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

NCT ID: NCT00004428

Last Updated: 2015-03-25

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

35 participants

Study Classification

INTERVENTIONAL

Study Start Date

1997-09-30

Study Completion Date

1999-10-31

Brief Summary

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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

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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.

Conditions

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Cystic Fibrosis

Study Design

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Allocation Method

RANDOMIZED

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Interventions

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CPX

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

PROTOCOL ENTRY CRITERIA:

* Mild cystic fibrosis
* Not pregnant or nursing Negative pregnancy test
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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SciClone Pharmaceuticals

INDUSTRY

Sponsor Role collaborator

FDA Office of Orphan Products Development

FED

Sponsor Role lead

Principal Investigators

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Eduardo Martins

Role: STUDY_CHAIR

SciClone Pharmaceuticals

References

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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.

Reference Type DERIVED
PMID: 37983082 (View on PubMed)

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.

Reference Type DERIVED
PMID: 33331662 (View on PubMed)

Other Identifiers

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SCICLONE-FDA-OP-97-1

Identifier Type: -

Identifier Source: secondary_id

199/13364

Identifier Type: -

Identifier Source: org_study_id

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