyses for this end point

    Secondary: Occurence of nephrocalcinosis

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    End point title
    		 Occurence of nephrocalcinosis
    End point description
    Nephrocalcinosis was diagnosed using renal ultrasound. At baseline, nephrocalcinosis was observed in a total of 19 kidneys in the OC5 group and in 11 kidneys in the Placebo group. No difference was observed between OC5 and Placebo with respect to reduction in nephrocalcinosis cases. In both treatment groups, nephrocalcinosis was observed in two fewer kidneys at the Week 8 clinical visit than at baseline.
    End point type
    Secondary
    End point timeframe
    Number of occurences at study baseline and after 8 weeks of treatment
    End point values
    		 Baseline OC5 Baseline Placebo OC5 group Placebo
    Number of subjects analysed
    14 [2]
    14 [3]
    14 [4]
    14 [5]
    Units: number of nephrocalcinosis
        Left kidney
    10
    6
    9
    5
        Right kidney
    9
    5
    8
    4
    Notes
    [2] - Baseline
    [3] - Baseline
    [4] - 8 weeks treatment
    [5] - 8 weeks treatment
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Each subject was questioned about AEs at each clinic visit/telephone follow-up following initiation of treatment.
    Adverse event reporting additional description
    Each subject was asked the question "Since your last clinic visit have you had any health problems?" The information could also be obtained from signs and symptoms detected during each examination, observed by the study personnel or spontaneous reports from the study subjects.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    16.1
    Reporting groups
    Reporting group title
    OC5 group
    Reporting group description
    		 Patients randomised to receive treatment with OC5.

    Reporting group title
    Placebo
    Reporting group description
    		 Patients randomised to placebo treatment.

    Serious adverse events
    		 OC5 group Placebo
    Total subjects affected by serious adverse events
         subjects affected / exposed
    3 / 14 (21.43%)
    0 / 14 (0.00%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Injury, poisoning and procedural complications
    Radius fracture
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 14 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Renal and urinary disorders
    Pyelonephritis
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 14 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Renal colic
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 14 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 OC5 group Placebo
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    10 / 14 (71.43%)
    12 / 14 (85.71%)
    General disorders and administration site conditions
    Chest pain
         subjects affected / exposed
    1 / 14 (7.14%)
    1 / 14 (7.14%)
         occurrences all number
    4
    1
    Influenza like illness
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    Pyrexia
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    Reproductive system and breast disorders
    Dysmenorrhoea
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    1
    Respiratory, thoracic and mediastinal disorders
    Oropharyngeal pain
         subjects affected / exposed
    2 / 14 (14.29%)
    1 / 14 (7.14%)
         occurrences all number
    2
    1
    Cough
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    2
    Epistaxis
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    Asthma
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    1
    Psychiatric disorders
    Depression
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    Investigations
    Red blood cells urine positive
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    1
    Injury, poisoning and procedural complications
    Radius fracture
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    Nervous system disorders
    Headache
         subjects affected / exposed
    1 / 14 (7.14%)
    5 / 14 (35.71%)
         occurrences all number
    1
    5
    Dizziness
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    Ear and labyrinth disorders
    Ear pain
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    1
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    3 / 14 (21.43%)
    2 / 14 (14.29%)
         occurrences all number
    3
    2
    Abdominal pain upper
         subjects affected / exposed
    1 / 14 (7.14%)
    1 / 14 (7.14%)
         occurrences all number
    4
    2
    Vomiting
         subjects affected / exposed
    2 / 14 (14.29%)
    0 / 14 (0.00%)
         occurrences all number
    3
    0
    Diarrhoea
         subjects affected / exposed
    0 / 14 (0.00%)
    2 / 14 (14.29%)
         occurrences all number
    0
    2
    Abdominal discomfort
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    Change of bowel habit
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    Constipation
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    Frequent bowel movements
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    Flatulence
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    1
    Skin and subcutaneous tissue disorders
    Acne
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    Rash
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    Skin reaction
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    Renal and urinary disorders
    Renal colic
         subjects affected / exposed
    2 / 14 (14.29%)
    0 / 14 (0.00%)
         occurrences all number
    3
    0
    Renal pain
         subjects affected / exposed
    2 / 14 (14.29%)
    0 / 14 (0.00%)
         occurrences all number
    2
    0
    Calculus urethral
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    Pyelonephritis
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    Musculoskeletal and connective tissue disorders
    Back pain
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    Myalgia
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    1
    Infections and infestations
    Rhinitis
         subjects affected / exposed
    0 / 14 (0.00%)
    2 / 14 (14.29%)
         occurrences all number
    0
    3
    Gastroenteritis
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    Nasopharyngitis
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    Upper respiratory tract infection
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    Laryngitis
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    1
    Tonsillitis
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    1

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    03 May 2013
    Amendment 1 (which related to Rev 3 of the protocol and was the first submitted protocol in France and the United Kingdom): This amendment removed inclusion criterion 4 (historic values of urinary oxalate); clarified details on telephone follow-up, clarified that ascorbic acid preparations were to be avoided during the study period, and defined abnormal safety laboratory limits for serum bicarbonate and blood pH.
    04 Sep 2013
    Amendment 2 (which related to Version 4 of the protocol): This amendment defined criteria of the early termination of the study, corrected an error in the table of drug potency, clarified instructions on how to handle potential infections due to study medication, included information on burden, risk threshold and risk:benefit assessment, and clarified instructions on how to handle any elevated formate levels.
    04 Sep 2013
    Amendment 3 and 4 (which related to Version 5 and 6 of the protocol in Germany and the United Kingdom respectively): These amendments were local to Germany and the UK, respectively. In Germany, Amendment 3 concerned sequential enrolment with adult subjects being recruited before children. In the UK, amendment 4 included a change of inclusion criterion 2 to state “Male or female subjects ≥5 years of age”.
    10 Jun 2014
    Amendment 5 (which related to Version 7, 8 and 9 of the protocol in France, Germany and the United Kingdom respectively): This amendment included the following changes: • Change in inclusion criteria number 3 to also include subjects with PH type II or III. • Correction of inclusion criteria number 4 to a mean urinary oxalate excretion ≥1 mmol/24 h/1.73 m2 (previously >1 mmol/24 h/1.73 m2). • Update of Section 7.2.2 (Prohibited Medications), Section 10.5.2 (Systemic Infections) and Section 11.1 (Study Population), with regard to antibiotic treatment. • Clarification in Section 5.2.2 and Section 9.1 that an additional urine collection during baseline may be scheduled in case of incomplete collection(s) or accidental loss of a collection during baseline. • Clarification of data imputation for end of treatment (Week 14) in Section 11.8.3. • Adjustment of the study timelines in line with actual study progress. Furthermore, due to issues with subject recruitment, it was necessary to add an eighth clinical site (Hôpital Robert Debré, Paris). A substantial amendment with information on the new site was sent to the French Ethics Committee SudEst II on 04 June 2014. Approval was given by SudEst II for the new site on 18 June 2014. A notification was also sent to ANSM on 04 June 2014 regarding the new clinical site. Approval was not required from ANSM. However, no subjects were recruited at this site.

    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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