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

Conditions

• Prion Disease

Eligibility Criteria

Sex

ALL

Age

18 Years - N/A

Healthy Volunteers

Not accepted

Interventions

• ION717 — DRUG
  ION717 will be administered by IT injection.
• Placebo — DRUG
  Placebo-matching ION717 will be administered by IT injection.

Study Details

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

Key Dates

Start date

Jan 4, 2024

Status verified

Apr 2026

Primary completion

Feb 28, 2027

Completion

Jun 30, 2030

Study Design

Enrollment

76 participants (estimated)

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Experimental: ION717 + Placebo, Regimen 1
  Participants will receive multiple doses of study drug (ION717 and placebo) during the 30-week treatment period; the order of doses is blinded. Participants will then receive multiple doses of ION717 during the 142-week open-label extension period.
• Experimental: ION717 + Placebo, Regimen 2
  Participants will receive multiple doses of study drug (ION717 and placebo) during the 30-week treatment period; the order of doses is blinded. Participants will then receive multiple doses of ION717 during the 142-week open-label extension period.
• Experimental: ION717, Regimen 3
  Participants will receive multiple doses of ION717 during the 30-week treatment period and the 142-week open-label extension period.

Primary Outcome Measure

Incidence of treatment-emergent adverse events. [ Time Frame: Baseline up to Week 33 ]

Central Contacts

• Ionis Pharmaceuticals, Inc.
  (844) 892-5665
  [email protected]

Locations (3)

Find similar trials in Boston, MA

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