What side effects are associated with this type of intervention?

Common treatments for Epidermolysis Bullosa (EB) include antimicrobial agents, wound care, and advanced therapies like stem cell transplantation and gene therapy. Antimicrobial treatments, such as topical antibiotics (e.g., mupirocin, clindamycin), are used to manage infections caused by S. aureus and P. aeruginosa. Known side effects of these treatments can include skin irritation, contact dermatitis, and, rarely, antibiotic resistance. Advanced therapies like stem cell transplantation may lead to severe complications, including graft-versus-host disease and high mortality rates. Gene therapy, while promising, can cause mild adverse effects such as fever, rash, and itching.

What prior FDA approvals exist?

There is no specific mention of prior FDA approvals for treatments of Epidermolysis Bullosa with antimicrobial action against S. aureus and P. aeruginosa in the provided context. However, treatments for EB often involve managing infections and promoting wound healing. For instance, silver-impregnated dressings are used to minimize infection risk in EB wounds. Additionally, other antimicrobial agents like topical clindamycin, erythromycin, and mupirocin are commonly used to treat skin infections, although their specific FDA approval for EB is not detailed in the context.

What other types of research exist?

Promising alternatives to APR-TD011 for Epidermolysis Bullosa patients include: 1) ActiMaris (AM) hypertonic ionised sea water solution, which has shown efficacy in wound cleansing, disinfection, and reducing inflammation. 2) Cornstarch-based wound dressing with hyaluronic acid and propolis, which exhibited significant antibacterial activity against S. aureus and other pathogens, and accelerated wound healing in vivo. 3) Antimicrobial photodynamic therapy (APDT) with RLP068/Cl, which demonstrated significant reduction in bacterial counts and complete re-epithelialization in infected wounds. These alternatives offer effective antimicrobial action and support wound healing.

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Antimicrobial Wound Cleansing Spray

Antimicrobial Wound Cleansing Spray for Epidermolysis Bullosa

Q: What is the mechanism of action of this treatment?

The most common treatments for Epidermolysis Bullosa (EB) include antimicrobial therapies, protein replacement therapy, and gene therapy. Antimicrobial treatments, like the APR-TD011 spray, work by reducing bacterial colonization and infection, particularly targeting pathogens such as Staphylococcus aureus and Pseudomonas aeruginosa, which are common in EB wounds. This is crucial for EB patients as it helps prevent infections that can exacerbate skin damage and delay wound healing. Protein replacement therapy involves delivering recombinant collagen type VII to the skin, which helps restore the structural integrity of the skin in patients with defective or missing collagen. Gene therapy, such as the use of viral vectors to deliver functional genes, aims to correct the underlying genetic defects in EB, promoting long-term skin stability and reducing blister formation. These treatments are significant as they address both the symptoms and the root causes of EB, improving the quality of life for patients.

Phase < 1

Waitlist Available

Research Sponsored by Northwestern University

Eligibility Criteria Checklist

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

Must have

Patients 6 months of age or above with junctional or dystrophic epidermolysis bullosa

Target wound that has been present for at least 3 weeks and is at least 10 cm2

Must not have

Do not agree to avoid dilute bleach or vinegar baths, or other antiseptic use, at the target site starting at screening throughout the study

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 8 weeks

Awards & highlights

No Placebo-Only Group

Summary

This trial tests a special spray to clean and heal infected wounds in patients with a rare skin condition. The spray aims to reduce harmful bacteria, helping the wounds heal better.

Who is the study for?

This trial is for patients aged 6 months or older with junctional or dystrophic epidermolysis bullosa who have a wound at least 10 cm2 in size that's been present for over 3 weeks and is infected with Staphylococcus aureus or Pseudomonas aeruginosa. It excludes those on recent antibiotics, using antiseptics, bleach/vinegar baths, or topical treatments at the target site.

What is being tested?

The study tests APR-TD011 antimicrobial spray on wounds of people with JEB/DEB to see how it affects skin bacteria before, during, and after an 8-week treatment period. Participants will then stop using the spray for four weeks followed by a six-month open-label use phase where they can choose to use the spray as desired.

What are the potential side effects?

Potential side effects are not explicitly listed but may include reactions typical of topical treatments such as skin irritation, redness, itching or allergic reactions due to sensitivity to ingredients in the APR-TD011 spray solution.

Eligibility Criteria

Inclusion Criteria

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

Select...

I am over 6 months old and have junctional or dystrophic epidermolysis bullosa.

Select...

My wound is at least 3 weeks old and covers an area of 10 cm2 or more.

Select...

My wound is infected with Staphylococcus aureus or Pseudomonas aeruginosa.

Exclusion Criteria

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

Select...

I agree not to use dilute bleach, vinegar baths, or other antiseptics on the target site during the study.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 8 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~8 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and 8 weeks for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Change in microbiome species

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: EB participantsExperimental Treatment1 Intervention

Visits will include screening, pre-treatment (week 0), weeks 4 and 8, followed by a visit without use of the APR-TD011 for 4 weeks (week 12) for microbiome assessment off of therapy.

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 antimicrobial therapies, protein replacement therapy, and gene therapy. Antimicrobial treatments, like the APR-TD011 spray, work by reducing bacterial colonization and infection, particularly targeting pathogens such as Staphylococcus aureus and Pseudomonas aeruginosa, which are common in EB wounds. This is crucial for EB patients as it helps prevent infections that can exacerbate skin damage and delay wound healing. Protein replacement therapy involves delivering recombinant collagen type VII to the skin, which helps restore the structural integrity of the skin in patients with defective or missing collagen. Gene therapy, such as the use of viral vectors to deliver functional genes, aims to correct the underlying genetic defects in EB, promoting long-term skin stability and reducing blister formation. These treatments are significant as they address both the symptoms and the root causes of EB, improving the quality of life for patients.

Multidisciplinary care for patients with epidermolysis bullosa from birth to adolescence: experience of one Italian reference center.[Epidermolysis bullosa : Diagnosis and therapy].