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    Summary
    EudraCT Number:2021-003200-40
    Sponsor's Protocol Code Number:NGAM-11
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2021-06-11
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    H.4 THIRD COUNTRY IN WHICH THE TRIAL WAS FIRST AUTHORISED
    Expand All   Collapse All
    A. Protocol Information
    A.2EudraCT number2021-003200-40
    A.3Full title of the trial
    Multicenter, Prospective, Parallel Group, Open-label, Randomized Phase III Study to Evaluate Safety and Efficacy of Different PANZYGA Dose Regimens in Pediatric Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) Patients
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study to Evaluate Safety and Efficacy of Different PANZYGA Dose Regimens in Pediatric Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) Patients
    A.4.1Sponsor's protocol code numberNGAM-11
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorOctapharma
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportOctapharma
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationOctapharma
    B.5.2Functional name of contact pointClinical Trials Information
    B.5.3 Address:
    B.5.3.1Street Address1408 Main Street
    B.5.3.2Town/ cityAgawam
    B.5.3.3Post code01001
    B.5.3.4CountryUnited States
    B.5.6E-mailctgov@clinicalresearchmgt.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation Yes
    D.2.1.1.1Trade name Panzyga
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product namePanzyga
    D.3.4Pharmaceutical form
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNPanzyga
    D.3.9.3Other descriptive nameHUMAN NORMAL IMMUNOGLOBULIN (IV)
    D.3.9.4EV Substance CodeSUB12041MIG
    D.3.10 Strength
    D.3.10.1Concentration unit % percent
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) Yes
    D.3.11.7Plasma derived medicinal product Yes
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Chronic inflammatory demyelinating polyradiculoneuropathy
    E.1.1.1Medical condition in easily understood language
    Chronic inflammatory demyelinating polyradiculoneuropathy
    E.1.1.2Therapeutic area Diseases [C] - Immune System Diseases [C20]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the efficacy of 2 PANZYGA dose regimens in pediatric CIDP patients based on the percentage of patients with CIDP improvement
    E.2.2Secondary objectives of the trial
    - To evaluate the efficacy of 2 PANZYGA dose regimens by determining the percentage of patients with CIDP relapse.
    - To evaluate the safety of PANZYGA (occurrence of treatment-emergent adverse events [TEAEs] and infusion related TEAES).
    - To further evaluate the efficacy of PANZYGA (including time to improvement and time to relapse and subgroup analyses).
    - To assess the effect of PANZYGA on the modified Rankin scale (mRS).
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Age ≥2 years and ≤17 years.
    2. Patients with a diagnosis of definite or probable CIDP based on European Neuromuscular Center (ENMC) criteria.
    3. Clinical history of functional impairment due to CIDP, corresponding to an mRS score ≥2, but ≤5.
    4. Voluntarily given written informed consent (provided by patient’s parent or legal guardian) or assent (provided by patient, if age-appropriate per Independent Ethics Committee [IEC]/Institutional Research Board [IRB] requirements).
    E.4Principal exclusion criteria
    1. Patients with previously diagnosed CIDP who lack any CIDP symptoms.
    2. Patients with a known history of inherited neuropathy or a family history of inherited neuropathy.
    3. Patients who have previously failed immunoglobulin therapy for CIDP.
    4. Patients who received intravenous immunoglobulin (IVIG) within 8 weeks prior to the Baseline visit (washout phase). However, if a patient has clinical evidence of confirmed CIDP relapse during the washout phase (consistent with an in-crease in mRS of >1), they are eligible for trial enrolment.
    5. Patients with a history of deep vein thrombosis (DVT) in the past year, or pulmonary embolism ever.
    6. Patients on unstable (change in prescribed dose within the last 8 weeks) corticosteroids or rituximab use.
    7. Patients with known or suspected hypersensitivity, anaphylaxis or severe systemic response to immune-globulins, blood or plasma derived products, or any component of PANZYGA.
    8. Female patients who are breastfeeding, pregnant, or planning to become pregnant, or are unwilling to use an effective birth control method while on the study (acceptable methods of birth control for this study include: intrauterine device[IUD], hormonal contraception, male or female condom, spermicide gel, diaphragm, sponge, or cervical cap).
    9. Known human immunodeficiency virus (HIV), hepatitis B virus (HBV), and/or hepatitis C virus (HCV) infections.
    10. Severe liver and/or kidney disease (alanine aminotransferase [ALT] > 3 × upper limit of normal [ULN]; aspartate aminotransferase [AST] > 3 × ULN; and/or creatinine levels >44 μmol/L for children ages 2–3 years, >62 μmol/L for children ages 4–10, and >89 μmol/L for children ages 11–17 years.
    11. Known immunoglobulin (IgA) deficiency and antibodies against IgA.
    12. History of alcohol or drug abuse in the previous year, as per Investigator’s opinion.
    13. Unable or unwilling to comply with the study protocol.
    14. Receipt of any other investigational medicinal product (IMP) within 3 months be-fore study entry.
    15. Any other condition(s) that, in the Investigator’s opinion, makes it undesirable for the patient to participate in the study or may interfere with protocol compliance
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy endpoint of this study is the percentage (%) of patients with CIDP improvement at Week 24. CIDP improvement is defined as the following: Decrease in mRS of ≥1 point from the most recent score
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 24
    E.5.2Secondary end point(s)
    The secondary efficacy endpoints of this study are:
    - The percentage (%) of patients with CIDP relapse. CIDP relapse is defined as an increase in mRS by >1 point from most recent score.
    - Time to CIDP relapse or withdrawal for any other reason.
    - The percentage of patients with excellent response, defined by a mRS score of 0 or 1 in each arm at Week 24.

    The secondary safety endpoints of this study are:
    - Occurrence of all TEAEs.
    - Percentage of patients with TEAEs, serious TEAEs, and related TEAEs.
    - Rate of TEAEs, serious TEAEs, and related TEAEs per infusion.
    - Rate of mild, moderate, and severe TEAEs per infusion
    - Short-term tolerance variables, including vital signs.
    - Safety laboratory variables
    E.5.2.1Timepoint(s) of evaluation of this end point
    24 weeks
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Yes
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 Will this trial be conducted at a single site globally? No
    E.8.4 Will this trial be conducted at multiple sites globally? Yes
    E.8.6 Trial involving sites outside the EEA
    E.8.6.2Trial being conducted completely outside of the EEA Yes
    E.8.6.3Specify the countries outside of the EEA in which trial sites are planned
    United States
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months9
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 30
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 15
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 15
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients No
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Pediatric patients
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 30
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    None
    G. Investigator Networks to be involved in the Trial
    H.4 Third Country in which the Trial was first authorised
    H.4.1Third Country in which the trial was first authorised: United States
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