Trial of Inhaled Molgramostim in Cystic Fibrosis Subjects With Nontuberculous Mycobacterial Infec… (NCT03597347) | Clinical Trial Compass
TerminatedPhase 2
Trial of Inhaled Molgramostim in Cystic Fibrosis Subjects With Nontuberculous Mycobacterial Infection
Stopped: Recruitment for trial stopped early due to limitations from COVID-19, additionally changes in the standard of care for CF patients during the study period resulted in confounding of the primary endpoint.
United States14 participantsStarted 2019-06-20
Plain-language summary
A study to evaluate the efficacy of inhaled molgramostim administered open-label to adult cystic fibrosis (CF) subjects with chronic pulmonary nontuberculous mycobacterial (NTM) infection, with or without ongoing antimycobacterial guideline based combination therapy.
Who can participate
Age range18 Years
SexALL
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Inclusion criteria
✓. Written informed consent obtained from participant.
✓. Confirmed diagnosis of CF according to the Cystic Fibrosis Foundation (CFF) 2017 Consensus Guidelines.
✓. History of chronic pulmonary infection with M. avium complex (MAC) or M. abscessus complex (MABSC) (defined as at least three positive NTM cultures (sputum or BAL for the same species (MAC) or subspecies (MABSC) within the 2 years prior to the screening visit, with at least one positive within the past 6 months and a minimum of 50% of NTM cultures positive over the past 2 years) that does not demonstrate response to current treatment course based on decreasing NTM burden or frequency of positive cultures, and in the opinion of the Investigator is unlikely to resolve with current treatment course.
✓. Subject fulfills criteria for inclusion in one of the following groups:
✓. Ability to produce sputum or be willing to undergo an induction protocol that produces sputum for clinical evaluation.
✓. An additional sputum culture performed by the central laboratory, which is positive for the same species (MAC) or subspecies (MABSC) of NTM as before the trial within 10 weeks of Baseline.
✓. CF which in the Investigator's opinion is clinically stable and not expected to require lung transplantation within the next year.
What they're measuring
1
Number of Participants With Sputum NTM Culture Conversion to Negative
. FEV1 ≥ 30% of predicted at screening that is normalized for age, gender, race, and height, using the Global Lung Function Initiative (GLI) equation.
Exclusion criteria
✕. Use of non-maintenance antibiotic for a concurrent pulmonary or extrapulmonary infection within 28 days prior to the Baseline visit.
✕. Use of a maintenance antibiotic regimen containing azithromycin for a concurrent non-NTM pulmonary infection within 28 days prior to the Baseline visit. For subjects in Group 1, azithromycin is allowed if part of ongoing multidrug NTM guideline-based antimycobacterial regimen.
✕. Prior therapy with inhaled or systemic granulocyte macrophage colony stimulating factor (GM-CSF).
✕. Subjects with hemoptysis of ≥60 mL in a 24-hour period within 4 weeks prior to Screening.
✕. Life expectancy of less than 6 months according to Investigator's judgement.
✕. History of, or present, myeloproliferative disease, leukemia or other hematological malignancy.
✕. Active pulmonary malignancy (primary or metastatic); or any malignancy requiring chemotherapy or radiation therapy within 1 year prior to Screening or anticipated during the study period.
✕. Active autoimmune disorder or other condition requiring therapy associated with significant immunosuppression, e.g. such as systemic corticosteroids at a dose equivalent of 10 mg/day or more of prednisolone or other significant immunosuppressant medications, within 3 months prior to Screening or anticipated during the study period. Inhaled or topical corticosteroids, or brief courses (\<14 days) of systemic corticosteroids for pulmonary exacerbations or other self-limited conditions are permitted.