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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06016088




Registration number
NCT06016088
Ethics application status
Date submitted
21/08/2023
Date registered
29/08/2023

Titles & IDs
Public title
A Double-Blind, Active-Controlled, Multiple-Ascending Dose Study of Aerosolized RSP-1502 in Subjects With CF and Chronic PA Lung Infection
Scientific title
A Double-Blind, Active-Controlled, Multiple-Ascending Dose, Phase 1b Study of Aerosolized RSP-1502 Delivered Via the PARI LC Plus® Nebulizer in Subjects With Cystic Fibrosis and Chronic Pseudomonas Aeruginosa Lung Infection
Secondary ID [1] 0 0
RESPIR-102
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Cystic Fibrosis Lung 0 0
Respiratory Infections, Recurrent, Chronic 0 0
Pseudomonas Aeruginosa 0 0
Condition category
Condition code
Infection 0 0 0 0
Studies of infection and infectious agents
Infection 0 0 0 0
Other infectious diseases
Infection 0 0 0 0
Sexually transmitted infections
Respiratory 0 0 0 0
Other respiratory disorders / diseases
Human Genetics and Inherited Disorders 0 0 0 0
Cystic fibrosis
Oral and Gastrointestinal 0 0 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
Inflammatory and Immune System 0 0 0 0
Connective tissue diseases
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - RSP-1502
Treatment: Drugs - Tobramycin inhalation solution

Experimental: 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.

Active comparator: Active Control - • Tobramycin Inhalation Solution 300 mg.


Treatment: Drugs: 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.

Treatment: Drugs: Tobramycin inhalation solution
Tobramycin inhalation solution is 300 mg tobramycin in 5 mL solution.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Treatment-emergent adverse events
Timepoint [1] 0 0
Day 1 through Day 28
Primary outcome [2] 0 0
Treatment-emergent serious adverse events
Timepoint [2] 0 0
Day 1 through Day 28
Primary outcome [3] 0 0
Changes in post-dose spirometry
Timepoint [3] 0 0
Day 1, Day 2, and Day 14
Primary outcome [4] 0 0
Pulmonary exacerbations
Timepoint [4] 0 0
Day 1 through Day 28
Primary outcome [5] 0 0
Changes in post-dose electrocardiogram results
Timepoint [5] 0 0
Day 1 and Day 2
Secondary outcome [1] 0 0
Pharmacokinetic parameters for CaEDTA
Timepoint [1] 0 0
Day 1
Secondary outcome [2] 0 0
Pharmacokinetic parameters for tobramycin
Timepoint [2] 0 0
Day 1

Eligibility
Key inclusion criteria
* Males or females aged =18 years of age.
* 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 year preceding screening.
* P. aeruginosa-positive sputum culture at screening.
* Forced expiratory volume in 1 second (FEV1) = 40 and = 90% 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.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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 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.
* 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 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.
* Smoking or vaping tobacco or any substance within 6 months prior to screening and anticipated inability to refrain from smoking throughout the study.
* Female subjects who are pregnant, lactating, or have a positive serum 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/04/2024
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/04/2025
Actual
Sample size
Target
52
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,SA,WA
Recruitment hospital [1] 0 0
The Alfred Hospital - Camperdown
Recruitment hospital [2] 0 0
Westmead Hospital - Westmead
Recruitment hospital [3] 0 0
Royal Adelaide Hospital - Adelaide
Recruitment hospital [4] 0 0
Lung Institute of Western Australia - Nedlands
Recruitment postcode(s) [1] 0 0
- Camperdown
Recruitment postcode(s) [2] 0 0
- Westmead
Recruitment postcode(s) [3] 0 0
- Adelaide
Recruitment postcode(s) [4] 0 0
- Nedlands
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Georgia
Country [4] 0 0
United States of America
State/province [4] 0 0
Louisiana
Country [5] 0 0
United States of America
State/province [5] 0 0
Minnesota
Country [6] 0 0
United States of America
State/province [6] 0 0
Missouri
Country [7] 0 0
United States of America
State/province [7] 0 0
New York
Country [8] 0 0
United States of America
State/province [8] 0 0
Ohio
Country [9] 0 0
United States of America
State/province [9] 0 0
Pennsylvania
Country [10] 0 0
United States of America
State/province [10] 0 0
Texas

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Respirion Pharmaceuticals Pty Ltd
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Brian Jones, PhD
Address 0 0
Country 0 0
Phone 0 0
215-732-5452
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Undecided
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT06016088