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

PHASE1PHASE2ACTIVE_NOT_RECRUITING

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of intrathecal (IT) delivery of ION717.

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Study details:

This is a first-in-human, randomized, multi-center study in participants with prion disease. Eligible participants will receive ION717 and placebo. The study will consist of a screening period of up to 6 weeks, a 30-week double-blind treatment period, a 70-week open-label extension period and a 32-week post-treatment period.

During the double-blind period, the order of ION717 and placebo doses will be randomized and blinded to participants, study sites and the Sponsor. During the open-label extension period, all participants will receive ION717. Multiple dose levels will be tested.

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Eligibility criteria

Researchers look for people who fit a certain description, called eligibility criteria. See if you qualify.

Inclusion criteria

A confirmed diagnosis of probable or definite prion disease.

Early-stage prion disease at the time of Screening.

Willing to meet all study requirements, including travel to Study Center, procedures, measurements and visits.

Patients must have a caregiver who is ≥ 18 years old and who is able and willing to facilitate the patient's involvement, to the best of their ability, for the duration of the trial; caregivers must also be able and willing to provide information about themselves and the patient for the duration of the trial.

Aged ≥ 18 at the time of informed consent.

Exclusion criteria

Clinically significant abnormalities in medical history, laboratory tests or physical examination that would render a patient unsuitable for inclusion.

Any contraindication or unwillingness to undergo an MRI.

Obstructive hydrocephalus, presence of a functional ventriculoperitoneal shunt for the drainage of cerebrospinal fluid (CSF) or an implanted central nervous system (CNS) catheter.

Known brain or spinal disease that would interfere with the LP process, CSF circulation or safety assessment.

Have any other condition, which, in the opinion of the Investigator would make the patient unsuitable for inclusion or could interfere with the patient participating in or completing the study.

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Eligibility

Age eligible for study : 18 and older

Healthy volunteers accepted : No

Gender eligible for study: All

Things to know

Study dates

Study start: 2024-01-04

Primary completion: 2025-07-01

Study completion finish: 2027-07-01

Study type

TREATMENT

Phase

PHASE1

PHASE2

Trial ID

NCT06153966

Intervention or treatment

DRUG: ION717

DRUG: Placebo

Conditions

• Prion Disease

Condition: Prion Disease
DRUG: ION717 and other drugs
Parkville, Not Specified, Australia
Sponsor: Ionis Pharmaceuticals, Inc.

You may be eligible to participate in this trial based on your search. Check eligibility by answering some questions.

Are you running this trial? If you're a clinic or sponsor, you can claim this study. Claim or learn more.

Find a site

Closest Location:

Royal Melbourne Hospital

Research sites nearby

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Royal Melbourne Hospital
Parkville, Not Specified, Australia

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Participant Group/Arm	Intervention/Treatment
EXPERIMENTAL: ION717 + Placebo, Regimen 1 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.	DRUG: ION717 ION717 will be administered by IT injection.
EXPERIMENTAL: ION717 + Placebo, Regimen 2 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.	DRUG: ION717 ION717 will be administered by IT injection.

What is the study measuring?

Primary outcome

Primary Outcome Measure	Primary Outcome Description	Primary Outcome Time Frame
Incidence of treatment-emergent adverse events.	Not Specified	Baseline up to Week 33

Secondary outcome

Secondary Outcome Measure	Secondary Outcome Description	Secondary Outcome Time Frame
Maximum Observed Plasma Concentration (Cmax) of ION717	Not Specified	on Day 1 and Week 9
Area Under the Plasma Concentration-time Curve (AUC) of ION717	Not Specified	on Day 1 and Week 9
Half-life (t1/2λz) of ION717 in Plasma	Not Specified	on Day 1 and Week 9
Cerebrospinal fluid (CSF) Concentration of ION717	Not Specified	Pre-dose and at multiple points post-dose up to Week 33
Amount of ION717 Excreted in Urine	Not Specified	Post-dose on Day 1
Percent Change from Baseline in Prion Protein (PrP) Concentration in CSF	Not Specified	Pre-dose and at multiple points post-dose up to Week 33

Frequently Asked Questions

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You may be eligible to participate in this trial based on your search.Apply for study

Are you running this trial? If you're a clinic or sponsor, you can claim this study.Claim this trial

References

Clinical Trials Gov: PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717