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ADENO-ASSOCIATED VIRAL VECTOR SEROTYPE RH10 ENCODING MIRNA AGAINST SOD1 MRNA

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.

Table of contents

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[1].

It is an interventional study, which means researchers are giving a study treatment and then watching what happens[1].

The condition being studied is amyotrophic lateral sclerosis (ALS), with a focus on the SOD1 form of the disease[1].

The brief study goal is to evaluate the safety and tolerability of ascending doses of intrathecally administered AMT-162 in participants with SOD1-ALS[1].

Who can participate

This trial is for adults with SOD1 ALS[1].

The source data does not list more detailed entry rules, such as exact age limits, disease stage, or other medical requirements[1].

Because the study is focused on the SOD1 form of ALS, it is not a general ALS trial for all patients with the disease[1].

Trial phase and study design

This is a Phase 1 trial[1].

Phase 1 studies are early clinical trials that mainly look at safety, tolerability, and basic signs of how the treatment behaves in people[1].

The study status is listed as Authorised[1].

The planned enrollment is 20 participants, so this is a small study[1].

The intervention is listed as ADENO-ASSOCIATED VIRAL VECTOR SEROTYPE RH10 ENCODING MIRNA AGAINST SOD1 MRNA for intrathecal use[1].

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[1].

Researchers are also tracking changes in clinical safety laboratory tests[1].

They are measuring changes in vital signs, such as blood pressure, pulse, and temperature[1].

The study also includes changes in electrocardiogram (ECG) results, which show the heart’s electrical activity[1].

These measurements help researchers see whether the treatment appears safe in the study setting[1].

Study status and size

The trial is marked as Authorised[1].

Only one trial record is provided in the source data, so the article is based on this single study[1].

With an enrollment of 20, the study is designed to gather early information rather than answer all questions about the treatment[1].

Patient-focused terms

Intrathecal use means the treatment is given into the fluid around the spinal cord[1].

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[1].

SOD1 ALS means ALS that is linked to the SOD1 gene, and this is the specific patient group being studied here[1].

Trial ID Phase Condition studied Status Enrollment
NCT06100276 Phase 1 Amyotrophic Lateral Sclerosis (ALS), SOD1 ALS Authorised 20

FAQ

  • What is being studied in these clinical trials? The trials are studying ADENO-ASSOCIATED VIRAL VECTOR SEROTYPE RH10 ENCODING MIRNA AGAINST SOD1 MRNA in adults with SOD1 ALS. The main focus is safety, tolerability, and early signs of effect.
  • Who can take part in the trial? The trial is for adults with SOD1 ALS. It is not described as open to all people with ALS, only to those with the SOD1 form.
  • What phase is the trial? This is a Phase 1 trial. Phase 1 studies are early trials that mainly check safety and how well people tolerate the study treatment.
  • What outcomes are researchers measuring? Researchers are measuring treatment-emergent adverse events, safety laboratory tests, vital signs, and ECG results. These help show whether the treatment appears safe in the study setting.
  • What condition is the trial targeting? The trial is targeting amyotrophic lateral sclerosis, also called ALS, specifically the SOD1 form. ALS is a serious disease that affects nerve cells and muscle control.

Ongoing Clinical Trials on ADENO-ASSOCIATED VIRAL VECTOR SEROTYPE RH10 ENCODING MIRNA AGAINST SOD1 MRNA

  • Study on the Safety and Effects of AMT-162 Gene Therapy for Adults with SOD1 ALS

    Not recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    Sweden

Glossary

  • Amyotrophic Lateral Sclerosis (ALS): A serious disease that affects nerve cells and leads to weakness and trouble moving, speaking, swallowing, or breathing.
  • SOD1 ALS: A form of ALS linked to the SOD1 gene. The trial is focused on this specific type of ALS.
  • Phase 1: An early stage of clinical research. The main goal is to learn about safety and tolerability.
  • Interventional study: A trial where researchers give a study treatment and then measure what happens.
  • Intrathecal use: Given into the fluid around the spinal cord. This is a direct way to deliver a study treatment.
  • Safety: How well a treatment can be used without causing unacceptable harm.
  • Tolerability: How well people are able to handle a treatment, including how side effects affect them.
  • Treatment-emergent adverse events (TEAEs): Health problems that start or get worse after a person begins the study treatment.
  • Laboratory tests: Blood or other tests used to check body function and overall health.
  • Vital signs: Basic body measurements such as blood pressure, pulse, and temperature.
  • Electrocardiogram (ECG): A test that records the heart’s electrical activity.