Recessive Dystrophic Epidermolysis BullosaNCT07700966
A Long-Term Safety Follow-up Registry for Patients Who Received Treatment With Prademagene Zamikeracel for Recessive Dystrophic Epidermolysis Bullosa (RDEB) in the Post-Marketing Setting
An observational study of Recessive Dystrophic Epidermolysis Bullosa, run by Abeona Therapeutics, Inc at 4 sites in United States.
Trial summary
The goal of this observational study is to evaluate the long-term safety and wound healing durability (up to 15 years) for participants with Recessive Dystrophic Epidermolysis Bullosa (RDEB) who were treated with pz-cel. The main questions it aims to answer are: The long-term safety profile of pz-cel. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Recessive Dystrophic Epidermolysis Bullosa
- What is being tested?
- Prademagene Zamikeracel
- How many people
- 250 participants
- Where
- 4 sitesUnited States
- Study dates
- 25 Jul 2025to Apr 2045
- How long will I take part?
- 15 Years
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: RDEB patients who received pz-cel treatment
Who may be able to join
- 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.
Who cannot join
- Inability to properly follow the protocol as determined by the Principal Investigator (PI).
Trial locations and contacts
4 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States4 sites, 4 recruiting
- Recruiting
Children's Hospital Colorado
Aurora, Colorado, 80045
Matthew Brien, 7207770802, matthew.brien@childrenscolorado.org
Anna Bruckner
- Recruiting
Ann & Robert H. Lurie Children's Hospital of Chicago - Department of Dermatology
Chicago, Illinois, 60611
Alyssa Appleman, 3125035927, alyssa.appleman@northwestern.edu
Amy Paller
- Recruiting
University of Texas Medical Branch
Galveston, Texas, 77555
Lindsay Allen, 4092662678, listurm@utmb.edu
Lindy Ross
- Recruiting
Lucile Packard Children's Hospital
Palo Alto, California, 94304
Thomas Buschbacher, 650-313-0354, tbusch@stanford.edu
Joyce Teng
About this study
The goal of this observational study is to evaluate the long-term safety and wound healing durability (up to 15 years) for participants with Recessive Dystrophic Epidermolysis Bullosa (RDEB) who were treated with pz-cel. The main questions it aims to answer are:
- The long-term safety profile of pz-cel.
- The wound healing durability of pz-cel.
- The occurrence of all malignancies after treatment with pz-cel, regardless of type or location, in individuals who received pz-cel treatment for RDEB in the post-marketing setting.
Read more
Participants will then be followed annually up to 15 years post-treatment. Assessments will occur via remote data collection. If the participants provider determines an office visit is necessary after the first year, some of this data can be collected in person as well. Annual assessments will include, but are not limited to:
- Review of Adverse Events (AEs), Serious Adverse Events (SAEs), hospitalizations, and any evidence of malignancies and potential retroviral infection
- Wound-healing durability data collection via the modified Caregiver Global Impression of Change for Wound Healing
- Any changes in concomitant medications and procedures.
- Being tested
- Prademagene Zamikeracel
- Medical subject headings
- Epidermolysis Bullosa Dystrophica
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Prademagene ZamikeracelCombination product
Also called EB-101, Pz-cel
ZEVASKYN® (prademagene zamikeracel) is a type of gene therapy made from your own skin cells that is surgically applied to treat wounds in both children and adults who have recessive dystrophic epidermolysis bullosa (RDEB).
| Study group | Role | What they receive |
|---|---|---|
| Patients with RDEB who received pz-cel treatment in the post-marketing setting | Not described Receives: Combination Product: Prademagene Zamikeracel |
What is being measured
The primary outcome is the main question the study is designed to answer.
The number and incidence of treatment-related malignancies.
Measured: From enrollment to 15 years post-treatment
The number and incidence of treatment-emergent SAEs, including systematic and wound specific SAEs
Measured: From enrollment to 15 years post-treatment
The number and incidences of treatment-related SAEs related to pz-cel, including systematic and wound-specific SAEs.
Measured: From enrollment to 15 years post-treatment
The number and incidence of positive RCR testing results required for AEs and SAEs where retroviral infection is a consideration
Measured: From enrollment to 15 years post-treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Jul 2026
- Study start25 Jul 2025
- Primary completionApr 2045 (estimated)
- Study completionApr 2045 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Recessive Dystrophic Epidermolysis BullosaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 250 participants (estimated)
- Target duration
- 15 Years
Sponsor and investigators
- Lead sponsor
- Abeona Therapeutics, Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07700966
- Sponsor's protocol ID
- Pz-cel-RY-401
- Status last verified
- Jul 2026
- First submitted
- 8 Jul 2026
- Last updated
- 14 Jul 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Long-Term Safety Follow-up Registry for Patients Who Received Treatment With Prademagene Zamikeracel for Recessive Dystrophic Epidermolysis Bullosa (RDEB) in the Post-Marketing Setting
- ClinicalTrials.gov ID
- NCT07700966
- Acronym / sponsor ID
- Pz-cel-RY-401
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 250 participants (estimated)
- Lead sponsor
- Abeona Therapeutics, IncIndustry (pharma, biotech, devices)
- Study start
- 25 Jul 2025 (actual)
- Primary completion
- Apr 2045 (estimated)
- Study completion
- Apr 2045 (estimated)
- First posted
- 14 Jul 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The goal of this observational study is to evaluate the long-term safety and wound healing durability (up to 15 years) for participants with Recessive Dystrophic Epidermolysis Bullosa (RDEB) who were treated with pz-cel. The main questions it aims to answer are:
- The long-term safety profile of pz-cel.
- The wound healing durability of pz-cel.
- The occurrence of all malignancies after treatment with pz-cel, regardless of type or location, in individuals who received pz-cel treatment for RDEB in the post-marketing setting.
Participants will then be followed annually up to 15 years post-treatment. Assessments will occur via remote data collection. If the participants provider determines an office visit is necessary after the first year, some of this data can be collected in person as well. Annual assessments will include, but are not limited to:
- Review of Adverse Events (AEs), Serious Adverse Events (SAEs), hospitalizations, and any evidence of malignancies and potential retroviral infection
- Wound-healing durability data collection via the modified Caregiver Global Impression of Change for Wound Healing
- Any changes in concomitant medications and procedures.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Target follow-up
- 15 Years
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Patients with RDEB who received pz-cel treatment in the post-marketing setting | – | Combination Product: Prademagene Zamikeracel |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- RDEB patients who received pz-cel treatment
Key inclusion criteria (2 listed)
- 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.
Key exclusion criteria (1 listed)
- Inability to properly follow the protocol as determined by the Principal Investigator (PI).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Prademagene ZamikeracelAlso: EB-101, Pz-celZEVASKYN® (prademagene zamikeracel) is a type of gene therapy made from your own skin cells that is surgically applied to treat wounds in both children and adults who have recessive dystrophic epidermolysis bullosa (RDEB). | Combination product | Patients with RDEB who received pz-cel treatment in the post-marketing setting |
Primary outcome
The number and incidence of treatment-related malignancies.
Time frame: From enrollment to 15 years post-treatment
The number and incidence of treatment-emergent SAEs, including systematic and wound specific SAEs
Time frame: From enrollment to 15 years post-treatment
The number and incidences of treatment-related SAEs related to pz-cel, including systematic and wound-specific SAEs.
Time frame: From enrollment to 15 years post-treatment
The number and incidence of positive RCR testing results required for AEs and SAEs where retroviral infection is a consideration
Time frame: From enrollment to 15 years post-treatment
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Jul 2026.
- Individual participant dataSharing planned: No
Related trials for Recessive Dystrophic Epidermolysis Bullosa
Not yet recruitingPhase 4
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A Long-Term Extension Study for Participants Previously Treated With EB-101 for the Treatment of RDEB
RDEB
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Treatment of Dysphagia in Patients With Dystrophic Epidermolysis Bullosa With Budesonide: a Retrospective Bicentric Study Nice - Paris St Louis / Necker (EBUDE Study)
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Targeting Collagen VII Antibodies With IV IgG in Dystrophic Epidermolysis Bullosa
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Targeting Collagen VII Antibodies in Bullous Diseases Using Efgartigimod IV (VYVGART)
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Recessive Dystrophic Epidermolysis Bullosa, run by Abeona Therapeutics, Inc at 4 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 14 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Angela Iheanacho (646-813-7166, aiheanacho@abeonatherapeutics.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The number and incidence of treatment-related malignancies. (time frame: From enrollment to 15 years post-treatment). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Jul 2026.