Please note that the copy function is not enabled for this field.
If you wish to
modify
existing outcomes, please copy and paste the current outcome text into the Update field.
LOGIN
CREATE ACCOUNT
MY TRIALS
LOGIN
CREATE ACCOUNT
MY TRIALS
REGISTER TRIAL
FAQs
HINTS AND TIPS
DEFINITIONS
Register a trial
The ANZCTR website will be unavailable from 1pm until 3pm (AEDT) on Wednesday the 30th of October for website maintenance. Please be sure to log out of the system in order to avoid any loss of data.
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this
information for consumers
Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT03421743
Registration number
NCT03421743
Ethics application status
Date submitted
16/01/2018
Date registered
5/02/2018
Titles & IDs
Public title
Pilot Trial of Inhaled Molgramostim in Non-tuberculous Mycobacterial (NTM) Infection
Query!
Scientific title
An Open-label, Non-controlled, Multicentre, Pilot Clinical Trial of Inhaled Molgramostim in Subjects With Antibiotic-resistant Non-tuberculosis Mycobacterial (NTM) Infection
Query!
Secondary ID [1]
0
0
2017-003374-14
Query!
Secondary ID [2]
0
0
SAV008-01
Query!
Universal Trial Number (UTN)
Query!
Trial acronym
OPTIMA
Query!
Linked study record
Query!
Health condition
Health condition(s) or problem(s) studied:
Mycobacterium Infections, Nontuberculous
0
0
Query!
Condition category
Condition code
Infection
0
0
0
0
Query!
Studies of infection and infectious agents
Query!
Infection
0
0
0
0
Query!
Other infectious diseases
Query!
Infection
0
0
0
0
Query!
Sexually transmitted infections
Query!
Intervention/exposure
Study type
Interventional
Query!
Description of intervention(s) / exposure
Treatment: Drugs - Inhaled molgramostim
Treatment: Drugs - Antimycobacterial regimen
Experimental: Inhaled molgramostim/antimycobacterials - Inhaled molgramostim administered in participants 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
Experimental: Inhaled molgramostim - Inhaled molgramostim administered in participants who remain sputum culture positive but have stopped a multidrug 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
Treatment: Drugs: Antimycobacterial regimen
Multidrug NTM guideline-based antimycobacterial regimen
Query!
Intervention code [1]
0
0
Treatment: Drugs
Query!
Comparator / control treatment
Query!
Control group
Query!
Outcomes
Primary outcome [1]
0
0
Number of Participants With Sputum Culture Conversion to Negative
Query!
Assessment method [1]
0
0
Sputum culture conversion is defined as at least 3 consecutive sputum samples without growth of NTM during the treatment period.
Query!
Timepoint [1]
0
0
48 weeks
Query!
Secondary outcome [1]
0
0
Number of Participants With Sputum Smear Conversion to Negative
Query!
Assessment method [1]
0
0
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 participants who were smear positive at Baseline.
Query!
Timepoint [1]
0
0
48 weeks
Query!
Secondary outcome [2]
0
0
Number of Participants With Durable Sputum Culture Conversion
Query!
Assessment method [2]
0
0
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.
Query!
Timepoint [2]
0
0
60 weeks
Query!
Secondary outcome [3]
0
0
Number of Participants With Durable Sputum Smear Conversion
Query!
Assessment method [3]
0
0
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 participants who were smear positive at Baseline.
Query!
Timepoint [3]
0
0
60 weeks
Query!
Secondary outcome [4]
0
0
Change From Baseline in Symptom Scores (Assessed Using Lower Respiratory Tract Infections - Visual Analogue Scale)
Query!
Assessment method [4]
0
0
For each of the clinical symptoms of Lower Respiratory Tract Infections (dyspnea, fatigue, cough, pain, and sputum), the participant assessed the severity using a 10 cm visual analogue scale (VAS) ranging from 0 = no symptoms to 10 = worst possible symptoms. A total LRTI score was calculated by summing up the score of each symptom (i.e., total LRTI score ranged from 0 to 50).
Query!
Timepoint [4]
0
0
Baseline to Week 48
Query!
Secondary outcome [5]
0
0
Change From Baseline in Symptom Scores (Assessed Using Quality of Life Questionnaire - Bronchiectasis (QOL-B))
Query!
Assessment method [5]
0
0
The QOL-B questionnaire including 37 items on 8 scales (emotional functioning, health perceptions, physical functioning, respiratory symptoms, role functioning, social functioning, treatment burden, vitality) was used to assess participant's quality of life (QoL). Each of the 37 items is scored from 1 to 4, and each of the 8 scale scores is standardised on a 0-100 point scale, with higher scores representing fewer symptoms or better functioning and QoL. Scales contain between 3 and 9 items, thus changing 1 answer category will correspond to a change of 11.1 to 3.7 points.
Query!
Timepoint [5]
0
0
Baseline to Week 48
Query!
Secondary outcome [6]
0
0
Change From Baseline in Global Rating of Health (GRH)
Query!
Assessment method [6]
0
0
GRH was assessed as an interviewer questionnaire, which assesses global health as "excellent, good, fair or poor". For analysis, these were scored as 1 (poor) to 4 (excellent).
Query!
Timepoint [6]
0
0
Baseline to Week 48
Query!
Secondary outcome [7]
0
0
Change From Baseline in Body Weight
Query!
Assessment method [7]
0
0
Query!
Timepoint [7]
0
0
Baseline to Week 48
Query!
Secondary outcome [8]
0
0
Change From Baseline in 6-Minute Walking Distance (6MWD)
Query!
Assessment method [8]
0
0
The 6-minute walk test (6MWT) was performed in accordance with the European Respiratory Society/American Thoracic Society (ERS/ATS) guideline by technicians with documented training and experience. The 6MWT was performed twice at each visit.
Query!
Timepoint [8]
0
0
Baseline to Week 48
Query!
Secondary outcome [9]
0
0
Change From Baseline in Dyspnea Scores During a 6MWT
Query!
Assessment method [9]
0
0
Query!
Timepoint [9]
0
0
Baseline to Week 48
Query!
Secondary outcome [10]
0
0
Number of Adverse Events (AEs) During the Trial Period
Query!
Assessment method [10]
0
0
All trial subjects were carefully monitored for the occurrence of AEs during the trial period from Baseline (Visit 2) to the 12-week Follow-up visit (Visit 15). AEs were collected by the investigator by a non-leading question and by reporting events directly observed or spontaneously volunteered by participants. Participants were also encouraged to contact the clinic in between visits if they experienced AEs or had any concerns.
Query!
Timepoint [10]
0
0
60 weeks
Query!
Secondary outcome [11]
0
0
Number of Serious AEs (SAEs) During the Trial Period
Query!
Assessment method [11]
0
0
SAEs were defined as any untoward medicinal occurrence or effect that at any dose:
* Results in death
* Is life-threatening
* Requires hospitalisation or prolongation of existing hospitalisation
* Results in persistent or significant disability or incapacity
* Is a congenital abnormality or birth defect
* May jeopardise the participant or may require medical intervention to prevent one or more of the outcomes listed above (Important Medical Events).
Query!
Timepoint [11]
0
0
60 weeks
Query!
Secondary outcome [12]
0
0
Number of Adverse Drug Reactions (ADRs) During the Trial Period
Query!
Assessment method [12]
0
0
All AEs were assessed by the investigator for causality (unlikely, possible, probable, not applicable) according to current regulatory standards. AEs which had a 'possible' or 'probable' causality were classified as ADRs.
Query!
Timepoint [12]
0
0
60 weeks
Query!
Secondary outcome [13]
0
0
Number of Severe AEs During the Trial Period
Query!
Assessment method [13]
0
0
All AEs were assessed by the investigator for severity (mild, moderate, severe) according to current regulatory standards.
Query!
Timepoint [13]
0
0
60 weeks
Query!
Secondary outcome [14]
0
0
Number of Participants Withdrawn From Treatment Due to an AE During the Trial Period
Query!
Assessment method [14]
0
0
Query!
Timepoint [14]
0
0
60 weeks
Query!
Secondary outcome [15]
0
0
Change From Baseline in Diffusing Capacity of the Lung for Carbon Monoxide (DLCO)
Query!
Assessment method [15]
0
0
Query!
Timepoint [15]
0
0
Baseline to Week 48
Query!
Secondary outcome [16]
0
0
Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1) (% Predicted)
Query!
Assessment method [16]
0
0
Query!
Timepoint [16]
0
0
Baseline to Week 48
Query!
Secondary outcome [17]
0
0
Change From Baseline in Forced Vital Capacity (FVC) (% Predicted)
Query!
Assessment method [17]
0
0
Query!
Timepoint [17]
0
0
Baseline to Week 48
Query!
Secondary outcome [18]
0
0
Number of Subjects With Development of Anti-GM-CSF Antibodies in Serum
Query!
Assessment method [18]
0
0
Analyses for anti-GM-CSF antibodies were performed at a central laboratory.
Query!
Timepoint [18]
0
0
60 weeks
Query!
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
Query!
Minimum age
18
Years
Query!
Query!
Maximum age
No limit
Query!
Query!
Sex
Both males and females
Query!
Can healthy volunteers participate?
No
Query!
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.
Query!
Study design
Purpose of the study
Treatment
Query!
Allocation to intervention
Non-randomised trial
Query!
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Query!
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Query!
Masking / blinding
Open (masking not used)
Query!
Who is / are masked / blinded?
Query!
Query!
Query!
Query!
Intervention assignment
Parallel
Query!
Other design features
Query!
Phase
Phase 2
Query!
Type of endpoint/s
Query!
Statistical methods / analysis
Query!
Recruitment
Recruitment status
Completed
Query!
Data analysis
Query!
Reason for early stopping/withdrawal
Query!
Other reasons
Query!
Date of first participant enrolment
Anticipated
Query!
Actual
1/03/2018
Query!
Date of last participant enrolment
Anticipated
Query!
Actual
Query!
Date of last data collection
Anticipated
Query!
Actual
13/01/2020
Query!
Sample size
Target
Query!
Accrual to date
Query!
Final
32
Query!
Recruitment in Australia
Recruitment state(s)
NSW,QLD,WA
Query!
Recruitment hospital [1]
0
0
Concord Repatriation General Hospital - Concord
Query!
Recruitment hospital [2]
0
0
The Prince Charles Hospital - Chermside West
Query!
Recruitment hospital [3]
0
0
Greenslopes Private Hospital - Greenslopes
Query!
Recruitment hospital [4]
0
0
Royal Perth Hospital - Perth
Query!
Recruitment postcode(s) [1]
0
0
2139 - Concord
Query!
Recruitment postcode(s) [2]
0
0
4032 - Chermside West
Query!
Recruitment postcode(s) [3]
0
0
4120 - Greenslopes
Query!
Recruitment postcode(s) [4]
0
0
6000 - Perth
Query!
Recruitment outside Australia
Country [1]
0
0
United Kingdom
Query!
State/province [1]
0
0
London
Query!
Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Query!
Name
Savara Inc.
Query!
Address
Query!
Country
Query!
Ethics approval
Ethics application status
Query!
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 non-tuberculous mycobacterial (NTM) infection. Participants will be treated for 24-weeks with inhaled molgramostim and 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.
Query!
Trial website
https://clinicaltrials.gov/study/NCT03421743
Query!
Trial related presentations / publications
Query!
Public notes
Query!
Contacts
Principal investigator
Name
0
0
Grant Waterer, Prof.
Query!
Address
0
0
Royal Perth Hospital
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for public queries
Name
0
0
Query!
Address
0
0
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for scientific queries
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
Query!
No/undecided IPD sharing reason/comment
Query!
What supporting documents are/will be available?
No Supporting Document Provided
Type
Other Details
Attachment
Study protocol
https://cdn.clinicaltrials.gov/large-docs/43/NCT03421743/Prot_000.pdf
Statistical analysis plan
https://cdn.clinicaltrials.gov/large-docs/43/NCT03421743/SAP_001.pdf
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results are available at
https://clinicaltrials.gov/study/NCT03421743