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Tests treatment safety and results for Cystic Fibrosis, 10011762
Official title A Study to Evaluate Safety and Efficacy of Subcutaneous Administration of Anakinra in Patients With CF
ClinicalTrials.gov ID: NCT03925194
What this study is testing
What is Anakinra?
Anakinra is an investigational medicine, given as an once-daily, being studied as a potential treatment for cystic fibrosis, 10011762.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- OBJECTIVES Primary: To evaluate efficacy of treatment with anakinra in subjects with CF who are ≥ 12 years of age by means of lung clearance index (LCI). Secondary To evaluate safety and tolerability of treatment with anakinra as well as to investigate further effects of anakinra on lung function and quality of life (QOL) in subjects with CF.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 12 and older
You may be able to join if
- Age ≥ 18 years (1st cohort). If justified by interim analysis, 18 \> age ≥ 12 years (2nd cohort),
- Informed consent of the patient (if applicable) and/or all legal guardians,
- Sufficient fluency of patient and/or his/her representative in German language to comply with study-specific procedures (e.g. to complete required...
- Confirmed diagnosis of cystic fibrosis, fulfilling at least one of the following three criteria:
- sweat chloride ≥ 60mEq/L,
You likely can't join if
- Expected non-compliance, i.e. inability or unwillingness to comply with study-specific procedures,
- Known allergy to anakinra or any ingredient of the pharmaceutical formulation of Kineret®,
- Planned immunization with attenuated (live) vaccine(s) during the treatment with the IMP or completed immunization with attenuated (live) vaccine(s)...
- Renal failure (creatinine in serum above ULN),
- History of tuberculosis or repeated detection of non-tuberculous mycobacteria from airway samples in the last 12 months before start of each...
- History of detection of Burkholderia cenocepacia species in the last 12 months before start of each treatment period,
See the full eligibility criteria
- Age ≥ 18 years (1st cohort). If justified by interim analysis, 18 \> age ≥ 12 years (2nd cohort),
- Informed consent of the patient (if applicable) and/or all legal guardians,
- Sufficient fluency of patient and/or his/her representative in German language to comply with study-specific procedures (e.g. to complete required quality of life questionnaires),
- Confirmed diagnosis of cystic fibrosis, fulfilling at least one of the following three criteria:
- sweat chloride ≥ 60mEq/L,
- two CF causing mutations in the CFTR gene,
- alterations of transepithelial potential difference of nasal or rectal epithelia typical for CF,
- FEV1 ≥ 50 % pred. at screening,
- LCI2.5 ≥ 7.05 at screening,
- Ability to perform reproducible multiple breath washout and spirometry,
- Oxyhaemoglobin saturation of ≥ 90% on room air at screening,
- No changes in the medication for cystic fibrosis lung disease for at least 4 weeks prior to the first administration of the IMP of each treatment period (in case of medication changes in Period 1 and/or the washout...
- Adequate bone marrow function assessed on the basis of: neutrophils \>1.5 x 109/L, platelets \>100 x 109/L, hemoglobin \>9.0 g/dL,
- Adequate liver function assessed on the basis of: GGT, ASAT, and ALAT \<3 x upper limit of normal (ULN),
- Adequate blood clotting assessed on the basis of: aPTT \<39 sec., INR \<1.2,
- Negative serology for HIV (anti-HIV 1/2 IgG/IgM and p24-Ag), HBV (anti-HBs quantitative and anti-HBc IgG/IgM) and HCV (anti-HCV IgG), negative Interferon-gamma release assay,
- Negative Beta-HCG blood/urine test in women of childbearing potential (of childbearing potential are females who have experienced menarche and are not permanently sterile or postmenopausal (postmenopausal: 12...
- Use of adequate contraception in sexually active female people (sexual abstinence, hormonal contraceptives or intrauterine device).
- Expected non-compliance, i.e. inability or unwillingness to comply with study-specific procedures,
- Known allergy to anakinra or any ingredient of the pharmaceutical formulation of Kineret®,
- Planned immunization with attenuated (live) vaccine(s) during the treatment with the IMP or completed immunization with attenuated (live) vaccine(s) within 4 weeks prior to the first administration of the IMP,
- Renal failure (creatinine in serum above ULN),
- History of tuberculosis or repeated detection of non-tuberculous mycobacteria from airway samples in the last 12 months before start of each treatment period,
- History of detection of Burkholderia cenocepacia species in the last 12 months before start of each treatment period,
- Colonization with multi-resistant Staphylococcus aureus (MRSA) and/or 4-multi-resistant gram negative (MRGN) Pseudomonas aeruginosa is only an exclusion criterion if the treating physician judges that this is an...
- Acute bronchopulmonary exacerbation (defined by modified Fuchs criteria (1) (see Appendix 1), modification includes all ways of application of an antibiotic (e.g., oral, i.v., inhaled)) within 14 days prior to the...
- Signs of other active infection within 14 days prior to the screening and before start of each treatment period (clinical symptoms (e.g. burning sensation while urinating, skin, wound or dental infection) and/or fever...
- Immunosuppressive treatment due to organ transplantation, rheumatic or autoimmune diseases as well as treatment with Anakinra in the last 3 months before Day 1 of Period 1,
- Participation in another treatment trial within the last 30 days prior to screening,
- Current oral corticosteroid use,
- Current oxygen supplementation,
- Current treatment with etanercept,
- Medical history of lung transplantation,
- Pregnant or nursing females (females of childbearing potential must have a negative pregnancy test at Screening),
- Known hypersensitivity to hypertonic saline (used for induction of sputum).
The study team makes the final eligibility decision.
Where it's taking place
- Heidelberg, Baden-Wurttemberg, Germany
- Essen, North Rhine-Westphalia, Germany
- Berlin, State of Berlin, Germany
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 12 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Heidelberg, Baden-Wurttemberg, Germany; Essen, North Rhine-Westphalia, Germany; Berlin, State of Berlin, Germany. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.