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

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ClinicalTrials.gov Identifier: NCT00590538
Recruitment Status : Terminated (12/15/2008 Voluntarily placed on inactive status-requested by the PI)
First Posted : January 10, 2008
Results First Posted : June 30, 2011
Last Update Posted : June 30, 2011
Sponsor:
Collaborator:
Cystic Fibrosis Foundation
Information provided by:
Children's Hospital of Philadelphia

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Basic Science
Condition Cystic Fibrosis
Interventions Drug: Sodium 4-Phenylbutyrate
Drug: Genistein (Unconjugated Isoflavones 100)
Drug: Placebo
Enrollment 9
Recruitment Details This study was terminated by the PI before completing enrollment
Pre-assignment Details  
Arm/Group Title Phenylbutyrate or Placebo
Hide Arm/Group Description

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).

Period Title: Overall Study
Started 9 [1]
Completed 7
Not Completed 2
Reason Not Completed
Study terminated before completed             2
[1]
STUDY NOT UNBLINDED AT TIME OF TERMINATION; THEREFORE NUMBER ASSIGNED TO EACH GROUP IS NOT KNOWN.
Arm/Group Title Phenylbutyrate or Placebo
Hide Arm/Group Description

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).

Overall Number of Baseline Participants 9
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 9 participants
<=18 years
0
   0.0%
Between 18 and 65 years
9
 100.0%
>=65 years
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 9 participants
Female
6
  66.7%
Male
3
  33.3%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 9 participants
9
1.Primary Outcome
Title Change in Voltage (mVolt) in Nasal Epithelium
Hide Description

The basis of analysis for the primary outcome measure will be the comparison of data from both the standard CF Nasal Potential Difference (NPD) Protocol compared to a modified NPD protocol including the perfusion of Genistein.

The NPD response will be compared from baseline to after study drug. NPD responses will then be compared between the Phenylbutrate group and the placebo group.

Time Frame Baseline and 2 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
No analysis was completed on data collected; AND study was never unblinded therefore details not available.
Arm/Group Title Phenylbutyrate or Placebo
Hide Arm/Group Description:

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).

Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
2.Secondary Outcome
Title Change in FEV1 (Forced Expiratory Volume in 1 Second) in Spirometry.
Hide Description Outcome measure will be obtained from standard Pulmonary Function testing.
Time Frame baseline and 2 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
No analysis was completed on data collected.
Arm/Group Title Phenylbutyrate or Placebo
Hide Arm/Group Description:

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).

Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
3.Secondary Outcome
Title Change in FVC (Forced Vital Capacity)in Spirometry.
Hide Description Outcome measure will be obtained from standard Pulmonary Function testing.
Time Frame baseline and 2 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
No analysis was completed on data collected.
Arm/Group Title Phenylbutyrate or Placebo
Hide Arm/Group Description:

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).

Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
4.Secondary Outcome
Title Number of Participants With Adverse Events
Hide Description Adverse Events will be assessed and outcome measure obtained by completion of Interval history, physical and mental status examinations of every participant.
Time Frame up to 2 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Phenylbutyrate or Placebo
Hide Arm/Group Description:

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).

Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
5.Secondary Outcome
Title Number of Participants With Abnormal Laboratory Safety Tests
Hide Description Outcome measure will be obtained by completion of routine metabolic and hematological laboratory parameters for every participant. Metabolic testing willl include a CMP (comprehensive metabolic panel, ALT (alanine aminotransferase test), GGT (gamma-glutamyl transpeptidase), and Uric Acid; Hematological testing will include a complete blood count (CBC), and partial thromboplastin (PT/PTT).
Time Frame up to 2 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Phenylbutyrate or Placebo
Hide Arm/Group Description:

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).

Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Phenylbutyrate or Placebo
Hide Arm/Group Description

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).

All-Cause Mortality
Phenylbutyrate or Placebo
Affected / at Risk (%)
Total   --/-- 
Hide Serious Adverse Events
Phenylbutyrate or Placebo
Affected / at Risk (%)
Total   0/9 (0.00%) 
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Phenylbutyrate or Placebo
Affected / at Risk (%)
Total   0/9 (0.00%) 
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.
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.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Ronald Rubenstein, MD, PhD
Organization: The Children's Hospital of Philadelphia
Phone: 1-215-590-1281
EMail: rubensteinr@email.chop.edu
Layout table for additonal information
Responsible Party: Ronald Rubenstein, M.D., PhD., The Children's Hospital of Philadelphia
ClinicalTrials.gov Identifier: NCT00590538    
Other Study ID Numbers: 2002-10-3023
RUBENS01A0 ( Other Identifier: Children's Hospital of Philadelphia )
First Submitted: December 27, 2007
First Posted: January 10, 2008
Results First Submitted: November 15, 2010
Results First Posted: June 30, 2011
Last Update Posted: June 30, 2011