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Study on the Safety and Effects of Acetylcysteine Amide for Patients Aged 12 and Over with Hereditary Cystatin C Amyloid Angiopathy

Investigational
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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a rare condition called Hereditary Cystatin C Amyloid Angiopathy (HCCAA). This is a genetic disorder that can lead to problems with blood vessels in the brain, sometimes causing bleeding. The trial will test a new treatment called NPI-001, also known as acetylcysteine amide, which is taken as a tablet. The purpose of the study is to evaluate the safety, tolerability, and effectiveness of this treatment in patients with HCCAA.

Participants in the study will take the medication NPI-001 orally for a period of up to 12 months. During this time, they will have regular check-ups to monitor their health and any side effects. The study will also look at how often brain bleeding events occur in patients taking the medication. This will help researchers understand if the treatment can reduce these events and improve the condition.

Throughout the study, participants will undergo various assessments, including blood tests, skin biopsies, and MRI scans of the brain. These tests will help track changes in the body and the effects of the treatment. The study aims to provide valuable information on the potential benefits of NPI-001 for people with Hereditary Cystatin C Amyloid Angiopathy.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Participation begins after providing informed consent. Eligibility includes being 12 years or older, of Icelandic ancestry, and carrying a specific genetic mutation.

    Initial assessments include skin biopsies, blood tests, and MRI evaluations of the brain.

  2. Step 2

    Medication administration

    The medication NPI-001 is administered orally in the form of a tablet.

    The dosage and frequency are determined by the study protocol and monitored throughout the trial.

  3. Step 3

    Monitoring and assessments

    Regular monitoring includes checking for any adverse effects, changes in vital signs, and conducting physical and neurological examinations.

    Blood and urine tests are performed to assess safety and measure specific protein levels.

  4. Step 4

    Efficacy evaluation

    The study evaluates the frequency of cerebral bleeding events and other clinical outcomes.

    Additional assessments include cognitive function tests and imaging studies to monitor brain health.

  5. Step 5

    Follow-up and conclusion

    The trial is expected to continue until March 2025, with follow-up assessments scheduled throughout the study period.

    Final evaluations will determine the overall safety and effectiveness of the treatment.

Who can join the trial?

9 criteria

  • The patient must be male or female, aged 12 or older, and of Icelandic ancestry. If the patient is between 12-17 years old, they can only join if a special committee approves it after checking safety in adults for at least 3 months.
  • The patient must have a specific change in their genes, known as the L68Q mutation, in the cystatin C gene. This is confirmed through genetic testing.
  • The patient must have previously been found to have cystatin C/amyloid protein complexes in their skin. These are specific protein build-ups.
  • The patient must have mild cognitive impairment, meaning they have some memory or thinking problems but can still follow the study instructions.
  • The patient must agree to have skin samples taken at the start and during the study, up to 12 months.
  • The patient must agree to have blood tests at the start and during the study, up to 12 months.
  • The patient must agree to have MRI scans of the brain. An MRI is a type of scan that uses magnets and radio waves to take pictures of the inside of the body.
  • The patient must give informed consent, which means they understand the study and agree to participate.
  • The patient must agree to use birth control methods as required by local rules. This applies to women who can have children and men who can father children. They must either have had certain surgeries to prevent pregnancy, not have intercourse, or use two acceptable birth control methods during the study and for 4 weeks after the last visit. Acceptable methods include hormonal birth control, devices placed in the uterus, barrier methods like condoms, or surgeries like tubal ligation or vasectomy.

Who cannot join the trial?

10 criteria

  • Patients who are not diagnosed with Hereditary Cystatin C Amyloid Angiopathy (HCCAA) cannot participate. This is a specific condition affecting blood vessels in the brain.
  • Individuals under the age of 12 are not eligible to join the study.
  • Participants must not have any other medical conditions that could interfere with the study or its results.
  • Pregnant or breastfeeding women are excluded from participating in the trial.
  • Patients who are unable to take the study medication orally are not eligible.
  • Individuals who have participated in another clinical trial within a certain time frame before this study may be excluded.
  • Anyone with a history of allergic reactions to the study medication or its ingredients cannot participate.
  • Patients with severe liver or kidney problems may be excluded from the study.
  • Individuals with certain mental health conditions that could affect their ability to follow study instructions may not be eligible.
  • Participants who are unable to provide informed consent, which means they cannot understand and agree to the study's requirements, are excluded.
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Investigated drugs

NPI-001 (AT-001) is a medication being studied for its safety, tolerability, and effectiveness in patients with Hereditary Cystatin C Amyloid Angiopathy (HCCAA). This medication is taken orally and is being tested to see if it can help reduce the frequency of cerebral bleeding events in patients aged 12 and over who have this condition.

What is already known about the treatment

NPI-001 – This medication is administered orally and is currently being studied for its safety, tolerability, and effectiveness in treating Hereditary Cystatin C Amyloid Angiopathy (HCCAA). It is in the clinical trial phase, with ongoing research to determine its impact on reducing cerebral bleeding events in patients aged 12 and older. NPI-001 is designed to target the underlying mechanisms of HCCAA, potentially altering the disease process at a molecular level. It falls under the pharmacological classification of investigational drugs, as it is still being evaluated for its therapeutic benefits and safety profile.

Investigated diseases

Hereditary Cystatin C Amyloid Angiopathy (HCCAA) – This is a rare genetic disorder characterized by the accumulation of amyloid proteins in the walls of blood vessels in the brain. The buildup of these proteins can lead to weakening of the blood vessels, making them prone to bleeding. Individuals with HCCAA may experience recurrent cerebral hemorrhages, which can cause neurological symptoms such as headaches, seizures, or cognitive changes. The disease typically manifests in early adulthood, but symptoms can vary widely among affected individuals. Over time, the repeated bleeding events can lead to progressive neurological impairment. The condition is inherited in an autosomal dominant pattern, meaning a single copy of the altered gene in each cell is sufficient to cause the disorder.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IITrial ID2023-503969-36-01Protocol code2023-503969-36-01Estimated enrolment25 patientsSponsorArctic Therapeutics ehf.

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On this site, “treatment” means an investigational medicine being studied in a clinical trial. Its safety and efficacy for the use being studied have not yet been confirmed, some participants may receive a placebo or a comparator medicine, and taking part does not guarantee any health benefit. The decision to take part is made by the doctor at the research site. This site is for information only and does not replace medical advice.

This service is not affiliated with the European Commission, the EMA, or the official CTIS system. Most information comes from publicly available international clinical-trial registries, supplemented by data from academic sites, national regulators and commercial sponsors. On this site, “treatment” and “therapy” mean a medicine being tested in a clinical trial. Its safety and effectiveness in the use being studied are not yet confirmed, some participants may receive a placebo or a comparator, and taking part does not guarantee a health benefit. The doctor at the research site decides who can take part. This site provides information, not medical advice. Certain content and visual elements on this website have been generated or enhanced using artificial intelligence (AI).