Safety and Tolerability of Inhaled Nitric Oxide in Patients With Cystic Fibrosis

NCT00570349 | Completed Phase 1 Results DMC

• 1 other identifier: INOT 50
• Study Type: interventional
• Target: 18
• Locations: 1 country
• Sites: 1

Brief Summary

The primary objective of the trial is to assess the safety and tolerability of inhaled nitric oxide (NO) when administered by nasal cannula over a 44 hour period to clinically stable Cystic Fibrosis (CF) subjects. Toxicity is to be defined as a drop in oxygen saturations, a decline in forced expiratory volume in one second (FEV1), or an increase in methemoglobin.

Conditions

Cystic Fibrosis

Keywords

Inhaled Nitric oxide; Cystic Fibrosis

Outcome Measures

Primary Outcomes (3)

• Safety and Tolerability of Drug, Assessed by Change in Methemoglobin Levels

  Methemoglobin level assessments were measured through blood draws - hematology. This test measures the amount of methemoglobin (a type of hemoglobin that is unable to transport oxygen to tissues) in blood. Normal methemoglobin percentage range 1% - 2%.

  Baseline and 48 hours

• Change in Oxygen Saturation

  Safety and tolerability of drug assessed by decreased oxygen saturation was measured through pulse oximeter, which measure the amount of oxygen in the blood. Normal range percentage is 95 - 100%

  Baseline and 48 hours

• Change in Forced Expiratory Volume in 1 Second (FEV1)

  Decrease in forced expiratory volume in 1 second was measured through spirometer. Spirometer measures the volume of air inspired and expired by the lungs.

  Baseline and 48 hours

Study Arms (3)

Low Dose Cohort

EXPERIMENTAL

Subjects in the low dose cohort receive 20 part per million (ppm) of nitric oxide via nasal cannula over a 44 hour period.

Drug: Nitric Oxide for Inhalation

High-Dose Cohort

EXPERIMENTAL

Subjects in the high dose cohort receive 40 ppm of nitric oxide via nasal cannula over a 44 hour period.

Drug: Nitric Oxide for Inhalation

Nitrogen

PLACEBO COMPARATOR

100% Nitrogen (placebo) will be administer at 20 ppm or 40 ppm via nasal cannula over a 44 hour period.

Drug: Nitrogen

Interventions

Nitric Oxide for Inhalation DRUG

Nitric oxide will be administered at 20 ppm via nasal cannula over a 44 hour period.

Also known as: INO

Low Dose Cohort

Nitrogen DRUG

100% nitrogen (placebo) will be administered at 20 ppm or 40 ppm via nasal cannula over a 44 hour period.

Also known as: Nitrogen (N2) Grade 5

Nitrogen

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Confirmed diagnosis of CF
• years of age and older
• FEV1 greater than 40% of predicted
• Resting awake oxygen saturation of at least 88%
• Stable pulmonary disease as defined by both clinical impression and having had no recent hospitalizations or changes in antibiotic regimen within 1 month prior to enrollment
• Signed informed consent form

You may not qualify if:

• Pulmonary exacerbation resulting in antibiotic treatment (except prophylactic antibiotics) within 1 month of enrollment
• Isolation of B. cepacia from a respiratory tract culture within 6 months
• Severe nasal obstruction at the time of screening
• Receipt of any aerosolized experimental or investigational drugs within 1 month of enrollment
• Pregnancy (a negative pregnancy test must be documented prior to enrollment if applicable)
• Patients who have received treatment with nitric oxide for inhalation within 24 hours prior to study initiation or other investigational medications within 24 hours.

Sponsors & Collaborators

• Mallinckrodt: lead

Study Sites (1)

The Children's Hospital

Denver, Colorado, 80218, United States

Location

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

Nitric Oxide; Inhalation; Nitrogen

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

Intervention Hierarchy (Ancestors)

Reactive Nitrogen Species; Free Radicals; Inorganic Chemicals; Nitrogen Oxides; Nitrogen Compounds; Oxides; Oxygen Compounds; Organic Chemicals; Respiratory Mechanics; Respiration; Respiratory Physiological Phenomena; Circulatory and Respiratory Physiological Phenomena; Elements; Gases

Results Point of Contact

• Title: Medical Information Call Center
• Organization: Mallinckrodt Pharmaceuticals

clinicaltrials@mnk.com

800-556-3314

Study Officials

• Scott Sagel, MD

  The Children's Hospital

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 6, 2007
• First Posted: December 10, 2007
• Study Start: July 1, 2004
• Primary Completion: December 1, 2008
• Study Completion: December 1, 2008
• Last Updated: November 13, 2019
• Results First Posted: December 1, 2010

Record last verified: 2016-05

Locations

US (1)