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

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ClinicalTrials.gov Identifier: NCT01432561
Recruitment Status : Completed
First Posted : September 13, 2011
Results First Posted : October 9, 2013
Last Update Posted : October 9, 2013
Sponsor:
Collaborator:
Raptor Pharmaceuticals Corp.
Information provided by (Responsible Party):
Ranjan Dohil, University of California, San Diego

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label)
Conditions Cystinosis
Nephropathic Cystinosis
Intervention Drug: Cysteamine bitartrate
Enrollment 8

Recruitment Details Recruitment period: 09/19/2011 - 11/21/2012 Recruitment Locations: medical centers
Pre-assignment Details 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.
Arm/Group Title Cysteamine Bitartrate
Hide Arm/Group Description

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.

Period Title: Overall Study
Started 8
Completed 8
Not Completed 0
Arm/Group Title Cysteamine Bitartrate
Hide Arm/Group Description

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 Number of Baseline Participants 8
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 8 participants
<=18 years
0
   0.0%
Between 18 and 65 years
8
 100.0%
>=65 years
0
   0.0%
Age Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 8 participants
29.9  (8.5)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 8 participants
Female
3
  37.5%
Male
5
  62.5%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 8 participants
8
1.Primary Outcome
Title Cysteamine Absorption: Area Under the Plasma Concentration Curve (AUC)
Hide Description 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.
Time Frame 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Cysteamine Bitartrate
Hide Arm/Group Description:
Cysteamine bitartrate : 500 mg total, single dose taken orally on visits 2, 3 & 4
Overall Number of Participants Analyzed 8
Mean (Standard Deviation)
Unit of Measure: min*uM
Fasted 3618  (372)
Fed High-Fat/Calorie 2799  (405)
Fed High-Protein 2457  (353)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Cysteamine Bitartrate
Comments High-fat/calorie compared to fasted condition
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value .04
Comments [Not Specified]
Method ANOVA
Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Cysteamine Bitartrate
Comments High-protein compared to fasted condition
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value .005
Comments [Not Specified]
Method ANOVA
Comments [Not Specified]
2.Primary Outcome
Title Peak Plasma Cysteamine Concentration (Cmax)
Hide Description 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.
Time Frame 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Cysteamine Bitartrate
Hide Arm/Group Description:
Cysteamine bitartrate : 500 mg total, single dose taken orally on visits 2, 3 & 4
Overall Number of Participants Analyzed 8
Mean (Standard Deviation)
Unit of Measure: uM
Fasted 26.3  (3.5)
Fed High-Fat/Calorie 22.4  (5.6)
Fed High-Protein 17.2  (2.6)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Cysteamine Bitartrate
Comments High-fat/calorie compared to fasted condition
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value .16
Comments [Not Specified]
Method ANOVA
Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Cysteamine Bitartrate
Comments High-protein compared to fasted condition
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value .036
Comments [Not Specified]
Method ANOVA
Comments [Not Specified]
3.Primary Outcome
Title Time to Peak Plasma Cysteamine Concentration (Tmax)
Hide Description 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.
Time Frame 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Cysteamine Bitartrate
Hide Arm/Group Description:
Cysteamine bitartrate : 500 mg total, single dose taken orally on visits 2, 3 & 4
Overall Number of Participants Analyzed 8
Mean (Standard Deviation)
Unit of Measure: minutes
Fasted 71.2  (12.9)
Fed High-Fat/Calorie 106.9  (28.8)
Fed High-Protein 120  (23.4)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Cysteamine Bitartrate
Comments Fed high-fat/calorie compared to fasted condition.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value .30
Comments [Not Specified]
Method Non-parametric Hodges-Lehmann method
Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Cysteamine Bitartrate
Comments High-protein compared to fasted condition
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value .05
Comments [Not Specified]
Method Non-parametric Hodges-Lehmann method
Comments [Not Specified]
Time Frame 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.
Adverse Event Reporting Description

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 .

 
Arm/Group Title Cysteamine Bitartrate
Hide Arm/Group Description

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.

All-Cause Mortality
Cysteamine Bitartrate
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Cysteamine Bitartrate
Affected / at Risk (%) # Events
Total   0/8 (0.00%)    
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Cysteamine Bitartrate
Affected / at Risk (%) # Events
Total   3/8 (37.50%)    
Gastrointestinal disorders   
Nausea   3/8 (37.50%)  3
Abdominal pain   1/8 (12.50%)  1
Indicates events were collected by systematic assessment
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
Name/Title: Dr. Ranjan Dohil, P.I.
Organization: University of California San Diego
Phone: 619-471-9554
Responsible Party: Ranjan Dohil, University of California, San Diego
ClinicalTrials.gov Identifier: NCT01432561     History of Changes
Other Study ID Numbers: 111011
First Submitted: September 9, 2011
First Posted: September 13, 2011
Results First Submitted: July 3, 2013
Results First Posted: October 9, 2013
Last Update Posted: October 9, 2013