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

NCT01432561 | Completed Results

• 1 other identifier: 111011
• Study Type: interventional
• Target: 8
• Locations: 1 country
• Sites: 1

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

Outcome Measures

Primary Outcomes (3)

• 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

Study Arms (1)

Cysteamine bitartrate

EXPERIMENTAL

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

Drug: Cysteamine bitartrate

Interventions

Cysteamine bitartrate DRUG

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

Also known as: Cystagon, Cysteamine

Cysteamine bitartrate

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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;

Sponsors & Collaborators

• University of California, San Diego: lead
• Raptor Pharmaceuticals Corp.: collaborator

Study Sites (1)

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

La Jolla, California, 92093, United States

Location

Related Publications (4)

• 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.

  PMID: 12110740 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.

  PMID: 3550461 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.

  PMID: 8455682 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.

  PMID: 3393396 BACKGROUND

MeSH Terms

Conditions

Cystinosis; Lysosomal Storage Diseases; Metabolic Diseases; Metabolism, Inborn Errors

Interventions

Cysteamine

Condition Hierarchy (Ancestors)

Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Mercaptoethylamines; Ethylamines; Amines; Organic Chemicals; Sulfhydryl Compounds; Sulfur Compounds

Results Point of Contact

• Title: Dr. Ranjan Dohil, P.I.
• Organization: University of California San Diego

rdohil@ucsd.edu

619-471-9554

Study Officials

• Ranjan Dohil, M.D.

  University of California, San Diego

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: September 9, 2011
• First Posted: September 13, 2011
• Study Start: September 1, 2011
• Primary Completion: December 1, 2011
• Study Completion: December 1, 2011
• Last Updated: October 9, 2013
• Results First Posted: October 9, 2013

Record last verified: 2013-09

Locations

US (1)