EpiFix for Pyoderma Gangrenosum

Recruiting at 2 trial locations

Overseen By Alison Smith, MD, PhD

Age: 18+

Sex: Any

Trial Phase: Phase 4

Sponsor: Louisiana State University Health Sciences Center in New Orleans

Disqualifiers: Pregnant, Prisoners, Under 18, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 8

Duration: 2 weeks

20 Participants Needed

Trial Summary

What is the purpose of this trial?

Pyoderma gangrenosum is a chronic debilitating ulcerative neutrophilic condition usually affecting the skin. The pathogenesis of pyoderma gangrenosum is poorly understood. Pyoderma gangrenosum has been estimated to have an incidence rate of 3-10 per million populations per year. Mortality estimates range up to 30%. The non-healing wounds present a significant social and economic burden on affected patients. We hypothesize that EpiFix (dehydrated human amnion/chorion membrane) will be beneficial in treating patients with pyoderma gangrenosum. Potentially eligible patients will be pre-screened in clinic. The pre-screening process will include a medical history, including review of co-morbid medical problems and home medications, and a physical exam of the wounds. The first stage of the study will involve a surgery in which the pyoderma gangrenosum wounds are going to be treated with the EpiFix. About a week later, the patient will then undergo another surgery where skin grafts will be placed on the wounds that were treated in the first surgery. In each surgery, samples of the wound will be taken, and genetic information from these samples will be studied to identify the genes that are active before and after treating the wound with EpiFix. Routine post-surgery clinic visits will then be used to monitor the healing of the wounds and make sure that no extra medical treatment is needed. Study staff will evaluate wounds twice a week for the first two weeks, then once a week until 6 weeks. Following this, you will then be followed every 4 weeks up to 22 weeks. These visits to the clinic will include taking wound measurements, taking digital photos, asking about level of pain and evaluating development of infection. Patient consent will be obtained to take any photos.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial staff during the pre-screening process.

What data supports the effectiveness of the treatment EpiFix for Pyoderma Gangrenosum?

In a case report, a patient with Pyoderma Gangrenosum experienced significant pain reduction and wound healing after using dehydrated human amnion/chorion membrane (dHACM), with the wound size decreasing by more than half over two months. This suggests that dHACM, used in EpiFix, may help in managing this condition. ^{1 2 3 4 5}

Is EpiFix (dehydrated human amnion/chorion membrane) generally safe for use in humans?

EpiFix, also known as dehydrated human amnion/chorion membrane, has been used in various medical treatments and is generally considered safe for human use. It has been applied in wound healing and other conditions, with no significant safety concerns reported in the available literature. ^{6 7 8 9 10}

How does the treatment EpiFix differ from other treatments for pyoderma gangrenosum?

EpiFix is unique because it uses a dehydrated human amnion/chorion membrane, which is a type of bioengineered skin graft, to promote healing of skin ulcers. This is different from the more common treatments like systemic corticosteroids or cyclosporine, which are drugs that suppress the immune system to reduce inflammation. ^{10 11 12 13 14}

Research Team

Alison Smith, MD, PhD

Principal Investigator

Louisiana State University Health Sciences Center - New Orleans

Eligibility Criteria

This trial is for adults over 18 with pyoderma gangrenosum, a rare skin condition causing painful ulcers. Participants must be pre-screened in clinic, reviewing medical history and current medications, along with a physical exam of the wounds.

Inclusion Criteria

I have been diagnosed with pyoderma gangrenosum.

I am over 18 years old.

Exclusion Criteria

Members of vulnerable populations (pregnant women and prisoners)

I am under 18 years old.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

1-2 weeks

1 visit (in-person)

Pre-Screening

Pre-screening process includes medical history review, co-morbid medical problems, home medications, and physical exam of the wounds

1 week

1 visit (in-person)

Treatment

Initial surgery with EpiFix treatment followed by a second surgery for skin grafts; wound samples collected for genetic analysis

2 weeks

2 surgeries (in-person)

Post-Surgery Monitoring

Routine clinic visits to monitor wound healing, take measurements, photos, and evaluate pain and infection

6 months

Weekly visits initially, then every 4 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• EpiFix

Trial Overview The study tests EpiFix (dehydrated human amnion/chorion membrane) on wound treatment. It involves two surgeries: first to apply EpiFix to the wounds, then a week later to place skin grafts. Wound healing is monitored through regular clinic visits and analysis of genetic information from wound samples.

Participant Groups

Group I: Treatment Arm Experimental Treatment 1 Intervention

Patient's pyoderma gangrenosum wounds are treated surgically with EpiFix (dehydrated human amnion/chorion membrane). In another surgery about one week later, we will be covering the treated wounds with split-thickness skin grafts. During each surgery, we will be collecting wound samples for genetic analysis. Routine post-surgery clinic visits will be used to monitor wound healing over a 6-month period.

EpiFix is already approved in United States for the following indications:

Approved in United States as EpiFix for:

• Wound healing
• Chronic wounds
• Diabetic foot ulcers
• Venous leg ulcers
• Pressure ulcers

Who Is Running the Clinical Trial?

Louisiana State University Health Sciences Center in New Orleans

Lead Sponsor

Trials

123

Recruited

42,400+

Findings from Research

In a retrospective case series involving 5 patients with pyoderma gangrenosum (PG) whose wounds had not healed for at least 2.5 months despite standard treatments, the application of dehydrated human amnion/chorion membrane (DHACM) led to successful wound closure in all cases.

The study suggests that DHACM could be an effective alternative treatment for PG ulcers that do not respond to conventional therapies, highlighting the need for further formal evaluation of this approach.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pyoderma gangrenosum and dehydrated human amnion/chorion membrane: a potential tool for an orphan disease. Alston, D., Eggiman, E., Forsyth, RA., et al.[2022]

A 77-year-old woman with refractory pyoderma gangrenosum experienced significant pain relief and wound healing after the application of dehydrated human amnion/chorion membrane (dHACM) allograft, reducing her pain from 10 to 0 within days.

After incorporating dHACM into her treatment plan alongside immunosuppressive therapy, the size of her wound decreased by over 56% within two months, highlighting the potential efficacy of dHACM in managing difficult wounds associated with pyoderma gangrenosum.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Dehydrated Human Amnion/Chorion Membrane as Adjunctive Therapy in the Multidisciplinary Treatment of Pyoderma Gangrenosum: A Case Report. Snyder, RJ., Ead, J., Glick, B., et al.[2017]

A 30-year-old woman developed chronic, nonhealing ulcers consistent with pyoderma gangrenosum after undergoing surgical biopsies, highlighting the condition's potential to worsen following trauma or surgical intervention.

Treatment with a high-potency topical steroid over 9 weeks led to gradual healing of the ulcers, suggesting that topical steroids can be an effective management option for pyoderma gangrenosum.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Treatment of postsurgical pyoderma gangrenosum with a high-potency topical steroid. Hawryluk, EB., Penn, SK., Wasko, MC., et al.[2013]

This case series presents the successful use of viable cryopreserved umbilical cord tissue (vCUT) in treating three patients with pyoderma gangrenosum (PG), leading to complete healing of their ulcers.

The findings suggest that vCUT may be an effective adjunctive treatment for managing the challenging wounds associated with PG, which often do not respond well to conventional therapies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Surgical Management of Lower Extremity Pyoderma Gangrenosum With Viable Cryopreserved Umbilical Tissue: A Case Series. Fridman, R., Bar-David, T., Larsen, J., et al.[2021]

Plasma exchange treatment for pyoderma gangrenosum showed promising results in 3 out of 4 patients, with significant pain relief and healing of lesions after just one session, especially when combined with corticosteroids.

The treatment effectively halted disease progression and reduced the need for surgical interventions, indicating that plasma exchange can be a valuable option for managing this condition, although results may vary among individuals.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Pyoderma gangrenosum: treatment with plasma exchange (4 cases)]. Gérard, A., Schooneman, F., Voiriot, P., et al.[2006]

Pyoderma gangrenosum (PG) is a rare and painful skin condition that often occurs alongside other diseases, such as inflammatory bowel disease, and is characterized by rapidly progressing ulcers.

Treatment for PG primarily involves systemic corticosteroids and cyclosporin A, with additional options like anti-TNF therapy showing promise, but the prognosis remains unpredictable due to the lack of large-scale clinical trials.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pyoderma gangrenosum--a review. Wollina, U.[2022]

Pyoderma gangrenosum (PG) is a rare skin condition that can occur in children, with this case being the youngest reported instance at just six months old, associated with chronic recurrent multifocal osteomyelitis (CRMO).

Approximately 50% of PG patients have an underlying systemic disease, with common associations including inflammatory bowel disease and arthritis, highlighting the importance of recognizing PG as a potential indicator of other health issues.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pyoderma gangrenosum in a six-month-old boy. Koturoğlu, G., Vardar, F., Ozkinay, F., et al.[2016]

Pyoderma gangrenosum (PG) can occur in pregnant women after a caesarean section and may be misdiagnosed as a surgical site infection (SSI), as seen in two cases where patients exhibited wound ulceration despite antibiotic treatment.

In both cases, a skin biopsy confirmed PG, and treatment with oral steroids led to significant improvement, highlighting the importance of accurate diagnosis and appropriate treatment for PG, which differs from that of SSI.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Incisional pyoderma gangrenosum after caesarean section: Two case reports. Murata, T., Kyozuka, H., Fukuda, T., et al.[2022]

Pyoderma gangrenosum (PG) is a complex skin condition characterized by painful ulcers and is often associated with underlying diseases like inflammatory bowel disease and certain malignancies, making accurate diagnosis crucial to avoid harmful treatments.

While there is no one-size-fits-all treatment for PG, effective management often involves systemic immunosuppressive therapies such as corticosteroids and cyclosporine, with emerging options like TNF-alpha blockers showing promise for difficult cases.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pyoderma gangrenosum. Conrad, C., Trüeb, RM.[2019]

Pyoderma gangrenosum is a serious skin condition often linked to underlying diseases like inflammatory bowel disease, and its diagnosis relies on clinical presentation and exclusion of similar conditions.

The most effective treatments include systemic corticosteroids and cyclosporine, with combinations of these and other drugs used for resistant cases, although the overall prognosis remains unpredictable.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Clinical management of pyoderma gangrenosum. Wollina, U.[2018]

A 21-year-old woman developed Pyoderma Gangrenosum (PG) after a caesarean section, which led to severe necrosis and complications, highlighting how PG can mimic infections and delay diagnosis.

High-dose corticosteroids were effective in rapidly treating PG, emphasizing the need for timely recognition to prevent further complications and morbidity from unnecessary surgical interventions.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pseudo-wound infection after a caesarean section: Case report of unrecognized Pyoderma Gangrenosum. van Donkelaar, CE., de Haan, JMH., Lange, JFM., et al.[2021]

In a study of five adult patients with steroid-refractory pyoderma gangrenosum, canakinumab treatment led to significant clinical improvement, with four out of five patients experiencing a decrease in lesion size and three achieving complete remission by week 16.

The results suggest that interleukin-1β is a key factor in the disease's pathology, indicating that canakinumab, which targets this cytokine, may be an effective treatment option for patients who do not respond to steroids.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Canakinumab in adults with steroid-refractory pyoderma gangrenosum. Kolios, AG., Maul, JT., Meier, B., et al.[2019]

A 58-year-old woman with biopsy-confirmed pyoderma gangrenosum (PG) experienced rapid progression of painful skin lesions, which were effectively treated with a combination of systemic steroids, topical negative pressure treatments, surgical revision, and partial skin grafting.

After this comprehensive treatment approach, the patient remained pain-free and showed no recurrence of PG for 11 months, indicating the efficacy of the treatment strategy in managing this rare autoimmune condition.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Topical negative pressure and skin transplantation for the treatment of pyoderma gangrenosum]. Gámiz, RC., Nielsen, AM.[2013]

Pyoderma gangrenosum (PG) is a rare skin condition with an incidence of 3-10 cases per million people per year, often associated with underlying diseases like inflammatory bowel diseases and hematological malignancies, indicating a potential immune system dysregulation.

The condition can be triggered by minor trauma in 20-30% of cases, and while the prognosis is generally good, it can vary significantly based on associated underlying disorders; treatment options include topical steroids for mild cases and systemic therapies like glucocorticoids for severe cases.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pyoderma gangrenosum: a systematic review. Cozzani, E., Gasparini, G., Parodi, A.[2022]

References

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

Pyoderma gangrenosum and dehydrated human amnion/chorion membrane: a potential tool for an orphan disease. [2022]

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

Dehydrated Human Amnion/Chorion Membrane as Adjunctive Therapy in the Multidisciplinary Treatment of Pyoderma Gangrenosum: A Case Report. [2017]

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

Treatment of postsurgical pyoderma gangrenosum with a high-potency topical steroid. [2013]

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

Surgical Management of Lower Extremity Pyoderma Gangrenosum With Viable Cryopreserved Umbilical Tissue: A Case Series. [2021]

5. France pubmed.ncbi.nlm.nih.gov

[Pyoderma gangrenosum: treatment with plasma exchange (4 cases)]. [2006]

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

Pyoderma gangrenosum--a review. [2022]

7. Turkey pubmed.ncbi.nlm.nih.gov

Pyoderma gangrenosum in a six-month-old boy. [2016]

8. Netherlands pubmed.ncbi.nlm.nih.gov

Incisional pyoderma gangrenosum after caesarean section: Two case reports. [2022]

9. Germany pubmed.ncbi.nlm.nih.gov

Pyoderma gangrenosum. [2019]

10. New Zealand pubmed.ncbi.nlm.nih.gov

Clinical management of pyoderma gangrenosum. [2018]

11. Netherlands pubmed.ncbi.nlm.nih.gov

Pseudo-wound infection after a caesarean section: Case report of unrecognized Pyoderma Gangrenosum. [2021]

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

Canakinumab in adults with steroid-refractory pyoderma gangrenosum. [2019]

13. Denmark pubmed.ncbi.nlm.nih.gov

[Topical negative pressure and skin transplantation for the treatment of pyoderma gangrenosum]. [2013]

14. Italy pubmed.ncbi.nlm.nih.gov

Pyoderma gangrenosum: a systematic review. [2022]