In short
Clinical trials are studying ADENO-ASSOCIATED VIRAL VECTOR SEROTYPE RH10 ENCODING MIRNA AGAINST SOD1 MRNA in adults with SOD1 ALS. The main goal is to check safety and tolerability, and to look for early signs of benefit. These trials focus on people with amyotrophic lateral sclerosis linked to SOD1.
Key points
- Clinical trials are studying ADENO-ASSOCIATED VIRAL VECTOR SEROTYPE RH10 ENCODING MIRNA AGAINST SOD1 MRNA in adults with SOD1 ALS. The available trial is a Phase 1 study, so it is mainly designed to check safety and tolerability. Researchers are also looking at early signs of benefit while giving the treatment intrathecally, meaning into the fluid around the spinal cord. The main outcomes include treatment-emergent adverse events, laboratory tests, vital signs, and ECG results. The study is authorised and plans to enroll 20 participants.
Trial overview
The trial listed for ADENO-ASSOCIATED VIRAL VECTOR SEROTYPE RH10 ENCODING MIRNA AGAINST SOD1 MRNA is called Safety and exploratory efficacy of AMT-162 for adults with SOD1 ALS.
It is an interventional study, which means researchers are giving a study treatment and then watching what happens.
The condition being studied is amyotrophic lateral sclerosis (ALS), with a focus on the SOD1 form of the disease.
The brief study goal is to evaluate the safety and tolerability of ascending doses of intrathecally administered AMT-162 in participants with SOD1-ALS.
Who can participate
This trial is for adults with SOD1 ALS.
The source data does not list more detailed entry rules, such as exact age limits, disease stage, or other medical requirements.
Because the study is focused on the SOD1 form of ALS, it is not a general ALS trial for all patients with the disease.
Trial phase and study design
This is a Phase 1 trial.
Phase 1 studies are early clinical trials that mainly look at safety, tolerability, and basic signs of how the treatment behaves in people.
The study status is listed as Authorised.
The planned enrollment is 20 participants, so this is a small study.
The intervention is listed as ADENO-ASSOCIATED VIRAL VECTOR SEROTYPE RH10 ENCODING MIRNA AGAINST SOD1 MRNA for intrathecal use.
What is being measured
The main outcomes are focused on treatment-emergent adverse events (TEAEs), which are health problems that start or get worse after treatment begins.
Researchers are also tracking changes in clinical safety laboratory tests.
They are measuring changes in vital signs, such as blood pressure, pulse, and temperature.
The study also includes changes in electrocardiogram (ECG) results, which show the heart’s electrical activity.
These measurements help researchers see whether the treatment appears safe in the study setting.
Study status and size
The trial is marked as Authorised.
Only one trial record is provided in the source data, so the article is based on this single study.
With an enrollment of 20, the study is designed to gather early information rather than answer all questions about the treatment.
Patient-focused terms
Intrathecal use means the treatment is given into the fluid around the spinal cord.
Exploratory efficacy means the study may look for early signs that the treatment could help, but that is not the main purpose of a Phase 1 trial.
SOD1 ALS means ALS that is linked to the SOD1 gene, and this is the specific patient group being studied here.