RecruitingRare disease

Long-Term Safety Study of NTLA-2001 for Patients with Hereditary Transthyretin Amyloidosis with Polyneuropathy or Transthyretin Amyloidosis-Related Cardiomyopathy

Fast replyInvestigationalNo placebo

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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 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.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Initial participation
Participation begins after completing or discontinuing a previous study involving NTLA-2001. Consent for this follow-up study is required.
Commitment to attend study visits and adhere to the follow-up schedule is necessary.
Step 2
Treatment overview
The study involves monitoring the long-term safety of NTLA-2001, a treatment administered through intravenous infusion.
NTLA-2001 is a dispersion for infusion containing ziclumeran, which targets the human TTR gene.
Step 3
Monitoring and assessments
Regular assessments will be conducted to monitor the incidence of treatment-related serious adverse events (SAEs) and protocol-specified clinical events of interest (CEIs).
Secondary assessments include monitoring biomarkers over time, such as serum transthyretin (TTR) and serum prealbumin levels.
Step 4
Study duration
The study is expected to continue until August 23, 2038.
Participants will be required to follow the study protocol throughout this period.

Who can join the trial?

3 criteria

The participant must have completed or stopped participating in a previous study sponsored by Intellia, where they received a full or partial dose of a treatment called NTLA-2001.
The participant must have given their agreement, known as informed consent, to take part in this follow-up study. Informed consent means they understand the study and agree to participate.
The participant must be willing to attend study visits, follow the schedule required by the study, and meet all the study requirements.

Who cannot join the trial?

2 criteria

Patients who have not been previously treated for the condition.
Patients who are part of a vulnerable population, which means groups that might need special protection or care.

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Verified sites

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Hopital Beaujon
Verified
Clichy, France
Region Vaesterbotten
Responsive
Umea, Sweden

Trial locations

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Investigated drugs

NTLA-2001 is a medication being studied for its long-term safety in participants who have already received treatment with it. This medication is part of a clinical trial to understand how it affects patients over an extended period. The goal is to ensure that NTLA-2001 is safe for continued use and to monitor any potential side effects that may arise after the initial treatment phase.

What is already known about the treatment

NTLA-2001 – This medication is administered through an intravenous infusion, allowing it to enter the bloodstream directly. It is currently under investigation in clinical trials, with a focus on its long-term safety for individuals who have already received treatment. The main therapeutic indications for NTLA-2001 include Hereditary Transthyretin Amyloidosis with Polyneuropathy (ATTRv-PN) and Transthyretin Amyloidosis-Related Cardiomyopathy (ATTR-CM). At the molecular level, NTLA-2001 works by targeting and editing specific genes associated with the production of abnormal proteins that cause these conditions. It is classified as a gene-editing therapy, representing a novel approach in the treatment of genetic disorders.

Investigated diseases

Hereditary Transthyretin Amyloidosis with Polyneuropathy (ATTRv-PN)
This is a genetic disorder where abnormal protein deposits, known as amyloid, accumulate in the body's tissues and organs. The condition primarily affects the peripheral nerves, leading to symptoms such as numbness, tingling, and pain in the hands and feet. Over time, it can progress to muscle weakness and loss of reflexes. As the disease advances, it may also impact the autonomic nervous system, causing issues like digestive problems and blood pressure irregularities. The progression of symptoms can vary widely among individuals.
Transthyretin Amyloidosis-Related Cardiomyopathy (ATTR-CM)
This condition involves the buildup of amyloid proteins in the heart tissue, leading to a form of heart disease known as cardiomyopathy. It results in the thickening and stiffening of the heart walls, which can impair the heart's ability to pump blood effectively. Symptoms often include fatigue, shortness of breath, and swelling in the legs and feet. As the disease progresses, it can lead to heart rhythm abnormalities and heart failure. The rate of progression and severity of symptoms can differ among affected individuals.