E.1 Medical condition or disease under investigation |
E.1.1 | Medical 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 |
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E.1.1.1 | Medical condition in easily understood language |
Patients with common variable immunodeficiency who suffer from inflammatory, non- infectious lung disease diagnosed by chest CT |
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E.1.1.2 | Therapeutic area | Diseases [C] - Immune System Diseases [C20] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 18.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10022611 |
E.1.2 | Term | Interstitial lung disease |
E.1.2 | System Organ Class | 10038738 - Respiratory, thoracic and mediastinal disorders |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 18.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10021449 |
E.1.2 | Term | Immunodeficiency common variable |
E.1.2 | System Organ Class | 10021428 - Immune system disorders |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To assess the safety of Abatacept for interstitial lung disease in patients with CVID and related disorders |
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E.2.2 | Secondary objectives of the trial |
To assess the efficacy of Abatacept on interstitial lung disease in patients with CVID and related disorders |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Diagnosis of CVID according to ESID/PAGID criteria or diagnosis of related disorders which fulfill the diagnostic criteria for CVID 2. Interstitial lung disease or granuloma diagnosed by chest CT positive for nodules, lines or ground-glass signs 3. Age 18 years and above 4. Signed written informed consent 5. Not responsive to steroid monotherapy for a minimum of three months or intolerance of steroid therapy 6. Need for intervention 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
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E.4 | Principal exclusion criteria |
1. Patient without legal capacity who is unable to understand the nature, significance and consequences of the study 2. Other immunosuppressive therapy including biologicals beyond steroid at screening phase 3. Previous treatment with Abatacept 4. Active Hepatitis B infection or tuberculosis infection 5. Other Uncontrolled infection 6. Lymphoma within the past 5 years 7. Pregnancy indicated by positive urine pregnancy test at screening 8. Breast feeding patients 9. Fertile patients refusing to use safe contraceptive methods during the study 10. Simultaneous participation in other interventional clinical trials
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E.5 End points |
E.5.1 | Primary end point(s) |
(1) Number and type of severe infections (intravenous treatment, hospitalization, ICU, opportunistic infection, death) under treatment (2) Number, type and severity of all other (serious) adverse events ((S)AE) in relation to study drug
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
(1) 12 months retrospective documentation before BaselineVisit (Day 0), BaselineVisit (Day 0), 3 months (Visit 1), 6 months (Visit 2), 12 months (Final Visit) (2) during screening up to 3 months before BaselineVisit (Day 0), BaselineVisit (Day 0), 3 months (Visit 1), 6 months (Visit 2), 12 months (Final Visit), 13 months (Follow-up via phone call)
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E.5.2 | Secondary end point(s) |
(1) Lung function parameters (DLCOcSB, FVC, DLCOc/VA, TLC SB, Predicted forced expiratory volume in one second FEV1 [L]) (2) pO2 at rest and under exercise (six minute walking test) (3) Laboratory parameters: sIL-2-receptor, Neopterin (4) CT scan lung: Global and specific score for nodules, lines, consolidation and ground glass (5) Cumulative steroid dose (6) Quality of life measured by SF36, CRDQ and SGRQ (7) Overall Survival (8) Immune phenotype of B- and T-cells
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
(1), (2, pO2 at rest), (3): 12 months retrospective documentation before BaselineVisit (Day 0), BaselineVisit (Day 0), 3 months (Visit 1), 6 months (Visit 2), 12 months (Final Visit) (2) pO2 under exercise: 12 months retrospective documentation before BaselineVisit (Day 0), BaselineVisit (Day 0), 12 months (Final Visit) (4) 12 months retrospective documentation before BaselineVisit (Day 0), BaselineVisit (Day 0), 12 months (Final Visit) (5) BaselineVisit (Day 0), 3 months (Visit 1), 6 months (Visit 2), 12 months (Final Visit) (6) SF36: BaselineVisit (Day 0), 12 months (Final Visit); CRDQ and SGRQ: BaselineVisit (Day 0), 6 months (Visit 2), 12 months (Final Visit) (7) All visits (8) BaselineVisit (Day 0), 12 months (Final Visit)
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 1 |
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.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.7 | Trial 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
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LVLS, however a follow-up visit 4 weeks after study end will be performed by telephone call by the study nurse. All (S)AEs will be documented. |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 2 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |