RSP-1502 Inhalation for Cystic Fibrosis
Recruiting in Palo Alto (17 mi)
+18 other locations
Age: Any Age
Sex: Any
Travel: May Be Covered
Time Reimbursement: Varies
Trial Phase: Phase 1 & 2
Recruiting
Sponsor: Respirion Pharmaceuticals Pty Ltd
Must not be taking: Nephrotoxic, Neurotoxic, Ototoxic, others
Disqualifiers: HIV, Active Hepatitis B/C, others
No Placebo Group
Trial Summary
What is the purpose of this trial?A double-blind, active-controlled, multiple-ascending dose, safety study of aerosolized RSP-1502 in subjects with cystic fibrosis Pseudomonas aeruginosa lung infection.
Will I have to stop taking my current medications?
You may need to stop taking certain medications. Specifically, you must stop all inhaled tobramycin from 28 days before the study until 28 days into the study, and all other inhaled antibiotics from 14 days before the study until 28 days into the study. If you are taking medications with known nephrotoxic, neurotoxic, or ototoxic potential, you may need to stop those as well, unless you are on low dose azithromycin with inhaled tobramycin and have no evidence of ototoxicity.
What evidence supports the effectiveness of the drug RSP-1502 for cystic fibrosis?
Research shows that inhaled tobramycin, a component of RSP-1502, significantly improves lung function and reduces infection in cystic fibrosis patients. It also lowers the need for hospital visits and additional antibiotics, enhancing patients' quality of life.
12345Is RSP-1502 inhalation safe for humans?
Tobramycin inhalation solution, also known as TOBI, has been used safely in cystic fibrosis patients, showing a safety profile comparable to other forms of the drug. Clinical studies indicate it is generally safe for use in humans, with a reduced mortality rate in cystic fibrosis patients compared to those not using it.
13467How is the drug RSP-1502 different from other cystic fibrosis treatments?
RSP-1502 is unique because it is an inhalation solution of tobramycin, which is specifically designed to treat lung infections in cystic fibrosis patients by delivering a high dose directly to the lungs, reducing systemic side effects. This approach can improve patient adherence due to its convenient administration and has shown to significantly improve lung function and reduce mortality compared to placebo.
13568Eligibility Criteria
Adults over 18 with cystic fibrosis and chronic lung infection caused by P. aeruginosa, who haven't had significant respiratory symptoms or changes in their CF treatments for the past month. They must be able to avoid other inhaled antibiotics during the study and have a stable organ function as determined by tests. Women of childbearing age should use contraception, men should either be infertile or agree to use condoms.Inclusion Criteria
I can stop using inhaled tobramycin from Day 28 to Day 28 of the study.
I am a male and will use condoms or am infertile.
Your blood, urine, and other test results must be normal and not interfere with the study assessments.
+9 more
Exclusion Criteria
You have tested positive for COVID-19 using a rapid test at the screening or on the first day of the trial.
I am currently on medication that could harm my kidneys, nerves, or hearing.
I do not currently have a lung infection with rapidly worsening bacteria.
+15 more
Trial Timeline
Screening
Participants are screened for eligibility to participate in the trial
2-4 weeks
Treatment
Participants receive aerosolized RSP-1502 or active control via inhalation for 14 days
2 weeks
Daily visits for dosing
Follow-up
Participants are monitored for safety and effectiveness after treatment
2 weeks
1-2 visits (in-person)
Participant Groups
The trial is testing RSP-1502, an aerosolized drug combined with different doses of CaEDTA compared to Tobramycin inhalation solution. Participants will inhale their assigned treatment twice daily for two weeks while being monitored for safety and effectiveness. The best dose will be chosen based on tolerance levels observed across groups.
2Treatment groups
Experimental Treatment
Active Control
Group I: RSP-1502Experimental Treatment1 Intervention
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 II: Active ControlActive Control1 Intervention
• Tobramycin Inhalation Solution 300 mg.
Find a Clinic Near You
Research Locations NearbySelect from list below to view details:
Dell Children's Medical Center of Central TexasAustin, TX
Columbia University Cystic Fibrosis ProgramNew York, NY
Stanford University Medical CenterPalo Alto, CA
Washington University School of MedicineSaint Louis, MO
More Trial Locations
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Who Is Running the Clinical Trial?
Respirion Pharmaceuticals Pty LtdLead Sponsor
References
Mortality of cystic fibrosis patients treated with tobramycin solution for inhalation. [2019]Tobramycin solution for inhalation (TOBI; TSI) is indicated to treat patients with cystic fibrosis who are infected with Pseudomonas aeruginosa. Preliminary findings from a randomized trial indicate that patients who received TSI had about half the mortality rate of those assigned to placebo.
Tobramycin for inhalation in cystic fibrosis: Beyond respiratory improvements. [2013]European consensus guidelines recommend nebulised antibiotics for maintenance therapy in patients with cystic fibrosis and chronic Pseudomonas aeruginosa infection. Two formulations of tobramycin for inhalation are available in Europe (Tobi; Novartis AG, Switzerland; Bramitob; Chiesi Farmaceutici S.p.A., Italy). Data from a series of randomised controlled studies in patients with mild-to-moderate cystic fibrosis and chronic P. aeruginosa infection show that both Tobi and Bramitob significantly improve lung function and reduce the density of P. aeruginosa in sputum. Bacterial resistance may develop but does not seem to be clinically important. Other benefits, such as improved patient nutritional status and reductions in the need for hospitalisation, antipseudomonal antibiotics and productivity losses have also been documented with Tobi and Bramitob. Both formulations of inhaled tobramycin are well tolerated with no evidence of renal or ototoxicity. Improved patient compliance may be achieved through reducing nebulisation time, either by using Bramitob, which is formulated in a smaller volume than Tobi, or with new generation nebulisers. In conclusion, inhaled tobramycin not only improves lung function in patients with cystic fibrosis, but also offers other benefits which have implications for healthcare costs and patient quality of life.
[TOBI Podhaler for treating chronic Pseudomonas aeruginosa infection in cystic fibrosis patients]. [2020]TOBI Podhaler is the first dry powder formulation of tobramycin for inhaled therapy of chronic Pseudomonas aeruginosa infection in cystic fibrosis patients from the age of 6 years. Clinical studies show a safety and efficacy profile comparable to tobramycin inhaled solution (TOBI). The short administration time and the convenient use of the system may significantly decrease the treatment burden. This paper gives a short review of the clinical studies and some practical information.
In vitro aerodynamic characterization of the dose emitted during nebulization of tobramycin high strength solution by novel and jet nebulizer delivery systems. [2018]Chronic infections with Pseudomonas aeruginosa are a leading cause of morbidity in patients with cystic fibrosis (CF). The aim of tobramycin inhalation therapy in CF patients with chronic pulmonary infection is to deliver high amounts of drug directly to the site of infection. TOBI(®) is a tobramycin nebulizer solution (300 mg/5 ml) approved by FDA for maintenance therapy for patient with CF. The 20% tobramycin sulfate solution was reported as the optimal and maximal concentration.
Inhaled tobramycin (TOBI): a review of its use in the management of Pseudomonas aeruginosa infections in patients with cystic fibrosis. [2018]Specifically formulated for nebulisation using the PARI LC PLUS reusable nebuliser, tobramycin solution for inhalation (TSI) [TOBI] provides a high dose of tobramycin (an aminoglycoside antibacterial with good activity against Pseudomonas aeruginosa) to the lungs of patients with cystic fibrosis, while maintaining low serum concentrations of the drug, thus reducing the risk of systemic toxicity. Intermittent (28-day on/28-day off) treatment with TSI 300 mg twice daily significantly (p or =6 years with cystic fibrosis and chronic P. aeruginosa infection. Improvements in lung function were most marked in adolescent patients (aged 13-17 years) in placebo-controlled trials. Improvements were maintained for up to 96 weeks in patients in an open-label extension study. Fewer TSI than placebo recipients required parenteral antipseudomonal agents or hospitalisation. In addition, TSI 300 mg twice daily for 28 days reduced P. aeruginosa density in the lower airways of patients aged
Clinical use of tobramycin inhalation solution (TOBI®) shows sustained improvement in FEV1 in cystic fibrosis. [2021]Tobramycin inhalation solution (TIS; TOBI®) has improved forced expiratory volume in 1 sec (FEV1 ) in cystic fibrosis (CF) trials. Using data from the Epidemiologic Study of CF (ESCF), we assessed the change in level and trend of FEV1 % predicted (pred) over a 2-year period associated with initiation of TIS during routine clinical practice.
A pilot study of the safety and efficacy of tobramycin solution for inhalation in patients with severe bronchiectasis. [2022]To evaluate the efficacy and safety of tobramycin solution for inhalation (TSI) in patients with severe bronchiectasis.
Dose administration maneuvers and patient care in tobramycin dry powder inhalation therapy. [2018]The purpose of this work was to study a new dry powder inhaler (DPI) of tobramycin capable to simplify the dose administration maneuvers to maximize the cystic fibrosis (CF) patient care in antibiotic inhalation therapy. For the purpose, tobramycin/sodium stearate powder (TobraPS) having a high drug content, was produced by spray drying, characterized and the aerodynamic behavior was investigated in vitro using different RS01 DPI inhalers. The aerosols produced with 28, 56 or 112 mg of tobramycin in TobraPS powder using capsules size #3, #2 or #0 showed that there was quasi linear relationship between the amount loaded in the device and the FPD. An in vivo study in healthy human volunteers showed that 3-6 inhalation acts were requested by the volunteers to inhale 120 mg of TobraPS powder loaded in a size #0 capsule aerosolized with a prototype RS01 device, according to their capability to inhale. The amount of powder emitted at 4 kPa pressure drop at constant air flow well correlated with the in vivo emission at dynamic flow, when the same volume of air passed through the device. The novel approach for the administration of 112 mg of tobramycin in one capsule could improve the convenience and adherence of the CF patient to the antibiotic therapy.