AGLE 102 for Epidermolysis Bullosa

Recruiting at 3 trial locations

Overseen By Mei Chen, Ph.D.

Age: Any Age

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Aegle Therapeutics

Must not be taking: Systemic steroids, Immunosuppressive agents

Disqualifiers: Systemic infection, Autoimmune disease, Malignancy, others

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

Compensation: Varies

Number of Visits: 10

Duration: 10 weeks

8 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a new treatment called AGLE-102 to assess its safety and effectiveness for people with Epidermolysis Bullosa (EB), a condition that causes painful skin lesions. The study aims to determine if AGLE-102, developed from stem cells, can help heal these lesions. Participants will be randomly assigned to receive either the treatment or a standard care comparison. Individuals with a confirmed diagnosis of Dystrophic Epidermolysis Bullosa (DEB) and specific types of skin wounds for at least four weeks may be eligible to join. As a Phase 1 trial, this research focuses on understanding how AGLE-102 works in people, offering participants the opportunity to be among the first to receive this new treatment.

Will I have to stop taking my current medications?

The trial requires that participants do not use systemic steroids or immunosuppressive agents. If you are taking these medications, you may need to stop them to participate in the trial.

Is there any evidence suggesting that AGLE-102 is likely to be safe for humans?

Research has shown that AGLE-102, a treatment derived from donor stem cells, is being tested for safety in people with Epidermolysis Bullosa (EB). In other studies, AGLE-102 produced encouraging results. For example, one patient with a severe burn experienced 99% healing within a week after just one dose. This suggests the treatment might be safe. However, as the trial remains in the early stages, more information is needed to confirm its safety for all patients. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for Epidermolysis Bullosa, which typically focus on wound care and symptom management, AGLE-102 targets the underlying cause of the condition. This investigational treatment is unique because it utilizes a novel mechanism of action that aims to promote skin healing at a molecular level. Researchers are excited about AGLE-102 because it has the potential to significantly improve wound healing and quality of life for patients, offering a more targeted approach than current therapies.

What evidence suggests that AGLE-102 might be an effective treatment for Epidermolysis Bullosa?

Research has shown that AGLE-102, a new treatment made from special cell products, may help with skin problems. One study found that a patient with a deep second-degree burn healed 99% in just seven days after a single dose of AGLE-102. This treatment originates from cells known for repairing tissue and reducing swelling. Early results suggest that AGLE-102 might aid in healing wounds in people with epidermolysis bullosa (EB), a condition that makes skin very fragile. The treatment remains under investigation in this trial, but the initial findings are promising. ^{1 2 4 6 7}

Who Is on the Research Team?

David T Woodley, MD

Principal Investigator

University of Southern California Dept of Dermatology

Are You a Good Fit for This Trial?

This trial is for individuals with Dystrophic Epidermolysis Bullosa (DEB), including severe and milder forms. Adults initially, but may include children aged 6+ later on. Participants need active wounds between 10-50 cm2 on certain body areas and must not be drug users or pregnant women unwilling to use birth control.

Inclusion Criteria

Subjects must have a negative urine test for drugs of abuse at the screening visit.

I have been diagnosed with DEB through specific tests.

I have an open wound between 10 and 50 cm2 on my arm, leg, or trunk.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

1 visit (in-person)

Treatment

Participants receive up to six administrations of EVs approximately two weeks apart over a period of 10 weeks

10 weeks

Up to 6 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment, with visits every four weeks

12 weeks

3 visits (in-person)

What Are the Treatments Tested in This Trial?

Interventions

• AGLE-102

Trial Overview AGLE-102, a therapy derived from healthy donor stem cells' extracellular vesicles, is being tested for safety and effectiveness in healing lesions caused by EB. This early-phase study will take place across multiple centers without randomizing participants.

How Is the Trial Designed?

Group I: AGLE 102 Experimental Treatment 1 Intervention

Treatment arm

Who Is Running the Clinical Trial?

Aegle Therapeutics

Lead Sponsor

Trials

2

Recruited

10+

Published Research Related to This Trial

Beremagene geperpavec (B-VEC) is a promising topical treatment for recessive dystrophic epidermolysis bullosa (RDEB), effectively restoring collagen VII (C7) expression and promoting wound healing in a clinical trial involving nine patients over 12 weeks.

The treatment was found to be safe, with no serious adverse events related to B-VEC, and it successfully met primary and secondary objectives related to wound healing and C7 expression.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

In vivo topical gene therapy for recessive dystrophic epidermolysis bullosa: a phase 1 and 2 trial. Gurevich, I., Agarwal, P., Zhang, P., et al.[2022]

In a study involving 15 patients with inherited epidermolysis bullosa (EB), tissue-engineered skin (Apligraf) demonstrated a high healing rate, with 79% of wounds healed by day 7 and 82% healed after 6 weeks, indicating its efficacy in wound treatment.

The treatment was safe, with no signs of rejection or infection, and patients reported improved quality of life and faster, less painful healing compared to standard dressings.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Tissue-engineered skin (Apligraf) in the healing of patients with epidermolysis bullosa wounds. Falabella, AF., Valencia, IC., Eaglstein, WH., et al.[2019]

In a clinical trial involving 8 patients with recessive dystrophic epidermolysis bullosa (RDEB), allogeneic epidermal skin grafts significantly reduced chronic wound sizes by 75% at 6 weeks, 95% at 12 weeks, and 100% at 52 weeks post-grafting, demonstrating effective wound healing.

The study found that the donor epidermal grafts persisted for over a year in some patients and showed signs of integrating with the host tissue, suggesting that these grafts may not only provide immediate healing but also promote long-term regeneration through the recruitment of donor-derived cells.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Immune tolerance of allogeneic haematopoietic cell transplantation supports donor epidermal grafting of recessive dystrophic epidermolysis bullosa chronic wounds. Ebens, CL., McGrath, JA., Riedl, JA., et al.[2022]

In a study of 3,280 patients with inherited epidermolysis bullosa (EB), corneal erosions and blisters were the most common ocular issues, particularly affecting 74.10% of patients with recessive dystrophic EB and 47.50% of those with junctional EB.

The cumulative risk of developing nonscarring corneal lesions by age 25 is 83.18% for junctional EB, indicating that regular eye examinations are crucial for managing ocular health in EB patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Eye involvement in inherited epidermolysis bullosa: experience of the National Epidermolysis Bullosa Registry. Fine, JD., Johnson, LB., Weiner, M., et al.[2007]

The study identified a point prevalence of epidermolysis bullosa (EB) in New Zealand at 19.5 per million, with the majority of cases being EB simplex (53%) and dystrophic EB (43%), indicating a significant presence of this condition in the population.

Advancements in genomic testing have improved the diagnostic rate for EB, highlighting the importance of genetic analysis in understanding the condition and suggesting that current prevalence rates may be underestimated due to methodological limitations.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Epidemiological, clinical, pathological and genetic characteristics of epidermolysis bullosa in New Zealand. Gear, R., Poke, G., Neas, K., et al.[2022]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT04173650

MSC EVs in Dystrophic Epidermolysis Bullosa STUDY DESIGN: This is a phase 1/2A, randomized, multi-center, study to assess the effectiveness and safety of AGLE-102 on lesions in subjects with EB.

2. aegletherapeutics.com aegletherapeutics.com/pipeline/

Pipeline We are initially developing AGLE-102TM as a first-in-class therapy for a rare, monogenetic, pediatric disease called dystrophic epidermolysis bullosa (DEB).

3. prnewswire.com prnewswire.com/news-releases/aegle-therapeutics-corp-announces-positive-data-for-the-first-patient-in-a-phase-12a-clinical-trial-dosed-with-agle-102-a-novel-extracellular-vesicle-therapy-302027568.html

Aegle Therapeutics Corp. Announces Positive Data for the ... First patient treated with AGLE-102 demonstrated 99% epithelialization in a deep second-degree burn within 7 days of receiving a single dose ...

4. synapse.patsnap.com synapse.patsnap.com/article/aegle-therapeutics-begins-dosing-in-agle-102-trial-for-deb

Aegle Therapeutics Begins Dosing in AGLE-102 Trial for ... Previous clinical trials have reported promising results in the treatment of burns. Additionally, AGLE-102 is undergoing preclinical evaluation ...

5. clinicaltrials.gov clinicaltrials.gov/study/NCT04173650?term=AREA%5BBasicSearch%5D(epidermolysis%20bullosa)&rank=8

MSC EVs in Dystrophic Epidermolysis Bullosa STUDY DESIGN: This is a phase 1/2A, randomized, multi-center, study to assess the effectiveness and safety of AGLE-102 on lesions in subjects ...

6. epidermolysisbullosanews.com epidermolysisbullosanews.com/news/dosing-starts-trial-topical-treatment-agle-102-deb-lesions/

Dosing starts in trial of topical treatment AGLE-102 for DEB ... The Phase 1/2a study (NCT04173650) is assessing the safety and efficacy of the therapy, applied topically to skin lesions, against a placebo in ...

7. synapse.patsnap.com synapse.patsnap.com/blog/first-patient-treated-in-aegle-therapeutics-phase-12a-agle-102-trial

First Patient Treated in Aegle Therapeutics' Phase 1/2a ... Aegle Therapeutics Corp. reveals the initial patient treated in the Phase 1/2a study of AGLE-102™ for Dystrophic Epidermolysis Bullosa.