Safety and efficacy of Abatacept in patients with treatment-resistant sarcoidosis

Phase 1

• Conditions: Steroid-refractory Sarcoidosis; MedDRA version: 20.0Level: PTClassification code 10037430Term: Pulmonary sarcoidosisSystem Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders; Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]

• Registration Number: EUCTR2016-003360-39-DE
• Lead Sponsor: niversitätsklinikum Freiburg

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2017-04-11
• Last Update Posted: 26 April 2021

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 30

Inclusion Criteria

1. Signed written informed consent
2. Ability to understand the nature, significance and consequences of the study and to comply with them
3. Age 18 years or above; male or female patients
4. Diagnosis of sarcoidosis according to the current applicable ATS/WASOG guidelines
5. Immunosuppressive therapy (= 5 mg prednisolone equivalent per day or any additional immunosuppressive therapy independently of the steroid dose) within the last 3 months prior screening for pulmonary involvement as defined by one of the following
a. Radiographic alterations in HRCT compatible with sarcoidosis
b. Lung function impairment caused by sarcoidosis (e.g. reduced TLC, FVC, reduced DLCO, reduced pO2 at rest or exercise-induced)
6. King’s Sarcoidosis Questionnaire (KSQ), GHS L module: Score < 80
7. Need for therapy escalation beyond 5 mg prednisolone equivalent according to the physician’s appraisal, e.g.:
a. Disease activity only controlled by > 5 mg prednisolone equivalent
b. Disease progression under established immunosuppressive therapy according to clinician’s appraisal (e.g. lung function deterioration, CT-graphic progression, increase of activity parameters)
c. Need for change of immunosuppressive therapy because of therapy-associated side effects
8. Elevated sIL2 receptor levels and/or elevated Neopterin as sign of T-cell activation within 6 months prior to date of informed consent
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 25
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 5

Exclusion Criteria

1. Severe lung functional impairment according to the treating physician interfering substantially with participation in the trial
2. End stage fibrotic lung disease without expected improvement to immunosuppressive therapy as judged by the treating physician.
3. Concomitant lung disease (e.g. COPD, asthma) that interferes with clinical assessment as judged by the treating physician
4. Concurrent immunosuppressive therapy other than corticosteroids and impossibility to allow a sufficient wash-out phase.
5. For biologicals a wash-out phase of two months is mandatory.
6. Previous treatment with Abatacept
7. Treatment with another investigational drug within 4 weeks or 5 half-lives prior to first application of trial medication.
8. Recurrent or active current bacterial, viral or fungal infection (excluding fungal infections of the nails), for example but not limited to active hepatitis B and C, typical or atypical mycobacteriosis or herpes zoster infections.
9. History of or active psychiatric disease (including known history of or current active abuse of drugs, chemicals or alcohol) interfering with the safe participation in the study.
10. History of or current primary or secondary immunodeficiency that could not be attributable to treatment-related immunodeficiency
11. Serious uncontrolled concomitant diseases not caused by sarcoidosis. Examples are cardiovascular (e.g. severe uncontrolled hypertension, instable angina pectoris, severe ischemic heart disease), central nervous system (e.g. stroke, dementia), hepatic, renal, endocrine (e.g. uncontrolled diabetes), gastrointestinal (e.g. complicated diverticulitis, Crohn’s disease, colitis ulcerosa)
12. Known malignancy or high clinical suspicion on malignant disease
13. Lymphoma within the last five years
14. Contraindications against treatment with Abatacept
15. Simultaneous application of live vaccines
16. Simultaneous participation in other interventional clinical trials
17. Pregnancy indicated by positive urine pregnancy test
18. Breast-feeding patients
19. Fertile patients refusing to use safe contraceptive methods during the study

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified