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

Cystinosis Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01432561
• Other study ID #: 111011
• Status: Completed
• Phase: N/A
• First received: September 9, 2011
• Last updated: September 23, 2013
• Start date: September 2011
• Est. completion date: December 2011

Study information

• Verified date: September 2013
• Source: University of California, San Diego
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Institutional Review Board
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 8
• Est. completion date: December 2011
• Est. primary completion date: December 2011
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: Both
• Age group: 18 Years and older

Eligibility

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.

Study Design

Endpoint Classification: Bio-availability Study, Intervention Model: Single Group Assignment, Masking: Open Label

Related Conditions & MeSH terms

• Cystinosis
• Fanconi Syndrome
• Nephropathic Cystinosis

Intervention

Drug:
• Cysteamine bitartrate
500 mg total, single dose taken orally on visits 2, 3 & 4 which must occur within a 14 day period.

Locations

Country	Name	City	State
United States	University of California, San Diego Center for Clinical Research Services (CCR)	La Jolla	California

Sponsors (2)

Lead Sponsor	Collaborator
University of California, San Diego	Raptor Pharmaceuticals Corp.

Country where clinical trial is conducted

United States, 

References & Publications (4)

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. — View Citation

Gahl WA, Thoene JG, Schneider JA. Cystinosis. N Engl J Med. 2002 Jul 11;347(2):111-21. Review. — View Citation

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. — View Citation

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. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
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.	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	No
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.	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	No
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.	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	No