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    Summary
    EudraCT Number:2022-004155-13
    Sponsor's Protocol Code Number:BMS_IM101-931
    National Competent Authority:Germany - PEI
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2023-01-27
    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 ConcernedGermany - PEI
    A.2EudraCT number2022-004155-13
    A.3Full title of the trial
    Study to investigate the restoring of an immunological balance during therapy with abatacept
    Abatacept restores immune system equilibrium (ARISE)
    Studie zur Untersuchung der Wiederherstellung eines immunologischen Gleichgewichts unter der Therapie mit Abatacept
    Abatacept stellt das immunologische Gleichgewicht wieder her (ARISE)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study to investigate the restoring of an immunological balance during therapy with abatacept
    Abatacept restores immune system equilibrium (ARISE)
    Studie zur Untersuchung der Wiederherstellung eines immunologischen Gleichgewichts unter der Therapie mit Abatacept
    Abatacept stellt das immunologische Gleichgewicht wieder her (ARISE)
    A.3.2Name or abbreviated title of the trial where available
    ARISE
    A.4.1Sponsor's protocol code numberBMS_IM101-931
    A.5.4Other Identifiers
    Name:DRKSNumber:DRKS00027556
    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 SponsorUniversitätsklinikum Freiburg
    B.1.3.4CountryGermany
    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 supportBristol-Myers Squibb GmbH & Co. KGaA
    B.4.2CountryGermany
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationUniversitätsklinikum Freiburg
    B.5.2Functional name of contact pointCoordinating Investigator
    B.5.3 Address:
    B.5.3.1Street AddressBreisacher Str. 115
    B.5.3.2Town/ cityFreiburg i. Br.
    B.5.3.3Post code79106
    B.5.3.4CountryGermany
    B.5.4Telephone number+49761270-77640
    B.5.5Fax number+49761270-77600
    B.5.6E-mailcci-studien@uniklinik-freiburg.de
    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 ORENCIA® Injektionslösung in einer Fertigspritze
    D.2.1.1.2Name of the Marketing Authorisation holderBristol-Myers-Squibb Pharma EEIG
    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 Solution for injection in pre-filled syringe
    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 INNAbatacept
    D.3.9.1CAS number 332348-12-6
    D.3.9.4EV Substance CodeSUB20635
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number125
    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) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product Yes
    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 Yes
    D.3.11.13.1Other medicinal product typeFusion protein
    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
    CVID patients confirmed according to ESID/PAGID criteria or related disorders which fulfill the diagnostic criteria for CVID and interstitial lung disease or granuloma diagnosed by chest CT positive for nodules, lines or ground-glass signs.
    OR
    CVID patients confirmed according to ESID/PAGID criteria or related disorders which fulfill the diagnostic criteria for CVID and Enteropathy diagnosed by endoscopy, clinical manifestation of diarrhea, severe bloating or/and malabsorption.
    In diese Studie werden Patienten mit CVID und verwandten Erkrankungen sowie ILD oder Enteropathie eingeschlossen. CVID wird nach den ESID/PAGID-Kriterien diagnostiziert, wie auch a) interstitielle Lungenerkrankung mit oder ohne Granulome, diagnostiziert durch Thorax-CT mit nachweisbaren Noduli, retikuläre Veränderungen oder Milchglastrübungen ODER
    b) eine zöliakieähnliche Enteropathie, die durch eine histologisch nachgewiesene lymphozytäre Enteropathie diagnostiziert wird.
    E.1.1.1Medical condition in easily understood language
    Common variable immunodeficiency (CVID). The common variable immunodeficiency (CVID) is a chronic disease caused by a malfunction of the immune system.
    Variabler Immundefekt. Das variable Immundefektsyndrom (common variable immunodeficiency = CVID) ist eine chronische Erkrankung, die auf einer Fehlsteuerung des Abwehrsystems beruht.
    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 20.1
    E.1.2Level PT
    E.1.2Classification code 10021449
    E.1.2Term Immunodeficiency common variable
    E.1.2System Organ Class 10021428 - Immune system disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To identify predictive, early markers of immune restoration under abatacept therapy in patients with CVID and related disorders.
    E.2.2Secondary objectives of the trial
    1) To molecularly describe immune restoration in CVID patients undergoing abatacept treatment by deep phenotyping
    2) To document safety of abatacept treatment in CVID patients with interstitial lung disease and /or autoimmune enteropathy
    3) To document efficacy of abatacept treatment in CVID patients with interstitial lung disease and /or autoimmune enteropathy
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Diagnosis of CVID according to ESID/PAGID criteria or diagnosis of related disorders which fulfill the diagnostic criteria for CVID
    2. A) Interstitial lung disease or granuloma diagnosed by chest CT positive for nodules, lines or ground-glass signs AND/OR
    B) Celiac-like enteropathy diagnosed by histologically proven lymphocytic enteropathy
    3. Male and female patients of age 18 years and above
    4. Signed written informed consent
    5. Patient able to understand and willing to follow the protocol, including s.c. application and adequate storage of study drug
    6. Patients receiving regular immunoglobulin replacement therapy with trough levels of IgG above 4g/l
    7. Patients have failed steroid monotherapy (including topical steroids for enteropathy) for a minimum of three months or do not tolerate steroid treatment
    8. Need for intervention due to pulmonary manifestation measured by either reduced oxygen saturation, drop of oxygen saturation under exercise or reduced DLCOcSB
    OR
    progress of interstitial lung disease measured by progressive
    changes in the CT scan of the lung
    OR
    Need for intervention due to gastrointestinal manifestation measured by stool frequency above three stools/day OR signs of malabsorption OR severe bloating
    OR
    Marsh score of 3 in the duodenal histology
    E.4Principal exclusion criteria
    Exclusion criteria:
    1. Patient without legal capacity who is unable to understand the nature, significance and consequences of the study
    2. Known diagnosis of a monogenic defect in CTLA-4 or LRBA
    3. Other immunosuppressive therapy including biologicals beyond steroid at screening phase. Between treatment with other biologicals or DMARDs and start of abatacept trial treatment the wash out period of the pretreatment must be kept. In case of pretreatment with rituximab, therapy must be stopped at least 3 months before inclusion into trial
    4. Previous treatment with abatacept
    5. Chronic or active infection or any major episode of infection requiring hospitalization or treatment with intravenous (i.v.) antibiotics within 30 days prior to registration
    6. Acute or uncontrolled chronic bacterial including tuberculosis or viral infection (including HIV, HBV, HCV, HEV, SARS-CoV2. Patients with a chronic Norovirus infection can be included).
    7. Subjects who have a present malignancy or previous malignancy within the last 5 years prior to screening (except documented history of cured non-metastatic squamous or basal cell skin carcinoma or cervical carcinoma in situ). Subjects who had a screening procedure that is suspicious for malignancy, and in whom the possibility of malignancy cannot be reasonably excluded following additional clinical, laboratory or other diagnostic evaluations
    8. Receipt of a live replication-competent virus vaccine within 3 months prior to first application of trial medication
    9. Serious uncontrolled concomitant disease not caused by CVID
    10. Prior hematopoietic stem cell transplantation (HSCT) or HSCT planned within next 12 months
    11. Known intolerance to study medication or any of the excipients
    12. Lack of consent to perform gastroscopy including biopsies in a patient with enteropathy
    13. Current or planned pregnancy, nursing period; WOCBP need to keep anti-conceptive measures until 14 weeks after EOT

    E.5 End points
    E.5.1Primary end point(s)
    Identification of predictive, early markers of immune restoration under abatacept therapy in patients with CVID and related disorders.
    E.5.1.1Timepoint(s) of evaluation of this end point
    After 6 months of treatment with Orencia within the clinical trial.
    E.5.2Secondary end point(s)
    1) In regard to immune reconstitution:
    - Differential signatures of RNA seq profiles of peripheral blood B and T cell populations before vs after treatment
    - Differential immune phenotype of peripheral blood B and T cell populations before vs after treatment
    - Differential serum protein composition before vs. after treatment
    2) In regard to safety:
    Number and type of severe infections (requiring intravenous treatment, hospitalization, ER and ICU treatment, opportunistic infection, death) and number, type and severity of all other (serious) adverse events ((S)AE) in relation to study drug
    3) In regard to efficacy in patients with ILD:
    - Borg Dyspnea scale
    - Lung function parameter (DLCOcSBVA), FVC, DLCOc/VA, TLC SB, pO2 at rest and under exercise (six-minute walking test)
    - Laboratory parameters: sIL-2-Receptor, Neopterin
    - CT scan lung: Global and specific scores for nodules, lines, consolidation and ground glass
    4) In regard to efficacy in patients with autoimmune enteropathy:
    - Stool frequency and consistency
    - Inflammatory Bowel Disease Questionnaire (IBDQ)
    - Gastrointestinal histology (for screening historical biopsies can be used)
    5) In regard to efficacy in all patients:
    - Relative reduction of steroid dose
    - Quality of life measured by SF-36, Chronic fatigue score(FACIT)
    - Overall survival
    E.5.2.1Timepoint(s) of evaluation of this end point
    After 6 months of treatment with Orencia within the clinical trial.
    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) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    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 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    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 years
    E.8.9.1In the Member State concerned months18
    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 No
    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) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 9
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 1
    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.4.1In the member state10
    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. After the end of the trial, further treatment is at the discretion of the treating physician according to routine.
    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 Decision2023-02-27
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2023-04-20
    P. End of Trial
    P.End of Trial StatusCompleted
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