PK and PD Study of NPI-001 and Cysteamine Bitartrate (NCT05994534) | Clinical Trial Compass
Active — Not RecruitingPhase 1/2
PK and PD Study of NPI-001 and Cysteamine Bitartrate
Australia12 participantsStarted 2023-10-29
Plain-language summary
Safety, pharmacokinetics, and pharmacodynamics of NPI-001 oral solution in cystinosis patients compared with cysteamine.
Who can participate
Age range
10 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Males or females, any race, ≥ 10 years of age.
. Diagnosis of nephropathic cystinosis and able to cease cysteamine therapy for 2 days.
. Females will be nonpregnant and nonlactating, and females of childbearing potential and males will agree to use contraception as detailed in the protocol.
. Able to comprehend and willing to sign an informed consent /assent form and to abide by the study restrictions (travel as necessary, clinical phase 1 unit or similar for up to 3 days).
Exclusion criteria
. Have undergone kidney transplantation.
. Are receiving dialysis treatment.
. History of significant hypersensitivity to NAC or any ingredient of NPI-001 oral solution.
. Participation in a clinical study involving administration of an investigational drug (new chemical entity) in the 30 days prior to Day 1.
. Inability to provide blood samples, including difficulty with venous access.
. Subjects who, in the opinion of the Investigator and/or Sponsor (or designee), should not participate in this study.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.