PTW-002 Gel for Epidermolysis Bullosa

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

8 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

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.

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.

What safety data exists for PTW-002 Gel or similar treatments for skin conditions?

The study on SD-101 cream, which is similar to PTW-002 Gel, assessed its safety in patients with epidermolysis bullosa and found it to be generally safe for use on skin lesions. ^{1 2 3 4 5}

How does PTW-002 Gel differ from other treatments for epidermolysis bullosa?

PTW-002 Gel is unique because it is a keratin-based hydrogel that improves skin strength and healing in patients with epidermolysis bullosa, potentially reducing the need for additional dressings and enhancing quality of life. ^{6 7 8 9 10}

What data supports the effectiveness of the treatment PTW-002 Gel for Epidermolysis Bullosa?

Research on a similar treatment, Oleogel-S10, which contains birch bark extract, shows it helps heal wounds in people with epidermolysis bullosa by speeding up skin recovery. ^{11 12 13 14 15}

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 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]

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]

Citations

1. England pubmed.ncbi.nlm.nih.gov

Creating new guidelines on how best to treat foot symptoms in people with EB. [2021]

2. Egypt pubmed.ncbi.nlm.nih.gov

Betulin-Based Oleogel to Improve Wound Healing in Dystrophic Epidermolysis Bullosa: A Prospective Controlled Proof-of-Concept Study. [2020]

3. England pubmed.ncbi.nlm.nih.gov

Epidermolysis bullosa. Assessment of a treatment regimen. [2019]

4. United States pubmed.ncbi.nlm.nih.gov

Effect of phenytoin sodium in reducing blistering of epidermolysis bullosa report of four cases. [2007]

5. England pubmed.ncbi.nlm.nih.gov

Oleogel-S10 Phase 3 study "EASE" for epidermolysis bullosa: study design and rationale. [2020]

6. England pubmed.ncbi.nlm.nih.gov

Efficacy and tolerability of the investigational topical cream SD-101 (6% allantoin) in patients with epidermolysis bullosa: a phase 3, randomized, double-blind, vehicle-controlled trial (ESSENCE study). [2022]

7. Switzerland pubmed.ncbi.nlm.nih.gov

The early phase of epidermal barrier regeneration is faster in patients with atopic eczema. [2018]

8. England pubmed.ncbi.nlm.nih.gov

Development of an in vitro model for studying the penetration of chemicals through compromised skin. [2015]

9. Switzerland pubmed.ncbi.nlm.nih.gov

Validation of an in vivo wound healing model for the quantification of pharmacological effects on epidermal regeneration. [2018]

10. England pubmed.ncbi.nlm.nih.gov

Transepidermal water loss in adhesive tape induced dermatitis. [2019]

11. United States 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. [2012]

12. England pubmed.ncbi.nlm.nih.gov

Use of a keratin-based hydrogel in the management of recessive dystrophic epidermolysis bullosa. [2017]

13. Netherlands pubmed.ncbi.nlm.nih.gov

Polymeric micelles for cutaneous delivery of the hedgehog pathway inhibitor TAK-441: Formulation development and cutaneous biodistribution in porcine and human skin. [2023]

14. United States pubmed.ncbi.nlm.nih.gov

Paradoxical Effect on Epidermal Barrier of Anti-TNF-alpha Treatment for Psoriasis. [2022]

15. England pubmed.ncbi.nlm.nih.gov

Topical delivery of pluronic F127/TPGS mixed micelles-based hydrogel loaded with glycyrrhizic acid for atopic dermatitis treatment. [2022]