A Double-Blind, Active-Controlled, Multiple-Ascending Dose Study of Aerosolized RSP-1502 in Subjects With CF and Chronic PA Lung Infection

NCT ID: NCT06016088

Last Updated: 2025-12-29

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

72 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-04-01

Study Completion Date

2026-06-30

Brief Summary

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A double-blind, active-controlled, multiple-ascending dose, safety study of aerosolized RSP-1502 in subjects with cystic fibrosis Pseudomonas aeruginosa lung infection.

Detailed Description

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This dose escalation safety study will evaluate several doses of RSP-1502 or active control administered by inhalation for 14 days. Following determination of the MTD, a dose expansion cohort will receive RSP-1502 at the MTD versus active control administered by inhalation for 14 days. All subjects will be followed for 14 days after completion of dosing.

Conditions

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Cystic Fibrosis Lung Respiratory Infections, Recurrent, Chronic Pseudomonas Aeruginosa

Keywords

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Pseudomonas aeruginosa cystic fibrosis pulmonary infection tobramycin lung infection EDTA edetate calcium disodium

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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RSP-1502

Cohorts 1-4 will receive RSP-1502 (300 mg tobramycin plus an ascending dose of CaEDTA).

Cohort 5 will receive 300 mg tobramycin + CaEDTA at the MTD.

Group Type EXPERIMENTAL

RSP-1502

Intervention Type DRUG

RSP-1502 is a sterile, preservative free solution to be administered by inhalation via a nebulizer. Each dose of RSP-1502 contains the active components tobramycin (300 mg) and CaEDTA in a 5 mL solution.

Active Control

• Tobramycin Inhalation Solution 300 mg.

Group Type ACTIVE_COMPARATOR

Tobramycin inhalation solution

Intervention Type DRUG

Tobramycin inhalation solution is 300 mg tobramycin in 5 mL solution.

Interventions

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RSP-1502

RSP-1502 is a sterile, preservative free solution to be administered by inhalation via a nebulizer. Each dose of RSP-1502 contains the active components tobramycin (300 mg) and CaEDTA in a 5 mL solution.

Intervention Type DRUG

Tobramycin inhalation solution

Tobramycin inhalation solution is 300 mg tobramycin in 5 mL solution.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Males or females aged ≥18 years of age for cohorts 1-4; males or females ≥12 years of age for cohort 5.
* Diagnosis of CF based on the following: historical positive sweat chloride value ≥ 60 mEq/L, and/or genotype with two identifiable mutations consistent with CF, accompanied by one or more clinical features consistent with the CF phenotype.
* History of P. aeruginosa-positive sputum cultures or throat swabs with at least 50% positive in the 2 years preceding screening.
* P. aeruginosa-positive sputum culture at screening.
* Forced expiratory volume in 1 second (FEV1) ≥ 30 and ≤ 120% predicted per Global Lung Function Initiative (GLI) equation, pre- or post-bronchodilator.
* Must be able to withhold all other inhaled tobramycin from Day -28 to Day 28 of study participation. Must be able to withhold all other inhaled antibiotics from Day -14 to Day 28.
* Medically stable with no evidence of significant new or acute respiratory symptoms within 30 days prior to screening.
* Hematology, clinical chemistry, and urinalysis results with no clinically significant abnormalities that would interfere with the study assessments at screening as determined by the investigator.
* Female subjects of childbearing potential, defined as not surgically sterile or at least 2 years postmenopausal, must agree to use one of the following forms of contraception from screening through the Day 28 visit: hormonal (oral, implant, or injection) begun \> 30 days prior to screening, barrier (condom, diaphragm with spermicide), intrauterine device, or vasectomized partner (6 months minimum).
* Male subjects must show documentation of infertility or agree to use condoms during study participation.
* Must be able to communicate with site personnel and to understand and voluntarily sign the Informed Consent Form, and be capable and willing to complete all study visits and perform all study required procedures.

Exclusion Criteria

* A history of previous allergy or sensitivity to components of RSP 1502.
* A history of intolerance to inhaled tobramycin (TOBI®, BETHKIS®, TOBI® Podhaler®, tobramycin inhalation solution).
* eGFR \< 40 mL/min, or serum total bilirubin \> 2X or serum transaminases \> 3X the upper limit of normal range at screening.
* Currently taking other medications with known nephrotoxic, neurotoxic, or ototoxic potential (subjects receiving inhaled tobramycin in conjunction with low dose azithromycin prior to study participation without evidence of ototoxicity may continue taking low dose azithromycin during the study).
* Currently taking ethacrynic acid, furosemide, urea, or intravenous mannitol.
* Lung infection with organisms associated with a more rapid decline in pulmonary status (including, but not limited to, Burkholderia cenocepacia, Burkholderia dolosa, and Mycobacterium abscessus). For subjects who have had a history of a positive culture, the investigator will apply the following criteria to establish whether the subject is free of infection with such organisms:

1. The subject has not had a respiratory tract culture positive for these organisms within the 12 months before the date of informed consent.
2. The subject has had at least 2 respiratory tract cultures negative for such organisms within the 12 months before the date of informed consent, with the first and last of these separated by at least 3 months, and the most recent one within the 6 months before the date of informed consent.
* Consistent inability to produce sputum and unwillingness to perform sputum induction.
* Any acute upper or lower respiratory tract infection or pulmonary exacerbation requiring changes in therapy (including systemic antibiotics), or other significant clinical/laboratory/radiological/spirometric sign of unstable or unexpectedly deteriorating respiratory disease within 30 days prior to the first study drug administration.
* Initiation or adjustment of chronic airway medications (eg, inhaled corticosteroids; chronic suppressive antibacterial treatment) or airway clearance regimen (eg, nebulized saline, rhDNase, initiation of mechanical vest or handheld airway clearance device) within 28 days prior to screening. Individuals can be rescreened 28 days after these agents/therapies have been established for at least 28 days.
* Is immunocompromised due to illness, or solid or hematological organ transplant.
* Requires systemic prednisone (or equivalent) \> 10 mg daily.
* Vaping or smoking tobacco or any other substance within 1 month prior to screening and anticipated inability to refrain from vaping or smoking throughout the study.
* Female subjects who are pregnant, lactating, or have a positive urine human chorionic gonadotropin (pregnancy) test, as determined by laboratory testing.
* HIV positive.
* Active Hepatitis B or C.
* History of recreational drug or alcohol use/abuse which in the opinion of the investigator will compromise the patient's ability to comply with the study protocol.
* Participation in a clinical study with administration of an investigational drug product within the previous 30 days, or five half-lives of the previously administered investigational product.
* Has any other medical condition(s) which, in the opinion of the Principal Investigator, would jeopardize the safety of the study subject or impact the validity of the study results.
Minimum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Respirion Pharmaceuticals Pty Ltd

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Tucson Cystic Fibrosis Center

Tucson, Arizona, United States

Site Status RECRUITING

Center for Cystic Fibrosis at Keck Medical Center of USC

Los Angeles, California, United States

Site Status RECRUITING

Stanford University Medical Center

Palo Alto, California, United States

Site Status RECRUITING

Augusta University

Augusta, Georgia, United States

Site Status RECRUITING

The Cystic Fibrosis Institute

Northfield, Illinois, United States

Site Status RECRUITING

Tulane University

New Orleans, Louisiana, United States

Site Status RECRUITING

The Minnesota Cystic Fibrosis Center

Minneapolis, Minnesota, United States

Site Status RECRUITING

Washington University School of Medicine

St Louis, Missouri, United States

Site Status RECRUITING

Columbia University Cystic Fibrosis Program

New York, New York, United States

Site Status RECRUITING

Rainbow Babies and Children's Hospital / University Hospitals Cleveland Medical Center

Cleveland, Ohio, United States

Site Status RECRUITING

Nationwide Children's Hospital

Columbus, Ohio, United States

Site Status RECRUITING

University of Pennsylvania

Philadelphia, Pennsylvania, United States

Site Status RECRUITING

Dell Children's Medical Center of Central Texas

Austin, Texas, United States

Site Status RECRUITING

Royal Prince Albert Hospital

Camperdown, New South Wales, Australia

Site Status RECRUITING

Westmead Hospital

Westmead, New South Wales, Australia

Site Status RECRUITING

Queensland Children's Hospital

Brisbane, Queensland, Australia

Site Status RECRUITING

The Prince Charles Hospital

Brisbane, Queensland, Australia

Site Status RECRUITING

Royal Adelaide Hospital

Adelaide, South Australia, Australia

Site Status RECRUITING

The Alfred Hospital

Melbourne, Victoria, Australia

Site Status RECRUITING

The Royal Children's Hospital

Parkville, Victoria, Australia

Site Status RECRUITING

Lung Institute of Western Australia

Nedlands, Western Australia, Australia

Site Status RECRUITING

The Kids Research Institute Australia, Perth Children's Hospital

Perth, Western Australia, Australia

Site Status RECRUITING

Countries

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United States Australia

Central Contacts

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Brian Jones, PhD

Role: CONTACT

Phone: 215-732-5452

Email: [email protected]

Sarah Coquillette

Role: CONTACT

Email: [email protected]

Facility Contacts

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Elizabeth Ryan

Role: primary

Lynn Fukushima

Role: primary

Lani Demchak

Role: primary

Heidi Stapp

Role: primary

Karolina Roszko

Role: primary

Shae Williams

Role: primary

Mary Bailey

Role: primary

Taylor Haas

Role: primary

Cayla Boodram

Role: primary

Cindy Schaefer

Role: primary

Terri Johnson

Role: primary

Kishan Patel

Role: primary

Kristina Adrean

Role: primary

Simone Visser, Prof.

Role: primary

Tracey Burns

Role: primary

Claire Wainwright, Prof.

Role: primary

Iain Smith

Role: primary

Judith Morton, Prof.

Role: primary

Peter Wark, Prof.

Role: primary

Philip Robinson, Prof.

Role: primary

Elissa Kony

Role: backup

Siobhain Mulrennan, Prof.

Role: primary

David Hancock, Prof.

Role: primary

Other Identifiers

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RESPIR-102

Identifier Type: -

Identifier Source: org_study_id