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

NCT05101915 | Terminated Phase 2 DMC

• 1 other identifier: P02702
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

Brief 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

Conditions

Cystic Fibrosis

Keywords

Mycobacterium abscessus

Outcome Measures

Primary Outcomes (2)

• Mycobacterial load in induced sputum samples

  The primary efficacy endpoint is the change in mycobacterial load in induced sputum samples as assessed by log10 change in M. abscessus cfu/g sputum from Baseline to End of Treatment.

  Through study completion, average one year

• Safety and tolerability

  Safety and tolerability will be assessed by clinical safety laboratory measurements, physical examinations, vital signs, concomitant medications; cumulative incidence of adverse events (AEs), serious adverse events (SAEs) and severe AEs.

  Through study completion, average one year

Secondary Outcomes (3)

• Change in mycobacterial load in spontaneously expectorated daily sputum samples

  Through study completion, average one year

• Proportion of individuals achieving a ≥2 log10 decrease in the mycobacterial load - induced samples

  Through study completion, average one year

• Proportion of individuals achieving a ≥2 log10 decrease in the mycobacterial load - spontaneous samples

  Through study completion, average one year

Study Arms (1)

Interventional

EXPERIMENTAL

Single arm trial involving all patients receiving IMP

Drug: RESP301

Interventions

RESP301 DRUG

Inhaled IMP delivered via nebulisation

Interventional

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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)

You may not qualify if:

• 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.

Sponsors & Collaborators

• Papworth Hospital NHS Foundation Trust: lead

Study Sites (1)

Royal Papworth Hospital NHS Foundation Trust

Cambridge, Cambridgeshire, CB30AY, United Kingdom

Location

MeSH Terms

Conditions

Cystic Fibrosis; Mycobacterium Infections, Nontuberculous

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases; Mycobacterium Infections; Actinomycetales Infections; Gram-Positive Bacterial Infections; Bacterial Infections; Bacterial Infections and Mycoses; Infections

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER GOV
• Responsible Party: SPONSOR

Model Details: This is a single centre, non-randomized, open label study

Study Record Dates

• First Submitted: September 13, 2021
• First Posted: November 1, 2021
• Study Start: November 1, 2021
• Primary Completion: January 31, 2024
• Study Completion: April 23, 2024
• Last Updated: May 10, 2024

Record last verified: 2024-05

Locations

GB (1)