Clinical trial NCT00041574: Hospital-Based Program for Treatment of Severe Cardiopulmonary Disease With Inhaled Nitric Oxide
Official title: Development of a Hospital-Based Home Program for the Use of Inhaled Nitric Oxide in the Chronic Management of Severe Cardiopulmonary Diseases
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Pulmonary Hypertension
Lung Disease
Sickle Cell Disease
Cardiac Transplant
Lung Transplant
Lead sponsor: Mallinckrodt
Sponsor class: INDUSTRY
First posted: 2002-07-11
Enrollment: 7
Sex: ALL
Minimum age (years): 5
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of this program is to evaluate the logistic issues and patient requirements for chronic pulsed INOmax delivery in ambulatory, home-care patients. To understand patient needs, patients with a variety of underlying diseases will be included. Safety of chronic therapy will be monitored by serial measurements of methemoglobin, platelet function assay and reported adverse events.

Detailed description: This study will help develop a comprehensive home care program using INOmax delivery systems. This study will enroll a limited number of patients (5-7) from several disease categories in order to develop the experience with testing acute and chronic responsiveness to INO therapy. The following groups will be tested:

1. Primary pulmonary hypertension (PPH)
2. Severe pulmonary hypertension due to congenital (anatomic) heart disease
3. Pulmonary hypertension or severe impairment of gas exchange in infants and children with chronic lung disease
4. Pulmonary hypertension or severe impairment of gas exchange due to severe chronic lung disease in adults
5. Pulmonary hypertension or severe impairment of gas exchange in hematologic and transplant disorders - young children and adults with sickle cell disease, chronic lung disease after bone marrow transplant, cardiac transplant, or lung transplant

Eligibility criteria: Inclusion criteria:

* Patients with pulmonary arterial hypertension (PAH) as defines by a mean pulmonary artery pressure (PAP) of 25 mmHg or greater by cardiac catheterization within the previous 12 months AND/OR Patients with chronic lung disease with severe impairment of gas exchange unresponsive to conventional medical management.
* If patient is receiving prostacyclin infusion treatment it must have been started at least one month prior to starting INO therapy. Patients will not be withdrawn from the study if started on other medications for treatment of pulmonary hypertension.
* Ability to establish a clinical investigator and a drug shipment site in the local area.

Exclusion Criteria:

* The Principal Investigator has determined that the patient and/or caregiver is/are unable to manage safe administration of home nitric oxide therapy and maintain appropriate drug accountability and storage.
* Women who are pregnant or nursing.

Primary outcomes:
measure: Methemoglobin level
timeFrame: 24 and 72 hours after treatment start, then at 1, 3, 6, 9 and 12 months
Secondary outcomes:
measure: Vital Signs
timeFrame: taken at 1, 2, 3, 4, 24, and 72 hours after treatment begins, then at 1, 3, 6, 9 and 12 months
measure: Pulse Oximetry
timeFrame: taken at 1, 2, 3, 4, 24, and 72 hours after treatment start and then at 1, 3, 6, 9 and 12 months
measure: Adverse Event Occurence
timeFrame: Study Duration
measure: Echocardiogram
timeFrame: At 72 hours after treatment start, then at 3, 6, 9 and 12 months
Other outcomes:
Locations:
city: Denver
country: United States
facility: The Children's Hospital
state: Colorado
city: Denver
country: United States
facility: University of Colorado Hospital
state: Colorado
city: Providence
country: United States
facility: Rhode Island Hospital
state: Rhode Island
Study officials:
affiliation: Children's Hospital Colorado
name: Steven H Abman, MD
role: PRINCIPAL_INVESTIGATOR
References: