Trial Outcomes & Findings for Study in Healthy Adults to Determine the Effect That Food Has on the Absorption and Delivery of the Drug Cystagon™ (NCT NCT01432561)

Results Overview

Subjects were randomized to one of two possible treatment sequences using block randomization: Sequence 1 - fasted, high-fat, high-protein or Sequence 2 - high-protein, high-fat, fasted. Sequence assignment determined the treatment condition corresponding to Period I, II \& III visits.

Recruitment status

COMPLETED

Study phase

NA

Target enrollment

8 participants

Primary outcome timeframe

0, 15, 30, 45, 60, 75, 90, 105, 120, 135, 150, 165, 180 minutes, and 3.5, 4, 4.5, 5, 6 hours post-dose

Results posted on

2013-10-09

Participant Flow

Recruitment period: 09/19/2011 - 11/21/2012 Recruitment Locations: medical centers

Screening for liver function and H pylori infection and brief medical history in order to determine if all inclusion and exclusion criteria were met. One enrollee met all inclusion criteria but clinical lab test revealed presence of H Pylori, an exclusion factor. Two enrollees withdrew from the trial prior to assignment due to scheduling conflicts.

Participant milestones

Participant milestones
Measure	Cysteamine Bitartrate Cysteamine bitartrate, 500mg once a day, three days. Cysteamine bitartrate : 500 mg total, single dose taken orally on visits 2, 3 \& 4 which must occur within a 14 day period.
Overall Study STARTED	8
Overall Study COMPLETED	8
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Study in Healthy Adults to Determine the Effect That Food Has on the Absorption and Delivery of the Drug Cystagon™

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Cysteamine Bitartrate n=8 Participants Cysteamine bitartrate, 500mg once a day, three days. Cysteamine bitartrate : 500 mg total, single dose taken orally on visits 2, 3 \& 4 which must occur within a 14 day period.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	8 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age Continuous	29.9 years STANDARD_DEVIATION 8.5 • n=5 Participants
Sex: Female, Male Female	3 Participants n=5 Participants
Sex: Female, Male Male	5 Participants n=5 Participants
Region of Enrollment United States	8 participants n=5 Participants

PRIMARY outcome

Timeframe: 0, 15, 30, 45, 60, 75, 90, 105, 120, 135, 150, 165, 180 minutes, and 3.5, 4, 4.5, 5, 6 hours post-dose

Subjects were randomized to one of two possible treatment sequences using block randomization: Sequence 1 - fasted, high-fat, high-protein or Sequence 2 - high-protein, high-fat, fasted. Sequence assignment determined the treatment condition corresponding to Period I, II \& III visits.

Outcome measures

Outcome measures
Measure	Cysteamine Bitartrate n=8 Participants Cysteamine bitartrate : 500 mg total, single dose taken orally on visits 2, 3 \& 4
Cysteamine Absorption: Area Under the Plasma Concentration Curve (AUC) Fasted	3618 min*uM Standard Deviation 372
Cysteamine Absorption: Area Under the Plasma Concentration Curve (AUC) Fed High-Fat/Calorie	2799 min*uM Standard Deviation 405
Cysteamine Absorption: Area Under the Plasma Concentration Curve (AUC) Fed High-Protein	2457 min*uM Standard Deviation 353

PRIMARY outcome

Timeframe: 0, 15, 30, 45, 60, 75, 90, 105, 120, 135, 150, 165, 180 minutes, and 3.5, 4, 4.5, 5, 6 hours post-dose

Subjects were randomized to one of two possible treatment sequences using block randomization: Sequence 1 - fasted, high-fat, high-protein or Sequence 2 - high-protein, high-fat, fasted. Sequence assignment determined the treatment condition corresponding to Period I, II \& III visits.

Outcome measures

Outcome measures
Measure	Cysteamine Bitartrate n=8 Participants Cysteamine bitartrate : 500 mg total, single dose taken orally on visits 2, 3 \& 4
Peak Plasma Cysteamine Concentration (Cmax) Fasted	26.3 uM Standard Deviation 3.5
Peak Plasma Cysteamine Concentration (Cmax) Fed High-Fat/Calorie	22.4 uM Standard Deviation 5.6
Peak Plasma Cysteamine Concentration (Cmax) Fed High-Protein	17.2 uM Standard Deviation 2.6

PRIMARY outcome

Timeframe: 0, 15, 30, 45, 60, 75, 90, 105, 120, 135, 150, 165, 180 minutes, and 3.5, 4, 4.5, 5, 6 hours post-dose

Subjects were randomized to one of two possible treatment sequences using block randomization: Sequence 1 - fasted, high-fat, high-protein or Sequence 2 - high-protein, high-fat, fasted. Sequence assignment determined the treatment condition corresponding to Period I, II \& III visits.

Outcome measures

Outcome measures
Measure	Cysteamine Bitartrate n=8 Participants Cysteamine bitartrate : 500 mg total, single dose taken orally on visits 2, 3 \& 4
Time to Peak Plasma Cysteamine Concentration (Tmax) Fasted	71.2 minutes Standard Deviation 12.9
Time to Peak Plasma Cysteamine Concentration (Tmax) Fed High-Fat/Calorie	106.9 minutes Standard Deviation 28.8
Time to Peak Plasma Cysteamine Concentration (Tmax) Fed High-Protein	120 minutes Standard Deviation 23.4

Adverse Events

Cysteamine Bitartrate

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Cysteamine Bitartrate n=8 participants at risk Cysteamine bitartrate, 500mg once a day, three days. Cysteamine bitartrate : 500 mg total, single dose taken orally on visits 2, 3 \& 4 which must occur within a 14 day period.
Gastrointestinal disorders Nausea	37.5% 3/8 • Number of events 3 • Adverse event data collected beginning date of enrollment and continuing to study withdrawal/termination or up to 7 days after last study visit, as applicable. Total duration of data collection varied among the subjects based on clinic scheduling. At study visits, subjects queried at specified intervals before and after drug administration (t=0, 30, 60, 90, 120 and 180 minutes) via a brief survey. Spontaneous A.E. observed and noted also. Between study visits, A.E.'s as reported by participants at the beginning of each study visit and 7 days after the final study visit were noted .
Gastrointestinal disorders Abdominal pain	12.5% 1/8 • Number of events 1 • Adverse event data collected beginning date of enrollment and continuing to study withdrawal/termination or up to 7 days after last study visit, as applicable. Total duration of data collection varied among the subjects based on clinic scheduling. At study visits, subjects queried at specified intervals before and after drug administration (t=0, 30, 60, 90, 120 and 180 minutes) via a brief survey. Spontaneous A.E. observed and noted also. Between study visits, A.E.'s as reported by participants at the beginning of each study visit and 7 days after the final study visit were noted .

Additional Information

Dr. Ranjan Dohil, P.I.

University of California San Diego

Phone: 619-471-9554

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place