Status:
COMPLETED
Pilot Trial of Inhaled Molgramostim in Non-tuberculous Mycobacterial (NTM) Infection
Lead Sponsor:
Savara Inc.
Conditions:
Mycobacterium Infections, Nontuberculous
Eligibility:
All Genders
18+ years
Phase:
PHASE2
Brief Summary
The trial is an open-label, non-controlled, multicenter, pilot clinical trial of inhaled molgramostim (recombinant human granulocyte-macrophage colony stimulating factor; rhGM-CSF) in subjects with pe...
Detailed Description
The study will comprise a Screening Visit, a Baseline Visit, a 24-week treatment period and a 12-week follow-up period. 30 adult participants with a history of chronic NTM infection with at least 2 po...
Eligibility Criteria
Inclusion
- History of chronic pulmonary infection with MAC or M. abscessus (defined as at least 2 documented positive sputum cultures in the prior 2 years, of which at least one was obtained in the 6 months prior to Screening).
- Subject fulfills one of the following criteria:
- Subjects who remain sputum culture positive while currently on a multidrug NTM guideline based antimycobacterial regimen, which has been ongoing for at least 6 months prior to the Baseline Visit
- Subjects who remain sputum culture positive but have either stopped a multidrug NTM guideline based antimycobacterial regimen at least 28 days prior to Screening due to lack of response or intolerance, or never started such treatment.
- Ability to produce at least 2 mL of sputum or be willing to undergo an induction that produces at least 2 mL of sputum for clinical evaluation.
- Female or male ≥18 years of age.
- Females who have been post-menopausal for more than 1 year or females of childbearing potential after a confirmed menstrual period using a highly efficient method of contraception (i.e. a method with less than 1% failure rate such as combined hormonal contraception, progesterone-only hormonal contraception, intrauterine device, intrauterine hormone- releasing system, bilateral tubal occlusion, vasectomized partner, sexual abstinence), during and until thirty (30) days after last dose of trial treatment. Females of childbearing potential must have a negative serum pregnancy test at Screening (Visit 1) and a negative urine pregnancy test at dosing at Baseline (Visit 2) and must not be lactating.
- Males agreeing to use condoms during and until thirty (30) days after last dose of medication, or males having a female partner who is using adequate contraception as described above.
- Willing and able to provide signed informed consent.
- Willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other trial procedures specified in the protocol as judged by the investigator
Exclusion
- Subjects diagnosed with cystic fibrosis.
- Prior therapy with inhaled or systemic GM-CSF.
- Subjects with hemoptysis of ≥60 mL in a 24 hour period within 4 weeks prior to Screening.
- Concurrent disease with a life expectancy of less than 6 months.
- 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 one year prior to Screening or anticipated during the study period.
- Active allergic bronchopulmonary mycosis or connective tissue disease, inflammatory bowel disease or other autoimmune disorder requiring therapy associated with significant immunosuppression, such as systemic corticosteroids at a dose equivalent of 10 mg/day or more of prednisolone, within 3 months prior to Screening or anticipated during the study period.
- Pulmonary tuberculosis requiring treatment or treated within 2 years prior to Screening.
- HIV infection or other disease associated with significant immunodeficiency.
- History of lung transplantation.
- Any change in chronic NTM multi-drug antimycobacterial regimen within 28 days prior to Screening.
- Treatment with any investigational medicinal product within 3 months of Screening.
- Previous experience of severe and unexplained side-effects during aerosol delivery of any kind of medicinal product
- Any other serious medical condition which in the opinion of the investigator would make the subject unsuitable for the trial.
Key Trial Info
Start Date :
March 1 2018
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
January 13 2020
Estimated Enrollment :
32 Patients enrolled
Trial Details
Trial ID
NCT03421743
Start Date
March 1 2018
End Date
January 13 2020
Last Update
July 3 2024
Active Locations (5)
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1
Concord Repatriation General Hospital
Concord, New South Wales, Australia, 2139
2
The Prince Charles Hospital
Chermside West, Queensland, Australia, 4032
3
Greenslopes Private Hospital
Greenslopes, Queensland, Australia, 4120
4
Royal Perth Hospital
Perth, Western Australia, Australia, 6000