Phase I Randomized Study of CPX for the Treatment of Adul... | NCT00004428

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

NCT00004428

Sponsor

FDA Office of Orphan Products Development

Status

Completed

Last Update Posted

Mar 25, 2015Estimated

Enrollment

Phase

Phase 1

Conditions

Cystic Fibrosis

Interventions

Countries

Not provided

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

ID	Type	Description	Link
SCICLONE-FDA-OP-97-1

Status Module

Record Verification Date

Jan 2000

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Sep 1997

Primary Completion Date

Not provided

Completion Date

Oct 1999

First Submitted Date

Oct 18, 1999

First Submission Date that Met QC Criteria

Oct 18, 1999

First Posted Date

Oct 19, 1999Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Mar 24, 2015

Last Update Posted Date

Mar 25, 2015Estimated

Name	Class
SciClone Pharmaceuticals	INDUSTRY

Description Module

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.

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 1

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

Name	Type	Description	Arm Group Labels	Other Names
CPX	Drug

Eligibility Module

Eligibility Criteria

PROTOCOL ENTRY CRITERIA:

Mild cystic fibrosis
Not pregnant or nursing Negative pregnancy test

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

Not provided

Standard Ages

AdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Name	Affiliation	Role
Eduardo Martins	SciClone Pharmaceuticals	Study Chair

References Module

Citations

PubMed Identifier	Type	Citation	Retractions
37983082	Derived	Heneghan M, 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. 2023 Nov 20;11(11):CD010966. doi: 10.1002/14651858.CD010966.pub4.
33331662	Derived

ID	Term
D003550	Cystic Fibrosis
D012140	Respiratory Tract Diseases
D030342	Genetic Diseases, Inborn
D035583	Rare Diseases

ID	Term
D010182	Pancreatic Diseases
D004066	Digestive System Diseases
D008171	Lung Diseases
D009358	Congenital, Hereditary, and Neonatal Diseases and Abnormalities