Study of a Nebulised Nitric Oxide Generating Solution in Patients With Mycobacterium Abscessus (NOMAB)

NHS Foundation Trust

Status and phase

Terminated

Phase 2

Conditions

Cystic Fibrosis

Treatments

Drug: RESP301

Study type

Interventional

Funder types

Other

Identifiers

NCT05101915

P02702

Details and patient eligibility

About

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

Full description

Investigators will undertake an eighteen-week single centre, open label study in participants with cystic fibrosis infected with Mycobacterium abscessus (M. abscessus)-pulmonary disease (-PD).

The study will treat particpants with cystic fibrosis (CF) attending the Adult Cystic Fibrosis Centre at the Royal Papworth Hospital, Cambridge, United Kingdom. Participants will be consented and screened for the RESP301-003 study to enable approximately 12 participants to commence treatment with RESP301.

Participants will have M abscessus-PD as defined by the ATS/IDSA, specifically: (i) two or more positive sputum cultures for M. abscessus; (ii) radiological change consistent with NTM-PD; and (iii) symptoms consistent with NTM-PD, after exclusion of other causes.

Participants will be recruited who (1) have not commenced antibiotic treatment for M. abscessus-PD or (2) have treatment refractory M. abscessus-PD (defined as remaining sputum culture positive after 6 months or more of treatment). Treatment-refractory participants will be suitable for enrolment in the study if date of first dosing is at least 2 months since a change in M. abscessus treatment (or 4 months since change of Clofazimine).

Enrollment

6 patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Adult patients of ≥18 years at time of informed consent
Patients with a clinical diagnosis of CF and confirmed by genetic testing
Diagnosis of treatment naïve or treatment refractory M. abscessus-PD
Signed informed consent documentation (indicating an understanding of the purpose and a willingness to meet the requirements for participation in the study)

Exclusion criteria

FEV1 <40% predicted
Methaemoglobin concentration > 2%
Use of nitric oxide donor medications such as prilocaine, sodium nitroprusside, and nitroglycerine within 30 days of proposed first treatment
Use of phosphodiesterase inhibitors (e.g., sildenafil) within 30 days of proposed first treatment
Evidence of pulmonary hypertension
History of frequent low volume or massive haemoptysis
Liver disease (i.e. liver cirrhosis, portal hypertension)
Subjects who have undergone organ transplantation
Pregnancy or lactation (female participants only)
Subjects who will not use appropriate forms of contraception for the duration of the study
Contraindication or unable to complete lung function testing
Contraindication or unable to tolerate nebulised hypertonic saline
Changes to previous NTM antibiotic regimen within two months of first dose of study treatment (or 4 months for clofazimine)
Subject has received investigational treatment as part of another interventional clinical trial within two months of the proposed first day of treatment
Required antibiotic treatment for a pulmonary exacerbation within 2 weeks of enrolment to the study.
Inability to undergo study related activities and / or commitments
Any subject who in the opinion of the investigator would not be best served by participating in this clinical trial.