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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)
Sponsor:
Collaborator:
Cystic Fibrosis Foundation
Information provided by:
Children's Hospital of Philadelphia
ClinicalTrials.gov Identifier:
NCT00590538
First received: December 27, 2007
Last updated: June 29, 2011
Last verified: June 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
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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).

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
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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).

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
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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
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  Other Adverse Events
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  More Information
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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