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Trial record 1 of 1 for:    NCT05101915
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Phase II Study of a Nebulised Nitric Oxide Generating Solution in Patients With Cystic Fibrosis (NOMAB)

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ClinicalTrials.gov Identifier: NCT05101915
Recruitment Status : Recruiting
First Posted : November 1, 2021
Last Update Posted : June 27, 2022
Sponsor:
Information provided by (Responsible Party):
Papworth Hospital NHS Foundation Trust

Study Description
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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

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Drug: RESP301 Phase 2

Detailed 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).

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 12 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description: This is a single centre, non-randomized, open label study
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase II Open Label Study of a Nebulised Nitric Oxide Generating Solution in Patients With Cystic Fibrosis
Actual Study Start Date : November 1, 2021
Estimated Primary Completion Date : October 1, 2022
Estimated Study Completion Date : December 26, 2022

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis

Arms and Interventions
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Arm Intervention/treatment
Experimental: Interventional
Single arm trial involving all patients receiving IMP
 Drug: RESP301
Inhaled IMP delivered via nebulisation


Outcome Measures
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Primary Outcome Measures :
  1. Mycobacterial load in induced sputum samples [ Time Frame: Through study completion, average one year ]
    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.

  2. Safety and tolerability [ Time Frame: Through study completion, average one year ]
    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.


Secondary Outcome Measures :
  1. Change in mycobacterial load in spontaneously expectorated daily sputum samples [ Time Frame: Through study completion, average one year ]
    The change in mycobacterial load in spontaneously expectorated daily sputum samples from Baseline to Final Week of Treatment. For this secondary endpoint, Baseline is defined as the average M. abscessus cfu/g sputum in samples from the mornings of Days -14 to -1 inclusive. The final seven days of treatment are days 22 to 28 inclusive; the sputum samples are produced on the following mornings

  2. Proportion of individuals achieving a ≥2 log10 decrease in the mycobacterial load - induced samples [ Time Frame: Through study completion, average one year ]
    Proportion of individuals achieving a ≥2 log10 decrease in the mycobacterial load as assessed by change in M. abscessus cfu/g in the induced sputum samples from Baseline to End of Treatment.

  3. Proportion of individuals achieving a ≥2 log10 decrease in the mycobacterial load - spontaneous samples [ Time Frame: Through study completion, average one year ]
    Proportion of individuals achieving a ≥2 log10 decrease in the mycobacterial load as assessed by change in M. abscessus cfu/g in the spontaneously expectorated sputum samples from Baseline to Final Week of Treatment.


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05101915


Contacts
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Contact: Sarah Fielding 01223 639719 sarah.fielding2@nhs.net
Contact: Victoria Hughes 01223639678 victoria.hughes1@nhs.net

Locations
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United Kingdom
Royal Papworth Hospital NHS Foundation Trust Recruiting
Cambridge, Cambridgeshire, United Kingdom, CB30AY
Contact: Sarah Fielding    01223639719    sarah.fielding2@nhs.net   
Contact: Victoria Hughes       victoria.hughes1@nhs.net   
Sponsors and Collaborators
Papworth Hospital NHS Foundation Trust
More Information
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Responsible Party: Papworth Hospital NHS Foundation Trust
ClinicalTrials.gov Identifier: NCT05101915    
Other Study ID Numbers: P02702
First Posted: November 1, 2021    Key Record Dates
Last Update Posted: June 27, 2022
Last Verified: June 2022

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Cystic Fibrosis
Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
 Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases