What is the mechanism of action of this treatment?

Common treatments for Epidermolysis Bullosa (EB) often involve cell-based therapies aimed at promoting tissue repair and reducing blister formation. Allogeneic mesenchymal stromal cells (MSCs), such as those in allo-APZ2-OTS, work by homing to damaged tissues, modulating the immune response, and promoting healing through anti-inflammatory and tissue-regenerative properties. Fibroblast injections can reduce erosion areas and accelerate wound healing by enhancing extracellular matrix production. Thymosin beta4, applied topically, aids in wound healing by promoting cell migration and reducing inflammation. These treatments are crucial for EB patients as they address the underlying issues of poor wound healing and chronic blistering, improving quality of life and reducing complications.

What side effects are associated with this type of intervention?

Common treatments for Epidermolysis Bullosa (EB) similar to allo-APZ2-OTS, such as allogeneic mesenchymal stromal cells (MSCs) and other stem cell therapies, have shown potential in promoting wound healing and reducing blister formation. However, these treatments can have side effects, including mild lymphadenopathy, hypersensitivity reactions, and complications related to the procedure itself. In some cases, severe adverse events like mortality have been reported, particularly with aggressive preparative regimens. Further studies are needed to confirm the long-term safety and efficacy of these therapies.

What prior FDA approvals exist?

As of now, there are no specific FDA-approved treatments for Epidermolysis Bullosa that are directly analogous to allo-APZ2-OTS, an allogeneic cell therapy. However, related treatments under investigation include stem cell therapies, such as allogeneic bone marrow transplantation and mesenchymal stem cell (MSC) therapy, which have shown promise in clinical trials for improving wound healing and reducing blister formation in patients with EB. These therapies aim to leverage the regenerative and immunomodulatory properties of stem cells to repair and regenerate damaged tissues.

What other types of research exist?

Promising alternatives to allo-APZ2-OTS for Epidermolysis Bullosa patients include: 1) Allogeneic bone marrow transplantation with nonmyeloablative regimens, which has shown improved wound healing and reduced blistering in clinical trials. 2) Systemic administration of bone marrow-derived nonhematopoietic mesenchymal stem cells (MSCs), specifically ABCB5+ MSCs, which have demonstrated immunomodulatory, anti-inflammatory, and tissue-healing properties. 3) Allogeneic fibroblast injections, which have shown a significant reduction in erosion area and time to healing in randomized controlled trials.

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Mesenchymal Stromal Cells

Stem Cell Therapy for Epidermolysis Bullosa

Phase 3

Recruiting

Research Sponsored by RHEACELL GmbH & Co. KG

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Male or female subjects from 6 months of age with a diagnosis of RDEB confirmed by genetic testing or by a skin biopsy with immunofluorescence mapping

Subject with a target wound meeting the following criteria: 5-50 cm2, ≥ 21 days and < 9 months, no signs of acute infection

Must not have

Any current tumor diseases, including squamous cell carcinoma and basal cell carcinoma

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 12 months

Awards & highlights

Pivotal Trial

Summary

This trial is testing a special cell therapy given through an IV to people with a severe genetic skin condition called RDEB. The therapy aims to help heal and grow new, healthy skin. Researchers are evaluating its safety and effectiveness. Previous tests have shown increased collagen expression and faster wound healing in patients with RDEB.

Who is the study for?

This trial is for males and females from 6 months old with confirmed recessive dystrophic epidermolysis bullosa (RDEB). Participants must have a specific wound type, be in good general health, and understand the study. Women who can have children need a negative pregnancy test and agree to use birth control.

What is being tested?

The trial tests allo-APZ2-OTS, an intravenous treatment for RDEB against a placebo. The goal is to see if it's safe and works better than not receiving the active treatment.

What are the potential side effects?

While specific side effects are not listed here, potential risks may include reactions related to infusion or immune response due to the nature of stem cell treatments.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I have RDEB confirmed by genetic testing or skin biopsy.

Select...

My wound is between 5-50 cm2, older than 21 days but less than 9 months, and not infected.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

I have a current diagnosis of skin cancer.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 12 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~12 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 12 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Proportion of subjects with complete target wound closure

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: VerumExperimental Treatment1 Intervention

Group II: PlaceboPlacebo Group1 Intervention

0 / 3Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for Epidermolysis Bullosa (EB) often involve cell-based therapies aimed at promoting tissue repair and reducing blister formation. Allogeneic mesenchymal stromal cells (MSCs), such as those in allo-APZ2-OTS, work by homing to damaged tissues, modulating the immune response, and promoting healing through anti-inflammatory and tissue-regenerative properties. Fibroblast injections can reduce erosion areas and accelerate wound healing by enhancing extracellular matrix production. Thymosin beta4, applied topically, aids in wound healing by promoting cell migration and reducing inflammation. These treatments are crucial for EB patients as they address the underlying issues of poor wound healing and chronic blistering, improving quality of life and reducing complications.

Can Intralesional Epidermal Growth Factor Reduce Skin Graft Applications in Patients with Diabetic Foot Ulcer?Human cultured epidermis accelerates wound healing regardless of its viability in a diabetic mouse model.Therapeutic effect of the epidermal growth factor on diabetic foot ulcer and the underlying mechanisms.