AGLE 102 for Epidermolysis Bullosa
Recruiting in Palo Alto (17 mi)
+2 other locations
Overseen byDavid T Woodley, MD
Age: Any Age
Sex: Any
Travel: May Be Covered
Time Reimbursement: Varies
Trial Phase: Phase 1 & 2
Recruiting
Sponsor: Aegle Therapeutics
Must not be taking: Systemic steroids, Immunosuppressive agents
Disqualifiers: Systemic infection, Autoimmune disease, Malignancy, others
No Placebo Group
Trial Summary
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.
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.
12345Eligibility 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.
+7 more
Trial 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)
Participant Groups
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.
1Treatment groups
Experimental Treatment
Group I: AGLE 102Experimental Treatment1 Intervention
Treatment arm
Find a Clinic Near You
Research Locations NearbySelect from list below to view details:
Department of Dermatology USC /Norris Comprehensive Cancer Center University of Southern CaliforniaLos Angeles, CA
Children's Hospital of PhiladelphiaPhiladelphia, PA
Phoenix Children's HospitalPhoenix, AZ
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Who Is Running the Clinical Trial?
Aegle TherapeuticsLead Sponsor
References
Eye involvement in inherited epidermolysis bullosa: experience of the National Epidermolysis Bullosa Registry. [2007]To determine the frequency of ocular manifestations in inherited epidermolysis bullosa (EB) within the continental United States and to define the estimated cumulative risks of developing nonscarring (blisters or erosions) and scarring corneal manifestations within each major EB subtype over time.
Immune tolerance of allogeneic haematopoietic cell transplantation supports donor epidermal grafting of recessive dystrophic epidermolysis bullosa chronic wounds. [2022]Chronic wounds, a common morbidity in recessive dystrophic epidermolysis bullosa (RDEB), lack definitive therapies.
Epidemiological, clinical, pathological and genetic characteristics of epidermolysis bullosa in New Zealand. [2022]To establish the epidemiological, clinical, pathological and genetic characteristics of epidermolysis bullosa (EB) in New Zealand (NZ).
In vivo topical gene therapy for recessive dystrophic epidermolysis bullosa: a phase 1 and 2 trial. [2022]Recessive dystrophic epidermolysis bullosa (RDEB) is a lifelong genodermatosis associated with blistering, wounding, and scarring caused by mutations in COL7A1, the gene encoding the anchoring fibril component, collagen VII (C7). Here, we evaluated beremagene geperpavec (B-VEC), an engineered, non-replicating COL7A1 containing herpes simplex virus type 1 (HSV-1) vector, to treat RDEB skin. B-VEC restored C7 expression in RDEB keratinocytes, fibroblasts, RDEB mice and human RDEB xenografts. Subsequently, a randomized, placebo-controlled, phase 1 and 2 clinical trial (NCT03536143) evaluated matched wounds from nine RDEB patients receiving topical B-VEC or placebo repeatedly over 12 weeks. No grade 2 or above B-VEC-related adverse events or vector shedding or tissue-bound skin immunoreactants were noted. HSV-1 and C7 antibodies sometimes presented at baseline or increased after B-VEC treatment without an apparent impact on safety or efficacy. Primary and secondary objectives of C7 expression, anchoring fibril assembly, wound surface area reduction, duration of wound closure, and time to wound closure following B-VEC treatment were met. A patient-reported pain-severity secondary outcome was not assessed given the small proportion of wounds treated. A global assessment secondary endpoint was not pursued due to redundancy with regard to other endpoints. These studies show that B-VEC is an easily administered, safely tolerated, topical molecular corrective therapy promoting wound healing in patients with RDEB.
Tissue-engineered skin (Apligraf) in the healing of patients with epidermolysis bullosa wounds. [2019]At present, wound treatment of inherited epidermolysis bullosa (EB) is only supportive.