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Trial record 12 of 32 for:    CYSTEAMINE

Pilot Study of Safety, Tolerability, Pharmacokinetics/Pharmacodynamics of RP103 Compared to Cystagon® in Patients With Cystinosis

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ClinicalTrials.gov Identifier: NCT00872729
Recruitment Status : Completed
First Posted : March 31, 2009
Results First Posted : October 28, 2014
Last Update Posted : May 19, 2017
Sponsor:
Information provided by (Responsible Party):
Horizon Pharma USA, Inc.

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Cystinosis
Interventions Drug: Cystagon®
Drug: RP103
Enrollment 9
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Cystagon® and RP103
Hide Arm/Group Description Reference Product: Cystagon® (Cysteamine Bitartrate) Capsules,150 mg/50 mg; Test Product: RP103 (Cysteamine Bitartrate) Delayed-release Capsules,75 mg; Single-dose, open-label, nonrandomized, 2-period, crossover study of cysteamine bitartrate delayed-release capsules (RP103) and Cystagon®. Subjects were enrolled sequentially and received Cystagon® first followed by RP103.
Period Title: Overall Study
Started 9
Completed 9
Not Completed 0
Arm/Group Title Cystagon® and RP103
Hide Arm/Group Description Reference Product: Cystagon® (Cysteamine Bitartrate) Capsules,150 mg/50 mg; Test Product: RP103 (Cysteamine Bitartrate) Delayed-release Capsules,75 mg.
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
8
  88.9%
Between 18 and 65 years
1
  11.1%
>=65 years
0
   0.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 9 participants
12.8  (4.76)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 9 participants
Female
2
  22.2%
Male
7
  77.8%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 9 participants
9
1.Primary Outcome
Title Plasma Pharmacokinetic Parameter: Cmax of Cysteamine
Hide Description [Not Specified]
Time Frame 12 hours post RP103 dosing and 7 hours post 1st Cystagon® dosing
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Cystagon® RP103
Hide Arm/Group Description:
Reference Product: Cystagon® (Cysteamine Bitartrate) Capsules, 150 mg/50 mg.
Test Product: RP103 (Cysteamine Bitartrate) Delayed-release Capsules, 75 mg.
Overall Number of Participants Analyzed 9 9
Geometric Mean (Geometric Coefficient of Variation)
Unit of Measure: umol/L
42.02
(51%)
33.06
(55%)
2.Primary Outcome
Title Plasma Pharmacokinetic Parameter: Tmax of Cysteamine
Hide Description [Not Specified]
Time Frame 12 hours post RP103 dosing and 7 hours post 1st Cystagon® dosing
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Cystagon® RP103
Hide Arm/Group Description:
Reference Product: Cystagon® (Cysteamine Bitartrate) Capsules, 150 mg/50 mg.
Test Product: RP103 (Cysteamine Bitartrate) Delayed-release Capsules, 75 mg.
Overall Number of Participants Analyzed 9 9
Median (Full Range)
Unit of Measure: hour
1.00
(0.50 to 2.00)
3.00
(0.00 to 5.13)
3.Primary Outcome
Title Plasma Pharmacokinetic Parameter: AUC(0-t) of Cysteamine
Hide Description t = 6 for Cystagon and t = 12 for RP103. Cystagon is dosed every 6 hours and there is no measurement after 6 hours and up to 12 hours.
Time Frame 12 hours post RP103 dosing and 6 hours post 1st Cystagon® dosing
Hide Outcome Measure Data
Hide Analysis Population Description
Subjects were enrolled sequentially according to the study design. A mixed-effects linear model was used to assess differences between the RP103 and Cystagon treatment groups. Sample size is based on feasibility rather than statistical considerations.
Arm/Group Title Cystagon® RP103
Hide Arm/Group Description:
Reference Product: Cystagon® (Cysteamine Bitartrate) Capsules, 150 mg/50 mg.
Test Product: RP103 (Cysteamine Bitartrate) Delayed-release Capsules, 75 mg.
Overall Number of Participants Analyzed 9 9
Geometric Mean (Geometric Coefficient of Variation)
Unit of Measure: umol•h/L
107.89
(58%)
119.17
(46%)
4.Primary Outcome
Title Pharmacodynamic Parameter: Changes of White Blood Cell (WBC) Cystine Level From Baseline
Hide Description

The pharmacodynamic (PD) parameter measures the changes of WBC cystine level from the baseline.

Cystine is a disulfide amino acid formed through oxidation of two molecules of cysteine; hence, cystine’s concentration is commonly given in half-cystine equivalents to avoid confusion.

The level of cystine in WBC/leukocytes is expressed in units of nmol half-cystine/mg protein (nmol ½ cystine/mg protein). Half-cystine is quantified by a reduction of cystine followed by an assay for cysteine, which is then normalized by the total cellular protein content within the sample using methods of such as Lowry assay, bicinchoninic acid assay, or Bradford.

Time Frame up to 12 hours post Cystagon® dosing and RP103 dosing
Hide Outcome Measure Data
Hide Analysis Population Description
Sample size is based on feasibility rather than statistical considerations. Analysis time differences (0-6 hours) is due to different absorption characteristics of Cysteamine between RP103 and Cystagon. Cystagon is dosed every 6 hours and there is no measurement after 6 hours and up to 12 hours.
Arm/Group Title Cystagon® (0.5 Hour) RP103 (0.5 Hour) Cystagon® (1 Hour) RP103 (1 Hour) Cystagon® (2 Hour) RP103 (2 Hour) Cystagon® (2.5 Hour) RP103 (2.5 Hour) Cystagon® (3 Hour) RP103 (3 Hour) Cystagon® (4 Hour) RP103 (4 Hour) Cystagon® (6 Hour) RP103 (6 Hour) Cystagon® (8 Hour) RP103 (8 Hour) Cystagon® (10 Hour) RP103 (10 Hour) Cystagon® (12 Hour) RP103 (12 Hour)
Hide Arm/Group Description:
Reference Product: Cystagon® (Cysteamine Bitartrate) Capsules, 150 mg/50 mg.
Test Product: RP103 (Cysteamine Bitartrate) Delayed-release Capsules, 75 mg.
Reference Product: Cystagon® (Cysteamine Bitartrate) Capsules, 150 mg/50 mg.
Test Product: RP103 (Cysteamine Bitartrate) Delayed-release Capsules, 75 mg.
Reference Product: Cystagon® (Cysteamine Bitartrate) Capsules, 150 mg/50 mg.
Test Product: RP103 (Cysteamine Bitartrate) Delayed-release Capsules, 75 mg.
Reference Product: Cystagon® (Cysteamine Bitartrate) Capsules, 150 mg/50 mg.
Test Product: RP103 (Cysteamine Bitartrate) Delayed-release Capsules, 75 mg.
Reference Product: Cystagon® (Cysteamine Bitartrate) Capsules, 150 mg/50 mg.
Test Product: RP103 (Cysteamine Bitartrate) Delayed-release Capsules, 75 mg.
Reference Product: Cystagon® (Cysteamine Bitartrate) Capsules, 150 mg/50 mg.
Test Product: RP103 (Cysteamine Bitartrate) Delayed-release Capsules, 75 mg.
Reference Product: Cystagon® (Cysteamine Bitartrate) Capsules, 150 mg/50 mg.
Test Product: RP103 (Cysteamine Bitartrate) Delayed-release Capsules, 75 mg.
Reference Product: Cystagon® (Cysteamine Bitartrate) Capsules, 150 mg/50 mg.
Test Product: RP103 (Cysteamine Bitartrate) Delayed-release Capsules, 75 mg.
Reference Product: Cystagon® (Cysteamine Bitartrate) Capsules, 150 mg/50 mg.
Test Product: RP103 (Cysteamine Bitartrate) Delayed-release Capsules, 75 mg.
Reference Product: Cystagon® (Cysteamine Bitartrate) Capsules, 150 mg/50 mg.
Test Product: RP103 (Cysteamine Bitartrate) Delayed-release Capsules, 75 mg.
Overall Number of Participants Analyzed 6 0 3 3 6 0 0 6 3 3 6 6 9 6 0 3 0 3 3 6
Mean (Standard Deviation)
Unit of Measure: nmol 1/2 cystine/mg protein
0.11  (0.24) -0.10  (0.02) 0.87  (0.33) -0.07  (0.28) 0.19  (0.48) -0.14  (0.06) 0.43  (0.70) -0.01  (0.15) 0.10  (0.50) 0.30  (0.34) 0.22  (0.41) 0.20  (0.31) 0.45  (0.36) 0.37  (0.18) 0.72  (0.47)
Time Frame After a dose of study drug (RP103 or Cystagon®) on or after study entry, for up to 10 days.
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Cystagon® RP103
Hide Arm/Group Description Reference Product: Cystagon® (Cysteamine Bitartrate) Capsules, 150 mg/50 mg; Test Product: RP103 (Cysteamine Bitartrate) Delayed-release Capsules, 75 mg
All-Cause Mortality
Cystagon® RP103
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Cystagon® RP103
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/9 (0.00%)      0/9 (0.00%)    
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Cystagon® RP103
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   8/9 (88.89%)      2/9 (22.22%)    
Gastrointestinal disorders     
Vomiting  1  4/9 (44.44%)  4 0/9 (0.00%)  0
Nausea  2  3/9 (33.33%)  3 0/9 (0.00%)  0
Abdominal pain  2  1/9 (11.11%)  1 0/9 (0.00%)  0
Breath odor  2  1/9 (11.11%)  1 0/9 (0.00%)  0
Constipation  2  1/9 (11.11%)  1 0/9 (0.00%)  0
Diarrhoea  2  1/9 (11.11%)  1 0/9 (0.00%)  0
General disorders     
Thirst  2  3/9 (33.33%)  4 2/9 (22.22%)  2
Injury, poisoning and procedural complications     
Contusion  2  1/9 (11.11%)  1 0/9 (0.00%)  0
Metabolism and nutrition disorders     
Anorexia  2  1/9 (11.11%)  1 0/9 (0.00%)  0
Hypokalaemia  2  1/9 (11.11%)  1 0/9 (0.00%)  0
Skin and subcutaneous tissue disorders     
Skin odour abnormal  2  1/9 (11.11%)  1 0/9 (0.00%)  0
Vascular disorders     
Pallor  2  1/9 (11.11%)  1 0/9 (0.00%)  0
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 11.1
2
Term from vocabulary, MedDRA (11.1)
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS 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.
Investigators may publish or disclose study data, with the restriction that sponsor may embargo such communications for a period up to 60 days from the time submitted to sponsor.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Evelyn Olson, Director
Organization: Horizon Orphan LLC
Phone: 224-383-3000
EMail: clinicaltrials@horizonpharma.com
Layout table for additonal information
Responsible Party: Horizon Pharma USA, Inc.
ClinicalTrials.gov Identifier: NCT00872729     History of Changes
Other Study ID Numbers: RP103-01
First Submitted: March 27, 2009
First Posted: March 31, 2009
Results First Submitted: October 5, 2012
Results First Posted: October 28, 2014
Last Update Posted: May 19, 2017