Cystic FibrosisPhase 1/2NCT00558844

Safety/Tolerability Study of Arikayce™ in Cystic Fibrosis Patients With Chronic Infection Due to Pseudomonas Aeruginosa

A Phase 1/2 study testing Arikayce™ 560 mg, Arikayce™ 70 mg and Arikayce™ 140 mg in people with Cystic Fibrosis, run by Insmed Incorporated at 19 sites in United States.

Completed

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

Official record

Search all trials

Trial summary

This is a study to determine the safety and tolerability of 28 days of daily dosing of 560 mg of Arikayce™ versus placebo and daily dosing of 70 mg and 140 mg of Arikayce™ versus placebo in patients who have Cystic fibrosis (CF) and chronic infection due to pseudomonas aeruginosa. Read more about this study

Who is it for?: Ages 6 years and older
Conditions the study is for: Cystic Fibrosis
What is being tested?: Arikayce™ 560 mg, Arikayce™ 70 mg and Arikayce™ 140 mg
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 41 participants
Where: 19 sitesUnited States
Study dates: Jan 2008to Jun 2009
Phase: Phase 1/2
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 6 years and older
All sexes
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

Male or female study subjects must be adults (≥ 6 years of age)
Confirmed diagnosis of CF
History of chronic infection with P.aeruginosa
FEV1 ≥40% of predicted at Screening
Ability to comply with study medication use, study visits and procedures
Ability to produce 0.5 grams of sputum

Who cannot join

Administration of any investigational drug within 8 weeks to Study Day 1
Emergency room visit or hospitalization for CF or respiratory-related illness within 4 weeks prior to screening
History of alcohol, medication or illicit drug abuse within 1 yr. to screening
History of lung transplantation
Female of childbearing potential who are not practicing an acceptable method of birth control or who are lactating
Positive Pregnancy test

Show all 11Show fewer

Use of any anti-pseudomonal antibiotics within 28 days prior to Study Day 1
Initiation of chronic therapy within 28 days prior to Study Day 1
History of sputum or throat swab culture yielding Burkholderia cepacia within 2 years prior to screening
History of mycobacterial and/or Aspergillus infection requiring treatment within 2 years prior to screening
History of biliary cirrhosis with portal hypertension, or splenomegaly

Trial locations and contacts

19 sites in 1 country.

City, postcode or hospital

19 sites in 1 country

United States19 sites

Study site
Albuquerque, New Mexico
Study site
Ann Arbor, Michigan
Study site
Baltimore, Maryland
Study site
Birmingham, Alabama
Study site
Boston, Massachusetts
Study site
Indianapolis, Indiana
Study site
Iowa City, Iowa
Study site
Jackson, Mississippi
Study site
Los Angeles, California
Study site
Miami, Florida
Study site
Minneapolis, Minnesota
Study site
Morristown, New Jersey
Study site
New Brunswick, New Jersey
Study site
Orlando, Florida
Study site
Philadelphia, Pennsylvania
Study site
Rochester, New York
Study site
Seattle, Washington
Study site
Sioux Falls, South Dakota
Study site
St Louis, Missouri

Other trials in United States

About this study

Official title: Phase 1b/2a Multidose Safety and Tolerability Study of Liposomal Amikacin for Inhalation (Arikayce™) in Cystic Fibrosis Patient With Chronic Infections Due to Pseudomonas Aeruginosa.

Read the detailed description

CF is a gentic disease resulting from mutations in a 230 kb gene on chromosome 7 known as the cystic fibrosis transmembrane conductance regulator (CFTR). Study subjects with CF manifest pathological changes in a variety or organs that express CFTR. The lungs are frequently affected, the sequelae being chronic infections and airway inflammation. The principal goal of both treatment of subjects with CF is to slow the chronic deterioration of lung function.

Study subjects will be randomized to receive either study drug or placebo (1.5% NaCl) by inhalation via a PARI eFlow nebulizer. Each subject will complete 28 days of daily dosing. All study patients will be followed for safety, pharmacokinetics, clinical and microbiologic activity for 56 days post completion of study treatment. For the two lower doses (70 mg and 140 mg): patients received drug for 28 days, followed by a 28 day safety evaluation. For 560 mg: patients received drug for 28 days, followed by a 56 day safety evaluation. The total study period will be up to 84 days, with screening visit occurring within the preceding 14 days prior to study day 1. Patients will be clinically evaluated during the first 48 hours post first study dose and weekly for the 28 day treatment period and during the follow up visits at study days 35, 42, 49, 56, 70 and 85 days to determine safety and tolerability, pharmacokinetics (PK) and clinical and microbiologic activity.

Clinical laboratory parameters, audiology testing, clinical adverse events and pulmonary function will be evaluated for all study subjects in order to determine the qualitative and quantitative safety and tolerability of Arikayce™ compared to placebo. Serum, urine and sputum specimens will be collected at periodic intervals to assess PK. Additionally, sputum samples will be collected to determine changes in bacterial density. Pulmonary function testing and CFQ-R measurements will be assessed at selected time points throughout the study. An exploratory evaluation of a Cystic Fibrosis Symptom Diary (CFSD) will also be implemented. Arikace™,Arikayce™, Liposomal Amikacin for Inhalation (LAI), and Amikacin Liposome Inhalation Suspension (ALIS) may be used interchangeably throughout this study and other studies evaluating amikacin liposomal inhalation suspension.

Conditions: Cystic Fibrosis
Being tested: Arikayce™ 560 mg, Placebo for 560 mg, Arikayce™ 70 mg, Arikayce™ 140 mg, Placebo for 70 mg / 140 mg
Keywords: Cystic Fibrosis, Respiratory Infections, Pulmonary Cystic Fibrosis, CFTR
Medical subject headings: Cystic Fibrosis, Respiratory Tract Infections

What participants receive

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

Arikayce™ 560 mgDrug
Arikayce™ at 560 mg
Subjects will be randomly assigned to study drug dose of of Arikayce™ or placebo in accordance with a code provided by the Sponsor/CRO. Randomization will be made in a 2:1 allocation between Arikayce™ and placebo. They will be blinded whether they receive Arikayce™ or Placebo Study subjects will receive Arikayce™ or placebo on Days 1 through Day 28. Drug is administered once a day via a nebulizer.
Placebo for 560 mgDrug
Matching placebo
Subjects will be randomly assigned to study drug dose of of Arikayce™ or placebo in accordance with a code provided by the Sponsor/CRO. Randomization will be made in a 2:1 allocation between Arikayce™ and placebo. They will be blinded whether they receive Arikayce™ or Placebo Study subjects will receive Arikayce™ or placebo on Days 1 through Day 28. Drug is administered once a day via a nebulizer.
Arikayce™ 70 mgDrug
Subjects will be randomly assigned to study drug dose of of Arikayce™ or placebo in accordance with a code provided by the Sponsor/CRO. Randomization will be made in a 1:1:1 allocation between Arikayce™ and placebo. They will be blinded whether they receive Arikayce™ or Placebo Study subjects will receive Arikayce™ or placebo on Days 1 through Day 28. Drug is administered once a day via a nebulizer.
Arikayce™ 140 mgDrug
Subjects will be randomly assigned to study drug dose of of Arikayce™ or placebo in accordance with a code provided by the Sponsor/CRO. Randomization will be made in a 1:1:1 allocation between Arikayce™ and placebo. They will be blinded whether they receive Arikayce™ or Placebo Study subjects will receive Arikayce™ or placebo on Days 1 through Day 28. Drug is administered once a day via a nebulizer.
Placebo for 70 mg / 140 mgDrug
Matching placebo
Subjects will be randomly assigned to study drug dose of of Arikayce™ or placebo in accordance with a code provided by the Sponsor/CRO. Randomization will be made in a 1:1:1 allocation between Arikayce™ and placebo. They will be blinded whether they receive Arikayce™ or Placebo Study subjects will receive Arikayce™ or placebo on Days 1 through Day 28. Drug is administered once a day via a nebulizer.

Study groups in NCT00558844
Study group	Role	What they receive
A	Active comparator	Arikayce™ at 560 mg Subjects randomized 2:1 to receive Arikayce 560 mg or Placebo. Receives: Drug: Arikayce™ 560 mg
B	Placebo comparator	Matching placebo for 560 mg Subjects randomized 2:1 to receive Arikayce 560 mg or Placebo. Receives: Drug: Placebo for 560 mg
C	Active comparator	Arikayce™ at 70 mg Subjects randomized 1:1:1 to receive Arikayce 70 mg, Arikayce 140 mg or Placebo. Receives: Drug: Arikayce™ 70 mg
D	Active comparator	Arikayce™ at 140 mg Subjects randomized 1:1:1 to receive Arikayce 70 mg, Arikayce 140 mg or Placebo. Receives: Drug: Arikayce™ 140 mg
E	Placebo comparator	Matching placebo for 70 mg/140 mg Subjects randomized 1:1:1 to receive Arikayce 70 mg, Arikayce 140 mg or Placebo. Receives: Drug: Placebo for 70 mg / 140 mg

What is being measured

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

Number of Participants With Treatment-Emergent Adverse Events
To evaluate the safety and tolerability of 28 days of daily dosing of nebulized Arikayce™, liposomal amikacin for inhalation.
Measured: 56 days

Secondary outcomes (8)

Pharmacokinetics of Arikayce™ in Serum
Measure PK parameter (Cmax) of Arikayce in serum
Measured: Day 1, Day 14 and Day 28
Pharmacokinetics (PK) of Arikayce™ in Sputum
Measure PK parameters (sputum concentration) of Arikayce in sputum, pre- and post-dose
Measured: Day 1 post-dose, Day 14 pre- and post-dose, Day 28 pre- and post-dose
Pharmacokinetics (PK) of Arikayce™ in Urine
Measure PK parameter (Ae0-24) of Arikayce in urine
Measured: Day 1, Day 14 and Day 28
Pharmacokinetics (PK) of Arikayce™ in Serum
Measure PK parameter (AUC) of Arikayce in Serum
Measured: Day 1, Day 14 and Day 28
Pulmonary Function: Pre-Dose FEV1 (%-Predicted)
Relative Change (%) from Baseline to Day 28, Day 56, Day 70, and Day 84 in Pulmonary Function
Measured: Baseline, Day 28, Day 56, Day 70 and Day 84
Density of Pseudomonas Aeruginosa in Sputum
Change (log10 CFU) from Baseline by Study Day and Treatment Arm
Measured: Day 7, Day 14, Day 21, Day 28 and Day 35
Duration of Systemic Anti-Pseudomonal Rescue Therapy
Measured: Through study duration, approximately 84 days
CFQ-R Respiratory Scale (Relative Change % From Baseline)
Quality of Life was measured by the absolute change from baseline in the Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory scale. Disease specific instrument designed to measure impact on overall health, daily life, perceived well-being and symptoms in patients with a diagnosis of cystic fibrosis. Scores range from 0 to 100, with higher scores indicating better health. Scores for each Health Related Quality of Life (HRQoL) domain; after recoding, each item is summed to generate a domain score and standardized.
Measured: Day 15, Day 28 and Day 42

Timeline

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

Jan 2008Jun 2009

First posted15 Nov 2007 (estimated)
Study startJan 2008
Primary completionJun 2009
Study completionJun 2009

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 (2)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 1/2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind
Who is masked: Participant, Care provider, Investigator, Outcomes assessor
Enrolment: 41 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00558844
Sponsor's protocol ID: TR02-106
Status last verified: May 2019
First submitted: 13 Nov 2007
Last updated: 4 Jun 2019
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes

Publications

Okusanya OO, Bhavnani SM, Hammel JP, Forrest A, Bulik CC, Ambrose PG, Gupta R. Evaluation of the pharmacokinetics and pharmacodynamics of liposomal amikacin for inhalation in cystic fibrosis patients with chronic pseudomonal infections using data from two phase 2 clinical studies. Antimicrob Agents Chemother. 2014 Sep;58(9):5005-15. doi: 10.1128/AAC.02421-13. Epub 2014 Mar 31.
PubMed 24687506
Clancy JP, Dupont L, Konstan MW, Billings J, Fustik S, Goss CH, Lymp J, Minic P, Quittner AL, Rubenstein RC, Young KR, Saiman L, Burns JL, Govan JR, Ramsey B, Gupta R; Arikace Study Group. Phase II studies of nebulised Arikace in CF patients with Pseudomonas aeruginosa infection. Thorax. 2013 Sep;68(9):818-25. doi: 10.1136/thoraxjnl-2012-202230. Epub 2013 Jun 8.
PubMed 23749840

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 4 Jun 2019. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Phase 1b/2a Multidose Safety and Tolerability Study of Liposomal Amikacin for Inhalation (Arikayce™) in Cystic Fibrosis Patient With Chronic Infections Due to Pseudomonas Aeruginosa.
ClinicalTrials.gov ID: NCT00558844
Acronym / sponsor ID: TR02-106
Conditions: Cystic Fibrosis
Keywords: Cystic Fibrosis, Respiratory Infections, Pulmonary Cystic Fibrosis, CFTR
Phase: Phase 1/2
Study type: Interventional
Recruitment status: Completed
Enrolment: 41 participants (actual)
Lead sponsor: Insmed IncorporatedIndustry (pharma, biotech, devices)
Study start: Jan 2008
Primary completion: Jun 2009 (actual)
Study completion: Jun 2009 (actual)
First posted: 15 Nov 2007 (estimated)
Results: Posted 4 Jun 2019

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: 5

Arm	Type	Receives
A	Active comparator	Drug: Arikayce™ 560 mgArikayce™ at 560 mg Subjects randomized 2:1 to receive Arikayce 560 mg or Placebo.
B	Placebo comparator	Drug: Placebo for 560 mgMatching placebo for 560 mg Subjects randomized 2:1 to receive Arikayce 560 mg or Placebo.
C	Active comparator	Drug: Arikayce™ 70 mgArikayce™ at 70 mg Subjects randomized 1:1:1 to receive Arikayce 70 mg, Arikayce 140 mg or Placebo.
D	Active comparator	Drug: Arikayce™ 140 mgArikayce™ at 140 mg Subjects randomized 1:1:1 to receive Arikayce 70 mg, Arikayce 140 mg or Placebo.
E	Placebo comparator	Drug: Placebo for 70 mg / 140 mgMatching placebo for 70 mg/140 mg Subjects randomized 1:1:1 to receive Arikayce 70 mg, Arikayce 140 mg or Placebo.

Who may qualify

Ages: 6 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (6 listed)

Male or female study subjects must be adults (≥ 6 years of age)
Confirmed diagnosis of CF
History of chronic infection with P.aeruginosa
FEV1 ≥40% of predicted at Screening
Ability to comply with study medication use, study visits and procedures

Key exclusion criteria (11 listed)

Administration of any investigational drug within 8 weeks to Study Day 1
Emergency room visit or hospitalization for CF or respiratory-related illness within 4 weeks prior to screening
History of alcohol, medication or illicit drug abuse within 1 yr. to screening
History of lung transplantation
Female of childbearing potential who are not practicing an acceptable method of birth control or who are lactating

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
Arikayce™ 560 mgArikayce™ at 560 mg Subjects will be randomly assigned to study drug dose of of Arikayce™ or placebo in accordance with a code provided by the Sponsor/CRO. Randomization will be made in a 2:1 allocation between Arikayce™ and placebo. They will be blinded whether they receive Arikayce™ or Placebo Study subjects will receive Arikayce™ or placebo on Days 1 through Day 28. Drug is administered once a day via a nebulizer.	Drug	A
Placebo for 560 mgMatching placebo Subjects will be randomly assigned to study drug dose of of Arikayce™ or placebo in accordance with a code provided by the Sponsor/CRO. Randomization will be made in a 2:1 allocation between Arikayce™ and placebo. They will be blinded whether they receive Arikayce™ or Placebo Study subjects will receive Arikayce™ or placebo on Days 1 through Day 28. Drug is administered once a day via a nebulizer.	Drug	B
Arikayce™ 70 mgSubjects will be randomly assigned to study drug dose of of Arikayce™ or placebo in accordance with a code provided by the Sponsor/CRO. Randomization will be made in a 1:1:1 allocation between Arikayce™ and placebo. They will be blinded whether they receive Arikayce™ or Placebo Study subjects will receive Arikayce™ or placebo on Days 1 through Day 28. Drug is administered once a day via a nebulizer.	Drug	C
Arikayce™ 140 mgSubjects will be randomly assigned to study drug dose of of Arikayce™ or placebo in accordance with a code provided by the Sponsor/CRO. Randomization will be made in a 1:1:1 allocation between Arikayce™ and placebo. They will be blinded whether they receive Arikayce™ or Placebo Study subjects will receive Arikayce™ or placebo on Days 1 through Day 28. Drug is administered once a day via a nebulizer.	Drug	D
Placebo for 70 mg / 140 mgMatching placebo Subjects will be randomly assigned to study drug dose of of Arikayce™ or placebo in accordance with a code provided by the Sponsor/CRO. Randomization will be made in a 1:1:1 allocation between Arikayce™ and placebo. They will be blinded whether they receive Arikayce™ or Placebo Study subjects will receive Arikayce™ or placebo on Days 1 through Day 28. Drug is administered once a day via a nebulizer.	Drug	E

Primary outcome

Number of Participants With Treatment-Emergent Adverse Events
Time frame: 56 days
To evaluate the safety and tolerability of 28 days of daily dosing of nebulized Arikayce™, liposomal amikacin for inhalation.

8 secondary outcomes

Pharmacokinetics of Arikayce™ in Serum
Time frame: Day 1, Day 14 and Day 28
Pharmacokinetics (PK) of Arikayce™ in Sputum
Time frame: Day 1 post-dose, Day 14 pre- and post-dose, Day 28 pre- and post-dose
Pharmacokinetics (PK) of Arikayce™ in Urine
Time frame: Day 1, Day 14 and Day 28
Pharmacokinetics (PK) of Arikayce™ in Serum
Time frame: Day 1, Day 14 and Day 28
Pulmonary Function: Pre-Dose FEV1 (%-Predicted)
Time frame: Baseline, Day 28, Day 56, Day 70 and Day 84
Density of Pseudomonas Aeruginosa in Sputum
Time frame: Day 7, Day 14, Day 21, Day 28 and Day 35
Duration of Systemic Anti-Pseudomonal Rescue Therapy
Time frame: Through study duration, approximately 84 days
CFQ-R Respiratory Scale (Relative Change % From Baseline)
Time frame: Day 15, Day 28 and Day 42

Results and safety

Results posted to the registry on 4 Jun 2019, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Arikayce™ at 560 mg	15	12
Placebo at 560 mg	7	7
Arikayce™ at 70 mg	7	6
Arikayce™ at 140 mg	5	5
Placebo at 70mg/140 mg	7	6

Primary outcome: Number of Participants With Treatment-Emergent Adverse Events

Time frame: 56 days. Count of participants. Unit: Participants

Measure	Arikayce™ at 560 mg n = 15	Placebo at 560 mg n = 7	Arikayce™ at 70 mg n = 7	Arikayce™ at 140 mg n = 5	Placebo at 70mg/140 mg n = 7
Patients with AEs	14	7	7	4	6
Patients with Treatment Related AEs	7	2	2	2	2
Deaths	0	0	0	0	0
Patients with Serious AEs	5	2	0	1	1
Patients Permanently Discontinuing due to AEs	4	0	0	0	0

Population analysed: Analyses were performed using the modified intent-to-treat (mITT) population, defined as all randomized subjects who received at least one dose of study drug

8 secondary outcomes are reported in the full results.

Adverse events

Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Arikayce™ at 560 mg	5 of 15 (33.3%)	14 of 15 (93.3%)	0 of 15
Placebo at 560 mg	2 of 7 (28.6%)	7 of 7 (100%)	0 of 7
Arikayce™ at 70 mg	0 of 7 (0%)	7 of 7 (100%)	0 of 7
Arikayce™ at 140 mg	1 of 5 (20%)	4 of 5 (80%)	0 of 5
Placebo at 70 mg/140 mg	1 of 7 (14.3%)	6 of 7 (85.7%)	0 of 7

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 4 Jun 2019.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Background: PubMed 24687506Okusanya OO, Bhavnani SM, Hammel JP, Forrest A, Bulik CC, Ambrose PG, Gupta R. Evaluation of the pharmacokinetics and pharmacodynamics of liposomal amikacin for inhalation in cystic fibrosis patients with chronic pseudomonal infections using data from two phase 2 clinical studies. Antimicrob Agents Chemother. 2014 Sep;58(9):5005-15. doi: 10.1128/AAC.02421-13. Epub 2014 Mar 31.
Derived from this study: PubMed 23749840Clancy JP, Dupont L, Konstan MW, Billings J, Fustik S, Goss CH, Lymp J, Minic P, Quittner AL, Rubenstein RC, Young KR, Saiman L, Burns JL, Govan JR, Ramsey B, Gupta R; Arikace Study Group. Phase II studies of nebulised Arikace in CF patients with Pseudomonas aeruginosa infection. Thorax. 2013 Sep;68(9):818-25. doi: 10.1136/thoraxjnl-2012-202230. Epub 2013 Jun 8.
Related InfoLink 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.

Gina Eagle, MD study director

Insmed Incorporated

19 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Arikayce™ 560 mg, Arikayce™ 70 mg and Arikayce™ 140 mg in people with Cystic Fibrosis, run by Insmed Incorporated at 19 sites in United States.

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 6 inclusion and 11 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 4 Jun 2019. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 19 sites in United States. 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 combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

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 4 Jun 2019). 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 Treatment-Emergent Adverse Events (time frame: 56 days). 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 NCT00558844, which also lists 2 linked publications.

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 4 Jun 2019.