Mycobacterium Infections, NontuberculousPhase 2OPTIMANCT03421743
Pilot Trial of Inhaled Molgramostim in Non-tuberculous Mycobacterial (NTM) Infection
A Phase 2 study testing Inhaled molgramostim and Antimycobacterial regimen in people with Mycobacterium Infections, Nontuberculous, run by Savara Inc. at 5 sites in 2 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Mycobacterium Infections, Nontuberculous
- What is being tested?
- Inhaled molgramostim and Antimycobacterial regimen
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 32 participants
- Where
- 5 sitesin 2 countries
- Study dates
- 1 Mar 2018to 13 Jan 2020
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Show all 10Show fewer
- 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
Who cannot join
- 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.
Show all 14Show fewer
- 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.
Trial locations and contacts
5 sites in 2 countries.
5 sites in 2 countries
Australia4 sites
The Prince Charles Hospital
Chermside West, Queensland, 4032
Concord Repatriation General Hospital
Concord, New South Wales, 2139
Greenslopes Private Hospital
Greenslopes, Queensland, 4120
Royal Perth Hospital
Perth, Western Australia, 6000
About this study
Official title: An Open-label, Non-controlled, Multicentre, Pilot Clinical Trial of Inhaled Molgramostim in Subjects With Antibiotic-resistant Non-tuberculosis Mycobacterial (NTM) Infection
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.
Read the 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 positive cultures in the prior 2 years, of which at least one is within the last 6 months prior to Screening, will be considered for enrollment. Participants should provide a positive NTM sputum culture at Screening to be eligible.
Two subgroups of participants will be recruited:
- Group 1: 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.
- Group 2: Participants 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.
The treatment period will consist of 14 trial visits (Screening [within 10 weeks of Baseline], Baseline, and every 4 weeks to Week 48 [visits at Weeks 28, 36 and 44 included telephone contact, others included clinic visits]) and a Follow-up visit 12 weeks after the end of treatment.
At the Baseline visit, eligible participants will start treatment with molgramostim nebulizer solution, 300 μg, administered by inhalation using an eFlow Nebulizer System. At each visit, sputum samples will be collected for staining and microscopy, and microbiological culture. In addition, clinical assessments including body weight, patient reported outcomes, and diffusion capacity of the lung for carbon monoxide (DLCO) will be conducted at each clinic visit. Spirometry will be assessed at Baseline, and at Weeks 12, 24, 32, 40 and 48. A 6-minute walk test (6-MWT) will be conducted at Baseline, at Weeks 12, 24, 48 and at the 12-week Follow-up visit. Safety laboratory samples will be collected at Screening, Baseline and at Weeks 4, 12, 24, 32, 40, 48 and at the 12-week Follow-up visit. Anti-GM-CSF antibodies will be assessed at Baseline, at Week 4, 12, 24, 32, 48, and at the 12-week Follow-up visit.
- Conditions
- Mycobacterium Infections, Nontuberculous
- Being tested
- Inhaled molgramostim, Antimycobacterial regimen
- Medical subject headings
- Mycobacterium Infections, Nontuberculous
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Inhaled molgramostimDrug
Also called rh-GM-CSF
300 µg / dose molgramostim (recombinant human GM-CSF) for inhalation
Antimycobacterial regimenDrug
Multidrug NTM guideline-based antimycobacterial regimen
| Study group | Role | What they receive |
|---|---|---|
| Inhaled molgramostim/antimycobacterials | Experimental | 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 Receives: Drug: Inhaled molgramostim; Drug: Antimycobacterial regimen |
| Inhaled molgramostim | Experimental | 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 Receives: Drug: Inhaled molgramostim |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Sputum Culture Conversion to Negative
Sputum culture conversion is defined as at least 3 consecutive sputum samples without growth of NTM during the treatment period.
Measured: 48 weeks
Secondary outcomes (18)
Number of Participants With Sputum Smear Conversion to Negative
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.
Measured: 48 weeks
Number of Participants With Durable Sputum Culture Conversion
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.
Measured: 60 weeks
Number of Participants With Durable Sputum Smear Conversion
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.
Measured: 60 weeks
Change From Baseline in Symptom Scores (Assessed Using Lower Respiratory Tract Infections - Visual Analogue Scale)
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).
Measured: Baseline to Week 48
Change From Baseline in Symptom Scores (Assessed Using Quality of Life Questionnaire - Bronchiectasis (QOL-B))
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.
Measured: Baseline to Week 48
Change From Baseline in Global Rating of Health (GRH)
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).
Measured: Baseline to Week 48
Change From Baseline in Body Weight
Measured: Baseline to Week 48
Change From Baseline in 6-Minute Walking Distance (6MWD)
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.
Measured: Baseline to Week 48
Change From Baseline in Dyspnea Scores During a 6MWT
Measured: Baseline to Week 48
Number of Adverse Events (AEs) During the Trial Period
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.
Measured: 60 weeks
Number of Serious AEs (SAEs) During the Trial Period
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).
Measured: 60 weeks
Number of Adverse Drug Reactions (ADRs) During the Trial Period
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.
Measured: 60 weeks
Number of Severe AEs During the Trial Period
All AEs were assessed by the investigator for severity (mild, moderate, severe) according to current regulatory standards.
Measured: 60 weeks
Number of Participants Withdrawn From Treatment Due to an AE During the Trial Period
Measured: 60 weeks
Change From Baseline in Diffusing Capacity of the Lung for Carbon Monoxide (DLCO)
Measured: Baseline to Week 48
Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1) (% Predicted)
Measured: Baseline to Week 48
Change From Baseline in Forced Vital Capacity (FVC) (% Predicted)
Measured: Baseline to Week 48
Number of Subjects With Development of Anti-GM-CSF Antibodies in Serum
Analyses for anti-GM-CSF antibodies were performed at a central laboratory.
Measured: 60 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Feb 2018
- Study start1 Mar 2018
- Primary completion15 Oct 2019
- Study completion13 Jan 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Mycobacterium Infections, NontuberculousStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Model description
- Open-label, non-controlled, multicenter, pilot clinical trial
- Masking
- None (open label)
- Enrolment
- 32 participants (actual)
Sponsor and investigators
- Lead sponsor
- Savara Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Grant Waterer, Prof., Principal investigator, Royal Perth Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03421743
- Sponsor's protocol ID
- SAV008-01
- Other IDs
- 2017-003374-14, 2017
- Acronym
- OPTIMA
- Status last verified
- Mar 2023
- First submitted
- 16 Jan 2018
- Last updated
- 3 Jul 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Thomson RM, Loebinger MR, Burke AJ, Morgan LC, Waterer GW, Ganslandt C. OPTIMA: An Open-Label, Noncomparative Pilot Trial of Inhaled Molgramostim in Pulmonary Nontuberculous Mycobacterial Infection. Ann Am Thorac Soc. 2024 Apr;21(4):568-576. doi: 10.1513/AnnalsATS.202306-532OC.
PubMed 37948736
Study at a glance
From the registry record, last updated by the sponsor on 3 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label, Non-controlled, Multicentre, Pilot Clinical Trial of Inhaled Molgramostim in Subjects With Antibiotic-resistant Non-tuberculosis Mycobacterial (NTM) Infection
- ClinicalTrials.gov ID
- NCT03421743
- Acronym / sponsor ID
- OPTIMA / SAV008-01
- Conditions
- Mycobacterium Infections, Nontuberculous
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 32 participants (actual)
- Lead sponsor
- Savara Inc.Industry (pharma, biotech, devices)
- Study start
- 1 Mar 2018 (actual)
- Primary completion
- 15 Oct 2019 (actual)
- Study completion
- 13 Jan 2020 (actual)
- First posted
- 5 Feb 2018 (actual)
- Results
- Posted 3 Jul 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 positive cultures in the prior 2 years, of which at least one is within the last 6 months prior to Screening, will be considered for enrollment. Participants should provide a positive NTM sputum culture at Screening to be eligible.
Two subgroups of participants will be recruited:
- Group 1: 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.
- Group 2: Participants 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.
The treatment period will consist of 14 trial visits (Screening [within 10 weeks of Baseline], Baseline, and every 4 weeks to Week 48 [visits at Weeks 28, 36 and 44 included telephone contact, others included clinic visits]) and a Follow-up visit 12 weeks after the end of treatment.
At the Baseline visit, eligible participants will start treatment with molgramostim nebulizer solution, 300 μg, administered by inhalation using an eFlow Nebulizer System. At each visit, sputum samples will be collected for staining and microscopy, and microbiological culture. In addition, clinical assessments including body weight, patient reported outcomes, and diffusion capacity of the lung for carbon monoxide (DLCO) will be conducted at each clinic visit. Spirometry will be assessed at Baseline, and at Weeks 12, 24, 32, 40 and 48. A 6-minute walk test (6-MWT) will be conducted at Baseline, at Weeks 12, 24, 48 and at the 12-week Follow-up visit. Safety laboratory samples will be collected at Screening, Baseline and at Weeks 4, 12, 24, 32, 40, 48 and at the 12-week Follow-up visit. Anti-GM-CSF antibodies will be assessed at Baseline, at Week 4, 12, 24, 32, 48, and at the 12-week Follow-up visit.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- Open-label, non-controlled, multicenter, pilot clinical trial
| Arm | Type | Receives |
|---|---|---|
| Inhaled molgramostim/antimycobacterials | Experimental | Drug: Inhaled molgramostim, Drug: Antimycobacterial regimenInhaled 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 |
| Inhaled molgramostim | Experimental | Drug: Inhaled molgramostimInhaled 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 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- 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:
- 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.
Key exclusion criteria (14 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Inhaled molgramostimAlso: rh-GM-CSF300 µg / dose molgramostim (recombinant human GM-CSF) for inhalation | Drug | Inhaled molgramostim, Inhaled molgramostim/antimycobacterials |
| Antimycobacterial regimenMultidrug NTM guideline-based antimycobacterial regimen | Drug | Inhaled molgramostim/antimycobacterials |
Primary outcome
Number of Participants With Sputum Culture Conversion to Negative
Time frame: 48 weeks
Sputum culture conversion is defined as at least 3 consecutive sputum samples without growth of NTM during the treatment period.
18 secondary outcomes
Number of Participants With Sputum Smear Conversion to Negative
Time frame: 48 weeks
Number of Participants With Durable Sputum Culture Conversion
Time frame: 60 weeks
Number of Participants With Durable Sputum Smear Conversion
Time frame: 60 weeks
Change From Baseline in Symptom Scores (Assessed Using Lower Respiratory Tract Infections - Visual Analogue Scale)
Time frame: Baseline to Week 48
Change From Baseline in Symptom Scores (Assessed Using Quality of Life Questionnaire - Bronchiectasis (QOL-B))
Time frame: Baseline to Week 48
Change From Baseline in Global Rating of Health (GRH)
Time frame: Baseline to Week 48
Change From Baseline in Body Weight
Time frame: Baseline to Week 48
Change From Baseline in 6-Minute Walking Distance (6MWD)
Time frame: Baseline to Week 48
Change From Baseline in Dyspnea Scores During a 6MWT
Time frame: Baseline to Week 48
Number of Adverse Events (AEs) During the Trial Period
Time frame: 60 weeks
Number of Serious AEs (SAEs) During the Trial Period
Time frame: 60 weeks
Number of Adverse Drug Reactions (ADRs) During the Trial Period
Time frame: 60 weeks
Number of Severe AEs During the Trial Period
Time frame: 60 weeks
Number of Participants Withdrawn From Treatment Due to an AE During the Trial Period
Time frame: 60 weeks
Change From Baseline in Diffusing Capacity of the Lung for Carbon Monoxide (DLCO)
Time frame: Baseline to Week 48
Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1) (% Predicted)
Time frame: Baseline to Week 48
Change From Baseline in Forced Vital Capacity (FVC) (% Predicted)
Time frame: Baseline to Week 48
Number of Subjects With Development of Anti-GM-CSF Antibodies in Serum
Time frame: 60 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Jul 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 37948736Thomson RM, Loebinger MR, Burke AJ, Morgan LC, Waterer GW, Ganslandt C. OPTIMA: An Open-Label, Noncomparative Pilot Trial of Inhaled Molgramostim in Pulmonary Nontuberculous Mycobacterial Infection. Ann Am Thorac Soc. 2024 Apr;21(4):568-576. doi: 10.1513/AnnalsATS.202306-532OC.
- Individual participant dataSharing planned: No
Related trials for Mycobacterium Infections, Nontuberculous
RecruitingObservational study
Study of Mycobacterial Infections
Mycobacterium Infections
Not yet recruitingPhase 2
Telacebec in Adults With Multibacillary Leprosy (TEBULA Leprosy Trial)
Leprosy, Multibacillary
Enrolling by invitationObservational study
Prospective Healthcare-Associated Links in Transmission of Nontuberculous Mycobacteria
Cystic Fibrosis, Nontuberculous Mycobacterial Pulmonary Infection
Active, not recruitingObservational study
Mycobacterial and Opportunistic Infections in HIV-Negative Thai and Taiwanese Patients Associated With Autoantibodies to Interferon-gamma
Nontuberculous Mycobacteria, Mycobacterium Tuberculosis
RecruitingPhase 2
A Study to Investigate Pharmacokinetics, Pharmacodynamics, and Safety of Subcutaneous Anifrolumab in Pediatric Participants 5 to < 18 Years of Age With Systemic Lupus Erythematosus
Systemic Lupus Erythematosus
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Grant Waterer, Prof. principal investigator
Royal Perth Hospital
5 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Inhaled molgramostim and Antimycobacterial regimen in people with Mycobacterium Infections, Nontuberculous, run by Savara Inc. at 5 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 3 Jul 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in Australia, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 3 Jul 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Sputum Culture Conversion to Negative (time frame: 48 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03421743, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Jul 2024.