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

Study Overview
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Brief Summary
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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.

Official Title
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Food-Effect on Bioavailability of Cystagon™ in Normal, Healthy Adults

Conditions
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Cystinosis, Nephropathic Cystinosis

Intervention / Treatment
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* Drug: Cysteamine bitartrate


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Male or female, smoker (no more than 25 cigarettes daily) or non-smoker, 18 years of age and older, with BMI > 18 and < 30.0. Females of childbearing potential who are sexually active must be willing to use two forms of contraceptive methods throughout the study and for 14days after the last study drug administration. Minimum weight of 50 kg. Good health, defined as not having history of any chronic illness and not requiring any regular medication/therapy. Must swallow tablets on a regular basis. 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: Pancreatitis Inflammatory bowel disease Malabsorption Severe liver disease Unstable heart disease, e.g., myocardial infarction, heart failure, arrhythmias. Unstable diabetes mellitus Any bleeding disorder. Zollinger-Ellison syndrome Malignant disease 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. -

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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Accepts Healthy Volunteers

Study Plan
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How is the study designed?
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Design Details

Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Cysteamine bitartrate<br>Cysteamine bitartrate, 500mg once a day, three days. | Drug: Cysteamine bitartrate<br>* 500 mg total, single dose taken orally on visits 2, 3 & 4 which must occur within a 14 day period.<br>* Other names: Cysteamine;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | 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 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Lysosomal Storage Diseases, Metabolic Diseases, Metabolism, Inborn Errors