Cystinosis Cystinosis
Conditions
Interventions
Single-dose administration of Nicystagon capsules (cysteamine bitartrate), single-dose administration of enteric-coated Nicystagon capsules, and single-dose co-administration of both formulations.
Cystinosis
Sponsors
Okamura Tadashi
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1.Japanese who are 18 years or older at the time of obtaining consent. 2. Participants with a confirmed diagnosis of cystinosis. 3. Participants receiving cysteamine bitartrate (Nicystagon) therapy with a stable dose and regimen for at least three months. 4. Participants who have provided written informed consent.
Exclusion criteria
Exclusion criteria: 1.Participants with gastrointestinal conditions that may affect drug absorption,including a history of gastrointestinal surgery, severe malabsorption syndrome,or active gastric or duodenal ulcers. 2.Participants with severe complications, such as severe liver dysfunction or poorly controlled heart disease, for whom participation in the study is judged to pose safety risks. 3.Participants for whom it is difficult to secure peripheral venous access for frequent blood sampling. 4.Any other participants deemed inappropriate for participation in the study by the principal investigator.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Leukocyte cystine concentration at 5 hours after administration | — |
Secondary
| Measure | Time frame |
|---|---|
| -Pharmacokinetic parameters of plasma cysteamine (Cmax, Tmax, and AUC0-6h). -Difference in the severity of nausea (with or without vomiting) during the post-nisistagon administration period and the post-study drug administration period. -Leukocyte cystine concentration after administration of the study drug. | — |
Contacts
Public ContactDaisuke Katagiri
National Center for Global Health and Medicine, Japan Institute for Health Security (JIHS)
Outcome results
None listed