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

FDA Office of Orphan Products Development

Status and phase: Completed; Phase 1

Conditions: Cystic Fibrosis

Treatments: Drug: CPX

Study type: Interventional

Funder types: Other U.S. Federal agency; Industry

Identifiers

NCT00004428

199/13364

SCICLONE-FDA-OP-97-1

Details and patient eligibility

About

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.

Full 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.

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

PROTOCOL ENTRY CRITERIA:

• Mild cystic fibrosis
• Not pregnant or nursing Negative pregnancy test