Trial Outcomes & Findings for Phenylbutyrate/Genistein Duotherapy in Delta F508-Heterozygotes (for Cystic Fibrosis) (NCT NCT00590538)

Results Overview

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.

Recruitment status

TERMINATED

Study phase

PHASE1/PHASE2

Target enrollment

9 participants

Primary outcome timeframe

Baseline and 2 weeks

Results posted on

2011-06-30

Participant Flow

This study was terminated by the PI before completing enrollment

Participant milestones

Participant milestones
Measure	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).
Overall Study STARTED	9
Overall Study COMPLETED	7
Overall Study NOT COMPLETED	2

Reasons for withdrawal

Reasons for withdrawal
Measure	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).
Overall Study Study terminated before completed	2

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Phenylbutyrate or Placebo n=9 Participants 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).
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	9 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	6 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants
Region of Enrollment United States	9 participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline and 2 weeks

Population: No analysis was completed on data collected; AND study was never unblinded therefore details not available.

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.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: baseline and 2 weeks

Population: No analysis was completed on data collected.

Outcome measure will be obtained from standard Pulmonary Function testing.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: baseline and 2 weeks

Population: No analysis was completed on data collected.

Outcome measure will be obtained from standard Pulmonary Function testing.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: up to 2 weeks

Adverse Events will be assessed and outcome measure obtained by completion of Interval history, physical and mental status examinations of every participant.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: up to 2 weeks

Population: 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.

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).

Outcome measures

Outcome data not reported

Adverse Events

Phenylbutyrate or Placebo

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Ronald Rubenstein, MD, PhD

The Children's Hospital of Philadelphia

Phone: 1-215-590-1281

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place