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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

ConditionRecessive Dystrophic Epidermolysis Bullosa
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeNot listed
SponsorAbeona Therapeutics, Inc

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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.

Disqualifiers

None

Trial population

RDEB patients who received pz-cel treatment

Trial design

Design model

Case-only

Time perspective

Prospective

Treatments tested in this trial

  • Prademagene Zamikeracel

    Combination product

    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).

Treatment groups

250 Participants
are divided into 1 treatment group
Group A: Patients with RDEB who received pz-cel treatment in the post-marketing setting1 intervention

Trial outcomes

Primary outcomes

1

The number and incidence of treatment-related malignancies.

Time frame
From enrollment to 15 years post-treatment
2

The number and incidence of treatment-emergent SAEs, including systematic and wound specific SAEs

Time frame
From enrollment to 15 years post-treatment
3

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
4

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

Secondary outcomes

Other outcomes

1

Categorical impression of change in wound healing over time via the modified Caregiver Global Impression of Change for Wound Healing (mCrGI-WH)

Time frame
Yearly, from 1- year post-treatment to 15- years post-treatment

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Abeona Therapeutics, Inc

Lead sponsor

Source ClinicalTrials.gov
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