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One-Patient trial hopes to fix rare nerve disease at its genetic root

NCT ID NCT07223632

Disease control Sponsor: Vanda Pharmaceuticals Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Aug 20, 2026 · Updated 2 times

Summary

This study tests a custom-made drug (VCA-894A) in a single person with a rare genetic nerve disease called CMT2S. The drug is designed to correct a specific genetic error and restore a missing protein. The main goals are to check if the treatment is safe and if it can improve muscle function.

Why investors are watching

Vanda Pharmaceuticals is testing VCA-894A, an experimental drug for a rare genetic nerve disease called CMT2S, in a single patient. This small trial matters because a positive safety result could validate the company's approach to treating this condition, which has no approved therapy.

If it works: If the drug proves safe and shows signs of working in this one patient, Vanda could advance to larger studies and potentially become the first company with a treatment for CMT2S.

If it fails: The trial involves only one person, so results may not apply to other patients. If the drug fails or causes harm, Vanda would need to restart its efforts, and the setback could hurt investor confidence in its pipeline.

AI-written from the trial record. Speculative, and not investment advice.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

1 person

The number who actually took part.

Started

May 2025

Expected to finish

Mar 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

Children (under 18), adults (18 to 64) and older adults (65 and over)

Sex

Female participants only

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Ability and acceptance to provide written informed consent. * Genetically confirmed diagnosis of CMT2S with confirmed IGHMBP2 intronic cryptic splice variant c. 1235+894C\>A. Exclusion Criteria: * Significant clinical deterioration of the patient's neurologic status, as judged by the Investigator. * Non-reversible conditions that are contraindications to lumbar puncture. * Pregnancy, recent pregnancy (within 6 weeks), or women who are breastfeeding.

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Conditions

The condition(s) this trial relates to.

Charcot-Marie-Tooth disease Charcot-Marie-Tooth disease axonal type 2S neuromuscular disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Charcot Marie Tooth disease (CMT) Neuromuscular diseases (NMD)

Contacts and locations

Locations

  • Vanda Investigational Site

    Madison, Wisconsin, 53792, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.