PTW-002 Gel for Epidermolysis Bullosa

Not currently recruiting at 2 trial locations

Overseen By Ramsey Johnson

Age: Any Age

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Phoenicis Therapeutics

Must not be taking: Aminoglycosides, Anticoagulants

Disqualifiers: Pregnancy, Carcinoma, Hepatic disease, others

Compensation: Varies

Number of Visits: Unknown

Duration: 8 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new gel, PTW-002, for treating Dystrophic Epidermolysis Bullosa (DEB), a rare skin condition that causes fragile skin prone to blistering. The study aims to determine the gel's safety, mechanism, and potential to improve the condition. Participants will receive either the active gel or a placebo gel that looks identical but lacks the active ingredient. The trial seeks individuals with DEB due to a specific genetic mutation and a wound that shows healing activity without infection. As a Phase 1, Phase 2 trial, this research focuses on understanding the treatment's effects in people and measuring its effectiveness in an initial, smaller group, offering participants a chance to contribute to groundbreaking research.

Will I have to stop taking my current medications?

The trial requires that you stop using aminoglycosides (a type of antibiotic) at least 7 days or 5 half-lives before the study starts, except for eye drops. If you're using any investigational drugs or devices, you must stop them 28 days or 5 half-lives before the study begins.

Is there any evidence suggesting that PTW-002 gel is likely to be safe for humans?

Research has shown that a similar treatment to PTW-002 Gel, called SD-101 cream, was tested for safety in people with epidermolysis bullosa. That study found SD-101 cream to be generally safe.

PTW-002 Gel is now undergoing early trials, where researchers focus heavily on safety. Its progression to this stage indicates some safety in earlier tests, but it remains under close observation for any issues.

Prospective trial participants should know that researchers carefully monitor any possible side effects or reactions. This vigilance helps ensure participant safety while scientists gather more information. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

PTW-002 Gel is unique because it offers a new poloxamer hydrogel delivery method for treating Epidermolysis Bullosa, a condition that currently sees limited effective topical treatments. Unlike standard care options that might involve wound care and pain management, PTW-002 is designed to be directly applied to the skin, which could potentially offer localized treatment with fewer systemic side effects. Researchers are particularly excited about PTW-002 because its direct application could enhance the healing process of fragile skin, offering a targeted approach that might improve the quality of life for patients with this challenging condition.

What evidence suggests that PTW-002 gel might be an effective treatment for Dystrophic Epidermolysis Bullosa?

Research suggests that PTW-002 gel, which participants in this trial may receive, might help treat Dystrophic Epidermolysis Bullosa (DEB), a condition where the skin is fragile and easily blisters. Studies have shown that PTW-002 fixes specific genetic issues related to this skin problem. The gel is applied directly to the skin, and early results indicate it could help wounds heal by strengthening the skin. Although more human data is needed, its mechanism appears promising for addressing the root cause of DEB. ^{1 2 3 6 7}

Who Is on the Research Team?

Hal Landy, MD

Principal Investigator

Phoenicis Therapeutics

Are You a Good Fit for This Trial?

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.

Inclusion Criteria

Patients, and/or their legal guardian(s), if the patient is under the legal age of consent, must provide written Informed Consent or Assent, in accordance with national and/or local laws, prior to the conduct of any study related procedures. In addition, if applicable, a minor child must provide informed Assent in accordance with national and/or local laws and in compliance with the recommendations of the approving Institutional Review Board.

Have a caregiver or support person available, who can follow study instructions in compliance with the protocol and attend study site visits with the patient as required, in the opinion of the Investigator.

I have RDEB or DDEB with a specific genetic mutation.

Exclusion Criteria

I have had gene therapy applied to my skin.

I have not had cancer in the TWA or any cancer in the last 5 years, except for treated skin cancer.

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

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive multiple doses of PTW-002 or placebo to evaluate safety, proof of mechanism, preliminary efficacy, and systemic exposure

8 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• PTW-002

Trial Overview 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.

How Is the Trial Designed?

Group I: PTW-002 10 mg/g gel Experimental Treatment 1 Intervention

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

Group II: Placebo Placebo Group 1 Intervention

Matching placebo poloxamer hydrogel for topical administration (cutaneous use)

Who Is Running the Clinical Trial?

Phoenicis Therapeutics

Lead Sponsor

Trials

3

Recruited

60+

Published Research Related to This Trial

In a study involving 20 subjects with mild to moderate atopic dermatitis, a water-based hydrogel vehicle significantly improved skin hydration compared to a moisturizing lotion (Eucerin Lotion®).

The hydrogel did not worsen transepidermal water loss (TEWL), while the comparator lotion actually increased TEWL, indicating that the hydrogel is a suitable option for preserving epidermal barrier function in patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Transepidermal water loss (TEWL) and corneometry with hydrogel vehicle in the treatment of atopic dermatitis: a randomized, investigator-blind pilot study. Kircik, LH.[2012]

The study developed a mixed micelles-based hydrogel (GL-MMs-gel) for delivering glycyrrhizic acid (GL) topically, which showed significantly improved skin permeability compared to a traditional GL solution-based gel (GL-sol-gel).

In a mouse model of atopic dermatitis, GL-MMs-gel was more effective in reducing symptoms such as skin lesions and inflammation, indicating its potential as a superior treatment option for atopic dermatitis.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Topical delivery of pluronic F127/TPGS mixed micelles-based hydrogel loaded with glycyrrhizic acid for atopic dermatitis treatment. Shen, C., Shen, B., Zhu, J., et al.[2022]

In a study involving 19 patients with various types of epidermolysis bullosa, 90% of hospitalized patients showed significant improvement, with a 76% reduction in blister numbers during their stay.

While the treatment led to accelerated healing of blisters for about 50% of outpatients over 5-11 months, it had limited long-term effects on preventing new blister formation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Epidermolysis bullosa. Assessment of a treatment regimen. Haber, RM., Ramsay, CA., Boxall, LB.[2019]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT05529134

Study of PTW-002 in Patients With Dominant or Recessive ... A double-blind, randomized, intra-patient placebo- controlled, multiple dose study of PTW-002 evaluating safety, proof of mechanism, preliminary efficacy, and ...

2. withpower.com withpower.com/trial/phase-2-epidermolysis-bullosa-9-2022-a38d2

PTW-002 Gel for Epidermolysis Bullosa · Info for Participants The trial requires that you stop using aminoglycosides (a type of antibiotic) at least 7 days or 5 half-lives before the study starts, except for eye drops.

3. medthority.com medthority.com/clinical-trials/epidermolysis-bullosa/study-of-ptw-002-in-patients-with-dominant-or-recessive-dystrophic-epidermolysis-bullosa-due-to-mutations-in-exon-73-of-the-col7a1-gene/

Study of PTW-002 in Patients With Dominant or Recessive ... A double-blind, randomized, intra-patient placebo- controlled, multiple dose study of PTW-002 evaluating safety, proof of mechanism, ...

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10277004/

Innovations in the Treatment of Dystrophic Epidermolysis ... In this review, we provide an overview and update on various therapeutic innovations for DEB, including gene therapy, cell-based therapy, protein therapy, and ...

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10630779/

CRISPR-Cas9-based non-viral gene editing therapy for ... It has finished phase 3 randomized double-blind phase study with 223 patients and demonstrated its therapeutic effect on accelerated wound healing in EB. The ...

6. academic.oup.com academic.oup.com/bjd/article/188/1/12/6763699

Efficacy and safety of Oleogel-S10 (birch triterpenes) for ... The EASE study is the largest clinical trial to be conducted in EB. It was designed to determine the efficacy and safety of Oleogel-S10 in ...

7. trial.medpath.com trial.medpath.com/clinical-trial/b048e6656f35e227/nct05529134-ptw-002-dystrophic-epidermolysis-bullosa

Study of PTW-002 in Patients With Dominant or Recessive ... A double-blind, randomized, intra-patient placebo- controlled, multiple dose study of PTW-002 evaluating safety, proof of mechanism, preliminary efficacy, ...