MSC EVs in Dystrophic Epidermolysis Bullosa
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|ClinicalTrials.gov Identifier: NCT04173650|
Recruitment Status : Not yet recruiting
First Posted : November 22, 2019
Last Update Posted : March 24, 2020
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 vs placebo in the treatment of lesions in subjects with Epidermolysis Bullosa (EB).
STUDY DESIGN: This is a phase 1/2A, multi-center, randomized, vehicle controlled, study to assess the effectiveness and safety of AGLE-103 vs placebo on lesions in subjects with EB.
|Condition or disease||Intervention/treatment||Phase|
|Dystrophic Epidermolysis Bullosa||Drug: AGLE 102||Phase 1 Phase 2|
STUDY DESIGN: This is a phase 1/2A, multi-center, randomized, vehicle controlled, study to assess the effectiveness and safety of AGLE-102 vs placebo on lesions in subjects with EB.
AGLE-102 will be applied topically, once a day to the entire body for a period of 60 days. Subjects will come into the study site to have 1 target lesion selected at baseline. Selected target lesion must be at least 21 days old (size 5 to 50 cm). Photographic confirmation of the target lesion location will be collected at baseline, and the picture saved from the first visit will be used to confirm location of the target lesion at subsequent visits. The subject will return to the study site for visit 2 (14 days +7 days from baseline), visit 3 (30 days + 7 days from baseline) and visit 4 (60 days ±7 days from baseline) to have the target lesion, previously identified at baseline, re-assessed for the level of healing. In addition, itching, pain, Body Surface Area (BSA), target lesion closure, and scaring of healed target lesion will also be assessed at each visit. The ARANZ SilhouetteStarTM will be used to measure the target lesion at all visits.
Primary End Point: The primary end point is to demonstrate in subjects with EB, the safety and efficacy of AGLE-102 vs placebo in lesion closure.
Secondary End Points: Are to assess the change in itching, pain, BSA of blisters or lesions, target lesion closure, and extent of scarring of healed target lesion after treatment with AGLE-102 vs placebo.
PLANNED SAMPLE SIZE: 10 subjects will be treated on the protocol with AGLE-102.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||10 participants|
|Intervention Model:||Parallel Assignment|
|Intervention Model Description:||This is a phase 1/2A, multi-center, open label, study to assess the effectiveness and safety of AGLE-102 on lesions in subjects with EB.|
|Masking:||None (Open Label)|
|Official Title:||A Safety Study of the Administration of MSC Extracellular Vesicles in the Treatment of Dystrophic Epidermolysis Bullosa Wounds|
|Estimated Study Start Date :||September 2020|
|Estimated Primary Completion Date :||May 2021|
|Estimated Study Completion Date :||November 2021|
Experimental: AGLE 102
Drug: AGLE 102
Exosomes from MSCs
- Dose Limiting Toxicity [ Time Frame: 8 months ]This study examines a dose escalation, Dose Limiting Toxicity (as defined in the NCI/CTCAE v4.0 grading scale)
- Wound size evaluation [ Time Frame: 8 months ]
1. Wound Size Evaluation
- Target wounds will be measured using Silhouette® (Aranz Medical
- Direct wound tracings will also be performed.