A Double-blind Study Evaluating the Efficacy, Safety, and Tolerability of Zorevunersen in Patients With Dravet Syndrome

Conditions

• Dravet Syndrome

Eligibility Criteria

Sex

ALL

Age

2 Years - 17 Years

Healthy Volunteers

Not accepted

Interventions

• zorevunersen — DRUG
  Treatment Period 1: Zorevunersen group will receive study drug by intrathecal (IT) administration on Day 1 (after the 8-week Baseline Period), Day 57 (Week 8), Day 169 (Week 24), and Day 281 (Week 40) at a dose level of 70 mg on Day 1 and Day 57, and 45 mg on Day 169 and Day 281. Treatment Period 2: Group assigned to zorevunersen in Treatment Period 1 will receive 45 mg of zorevunersen on Day 393 (Week 56), Day 477 (Week 68), and Day 589 (Week 84).
• Sham Comparator — OTHER
  Treatment Period 1: Sham group will not have drug administered. Sham group will have a procedure intended to mimic the drug administration. Treatment Period 2: Group assigned to sham in Treatment Period 1 will receive 70 mg of zorevunersen on Day 393 (Week 56) and on Day 477 (Week 68), and 45 mg of zorevunersen Day 589 (Week 84).

Study Details

The purpose of the study is to evaluate the efficacy, safety, and tolerability of zorevunersen in Patients with Dravet syndrome.

Key Dates

First listed

Mar 12, 2025

Start date

Jun 4, 2025

Status verified

Jun 2026

Primary completion

Mar 31, 2027

Completion

Oct 31, 2028

Study Design

Enrollment

170 participants (estimated)

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Experimental: Zorevunersen
  Eligible patients will be randomly assigned in a 1:1 ratio to zorevunersen:sham in Treatment Period 1 (approximately 52 weeks). Upon the completion of Treatment Period 1 all eligible patients, will enter Treatment Period 2 and receive zorevunersen, regardless of initial treatment assignment.
• Sham Comparator: Sham Comparator
  Eligible patients will be randomly assigned in a 1:1 ratio to zorevunersen:sham

Primary Outcome Measure

Measurement of Seizure Change [ Time Frame: Week 28 ]

Central Contacts

• Emperor Information Center
  1-781-430-8200
  [email protected]

Locations (28)

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