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    Clinical Trial Results:
    Lumasiran in hyperoxalaemic patients on haemodialysis

    Summary
    EudraCT number
    		 2022-002681-32
    Trial protocol
    		 DE  
    Global end of trial date
    20 Jan 2025

    Results information
    Results version number
    		 v1(current)
    This version publication date
    20 Feb 2026
    First version publication date
    20 Feb 2026
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    20018510
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT06225544
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Charité - Universitätsmedizin
    Sponsor organisation address
    Charitéplatz 1, Berlin, Germany, 10117
    Public contact
    Gerlineke Hawkins-van der Cingel, Campus Virchow-Klinikum Medizinische Klinik m.S. Nephrologie und Internistische Intensivmedizin, 49 304507530067, gerlineke.hawkins-van-der-cingel@charite.de
    Scientific contact
    Gerlineke Hawkins-van der Cingel, Campus Virchow-Klinikum Medizinische Klinik m.S. Nephrologie und Internistische Intensivmedizin, 49 304507530067, gerlineke.hawkins-van-der-cingel@charite.de
    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
    16 Dec 2025
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    20 Jan 2025
    Global end of trial reached?
    Yes
    Global end of trial date
    20 Jan 2025
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To assess if this medication can successfully lower plasma oxalate levels in patients with End Stage Kidney Disease on haemodialysis.
    Protection of trial subjects
    The study was conducted in accordance with the ICH E6 (R2) Guideline for Good Clinical Practice (GCP),with applicable local regulations (including European Directive 2001/20/EC, German Medicinal Products Act (AMG)), and with the ethical principles that have their origins in the Declaration of Helsinki.
    Background therapy
    		 Cardiovascular death remains the leading cause of mortality for patients with ESKD, with minimal advances to ameliorate this in recent years. Whilst traditional risk factors such as hypercholesterolemia and atherosclerosis are important, this does not fully explain the significantly increased risk for patients with ESKD. In the general population, the main causes of cardiovascular death are stroke and myocardial infarction, however the dialysis population is more likely to die from sudden cardiac death or recurrent heart failure. There is increasing scientific evidence that aside from traditional cardiovascular risk factors such as altered lipid metabolism; the dysregulated metabolism of certain amino acids play a role in inflammation and atherosclerosis. Reduced glycine-oxalate ratios have been demonstrated in both human and mice models with atherosclerosis. AGXT is a main driver of glycine biosynthesis and when deficient leads to rising glyoxylate which is subsequently converted to oxalate. In brief, this triggers pro-atherogenic pathways, increased inflammatory pathway signalling and superoxide accumulation. From our own preliminary studies we know that pre-dialysis oxalate levels do recover between dialysis sessions, indicating that supra-normal oxalate levels persist for the majority of the time for our patients. As laboratory work continues to investigate the role of oxalate in atherosclerosis, there remains limited improvements in cardiovascular outcomes for haemodialysis patients over recent decades. If this preliminary study can show that a new oxalate lowering agent is well tolerated and efficacious in this population, it would pave the way for larger trials to establish if lowering excessive serum oxalate can successfully lower cardiovascular mortality and morbidity.
    Evidence for comparator
    		 -
    Actual start date of recruitment
    01 Mar 2024
    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
    Germany: 29
    Worldwide total number of subjects
    29
    EEA total number of subjects
    29
    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)
    15
    From 65 to 84 years
    14
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 The trial was conducted at 2 site in Germany from March 1, 2024 to January 20, 2025.

    Pre-assignment
    Screening details
    		 36 patients were screened aged between ≥18 and ≤80 years old with ESKD and who are receiving chronic haemodialysis treatment, of whom 29 were randomised.

    Period 1
    Period 1 title
    overall trail (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Carer, Assessor

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Treatment group
    Arm description
    		 Lumasiran, an RNA interference (RNAi) therapeutic agent. Lumasiran targets the HAO1 gene (hydroxyacid oxidase 1 gene) which encodes glycolate oxidase. By degrading the mRNA for glycolate oxidase, this reduces the hepatic production of oxalate and increases the concentration of its precursor glycolate which is more readily excreted. Thus, reducing both the urinary and plasma oxalate levels. Lumasiran is conjugated to GalNAc which is a carbohydrate with a very high affinity for a highly expressed receptor in the liver, allowing for a targeted delivery system to the liver.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Lumasiran
    Investigational medicinal product code
    EMEA/H/C/005040
    Other name
    Lumasiran sodium
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Lumasiran was injected at the end of the dialysis session at a dose of 3mg/kg, monthly loading doses for 3 months followed by the first of the quarterly maintenance doses starting a month after the last loading dose. Patients received at the end of the first haemodialysis session of the week (i.e., after the long interval, either on a Monday or Tuesday). loading dose

    Arm title
    		 Placebo
    Arm description
    		 -
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Placebo (0.9% Sodium Chloride ) was injected at the end of the dialysis session. It was given given as three monthly loading doses followed by one further maintenance dose.

    Number of subjects in period 1
    		Treatment group Placebo
    Started
    16
    13
    Completed
    13
    13
    Not completed
    3
    0
         Adverse event, serious fatal
    2
    -
         Protocol deviation
    1
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Treatment group
    Reporting group description
    		 Lumasiran, an RNA interference (RNAi) therapeutic agent. Lumasiran targets the HAO1 gene (hydroxyacid oxidase 1 gene) which encodes glycolate oxidase. By degrading the mRNA for glycolate oxidase, this reduces the hepatic production of oxalate and increases the concentration of its precursor glycolate which is more readily excreted. Thus, reducing both the urinary and plasma oxalate levels. Lumasiran is conjugated to GalNAc which is a carbohydrate with a very high affinity for a highly expressed receptor in the liver, allowing for a targeted delivery system to the liver.

    Reporting group title
    Placebo
    Reporting group description
    		 -

    Reporting group values
    		 Treatment group Placebo Total
    Number of subjects
    		 16 13 29
    Age categorical
    Units: Subjects
        In utero
    		 0
        Preterm newborn infants (gestational age < 37 wks)
    		 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)
    		 0
        From 65-84 years
    		 0
        85 years and over
    		 0
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 56.8 ( 14.6 ) 66.7 ( 12.7 ) -
    Gender categorical
    Units: Subjects
        Female
    		 6 5 11
        Male
    		 10 8 18
    Diabetes mellitus Type 2
    Comorbidities
    Units: Subjects
        No
    		 13 11 24
        Yes
    		 3 2 5
    Ischaemic heart failure
    Comorbidities
    Units: Subjects
        No
    		 10 8 18
        Yes
    		 6 5 11
    Stroke
    Comorbidities
    Units: Subjects
        No
    		 14 12 26
        Yes
    		 2 1 3
    Peripheral vascular disease
    Comorbidities
    Units: Subjects
        No
    		 11 10 21
        Yes
    		 5 3 8
    Cardiac arrhythmia (no further specified)
    Comorbidities
    Units: Subjects
        No
    		 13 9 22
        Yes
    		 3 4 7
    Acute coronary syndrom
    Comorbidities
    Units: Subjects
        No
    		 16 12 28
        Yes
    		 0 1 1
    Structural heart disease
    Comorbidities
    Units: Subjects
        No
    		 11 7 18
        Yes
    		 5 6 11
    Renal history - Cause of ESKD
    *Other causes including: Obstructive disease/nephrectomy, IgA Nephropathy, FSGS, AA Amyloid, MCGN, Bartter syndrome, recurrent AKI & TIN
    Units: Subjects
        Diabetic nephropathy
    		 2 2 4
        Other causes including*
    		 13 6 19
        Polycystic kidney disease, autosomal dominant
    		 0 2 2
        Systemic lupus erythematosus
    		 1 1 2
        CKDu
    		 0 2 2
    Transplantation history
    Units: Subjects
        No
    		 16 10 26
        Yes
    		 0 3 3
    Current HD Access
    Units: Subjects
        Tunnelled haemodialysis catheter (THL)
    		 5 2 7
        Arteriovenous fistula (AVF)
    		 8 9 17
        Arteriovenous graft (AVG)
    		 3 2 5
    Beta blocker
    Regular Medications
    Units: Subjects
        No
    		 4 3 7
        Yes
    		 12 10 22
    ACE inhibitors / ARB
    Regular Medications
    Units: Subjects
        No
    		 6 4 10
        Yes
    		 10 9 19
    Anti-platelets
    Regular Medications
    Units: Subjects
        No
    		 12 9 21
        Yes
    		 4 4 8
    Anti-coagulation
    Units: Subjects
        No
    		 10 10 20
        Yes
    		 6 3 9
    Statins
    Units: Subjects
        No
    		 11 8 19
        Yes
    		 5 5 10
    24h urine volume
    Units: Subjects
        400 ml
    		 1 0 1
        500 ml
    		 3 0 3
        Anuric
    		 12 13 25
    BMI
    Units: kg/m²
        arithmetic mean (standard deviation)
    		 26.1 ( 5.8 ) 24.6 ( 4.1 ) -
    Systolic blood pressure
    Units: mmHg
        arithmetic mean (standard deviation)
    		 134.8 ( 24.6 ) 133.8 ( 18.2 ) -
    Diastolic blood pressure
    Units: mmHg
        arithmetic mean (standard deviation)
    		 74.4 ( 17.8 ) 66 ( 15.6 ) -
    Duration of dialysis
    Units: year
        arithmetic mean (standard deviation)
    		 20.8 ( 2.7 ) 18 ( 5.4 ) -
    Current average clearance
    Units: Kt/v
        arithmetic mean (standard deviation)
    		 1.4 ( 0.4 ) 1.5 ( 0.2 ) -
    Average IDW gains
    Units: kg
        arithmetic mean (standard deviation)
    		 2.2 ( 0.7 ) 2.7 ( 0.8 ) -
    Plasma Oxalate level
    Units: μmol/L
        arithmetic mean (standard deviation)
    		 40.7 ( 19.5 ) 33.7 ( 6.4 ) -

    End points

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    End points reporting groups
    Reporting group title
    Treatment group
    Reporting group description
    		 Lumasiran, an RNA interference (RNAi) therapeutic agent. Lumasiran targets the HAO1 gene (hydroxyacid oxidase 1 gene) which encodes glycolate oxidase. By degrading the mRNA for glycolate oxidase, this reduces the hepatic production of oxalate and increases the concentration of its precursor glycolate which is more readily excreted. Thus, reducing both the urinary and plasma oxalate levels. Lumasiran is conjugated to GalNAc which is a carbohydrate with a very high affinity for a highly expressed receptor in the liver, allowing for a targeted delivery system to the liver.

    Reporting group title
    Placebo
    Reporting group description
    		 -

    Primary: change Plasma Oxalate Levels by group and time point

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    End point title
    		 change Plasma Oxalate Levels by group and time point
    End point description
    End point type
    Primary
    End point timeframe
    from baseline up to 6 months
    End point values
    		 Treatment group Placebo
    Number of subjects analysed
    14 [1]
    13
    Units: μmol/L
    arithmetic mean (standard deviation)
        Month 1
    4.9 ( 19 )
    1.5 ( 5.1 )
        Month 2
    10.6 ( 20.8 )
    4.7 ( 4.1 )
        Month 3
    1.4 ( 8.3 )
    4.1 ( 5.4 )
        Month 4
    1.4 ( 9.3 )
    5.3 ( 5.7 )
        Month 5
    5.1 ( 7.8 )
    1.6 ( 7.6 )
        Month 6
    2.7 ( 6.7 )
    -3 ( 14 )
    Notes
    [1] - n= 14 Month 1-2 und n= 13 Month 3-6, because one person less due to lack of compliance
    Statistical analysis title
    		 Percentage change of plasma oxalate level
    Statistical analysis description
    The primary analysis (FAS) .The treatment effect along with a 95% confidence interval was estimated from a linear mixed model with percentage change of plasma oxalate level as response, and with treatment, time and their interaction as fixed effects, and patient as random effect adjusted for baseline plasma oxalate level. All estimates will be accompanied with 95% confidence intervals. The imputation was done separately by group.
    Comparison groups
    Treatment group v Placebo
    Number of subjects included in analysis
    27
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 < 0.05
    Method
    		 Mixed models analysis
    Parameter type
    		 Mean difference (final values)
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -
         upper limit
    		 -

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    overall trial
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 CTCAE
    Dictionary version
    5.0
    Reporting groups
    Reporting group title
    Treatment group
    Reporting group description
    		 -

    Reporting group title
    Placebo
    Reporting group description
    		 -

    Serious adverse events
    		 Treatment group Placebo
    Total subjects affected by serious adverse events
         subjects affected / exposed
    4 / 16 (25.00%)
    4 / 13 (30.77%)
         number of deaths (all causes)
    2
    0
         number of deaths resulting from adverse events
    0
    0
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Metastatic Urothelial carcinoma
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 13 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 1
    0 / 0
    Injury, poisoning and procedural complications
    Fracture
    Additional description: Fracture fibula
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 13 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Vascular disorders
    Peripheral ischemia
    Additional description: Acute limb ischaemia due to occlusion of right sided limb of EVAR prosthesis
         subjects affected / exposed
    0 / 16 (0.00%)
    1 / 13 (7.69%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Surgical and medical procedures
    Bowel resection after bowel ischemia
         subjects affected / exposed
    0 / 16 (0.00%)
    1 / 13 (7.69%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Elective femoral-popliteal bypass
         subjects affected / exposed
    0 / 16 (0.00%)
    1 / 13 (7.69%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Cardiac disorders
    Myocardial infarction
         subjects affected / exposed
    0 / 16 (0.00%)
    1 / 13 (7.69%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Multi-organ failure
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 13 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 1
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    pneumonia, atypical
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 13 (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: 1%
    Non-serious adverse events
    		 Treatment group Placebo
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    1 / 16 (6.25%)
    1 / 13 (7.69%)
    Skin and subcutaneous tissue disorders
    Pruritus
    Additional description: Mild itching
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 13 (0.00%)
         occurrences all number
    1
    0
    Infections and infestations
    Covid-19 infection
         subjects affected / exposed
    0 / 16 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    30 Apr 2024
    change of Chief Investigator

    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 planned recruitment number was not achieved. The reasons were the burden of comorbidity, particularly psychiatric comorbidity and reluctance for an injection/health anxiety post the COVID-19 pandemic.
    For support, Contact us.
    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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