Nebulised RESP30X Nitric Oxide Formulations in NCFB Patients with Pseudomonas Aeruginosa (Pa) (NOPA)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Thirty Respiratory Limited

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Respiratory Tract Diseases [C08]

Trial duration

completed 20 Apr 2026

Decision date (initial)

2024-04-22

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

Funding sources

Thirty Respiratory Limited (30 Respiratory)

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Others, Safety

To assess the safety and tolerability of RESP30X in NCFB participants with confirmed high-titre respiratory PPMs.

Secondary objectives 1

1. To characterise the PK of RESP30X in NCFB participants with confirmed high-titre respiratory PPMs.

Conditions and MedDRA coding

Non-Cystic Fibrosis Bronchiectasis with Pseudomonas Aeruginosa or other Potentially Pathogenic Micro-organisms

Study design 5 periods

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 8

1. 1. Provide written, informed consent prior to all study-related procedures and agree to undergo all study procedures.
2. 2. Aged between 18 and 75 years, inclusive.
3. 3. Clinical history of bronchiectasis affecting 1 or more lobes based on symptoms (cough, sputum productive and/or recurrent lower respiratory tract infections) as confirmed by historical computerised tomography (CT) scan and radiology report performed within the last 5 years.
4. 4. Confirmed high-titre respiratory PPMs (Part 1: Pa only) at screening ≥10^⁵ CFU/mL (as determined by central laboratory microbiological cultures).
5. 5. Individuals of childbearing potential (IOCBP) and their partners who engage in heterosexual intercourse must agree to use protocol defined method(s) of contraception.
6. 6. Patients who can produce spontaneous sputum on a daily basis.
7. 7. Patients who are able to self-administer the SABA inhaler and study nebuliser for IMP administration effectively in the investigator’s opinion, following training.
8. 8. Patients appropriately vaccinated against influenza and pneumococcus at least 14-days prior to Day 1. This applies only if the screening visit falls between September and March and the Influenza vaccine/pneumococcal vaccine for that year is available.

Exclusion criteria 31

1. 1. Currently receiving therapy with any inhaled antibiotic therapy. Patients who have previously received inhaled antibiotic therapy may be eligible if therapy was discontinued at least 28-days prior to screening.
2. 10. Taking medications that may induce methaemoglobinaemia or have received these within 30 days of screening.
3. 3. Participation in other clinical studies with investigational agents within 8 weeks prior to screening.
4. 4. Treatment with NO and other NO donor agents, phosphodiesterase inhibitors and lung surfactant drugs, within 30 days prior to screening.
5. 5. Treatment with immunosuppressive medications within 2 weeks prior to screening, or systemic corticosteroids, or immunoglobulin therapy for> 7 days within 2 weeks prior to screening.
6. 6. HIV positive AND: • CD4 < 350 cells/mm3
7. 7. FEV1 <55% predicted at the screening visit.
8. 8. Significant haemoptysis within 60 days of screening defined as an estimated volume of 50ml in a single occurrence.
9. 9. History of methaemoglobinaemia.
10. 22. Known allergy to active substance or any excipients or to auxiliary product.
11. 13. In the opinion of the investigator, patients with an acute exacerbation of NCFB
12. 11. Baseline SpMet >5%.
13. 12. Current smokers of tobacco products, marijuana, e-cigarettes/vaping.
14. 14. In the opinion of the investigator, any other clinically relevant active respiratory disease with the potential to compromise patient safety or confound interpretation of safety or efficacy outcomes.
15. 15. Asthma which requires treatment with Global Initiative for Asthma steps 4–5 suggested medications for the previous year, or systemic corticosteroids for ≥50% of the previous year.
16. 16. Patients with a diagnosis of primary ciliary dyskinesia
17. 17. Patients with a diagnosis of pulmonary hypertension.
18. 21. Conditions of increased risk for MetHb formation, significant anaemia or haemoglobinopathy.
19. 18. Patients with a current diagnosis of pulmonary TB based on clinical testing or symptoms. Patients with a history of pulmonary TB who have completed a course or eradication therapy at least 2 years prior to screening may be eligible if there is no clinical suspicion of recurrence. Patients with latent pulmonary TB are eligible provided they have received adequate treatment per local country guidelines.
20. 19. Patients with a diagnosis, or suspected diagnosis, of nontuberculous mycobacteria infection. Patients with a previous positive culture that is suspected to be a contaminant are eligible.
21. 23. Known hypersensitivity to NO.
22. 20. Symptomatic gastroesophageal reflux disease (GERD) causing NCFB disease.
23. 28. Baseline-corrected QTcF >450 msec (males) or 470 msec (females) or history of congenital long QT syndrome, Torsades de Pointes or other clinically significant abnormal ECG at Screening screening or Baseline baseline.
24. 29. History of solid organ transplantation.
25. 30. History of malignancy or treatment for malignancy within the past year.
26. 2. Treatment with systemic anti-infective therapy within 28-days prior to screening. Treatment with azithromycin may be permitted only if in accordance with Protocol Section 4.4.1.
27. 24. History of anaphylaxis to any medication or hospitalisation due to an adverse drug reaction (ADR)
28. 25. Patients who are pregnant or breast-feeding.
29. 26. Patients planning to conceive a child within the anticipated period of study participation and for at least 90 days after the last dose of IMP in the study.
30. 27. Patients with the following toxicities at screening as defined by the enhanced CTCAE toxicity table version 5.0, 27Nov2017. See protocol.
31. 30.Baseline-corrected QTcF >450 msec (males) or 470 msec (females) or history of congenital long QT syndrome, Torsades de Pointes or other clinically significant abnormal ECG at screening or baseline.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 3

1. 1. Incidence, intensity, causality, and seriousness of treatment-emergent adverse events (TEAEs).
2. 2. Changes in clinical laboratory values (haematology, clinical chemistry) at each time point.
3. 3. Changes in vital signs (blood pressure, heart rate, temperature, respiratory rate) and oxygen saturation (SpO2) at each time point.

Secondary endpoints 1

1. Plasma PK parameters peak serum concentration (Cmax), time to peak serum concentration (Tmax), area under the concentration-time curve from dosing (time 0) to time t (AUC0-t), area under the concentration-time curve from dosing (time 0) to end of dosing period (AUC0-tau), area under the concentration-time curve for total drug exposure from dosing (time 0) extrapolated to infinity (AUC0-inf), trough plasma concentration (Ctrough), and half-life (t1/2).

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 2

Auxiliary 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Thirty Respiratory Limited

Sponsor organisation

Thirty Respiratory Limited

Address

1 Red Place

City

London

Postcode

W1K 6PL

Country

United Kingdom

Scientific contact point

Organisation

Thirty Respiratory Limited

Contact name

Syed Jafri

Public contact point

Organisation

Thirty Respiratory Limited

Contact name

Syed Jafri

Third parties 13

Locations

3 EU/EEA countries · 5 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 28 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

3 submissions — initial application plus substantial / non-substantial modifications