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    Summary
    EudraCT Number:2021-005001-26
    Sponsor's Protocol Code Number:20-773/M
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2021-12-08
    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
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedNetherlands - Competent Authority
    A.2EudraCT number2021-005001-26
    A.3Full title of the trial
    Influencing Progression of Airway Disease in Primary Antibody Deficiency
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    The effect of high dose versus low dose antibody supplementation on airway disease in primary antibody defciency
    A.3.2Name or abbreviated title of the trial where available
    IPAD Trial
    A.4.1Sponsor's protocol code number20-773/M
    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 SponsorUMC Utrecht
    B.1.3.4CountryNetherlands
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportZonMW
    B.4.2CountryNetherlands
    B.4.1Name of organisation providing supportTakeda
    B.4.2CountryNetherlands
    B.4.1Name of organisation providing supportCSL Behring
    B.4.2CountryNetherlands
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationUMC Utrecht
    B.5.2Functional name of contact pointJoris van Montfrans
    B.5.3 Address:
    B.5.3.1Street AddressLundlaan 6
    B.5.3.2Town/ cityUtrecht
    B.5.3.3Post code3584EA
    B.5.3.4CountryNetherlands
    B.5.4Telephone number+31887554340
    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 Human normal immunoglobulin
    D.2.1.1.2Name of the Marketing Authorisation holderBaxter/CSL Behring/Sanquin/Takeda
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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.3.4Pharmaceutical form Concentrate and solvent for solution for injection/infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPInjection (Noncurrent)
    Intravenous use
    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 antibody deficiency:
    - Unclassified antibody deficiency (unPAD)
    - IgA deficiency
    - Specific polysaccharide antibody deficiency (SPAD)
    - IgG subclass deficiency (IgSD)
    - Common variable immunodeficiency (CVID)
    - Agammaglobulinemia (X-linked or otherwise)
    E.1.1.1Medical condition in easily understood language
    Antibody deficency not caused by another medical condition, such as cancer or infection, or immunosupressive or chemotherapeutic therapy.
    E.1.1.2Therapeutic area Diseases [C] - Immune System Diseases [C20]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Difference in mean AD and ILD scores (as measured with CT scanning) between t=0 and t=2 years in patients with standard vs higher Ig replacement therapy dosing.
    E.2.2Secondary objectives of the trial
    1. The incidence of symptomatic lower pulmonary infections in PAD patients with high Ig replacement therapy dosing versus standard Ig replacement therapy dosing.
    2. Number of physician diagnosed lower respiratory tract infections in patients with high vs standard Ig replacement therapy dosing.
    3. Number of hospital admissions and duration of hospital admissions for pulmonary complications (ao exacerbations of bronchiectasis)
    4. Outcomes of pulmonary function tests (specifically: Total Lung Capacity (TLC), Forced Expiratory Volume after 1 second (FEV1), CO diffusion) on t=0 and t=2 years in all patients
    5. Days missed from work / school in patients with high vs standard Ig replacement therapy dosing.
    6. Total therapeutic and preventive costs
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    In order to be eligible to participate in this study, a subject must meet all of the following criteria:
    1. Age 4-60 years
    2. Diagnosis of Primary Antibody Deficiency / Common Variable Immunodeficiency Disorder (see Appendix 1).
    3. Indication for immunoglobulin replacement therapy and/or treated with immunoglobulin replacement therapy
    4. Current IgG dosing 0.4 - 0.6 gr / kg / 3-4 weeks
    5. Receiving treatment and follow up for PAD by one of the physicians in the participating centers
    6. Written informed consent
    7. Normal lung status, or mild to medium severe pulmonary disease (measured by pulmonary function test and on CT scan, scored by an independent radiologist based on the following criteria):
    a. Baseline AD score < 5 and ILD score < 7, or;
    b. Baseline AD score > 5 and/or ILD score > 7 without clinical diagnosis of severe respiratory insufficiency (defined as: saturations in room air <92% and / or oxygen dependency).
    c. Baseline pulmonary function (FEV1 and FVC >70% expected for age and body weight / length)
    E.4Principal exclusion criteria
    A potential subject who meets any of the following criteria will be excluded from participation in this study:
    1. Age above 60 year
    2. Diagnosis of Combined Immunodeficiency (CID) disease at onset of study (see Appendix A). Explanation: Combined Immunodeficiency is featured by the occurrence of more viral infections and reactivations and thus less comparable to PAD.
    3. Severe pulmonary disease, determined by an independent radiologist:
    a. Baseline AD score > 5 and/or ILD score > 7, in combination with:
    i. Saccular bronchiectasis on CT scan, or;
    ii. Clinical diagnosis of severe respiratory insufficiency (defined as: (defined as: saturations in room air <92%, and/ or oxygen dependency).
    b. Baseline pulmonary function (FEV1 and FVC >70% expected for age and body weight / length)
    E.5 End points
    E.5.1Primary end point(s)
    The main outcome parameters of this study are rate of progression of AD an ILD after 24 months in patients receiving a higher dose of Ig medication versus patients receiving standard Ig dosing, and the associated decrease in health costs. To measure this main outcome parameter we will use a disease specific CT scoring method that was developed and validated specifically for monitoring of lung disease in PAD (9,10). The scoring method was shown to be accurate and reproducible.
    By protocol, CT scans are made at t = 0 and t = 2 years. It is however possible that clinical symptoms or situations arise during the study period within a participant, so that an additional CT scan is indicated. The reasons for making an extra CT scan (following current clinical practice guidelines) are:
    − Symptoms of dyspnea accompanied by low saturations (<92%)
    − A lung function (FVC or FEV1) below 80% of expected value for age and length / body weight
    − Abnormalities on a chest X-ray making a CT scan necessary (made for indications of extensive coughing, pain with breathing, or dyspnea for unknown reasons)

    If an extra CT scan is made, this may lead to an adjustment of clinical treatment (in which situation routine clinical guidelines will be followed). Any additional CT scans (apart from t=0 or t=2 years) will be evaluated during the analysis presented to the DSMB / DMB after one year. It is our experience that extra CT scanning is an unusual event in these patients. We thus expect a low number of additional CT scans. We thus expect that findings on additional CT scans will not lead to premature termination of the study. 
     
    The CT scan at t = 2 years must in principle be performed.
    E.5.1.1Timepoint(s) of evaluation of this end point
    At baseline and after 2 years
    E.5.2Secondary end point(s)
    1. Number of upper- and lower respiratory tract infections before onset of study and during study, i.e.: Sino pulmonary disease, otitis, pneumonia, collected by study CRF’s.
    2. Pulmonary symptoms will be evaluated on a daily basis (during two periods of 2 months)
    using a diary that can be directly entered in Castor.
    3. Days missed from school and work due to infections, measured by the PCQ (productivity cost questionnaire) instrument.
    4. Quality of life, measured with the EQ-5D questionnaire
    5. Ig dosing and IgG trough levels in intervention and in control group
    6. Total health costs (consisting of costs for health care professional visits, medication, hospitalizations, imaging and biochemical investigations) in intervention and control group, collected from electronic patient files.
    7. Immunological laboratory phenotype, collected from electronic patient files.
    8. Adverse events: reporting and monitoring of patients and Adverse Events (AEs), Serious Adverse Events (SAEs) and Suspected Unexpected Serious Adverse Reaction (SUSARs) will be done by an independent monitor.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Point 1 and 8 will be measured continiously.
    Point 2 will be evaluated during two periods of 2 months.
    Point 3,4 and 7 will be evaluated at baseline, after 12 months and after 24 months.
    Point 5 will be evaluated at baseline, after 1 month, after 2 months, after 6 months, after 8 months, after 12 months and after 24 months
    Point 7 will be evaluated at the end of the study
    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) No
    E.7.4Therapeutic use (Phase IV) Yes
    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 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 Yes
    E.8.2.3.1Comparator description
    Patients who do not receive a 33% dose increase of immunoglobuline replacement therapy
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned3
    E.8.5The trial involves multiple Member States No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    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.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    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: 50
    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: 25
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 25
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 50
    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 Yes
    F.3.3Specific vulnerable populations No
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    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.4.1In the member state100
    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
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2021-12-08
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-12-24
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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