PTW-002 Gel for Epidermolysis Bullosa
Recruiting at 2 trial locations
RJ
Overseen ByRamsey Johnson
Age: Any Age
Sex: Any
Trial Phase: Phase 1 & 2
Sponsor: Phoenicis Therapeutics
Trial Summary
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 data supports the effectiveness of the treatment PTW-002 Gel for Epidermolysis Bullosa?
What safety data exists for PTW-002 Gel or similar treatments for skin conditions?
How does PTW-002 Gel differ from other treatments for epidermolysis bullosa?
Research Team
HL
Hal Landy, MD
Principal Investigator
Phoenicis Therapeutics
Eligibility Criteria
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.
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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.
See 7 more
Timeline
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
Treatment Details
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.
Participant Groups
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)
Find a Clinic Near You
Who Is Running the Clinical Trial?
Phoenicis Therapeutics
Lead Sponsor
Trials
3
Recruited
60+
Findings from Research
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]
References
Creating new guidelines on how best to treat foot symptoms in people with EB. [2021]
Betulin-Based Oleogel to Improve Wound Healing in Dystrophic Epidermolysis Bullosa: A Prospective Controlled Proof-of-Concept Study. [2020]
Epidermolysis bullosa. Assessment of a treatment regimen. [2019]
Effect of phenytoin sodium in reducing blistering of epidermolysis bullosa report of four cases. [2007]
Oleogel-S10 Phase 3 study "EASE" for epidermolysis bullosa: study design and rationale. [2020]
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]
The early phase of epidermal barrier regeneration is faster in patients with atopic eczema. [2018]
Development of an in vitro model for studying the penetration of chemicals through compromised skin. [2015]
Validation of an in vivo wound healing model for the quantification of pharmacological effects on epidermal regeneration. [2018]
Transepidermal water loss in adhesive tape induced dermatitis. [2019]
Transepidermal water loss (TEWL) and corneometry with hydrogel vehicle in the treatment of atopic dermatitis: a randomized, investigator-blind pilot study. [2012]
Use of a keratin-based hydrogel in the management of recessive dystrophic epidermolysis bullosa. [2017]
Polymeric micelles for cutaneous delivery of the hedgehog pathway inhibitor TAK-441: Formulation development and cutaneous biodistribution in porcine and human skin. [2023]
Paradoxical Effect on Epidermal Barrier of Anti-TNF-alpha Treatment for Psoriasis. [2022]
Topical delivery of pluronic F127/TPGS mixed micelles-based hydrogel loaded with glycyrrhizic acid for atopic dermatitis treatment. [2022]
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