Safety, Tolerability and Efficacy of NPI-001 in Patients With Hereditary Cystatin C Amyloid Angio… (NCT07716865) | Clinical Trial Compass
Active — Not RecruitingPhase 2
Safety, Tolerability and Efficacy of NPI-001 in Patients With Hereditary Cystatin C Amyloid Angiopathy (HCCAA)
Iceland15 participantsStarted 2024-04-03
Plain-language summary
The study aims to measure safety, tolerability and biomarker-based efficacy of NPI-001 (AT-001) in Subjects with Hereditary Cystatin C Amyloid Angiopathy (HCCAA). In the study participants receive increasing dose or active treatment (250 mg vs 500mg vs 750 mg) or matched placebo in the form of tablets BID.
Who can participate
Age range
12 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
. Patient is male or female, aged 12 or older, and of Icelandic ancestry (see section 5.1 Selection of Trial Population). Subjects 12-17 years old will only qualify for inclusion if the DSMB approves lowering the minimum age following review of at least 3 months of safety in adults.
. Patient has been genotyped/sequenced and confirmed to carry the L68Q mutation in the cystatin C gene.
. Patients with previously established cystatin C/amyloid protein complexes in the skin
. Patients with mild cognitive impairment with cognitive function to follow the study protocol.
. Patient is willing to have a baseline and follow up skin biopsies according to the schedule of assessments, for up to 12 months, and up to 24 months if participating in the extension phase.\*
. Patient is willing to have a baseline and follow up blood tests according to the schedule of assessments, for up to 12 months, and up to 24 months if participating in the extension phase.\*
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.
What they're measuring
1
Incidence of Treatment-Emergent Adverse Events
Timeframe: Through study completion, up to 24 months
2
Frequency of cerebral bleeding events
Timeframe: Through study completion, up to 24 months
3
Safety labs results within normal range
Timeframe: Through study completion, up to 24 months
. Patient is willing to undergo MRI evaluations of the brain.\*
. Patient has provided informed consent for participation in trial.
Exclusion criteria
. Patient does not have L68Q mutation.
. Patients with moderate to severe cognitive impairment.
. Patient has clinically significant illness, mental or physical, that, in the opinion of the investigator, might confound the results of the study, pose additional risk to the patient by their participation, or prevent/impede the patient from completing the study.
. Patient has known sensitivity to NAC
. Coagulation/clotting parameters clinically significant outside the normal range (platelet counts, aPTT, PT)
. Subject is not willing to cease NAC supplementation at least 2 weeks prior to study participation.
. Patient is pregnant or breastfeeding.
. Known or suspected excessive alcohol or drug abuse.