Phase II Study of a Nebulised Nitric Oxide Generating Solution in Patients With Cystic Fibrosis

• Status: Not yet recruiting
• Trial end date: 2022-09-26
• Target enrollment: 0
• Participant gender: All

Summary

• To evaluate the change in M. abscessus cfu/g in induced sputum samples from baseline to the end of treatment with RESP301 in patients with cystic fibrosis who have treatment-naïve or treatment-refractory M. abscessus-pulmonary disease

• To assess the safety and tolerability of RESP301 during treatment (28 days) and follow up (84 days) in patients with cystic fibrosis who have treatment naïve or treatment refractory M. abscessus-pulmonary disease

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Papworth Hospital NHS Foundation Trust

Criteria

Inclusion Criteria:

1. Adult patients of ≥18 years at time of informed consent

2. Patients with a clinical diagnosis of CF and confirmed by genetic testing

3. Diagnosis of treatment naïve or treatment refractory M. abscessus-PD

4. Signed informed consent documentation (indicating an understanding of the purpose and a willingness to meet the requirements for participation in the study)

Exclusion Criteria:

1. FEV1 <30% predicted

2. Methaemoglobin concentration > 2%

3. Use of nitric oxide donor medications such as prilocaine, sodium nitroprusside, and nitroglycerine within 30 days of proposed first treatment

4. Use of phosphodiesterase inhibitors (e.g., sildenafil) within 30 days of proposed first treatment

5. Evidence of pulmonary hypertension

6. History of frequent low volume or massive haemoptysis

7. Liver disease (i.e. liver cirrhosis, portal hypertension)

8. Subjects who have undergone organ transplantation

9. Pregnancy or lactation (female participants only)

10. Subjects who will not use appropriate forms of contraception for the duration of the study

11. Contraindication or unable to complete lung function testing

12. Contraindication or unable to tolerate nebulised hypertonic saline

13. Changes to previous NTM antibiotic regimen within two months of first dose of study treatment (or 4 months for clofazimine)

14. Subject has received investigational treatment as part of another interventional clinical trial within two months of the proposed first day of treatment

15. Required antibiotic treatment for a pulmonary exacerbation within 2 weeks of enrolment to the study.

16. Inability to undergo study related activities and / or commitments

17. Any subject who in the opinion of the investigator would not be best served by participating in this clinical trial.