In short
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.
Key points
- Clinical trials of Acetylcysteine Amide are studying two main conditions: hereditary cystatin C amyloid angiopathy and Alzheimer’s disease. All listed studies are Phase 2, so they are looking at early signs of benefit while also checking safety. The hereditary cystatin C amyloid angiopathy trial includes people aged 12 and older, while the Alzheimer’s disease trial includes subjects with that diagnosis. Researchers are measuring safety, tolerability, brain bleeding events, MRI findings, lab tests, and other clinical checks. One hereditary cystatin C amyloid angiopathy trial is authorised, one is withdrawn, and the Alzheimer’s disease trial is authorised.
Trial overview
These clinical trials are studying Acetylcysteine Amide in people with hereditary cystatin C amyloid angiopathy (HCCAA) and Alzheimer’s disease. 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.
Hereditary cystatin C amyloid angiopathy trials
Two trial records focus on HCCAA, and both use oral Acetylcysteine Amide at 1500 mg. One study is authorised, and the other was withdrawn before completion.
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. 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.
The withdrawn HCCAA study had a similar goal, but it also planned to measure skin biomarkers, including cystatin C/amyloid protein complex deposition and related collagen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. It also planned to compare serious cerebral bleeding events with a historical rate, which means a past bleeding rate used as a comparison.
Alzheimer’s disease trial
Another authorised Phase 2 study is evaluating Acetylcysteine Amide in subjects with Alzheimer’s disease. 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.
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. The study also uses MRI imaging to assess ARIA H and ARIA E in the brain, and the primary outcome includes ARIA MRI classification.
Who can participate
The HCCAA trial is open to patients aged 12 and over with hereditary cystatin C amyloid angiopathy. The Alzheimer’s disease trial includes subjects with Alzheimer’s disease.
Based on the trial records, the studies are designed for people who already have the target condition, rather than for healthy volunteers.
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.
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. In the withdrawn HCCAA study, they also planned skin biopsy biomarker tests, including amyloid-cystatin C complexes and collagen-related markers.
In the Alzheimer’s disease trial, the main measures are safety, lab values, ECG, physical examination, vital signs, and MRI-based ARIA classification. These tests help researchers watch for brain imaging changes and other signs of how people respond to the study treatment.
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. The withdrawn HCCAA trial also listed 25 participants as its planned enrollment.
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.