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

• Register: ClinicalTrials.gov
• Last refreshed on: 13 June 2016
• Main ID: NCT01958944
• Date of registration: 06/10/2013
• Prospective Registration: Yes
• Primary sponsor: Advanced Inhalation Therapies Ltd
• Public title: Evaluation of the Safety and Tolerability of Inhaled Nitric Oxide to Subjects With Cystic Fibrosis
• Scientific 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
• Date of first enrolment: December 2013
• Target sample size: 9
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01958944
• Study type: Interventional
• Study design: Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 1/Phase 2

Countries of recruitment

Israel

Contacts

• Name: Hannah Blau, M.D
• Affiliation: Schneider Children's Medical Center, Israel

• Name: Asher Tal, M.D
• Affiliation: Soroka University Medical Center

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Subjects (Male or female) =10 years old

2. Confirmed diagnosis of CF

3. Resting awake oxygen saturation of at least 92% in room air

4. Approved and signed informed consent:

1. Subject aged over 10 years old (10 included) -

- signed an informed consent by the subject

- Parents/ legal guardian signed informed consent.

2. Subject aged over 18 years old (18 included) - • signed an informed consent by
the subject

5. 80% =FEV1= 30%

6. Confirmed to be colonized with Pseudomonas aeruginosa

Exclusion Criteria:

1. Subjects younger than 10 years old

2. FEV1< 30% or FEV1> 80%

3. Pulmonary exacerbation resulting in antibiotic treatment (except prophylactic
antibiotics) within1 month before enrollment

4. Subject is pregnant (when applicable, a negative pregnancy test result must be
verified prior to enrollment and during treatment)

5. Subjects diagnosed with methemoglobinemia, immunodeficiency and/ or heart disease.

6. 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.

7. History of frequent epistaxis (>1 episode/month)

8. Significant hemoptysis within 30 days (= 5 mL of blood in one coughing episode or >
30 mL of blood in a 24 hour period)

9. Methemoglobin level>3% at screening

10. Patients on systemic steroids (1mg/kg or > 20mg of prednisone per day) within 30 days
of screening;

11. Smokers;

12. History of illicit drug or medication abuse within 1 year of screening ;

13. history of lung transplantation;

14. Patients treated for high blood pressure

15. Subjects cannot comply with the study design

16. 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.

17. The subject is identified by the investigator as being unable or unwilling to perform
study procedures.



Age minimum: 10 Years
Age maximum: N/A
Gender: Both

Health Condition(s) or Problem(s) studied

Cystic Fibrosis

Intervention(s)

Drug: Nitric oxide

Primary Outcome(s)

Proportion of subjects (%) who prematurely discontinued the study due to adverse events or serious adverse events [Time Frame: 1 month]

Met-Hemoglobin percentage (MetHb)associated with inhaled NO [Time Frame: 1 month]

Proportion of subjects (%) who prematurely discontinued the study for any reason [Time Frame: 1 month]

Number of participants with adverse events associated with inhaled NO [Time Frame: 1 month]

Secondary Outcome(s)

Comparing the FEV1 improvement of =10 years old with CF before and after NO treatment [Time Frame: 1 month]

Secondary ID(s)

AIT_ CP_ CF 01

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Soroka University Medical Center

Schneider Children's Medical Center, Israel