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    Clinical Trial Results:
    Post-Marketing Study to Evaluate the Efficacy and Safety of PANZYGA in Pediatric Patients with Chronic Immune Thrombocytopenia (ITP)

    Summary
    EudraCT number
    		 2019-004063-49
    Trial protocol
    		 Outside EU/EEA  
    Global end of trial date
    06 Dec 2023

    Results information
    Results version number
    		 v1(current)
    This version publication date
    17 Aug 2024
    First version publication date
    17 Aug 2024
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    NGAM-10
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03866798
    WHO universal trial number (UTN)
    		 -
    Other trial identifiers
    		 IND : 14121, BLA: 125587
    Sponsors
    Sponsor organisation name
    Octapharma USA, Inc
    Sponsor organisation address
    117 West Century Road Paramus, New Jersey, United States, 07652
    Public contact
    Octapharma Pharmazeutika Produktionsges.m.b.H, Clinical Research & Development, 0043 (1) 610- 320, ClinicalRDVienna@groups.octapharma.com
    Scientific contact
    Octapharma Pharmazeutika Produktionsges.m.b.H, Clinical Research & Development, 0043 (1) 610- 320, ClinicalRDVienna@groups.octapharma.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
    17 May 2024
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    06 Dec 2023
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    The primary objective is to evaluate the efficacy of PANZYGA in increasing the platelet count in pediatric patients with chronic ITP.
    Protection of trial subjects
    This trial was conducted in accordance to the principles of ICH- GCP, ensuring that the rights, safety and well-being of patients are protected and in consistency with the Declaration of Helsinki, national regulatory requirements and FDA Code of Federal Regulation. Inclusion and exclusion criteria were carefully defined in order to protect subjects from contraindications, interactions with other medication and risk factors associated with the investigational medicinal product. Throughout the study safety was assessed, such as monitoring of AEs and SAEs, safety lab results, prior and concomitant medication and vital signs.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    21 Jan 2020
    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 States: 6
    Worldwide total number of subjects
    6
    EEA total number of subjects
    0
    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)
    6
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    0
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 8 sites were initiated in the USA of which 5 sites enrolled patients in the study.

    Pre-assignment
    Screening details
    		 6 Patients with documented diagnosis of Chronic Immune Thrombocytopenia (ITP) were screened between 21-Jan-2020 and 19-Sep-2023 according to predefined in- and exclusion criteria.

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

    Arms
    Arm title
    		 Panzyga
    Arm description
    		 Each patient was administered panzyga by intravenous (IV) infusion at a dose of 1 g/kg body weight panzyga on Day 1 and optionally another dose of 1 g/kg panzyga on Day 3; body-weight dependent doses were based on each patient’s weight collected at Baseline.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Panzyga
    Investigational medicinal product code
    Other name
    Immune Globulin Intravenous Human 10%
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Each patient was administered panzyga by intravenous (IV) infusion at a dose of 1 g/kg body weight panzyga on Day 1 and optionally another dose of 1 g/kg panzyga on Day 3; doses were based on each patient’s weight collected at Baseline. Total panzyga doses were 1 g/kg for patients receiving only 1 dose on Day 1 and 2 g/kg for those receiving the optional Day 3 dose. Prior to the Day 3 infusion, Investigators assessed each patient’s pre-infusion platelet count, hematocrit, and hemoglobin, and if the platelet count was >50 × 10^9/L, the Day 3 infusion was not administered; If the platelet count was still ≤50 × 10^9/L, the Day 3 infusion was administered. If any parameters indicated a clinically relevant change such that, in the Investigator’s opinion, it was not safe to administer the second infusion, the patient was to be withdrawn from study treatment and then followed for safety through Day 32.

    Number of subjects in period 1
    		Panzyga
    Started
    6
    Completed
    6

    Baseline characteristics

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

    Reporting group values
    		 Overall Period Total
    Number of subjects
    		 6 6
    Age categorical
    Units: Subjects
        Children (2-11 years)
    		 6 6
    Gender categorical
    Units: Subjects
        Female
    		 2 2
        Male
    		 4 4

    End points

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    End points reporting groups
    Reporting group title
    Panzyga
    Reporting group description
    		 Each patient was administered panzyga by intravenous (IV) infusion at a dose of 1 g/kg body weight panzyga on Day 1 and optionally another dose of 1 g/kg panzyga on Day 3; body-weight dependent doses were based on each patient’s weight collected at Baseline.

    Primary: Platelet Count Increase

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    End point title
    		 Platelet Count Increase [1]
    End point description
    The primary efficacy parameter was a clinical response defined as an increase in platelet count at least once to ≥50 × 10^9/L within 7 days after the first infusion, i.e., by Day 8.
    End point type
    Primary
    End point timeframe
    within 7 days after the first infusion, i.e., by Day 8.
    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: Due to the low number of enrolled patients, the analyses included only data listings, a small number of summary tables, and a figure. No statistical analysis performed.
    End point values
    		 Panzyga
    Number of subjects analysed
    6
    Units: Responder
        Yes
    4
        Yes (%)
    67
        No
    2
        No (%)
    33
    No statistical analyses for this end point

    Secondary: Time to Platelet Response (Platelet count ≥ 50 x 10^9/L)

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    End point title
    		 Time to Platelet Response (Platelet count ≥ 50 x 10^9/L)
    End point description
    Response was defined as an increase in platelet count at least once to ≥50 × 10^9/L within 7 days after the first infusion, i.e., by Day 8.
    End point type
    Secondary
    End point timeframe
    Days from first infusion to the first time reaching a platelet count of ≥50 × 10^9/L.
    End point values
    		 Panzyga
    Number of subjects analysed
    6
    Units: count of subjects
        2 days
    3
        3 days
    1
        non-responders
    2
    No statistical analyses for this end point

    Secondary: Duration of Platelet Response (Platelet Count Maintained ≥ 50 x 10^9/L)

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    End point title
    		 Duration of Platelet Response (Platelet Count Maintained ≥ 50 x 10^9/L)
    End point description
    Response was defined as an increase in platelet count at least once to ≥50 × 10^9/L within 7 days after the first infusion, i.e., by Day 8.
    End point type
    Secondary
    End point timeframe
    number of days the platelet count remained above ≥50 × 10^9/L.
    End point values
    		 Panzyga
    Number of subjects analysed
    6
    Units: count of subjects
        6 days
    1
        7 days
    1
        33 days
    1
        34 days
    1
        non-responders
    2
    No statistical analyses for this end point

    Secondary: Maximum Platelet Count

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    End point title
    		 Maximum Platelet Count
    End point description
    maximum platelet count during the study.
    End point type
    Secondary
    End point timeframe
    the maximum platelet count during the study up to 32 days
    End point values
    		 Panzyga
    Number of subjects analysed
    6
    Units: count of subjects
        99 (10^9/L)
    1
        11 (10^9/L)
    1
        516 (10^9/L)
    1
        386 (10^9/L)
    1
        204 (10^9/L)
    1
        30 (10^9/L)
    1
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    AEs were collected from the start of the first infusion for the whole duration of the study
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    26.1
    Reporting groups
    Reporting group title
    Panzyga
    Reporting group description
    		 Each patient was administered panzyga by intravenous (IV) infusion at a dose of 1 g/kg body weight panzyga on Day 1 and optionally another dose of 1 g/kg panzyga on Day 3; body-weight dependent doses were based on each patient’s weight collected at Baseline.

    Serious adverse events
    		 Panzyga
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 6 (0.00%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Panzyga
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    4 / 6 (66.67%)
    Nervous system disorders
    Headache
         subjects affected / exposed
    2 / 6 (33.33%)
         occurrences all number
    2
    Blood and lymphatic system disorders
    Neutropenia
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Gastrointestinal disorders
    Nausea
         subjects affected / exposed
    2 / 6 (33.33%)
         occurrences all number
    2
    Vomiting
         subjects affected / exposed
    2 / 6 (33.33%)
         occurrences all number
    2
    Mouth haemorrhage
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    2
    Dental caries
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Respiratory, thoracic and mediastinal disorders
    Epistaxis
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Oropharyngeal pain
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Rhinorrhoea
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Musculoskeletal and connective tissue disorders
    Myalgia
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    21 May 2019
    Amendment 1: Comments provided to Sponsor by the FDA review were implemented : - A plan for Sponsor expedited safety reporting to FDA was added, along with additional Investigator SAE reporting requirements, as specified in 21 CFR 312.32. - Instructions for handling infusion-related adverse events were modified such that in the event of Grade 2 or higher infusion-related reactions the PANZYGA infusion would be stopped; the infusions may be resumed at a lower rate after the symptoms subside. - A new section - Managing Hypersensitivity Reactions - Specification of Pre-medication Use Prior to PANZYGA Infusions - Additional vital sign assessments after the start of the PANZYGA infusion, specifically to add vital signs assessments prior to any infusion rate change and to assess them every hour - Thrombopoietin receptor agonist use is prohibited during Screening through Day 32 unless patients have been on a stable dose for 3 weeks. - Clarification regarding Long-term Anti-prolific Agents or Attenuated Androgen Therapy
    05 Aug 2019
    Amendment 2: - FDA reviewed Study Protocol No. NGAM-10 Version 02, including Amendment 01 and requested to amend the protocol such that in the event of severe hypersensitivity reactions or anaphylaxis, any further Panzyga administration should be discontinued and not resumed. In addition the following has been amended: -Increasing Exclusionary Time Requirement after Splenectomy was implemented - Allowable Time Windows for Changing Infusion Rates was implemented - Allowable Time Window for Vital Sign Assessments Taken at the End of the Infusion was implemented - Clarification of Body Weight and Height Assessments - Reconsent of Patients who Turn 18 years old during study participation - Allowing Safety Assessment Visits to be performed locally by the Patient’s Primary Care Physician and/or Local Laboratory Services
    27 Apr 2020
    Amendment 3: Protocol has been amended to address considerations specified in the FDA Guidance on the Conduct of Clinical Trials of Medical Products during COVID-19 Pandemic, March 2020, rev. April 2020.

    Interruptions (globally)
    Were there any global interruptions to the trial? Yes
    Date
    Interruption
    Restart date
    06 Dec 2023
    Due to feasibility constraints with respect to finding pediatric patients with chronic ITP who were eligible and willing to participate, the study was terminated early after 6 patients were enrolled and treated.
    -

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

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