What is the mechanism of action of this treatment?

The most common treatments for Prion Diseases focus on reducing the accumulation of misfolded prion proteins, which are central to the disease's pathology. Approaches include the use of antisense oligonucleotides (ASOs) and RNA interference (RNAi) to silence the expression of prion protein genes, thereby reducing the production of the pathogenic proteins. Intrathecal delivery, as studied in the ION717 trial, allows for direct administration to the central nervous system, potentially increasing treatment efficacy. These mechanisms are crucial for Prion Diseases patients because they target the root cause of the disease, aiming to halt or slow its progression, which is vital given the rapid and fatal nature of these conditions.

What side effects are associated with this type of intervention?

Common treatments for prion diseases, such as supportive care and investigational therapies, often focus on symptom management rather than curing the disease. Side effects of these treatments can vary. For instance, intrathecal delivery of medications, similar to the method used in the ION717 trial, can cause side effects such as headaches, nausea, and potential infections at the injection site. Other investigational treatments may include neuroprotective agents, which can have side effects like dizziness, fatigue, and gastrointestinal disturbances. Overall, the side effects depend on the specific treatment modality and the patient's overall health condition.

What prior FDA approvals exist?

There are no specific FDA-approved treatments for prion diseases mentioned in the provided context. Prion diseases are typically managed with supportive care, as there are no definitive treatments available. Research is ongoing to find effective therapies, including those that may involve mechanisms similar to ION717, such as targeted delivery methods.

What other types of research exist?

Promising novel alternatives to ION717 for prion diseases include pentosan polysulphate, which has shown potential in attenuating prion-related injury, and antisense oligonucleotide therapies, which are being explored for various neurodegenerative diseases. Discuss these options with your healthcare provider to determine their suitability and availability in 2024.

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ION717 for Prion Diseases

Phase 1 & 2

Recruiting

Research Sponsored by Ionis Pharmaceuticals, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Aged ≥ 18 at the time of informed consent.

A confirmed diagnosis of probable or definite prion disease.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Summary

This trial involves injecting a drug called ION717 into the spinal fluid of people with prion disease. The goal is to see how the drug behaves in the body and its effects on the disease. Prion disease has few treatment options, making this study important.

Who is the study for?

This trial is for adults over 18 with early-stage prion disease, confirmed as probable or definite. Participants need to commit to the study schedule and travel requirements. They must have a caregiver willing to support their involvement throughout the trial.

What is being tested?

The study is testing ION717, delivered directly into the spinal fluid (intrathecal delivery), against a placebo. It aims to assess how safe it is, how well tolerated by patients, its movement in the body (pharmacokinetics), and its effects on prion disease (pharmacodynamics).

What are the potential side effects?

While specific side effects are not listed here, common ones from intrathecal drug delivery may include headache, back pain, nausea, fever or infection at injection site. The safety profile of ION717 will be closely monitored.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am 18 years old or older.

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I have been diagnosed with prion disease.

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It seems like the provided text is incomplete. Could you please provide more details or context so I can assist you accurately?

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My condition is in the early stages of prion disease.

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I have a caregiver over 18 who can help me throughout the trial.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Trial Design

2Treatment groups

Experimental Treatment

Group I: ION717 + Placebo, Regimen 2Experimental Treatment2 Interventions

Participants will receive multiple doses of study drug (ION717 and placebo) during the 30-week double-blind treatment period; the order of doses is blinded. Participants will then receive multiple doses of ION717 during the 70-week open-label extension period.

Group II: ION717 + Placebo, Regimen 1Experimental Treatment2 Interventions

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Placebo

1995

Completed Phase 3

~2670

0 / 5Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Prion Diseases focus on reducing the accumulation of misfolded prion proteins, which are central to the disease's pathology. Approaches include the use of antisense oligonucleotides (ASOs) and RNA interference (RNAi) to silence the expression of prion protein genes, thereby reducing the production of the pathogenic proteins. Intrathecal delivery, as studied in the ION717 trial, allows for direct administration to the central nervous system, potentially increasing treatment efficacy. These mechanisms are crucial for Prion Diseases patients because they target the root cause of the disease, aiming to halt or slow its progression, which is vital given the rapid and fatal nature of these conditions.