Clinical trial NCT00274313: Safety Study of Inhaled 552-02 in Cystic Fibrosis Patients
Official title: A Study of the Safety and Pharmacokinetics of 552-02 Following 14 Days of Dosing By Inhalation in Patients With Cystic Fibrosis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Cystic Fibrosis
Keywords: Cystic Fibrosis
Lead sponsor: Parion Sciences
Sponsor class: INDUSTRY
First posted: 2006-01-10
Enrollment: 40
Sex: ALL
Minimum age (years): 14
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to evaluate the safety and tolerability of a new inhaled sodium-channel blocker called 552-02 in teens and adults with cystic fibrosis. 552-02 will be inhaled once a day for 14 days using a nebulizer. A small subgroup of patients will donate blood samples for pharmacokinetic analysis to see how 552-02 is absorbed into the blood and eliminated after 14 days of treatment.


Eligibility criteria: Inclusion Criteria:

1. Male and female patients aged \> 14 years.
2. Patients who are diagnosed with cystic fibrosis.
3. Patients who have a FEV1 ≥ 50% predicted (post-bronchodilator) at screening.
4. Patients who are able to perform reproducible spirometry according to ATS guidelines.
5. Patients who have an oxygen saturation of ≥ 92% on room air as determined by pulse oximetry at screening.

Exclusion Criteria:

1. Patients who have a FEV1 change ≥ 15% after bronchodilator use at screening.
2. Patients who have unstable lung disease as defined by the requirement for intravenous antibiotics during the four weeks prior to screening, a change in medical regimen within 14 days prior to administration of the first dose of study drug or during the 14 day treatment period, a FEV1 ≥ 15% below recent (within six months) clinical measurements, or significant new findings on chest radiograph (pneumothorax, lobar/segmental collapse) that are not considered a part of the usual, chronic progression of cystic fibrosis lung disease.
3. Patients on angiotensin converting enzyme (ACE) inhibitors.
4. Patients with renal insufficiency as evidenced by hyperkalemia (blood potassium levels greater than 5.5 mEq/L) or serum creatinine \> 2.0 mg/dL.
5. Patients who have a history of drug allergies to any medicine chemically related to the study drug (e.g. amiloride, Moduretic, Midamor; triamterene).
6. Patients who are pregnant, have a positive pregnancy test, or are nursing.
7. Patients who have had a lung transplant.

Primary outcomes:
measure: Safety assessments
measure: Blood and urine laboratory tests
measure: Pulmonary function tests
measure: Electrocardiograms
measure: Vital signs and pulse oximetry
Secondary outcomes:
measure: Plasma pharmacokinetics on Day 14 of the study.
Other outcomes:
Locations:
city: San Diego
country: United States
facility: University of California at San Diego
state: California
city: San Francisco
country: United States
facility: University of California at San Francisco Medical Center
state: California
city: Denver
country: United States
facility: The Children's Hospital
state: Colorado
city: Orlando
country: United States
facility: Nemours Children's Clinic
state: Florida
city: Tampa
country: United States
facility: University of South Florida
state: Florida
city: Chicago
country: United States
facility: Children's Memorial Hospital
state: Illinois
city: Omaha
country: United States
facility: University of Nebraska Medical Center
state: Nebraska
city: Morristown
country: United States
facility: Morristown Memorial Hospital
state: New Jersey
city: Syracuse
country: United States
facility: State University of New York Upstate
state: New York
city: Chapel Hill
country: United States
facility: University of North Carolina at Chapel Hill
state: North Carolina
city: Hershey
country: United States
facility: Hershey Medical Center
state: Pennsylvania
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Pittsburgh
country: United States
facility: University of Pittsburgh
state: Pennsylvania
city: Charleston
country: United States
facility: Medical University of South Carolina
state: South Carolina
city: Charlottesville
country: United States
facility: University of Virginia
state: Virginia
Study officials:
References: