Cystic FibrosisPhase 2NOMABNCT05101915
Study of a Nebulised Nitric Oxide Generating Solution in Patients With Mycobacterium Abscessus
A Phase 2 study testing RESP301 in people with Cystic Fibrosis, run by Papworth Hospital NHS Foundation Trust at 1 site in United Kingdom.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- RESP301
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- 1 Nov 2021to 23 Apr 2024
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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)
Who cannot join
- 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
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- 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.
Trial locations and contacts
1 site in 1 country.
United Kingdom1 site
Royal Papworth Hospital NHS Foundation Trust
Cambridge, Cambridgeshire, CB30AY
About this study
Official title: Phase II Open Label Study of a Nebulised Nitric Oxide Generating Solution in Patients With Mycobacterium Abscessus Pulmonary Disease
- 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
Read the 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).
- Conditions
- Cystic Fibrosis
- Being tested
- RESP301
- Keywords
- Mycobacterium abscessus
- Medical subject headings
- Cystic Fibrosis, Mycobacterium Infections, Nontuberculous
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RESP301Drug
Inhaled IMP delivered via nebulisation
| Study group | Role | What they receive |
|---|---|---|
| Interventional | Experimental | Single arm trial involving all patients receiving IMP Receives: Drug: RESP301 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: Through study completion, average one year
Secondary outcomes (3)
Change in mycobacterial load in spontaneously expectorated daily sputum samples
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
Measured: Through study completion, average one year
Proportion of individuals achieving a ≥2 log10 decrease in the mycobacterial load - induced samples
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.
Measured: Through study completion, average one year
Proportion of individuals achieving a ≥2 log10 decrease in the mycobacterial load - spontaneous samples
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.
Measured: Through study completion, average one year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Nov 2021
- Study start1 Nov 2021
- Primary completion31 Jan 2024
- Study completion23 Apr 2024
Why it stopped: TSC recommended early termination due to lack of futility of the trial.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- This is a single centre, non-randomized, open label study
- Masking
- None (open label)
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Papworth Hospital NHS Foundation Trust
- Sponsor type
- Other government
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05101915
- Sponsor's protocol ID
- P02702
- Acronym
- NOMAB
- Status last verified
- May 2024
- First submitted
- 13 Sep 2021
- Last updated
- 10 May 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 May 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Open Label Study of a Nebulised Nitric Oxide Generating Solution in Patients With Mycobacterium Abscessus Pulmonary Disease
- ClinicalTrials.gov ID
- NCT05101915
- Acronym / sponsor ID
- NOMAB / P02702
- Conditions
- Cystic Fibrosis
- Keywords
- Mycobacterium abscessus
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: TSC recommended early termination due to lack of futility of the trial.
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Papworth Hospital NHS Foundation TrustOther government
- Study start
- 1 Nov 2021 (actual)
- Primary completion
- 31 Jan 2024 (actual)
- Study completion
- 23 Apr 2024 (actual)
- First posted
- 1 Nov 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
- 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
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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- This is a single centre, non-randomized, open label study
| Arm | Type | Receives |
|---|---|---|
| Interventional | Experimental | Drug: RESP301Single arm trial involving all patients receiving IMP |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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)
Key exclusion criteria (17 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RESP301Inhaled IMP delivered via nebulisation | Drug | Interventional |
Primary outcome
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.
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.
3 secondary outcomes
Change in mycobacterial load in spontaneously expectorated daily sputum samples
Time frame: Through study completion, average one year
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 - spontaneous samples
Time frame: Through study completion, average one year
Results and safety
The study was stopped early (reason given: TSC recommended early termination due to lack of futility of the trial.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 May 2024.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing RESP301 in people with Cystic Fibrosis, run by Papworth Hospital NHS Foundation Trust at 1 site in United Kingdom.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 10 May 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: TSC recommended early termination due to lack of futility of the trial.). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Mycobacterial load in induced sputum samples (time frame: Through study completion, average one year). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 May 2024.