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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT03421743
Registration number
NCT03421743
Ethics application status
Date submitted
16/01/2018
Date registered
5/02/2018
Date last updated
24/03/2021
Titles & IDs
Public title
Pilot Trial of Inhaled Molgramostim in Nontuberculous Mycobacterial (NTM) Infection
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Scientific title
An Open-label, Non-controlled, Multicentre, Pilot Clinical Trial of Inhaled Molgramostim in Subjects With Antibiotic-resistant Non-tuberculosis Mycobacterial (NTM) Infection
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Secondary ID [1]
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SAV-008-01
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Universal Trial Number (UTN)
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Trial acronym
OPTIMA
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Mycobacterium Infections, Nontuberculous
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Condition category
Condition code
Infection
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Studies of infection and infectious agents
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Infection
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Other infectious diseases
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Infection
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Sexually transmitted infections
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Inhaled molgramostim
Experimental: Inhaled molgramostim/antimycobacterials - Inhaled molgramostim administered in subjects who remain sputum culture positive while currently on a multidrug Nontuberculous Mycobacterial (NTM) guideline based antimycobacterial regimen, which has been ongoing for at least 6 months prior to the Baseline Visit
Experimental: Inhaled molgramostim - Inhaled molgramostim administered in subjects who remain sputum culture positive but have stopped a multidrug Nontuberculous Mycobacterial (NTM) guideline based antimycobacterial regimen at least 28 days prior to Screening due to lack of response or intolerance, or who never started such treatment
Treatment: Drugs: Inhaled molgramostim
300 µg / dose molgramostim (recombinant human GM-CSF) for inhalation
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Number of subjects with sputum culture conversion to negative
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Assessment method [1]
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Sputum culture conversion is defined as at least three consecutive sputum samples without growth of Nontuberculous Mycobacteria (NTM) during the treatment period.
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Timepoint [1]
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48 weeks
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Secondary outcome [1]
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Number of subjects with sputum smear conversion to negative
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Assessment method [1]
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Sputum smear conversion is defined as at least three consecutive negative acid-fast bacilli (AFB) stained sputum smears on microscopy during the treatment period among subjects who were smear positive at Baseline.
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Timepoint [1]
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48 weeks
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Secondary outcome [2]
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Number of subjects with durable sputum culture conversion
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Assessment method [2]
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Durability is defined as sputum culture conversion at or before Week 24 and culture still negative for growth of NTM at 12-weeks follow-up.
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Timepoint [2]
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60 weeks
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Secondary outcome [3]
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Number of subjects with durable sputum smear conversion
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Assessment method [3]
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Durability is defined as sputum smear conversion at or before Week 48 and AFB stained smear still negative for NTM at 12-weeks follow-up among subjects who were smear positive at Baseline.
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Timepoint [3]
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60 weeks
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Secondary outcome [4]
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Change in semi-quantitative grade of number of NTM on microscopy of AFB stained sputum smears
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Assessment method [4]
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Timepoint [4]
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60 weeks
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Secondary outcome [5]
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Change in semi-quantitative grade of sputum cultures
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Assessment method [5]
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Timepoint [5]
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60 weeks
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Secondary outcome [6]
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Change in symptom scores (assessed using Lower Respiratory Tract Infections - Visual Analogue Scale (LRTI-VAS)
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Assessment method [6]
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Timepoint [6]
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60 weeks
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Secondary outcome [7]
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Change in Quality of Life scores (assessed using Quality of Life Questionnaire - Bronchiectasis (QOL-B))
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Assessment method [7]
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Timepoint [7]
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60 weeks
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Secondary outcome [8]
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Change in Global Rating of Health (GRH)
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Assessment method [8]
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Timepoint [8]
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60 weeks
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Secondary outcome [9]
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Change in body weight
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Assessment method [9]
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Timepoint [9]
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60 weeks
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Secondary outcome [10]
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Change in 6-minute walk distance (6MWD)
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Assessment method [10]
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Timepoint [10]
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60 weeks
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Secondary outcome [11]
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Change in oxygen desaturation during a 6-minute walk test (6MWT)
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Assessment method [11]
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Timepoint [11]
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60 weeks
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Secondary outcome [12]
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Change in Borg CR10 scores for dyspnea during a 6MWT
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Assessment method [12]
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Timepoint [12]
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60 weeks
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Secondary outcome [13]
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Number of adverse events (AEs) during the trial period
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Assessment method [13]
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Timepoint [13]
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60 weeks
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Secondary outcome [14]
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Number of serious AEs (SAEs) during the trial period
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Assessment method [14]
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Timepoint [14]
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60 weeks
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Secondary outcome [15]
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Number of adverse drug reactions (ADRs) during the trial period
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Assessment method [15]
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Timepoint [15]
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60 weeks
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Secondary outcome [16]
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Number of severe AEs during the trial period
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Assessment method [16]
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Timepoint [16]
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60 weeks
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Secondary outcome [17]
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Number of AEs leading to treatment discontinuation during the trial period
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Assessment method [17]
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Timepoint [17]
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60 weeks
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Secondary outcome [18]
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Change in white blood cell counts (WBC) in blood
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Assessment method [18]
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Timepoint [18]
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60 weeks
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Secondary outcome [19]
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Change in white cell differential counts in blood
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Assessment method [19]
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Timepoint [19]
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60 weeks
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Secondary outcome [20]
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Change in Diffusing Capacity of the Lung for Carbon Monoxide (DLCO)
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Assessment method [20]
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Timepoint [20]
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48 weeks
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Secondary outcome [21]
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Change in forced expiratory volume in 1 second (FEV1) (% predicted)
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Assessment method [21]
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Timepoint [21]
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48 weeks
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Secondary outcome [22]
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Change in forced vital capacity (FVC) (% predicted)
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Assessment method [22]
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Timepoint [22]
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48 weeks
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Secondary outcome [23]
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Number of subjects with development of anti-molgramostim antibodies in serum
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Assessment method [23]
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Timepoint [23]
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60 weeks
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Eligibility
Key inclusion criteria
1. 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).
2. 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.
3. 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.
4. Female or male =18 years of age.
5. 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.
6. 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.
7. Willing and able to provide signed informed consent.
8. 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
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Minimum age
18
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
1. Subjects diagnosed with cystic fibrosis.
2. Prior therapy with inhaled or systemic GM-CSF.
3. Subjects with hemoptysis of =60 mL in a 24 hour period within 4 weeks prior to
Screening.
4. Concurrent disease with a life expectancy of less than 6 months.
5. History of, or present, myeloproliferative disease, leukemia or other hematological
malignancy.
6. 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.
7. 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.
8. Pulmonary tuberculosis requiring treatment or treated within 2 years prior to
Screening.
9. HIV infection or other disease associated with significant immunodeficiency.
10. History of lung transplantation.
11. Any change in chronic NTM multi-drug antimycobacterial regimen within 28 days prior to
Screening.
12. Treatment with any investigational medicinal product within 3 months of Screening.
13. Previous experience of severe and unexplained side-effects during aerosol delivery of
any kind of medicinal product
14. Any other serious medical condition which in the opinion of the investigator would
make the subject unsuitable for the trial.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Non-randomised trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
1/03/2018
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
13/01/2020
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Sample size
Target
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Accrual to date
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Final
32
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Recruitment in Australia
Recruitment state(s)
NSW,QLD,WA
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Recruitment hospital [1]
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Concord Repatriation General Hospital - Concord
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Recruitment hospital [2]
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The Prince Charles Hospital - Chermside West
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Recruitment hospital [3]
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Greenslopes Private Hospital - Greenslopes
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Recruitment hospital [4]
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Royal Perth Hospital - Perth
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Recruitment postcode(s) [1]
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2139 - Concord
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Recruitment postcode(s) [2]
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4032 - Chermside West
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Recruitment postcode(s) [3]
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4120 - Greenslopes
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Recruitment postcode(s) [4]
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6000 - Perth
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Recruitment outside Australia
Country [1]
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United Kingdom
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State/province [1]
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London
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Savara Inc.
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Address
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Country
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Ethics approval
Ethics application status
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Summary
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 persistent pulmonary Nontuberculous Mycobacterial (NTM) infection. Subject
will be treated for 24-weeks with inhaled molgramostim and will be followed up for 12-weeks
after end of treatment. The primary aim of the trial is to investigate the efficacy of
inhaled molgramostim on NTM sputum culture conversion to negative.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT03421743
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Grant Waterer, Prof.
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Address
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Royal Perth Hospital
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Address
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Country
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Phone
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Fax
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Email
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT03421743
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