infection with nontuberculous mycobacteria Nontuberculous mycobacterial (NTM) disease
Conditions
Interventions
All participants will receive CFZ as a loading dose regimen of 300 mg once
daily for 4 weeks and will then continue with a standard dose of 100 mg once
daily until a total 4 months of treatment with
Sponsors
Afdeling Apotheek
Eligibility
Age
18 Years to 99 Years
Inclusion criteria
Inclusion criteria: - The participant is diagnosed with pulmonary or extrapulmonary NTM disease and is eligible for treatment with CFZ - The participant is at least 18 years of age - The participant has a body weight (in light clothing and with no shoes) of at least 45 kg - The participant is able and willing to provide written, informed consent
Exclusion criteria
Exclusion criteria: - The participant is in poor general condition where participation in the study cannot be accepted per discretion of the Investigator - There is evidence showing the participant has clinically significant metabolic, gastrointestinal, or other abnormalities that could possibly alter the PK of CFZ - The participant is diagnosed with cystic fibrosis - The participant has a prolongation of the QTc interval, > 450 milliseconds for males and > 460 milliseconds for females, on the screening ECG - The participant has abnormal alanine aminotransferase (ALT) and/or aspartate transferase (AST) levels > 3 times the upper limit of the laboratory reference range at screening - The participant is pregnant or is using inadequate contraceptive measures (if applicable) - The participant is breastfeeding (if applicable) - The participant has a known or suspected, current drug or alcohol abuse, that is, in the opinion of the Investigator, sufficient to compromise the safety or cooperation of the patient - The participant has as history of allergy/hypersensitivity to CFZ - The participant has received clofazimine in the past 3 months before inclusion with the exception of short-term use of no more than 7 days in the period of 1 to 3 months before inclusion
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The primary study parameters of this study are the PK parameters of CFZ at day 28 of treatment, including AUC0-24, Cmax, and C0, after a loading dose regimen of 300 mg once daily for 4 weeks in adult patients with pulmonary or extrapulmonary NTM disease. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary study parameters are: • Difference between the highest measured concentrations of CFZ in this study and a reference study (i.e. PERC study; EudraCT number 2015-003786-28) after approximately 1 and 4 months of treatment. • The predicted time to reach steady state concentrations with and without a loading dose. • The predicted PK parameters, including AUC0-24, Cmax and C0, after approximately 1 and 4 months of treatment in both this study and a reference cohort (i.e. PERC study). • The performance of the existing population PK model for CFZ derived in TB patients for the NTM population. • Safety and tolerability of CFZ in this study, assessed by the occurrence and grading of adverse events | — |
Countries
Netherlands
Outcome results
None listed