Carlos Milla
ISIS Study
Contact Information
Stanford University School of Medicine 300 Pasteur Drive Stanford, CA 94305Brief
The study objective is to assess whether 7% HS is an effective and safe therapy in infants with Cystic Fibrosis. A growing body of evidence supports the importance of intervention in Cystic Fibrosis (CF) lung disease during infancy, in order to potentially delay or prevent irreversible lung disease. Yet, aside from antimicrobial therapies, the CF community has no clinical trial evidence base with which to guide pulmonary therapies in infants and toddlers. Hypertonic Saline (HS) is the most attractive chronic maintenance therapy to investigate in infants because it addresses defective mucociliary clearance, an early step in the cascade of events leading to CF lung disease that is expected to be abnormal prior to the onset of airway infection and inflammation.
Recruiting Status:
RecruitingStanford Recruiting Status:
RecruitingCondition(s):
Intervention(s):
- Drug: 7% Hypertonic Saline
Phase:
N/AEligibility
Ages Eligible for Study:
4 months to 16 monthsGenders Eligible for Study:
Male and FemaleHealth of Volunteers:
People with the conditions listed in this trial can participate as controls.Key Inclusion Criteria:
1 Diagnosis of CF by one or more clinical features of CF, AND either (a) or (b) as follows: a) A documented sweat chloride > 60 mEq/L by quantitative pilocarpine iontophoresis b) A genotype with two identifiable mutations consistent with CF
2 Informed consent by parent or legal guardian
3 4 months to <16 months of age at Screening Visit
4 Ability to comply with medication use, study visits and study procedures as judged by the site investigator
5 Functional residual capacity (FRC) measurement at the Baseline visit meets acceptability criteria as per TDN SOP 504, Training and Qualification to Perform Infant Pulmonary Function Testing in TDN Clinical Trials
Key Exclusion Criteria:
1. Acute wheezing at Screening visit (prior to HS test dose) or at Baseline visit
2. Acute intercurrent respiratory infection, defined as an increase in cough, wheezing, or respiratory rate
with onset in 1 week preceding Screening visit or 3 weeks preceding Baseline visit
3. Oxygen saturation < 95% (<90% in Denver, Colorado due to altitude) at Screening visit (prior to HS test dose) or at Baseline visit
4. History of adverse reaction to sedation
5. Clinically significant upper airway obstruction as determined by the Site Investigator (e.g. severe laryngomalacia, markedly enlarged tonsils, significant snoring, diagnosed obstructive sleep apnea)
6. Severe gastroesophageal reflux, defined as persistent frequent emesis despite anti-reflux therapy
7. Other major organ dysfunction excluding pancreatic dysfunction
8. Physical findings that would compromise the safety of the subject or the quality of the study data as determined by site investigator
9. Intolerance of test dose of HS at screening visit, as defined in Study Protocol Section
10. Investigational drug use within 30 days of Baseline visit
11. Findings from the infant lung function measurements at the Baseline visit that would compromise the safety of the subject, as determined by the site investigator.
Additional Study Details
Official Title:
INFANT STUDY OF INHALED SALINE IN CYSTIC FIBROSISAnticipated start date:
8/1/2009Lead Sponsor:
Cystic Fibrosis Foundation TherapeuticsInvestigator(s):
- Carlos Milla
- Colleen Dunn RCP
- Dr. Jacquelyn Marie Zirbes DNP
- Zoe Davies RN
- Martha Hamilton
- Terry Earl Robinson
- Richard B Moss
- Carol K. Conrad
- Andrea Core
- Yan Ki Angela Leung
Study Type:
InterventionalPurpose:
TreatmentAllocation:
RandomizedMasking:
OpenControl:
noneAssignment:
ParallelEndpoints:
Safety/EfficacyPrimary Outcomes:
- average change in functional residual capacity (FRC) measured by plethysmography during infant pulmonary function testing (iPFT) from baseline to end of treatment (48 ? 4 weeks) between infants randomized to Hypertonic Saline and isotonic saline.
Secondary Outcomes:
- the number of pulmonary exacerbations requiring treatment with oral, inhaled or intravenous antibiotics between infants randomized to HS and IS.
- rates of adverse events, withdrawal, adherence to treatment, and new isolation of CF pathogens from respiratory cultures; and resting respiratory rate and room air oxygen saturations over the 48 week treatment period, between infants randomized to HS and IS. The rate of intolerance to the test dose of HS at the Screening visit will also be described.
Total Number to be Enrolled:
150Total Number to be Enrolled at Stanford:
15More Information
Locations & Contacts
Stanford Locations & Contacts:
Stanford University School of Medicine 300 Pasteur Drive Stanford, CA 94305Non-Stanford Locations:
The Stanford website does not have any locations outside of Stanford listed for this trial. You may want to check clinicaltrials.gov for posible additional locations.
This listing was last updated:
8/24/2009PLEASE NOTE:
Study Coordinators and Research Nurses cannot give medical advice over the phone. Telephone numbers are provided for obtaining additional information on specific clinical research trials only. If you have specific questions which require clinical expertise, please call your primary care physician. If you do not have a primary care physician please feel free to call the SHC Physician Referral Service at (800) 756-9000 or send an email.
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