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Cystic FibrosisPhase 1/2NCT01958944

Evaluation of the Safety and Tolerability of Inhaled Nitric Oxide to Subjects With Cystic Fibrosis

A Phase 1/2 study testing Nitric oxide in people with Cystic Fibrosis, run by Beyond Air Inc. at 2 sites in Israel.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

Cystic Fibrosis is defined as a genetic disorder affecting approximately 100,000 individuals worldwide. CF is caused by mutations in the CF Transmembrane Conductance Regulator (CFTR) gene. CF patients are highly prone to environmental opportunistic bacterial infections leading to prolonged and chronic lung infections. Read more about this study

Who is it for?
Ages 10 years and older
Conditions the study is for
Cystic Fibrosis
What is being tested?
Nitric oxide
Could I receive a placebo?
No, everyone receives an active treatment
How many people
9 participants
Where
2 sitesIsrael
Study dates
Dec 2013to Nov 2015
Phase
Phase 1/2
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 10 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Subjects (Male or female) ≥10 years old
  • Confirmed diagnosis of CF
  • Resting awake oxygen saturation of at least 92% in room air
  • Approved and signed informed consent:
  • Subject aged over 10 years old (10 included) -
  • signed an informed consent by the subject
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  • Parents/ legal guardian signed informed consent.
  • Subject aged over 18 years old (18 included) - • signed an informed consent by the subject
  • 80% ≥FEV1≥ 30%
  • Confirmed to be colonized with Pseudomonas aeruginosa

Who cannot join

  • Subjects younger than 10 years old
  • FEV1< 30% or FEV1> 80%
  • Pulmonary exacerbation resulting in antibiotic treatment (except prophylactic antibiotics) within1 month before enrollment
  • Subject is pregnant (when applicable, a negative pregnancy test result must be verified prior to enrollment and during treatment)
  • Subjects diagnosed with methemoglobinemia, immunodeficiency and/ or heart disease.
  • Use of an investigational drug within 30 days prior enrolment and/ or the subject is expected to participate in a new study within three months from enrollment to this study.
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  • History of frequent epistaxis (>1 episode/month)
  • Significant hemoptysis within 30 days (≥ 5 mL of blood in one coughing episode or > 30 mL of blood in a 24 hour period)
  • Methemoglobin level>3% at screening
  • Patients on systemic steroids (1mg/kg or > 20mg of prednisone per day) within 30 days of screening;
  • Smokers;
  • History of illicit drug or medication abuse within 1 year of screening ;
  • history of lung transplantation;
  • Patients treated for high blood pressure
  • Subjects cannot comply with the study design
  • Presence of a condition or abnormality that in the opinion of the Investigator would compromise the safety of the subject or the quality of the data.
  • The subject is identified by the investigator as being unable or unwilling to perform study procedures.

Trial locations and contacts

2 sites in 1 country.

Israel2 sites

  • Soroka university

    Beersheba, 84101

  • Schneider Children's Medical Center of Israel

    Petach Tikvah, 49202

Other trials in Israel

About this study

Official title: Phase II Prospective, Open Labeled, Multi-Center, Evaluation of the Safety and Tolerability of Nitric Oxide Given Intermittently Via Inhalation to Subjects With Cystic Fibrosis

Cystic Fibrosis is defined as a genetic disorder affecting approximately 100,000 individuals worldwide. CF is caused by mutations in the CF Transmembrane Conductance Regulator (CFTR) gene. CF patients are highly prone to environmental opportunistic bacterial infections leading to prolonged and chronic lung infections. This results in reduction in the life expectancy of CF patients due to excessive lung tissue destruction.

Nitric Oxide (NO) is a naturally produced antimicrobial agent which is part of the innate immune defense system of the lung. Both in vitro and in vivo studies had shown clearly that NO acts against a wide variety of microbes including drug resistant bacteria as well as viruses and fungi. Building on a successful phase I safety trial, the team aims to develop a combined drug-device strategy to combat lung infections caused by biofilm-forming bacteria. Unlike other inhaled drugs, NO is also a smooth muscle relaxant and avoids the concomitant bronchial constriction often associated with inhaled antibiotics. An added benefit of NO therapy is its mucolytic activity. We suggest that the combine broad spectrum antimicrobial activity, signaling and mucolytic properties of NO, delivered to the lungs of CF patients, will be directed at reducing bacterial resistance, microbial burden and biofilms as well as resulting in improved airway clearance of viscid sputum.

Read more

Primary Objectives: Assess the safety and the tolerability of NO intermittent inhalation treatment in ≥10 years old CF subjects. Secondary Objective: Assess the improvement in forced expiratory volume in 1 second (FEV1) before and after NO intermittent inhalation. Up to 10 subjects with Cystic Fibrosis will be enrolled into the study.

Treatment administration: The subjects will receive intermittent inhalation of NO in addition to standard treatment for 10 working days (no NO treatment will be given to the subjects during weekend days). The subjects will be asked to attend the CF clinic once a week for a period of two weeks in order to evaluate the parameters related to the study. Oxygen (O2), NO, nitrogen dioxide (NO2) and fraction of inspired oxygen (FiO2) delivered to the patient will be continuously monitored.

Conditions
Cystic Fibrosis
Being tested
Nitric oxide
Keywords
CF, Cystic Fibrosis, NO, nitric oxide, inhalation
Medical subject headings
Cystic Fibrosis, Respiratory Aspiration

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Nitric oxideDrug

Study groups in NCT01958944
Study groupRoleWhat they receive
Nitric oxide + standard treatmentExperimentalInhalation of 160 ppm NO for 30 minutes, 3 times daily, for a duration of 10 working days with the exclusion of weekend days (Friday \& Saturday) in which no treatment under this study will be provided.
Receives: Drug: Nitric oxide

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Met-Hemoglobin percentage (MetHb)associated with inhaled NO

    Measured: 1 month

  2. Number of participants with adverse events associated with inhaled NO

    Measured: 1 month

  3. Proportion of subjects (%) who prematurely discontinued the study for any reason

    Measured: 1 month

  4. Proportion of subjects (%) who prematurely discontinued the study due to adverse events or serious adverse events

    Measured: 1 month

Secondary outcomes (1)

  1. Comparing the FEV1 improvement of ≥10 years old with CF before and after NO treatment

    Measured: 1 month

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted9 Oct 2013 (estimated)
  2. Study startDec 2013
  3. Primary completionNov 2015
  4. Study completionNov 2015

Resources

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 1/2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
9 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01958944
Sponsor's protocol ID
AIT_ CP_ CF 01
Status last verified
Jun 2016
First submitted
6 Oct 2013
Last updated
3 Jun 2016
Results posted
No
Data monitoring committee
No

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Nitric oxide in people with Cystic Fibrosis, run by Beyond Air Inc. at 2 sites in Israel.

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 10 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: finished, as of the sponsor's last update on 3 Jun 2016. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites in Israel. 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 combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

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?

Met-Hemoglobin percentage (MetHb)associated with inhaled NO (time frame: 1 month). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01958944.

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 3 Jun 2016.

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