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Can a One-Time gene edit stop duchenne? Single-Patient study tests the idea

NCT ID NCT05514249

Cure Sponsor: Cure Rare Disease, Inc 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
Running, but no longer taking on new participants.
Completed This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Oct 20, 2026 · Last updated Oct 21, 2026 · Updated 1 time

Summary

Researchers are testing an experimental gene-editing therapy called CRD-TMH-001 in one person with a rare mutation of Duchenne muscular dystrophy. The participant receives a single intravenous dose. The study's main goal is to measure safety and tolerability by tracking serious and non-serious adverse events. This is an early-stage, single-patient study designed to understand the therapy's effects.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
CRD-TMH-001, an experimental gene-editing therapy given as a single intravenous dose
What this could lead to
If it works, this could point toward a one-time gene-editing treatment for a rare form of Duchenne muscular dystrophy.
What could go wrong
This is a single-patient study, so any result is highly preliminary and may not apply to others. Gene-editing therapies carry risks of immune reactions and unintended genetic changes.

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

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

1 person

The number who actually took part.

Started

Aug 2022

Finished

Oct 2022

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

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

Ages

18 to 28 years

Sex

Male 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: * Completion of informed consent * Confirmation of genetic mutation * Confirmation of absence of elevated AAV9 NAbs Exclusion Criteria: \- Any significant medical issue(s) (past or current) that would, in the opinion of the Principal Investigator (PI), prevent this patient from being dosed.

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Conditions

The condition(s) this trial relates to.

Duchenne muscular dystrophy muscular dystrophy

As listed by the trial registrant

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

Duchenne muscular dystrophy

Contacts and locations

Locations

  • UMass Chan Medical School

    Worcester, Massachusetts, 01655, United States

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