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Phase I Randomized Study of CPX for the Treatment of Adult Patients With Mild Cystic Fibrosis

This study has been completed.
SciClone Pharmaceuticals
Information provided by:
FDA Office of Orphan Products Development Identifier:
First received: October 18, 1999
Last updated: March 24, 2015
Last verified: January 2000

OBJECTIVES: I. Evaluate the safety of ascending doses of CPX administered to adult patients with mild cystic fibrosis.

II. Evaluate the pharmacokinetics of ascending doses of CPX in this patient population.

Condition Intervention Phase
Cystic Fibrosis Drug: CPX Phase 1

Study Type: Interventional
Study Design: Allocation: Randomized
Masking: Double
Primary Purpose: Treatment

Resource links provided by NLM:

Further study details as provided by FDA Office of Orphan Products Development:

Estimated Enrollment: 35
Study Start Date: September 1997
Estimated Study Completion Date: October 1999
Detailed Description:

PROTOCOL OUTLINE: This is a randomized, double blind, placebo controlled, dose escalation study.

There are 7 experimental cohorts, each treated with a different oral dose of CPX or placebo. Within each cohort, 4 patients receive a single dose of CPX and 1 patient receives placebo. Each patient is monitored 24 hours postdose. Escalation to the next dose level for each subsequent cohort begins only after the safety data obtained from the previous cohort is reviewed and found not to limit dose escalation.

All patients return for a follow up evaluation 1 week after dosing.


Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  • Mild cystic fibrosis
  • Not pregnant or nursing Negative pregnancy test
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT00004428

Sponsors and Collaborators
FDA Office of Orphan Products Development
SciClone Pharmaceuticals
Study Chair: Eduardo Martins SciClone Pharmaceuticals
  More Information Identifier: NCT00004428     History of Changes
Other Study ID Numbers: 199/13364
Study First Received: October 18, 1999
Last Updated: March 24, 2015

Keywords provided by FDA Office of Orphan Products Development:
cardiovascular and respiratory diseases
cystic fibrosis
genetic diseases and dysmorphic syndromes
rare disease

Additional relevant MeSH terms:
Cystic Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases processed this record on September 21, 2017