Cure My Disease

Don't miss your cure!

 Log in / Sign up

Gene therapy for 'Butterfly Skin' disease tracked for 15 years in major safety registry

NCT ID NCT07700966

Disease control Sponsor: Abeona Therapeutics, Inc Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
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
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jul 14, 2026 · Last updated Jul 15, 2026 · Updated 1 time

Summary

This registry follows people with recessive dystrophic epidermolysis bullosa (RDEB) who have been treated with prademagene zamikeracel, a gene therapy made from their own skin cells and surgically applied to wounds. The study monitors long-term safety and wound healing durability for up to 15 years after treatment. Participants are assessed annually, either remotely or in person, to track side effects, malignancies, and how well wounds heal over time.

What this could mean

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

Active substance
a gene therapy made from the patient's own skin cells, surgically applied to wounds
What this could lead to
If successful, this registry could confirm that prademagene zamikeracel provides durable wound healing and a favorable long-term safety profile for people with RDEB.
What could go wrong
This is an observational registry, not a controlled trial, so it cannot prove cause and effect. Long-term risks, including malignancies, are still being evaluated.

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.

Participants

About 250 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jul 2025

Expected to finish

Apr 2045

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.

Who is studied

RDEB patients who received pz-cel treatment

Ages

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

Sex

Anyone

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: * Willing and able to give consent/assent; if under the age of 18, guardian(s) is(are) willing and able to give consent. * Patients who received treatment with prademagene zamikeracel in the post-marketing setting. Exclusion Criteria: • Inability to properly follow the protocol as determined by the Principal Investigator (PI).

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for Recessive dystrophic epidermolysis bullosa are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

Conditions

The condition(s) this trial relates to.

epidermolysis bullosa dystrophica recessive dystrophic epidermolysis bullosa

As listed by the trial registrant

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

Recessive dystrophic epidermolysis bullosa

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    4 sites. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Ann & Robert H. Lurie Children's Hospital of Chicago - Department of Dermatology

    RECRUITING

    Chicago, Illinois, 60611, United States

  • Children's Hospital Colorado

    RECRUITING

    Aurora, Colorado, 80045, United States

  • Lucile Packard Children's Hospital

    RECRUITING

    Palo Alto, California, 94304, United States

  • University of Texas Medical Branch

    RECRUITING

    Galveston, Texas, 77555, United States

More trials for these conditions

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