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    Summary
    EudraCT Number:2019-002999-13
    Sponsor's Protocol Code Number:SCGAM-06
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2019-07-16
    Trial results View 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 number2019-002999-13
    A.3Full title of the trial
    CLINICAL PHASE 3 STUDY TO MONITOR THE SAFETY, TOLERABILITY, AND EFFICACY OF SUBCUTANEOUS HUMAN IMMUNOGLOBULIN (CUTAQUIG®) ADMINISTERED AT MODIFIED DOSING REGIMENS IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASES
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Safety, tolerability, and efficacy of subcutaneous human immunoglobulin (CUTAQUIG®) administered at modified dosing regimens in patients with primary immunodeficiency diseases
    A.4.1Sponsor's protocol code numberSCGAM-06
    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 organisationCRMG
    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 Cutaquig
    D.2.1.1.2Name of the Marketing Authorisation holderOctapharma
    D.2.1.2Country which granted the Marketing AuthorisationUnited States
    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 nameCutaquig
    D.3.4Pharmaceutical form
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNCutaquig
    D.3.9.3Other descriptive nameNORMAL IMMUNOGLOBULIN
    D.3.9.4EV Substance CodeSUB127253
    D.3.10 Strength
    D.3.10.1Concentration unit % (V/V) percent volume/volume
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number16.5
    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
    primary immunodeficiency
    E.1.1.1Medical condition in easily understood language
    primary immunodeficiency
    E.1.1.2Therapeutic area Diseases [C] - Immune System Diseases [C20]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10064859
    E.1.2Term Primary immunodeficiency syndrome
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The co-primary objectives of this study are to assess CUTAQUIG administered using
    the following infusion parameters:
    • Compare total IgG trough levels from weekly infusions to every other week
    infusions
    • Safety and tolerability when administered at increased infusion volumes at
    each infusion site
    • Safety and tolerability when administered at increased infusion flow rates at
    each infusion site
    • Safety and tolerability when administered on an every other week dosing
    regimen
    E.2.2Secondary objectives of the trial
    The secondary objectives of this study are to:
    • Assess the effect of CUTAQUIG on quality-of-life (QoL) measures
    • Obtain further data on the safety and efficacy of CUTAQUIG
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Age ≥2 years and ≤75 years.
    2. Confirmed diagnosis of primary immunodeficiency (PI) disease as defined by
    the European Society for Immunodeficiencies and Pan American Group for
    Immunodeficiency and requiring immunoglobulin replacement therapy due to
    hypogammaglobulinaemia or agammaglobulinaemia. Note: The exact type of
    PI disease will be recorded.
    3. Established on a consistent or stable mg/kg dose of any SCIG treatment for
    a minimum of 3 months prior to Screening. Note: patients entering Cohort 3
    must be on weekly SCIG infusions for a minimum of 12 weeks.
    4. Availability of the Immunoglobulin G (IgG) trough levels of 2 previous SCIG
    infusions within 1 year of Screening, with 1 trough level obtained within
    3 months prior to enrollment, and maintenance of trough serum IgG levels
    ≥5.0 g/L in 2 previous infusions. Patients with no prior IgG trough level within
    3 months prior to enrollment may use the Screening IgG trough level as their
    2nd reading.
    5. Voluntarily given, fully informed signed informed consent. For patients under
    the legal age of consent, voluntarily given, fully-informed, signed informed
    consent will be provided by patient’s parent or legal guardian, and assent will
    be provided by patient (per age-appropriate Institutional Review Board [IRB]
    requirements).
    6. Females of childbearing potential, who are not nursing and have no plans for
    pregnancy during the course of the study, have been using at least
    1 acceptable form of birth control for a minimum of 30 days prior to the
    Screening visit and must agree to use at least 1 acceptable method of
    contraception for 30 days after the last dose of CUTAQUIG. Acceptable
    methods include: intrauterine device (IUD), hormonal contraception, male or
    female condom, spermicide gel, diaphragm, sponge, cervical cap, or
    abstinence.
    7. For female patients of child-bearing potential, a negative result in a urine
    pregnancy test conducted at the Screening visit.
    8. Willingness to comply with all aspects of the protocol, including blood
    sampling, for the duration of the study.
    E.4Principal exclusion criteria
    1. Evidence of active infection within 4 weeks of Screening or during the
    Screening Period.
    2. Current or clinically-significant history of any cardiovascular, respiratory,
    hepatic, renal, gastrointestinal, endocrine, neurological, immunological
    (excluding PI), hematologic, and/or psychiatric disorder(s), or a history of any
    other illness that, in the opinion of the Investigator, might confound the results
    of the study, or pose additional risk to the patient by participation in the study.
    3. Known history of adverse reactions to immunoglobulin A (IgA) in other
    products.
    4. Body mass index (BMI) >40 kg/m2 for patients entering Cohort 2 or Cohort 3.
    There are no BMI restrictions for Cohort 1.
    5. Ongoing history of hypersensitivity or persistent reactions to blood or plasma
    derived products, or any component of the investigational product (such as
    Polysorbate 80).
    6. Requirement of any routine premedication for IgG administration.
    7. History of malignancies of lymphoid cells and immunodeficiency with
    lymphoma.
    8. Severe liver function impairment (aspartate aminotransferase [AST] or
    alanine aminotransferase [ALT] >3 times above upper limit of normal).
    9. Known protein-losing enteropathies or clinically significant proteinuria.
    10.Presence of renal function impairment (creatine >120 μM/L or creatinine
    >1.35 mg/dL), or predisposition for acute renal failure (eg, any degree of preexisting
    renal insufficiency or routine treatment with known nephritic drugs).
    11.Treatment with oral or parenteral steroids for ≥30 days, or when given
    intermittently or as bolus at daily doses ≥0.15 mg/kg when taken within
    30 days of Screening. Note: Short or intermittent courses of steroids (ie, a
    steroid burst) of >0.15 mg/kg/day is allowed for treatment of a short-term
    condition such as an asthma exacerbation.
    12.Treatment with immunosuppressive or immunomodulatory drugs (except
    Omalizumab).
    13.Use of HYQVIA (Immune Globulin Infusion 10% [Human] with Recombinant
    Human Hyaluronidase) within 3 months prior to first CUTAQUIG infusion.
    14.Live viral vaccination (such as measles, rubella, mumps, and varicella) within
    2 months prior to first CUTAQUIG infusion.
    15.Exposure to blood or any blood product or derivative, other than subcutaneous
    IgG used for regular PI disease treatment, within 3 months before
    the first CUTAQUIG infusion.
    16.Treatment with any investigational medicinal product within 3 months prior to
    first CUTAQUIG infusion. Note: Patients participating in Study SCGAM-03 will
    be allowed to enter this study without the 3-month waiting period for an
    Investigational Product. Patients receiving another SCIG product within
    3 months prior to the first CUTAQUIG infusion may be considered for
    enrollment after Sponsor approval.
    17.Presence of any condition that is likely to interfere with the evaluation of
    CUTAQUIG or satisfactory conduct of the trial.
    18.Known or suspected to abuse alcohol, drugs, psychotropic agents, or other
    chemicals within the past 12 months prior to first CUTAQUIG infusion.
    19.Known active or chronic hepatitis B, hepatitis C, or HIV infection. Past
    hepatitis B or hepatitis C infection that has been cured is allowed.
    E.5 End points
    E.5.1Primary end point(s)
    The co-primary objectives are to compare total IgG trough levels from weekly
    infusions to every other week infusions, to assess safety and tolerability of
    CUTAQUIG being administered according to 3 different infusion parameters, and to
    assess efficacy parameters when switching from weekly infusions to every other
    week infusions. These will be assessed using the following safety variables:
    • Change in individual total IgG trough levels from weekly infusions to every
    other week infusions
    • Occurrence of treatment-emergent AEs (TEAEs) throughout the entire
    Stabilization and Treatment Periods starting with the first infusion of
    investigational medicinal product (IMP)
    • Occurrence of TEAEs temporally associated with CUTAQUIG delivered
    during the Treatment Period
    • Occurrence of TEAEs temporally associated with CUTAQUIG delivered
    during the Stabilization Period
    • TEAEs by speed of infusion
    • Local infusion-site reactions
    • Laboratory parameters (hematology, clinical chemistry, and basic urinalysis)
    E.5.1.1Timepoint(s) of evaluation of this end point
    25 weeks
    E.5.2Secondary end point(s)
    Secondary objectives will evaluate quality of life, along with additional evaluations of
    CUTAQUIG safety and efficacy being administered according to 3 different infusion
    parameters. These will be assessed using the following variables:
    • Quality of Life questionnaires
    • Measurement of individual profiles of IgG trough levels over time (all cohorts)
    • Monitoring for all infectious disease occurrence, resolution, and antibiotic use
    • Occurrence of serious bacterial infections (SBIs)
    • Vital signs (blood pressure, pulse, body temperature, respiratory rate)
    E.5.2.1Timepoint(s) of evaluation of this end point
    25 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 No
    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 No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    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) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    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 No
    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 No
    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 years2
    E.8.9.2In all countries concerned by the trial months0
    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: 10
    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: 5
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 5
    F.1.2Adults (18-64 years) No
    F.1.2.1Number of subjects for this age range: 50
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 5
    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 Yes
    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 No
    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 65
    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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