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Acetylcysteine Amide

Clinical trials are studying Acetylcysteine Amide in people with hereditary cystatin C amyloid angiopathy and Alzheimer’s disease. These trials are looking at safety, tolerability, and signs of benefit, including bleeding events and brain imaging findings. Both adults and, in one study, people aged 12 and older are included.

Table of Contents

Trial overview

These clinical trials are studying Acetylcysteine Amide in people with hereditary cystatin C amyloid angiopathy (HCCAA) and Alzheimer’s disease.[1][2] All listed studies are Phase 2 interventional trials, which means the treatment is being given to participants and the researchers are looking for early signs of benefit as well as safety.[1][2]

Hereditary cystatin C amyloid angiopathy trials

Two trial records focus on HCCAA, and both use oral Acetylcysteine Amide at 1500 mg.[1][3] One study is authorised, and the other was withdrawn before completion.[1][3]

The authorised HCCAA study aims to assess safety, tolerability, and efficacy after 12 months of treatment, with an open-label extension phase that continues to 24 months.[1] Its main efficacy outcome is the frequency of clinical cerebral bleeding events, meaning brain bleeding events that can cause stroke-like problems, either hemorrhagic or ischemic.[1]

The withdrawn HCCAA study had a similar goal, but it also planned to measure skin biomarkers, including cystatin C/amyloid protein complex deposition and related collagen and cell surface marker changes.[3] It also planned to compare serious cerebral bleeding events with a historical rate, which means a past bleeding rate used as a comparison.[3]

Alzheimer’s disease trial

Another authorised Phase 2 study is evaluating Acetylcysteine Amide in subjects with Alzheimer’s disease.[2] This trial compares Acetylcysteine Amide with a placebo, which is a tablet that looks like the study drug but does not contain the active treatment.[2]

The main focus is safety and tolerability, using treatment-emergent adverse events, serious adverse events, clinical lab values, electrocardiogram results, physical examination, and vital signs.[2] The study also uses MRI imaging to assess ARIA H and ARIA E in the brain, and the primary outcome includes ARIA MRI classification.[2]

Who can participate

The HCCAA trial is open to patients aged 12 and over with hereditary cystatin C amyloid angiopathy.[1][3] The Alzheimer’s disease trial includes subjects with Alzheimer’s disease.[2]

Based on the trial records, the studies are designed for people who already have the target condition, rather than for healthy volunteers.[1][2]

What researchers measure

In the HCCAA study, researchers are checking safety with treatment-emergent adverse events, serious adverse events, vital signs, ECG findings, physical and neurological exams, and lab tests of blood and urine.[1]

In the same study, researchers are also checking efficacy, which means whether the treatment may help the disease, by measuring the number of cerebral bleeding events.[1] In the withdrawn HCCAA study, they also planned skin biopsy biomarker tests, including amyloid-cystatin C complexes and collagen-related markers.[3]

In the Alzheimer’s disease trial, the main measures are safety, lab values, ECG, physical examination, vital signs, and MRI-based ARIA classification.[2] These tests help researchers watch for brain imaging changes and other signs of how people respond to the study treatment.[2]

Trial status and size

The authorised HCCAA trial plans to enroll 25 participants, and the authorised Alzheimer’s disease trial plans to enroll 30 participants.[1][2] The withdrawn HCCAA trial also listed 25 participants as its planned enrollment.[3]

Across the available records, the development program for Acetylcysteine Amide is still early and small in size, with a focus on careful safety monitoring and a few disease-specific outcome measures.[1][2][3]

Trial ID Phase Condition studied Status Enrollment
2023-503969-36-01 Phase 2 Hereditary Cystatin C Amyloid Angiopathy (HCCAA) Authorised 25
2024-519497-39-00 Phase 2 Alzheimer’s Disease Authorised 30
2023-503969-36-00 Phase 2 Hereditary Cystatin C Amyloid Angiopathy (HCCAA) Withdrawn 25

FAQ

  • What is being studied in Acetylcysteine Amide trials? The trials are studying whether Acetylcysteine Amide is safe and well tolerated, and whether it may help in hereditary cystatin C amyloid angiopathy and Alzheimer’s disease. Researchers are also looking at bleeding events and biomarker changes.
  • Who can take part in these trials? One trial includes patients with hereditary cystatin C amyloid angiopathy who are 12 years and older. Another trial includes subjects with Alzheimer’s disease.
  • What phase are the trials in? Both listed trials are Phase 2 studies. Phase 2 trials usually look at early signs of benefit while also checking safety.
  • What outcomes are the researchers measuring? They are measuring treatment-emergent adverse events, serious adverse events, vital signs, ECG results, lab tests, neurological exams, MRI findings, and bleeding events. One trial also studies skin biomarker changes.
  • Are all the trials still active? No. One hereditary cystatin C amyloid angiopathy trial is authorised, one is withdrawn, and the Alzheimer’s disease trial is authorised. This means the studies are at different stages of progress.

Ongoing Clinical Trials on Acetylcysteine Amide

  • Study on the Safety and Effects of NPI-001 (Acetylcysteine Amide) for Patients Aged 50-85 with Mild Cognitive Impairment or Mild Alzheimer’s Disease

    Recruiting

    1
    Investigated drugs:
    Denmark Iceland

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

    Not yet recruiting

    1 1
    Investigated diseases:
    Investigated drugs:
    Iceland

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

    Not recruiting

    1 1
    Investigated diseases:
    Investigated drugs:
    Iceland

Glossary

  • Phase 2: A mid-stage clinical trial that checks early signs of benefit and continues to study safety in a small group of people.
  • Interventional study: A study where researchers give a treatment or placebo and then compare results.
  • Safety: How well a treatment can be used without causing harmful problems.
  • Tolerability: How well people can handle a treatment, including whether side effects are manageable.
  • Treatment-emergent adverse events: Health problems that start or get worse after treatment begins.
  • Serious adverse events: Severe health problems that may need hospital care or cause major harm.
  • Electrocardiogram (ECG): A test that records the heart’s electrical activity.
  • Vital signs: Basic body measurements such as blood pressure, pulse, temperature, and breathing rate.
  • Biomarker: A measurable sign in the body that can help show disease activity or response to treatment.
  • MRI: A scan that uses magnets and radio waves to make detailed pictures of the brain.
  • ARIA: Brain imaging changes seen on MRI; in these trials, the study checks ARIA H and ARIA E.
  • Historical rate: A past rate used for comparison with what happens in the study.

References

  1. https://clinicaltrials.gov/study/2023-503969-36-01
  2. https://clinicaltrials.gov/study/2024-519497-39-00
  3. https://clinicaltrials.gov/study/2023-503969-36-00