Autoimmune Pulmonary Alveolar ProteinosisPhase 2IMPALANCT02702180
Efficacy and Safety of Inhaled Molgramostim (rhGM-CSF) in Autoimmune Pulmonary Alveolar Proteinosis
A Phase 2 study testing Molgramostim and PARI eFlow nebulizer system in people with Autoimmune Pulmonary Alveolar Proteinosis, run by Savara Inc. at 30 sites in 18 countries.
Trial summary
This study evaluates inhaled molgramostim (recombinant human granulocyte macrophage-colony stimulating factor [rhGM-CSF]) in the treatment of autoimmune pulmonary alveolar proteinosis (aPAP) patients. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Autoimmune Pulmonary Alveolar Proteinosis
- What is being tested?
- Molgramostim and PARI eFlow nebulizer system
- Type of treatment
- Medical device
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 139 participants
- Where
- 30 sitesin 18 countries
- Study dates
- 21 Mar 2016to 27 Sep 2019
- 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
- aPAP diagnosed by computed tomography, or by biopsy, or by Broncho Alveolar Lavage (BAL), and by increased GM-CSF autoantibodies in serum.
- Stable or progressive aPAP during a minimum period of 2 months prior to the Baseline visit.
- Arterial oxygen tension (PaO2) <75 mmHg/<10 kilo Pascal (kPa) at rest, OR desaturation of >4 percentage points on the 6MWT
- An alveolar-arterial oxygen difference [(A-a)DO2] of minimum 25 mmHg/3.33 kPa
- Female or male ≥18 years of age
- Females who have been post-menopausal for >1 year or females of childbearing potential after a confirmed menstrual period using a highly efficient method of contraception (i.e. a method with <1% failure rate such as combined hormonal contraception, progesterone-only hormonal contraception, intrauterine device, intrauterine hormone-releasing system, bilateral tubal occlusion, vasectomised partner, sexual abstinence), during and until 30 days after last dose of double-blind 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
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- Males agreeing to use condoms during and until 30 days after last dose of double-blind 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
- Diagnosis of hereditary or secondary PAP
- WLL within 1 month of Baseline
- Treatment with GM-CSF within 3 months of Baseline
- Treatment with rituximab within 6 months of Baseline
- Treatment with plasmapheresis within 3 months of Baseline
- Treatment with any investigational medicinal product within 4 weeks of Screening
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- Concomitant use of sputum modifying drugs such as carbocysteine or ambroxol
- History of allergic reactions to GM-CSF
- Connective tissue disease, inflammatory bowel disease or other autoimmune disorder requiring treatment associated with significant immunosuppression, e.g. more than 10 mg/day systemic prednisolone
- Previous experience of severe and unexplained side-effects during aerosol delivery of any kind of medicinal product
- History of, or present, myeloproliferative disease or leukaemia
- Known active infection (viral, bacterial, fungal or mycobacterial)
- Apparent pre-existing concurrent pulmonary fibrosis
- Any other serious medical condition which in the opinion of the investigator would make the participant unsuitable for the trial
Trial locations and contacts
30 sites in 18 countries.
30 sites in 18 countries
Japan5 sites
Niigata University Medical and Dental Hospital
Niigata
National Hospital Organization Kinki-Chuo
Osaka
Tohoku University Hospital
Sendai
Aichi Medical University Hospital
Toyohashi
Kanagawa Cardiovascular and Respiratory Center
Yokohama
Germany4 sites
Westdeutsches Lungenzentrum am Universitätsklinikum Essen
Essen
Asklepios Fachkliniken München - Gauting
Gauting, 82131
Thoraxklinik am Universitätsklinikum Heidelberg
Heidelberg
Universitätsklinikum Schleswig-Holstein Zentralklinikum, Lübeck Medizinische Klinik III, Pneumologie
Lübeck, 23538
United States3 sites
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
University of Florida
Gainesville, Florida, 32610
UCLA
Los Angeles, California, 90095
South Korea3 sites
Seoul National University Hospital
Seoul, 0 3080
Asan Medical Center, Division of Pulmonary and Critical Care Medicine
Seoul, 05505
Samsung Medical Center, Division of Pulmonary and Critical Care Medicine
Seoul, 135-710
Portugal2 sites
Hospital de dia de Pneumologia
Lisbon
Hospital Sao Joao
Porto, 4200-319
Slovakia1 site
II. Pulmonary Department National Institut for TB, Lung Diseases and Chest Surgery
Vyšné Hágy, 05984
Turkey (Türkiye)1 site
Yedikule Pulmonary Diseases and Pulmonary Surgery Training and Research Hospital
Istanbul, 34020
About this study
Official title: A Randomised, Double-blind, Placebo-controlled Multicentre Clinical Trial of Inhaled Molgramostim in Autoimmune Pulmonary Alveolar Proteinosis Patients
This study evaluates inhaled molgramostim (recombinant human granulocyte macrophage-colony stimulating factor [rhGM-CSF]) in the treatment of autoimmune pulmonary alveolar proteinosis (aPAP) patients. A third of the patients will receive inhaled molgramostim once daily for 24 weeks, a third will receive inhaled molgramostim intermittently (7 days on, 7 days off) for 24 weeks and a third will receive inhaled matching placebo for 24 weeks.
Read the detailed description
The trial is a phase 2, randomized, double-blind, placebo-controlled multicentre clinical trial investigating efficacy and safety of inhaled molgramostim (rhGM-CSF) in patients with aPAP.
The trial will include 2 periods; a double-blind treatment period consisting of up to 8 trial visits (Screening, Baseline, and at Weeks 4, 8,12, 16, 20 and 24 after randomisation) and a open-label follow-up period consisting of up to 5 trial visits (at Weeks 4, 12, 24, 36 and 48 post-treatment).
In the double-blind treatment period, eligible subjects will be randomised to treatment for up to 24 weeks with either: 1) inhaled molgramostim (300 µg) once daily (MOL-OD), 2) inhaled molgramostim (300 µg) and matching placebo administered intermittently (7 days on and 7 days off) (MOL-INT) or 3) inhaled placebo once daily (PBO). During the follow-up period, all participants will receive inhaled molgramostim intermittently (7 days on, 7 days off). During the trial, whole lung lavage (WLL) may be applied as rescue therapy in case of significant clinical worsening.
- Being tested
- Molgramostim, Placebo, PARI eFlow nebulizer system
- Medical subject headings
- Pulmonary Alveolar Proteinosis, Acquired
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MolgramostimDrug
Also called rhGM-CSF
300 mcg molgramostim (rhGM-CSF) nebulizer solution for inhalation
PlaceboDrug
Placebo nebulizer solution for inhalation
PARI eFlow nebulizer systemDevice
PARI eFlow nebulizer system
| Study group | Role | What they receive |
|---|---|---|
| Double-blind molgramostim once daily | Experimental | Inhalation of molgramostim nebuliser solution 300 mcg once daily for 24 weeks Receives: Drug: Molgramostim; Device: PARI eFlow nebulizer system |
| Double-blind molgramostim intermittent | Experimental | Inhalation of molgramostim nebuliser solution 300 mcg for 7 days and placebo nebuliser solution for 7 days for 24 weeks (12 cycles) Receives: Drug: Molgramostim; Device: PARI eFlow nebulizer system |
| Double-blind placebo | Placebo comparator | Inhalation of placebo nebuliser solution once daily for 24 weeks Receives: Drug: Placebo; Device: PARI eFlow nebulizer system |
| Open-label molgramostim intermittent | Experimental | Inhalation of molgramostim nebuliser solution 300 mcg for 7 days and placebo nebuliser solution for 7 days for 24 or 48 weeks from completion of the double-blind period Receives: Drug: Molgramostim; Device: PARI eFlow nebulizer system |
What is being measured
The primary outcome is the main question the study is designed to answer.
Absolute Change From Baseline of Alveolar-arterial Oxygen Concentration (A-a(DO2)) After 24 Weeks of Treatment
Measurement of (A-a)DO2 was done by blood gas analysis. An arterial blood sample was collected in the supine position, after resting for at least 10 minutes (or longer if required to achieve stable oxygen saturation). The sample was analyzed for arterial oxygen tension (PaO2) and partial pressure of carbon dioxide (PaCO2). The calculation of (A-a)DO2 was done centrally by using a formula described in the protocol.
Measured: From baseline to 24 weeks
Secondary outcomes (8)
Change From Baseline in 6-minute Walking Distance (6MWD) After 24 Weeks of Treatment
The 6MWD was assessed by the use of 6-minute walking test (6MWT). The 6MWT was performed in accordance with the 2014 ATS/ERS guideline "field walking tests in chronic respiratory disease" (Holland et al. 2014) by technicians with documented training and experience.
Where possible, the test was conducted with the participant breathing ambient atmospheric air. If the participant required oxygen supplementation at rest, a titration procedure was carried out as part of the 6MWT at screening in order to determine the oxygen flow rate required for the participant to complete the test. This flow rate was to be used during subsequent tests, if possible.
Measured: From baseline to 24 weeks
Change From Baseline in St. George's Respiratory Questionnaire (SGRQ) Total Score After 24 Weeks of Treatment
The SGRQ is designed to measure health impairment in patients with asthma and chronic obstructive pulmonary disease (COPD). It consists of two parts, where Part 1 covers the participants' recollection of their symptoms over the preceding period (1 month recall used in this trial, Symptoms component) and Part 2 addresses the participants' current state in terms of disturbances to their daily physical activity (Activity component) and a wide range of disturbances of psycho-social function (Impact component). A total score as well as individual component scores were calculated. Scores (total and component) range from 0 to 100, with higher scores indicating more limitations.
Measured: From baseline to 24 weeks
Number of Whole Lung Lavage During 24 Weeks of Treatment
In all versions of the protocol (no participants were recruited under version 1.0), whole lung lavage (single lung or both lungs) was applied as rescue therapy. In protocol version 2.0, the criterion for performing whole lung lavage was an increase in (A-a)DO2 by more than 10 mmHg/1.33 kilopascal compared to baseline. In protocol version 3.0 onwards, the criterion for performing whole lung lavage was clinical worsening of aPAP based on symptoms, reduced exercise capacity and/or findings of hypoxemia or desaturation according to the investigator's judgement.
In protocol versions 2.0 and 3.0, participants undergoing whole lung lavage during the double-blind period were to discontinue double-blind treatment, encouraged to continue to follow the same visit schedule and, if required, receive further whole lung lavage at the investigator's discretion. In protocol version 4.0 and 5.0, participants undergoing whole lung lavage were to continue double-blind treatment.
Measured: From baseline to 24 weeks
Number of Adverse Events (AEs) During 24 Weeks of Treatment
Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose.
Information about AEs was collected by the investigator by a non-leading question such as "have you experienced any new health problems or worsening of existing conditions" and by reporting events directly observed or spontaneously volunteered by participants (e.g., in the Subject Diary Card, where subjects were asked to record any AEs and answer questions regarding lung toxicity and known systemic effects). Participants were also encouraged to contact the clinic in between visits if they experienced AEs or had any concerns.
All AEs were assessed by the investigator for severity (mild, moderate, severe), outcome (recovered, not recovered, recovered with sequelae, fatal, unknown) and causality (unlikely, possible, probable, not applicable) according to current regulatory standards.
Measured: From baseline to 24 weeks
Number of Serious Adverse Events (SAEs) During 24 Weeks of Treatment
SAEs are 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 anomaly or birth defect
- May jeopardise the subject or may require intervention to prevent one or more of the other outcomes listed above (Important Medical Events)
Measured: From baseline to 24 weeks
Number of Adverse Drug Reactions (ADRs) During 24 Weeks of Treatment
All AEs judged by either the reporting investigator or the sponsor as having a reasonable causal relationship to a medicinal product qualify as ADRs. The expression reasonable causal relationship means that a causal relationship between a medicinal product and an AE is at least a reasonable possibility i.e. the relationship cannot be ruled out.
Measured: From baseline to 24 weeks
Number of Severe AEs During 24 Weeks of Treatment
All AEs were assessed by the investigator for severity (mild, moderate, severe) according to current regulatory standards:
- Mild: The AE is easily tolerated and does not interfere with daily activity.
- Moderate: The AE interferes with daily activity, but the subject is still able to function. Medical intervention may be considered.
- Severe: The AE is incapacitating and requires medical intervention.
Measured: From baseline to 24 weeks
Number of Participants With at Least 1 AE Leading to Treatment Discontinuation During 24 Weeks of Treatment
Participants could be discontinued from treatment and assessments at any time, if deemed necessary by the investigator. Potential reasons for discontinuation of treatment included e.g. unacceptable AE.
Measured: From baseline to 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Mar 2016 (estimated)
- Study start21 Mar 2016
- Primary completion19 Apr 2019
- Study completion27 Sep 2019
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 Autoimmune Pulmonary Alveolar ProteinosisStudies 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 139 participants (actual)
Sponsor and investigators
- Lead sponsor
- Savara Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Cliff Morgan, MD, Principal investigator, Royal Brompton Hospital, London
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02702180
- Sponsor's protocol ID
- MOL-PAP-002
- Other IDs
- 2015-003878-33, 2015
- Acronym
- IMPALA
- Status last verified
- Apr 2023
- First submitted
- 28 Feb 2016
- Last updated
- 12 Apr 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- Yes
- Individual data sharing
- No
Publications
Trapnell BC, Inoue Y, Bonella F, Morgan C, Jouneau S, Bendstrup E, Campo I, Papiris SA, Yamaguchi E, Cetinkaya E, Ilkovich MM, Kramer MR, Veltkamp M, Kreuter M, Baba T, Ganslandt C, Tarnow I, Waterer G, Jouhikainen T; IMPALA Trial Investigators. Inhaled Molgramostim Therapy in Autoimmune Pulmonary Alveolar Proteinosis. N Engl J Med. 2020 Oct 22;383(17):1635-1644. doi: 10.1056/NEJMoa1913590. Epub 2020 Sep 7.
PubMed 32897035
Study at a glance
From the registry record, last updated by the sponsor on 12 Apr 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomised, Double-blind, Placebo-controlled Multicentre Clinical Trial of Inhaled Molgramostim in Autoimmune Pulmonary Alveolar Proteinosis Patients
- ClinicalTrials.gov ID
- NCT02702180
- Acronym / sponsor ID
- IMPALA / MOL-PAP-002
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 139 participants (actual)
- Lead sponsor
- Savara Inc.Industry (pharma, biotech, devices)
- Study start
- 21 Mar 2016 (actual)
- Primary completion
- 19 Apr 2019 (actual)
- Study completion
- 27 Sep 2019 (actual)
- First posted
- 8 Mar 2016 (estimated)
- Results
- Posted 15 Apr 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study evaluates inhaled molgramostim (recombinant human granulocyte macrophage-colony stimulating factor [rhGM-CSF]) in the treatment of autoimmune pulmonary alveolar proteinosis (aPAP) patients. A third of the patients will receive inhaled molgramostim once daily for 24 weeks, a third will receive inhaled molgramostim intermittently (7 days on, 7 days off) for 24 weeks and a third will receive inhaled matching placebo for 24 weeks.
Detailed description
The trial is a phase 2, randomized, double-blind, placebo-controlled multicentre clinical trial investigating efficacy and safety of inhaled molgramostim (rhGM-CSF) in patients with aPAP.
The trial will include 2 periods; a double-blind treatment period consisting of up to 8 trial visits (Screening, Baseline, and at Weeks 4, 8,12, 16, 20 and 24 after randomisation) and a open-label follow-up period consisting of up to 5 trial visits (at Weeks 4, 12, 24, 36 and 48 post-treatment).
In the double-blind treatment period, eligible subjects will be randomised to treatment for up to 24 weeks with either: 1) inhaled molgramostim (300 µg) once daily (MOL-OD), 2) inhaled molgramostim (300 µg) and matching placebo administered intermittently (7 days on and 7 days off) (MOL-INT) or 3) inhaled placebo once daily (PBO). During the follow-up period, all participants will receive inhaled molgramostim intermittently (7 days on, 7 days off). During the trial, whole lung lavage (WLL) may be applied as rescue therapy in case of significant clinical worsening.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Double-blind molgramostim once daily | Experimental | Drug: Molgramostim, Device: PARI eFlow nebulizer systemInhalation of molgramostim nebuliser solution 300 mcg once daily for 24 weeks |
| Double-blind molgramostim intermittent | Experimental | Drug: Molgramostim, Device: PARI eFlow nebulizer systemInhalation of molgramostim nebuliser solution 300 mcg for 7 days and placebo nebuliser solution for 7 days for 24 weeks (12 cycles) |
| Double-blind placebo | Placebo comparator | Drug: Placebo, Device: PARI eFlow nebulizer systemInhalation of placebo nebuliser solution once daily for 24 weeks |
| Open-label molgramostim intermittent | Experimental | Drug: Molgramostim, Device: PARI eFlow nebulizer systemInhalation of molgramostim nebuliser solution 300 mcg for 7 days and placebo nebuliser solution for 7 days for 24 or 48 weeks from completion of the double-blind period |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- aPAP diagnosed by computed tomography, or by biopsy, or by Broncho Alveolar Lavage (BAL), and by increased GM-CSF autoantibodies in serum.
- Stable or progressive aPAP during a minimum period of 2 months prior to the Baseline visit.
- Arterial oxygen tension (PaO2) <75 mmHg/<10 kilo Pascal (kPa) at rest, OR desaturation of >4 percentage points on the 6MWT
- An alveolar-arterial oxygen difference [(A-a)DO2] of minimum 25 mmHg/3.33 kPa
- Female or male ≥18 years of age
Key exclusion criteria (14 listed)
- Diagnosis of hereditary or secondary PAP
- WLL within 1 month of Baseline
- Treatment with GM-CSF within 3 months of Baseline
- Treatment with rituximab within 6 months of Baseline
- Treatment with plasmapheresis within 3 months of Baseline
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 |
|---|---|---|
| MolgramostimAlso: rhGM-CSF300 mcg molgramostim (rhGM-CSF) nebulizer solution for inhalation | Drug | Double-blind molgramostim intermittent, Double-blind molgramostim once daily, Open-label molgramostim intermittent |
| PlaceboPlacebo nebulizer solution for inhalation | Drug | Double-blind placebo |
| PARI eFlow nebulizer systemPARI eFlow nebulizer system | Device | Double-blind molgramostim intermittent, Double-blind molgramostim once daily, Double-blind placebo, Open-label molgramostim intermittent |
Primary outcome
Absolute Change From Baseline of Alveolar-arterial Oxygen Concentration (A-a(DO2)) After 24 Weeks of Treatment
Time frame: From baseline to 24 weeks
Measurement of (A-a)DO2 was done by blood gas analysis. An arterial blood sample was collected in the supine position, after resting for at least 10 minutes (or longer if required to achieve stable oxygen saturation). The sample was analyzed for arterial oxygen tension (PaO2) and partial pressure of carbon dioxide (PaCO2). The calculation of (A-a)DO2 was done centrally by using a formula described in the protocol.
8 secondary outcomes
Change From Baseline in 6-minute Walking Distance (6MWD) After 24 Weeks of Treatment
Time frame: From baseline to 24 weeks
Change From Baseline in St. George's Respiratory Questionnaire (SGRQ) Total Score After 24 Weeks of Treatment
Time frame: From baseline to 24 weeks
Number of Whole Lung Lavage During 24 Weeks of Treatment
Time frame: From baseline to 24 weeks
Number of Adverse Events (AEs) During 24 Weeks of Treatment
Time frame: From baseline to 24 weeks
Number of Serious Adverse Events (SAEs) During 24 Weeks of Treatment
Time frame: From baseline to 24 weeks
Number of Adverse Drug Reactions (ADRs) During 24 Weeks of Treatment
Time frame: From baseline to 24 weeks
Number of Severe AEs During 24 Weeks of Treatment
Time frame: From baseline to 24 weeks
Number of Participants With at Least 1 AE Leading to Treatment Discontinuation During 24 Weeks of Treatment
Time frame: From baseline to 24 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Apr 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 32897035Trapnell BC, Inoue Y, Bonella F, Morgan C, Jouneau S, Bendstrup E, Campo I, Papiris SA, Yamaguchi E, Cetinkaya E, Ilkovich MM, Kramer MR, Veltkamp M, Kreuter M, Baba T, Ganslandt C, Tarnow I, Waterer G, Jouhikainen T; IMPALA Trial Investigators. Inhaled Molgramostim Therapy in Autoimmune Pulmonary Alveolar Proteinosis. N Engl J Med. 2020 Oct 22;383(17):1635-1644. doi: 10.1056/NEJMoa1913590. Epub 2020 Sep 7.
- Individual participant dataSharing planned: No
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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.
Cliff Morgan, MD principal investigator
Royal Brompton Hospital, London
30 sites in 18 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Molgramostim and PARI eFlow nebulizer system in people with Autoimmune Pulmonary Alveolar Proteinosis, run by Savara Inc. at 30 sites in 18 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 9 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 12 Apr 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 30 sites in Japan, Germany, United States, South Korea, Portugal and 13 more countries. 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. Some participants in this study may receive a placebo. 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 15 Apr 2022). 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?
Absolute Change From Baseline of Alveolar-arterial Oxygen Concentration (A-a(DO2)) After 24 Weeks of Treatment (time frame: From baseline to 24 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 NCT02702180, 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 12 Apr 2023.