Study of Intrathecal ELP-02 for Charcot-Marie-Tooth Disease Type 4J (CMT4J)

Conditions

• Charcot-Marie-Tooth Disease Type 4J

Eligibility Criteria

Sex

ALL

Age

3 Years - 20 Years

Healthy Volunteers

Not accepted

Interventions

• ELP-02 — GENETIC
  ELP-02 is an AAV9-based gene therapy vector that expresses the fully functional form of FIG4 under the control of a synthetic promoter. ELP-02 will be delivered intrathecally and is designed to achieve stable, potentially life-long expression of FIG4 in non-dividing cells.

Study Details

First-in-human Phase 1/2, open-label study to evaluate the safety, tolerability, and efficacy of a single lumbar intrathecal dose of ELP-02 to individuals with CMT4J.

Key Dates

First listed

Mar 3, 2026

Start date

Jul 31, 2026

Status verified

Jul 2026

Primary completion

Dec 31, 2032

Completion

Dec 31, 2032

Study Design

Enrollment

8 participants (estimated)

Allocation

NA

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Arms

• Experimental: Treatment Arm
  Single lumbar intrathecal delivery of ELP-02

Primary Outcome Measure

Incidence of Grade 3 or Higher Unanticipated Treatment-Related Adverse Events [ Time Frame: 60 months ]

Central Contacts

• Rachel Thomas, RN
  1-833-335-7432
  [email protected]

Locations (2)

Find similar trials in Palo Alto, CA