12 Participants Needed

EB-101 for Epidermolysis Bullosa

Recruiting at 1 trial location
AT
Overseen ByAbeona Therapeutics
Age: Any Age
Sex: Any
Trial Phase: Phase 3
Sponsor: Abeona Therapeutics, Inc
Must be taking: Pain medications
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Pivotal Trial (Near Approval)This treatment is in the last trial phase before FDA approval
Prior Safety DataThis treatment has passed at least one previous human trial
Approved in 1 JurisdictionThis treatment is already approved in other countries

Trial Summary

Will I have to stop taking my current medications?

The trial requires that you stay on a stable pain medication regimen for at least 30 days before starting and throughout the study. Other medications are not specifically mentioned, so it's best to discuss with the trial team.

What data supports the effectiveness of the treatment EB-101 for Epidermolysis Bullosa?

Research on similar gene therapies for Epidermolysis Bullosa shows promise, with studies demonstrating long-term restoration of type VII collagen, which is crucial for skin integrity, in preclinical models. These findings suggest that gene delivery methods, like those used in EB-101, can effectively correct genetic defects in skin tissue, potentially improving symptoms in patients.12345

Is EB-101 safe for use in humans?

The treatment involving gene-modified cells, similar to EB-101, was well tolerated in a study with no serious adverse reactions or autoimmune responses observed, suggesting it is generally safe in humans.12345

How is the treatment EB-101 unique for epidermolysis bullosa?

EB-101 is unique because it involves using a patient's own skin cells that are genetically modified to correct the COL7A1 gene mutation, which is responsible for the skin fragility in epidermolysis bullosa. This ex-vivo gene therapy approach aims to restore the production of type VII collagen, a key structural protein, directly addressing the root cause of the condition rather than just alleviating symptoms.13467

What is the purpose of this trial?

To evaluate and further characterize the safety of EB-101 (LZRSE-Col7A1 gene-corrected keratinocyte sheets with type VII collagen \[C7\] expression) for the treatment of large, chronic RDEB wounds in new and previously EB-101 treated patients 12 months and older.

Research Team

AI

Angela Iheanacho, MS

Principal Investigator

Abeona Therapeutics, Inc

SA

Sarah Abdelwahab, MD

Principal Investigator

Abeona Therapeutics, Inc

Eligibility Criteria

This trial is for patients aged 6 and older with Recessive Dystrophic Epidermolysis Bullosa (RDEB) who have stable pain medication regimens and at least one wound larger than 20 cm2 that's been present for over six months. Participants must not be pregnant, use reliable birth control, and cannot have a history of certain allergies or recent investigational therapies.

Inclusion Criteria

I have two confirmed RDEB C7 mutations inherited recessively.
I have a wound larger than 20 cm2, present for over 6 months, and it's a stage 2 wound.
I am using reliable birth control and have a negative pregnancy test.
See 5 more

Exclusion Criteria

I have severe symptoms or lab results, but common issues like throat tightness, low iron, low protein, or pain/itch won't disqualify me.
I am too unwell to travel for treatment or participate in the study.
You are currently addicted to drugs or alcohol.
See 9 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

5 weeks
1 visit (in-person), 1 phone call

Treatment

One-time surgical application of up to 12 autologous, gene-corrected keratinocyte sheets

1 week
Inpatient stay for up to 7 days

Follow-up

Participants are monitored for safety and effectiveness after treatment

24 weeks
Phone call on Day 14; telehealth visits on Weeks 4, 8, and 18; clinic visits on Weeks 12 and 24

Treatment Details

Interventions

  • EB-101
  • Surgical application of RDEB wounds
Trial Overview The trial tests EB-101, which are engineered skin sheets applied surgically to treat wounds caused by RDEB. It aims to assess the safety of this treatment in both new patients and those previously treated with EB-101.
Participant Groups
1Treatment groups
Experimental Treatment
Group I: EB-101 Surgical application of RDEB woundsExperimental Treatment1 Intervention
New or Previously Treated RDEB Patients

EB-101 is already approved in United States for the following indications:

๐Ÿ‡บ๐Ÿ‡ธ
Approved in United States as Prademagene zamikeracel for:
  • Recessive dystrophic epidermolysis bullosa (RDEB)

Find a Clinic Near You

Who Is Running the Clinical Trial?

Abeona Therapeutics, Inc

Lead Sponsor

Trials
12
Recruited
1,100+

Findings from Research

A retroviral vector was successfully used to deliver the wild-type BP180 gene to keratinocytes from patients with junctional epidermolysis bullosa (JEB), restoring BP180 protein expression and normalizing cell adhesion in vitro.
When these genetically modified keratinocytes were used to regenerate skin on immune-deficient mice, the resulting tissue showed restored BP180 expression at the dermal-epidermal junction, indicating potential for future gene therapy approaches in treating JEB.
BP180 gene delivery in junctional epidermolysis bullosa.Seitz, CS., Giudice, GJ., Balding, SD., et al.[2023]
A new gene delivery method using retroviral vectors has shown long-term success in restoring type VII collagen in skin tissue affected by recessive dystrophic epidermolysis bullosa (RDEB), maintaining expression for over a year and through 12 skin turnover cycles.
This restoration corrected key features of RDEB, such as the formation of anchoring fibrils, indicating that this approach is both effective and safe, paving the way for future human clinical trials.
Long-term type VII collagen restoration to human epidermolysis bullosa skin tissue.Siprashvili, Z., Nguyen, NT., Bezchinsky, MY., et al.[2021]
In a phase I trial involving 4 adults with recessive dystrophic epidermolysis bullosa (RDEB), gene-modified fibroblasts were safely administered with no serious adverse or autoimmune reactions observed over 12 months.
The treatment led to a significant increase in type VII collagen (C7) expression in the injected skin of 3 out of 4 subjects, indicating potential efficacy for restoring skin integrity, with sustained effects noted in 2 subjects.
Safety and early efficacy outcomes for lentiviral fibroblast gene therapy in recessive dystrophic epidermolysis bullosa.Lwin, SM., Syed, F., Di, WL., et al.[2022]

References

BP180 gene delivery in junctional epidermolysis bullosa. [2023]
Long-term type VII collagen restoration to human epidermolysis bullosa skin tissue. [2021]
Safety and early efficacy outcomes for lentiviral fibroblast gene therapy in recessive dystrophic epidermolysis bullosa. [2022]
SIN retroviral vectors expressing COL7A1 under human promoters for ex vivo gene therapy of recessive dystrophic epidermolysis bullosa. [2021]
An RNA-targeted therapy for dystrophic epidermolysis bullosa. [2018]
Gene therapy of epidermolysis bullosa. [2023]
Fibroblasts show more potential as target cells than keratinocytes in COL7A1 gene therapy of dystrophic epidermolysis bullosa. [2012]
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