ASCEND: Safety and Tolerability of ION337 for the Treatment of Dravet Syndrome

Part of paid clinical trials in Ann Arbor, Michigan.

Sponsor
Ionis Pharmaceuticals, Inc.
Study ID
NCT07531745
Phase
PHASE1/PHASE2
Status
Recruiting
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Conditions

  • Dravet Syndrome

Eligibility Criteria

Sex
ALL
Age
2 Years - 12 Years
Healthy Volunteers
Not accepted

Interventions

  • ION337 — DRUG
    ION337 will be administered by ITB injection.

Study Details

The primary purpose of this study is to evaluate the safety and tolerability of ION337 in participants with Dravet syndrome (DS).

Key Dates

Start date
Apr 30, 2026
Status verified
May 2026
Primary completion
Dec 31, 2030
Completion
Dec 31, 2030

Study Design

Enrollment
32 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: Part 1: Single Ascending Dose (SAD): Dose Level 1
    Participants aged 2 to ≤ 12 years will receive a single intrathecal bolus (ITB) injection of ION337.
  • Experimental: Part 1: SAD: Dose Level 2
    Participants aged 2 to ≤ 12 will receive a single dose of ION337.
  • Experimental: Part 1: SAD: Dose Level 3
    Participants aged 2 to ≤ 12 will receive a single dose of ION337.
  • Experimental: Part 1: SAD: Dose Level 4
    Participants aged 2 to ≤ 12 will receive a single dose of ION337.
  • Experimental: Part 2: Multiple Ascending Dose (MAD): Dose Level 1-4
    Only participants who complete Part 1 will be eligible to participate in Part 2. Participants will receive multiple doses of ION337. Participants will begin treatment at the same dose level assigned in Part 1.

Primary Outcome Measure

Parts 1 and 2: Number of Participants with Treatment-emergent Adverse Events (TEAEs) and Serious TEAEs [ Time Frame: Part 1: up to 6 months; Part 2: up to 31 months ]

Central Contacts

Locations (3)

FacilityCityStateZIPSite coordinators
University of Michigan Health SystemAnn ArborMichigan48109-
Mayo ClinicRochesterMinnesota55905-
Le Bonheur Children's HospitalMemphisTennessee38103-

Find similar trials in Ann Arbor, MI

By research site
University of Michigan Health System· Ann Arbor, MIMayo Clinic· Rochester, MNLe Bonheur Children's Hospital· Memphis, TN

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