A Study to Evaluate Safety and Efficacy of Subcutaneous Administration of Anakinra in Patients With CF

NCT03925194 | Recruiting Phase 2 DMC

• 1 other identifier: EudraCT No.: 2016-004786-80-A
• Study Type: interventional
• Target: 52
• Locations: 1 country
• Sites: 3

Brief Summary

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.

Conditions

Cystic Fibrosis, 10011762

Outcome Measures

Primary Outcomes (1)

• Absolute pre-post change of the lung clearance index (LCI)

  28 days

Study Arms (2)

Anakinra

EXPERIMENTAL

Drug: Anakinra

Placebo

PLACEBO COMPARATOR

Drug: Anakinra

Interventions

Anakinra DRUG

Application of Anakinra once daily for 28 days

Anakinra; Placebo

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify 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 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 phase the wash-out may be extended for up to 12 weeks in order to fulfill this criterion),
• 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,

You may not qualify 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) 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,
• 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 screening and before start of each treatment period,
• 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 and/or deterioration of infection-specific laboratory parameters beyond changes driven by the underlying disease),
• 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 interventional 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),

Sponsors & Collaborators

• Heidelberg University: lead

Study Sites (3)

University Children's Hospital Heidelberg, Cystic Fibrosis Centre

Heidelberg, Baden-Wurttemberg, 69120, Germany

RECRUITING

Universitätsmedizin Essen, Ruhrlandklinik

Essen, North Rhine-Westphalia, 45239, Germany

RECRUITING

Charité - Universitätsmedizin Berlin

Berlin, State of Berlin, 13353, Germany

RECRUITING

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

Interleukin 1 Receptor Antagonist Protein

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Intervention Hierarchy (Ancestors)

Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Deputy of Principal Investigator (Olaf Sommerburg)

Study Record Dates

• First Submitted: April 19, 2019
• First Posted: April 24, 2019
• Study Start: December 1, 2022
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): December 1, 2026
• Last Updated: March 27, 2025

Record last verified: 2025-03

Data Sharing

• IPD Sharing: Will not share

Locations

DE (3)