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Placebo for Prion Diseases

Phase 1 & 2

Waitlist Available

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 baseline up to week 29

Awards & highlights

Study Summary

This trial will test the safety and effectiveness of delivering ION717 directly into the spinal canal.

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.Check my eligibility

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).See study design

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 ~ baseline up to week 29

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline up to week 29

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline up to week 29 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Incidence of treatment-emergent adverse events.

Secondary outcome measures

Amount of ION717 Excreted in Urine

Area Under the Plasma Concentration-time Curve (AUC) of ION717

Cerebrospinal fluid (CSF) Concentration of ION717

+3 more

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 24-week treatment period. The order of doses is blinded.

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

Participants will receive multiple doses of study drug (ION717 and placebo) during the 24-week treatment period. The order of doses is blinded.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Placebo

1995

Completed Phase 3

~2670

0 / 5Eligibility criteria met

Find a Location

Who is running the clinical trial?

Ionis Pharmaceuticals, Inc.Lead Sponsor

146 Previous Clinical Trials

15,226 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are there ongoing efforts to enlist individuals for participation in this current research study?

"Indeed, as per the information available on clinicaltrials.gov, this ongoing study is actively seeking individuals to participate. The initial posting of the trial was on December 1st, 2023 and it has been recently updated on December 19th, 2023. The research team aims to recruit a total of 56 patients from one designated location."

Answered by AI

What is the current number of individuals being recruited for participation in this research endeavor?

"Indeed, the details provided on clinicaltrials.gov indicate that this investigation is actively seeking eligible participants. The initial posting of the study occurred on December 1st, 2023 and it was most recently updated on December 19th, 2023. The research team aims to recruit a total of 56 patients from a single location."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717.

2024. "PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717.". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT06153966.

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