Phenylbutyrate/Genistein Duotherapy in Delta F508-Heterozygotes (for Cystic Fibrosis)

This study has been terminated.

( 12/15/2008 Voluntarily placed on inactive status-requested by the PI )

Study NCT00590538 Information provided by Children's Hospital of Philadelphia

First Received on December 27, 2007. Last Updated on June 29, 2011 History of Changes

Results First Received: November 15, 2010

Study Type:	Interventional
Study Design:	Allocation: Randomized; Endpoint Classification: Safety/Efficacy Study; Intervention Model: Parallel Assignment; Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor); Primary Purpose: Basic Science
Condition:	Cystic Fibrosis
Interventions:	Drug: Sodium 4-Phenylbutyrate Drug: Genistein (Unconjugated Isoflavones 100) Drug: Placebo

Participant Flow

Hide Participant Flow

Recruitment Details

Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
This study was terminated by the PI before completing enrollment

Pre-Assignment Details

Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups

	Description
Phenylbutyrate or Placebo	Subjects will be randomized to receive either the Phenylbutyrate or placebo tablets for 4 days. PLEASE NOTE: AT THE TIME OF TERMINATION BY PI, THE STUDY WAS NOT UNBLINDED SO IT IS NOT KNOWN WHICH PARTICIPANTS WERE ASSIGNED TO WHICH GROUP. Every participant will receive Genistein during the Nasal Potential Difference (NPD).

Description

Phenylbutyrate or Placebo

Subjects will be randomized to receive either the Phenylbutyrate or placebo tablets for 4 days.

PLEASE NOTE: AT THE TIME OF TERMINATION BY PI, THE STUDY WAS NOT UNBLINDED SO IT IS NOT KNOWN WHICH PARTICIPANTS WERE ASSIGNED TO WHICH GROUP.

Every participant will receive Genistein during the Nasal Potential Difference (NPD).

Participant Flow: Overall Study

	Phenylbutyrate or Placebo
STARTED	9 ^[1]
COMPLETED	7
NOT COMPLETED	2
Study terminated before completed	2

[1]	STUDY NOT UNBLINDED AT TIME OF TERMINATION; THEREFORE NUMBER ASSIGNED TO EACH GROUP IS NOT KNOWN.

Baseline Characteristics

Hide Baseline Characteristics

Reporting Groups

	Description
Phenylbutyrate or Placebo	Subjects will be randomized to receive either the Phenylbutyrate or placebo tablets for 4 days. PLEASE NOTE: AT THE TIME OF TERMINATION BY PI, THE STUDY WAS NOT UNBLINDED SO IT IS NOT KNOWN WHICH PARTICIPANTS WERE ASSIGNED TO WHICH GROUP. Every participant will receive Genistein during the Nasal Potential Difference (NPD).

Description

Phenylbutyrate or Placebo

Subjects will be randomized to receive either the Phenylbutyrate or placebo tablets for 4 days.

PLEASE NOTE: AT THE TIME OF TERMINATION BY PI, THE STUDY WAS NOT UNBLINDED SO IT IS NOT KNOWN WHICH PARTICIPANTS WERE ASSIGNED TO WHICH GROUP.

Every participant will receive Genistein during the Nasal Potential Difference (NPD).

Baseline Measures

	Phenylbutyrate or Placebo
Number of Participants [units: participants]	9
Age [units: participants]
<=18 years	0
Between 18 and 65 years	9
>=65 years	0
Gender [units: participants]
Female	6
Male	3
Region of Enrollment [units: participants]
United States	9

Outcome Measures

Show All Outcome Measures

1. Primary:

Change in Voltage (mVolt) in Nasal Epithelium [ Time Frame: Baseline and 2 weeks ]

Show Outcome Measure 1

2. Secondary:

Change in FEV1 (Forced Expiratory Volume in 1 Second) in Spirometry. [ Time Frame: baseline and 2 weeks ]

Show Outcome Measure 2

3. Secondary:

Change in FVC (Forced Vital Capacity)in Spirometry. [ Time Frame: baseline and 2 weeks ]

Show Outcome Measure 3

4. Secondary:

Number of Participants With Adverse Events [ Time Frame: up to 2 weeks ]

Show Outcome Measure 4

5. Secondary:

Number of Participants With Abnormal Laboratory Safety Tests [ Time Frame: up to 2 weeks ]

Show Outcome Measure 5

Serious Adverse Events

Show Serious Adverse Events

Other Adverse Events

Show Other Adverse Events

More Information

Hide More Information

Certain Agreements:

Principal Investigators are NOT employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No analysis was completed on data collected; More clinically efficacious compounds have been identified which suggested that completion of this study might not be as critical as when initially proposed; therefore, the study was terminated by PI.

Results Point of Contact:

Name/Title: Ronald Rubenstein, MD, PhD
Organization: The Children's Hospital of Philadelphia
phone: 1-215-590-1281
e-mail: rubensteinr@email.chop.edu

No publications provided

Responsible Party:	Ronald Rubenstein, M.D., PhD., The Children's Hospital of Philadelphia
ClinicalTrials.gov Identifier:	NCT00590538 History of Changes
Other Study ID Numbers:	2002-10-3023, RUBENS01A0
Study First Received:	December 27, 2007
Results First Received:	November 15, 2010
Last Updated:	June 29, 2011
Health Authority:	United States: Food and Drug Administration; United States: Institutional Review Board