AGLE 102 for Epidermolysis Bullosa

Recruiting at 2 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

10 Participants Needed

Trial Summary

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.

What safety data exists for AGLE-102 or similar treatments for epidermolysis bullosa?

The research does not provide specific safety data for AGLE-102, but a similar treatment, beremagene geperpavec (B-VEC), was found to be safely tolerated in a clinical trial for a related condition, with no significant adverse events reported. ^{1 2 3 4 5}

What is the purpose of this trial?

INVESTIGATIONAL PRODUCT: AGLE-102 is an allogeneic derived extracellular vesicle (EV) product derived from normal donor mesenchymal stem cells (MSCs). INDICATION AND RATIONALE: The aim of the study is to assess the safety and efficacy of AGLE-102 in the treatment of lesions in subjects with Epidermolysis Bullosa (EB). STUDY DESIGN: This is a phase 1/2A, non randomized, multi-center, study to assess the effectiveness and safety of AGLE-102 on lesions in subjects with EB.

Research Team

David T Woodley, MD

Principal Investigator

University of Southern California Dept of Dermatology

Eligibility Criteria

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

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)

Treatment Details

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.

Participant Groups

Group I: AGLE 102 Experimental Treatment 1 Intervention

Treatment arm

Who Is Running the Clinical Trial?

Aegle Therapeutics

Lead Sponsor

Trials

2

Recruited

10+

Findings from Research

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]

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]

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]

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]

References

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

Eye involvement in inherited epidermolysis bullosa: experience of the National Epidermolysis Bullosa Registry. [2007]

2. England pubmed.ncbi.nlm.nih.gov

Immune tolerance of allogeneic haematopoietic cell transplantation supports donor epidermal grafting of recessive dystrophic epidermolysis bullosa chronic wounds. [2022]

3. Australia pubmed.ncbi.nlm.nih.gov

Epidemiological, clinical, pathological and genetic characteristics of epidermolysis bullosa in New Zealand. [2022]

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

In vivo topical gene therapy for recessive dystrophic epidermolysis bullosa: a phase 1 and 2 trial. [2022]

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

Tissue-engineered skin (Apligraf) in the healing of patients with epidermolysis bullosa wounds. [2019]