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    Clinical Trial Results:
    Single-Arm Study to Assess the Efficacy of UVADEX® (Methoxsalen) Sterile Solution in Conjunction With the THERAKOS® CELLEX® Photopheresis System in Pediatric Patients With Steroid-Refractory Acute Graft Versus Host Disease (aGvHD)

    Summary
    EudraCT number
    		 2014-004806-14
    Trial protocol
    		 DE   HU   GB   IT   ES   AT  
    Global end of trial date
    16 Jul 2019

    Results information
    Results version number
    		 v2(current)
    This version publication date
    09 Sep 2020
    First version publication date
    07 Aug 2020
    Other versions
    		 v1
    Version creation reason
    • Correction of full data set
    To correct three typographical errors

    Trial information

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    Trial identification
    Sponsor protocol code
    TKS-2014-001
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT02524847
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Therakos, Inc., a Mallinckrodt Company
    Sponsor organisation address
    1425 U.S. Route 206, Bedminster, NJ, United States, 07921
    Public contact
    Medical Information Call Center, Therakos, Inc., a Mallinckrodt Company, 1 800-844-2830 Ext 5, ClinicalTrials@mnk.com
    Scientific contact
    Medical Information Call Center, Therakos, Inc., a Mallinckrodt Company, 1 800-844-2830 Ext 5, ClinicalTrials@mnk.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 Aug 2019
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    16 Jul 2019
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    To evaluate the efficacy of extracorporeal photopheresis (ECP) in pediatric participants with steroid-refractory aGvHD.
    Protection of trial subjects
    This trial was conducted in accordance with the ethical principles of Good Clinical Practice, according to the ICH Harmonized Tripartite Guideline, which has its foundation in the Declaration of Helsinki.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    20 Jan 2016
    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
    United States: 10
    Country: Number of subjects enrolled
    Spain: 3
    Country: Number of subjects enrolled
    United Kingdom: 1
    Country: Number of subjects enrolled
    Austria: 2
    Country: Number of subjects enrolled
    France: 3
    Country: Number of subjects enrolled
    Germany: 2
    Country: Number of subjects enrolled
    Italy: 8
    Worldwide total number of subjects
    29
    EEA total number of subjects
    19
    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)
    2
    Children (2-11 years)
    17
    Adolescents (12-17 years)
    9
    Adults (18-64 years)
    1
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 Participants were recruited by multiple treatment centers in the United States and Europe.

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

    Arms
    Arm title
    		 Methoxsalen with ECP
    Arm description
    		 Participants received methoxsalen 20 μg/ml in conjunction with ECP procedure three times per week for Weeks 1 to 4, and two times per week for Weeks 5 to 12.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Methoxsalen
    Investigational medicinal product code
    298-81-7
    Other name
    Methoxalen Sterile Solution
    Pharmaceutical forms
    Solution for blood fraction modification
    Routes of administration
    Extracorporeal use
    Dosage and administration details
    Participants received methoxsalen 20 μg/ml in conjunction with extracorporeal (ECP) use procedure three times per week for Weeks 1 to 4, and two times per week for Weeks 5 to 12.

    Number of subjects in period 1
    		Methoxsalen with ECP
    Started
    29
    Completed
    15
    Not completed
    14
         Condition no longer requires treatment
    4
         Adverse event, non-fatal
    4
         Death
    1
         Unsatisfactory therapeutic effect
    4
         Reason not specified
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Overall Study
    Reporting group description
    		 -

    Reporting group values
    		 Overall Study Total
    Number of subjects
    		 29 29
    Age categorical
    Units: Subjects
        Infants and toddlers (28 days-23 months)
    		 2 2
        Children (2-11 years)
    		 17 17
        Adolescents (12-17 years)
    		 9 9
        Adults (18-64 years)
    		 1 1
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 8.6 ± 5.02 -
    Gender categorical
    Units: Subjects
        Female
    		 12 12
        Male
    		 17 17
    Ethnicity (NIH/OMB)
    Units: Subjects
        Hispanic or Latino
    		 5 5
        Not Hispanic or Latino
    		 24 24
    Race
    Units: Subjects
        Asian
    		 1 1
        Black or African American
    		 2 2
        White
    		 22 22
        Other
    		 4 4

    End points

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    End points reporting groups
    Reporting group title
    Methoxsalen with ECP
    Reporting group description
    		 Participants received methoxsalen 20 μg/ml in conjunction with ECP procedure three times per week for Weeks 1 to 4, and two times per week for Weeks 5 to 12.

    Subject analysis set title
    Stage 0 = No GvHD rash
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    No GvHD rash

    Subject analysis set title
    Stage 1 = Maculopapular rash on < 25% body surface area (BSA)
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Maculopapular rash on < 25% body surface area (BSA)

    Subject analysis set title
    Stage 2 = Maculopapular rash on 25-50% BSA
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Maculopapular rash on 25-50% BSA

    Subject analysis set title
    Stage 3 = Maculopapular rash on >50% BSA
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Maculopapular rash on >50% BSA

    Subject analysis set title
    Stage 4 = Generalized erythroderma plus bullous formation
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Generalized erythroderma plus bullous formation, which are blisters bigger than 5 mm across

    Subject analysis set title
    Stage 0 = Bilirubin < 2.0 mg/dL
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Bilirubin < 2.0 mg/dL

    Subject analysis set title
    Stage 1 = Bilirubin 2.0-3.0 mg/dL
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Bilirubin 2.0-3.0 mg/dL

    Subject analysis set title
    Stage 2 = Bilirubin 3.1-6.0 mg/dL
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Bilirubin 3.1-6.0 mg/dL

    Subject analysis set title
    Stage 3 = Bilirubin 6.1-15.0 mg/dL
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Bilirubin 6.1-15.0 mg/dL

    Subject analysis set title
    Stage 4 = Bilirubin > 15.0 mg/dL
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Bilirubin > 15.0 mg/dL

    Primary: Number of Participants Achieving Overall Response (OR) Using the Modified International Bone Marrow Transplant Registry (IBMTR) Severity Index at Week 4

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    End point title
    		 Number of Participants Achieving Overall Response (OR) Using the Modified International Bone Marrow Transplant Registry (IBMTR) Severity Index at Week 4 [1]
    End point description
    OR using the modified IBMTR Severity Index is defined as complete response (CR) + partial response (PR) as follows: • CR: complete resolution of all signs and symptoms of aGvHD in all evaluable organs without addition of next-line systemic treatment • PR: improvement of 1 stage in 1 or more aGvHD target organs without progression in others and without addition of next-line systemic treatment
    End point type
    Primary
    End point timeframe
    4 weeks
    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: It was not possible to create an analysis module in this database because there was only one arm.
    End point values
    		 Methoxsalen with ECP
    Number of subjects analysed
    29
    Units: Participants
    16
    No statistical analyses for this end point

    Secondary: Number of Participants With Adverse Events

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    End point title
    		 Number of Participants With Adverse Events
    End point description
    Clinically significant changes in vital signs, laboratory values and investigations are reported as adverse events. Summary data are provided below, with details listed in the adverse events module.
    End point type
    Secondary
    End point timeframe
    16 weeks
    End point values
    		 Methoxsalen with ECP
    Number of subjects analysed
    29
    Units: Participants
        Any serious AE
    12
        Non-serious TEAE at 5% threshold
    17
        Death for any cause
    3
    No statistical analyses for this end point

    Secondary: Percentage of Participants Achieving Overall Response (OR) Using Modified IBMTR Severity Index at Week 8

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    End point title
    		 Percentage of Participants Achieving Overall Response (OR) Using Modified IBMTR Severity Index at Week 8
    End point description
    OR using the modified IBMTR Severity Index is defined as complete response (CR) + partial response (PR) as follows: • CR: complete resolution of all signs and symptoms of aGvHD in all evaluable organs without addition of next-line systemic treatment • PR: improvement of 1 stage in 1 or more aGvHD target organs without progression in others and without addition of next-line systemic treatment
    End point type
    Secondary
    End point timeframe
    8 weeks
    End point values
    		 Methoxsalen with ECP
    Number of subjects analysed
    19
    Units: percentage of participants
        number (confidence interval 95%)
    73.7 (48.8 to 90.9)
    No statistical analyses for this end point

    Secondary: Percentage of Participants Achieving Overall Response (OR) Using Modified IBMTR Severity Index at Week 12

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    End point title
    		 Percentage of Participants Achieving Overall Response (OR) Using Modified IBMTR Severity Index at Week 12
    End point description
    OR using the modified IBMTR Severity Index is defined as complete response (CR) + partial response (PR) as follows: • CR: complete resolution of all signs and symptoms of aGvHD in all evaluable organs without addition of next-line systemic treatment • PR: improvement of 1 stage in 1 or more aGvHD target organs without progression in others and without addition of next-line systemic treatment
    End point type
    Secondary
    End point timeframe
    12 weeks
    End point values
    		 Methoxsalen with ECP
    Number of subjects analysed
    14
    Units: percentage of participants
        number (confidence interval 95%)
    78.6 (49.2 to 95.3)
    No statistical analyses for this end point

    Secondary: Duration of Response (Days) Within 16 Weeks Using Modified IBMTR Severity Index

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    End point title
    		 Duration of Response (Days) Within 16 Weeks Using Modified IBMTR Severity Index
    End point description
    Duration of first response is presented for participants whose disease progressed. Duration of response is defined in the following way: Participants whose response failed: Date at which 1st disease progression occurs - date of 1st response +1. Participants whose response did not relapse: Date of 16-week follow-up or final assessment prior to week 16 (if participant withdrew early) - date of 1st response.
    End point type
    Secondary
    End point timeframe
    16 weeks
    End point values
    		 Methoxsalen with ECP
    Number of subjects analysed
    18
    Units: days
        median (full range (min-max))
    13.5 (4 to 50)
    No statistical analyses for this end point

    Secondary: Overall Response Rate (ORR) According to the Modified Glucksberg Criteria

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    End point title
    		 Overall Response Rate (ORR) According to the Modified Glucksberg Criteria
    End point description
    ORR is defined as the percentage of participants who achieve an overall response after 4 weeks, 8 weeks, and 12 weeks of ECP treatment according to a scoring algorithm applied to calculate the grade of aGvHD using the modified Glucksberg Criteria.
    End point type
    Secondary
    End point timeframe
    4 weeks, 8 weeks, and 12 weeks
    End point values
    		 Methoxsalen with ECP
    Number of subjects analysed
    29
    Units: percentage of participants
    number (confidence interval 95%)
        Week 4 (n=24)
    50 (29.1 to 70.9)
        Week 8 (n=19)
    63.2 (38.4 to 83.7)
        Week 12 (n=14)
    78.6 (49.2 to 95.3)
    No statistical analyses for this end point

    Secondary: Cumulative Dose of Daily Steroids

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    End point title
    		 Cumulative Dose of Daily Steroids
    End point description
    Steroids administered from diagnosis of aGvHD to 12 weeks after initiation of ECP treatment.
    End point type
    Secondary
    End point timeframe
    12 weeks
    End point values
    		 Methoxsalen with ECP
    Number of subjects analysed
    29
    Units: mg
        median (full range (min-max))
    1917 (192 to 6895)
    No statistical analyses for this end point

    Secondary: Number of Participants With Skin Rated as Stage 0 - 4 Using the Modified Glucksberg Criteria

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    End point title
    		 Number of Participants With Skin Rated as Stage 0 - 4 Using the Modified Glucksberg Criteria
    End point description
    Number of participants whose skin was rated as Stage 0 - 4 using the modified Glucksberg criteria based on the Graft versus Host Disease (GvHD) rash - Stages are defined in the reporting group titles shown in the table.
    End point type
    Secondary
    End point timeframe
    4 weeks, 8 weeks, and 12 weeks
    End point values
    		 Stage 0 = No GvHD rash Stage 1 = Maculopapular rash on < 25% body surface area (BSA) Stage 2 = Maculopapular rash on 25-50% BSA Stage 3 = Maculopapular rash on >50% BSA Stage 4 = Generalized erythroderma plus bullous formation
    Number of subjects analysed
    29
    29
    29
    29
    29
    Units: Participants
        Week 4 (n=23)
    9
    8
    3
    3
    0
        Week 8 (n=18)
    11
    5
    1
    1
    0
        Week 12 (n=14)
    10
    2
    1
    1
    0
    No statistical analyses for this end point

    Secondary: Number of Participants With Liver Rated as Stage 0 - 4 Using the Modified Glucksberg Criteria

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    End point title
    		 Number of Participants With Liver Rated as Stage 0 - 4 Using the Modified Glucksberg Criteria
    End point description
    Number of participants whose liver was rated as Stage 0 - 4 on the modified Glucksberg criteria - Stages are based on level of bilirubin, as defined in the table's arm/group descriptions.
    End point type
    Secondary
    End point timeframe
    4 weeks, 8 weeks, and 12 weeks
    End point values
    		 Stage 0 = Bilirubin < 2.0 mg/dL Stage 1 = Bilirubin 2.0-3.0 mg/dL Stage 2 = Bilirubin 3.1-6.0 mg/dL Stage 3 = Bilirubin 6.1-15.0 mg/dL Stage 4 = Bilirubin > 15.0 mg/dL
    Number of subjects analysed
    29
    29
    29
    29
    29
    Units: Participants
        Week 4 (n=21)
    19
    1
    1
    0
    0
        Week 8 (n=13)
    13
    0
    0
    0
    0
        Week 12 (n=12)
    12
    0
    0
    0
    0
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Up to 16 weeks
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    21.0
    Reporting groups
    Reporting group title
    Methoxsalen with ECP
    Reporting group description
    		 Participants received methoxsalen 20 μg/ml in conjunction with ECP procedure three times per week for Weeks 1 to 4, and two times per week for Weeks 5 to 12.

    Serious adverse events
    		 Methoxsalen with ECP
    Total subjects affected by serious adverse events
         subjects affected / exposed
    12 / 29 (41.38%)
         number of deaths (all causes)
    3
         number of deaths resulting from adverse events
    0
    Vascular disorders
    Hypertension
         subjects affected / exposed
    1 / 29 (3.45%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Nervous system disorders
    Haemorrhage Intracranial
         subjects affected / exposed
    1 / 29 (3.45%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    2 / 29 (6.90%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    General Physical Health Deterioration
         subjects affected / exposed
    1 / 29 (3.45%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Blood and lymphatic system disorders
    Autoimmune Haemolytic Anaemia
         subjects affected / exposed
    1 / 29 (3.45%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Pancytopenia
         subjects affected / exposed
    1 / 29 (3.45%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Immune system disorders
    Acute Graft Versus Host Disease
         subjects affected / exposed
    2 / 29 (6.90%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Acute Graft Versus Host Disease In Intestine
         subjects affected / exposed
    1 / 29 (3.45%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Acute Graft Versus Host Disease In Liver
         subjects affected / exposed
    1 / 29 (3.45%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Graft Versus Host Disease
         subjects affected / exposed
    1 / 29 (3.45%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Respiratory Failure
         subjects affected / exposed
    1 / 29 (3.45%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Renal and urinary disorders
    Cystitis haemorrhagic
         subjects affected / exposed
    1 / 29 (3.45%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Renal Failure
         subjects affected / exposed
    1 / 29 (3.45%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    1 / 29 (3.45%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Infections and infestations
    Aspergilloma
         subjects affected / exposed
    1 / 29 (3.45%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Bk Virus Infection
         subjects affected / exposed
    1 / 29 (3.45%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Cellulitis
         subjects affected / exposed
    1 / 29 (3.45%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Cystitis Viral
         subjects affected / exposed
    1 / 29 (3.45%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Cytomegalovirus Infection
         subjects affected / exposed
    1 / 29 (3.45%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Device Related Infection
         subjects affected / exposed
    1 / 29 (3.45%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Hepatitis E
         subjects affected / exposed
    1 / 29 (3.45%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Metabolism and nutrition disorders
    Hyperkalaemia
         subjects affected / exposed
    1 / 29 (3.45%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Hypokalaemia
         subjects affected / exposed
    1 / 29 (3.45%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Hypomagnesaemia
         subjects affected / exposed
    1 / 29 (3.45%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Methoxsalen with ECP
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    17 / 29 (58.62%)
    Investigations
    Immunoglobulins Decreased
         subjects affected / exposed
    2 / 29 (6.90%)
         occurrences all number
    3
    Vascular disorders
    Hypertension
         subjects affected / exposed
    4 / 29 (13.79%)
         occurrences all number
    7
    Hypotension
         subjects affected / exposed
    3 / 29 (10.34%)
         occurrences all number
    6
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    3 / 29 (10.34%)
         occurrences all number
    4
    Immune system disorders
    Hypogammaglobulinaemia
         subjects affected / exposed
    3 / 29 (10.34%)
         occurrences all number
    3
    Gastrointestinal disorders
    Abdominal Pain
         subjects affected / exposed
    4 / 29 (13.79%)
         occurrences all number
    5
    Nausea
         subjects affected / exposed
    4 / 29 (13.79%)
         occurrences all number
    8
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    4 / 29 (13.79%)
         occurrences all number
    5
    Psychiatric disorders
    Depression
         subjects affected / exposed
    2 / 29 (6.90%)
         occurrences all number
    2
    Infections and infestations
    Clostridium Difficile Infection
         subjects affected / exposed
    2 / 29 (6.90%)
         occurrences all number
    2
    Cytomegalovirus Infection
         subjects affected / exposed
    2 / 29 (6.90%)
         occurrences all number
    2
    Pneumonia
         subjects affected / exposed
    2 / 29 (6.90%)
         occurrences all number
    2
    Metabolism and nutrition disorders
    Hyperglycaemia
         subjects affected / exposed
    3 / 29 (10.34%)
         occurrences all number
    3
    Hypocalcaemia
         subjects affected / exposed
    3 / 29 (10.34%)
         occurrences all number
    4
    Hyperkalaemia
         subjects affected / exposed
    2 / 29 (6.90%)
         occurrences all number
    2
    Hypophosphataemia
         subjects affected / exposed
    2 / 29 (6.90%)
         occurrences all number
    2
    Malnutrition
         subjects affected / exposed
    2 / 29 (6.90%)
         occurrences all number
    2

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    15 Mar 2016
    Changes made to provide more detailed information for the investigators and ensure study success. Extended the duration of the trial, adding time points at which to collect measurements. Adjusted inclusion/exclusion criteria and recording requirements, and added a risk/benefit statement.
    27 Jul 2017
    Modified inclusion and exclusion criteria and extended the recording period for adverse events.

    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.
    This study did have a notable limitation in its single-group study design. This may limit a more robust assessment vs standard of care alone for primary endpoint of overall response and secondary endpoints steroid sparing and disease progression.
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