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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
Sponsor:
Information provided by (Responsible Party):
Aegle Therapeutics

Brief 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 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

Detailed Description:

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.

STUDY OBJECTIVES:

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 10 participants
Allocation: Non-Randomized
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)
Primary Purpose: Treatment
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


Arm Intervention/treatment
Experimental: AGLE 102
Treatment arm
Drug: AGLE 102
Exosomes from MSCs




Primary Outcome Measures :
  1. 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)


Secondary Outcome Measures :
  1. Wound size evaluation [ Time Frame: 8 months ]

    1. Wound Size Evaluation

    1. Target wounds will be measured using Silhouette® (Aranz Medical
    2. Direct wound tracings will also be performed.



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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Informed Consent signed by the subject or subject's legal representative.
  2. Diagnosis of EB - add specific details
  3. Subjects must be 6 months or older.
  4. Subject must have 1 target lesion (size 5 to 50 cm2).
  5. Target lesion must be 21 days or older.

Exclusion Criteria:

  1. Selected target lesion cannot have clinical evidence of local infection.
  2. Use of investigational drug within the 30 days before enrollment.
  3. Use of immunotherapy or cytotoxic chemotherapy within the 60 days before enrollment.
  4. Use of systemic or topical steroidal therapy within the 30 days before enrollment.
  5. Use of systemic antibiotics within the 7 days before enrollment.
  6. Current or former malignancy.
  7. Arterial or venous disorder resulting in ulcerated lesions.
  8. Diabetes mellitus.
  9. Pregnancy os breastfeeding during the study.
  10. Females of childbearing potential who are not abstinent and not practicing a medically acceptable method of contraception.
  11. Known history of cardiac, hepatic, or renal disease.
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Responsible Party: Aegle Therapeutics
ClinicalTrials.gov Identifier: NCT04173650    
Other Study ID Numbers: EB IND
First Posted: November 22, 2019    Key Record Dates
Last Update Posted: March 24, 2020
Last Verified: March 2020

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Aegle Therapeutics:
Exosomes
Additional relevant MeSH terms:
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Epidermolysis Bullosa
Epidermolysis Bullosa Dystrophica
Skin Abnormalities
Congenital Abnormalities
Skin Diseases, Genetic
Genetic Diseases, Inborn
Skin Diseases
Skin Diseases, Vesiculobullous
Collagen Diseases
Connective Tissue Diseases