Ignite Creation Date:
2025-12-24 @ 5:24 PM
Ignite Modification Date:
2025-12-24 @ 5:24 PM
Study NCT ID:
NCT04173650
Status:
RECRUITING
Last Update Posted:
2025-06-25
First Post:
2019-11-18
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
MSC EVs in Dystrophic Epidermolysis Bullosa
Sponsor:
Aegle Therapeutics
Organization:
Study Overview
Official Title:
Mesenchymal Stem Cell Extracellular Vesicles for the Treatment of Recessive Dystrophic Epidermolysis Bullosa Wounds
Status:
RECRUITING
Status Verified Date:
2025-06
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
INVESTIGATIONAL PRODUCT: AGLE-102 is an allogeneic 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, randomized, multi-center, study to assess the effectiveness and safety of AGLE-102 on lesions in subjects with EB.
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, randomized, multi-center, study to assess the effectiveness and safety of AGLE-102 on lesions in subjects with EB.
Detailed Description:
STUDY DESIGN: This is a prospective, open label, randomized, multi-center study, with intra-subject paired wound standard of care 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, no additional doses are given unless the wound reopens during the treatment period, in which case the original retreatment schedule resumes but is not extended. Subjects with wound closure identified at one of the treatment visits should return for all subsequent visits to verify wound closure and assess the scar quality of the wound, unless the closure is identified at the final study visit, in which case no additional visits are required.
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 (unless closure is identified at the final study visit). At the conclusion of the 10-weektreatment period, the wounds are followed every 4 weeks for a period of 12 weeks. 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.
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, no additional doses are given unless the wound reopens during the treatment period, in which case the original retreatment schedule resumes but is not extended. Subjects with wound closure identified at one of the treatment visits should return for all subsequent visits to verify wound closure and assess the scar quality of the wound, unless the closure is identified at the final study visit, in which case no additional visits are required.
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 (unless closure is identified at the final study visit). At the conclusion of the 10-weektreatment period, the wounds are followed every 4 weeks for a period of 12 weeks. 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.
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
False
Is an FDA AA801 Violation?: