Cystic FibrosisPhase 2CARE-CF1NCT03000348

A Study of the Dosing, Efficacy, and Safety of Oral Cysteamine in Adult Patients With Cystic Fibrosis Exacerbations

A Phase 2 study testing Cysteamine in people with Cystic Fibrosis, run by NovaBiotics Ltd. at 17 sites in 3 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Trial summary

This study investigates the use of cysteamine in the treatment of adults with Cystic Fibrosis who are experiencing an exacerbation of CF-associated lung disease. There are six different potential dosing regimens, including one that is placebo. Read more about this study

Who is it for?: Ages 18 years and older
Conditions the study is for: Cystic Fibrosis
What is being tested?: Cysteamine
Type of treatment: Pill or oral medicine
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 91 participants
Where: 17 sitesin 3 countries
Study dates: Dec 2016to Apr 2018
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

CF-associated lung disease with documented history of chronic infection with Gram-negative organism(s)
Established patient of the Principal Investigator's CF Multi Disciplinary Team (MDT)
Age ≥18 years
Weight >40 kg
FEV1 >30% of predicted within the 6 months prior to study exacerbation
At the baseline visit: experiencing a new exacerbation of CF-associated lung disease (based on Investigator assessment of ≥4 symptoms present on the Fuchs' criteria) requiring treatment that includes an aminoglycoside antibiotic

Show all 23Show fewer

Females of childbearing potential will be included if they are either sexually inactive (sexually abstinent for 14 days prior to the first study drug dose continuing through 28 days after the last study drug dose, or using one of the following highly effective contraceptive (i.e. results in <1% failure rate when used consistently and correctly) methods in this trial:
intrauterine device (IUD);
surgical sterilization of the partner (vasectomy for 6 months minimum);
combined (estrogen or progestogen containing) hormonal contraception associated with the inhibition of ovulation (either oral, intravaginal, or transdermal);
progestogen only hormonal contraception associated with the inhibition of ovulation (either oral, injectable, or implantable);
intrauterine hormone releasing system (IUS);
bilateral tubal occlusion.
Females of childbearing potential agree to remain sexually inactive or to keep the same birth control method for at least 28 days following the last dose.
A female of non-childbearing potential must have undergone one of the following sterilization procedures at least 6 months prior to the first study drug dose:
hysteroscopic sterilization;
bilateral tubal ligation or bilateral salpingectomy;
hysterectomy;
bilateral oophorectomy; or be postmenopausal with amenorrhea for at least 1 year prior to the first study drug dose and follicle stimulating hormone (FSH) serum levels consistent with postmenopausal status.
A non-vasectomized male subject agrees to use a condom with spermicide or abstain from sexual intercourse during the study until 90 days beyond the last dose of study medication and the female partner agrees to comply with inclusion 7 or 9. For a vasectomized male who has had his vasectomy 6 months or more prior to study start, it is required that they use a condom during sexual intercourse. A male who has been vasectomized less than 6 months prior to study start must follow the same restrictions as a non-vasectomized male.
If male, agrees not to donate sperm from the first study drug dose until 90 days after dosing.
Willing and able to comply with all protocol requirements and procedures, including induction of sputum, if necessary
Willing and able to provide signed and dated informed consent

Who cannot join

Hypersensitive to cysteamine or to any of the excipients
Hypersensitive to penicillamine
Transplant recipient
Participation in any other interventional clinical research study (participation in observational studies is not exclusionary) within 30 days of Baseline (Day 0), and any planned participation in an interventional clinical research study for the duration of this study
If female, pregnancy, planned pregnancy, or breast-feeding
Any other significant disease/disorder which, in the Investigator's opinion, either puts the patient at risk due to study participation, or may influence the results of the study or the patient's ability to participate in the study

Trial locations and contacts

17 sites in 3 countries.

Country

City, postcode or hospital

17 sites in 3 countries

United States8 sites

Albany Medical College
Albany, New York, 12208
Nationwide Children's Hospital
Columbus, Ohio, 43205
University of Florida
Gainesville, Florida, 32610
The Medical College of Wisconsin/Froedtert Hospital
Milwaukee, Wisconsin, 53226
West Virginia University
Morgantown, West Virginia, 26506
Central Florida Pulmonary
Orlando, Florida, 32803
San Francisco Critical Care Medical Group California Pacific Medical Center
San Francisco, California, 94115
Banner University of Arizona Medical Center
Tucson, Arizona, 85724

Other trials in United States

United Kingdom7 sites

Aberdeen Royal Infirmary
Aberdeen, Scotland, AB25 2ZN
Ninewells Hospital Scottish Adult Cystic Fibrosis Service
Dundee, DD1 9SY
Western General Hospital Edinburgh, CF Adults / CF Unit
Edinburgh, EH4 3HE
NHS GGC
Glasgow, G51 4TF
Raigmore Hospital
Inverness, IV2 3UJ
St. James University Hospital
Leeds, LS9 7TF
Royal Victoria Infirmary Adult CF Centre
Newcastle upon Tyne, NE1 4LP

Other trials in United Kingdom

Italy2 sites

Ospedale Padiatrico Bambino Gesu Centro Fibrosi Cistica
Roma, 00165
Azienda Ospedaliera Universitaria Integrato di Verona Borgo Trento Centro Fibrosi Cistica
Verona, 37126

Other trials in Italy

About this study

Official title: A Randomized, Double-Blind, Parallel Group, Placebo-Controlled Study Investigating the Optimal Dose Regimen, Efficacy, and Safety of Adding Oral Cysteamine in Adult Patients Being Treated for an Exacerbation of CF-associated Lung Disease

Read the detailed description

This is a multicenter, double-blind, randomized, placebo-controlled, 6-arm study to investigate the optimal dose regimen, efficacy, and safety of cysteamine in the treatment of adult patients with CF who are experiencing an exacerbation of CF-associated lung disease. Patients will be screened for the study and eligible patients will be randomized to receive either cysteamine or placebo as add-on therapy to their standard of care treatment for CF-associated lung disease.

Conditions: Cystic Fibrosis
Being tested: Cysteamine, Placebo Oral Capsule
Keywords: Exacerbation, CF, Lung disease, Lung infection, Gram negative, Bacterial Infection, pneumonia, bronchitis
Medical subject headings: Cystic Fibrosis, Lung Diseases, Bacterial Infections, Pneumonia, Bronchitis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

CysteamineDrug
Also called Lynovex, NM001, Lynovex Oral
Oral Cysteamine Capsule
Placebo Oral CapsuleDrug
Placebo Oral Capsule

Study groups in NCT03000348
Study group	Role	What they receive
High Dose, Once per day	Active comparator	Patient takes one oral dose of Cysteamine (high dose) per day, in the morning. The patient takes two oral placebo doses, one at mid-day and one in the evening. Receives: Drug: Cysteamine; Drug: Placebo Oral Capsule
High Dose, Twice per day	Active comparator	Patient takes two oral doses of Cysteamine (high dose) per day, one in the morning and one in the evening. The patient takes one oral placebo dose, at mid-day. Receives: Drug: Cysteamine; Drug: Placebo Oral Capsule
High Dose, Three times per day	Active comparator	Patient takes three oral doses of Cysteamine (high dose) per day, one in the morning, one at mid-day and one in the evening. Receives: Drug: Cysteamine
Placebo	Placebo comparator	Patient takes three oral doses of placebo, one in the morning, one at mid-day and one in the evening. Receives: Drug: Placebo Oral Capsule
Low Dose, Three times per day	Active comparator	Patient takes three oral doses of Cysteamine (low dose) per day, one in the morning, one at mid-day and one in the evening. Receives: Drug: Cysteamine; Drug: Placebo Oral Capsule
Mid-Range Dose, Three times per day	Active comparator	Patient takes three oral doses of Cysteamine (mid-range dose) per day, one in the morning, one at mid-day and one in the evening. Receives: Drug: Cysteamine; Drug: Placebo Oral Capsule

What is being measured

The primary outcome is the main question the study is designed to answer.

Change From Baseline in Sputum Bacterial Load
Change from baseline through to Day 21 in log10 cfu/ml transformed total gram negative sputum bacterial load
Measured: Baseline through Day 21/End of Study
Safety and Tolerability Assessed by the Number of Subjects With Adverse Events
Assessed by variables such as adverse events (AEs), laboratory assessments, physical examinations, and vital signs.
Measured: Baseline through Day 21/End of Study

Secondary outcomes (12)

Change From Baseline in Neutrophil Elastase Levels
Actual values and change from baseline in neutrophil elastase levels were summarized using descriptive statistics by visit for each treatment group and each TDD group for the ITT Population.
Measured: Baseline through Day 21/End of Study
Change From Baseline in Sputum IL8
Sputum IL-8 Levels by Visit - Covariate Adjusted ANCOVA with Observed Data ITT Population
Measured: Baseline through Day 21/End of Study
Change From Baseline in FEV1
Change from Baseline in FEV1 Percent Predicted (%) - Covariate Adjusted ANCOVA with Observed Data ITT Population
Measured: Baseline through Day 21/End of Study
Change From Baseline in BMI
BMI (kg/m^2) by Visit - ANCOVA with Observed Data ITT Population
Measured: Baseline through Day 21/End of Study
Change From Baseline in C-Reactive Protein
Change from baseline in C-Reactive Protein at visits 7, 14 and 21
Measured: Baseline through Day 21
Change From Baseline in Blood Leukocyte Count
Blood Leukocyte Count (10^9 leucocytes/L) by Visit - ANCOVA with Observed Data ITT Population
Measured: Baseline through Day 21/End of Study
Assessment of Blood Cysteamine Levels
Study Drug Plasma at Day 14 Safety Population
Measured: Day 14
Assessment of Sputum Cysteamine Levels
Study Drug Sputum Concentrations at Day 14 Safety Population
Measured: Day 14
Change From Baseline in CFRSD-CRISS
Mean Change from Baseline in Cystic Fibrosis Respiratory Symptom Diary (CFRSD)-Chronic Respiratory Infection Symptom Scale (CRISS) CRFSD-CRISS:The CFRSD is a 16-item PROM to evaluate the effect of treatment on the severity of symptoms of acute respiratory infections associated with CF (i.e., CFRSD-CRISS) and to assess the emotional and activity impacts of these symptoms. The overall CRISS score range is 0-100 with 100 being the most severe symptoms.The CFRSD-CRISS is a validated unidimensional scale based on a subset of 8 items from the CFRSD questionnaire that quantifies symptom severity for the previous 24 hours to capture the magnitude of symptoms in stable CF, during medically treated CF exacerbations, and during recover from an exacerbation. The 8 items on the CFRSD-CRISS were scored using a 5-point Likert scale ranging from 0 (no symptom) to 4 (the highest magnitude of severity). So score range of 0-32.
Measured: Baseline through to Day 21
Change From Baseline in CFQ-R
The CFQ-R is a disease-specific HRQOL (Health related quality of life) measure containing both generic and CF-specific scales and measures functioning during the previous 2 weeks. Each CFQ-R scale yielded standardized scores ranging from 0 to 100; higher scores indicated better HRQOL
Measured: Baseline through Day 21/End of Study
Change From Baseline in Jarad and Sequeiros Symptom Score Questionnaire
The Jarad and Sequeiros Symptom Questionnaire (Jarad, 2012) is a simple participant-completed questionnaire that assesses and evaluates change in participant symptoms related to different aspects of respiratory function during a CF exacerbation. The questionnaire consists of 4 questions, each answered on a 4-point scale ranging from 1 (best) to 4 (worst). A range of minimum 4 to maximum16.Jarad and Sequeiros Questionnaire Score - changes from baseline at day 7 and day 14
Measured: changes from baseline at day 7 and day 14
Change From Baseline in Weight
Weight (kg) by visit - ANCOVA with observed data
Measured: Baseline through Day 21/End of Study

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Dec 2016Apr 2018

First posted22 Dec 2016 (estimated)
Study startDec 2016
Primary completionApr 2018
Study completionApr 2018

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 Cystic FibrosisStudies 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: 91 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT03000348
Sponsor's protocol ID: NBTCS02
Acronym: CARE-CF1
Status last verified: Apr 2021
First submitted: 11 Dec 2016
Last updated: 14 Apr 2021
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes

Publications

Devereux G, Wrolstad D, Bourke SJ, Daines CL, Doe S, Dougherty R, Franco R, Innes A, Kopp BT, Lascano J, Layish D, MacGregor G, Murray L, Peckham D, Lucidi V, Lovie E, Robertson J, Fraser-Pitt DJ, O'Neil DA. Oral cysteamine as an adjunct treatment in cystic fibrosis pulmonary exacerbations: An exploratory randomized clinical trial. PLoS One. 2020 Dec 28;15(12):e0242945. doi: 10.1371/journal.pone.0242945. eCollection 2020.
PubMed 33370348

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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Study at a glance

From the registry record, last updated by the sponsor on 14 Apr 2021. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Randomized, Double-Blind, Parallel Group, Placebo-Controlled Study Investigating the Optimal Dose Regimen, Efficacy, and Safety of Adding Oral Cysteamine in Adult Patients Being Treated for an Exacerbation of CF-associated Lung Disease
ClinicalTrials.gov ID: NCT03000348
Acronym / sponsor ID: CARE-CF1 / NBTCS02
Conditions: Cystic Fibrosis
Keywords: Exacerbation, CF, Lung disease, Lung infection, Gram negative, Bacterial Infection, pneumonia, bronchitis
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 91 participants (actual)
Lead sponsor: NovaBiotics Ltd.Industry (pharma, biotech, devices)
Collaborators: Agility Clinical, Inc., PSR Group B.V.
Study start: Dec 2016
Primary completion: Apr 2018 (actual)
Study completion: Apr 2018 (actual)
First posted: 22 Dec 2016 (estimated)
Results: Posted 14 Apr 2021

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind (participant, care provider, investigator, outcomes assessor)
Number of arms: 6

Arm	Type	Receives
High Dose, Once per day	Active comparator	Drug: Cysteamine, Drug: Placebo Oral CapsulePatient takes one oral dose of Cysteamine (high dose) per day, in the morning. The patient takes two oral placebo doses, one at mid-day and one in the evening.
High Dose, Twice per day	Active comparator	Drug: Cysteamine, Drug: Placebo Oral CapsulePatient takes two oral doses of Cysteamine (high dose) per day, one in the morning and one in the evening. The patient takes one oral placebo dose, at mid-day.
High Dose, Three times per day	Active comparator	Drug: CysteaminePatient takes three oral doses of Cysteamine (high dose) per day, one in the morning, one at mid-day and one in the evening.
Placebo	Placebo comparator	Drug: Placebo Oral CapsulePatient takes three oral doses of placebo, one in the morning, one at mid-day and one in the evening.
Low Dose, Three times per day	Active comparator	Drug: Cysteamine, Drug: Placebo Oral CapsulePatient takes three oral doses of Cysteamine (low dose) per day, one in the morning, one at mid-day and one in the evening.
Mid-Range Dose, Three times per day	Active comparator	Drug: Cysteamine, Drug: Placebo Oral CapsulePatient takes three oral doses of Cysteamine (mid-range dose) per day, one in the morning, one at mid-day and one in the evening.

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (23 listed)

CF-associated lung disease with documented history of chronic infection with Gram-negative organism(s)
Established patient of the Principal Investigator's CF Multi Disciplinary Team (MDT)
Age ≥18 years
Weight >40 kg
FEV1 >30% of predicted within the 6 months prior to study exacerbation

Key exclusion criteria (6 listed)

Hypersensitive to cysteamine or to any of the excipients
Hypersensitive to penicillamine
Transplant recipient
Participation in any other interventional clinical research study (participation in observational studies is not exclusionary) within 30 days of Baseline (Day 0), and any planned participation in an interventional clinical research study for the duration of this study
If female, pregnancy, planned pregnancy, or breast-feeding

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
CysteamineAlso: Lynovex, NM001, Lynovex OralOral Cysteamine Capsule	Drug	High Dose, Once per day, High Dose, Three times per day, High Dose, Twice per day, Low Dose, Three times per day, Mid-Range Dose, Three times per day
Placebo Oral CapsulePlacebo Oral Capsule	Drug	High Dose, Once per day, High Dose, Twice per day, Low Dose, Three times per day, Mid-Range Dose, Three times per day, Placebo

Primary outcome

Change From Baseline in Sputum Bacterial Load
Time frame: Baseline through Day 21/End of Study
Change from baseline through to Day 21 in log10 cfu/ml transformed total gram negative sputum bacterial load
Safety and Tolerability Assessed by the Number of Subjects With Adverse Events
Time frame: Baseline through Day 21/End of Study
Assessed by variables such as adverse events (AEs), laboratory assessments, physical examinations, and vital signs.

12 secondary outcomes

Change From Baseline in Neutrophil Elastase Levels
Time frame: Baseline through Day 21/End of Study
Change From Baseline in Sputum IL8
Time frame: Baseline through Day 21/End of Study
Change From Baseline in FEV1
Time frame: Baseline through Day 21/End of Study
Change From Baseline in BMI
Time frame: Baseline through Day 21/End of Study
Change From Baseline in C-Reactive Protein
Time frame: Baseline through Day 21
Change From Baseline in Blood Leukocyte Count
Time frame: Baseline through Day 21/End of Study
Assessment of Blood Cysteamine Levels
Time frame: Day 14
Assessment of Sputum Cysteamine Levels
Time frame: Day 14
Change From Baseline in CFRSD-CRISS
Time frame: Baseline through to Day 21
Change From Baseline in CFQ-R
Time frame: Baseline through Day 21/End of Study
Change From Baseline in Jarad and Sequeiros Symptom Score Questionnaire
Time frame: changes from baseline at day 7 and day 14
Change From Baseline in Weight
Time frame: Baseline through Day 21/End of Study

Results and safety

Results posted to the registry on 14 Apr 2021, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Placebo	17	14
450mg, Once Per Day	11	7
150mg, Three Times Per Day	15	11
450mg, Twice Per Day	15	12
300mg, Three Times Per Day	16	11
450mg, Three Times Per Day	15	13

Primary outcome: Change From Baseline in Sputum Bacterial Load

Time frame: Baseline through Day 21/End of Study. Mean (Standard Deviation). Unit: log 10 cfu/ml

Measure	Placebo n = 17	450mg, Once Per Day n = 11	150mg, Three Times Per Day n = 15	450mg, Twice Per Day n = 15	300mg, Three Times Per Day n = 16	High Dose, Three Times Per Day n = 15
Day 7 change from baseline	-2.1 (2.887)	-1.03 (3.124)	-1.28 (1.978)	-1.7 (2.439)	-1.03 (1.997)	-0.25 (2.228)
Day 14 change from baseline	-1.38 (2.291)	0.12 (2.045)	-1.24 (2.686)	-1.32 (2.298)	-0.98 (1.894)	0.33 (2.270)
Day 21 change from baseline	-0.18 (1.238)	-1.22 (2.760)	-0.26 (1.639)	-1.04 (2.891)	-0.87 (1.677)	0.93 (2.348)

Population analysed: Intention to treat population

Primary outcome: Safety and Tolerability Assessed by the Number of Subjects With Adverse Events

Time frame: Baseline through Day 21/End of Study. Number. Unit: participants

Measure	Placebo n = 17	450mg, Once Per Day n = 11	150mg, Three Times Per Day n = 15	450mg, Twice Per Day n = 15	300mg, Three Times Per Day n = 16	450mg, Three Times Per Day n = 15
Number of AEs	30	29	40	33	45	36
Number of SAEs	1	1	2	1	0	1
1 or more AEs	9	10	11	12	10	13
Severity of AE mild	7	6	5	6	4	7
Severity of AE moderate	2	4	6	5	5	6
Severity of AE severe	0	0	0	1	1	0
AEs leading to drug discontinuation	1	1	1	1	1	1
Nausea	0	3	5	3	5	3

Population analysed: Summary of participant reported adverse events (AEs) by study group

12 secondary outcomes are reported in the full results.

Adverse events

Time frame: Adverse Events were collected from baseline (day 0, day study treatment commences) until day 21 (end of study/follow up). Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Placebo	1 of 17 (5.9%)	9 of 17 (52.9%)	0 of 17
High Dose, Once Per Day	1 of 11 (9.1%)	10 of 11 (90.9%)	0 of 11
Low Dose, Three Times Per Day	2 of 15 (13.3%)	11 of 15 (73.3%)	0 of 15
High Dose, Twice Per Day	1 of 15 (6.7%)	12 of 15 (80%)	0 of 15
Mid-Range Dose, Three Times Per Day	0 of 16 (0%)	10 of 16 (62.5%)	0 of 16
High Dose, Three Times Per Day	1 of 15 (6.7%)	13 of 15 (86.7%)	0 of 15

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 14 Apr 2021.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 33370348Devereux G, Wrolstad D, Bourke SJ, Daines CL, Doe S, Dougherty R, Franco R, Innes A, Kopp BT, Lascano J, Layish D, MacGregor G, Murray L, Peckham D, Lucidi V, Lovie E, Robertson J, Fraser-Pitt DJ, O'Neil DA. Oral cysteamine as an adjunct treatment in cystic fibrosis pulmonary exacerbations: An exploratory randomized clinical trial. PLoS One. 2020 Dec 28;15(12):e0242945. doi: 10.1371/journal.pone.0242945. eCollection 2020.
NovaBiotics WebsiteLink provided by the sponsor.

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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.

The record lists no contact. Use the official record to reach the sponsor.

17 sites in 3 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Cysteamine in people with Cystic Fibrosis, run by NovaBiotics Ltd. at 17 sites in 3 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 23 inclusion and 6 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 14 Apr 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 17 sites in United States, United Kingdom, Italy. 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 14 Apr 2021). 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?

Change From Baseline in Sputum Bacterial Load (time frame: Baseline through Day 21/End of Study). The study lists 2 primary outcomes in all. 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 NCT03000348, 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 14 Apr 2021.