Hereditary neuropathic amyloidosis – European Clinical Trials Information Network https://clinicaltrials.eu Bridging Patients with Clinical Trials Fri, 12 Jun 2026 10:37:05 +0000 en-US hourly 1 https://wordpress.org/?v=7.0 https://clinicaltrials.eu/wp-content/uploads/2024/12/cropped-EU_icon-32x32.png Hereditary neuropathic amyloidosis – European Clinical Trials Information Network https://clinicaltrials.eu 32 32 Long-Term Safety and Efficacy Study of Eplontersen in Patients with Hereditary Transthyretin-Mediated Amyloid Polyneuropathy https://clinicaltrials.eu/trial/long-term-safety-and-efficacy-study-of-eplontersen-in-patients-with-hereditary-transthyretin-mediated-amyloid-polyneuropathy/ Wed, 29 Apr 2026 15:00:55 +0000 https://clinicaltrials.eu/trial/long-term-safety-and-efficacy-study-of-eplontersen-in-patients-with-hereditary-transthyretin-mediated-amyloid-polyneuropathy/ 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.

]]> Study on NTLA-2001 for Patients with Hereditary Transthyretin Amyloidosis with Polyneuropathy https://clinicaltrials.eu/trial/study-on-ntla-2001-for-patients-with-hereditary-transthyretin-amyloidosis-with-polyneuropathy/ Wed, 29 Apr 2026 15:00:39 +0000 https://clinicaltrials.eu/trial/study-on-ntla-2001-for-patients-with-hereditary-transthyretin-amyloidosis-with-polyneuropathy/ This clinical trial is focused on studying a condition called Hereditary Transthyretin Amyloidosis with Polyneuropathy (ATTRv-PN) and another condition known as Transthyretin Amyloidosis-Related Cardiomyopathy (ATTR-CM). These are diseases where abnormal proteins build up in the body, affecting nerves and the heart. The treatment being tested is called NTLA-2001, which is a new type of therapy that uses a technology called CRISPR/Cas9 to edit genes. This treatment involves a special messenger RNA that helps guide the editing process to target the TTR gene, which is responsible for the disease.

The purpose of the study is to evaluate the safety and how well the body tolerates NTLA-2001, as well as to understand how the drug moves through and affects the body. The study is divided into two parts. In the first part, patients will receive a single dose of the treatment to see how they respond. In the second part, the study will expand to include more patients to further assess the treatment’s effects. Some patients who have already received a smaller dose of NTLA-2001 may receive a follow-up dose to gather more information.

Throughout the study, various assessments will be conducted to monitor the safety of the treatment and its impact on the patients’ conditions. These assessments will include checking for any immune reactions, measuring how the drug is processed in the body, and evaluating changes in the symptoms of polyneuropathy, which is a condition where multiple nerves are damaged. The study aims to provide valuable insights into the potential of NTLA-2001 as a treatment for these challenging diseases.

]]> Long-Term Safety Study of NTLA-2001 for Patients with Hereditary Transthyretin Amyloidosis with Polyneuropathy or Transthyretin Amyloidosis-Related Cardiomyopathy https://clinicaltrials.eu/trial/long-term-safety-study-of-ntla-2001-for-patients-with-hereditary-transthyretin-amyloidosis-with-polyneuropathy-or-transthyretin-amyloidosis-related-cardiomyopathy/ Wed, 29 Apr 2026 14:30:08 +0000 https://clinicaltrials.eu/trial/long-term-safety-study-of-ntla-2001-for-patients-with-hereditary-transthyretin-amyloidosis-with-polyneuropathy-or-transthyretin-amyloidosis-related-cardiomyopathy/ This clinical trial is focused on the long-term follow-up of individuals who have been treated with a medication called NTLA-2001. The study is specifically looking at two conditions: Hereditary Transthyretin Amyloidosis with Polyneuropathy and Transthyretin Amyloidosis-Related Cardiomyopathy. These are rare diseases where abnormal proteins build up in the body, affecting nerves and the heart. The treatment, NTLA-2001, is a type of gene therapy designed to target and modify the Transthyretin (TTR) gene, which is involved in these conditions.

The purpose of this study is to evaluate the long-term safety of NTLA-2001 in participants who have already received this treatment in previous studies. Participants will be monitored over time to check for any side effects related to the treatment and to observe specific health markers. The study involves regular follow-up visits where participants’ health will be assessed, but it does not involve any new treatments or medications beyond what they have already received.

Participants in this study have previously completed or discontinued a clinical study involving NTLA-2001 and have agreed to continue with follow-up visits. The study aims to gather important information about the long-term effects of the treatment, which will help in understanding its safety and impact on the conditions being studied. This information is crucial for developing future treatments and improving care for individuals with these rare diseases.

]]> Study on the Effectiveness and Safety of Vutrisiran and Patisiran for Patients with Hereditary Transthyretin Amyloidosis https://clinicaltrials.eu/trial/study-on-the-effectiveness-and-safety-of-vutrisiran-and-patisiran-for-patients-with-hereditary-transthyretin-amyloidosis/ Wed, 29 Apr 2026 14:28:27 +0000 https://clinicaltrials.eu/trial/study-on-the-effectiveness-and-safety-of-vutrisiran-and-patisiran-for-patients-with-hereditary-transthyretin-amyloidosis/ This clinical trial is focused on studying a condition known as Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis). This is a rare genetic disease where abnormal proteins build up in the body, leading to nerve damage and other health issues. The study is evaluating a treatment called ALN-TTRSC02, which is also known by its code name. This treatment is designed to help reduce the effects of the disease on the nervous system.

The purpose of the study is to assess how effective and safe ALN-TTRSC02 is for patients with hATTR Amyloidosis. Participants in the study will receive the treatment as a solution for injection. The study will also compare the effects of ALN-TTRSC02 with another treatment called Onpattro, which is given as a solution for infusion. Some participants may receive a placebo instead of the active treatment. The study will monitor changes in nerve function and quality of life over time.

Throughout the study, participants will undergo regular assessments to track their progress and any changes in their condition. The study aims to provide valuable information on how ALN-TTRSC02 can help manage the symptoms of hATTR Amyloidosis and improve the lives of those affected by this condition.

]]>