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NCT07447557 · ClinicalTrials.gov registry record · Phase 1

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

A Phase 1 study, sponsored by Elpida Therapeutics SPC.

Recruiting
Registry status
Phase 1
Development phase
8
Enrollment target

NCT07447557: Recruiting Phase 1 study, sponsored by Elpida Therapeutics SPC.

NCT07447557 is a Phase 1 study that is actively recruiting participants, run by Elpida Therapeutics SPC. The registered enrollment target is 8 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (87% lower). According to ClinicalTrials.gov, the official US trial registry.

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The verdict

NCT07447557, a Phase 1 study, is actively recruiting participants, sponsored by Elpida Therapeutics SPC.

RECRUITING
Registry status
Phase 1
Development phase
8 participants
Enrollment target

Study Summary

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.

Primary Outcome

Incidence and severity of unanticipated treatment-related adverse events graded as Grade 3 or higher will be assessed based on the occurrence of serious adverse events (SAEs) and adverse events (AEs), classified according to Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0.

Interventions

  • GENETIC ELP-02

Trial Details

FieldValue
Enrollment Target 8 participants
Start Date 2026-07
Est. Completion 2032-12
Phase Phase 1
Elpida Therapeutics SPC

4 total trials

What NCT07447557 shows while recruiting

NCT07447557 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 8 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (87% lower).

The record links to 0 conditions, and to 1 intervention - of which ELP-02 is the first listed.

NCT07447557 does not publish any study locations in the registry export this page uses.

Frequently Asked Questions

What is clinical trial NCT07447557 about?

NCT07447557 is a clinical study titled "Study of Intrathecal ELP-02 for Charcot-Marie-Tooth Disease Type 4J (CMT4J)". 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.

What is the current status of trial NCT07447557?

This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 8 participants. The study started on 2026-07. Estimated completion is 2032-12.

What interventions are being tested in trial NCT07447557?

The interventions under investigation include: ELP-02 (GENETIC).

Who is sponsoring clinical trial NCT07447557?

This trial is sponsored by Elpida Therapeutics SPC, which has 4 total clinical trials registered on ClinicalTrials.gov.

How this trial's enrollment target compares

Where NCT07447557's enrollment target sits among peer trials

8 1959th of 2000 higher than 29 of 2,000 other Phase 1 trials

participants (enrollment target), bucketed by value

Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.

Source ClinicalTrials.gov registry export · 2026-08-08

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Source: ClinicalTrials.gov NCT07447557, the US trial registry maintained by the National Library of Medicine. NCT07447557 (small enrollment · none site footprint · recruiting) retrieved and formatted by PlainTrial, see methodology.