Health TrialsbyHealthPanel

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

ConditionPrion Disease
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorIonis Pharmaceuticals, Inc.

About this trial

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

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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.

Disqualifiers

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.

Trial design

Design model

Parallel

Treatments tested in this trial

  • ION717

    Drug

    ION717 will be administered by IT injection.

  • Placebo

    Drug

    Placebo-matching ION717 will be administered by IT injection.

Treatment groups

76 Participants
are divided into 3 treatment groups
Group A: ION717 + Placebo, Regimen 1Experimental treatment 2 interventions
Group B: ION717 + Placebo, Regimen 2Experimental treatment 2 interventions
Group C: ION717, Regimen 3Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Incidence of treatment-emergent adverse events.

Time frame
Baseline up to Week 33

Secondary outcomes

1

Maximum Observed Plasma Concentration (Cmax) of ION717

Time frame
on Day 1 and Week 9
2

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

Time frame
on Day 1 and Week 9
3

Half-life (t1/2λz) of ION717 in Plasma

Time frame
on Day 1 and Week 9
4

Cerebrospinal fluid (CSF) Concentration of ION717

Time frame
Pre-dose and at multiple points post-dose up to Week 33

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Ionis Pharmaceuticals, Inc.

Lead sponsor

Source ClinicalTrials.gov
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