What side effects are associated with this type of intervention?

Common treatments for Epidermolysis Bullosa, particularly those targeting the COL7A1 gene, include gene therapy, protein replacement therapy, and the use of ABCB5+ mesenchymal stromal cells (MSCs). Gene therapy using autologous keratinocytes transduced with a retroviral vector containing the COL7A1 gene has shown no serious adverse events, though transient tissue-bound and circulating antibodies to type VII collagen were detected in one patient. Protein replacement therapy with recombinant collagen type VII has demonstrated potential but requires further research to confirm its safety. Treatment with ABCB5+ MSCs has been associated with mild lymphadenopathy and hypersensitivity reactions, which resolved upon treatment withdrawal.

What prior FDA approvals exist?

The FDA has granted orphan drug designation for several innovative treatments targeting the COL7A1 gene mutation in Epidermolysis Bullosa. Notably, gene therapy using ex vivo-expanded autologous keratinocytes transduced with a retroviral vector containing the COL7A1 gene (EB-101) has shown promise in clinical trials. Additionally, protein replacement therapy delivering recombinant collagen type VII has been evaluated in preclinical studies and represents a promising pathway toward clinical application. These therapies aim to address the underlying genetic defects and improve clinical outcomes for patients with dystrophic forms of EB.

What other types of research exist?

Promising alternatives to PTW-002 for Epidermolysis Bullosa patients include: 1) ABCB5+ mesenchymal stem cells (allo-APZ2-EB), which have shown reductions in disease activity and symptoms in a phase 1/2 trial. 2) Protein replacement therapy using recombinant collagen type VII, which has demonstrated the ability to restore collagen function and improve skin integrity in preclinical studies. 3) Gene therapy with ex vivo-expanded autologous keratinocytes transduced with a retroviral vector containing the COL7A1 gene (EB-101), which has shown promising results in wound healing and collagen expression in a phase 1/2 clinical trial.

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Topical Agent

PTW-002 Gel for Epidermolysis Bullosa

Phase 1 & 2

Waitlist Available

Research Sponsored by Phoenicis Therapeutics

Eligibility Criteria Checklist

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

Must have

Must not have

History of skin-based gene therapy to the TWA.

Use of aminoglycosides, by any route of administration, except eye drops, 7 days or 5 half-lives, whichever is longer, prior to Baseline visit.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Summary

This trial is testing a new treatment called PTW-002 for patients with a rare skin condition caused by a genetic mutation. The treatment aims to strengthen their skin and reduce blistering.

Who is the study for?

This trial is for children and adults with Dystrophic Epidermolysis Bullosa (DEB) due to specific gene mutations. Participants must have a caregiver, no signs of local infection in the target wound area, and agree to use contraception if applicable. Excluded are those with certain treatments or conditions that could affect safety or study results.

What is being tested?

The study tests PTW-002 gel against a placebo in patients with DEB. It's double-blind, meaning neither participants nor researchers know who gets the real treatment versus placebo. The goal is to assess safety, how well it works, and its effects on the body.

What are the potential side effects?

While not explicitly listed here, potential side effects may include reactions at the application site such as redness or irritation since PTW-002 is a topical medication being tested for skin conditions.

Eligibility Criteria

Exclusion Criteria

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

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I have had gene therapy applied to my skin.

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I haven't taken aminoglycoside antibiotics, except as eye drops, for 7 days or 5 half-lives before my baseline visit.

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I haven't used any experimental drugs or devices recently, nor plan to during the study.

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I have had cell therapy that needed specific medication.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: PTW-002 10 mg/g gelExperimental Treatment1 Intervention

PTW-002 poloxamer hydrogel for topical administration (cutaneous use), 10 mg/g gel

Group II: PlaceboPlacebo Group1 Intervention

Matching placebo poloxamer hydrogel for topical administration (cutaneous use)

0 / 4Eligibility 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

The most common treatments for Epidermolysis Bullosa (EB) include gene therapy, protein replacement therapy, and cell-based therapies. Gene therapy aims to correct the underlying genetic mutations, such as those in the COL7A1 gene, by introducing functional copies of the gene into the patient's cells, thereby restoring the production of type VII collagen essential for skin stability. Protein replacement therapy involves delivering recombinant type VII collagen to the skin to compensate for the defective or missing protein, improving skin adhesion and reducing blistering. Cell-based therapies, such as the use of allogeneic fibroblasts or mesenchymal stem cells, focus on enhancing skin repair and collagen deposition at the dermal-epidermal junction. These treatments are crucial for EB patients as they address the root cause of the disease, potentially offering long-term relief and improved quality of life.

Gene Replacement Therapies for Genodermatoses: A Status Quo.Small molecule drug development for rare genodermatoses - evaluation of the current status in epidermolysis bullosa.Molecular therapies for inherited epidermolysis bullosa.