CystinosisNCT01432561
Study in Healthy Adults to Determine the Effect That Food Has on the Absorption and Delivery of the Drug Cystagon™
A study testing Cysteamine bitartrate in people with Cystinosis and Nephropathic Cystinosis, run by University of California, San Diego at 1 site in United States.
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. Read more about this study
- Who is it for?
- Ages 18 years and olderHealthy volunteers can take part
- Conditions the study is for
- Cystinosis, Nephropathic Cystinosis
- What is being tested?
- Cysteamine bitartrate
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 1 siteUnited States
- Study dates
- Sep 2011to Dec 2011
- Phase
- Interventional
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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
Show all 22Show fewer
- 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.
- -
Trial locations and contacts
1 site in 1 country.
United States1 site
University of California, San Diego Center for Clinical Research Services (CCR)
La Jolla, California, 92093
About this study
Official title: Food-Effect on Bioavailability of Cystagon™ in Normal, Healthy Adults
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
- Being tested
- Cysteamine bitartrate
- Keywords
- Lysosomal Storage Diseases, Metabolic Diseases, Metabolism, Inborn Errors
- Medical subject headings
- Cystinosis, Lysosomal Storage Diseases, Metabolic Diseases, Metabolism, Inborn Errors
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Cysteamine bitartrateDrug
Also called Cystagon, Cysteamine
500 mg total, single dose taken orally on visits 2, 3 \& 4 which must occur within a 14 day period.
| Study group | Role | What they receive |
|---|---|---|
| Cysteamine bitartrate | Experimental | Cysteamine bitartrate, 500mg once a day, three days. Receives: Drug: Cysteamine bitartrate |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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.
Measured: 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
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Sep 2011 (estimated)
- Study startSep 2011
- Primary completionDec 2011
- Study completionDec 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for CystinosisStudies of the same condition, open and under way.
- More trials run by University of California, San DiegoThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of California, San Diego
- Sponsor type
- University, hospital or other
- Collaborators
- Raptor Pharmaceuticals Corp.
- Responsible party
- Principal investigator, Ranjan Dohil, University of California, San Diego
- Investigators
- Ranjan Dohil, M.D., Principal investigator, University of California, San Diego
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01432561
- Sponsor's protocol ID
- 111011
- Status last verified
- Sep 2013
- First submitted
- 9 Sep 2011
- Last updated
- 9 Oct 2013
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
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.
PubMed 12110740Gahl 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.
PubMed 3550461Markello 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.
PubMed 8455682Smolin 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.
PubMed 3393396
Study at a glance
From the registry record, last updated by the sponsor on 9 Oct 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Food-Effect on Bioavailability of Cystagon™ in Normal, Healthy Adults
- ClinicalTrials.gov ID
- NCT01432561
- Acronym / sponsor ID
- 111011
- Conditions
- Cystinosis, Nephropathic Cystinosis
- Keywords
- Lysosomal Storage Diseases, Metabolic Diseases, Metabolism, Inborn Errors
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 8 participants (actual)
- Lead sponsor
- University of California, San DiegoUniversity, hospital or other
- Collaborators
- Raptor Pharmaceuticals Corp.
- Study start
- Sep 2011
- Primary completion
- Dec 2011 (actual)
- Study completion
- Dec 2011 (actual)
- First posted
- 13 Sep 2011 (estimated)
- Results
- Posted 9 Oct 2013
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Cysteamine bitartrate | Experimental | Drug: Cysteamine bitartrateCysteamine bitartrate, 500mg once a day, three days. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (22 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Cysteamine bitartrateAlso: Cystagon, Cysteamine500 mg total, single dose taken orally on visits 2, 3 \& 4 which must occur within a 14 day period. | Drug | Cysteamine bitartrate |
Primary outcome
Cysteamine Absorption: Area Under the Plasma Concentration Curve (AUC)
Time frame: 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
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.
Peak Plasma Cysteamine Concentration (Cmax)
Time frame: 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
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.
Time to Peak Plasma Cysteamine Concentration (Tmax)
Time frame: 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
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.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Oct 2013.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 12110740Gahl WA, Thoene JG, Schneider JA. Cystinosis. N Engl J Med. 2002 Jul 11;347(2):111-21. doi: 10.1056/NEJMra020552. No abstract available.
- Background: PubMed 3550461Gahl 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.
- Background: PubMed 8455682Markello 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.
- Background: PubMed 3393396Smolin 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Ranjan Dohil, M.D. principal investigator
University of California, San Diego
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Cysteamine bitartrate in people with Cystinosis and Nephropathic Cystinosis, run by University of California, San Diego at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 22 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 9 Oct 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Yes. Results are posted in the registry (first posted 9 Oct 2013). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Cysteamine Absorption: Area Under the Plasma Concentration Curve (AUC) (time frame: 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). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01432561, which also lists 4 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Oct 2013.