Single Dose Escalation Study of TR02 (Sustained Lipid Inhalation Technology [SLIT™] Amikacin) in Participants With Cystic Fibrosis (CF) Having Chronic Infections of Pseudomonas Aeruginosa

A Phase 1 study testing SLIT™ Amikacin in people with Cystic Fibrosis, run by Insmed Incorporated.

Completed

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

Trial summary

The primary purpose of this study is to evaluate the safety and tolerability of three active doses of nebulized amikacin in a SLIT™ formulation. 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?: SLIT™ Amikacin
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 18 participants
Where: No sites listed yet
Study dates: 12 May 2004to 8 Feb 2005
Phase: Phase 1
Are there results?: None posted in the registry

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

Study participants must produce sputum that is positive for Pseudomonas aeruginosa.
Confirmed diagnosis of CF (positive sweat chloride >60 milliequivalents (mEq)/liter (by pilocarpine iontophoresis) and/or a genotype with two identifiable mutations consistent with CF accompanied by one or more clinical features with the CF phenotype.
Forced expiratory volume (FEV1) ≥40% predicted at Screening as calculated by the Knudsen reference equations.
Clinically stable with no evidence of current pulmonary exacerbation.

Who cannot join

History of lung transplantation.
Use of intravenous antibiotics or oral quinolones within 14 days of Screening.
Use of low dose oral antibiotics (e.g. tetracycline, sulfa) for acne or other conditions within 30 days of Screening.
Use of systemic corticosteroids (≥20 milligrams [mg] of prednisone per day) within 30 days of Screening.
Initiation of TOBI® (tobramycin), high dose ibuprofen, recombinant human DNase (rhDNase), or macrolide antibiotics within 60 days of Screening.
History of sputum or throat swab culture yielding Burkholderia cepacia complex within 2 years of Screening or growth of Burkholderia cepacia complex from the sputum or throat swab culture obtained at Screening.

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History of biliary cirrhosis, portal hypertension, or splenomegaly or splenomegaly on physical exam at Screening or enrollment.
History of daily continuous oxygen supplementation or requirement for more than 2 liter per minute (L/min) at night.
Note: Other inclusion/exclusion criteria may apply.

About this study

Official title: Safety and Tolerability Study of Single Dose Escalations of TR02 (SLIT™ Amikacin) by Inhalation in Cystic Fibrosis Study Patients With Chronic Infections of Pseudomonas Aeruginosa

The primary purpose of this study is to evaluate the safety and tolerability of three active doses of nebulized amikacin in a SLIT™ formulation.

Conditions: Cystic Fibrosis
Being tested: SLIT™ Amikacin, Placebo
Medical subject headings: Cystic Fibrosis

What participants receive

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

SLIT™ AmikacinDrug
Also called TR02
Amikacin administered via the Pari LC STAR™ nebulizer.
PlaceboDrug
Nebulized saline.

Study groups in NCT06238856
Study group	Role	What they receive
Cohort 1: Amikacin Dose 1 + Placebo	Experimental	Participants will receive a single dose of SLIT™ amikacin at Dose 1 or matching placebo by inhalation on Day 0. Receives: Drug: SLIT™ Amikacin; Drug: Placebo
Cohort 2: Amikacin Dose 2 + Placebo	Experimental	Participants will receive a single dose of SLIT™ amikacin at Dose 2 or matching placebo by inhalation on Day 0 upon initiation of Cohort 2. Receives: Drug: SLIT™ Amikacin; Drug: Placebo
Cohort 3: Amikacin Dose 3 + Placebo	Experimental	Participants will receive a single dose of SLIT™ amikacin at Dose 3 or matching placebo by inhalation on Day 0 upon initiation of Cohort 3. Receives: Drug: SLIT™ Amikacin; Drug: Placebo

What is being measured

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

Number of Participants who Experience a Treatment Emergent Adverse Event (TEAE)
Safety and tolerability of three active doses of nebulized amikacin in a SLIT™ formulation.
Measured: Up to Day 28

Secondary outcomes (4)

Area Under the Concentration-time Curve (AUC) of SLIT™ Amikacin in Serum
Measured: Pre-dose and at multiple time points post-dose up to Day 3
Percent Dose of SLIT™ Amikacin in Urine
Measured: At multiple time points post-dose up to Day 3
AUC of SLIT™ Amikacin in Sputum
Measured: Pre-dose and at multiple time points post-dose on Days 1, 2, 3, 8, 14, and 28
Change From Baseline in Sputum Density of Pseudomonas Aeruginosa
Measured: Baseline up to Day 28

Timeline

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

12 May 20048 Feb 2005

First posted2 Feb 2024
Study start12 May 2004
Primary completion8 Feb 2005
Study completion8 Feb 2005

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
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
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Sequential
Masking: Quadruple blind
Who is masked: Participant, Care provider, Investigator, Outcomes assessor
Enrolment: 18 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT06238856
Sponsor's protocol ID: TR02-101
Status last verified: Jan 2024
First submitted: 25 Jan 2024
Last updated: 2 Feb 2024
Results posted: No
Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No

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?

Official record

RecruitingPhase 2
A Study of 24 to 52 Weeks Treatment to Evaluate The Efficacy And Safety of Galvokimig in Adult Study Participants With Non-Cystic Fibrosis Bronchiectasis
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CFTR Modulator Effects on Bone and Muscle in Adults With Cystic Fibrosis
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Role of Genetic Factors in the Development of Lung Disease
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Early Study on Tamoxifen Safety/Tolerability in Cystic Fibrosis Patients Unable to Use CFTR Modulators.
Cystic Fibrosis - Complete

All Cystic Fibrosis trials

Study at a glance

From the registry record, last updated by the sponsor on 2 Feb 2024. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Safety and Tolerability Study of Single Dose Escalations of TR02 (SLIT™ Amikacin) by Inhalation in Cystic Fibrosis Study Patients With Chronic Infections of Pseudomonas Aeruginosa
ClinicalTrials.gov ID: NCT06238856
Acronym / sponsor ID: TR02-101
Conditions: Cystic Fibrosis
Phase: Phase 1
Study type: Interventional
Recruitment status: Completed
Enrolment: 18 participants (actual)
Lead sponsor: Insmed IncorporatedIndustry (pharma, biotech, devices)
Study start: 12 May 2004 (actual)
Primary completion: 8 Feb 2005 (actual)
Study completion: 8 Feb 2005 (actual)
First posted: 2 Feb 2024 (actual)
Results: Not posted in the source record

Clinical summary

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

The primary purpose of this study is to evaluate the safety and tolerability of three active doses of nebulized amikacin in a SLIT™ formulation.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Sequential
Masking: Quadruple blind (participant, care provider, investigator, outcomes assessor)
Number of arms: 3

Arm	Type	Receives
Cohort 1: Amikacin Dose 1 + Placebo	Experimental	Drug: SLIT™ Amikacin, Drug: PlaceboParticipants will receive a single dose of SLIT™ amikacin at Dose 1 or matching placebo by inhalation on Day 0.
Cohort 2: Amikacin Dose 2 + Placebo	Experimental	Drug: SLIT™ Amikacin, Drug: PlaceboParticipants will receive a single dose of SLIT™ amikacin at Dose 2 or matching placebo by inhalation on Day 0 upon initiation of Cohort 2.
Cohort 3: Amikacin Dose 3 + Placebo	Experimental	Drug: SLIT™ Amikacin, Drug: PlaceboParticipants will receive a single dose of SLIT™ amikacin at Dose 3 or matching placebo by inhalation on Day 0 upon initiation of Cohort 3.

Who may qualify

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

Key inclusion criteria (4 listed)

Study participants must produce sputum that is positive for Pseudomonas aeruginosa.
Confirmed diagnosis of CF (positive sweat chloride >60 milliequivalents (mEq)/liter (by pilocarpine iontophoresis) and/or a genotype with two identifiable mutations consistent with CF accompanied by one or more clinical features with the CF phenotype.
Forced expiratory volume (FEV1) ≥40% predicted at Screening as calculated by the Knudsen reference equations.
Clinically stable with no evidence of current pulmonary exacerbation.

Key exclusion criteria (9 listed)

History of lung transplantation.
Use of intravenous antibiotics or oral quinolones within 14 days of Screening.
Use of low dose oral antibiotics (e.g. tetracycline, sulfa) for acne or other conditions within 30 days of Screening.
Use of systemic corticosteroids (≥20 milligrams [mg] of prednisone per day) within 30 days of Screening.
Initiation of TOBI® (tobramycin), high dose ibuprofen, recombinant human DNase (rhDNase), or macrolide antibiotics within 60 days of Screening.

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
SLIT™ AmikacinAlso: TR02Amikacin administered via the Pari LC STAR™ nebulizer.	Drug	Cohort 1: Amikacin Dose 1 + Placebo, Cohort 2: Amikacin Dose 2 + Placebo, Cohort 3: Amikacin Dose 3 + Placebo
PlaceboNebulized saline.	Drug	Cohort 1: Amikacin Dose 1 + Placebo, Cohort 2: Amikacin Dose 2 + Placebo, Cohort 3: Amikacin Dose 3 + Placebo

Primary outcome

Number of Participants who Experience a Treatment Emergent Adverse Event (TEAE)
Time frame: Up to Day 28
Safety and tolerability of three active doses of nebulized amikacin in a SLIT™ formulation.

4 secondary outcomes

Area Under the Concentration-time Curve (AUC) of SLIT™ Amikacin in Serum
Time frame: Pre-dose and at multiple time points post-dose up to Day 3
Percent Dose of SLIT™ Amikacin in Urine
Time frame: At multiple time points post-dose up to Day 3
AUC of SLIT™ Amikacin in Sputum
Time frame: Pre-dose and at multiple time points post-dose on Days 1, 2, 3, 8, 14, and 28
Change From Baseline in Sputum Density of Pseudomonas Aeruginosa
Time frame: Baseline up to Day 28

Results and safety

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 2 Feb 2024.
Individual participant dataSharing planned: No

Related trials for Cystic Fibrosis

RecruitingPhase 2
A Study of 24 to 52 Weeks Treatment to Evaluate The Efficacy And Safety of Galvokimig in Adult Study Participants With Non-Cystic Fibrosis Bronchiectasis
Non-cystic Fibrosis Bronchiectasis
Active, not recruitingObservational study
CFTR Modulator Effects on Bone and Muscle in Adults With Cystic Fibrosis
Cystic Fibrosis, Bone Loss
RecruitingObservational study
Natural History of Bronchiectasis
Bronchiectasis, Cystic Fibrosis
RecruitingObservational study
Role of Genetic Factors in the Development of Lung Disease
Cystic Fibrosis, Pulmonary Fibrosis
RecruitingPhase 2
Early Study on Tamoxifen Safety/Tolerability in Cystic Fibrosis Patients Unable to Use CFTR Modulators.
Cystic Fibrosis - Complete

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.

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing SLIT™ Amikacin in people with Cystic Fibrosis, run by Insmed Incorporated.

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 4 inclusion and 9 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 2 Feb 2024. Status can change between updates, so confirm with the study contact before making plans.

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 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.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

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 who Experience a Treatment Emergent Adverse Event (TEAE) (time frame: Up to Day 28). 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 NCT06238856.

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 2 Feb 2024.