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| Name | Class |
|---|---|
| Raptor Pharmaceuticals Corp. | INDUSTRY |
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In order to meet FDA standards of safety and efficacy reporting for most new drugs, food-effect bioavailability (the impact that the presence of food in the digestive tract has on the rate and extent at which a drug is absorbed into the bloodstream and delivered to the site of action) must be collected. Cystagon™ is an FDA approved drug for the treatment of the rare disease cystinosis that became available in 1994, but there is inadequate knowledge of the food-effect on this drug's bioavailability. This study aims to investigate how food affects the absorption of Cystagon™ into the bloodstream of normal healthy adults.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Cysteamine bitartrate | Experimental | Cysteamine bitartrate, 500mg once a day, three days. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Cysteamine bitartrate | Drug | 500 mg total, single dose taken orally on visits 2, 3 & 4 which must occur within a 14 day period. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Cysteamine Absorption: Area Under the Plasma Concentration Curve (AUC) | 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. | 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 |
| Peak Plasma Cysteamine Concentration (Cmax) | 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. | 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 |
| Time to Peak Plasma Cysteamine Concentration (Tmax) | 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. | 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 |
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Inclusion Criteria:
Exclusion Criteria:
Evidence of Helicobacter pylori infection, presently, or within the last year.
Subjects with known hypersensitivity to cysteamine.
History, currently or within the past 3 months, of the following conditions:
Subjects whom may be pregnant or have health issues that make it unsafe for them participate, or whose concomitant medical problems preclude them from committing to the study schedule.
Use of an investigational drug within 30 days (or 90 days for biologics) prior to dosing.
Use of prescription medication within 14 days prior to the first dosing;
Use over-the-counter products including natural health products (e.g. food supplements and herbal supplements) within 7 days prior to the first dosing.
Donation of plasma within 7 days prior to dosing. Donation or loss of blood (excluding volume drawn at screening) of 50 mL to 499 mL of blood within 30 days, or more than 499 mL within 56 days prior to dosing.
Hemoglobin <13.5 g/dL (males) and <12.0 g/dL (females) and hematocrit <41.0% (males) and <36.0% (females) at screening.
Breast-feeding subject.
Immunization with a live attenuated vaccine 1 month prior to dosing or planned vaccination during the course of the study.
Presence of fever (body temperature >37.6°C) (e.g. a fever associated with a symptomatic viral or bacterial infection) within 2 weeks prior to dosing.
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| Name | Affiliation | Role |
|---|---|---|
| Ranjan Dohil, M.D. | University of California, San Diego | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of California, San Diego Center for Clinical Research Services (CCR) | La Jolla | California | 92093 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 12110740 | Background | Gahl WA, Thoene JG, Schneider JA. Cystinosis. N Engl J Med. 2002 Jul 11;347(2):111-21. doi: 10.1056/NEJMra020552. No abstract available. | |
| 3550461 | Background | Gahl WA, Reed GF, Thoene JG, Schulman JD, Rizzo WB, Jonas AJ, Denman DW, Schlesselman JJ, Corden BJ, Schneider JA. Cysteamine therapy for children with nephropathic cystinosis. N Engl J Med. 1987 Apr 16;316(16):971-7. doi: 10.1056/NEJM198704163161602. |
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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.
Recruitment period: 09/19/2011 - 11/21/2012 Recruitment Locations: medical centers
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| ID | Title | Description |
|---|---|---|
| FG000 | 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. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | 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. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Cysteamine Absorption: Area Under the Plasma Concentration Curve (AUC) | 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. | Posted | Mean | Standard Deviation | min*uM | 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 |
|
|
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 .
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | 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. |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Nausea | Gastrointestinal disorders | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. Ranjan Dohil, P.I. | University of California San Diego | 619-471-9554 | rdohil@ucsd.edu |
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| ID | Term |
|---|---|
| D003554 | Cystinosis |
| D016464 | Lysosomal Storage Diseases |
| D008659 | Metabolic Diseases |
| D008661 | Metabolism, Inborn Errors |
| ID | Term |
|---|---|
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D009750 | Nutritional and Metabolic Diseases |
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| ID | Term |
|---|---|
| D003543 | Cysteamine |
| ID | Term |
|---|---|
| D008624 | Mercaptoethylamines |
| D005021 | Ethylamines |
| D000588 | Amines |
| D009930 | Organic Chemicals |
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| 8455682 | Background | Markello TC, Bernardini IM, Gahl WA. Improved renal function in children with cystinosis treated with cysteamine. N Engl J Med. 1993 Apr 22;328(16):1157-62. doi: 10.1056/NEJM199304223281604. |
| 3393396 | Background | Smolin LA, Clark KF, Thoene JG, Gahl WA, Schneider JA. A comparison of the effectiveness of cysteamine and phosphocysteamine in elevating plasma cysteamine concentration and decreasing leukocyte free cystine in nephropathic cystinosis. Pediatr Res. 1988 Jun;23(6):616-20. doi: 10.1203/00006450-198806000-00018. |
| Participants |
|
| Age Continuous | Mean | Standard Deviation | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| Participants |
|
|
|
| Primary | Peak Plasma Cysteamine Concentration (Cmax) | 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. | Posted | Mean | Standard Deviation | uM | 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 |
|
|
|
|
| Primary | Time to Peak Plasma Cysteamine Concentration (Tmax) | 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. | Posted | Mean | Standard Deviation | minutes | 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 |
|
|
|
|
| 0 |
| 8 |
| 3 |
| 8 |
| Abdominal pain | Gastrointestinal disorders | Systematic Assessment |
|
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| D013438 |
| Sulfhydryl Compounds |
| D013457 | Sulfur Compounds |
| Title | Measurements |
|---|---|
|
| .036 |
| 95 |
| No |
| Superiority or Other |
| Title | Measurements |
|---|---|
|
| .05 |
| 95 |
| No |
| Superiority or Other |