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Safety, Tolerability and Efficacy of NPI-001 (AT-001) in Patients with Hereditary Cystatin C Amyloid Angiopathy (HCCAA)
EU CTIS ID: 2023-503969-36-00
What this study is testing
To assess safety and tolerability and efficacy of NPI-001 administered orally in HCCAA patients (ages 12 and over) To assess the effect of NPI-001 on frequency of cerebral bleeding events in comparison with historical rate (HR) To assess the effect of NPI-001 on biomarkers obtained from skin biopsies, including reduction in amyloid-cystatin C complexes and related collagen and skin cell surface activation markers compared with baseline levels, using cryoEM and Western blot approaches
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 1. 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.
- 2. Patient has been genotyped/sequenced and confirmed to carry the L68Q mutation in the cystatin C gene.
- 3. Patient is willing to have a baseline and follow up skin biopsies according to the schedule of assessments, for up to 12 months.
- 4. Patient is willing to have a baseline and follow up blood tests according to the schedule of assessments, for up to 12 months.
- 5. Patient is willing to undergo MRI evaluations of the brain.
- 6. Patient has provided informed consent for participation in trial
You likely can't join if
- 1. Patient does not have L68Q mutation
- 10. Patient is taking medications known to affect or be affected by CYP enzymes or transporters will be excluded to avoid any inference
- 2. 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.
- 3. Patient has known sensitivity to NAC
- 4. Subject is not willing to cease NAC supplementation at least 2 weeks prior to study participation.
- 5. Patient is pregnant or breastfeeding.
See the full eligibility criteria
- 1. 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.
- 2. Patient has been genotyped/sequenced and confirmed to carry the L68Q mutation in the cystatin C gene.
- 3. Patient is willing to have a baseline and follow up skin biopsies according to the schedule of assessments, for up to 12 months.
- 4. Patient is willing to have a baseline and follow up blood tests according to the schedule of assessments, for up to 12 months.
- 5. Patient is willing to undergo MRI evaluations of the brain.
- 6. Patient has provided informed consent for participation in trial
- 7. Patient is willing and able to use contraception consistent with local regulations regarding the methods for participants in the clinical trial. Both female participants of childbearing potential and male participants able to father children must have (or have a partner who has) had a bilateral oophorectomy, hysterectomy or bilateral salpingectomy; must abstain from intercourse; or must agree to practice 2 acceptable methods of contraception throughout the course of the study and 4 weeks after the last visit. Acceptable methods of contraception include hormonal contraception (i.e., birth control pills, injected hormones, dermal patch or vaginal ring), intrauterine device, barrier methods (diaphragm, condom), tubal ligation, and vasectomy.
- 2.3. Patients with mild cognitive impairment with cognitive function to follow the study protocol.
- 1. Patient does not have L68Q mutation
- 10. Patient is taking medications known to affect or be affected by CYP enzymes or transporters will be excluded to avoid any inference
- 2. 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.
- 3. Patient has known sensitivity to NAC
- 4. Subject is not willing to cease NAC supplementation at least 2 weeks prior to study participation.
- 5. Patient is pregnant or breastfeeding.
- 6. Known or suspected excessive alcohol or drug abuse
- 7. There is any concern by the investigator regarding the patient’s safety, compliance, or suitability with respect to his/her participation in the study.
- 8. Use of other investigational drugs at the time of enrollment, or within 5 half-lives of enrollment, or within 14 days, whichever is longer
- 1.2. Patients with moderate to severe cognitive impairment.
The study team makes the final eligibility decision.
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 65+ years, 18-64 years, 0-17 years. The study team makes the final eligibility decision.
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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.