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

NCT ID: NCT01432561

Last Updated: 2013-10-09

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: NA
• Total Enrollment: 8 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2011-09-30
• Study Completion Date: 2011-12-31

Brief Summary

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.

Conditions

Cystinosis; Nephropathic Cystinosis

Keywords

Lysosomal Storage Diseases; Metabolic Diseases; Metabolism, Inborn Errors

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Blinding Strategy: NONE

Study Groups

Cysteamine bitartrate

Cysteamine bitartrate, 500mg once a day, three days.

Group Type: EXPERIMENTAL

Cysteamine bitartrate

Intervention Type: DRUG

500 mg total, single dose taken orally on visits 2, 3 \& 4 which must occur within a 14 day period.

Interventions

Cysteamine bitartrate

500 mg total, single dose taken orally on visits 2, 3 \& 4 which must occur within a 14 day period.

Intervention Type: DRUG

Other Intervention Names

Cystagon; Cysteamine

Eligibility Criteria

Inclusion Criteria

1. 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.

2. 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.

3. Minimum weight of 50 kg.

4. Good health, defined as not having history of any chronic illness and not requiring any regular medication/therapy.

5. Must swallow tablets on a regular basis.

Exclusion Criteria

1. Evidence of Helicobacter pylori infection, presently, or within the last year.

2. Subjects with known hypersensitivity to cysteamine.

3. 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

4. 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.

5. Use of an investigational drug within 30 days (or 90 days for biologics) prior to dosing.

6. Use of prescription medication within 14 days prior to the first dosing;

7. Use over-the-counter products including natural health products (e.g. food supplements and herbal supplements) within 7 days prior to the first dosing.

8. 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.

9. Hemoglobin <13.5 g/dL (males) and <12.0 g/dL (females) and hematocrit <41.0% (males) and <36.0% (females) at screening.

10. Breast-feeding subject.

11. Immunization with a live attenuated vaccine 1 month prior to dosing or planned vaccination during the course of the study.

12. 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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• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: Yes

Sponsors

Raptor Pharmaceuticals Corp.

INDUSTRY

Sponsor Role: collaborator

University of California, San Diego

OTHER

Sponsor Role: lead

Responsible Party

Ranjan Dohil

Principal Investigator

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Ranjan Dohil, M.D.

Role: PRINCIPAL_INVESTIGATOR

University of California, San Diego

Locations

University of California, San Diego Center for Clinical Research Services (CCR)

La Jolla, California, United States

Countries

United States

References

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.

Reference Type: BACKGROUND

PMID: 12110740 (View on PubMed)

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.

Reference Type: BACKGROUND

PMID: 3550461 (View on PubMed)

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.

Reference Type: BACKGROUND

PMID: 8455682 (View on PubMed)

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.

Reference Type: BACKGROUND

PMID: 3393396 (View on PubMed)

Other Identifiers

111011

Identifier Type: -

Identifier Source: org_study_id