Back to resultsPhase I Randomized Study of CPX for the Treatment of Adult Patients With Mild Cystic Fibrosis
Primary Purpose
Cystic Fibrosis
Status
Completed
Phase
Phase 1
Locations
Study Type
Interventional
Intervention
CPX
Sponsored by
About this trial
This is an interventional treatment trial for Cystic Fibrosis focused on measuring cardiovascular and respiratory diseases, cystic fibrosis, genetic diseases and dysmorphic syndromes, rare disease
Eligibility Criteria
PROTOCOL ENTRY CRITERIA: Mild cystic fibrosis Not pregnant or nursing Negative pregnancy test
Sites / Locations
Outcomes
Primary Outcome Measures
Secondary Outcome Measures
Full Information
NCT ID
NCT00004428
First Posted
October 18, 1999
Last Updated
March 24, 2015
Sponsor
FDA Office of Orphan Products Development
Collaborators
SciClone Pharmaceuticals
1. Study Identification
Unique Protocol Identification Number
NCT00004428
Brief Title
Phase I Randomized Study of CPX for the Treatment of Adult Patients With Mild Cystic Fibrosis
Study Type
Interventional
2. Study Status
Record Verification Date
January 2000
Overall Recruitment Status
Completed
Study Start Date
September 1997 (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
October 1999 (undefined)
3. Sponsor/Collaborators
Name of the Sponsor
FDA Office of Orphan Products Development
Collaborators
SciClone Pharmaceuticals
4. Oversight
5. Study Description
Brief Summary
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.
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cystic Fibrosis
Keywords
cardiovascular and respiratory diseases, cystic fibrosis, genetic diseases and dysmorphic syndromes, rare disease
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Masking
Double
Allocation
Randomized
Enrollment
35 (false)
8. Arms, Groups, and Interventions
Intervention Type
Drug
Intervention Name(s)
CPX
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
PROTOCOL ENTRY CRITERIA:
Mild cystic fibrosis
Not pregnant or nursing Negative pregnancy test
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Eduardo Martins
Organizational Affiliation
SciClone Pharmaceuticals
Official's Role
Study Chair
12. IPD Sharing Statement
Citations:
PubMed Identifier
33331662
Citation
Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2020 Dec 17;12(12):CD010966. doi: 10.1002/14651858.CD010966.pub3.
Results Reference
derived
Learn more about this trial
Phase I Randomized Study of CPX for the Treatment of Adult Patients With Mild Cystic Fibrosis
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