MSC EVs in Dystrophic Epidermolysis Bullosa

Dystrophic Epidermolysis Bullosa Clinical Trial

Official title: A Safety Study of the Administration of Mesenchymal Stem Cell Extracellular Vesicles in the Treatment of Dystrophic Epidermolysis Bullosa Wounds

Status Not yet recruiting

Clinical Trial Summary

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, ascending dose, study to assess the effectiveness and safety of AGLE-102 on lesions in subjects with EB.

Clinical Trial Description

STUDY DESIGN: This trial is a prospective, open label, randomized, multi-center study, with intra-subject paired- wound control, intended to assess the safety and preliminary efficacy of administering EVs derived from allogeneic MSCs to 10-50 cm2 wounds in RDEB subjects with wounds persisting for at least four weeks.

After providing written informed consent (assent with parental written consent for pediatric subjects) and undergoing screening evaluations, eligible subjects have a single set of paired wounds identified that are determined by either subject/caregiver reporting, medical history or other reliable sources to have been present for a minimum of four weeks. One of each pair is randomized to treatment with EVs in conjunction with standard of care. The other wound of each pair receives standard of care alone, without EV treatment. All wounds on study continue to receive standard of care throughout the study. For the EV treated wounds, up to six administrations of EVs occur approximately two weeks apart over a period of 10 weeks. If the treated wound closes prior to six administrations, and remains closed for at least two weeks, no additional doses are given. Wound closure is determined by complete re-epithelialization that is not subject to re-injury during dressing changes or as a result of normal daily activities (e.g., wearing clothing, eating, sleeping). This is confirmed by the investigator at least two weeks after initial closure. After the initial 10- week treatment period, or from the time of closure of the treated wound if prior to the end of the treatment period, the wounds are followed every four weeks for a period of 12 additional weeks, for a total of up to 22 weeks of observation from the start of treatment. Regardless of the closure status of wounds or the timing of the last EV treatment, all subjects must present 10 weeks after the initial treatment to ascertain closure status of both the control and EV treated wounds. At no time during the study does closure status of the control wound influence the EV treatment or visit schedule.

STUDY OBJECTIVES:

Primary Objectives:

Determine the safety of applying multiple administrations of EVs derived from allogeneic MSCs to 10-50 cm2 RDEB wounds that have persisted for at least four weeks

Determine if EVs plus standard of care can promote wound healing in RDEB subjects compared to standard of care alone control

Secondary Objectives: Determine if there is clinical benefit of applying EVs to RDEB wounds

PLANNED SAMPLE SIZE: 8 subjects will be treated on the protocol with AGLE-102. ;

Related Conditions & MeSH terms

• Dystrophic Epidermolysis Bullosa
• Epidermolysis Bullosa
• Epidermolysis Bullosa Dystrophica

• NCT number: NCT04173650
• Study type: Interventional
• Source: Aegle Therapeutics
• Contact: Mei Chen, Ph.D.
• Phone: 323-865-0621
• Email: chenm@usc.edu
• Status: Not yet recruiting
• Phase: Phase 1/Phase 2
• Start date: April 2024
• Completion date: September 2025