RecruitingRare disease

Long-Term Safety and Efficacy Study of Eplontersen in Patients with Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

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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 condition known as Hereditary Transthyretin-Mediated Amyloid Polyneuropathy (hATTR-PN). This is a rare genetic disorder that affects the nerves, leading to symptoms like numbness, pain, and weakness. The study is investigating the long-term safety and effectiveness of a treatment called ION 682884, also known as eplontersen. Eplontersen is a type of medication called an antisense oligonucleotide, which is designed to target and reduce the production of a specific protein that contributes to the disease.

The purpose of the study is to evaluate how safe and tolerable eplontersen is when used over an extended period. Participants in the study will receive eplontersen through injections under the skin. The study will monitor various health indicators, such as blood tests, kidney function, and liver enzymes, to ensure the treatment is safe. Participants will also be asked about any side effects they experience and will undergo regular physical examinations and heart tests.

Throughout the study, changes in the participants' symptoms and quality of life will be assessed using specific questionnaires and scores. These assessments will help determine if the treatment is effective in managing the symptoms of hATTR-PN. The study will also compare these results to baseline measurements taken at the start of the study. Participants will continue to receive the treatment and be monitored for a period of time to gather comprehensive data on the long-term effects of eplontersen.

The research process

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

Step 1
Joining the study
Participation begins after satisfactory completion of a previous study with the medication ION-682884 or a related study with inotersen. Written informed consent is required, along with compliance with study requirements.
Participants must meet specific criteria, including non-pregnancy for females and the use of effective contraceptive methods for both males and females, if applicable.
Step 2
Medication administration
The medication eplontersen, also known as ION-682884, is administered as a solution for injection.
The route of administration is subcutaneous use, meaning the injection is given under the skin.
Step 3
Monitoring and assessments
Regular monitoring includes checking platelet count, kidney function, liver enzymes, and other laboratory tests.
Participants will be observed for any adverse events and changes in vital signs, weight, and physical examination findings.
Step 4
Evaluation of treatment effects
The study evaluates changes in neuropathy symptoms, quality of life, and other health measures from the baseline.
Secondary assessments include scores related to neuropathy impairment, quality of life, and physical health.
Step 5
Completion of the study
The study is estimated to conclude by September 2026.
Participants will continue to be monitored for safety and efficacy of the treatment throughout the study duration.

Who can join the trial?

10 criteria

Must have completed a previous study with the medication ION-682884 or a related study with a similar medication, as judged satisfactory by the study doctor and sponsor.
Must have signed a written agreement to participate in the study and be able to follow all study requirements.
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Females must not be pregnant or breastfeeding and must meet one of the following conditions:
- Have had surgery to prevent pregnancy (e.g., tubal occlusion, hysterectomy).
- Be post-menopausal, which means not having a menstrual period for 12 months without another medical reason.
- Be abstinent, meaning not engaging in sexual activity, if this is their usual lifestyle.
- If sexually active and able to have children, agree to use effective birth control methods and take pregnancy tests as required by the study.
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  Males must meet one of the following conditions:
  - Have had surgery to prevent fathering a child (e.g., bilateral orchidectomy).
  - Be abstinent, meaning not engaging in sexual activity, if this is their usual lifestyle.
  - If sexually active with a female partner who can have children, ensure the partner uses effective birth control methods during the study and for 24 weeks after the last dose of the medication.
  - Must be willing to take vitamin A supplements as required by the study.

Who cannot join the trial?

10 criteria

Patients who have other serious health conditions that could interfere with the study.
Patients who are currently participating in another clinical trial.
Patients who have had a recent major surgery or are planning to have surgery during the study period.
Patients who have a history of allergic reactions to similar medications.
Patients who are pregnant or breastfeeding.
Patients who have a history of substance abuse or alcohol dependency.
Patients who are unable to comply with the study procedures or follow-up visits.
Patients who have a known infection with certain viruses, such as HIV or hepatitis.
Patients who have a history of certain heart conditions.
Patients who have uncontrolled high blood pressure.

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Verified sites

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Verified
Assistance Publique Hopitaux De Marseille
Marseille, France
Verified
Bicetre Hospital
Le Kremlin-Bicetre, France
Responsive
Region Vaesterbotten
Umea, Sweden

Trial locations

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Investigated drugs

ION-682884 (eplontersen) is a medication being studied for its long-term safety and effectiveness in treating patients with hereditary transthyretin-mediated amyloid polyneuropathy (hATTR-PN). This condition is a rare genetic disorder that affects the nerves, leading to symptoms like pain, weakness, and loss of sensation. The trial aims to see how well patients tolerate the medication over an extended period and to monitor any side effects that may occur.

Eplontersen

What is already known about the treatment

Eplontersen – Eplontersen is administered as an injection, typically under the skin. It is currently being studied in clinical trials for its long-term safety and effectiveness in treating hereditary transthyretin-mediated amyloid polyneuropathy (hATTR-PN). This medication works by reducing the production of a specific protein that contributes to nerve damage in this condition. Eplontersen is classified as an antisense oligonucleotide, which means it targets and binds to RNA to prevent the production of harmful proteins.

Investigated diseases

Hereditary Transthyretin-Mediated Amyloid Polyneuropathy – This is a rare genetic disorder characterized by the buildup of amyloid proteins in the body's tissues, particularly affecting the nerves. It is caused by mutations in the transthyretin (TTR) gene, leading to the production of abnormal proteins that deposit in various organs and tissues. The disease primarily affects the peripheral nerves, leading to symptoms such as numbness, tingling, and pain in the limbs. Over time, it can progress to muscle weakness and loss of reflexes. The condition may also impact autonomic functions, causing issues like digestive problems and blood pressure fluctuations. As the disease advances, it can significantly affect mobility and quality of life.