MSC EVs in Dystrophic Epidermolysis Bullosa

NCT04173650 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: EB IND
• Study Type: interventional
• Target: 8
• Locations: 1 country
• Sites: 3

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.

Conditions

Dystrophic Epidermolysis Bullosa

Keywords

Exosomes; Extracellular Vesicles

Outcome Measures

Primary Outcomes (2)

• Treatment-emergent Adverse Events

  Incidence of adverse events after single and repeat administrations of EVs

  22 weeks

• Wound healing improvement

  Change in wound surface area relative to baseline compared with standard of care control

  10 weeks

Secondary Outcomes (5)

• Complete wound closure efficacy

  10 weeks

• Time from baseline to complete wound closure

  22 weeks

• Scar quality improvement

  22 weeks

• Pain relief efficacy

  22 weeks

• Quality of Life Improvement

  22 weeks

Study Arms (1)

AGLE 102

EXPERIMENTAL

Treatment arm

Drug: AGLE-102

Interventions

AGLE-102 DRUG

Extracellular vesicles from bone marrow derived mesenchymal stem cells - up to six topical administrations

Also known as: AGLE 102, Control

AGLE 102

Eligibility Criteria

• Age: 6 Months+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Subjects must be 6 months or older at screening.
• Subjects who have a confirmed diagnosis of DEB based on electron microscopy, immunomapping, or genetic testing. Subjects with severe DEB (e.g., RDEB patients with absent Col VII/no anchoring fibrils) and milder forms of DEB (e.g., RDEB patients with reduced Col VII and/or anchoring fibril levels) are eligible.
• Presence for at least four weeks of two 10 - 50 cm2 wounds (unroofed EB erosions) at least 4 inches apart that can be considered similar enough in size, wound location and expected healing characteristics to be a matched pair. Neither index wound may have been treated with any local (topical, subcutaneous, etc) or systemic therapeutic other than standard of care treatments described in this protocol (i.e. local or systemic antimicrobials to control infection, topical anesthetic and oral analgesics if required) in the past 3 months prior to screening.
• Females of childbearing potential must have a negative urine or serum pregnancy test at screening and agree to continue use of an acceptable form of birth control throughout the duration of the study. Acceptable forms of birth control include oral, implant, injectable, and transdermal contraceptives; an intrauterine device; or other forms considered acceptable by the investigator. A female subject is eligible to participate if she is not pregnant, is not a woman or childbearing potential (WOCBP), or is a WOCBP who agrees to follow the contraceptive guidance above.
• Post-pubertal males who agree to use an acceptable method of contraception for the duration of the study.
• Subjects 18 years of age and older must be capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol. Subjects under the age of 18 years must be capable of giving assent (if age-appropriate), and/or they must have a guardian capable of providing consent.
• Willing to comply with the protocol requirements.
• Note: Reference to Appendix 3 can be located in the protocol

You may not qualify if:

• Concomitant treatment at the time of screening or at any time during the study of either study wound (control or AGLE-102 treated) with local or systemic therapy other than standard of care treatments described in this protocol (i.e. local or systemic antimicrobials to control infection, topical anesthetic and oral analgesics if required). Concomitant treatment of non-study wounds with local therapy during the study is acceptable. Systemic treatment of RDEB wounds (except as noted for standard of care) during the study is not acceptable. Subjects unwilling to abstain from prohibited concomitant treatment of study wounds (treated or control) and/or systemic treatment of RDEB wounds during the study period are excluded.
• Either study wound located within 6 inches of wounds treated concurrently with any other local therapy.
• Clinical evidence of systemic infection.
• History of bone marrow transplantation.
• Diagnosed clinically significant autoimmune disease
• Wound that extends across the fingers, toes, pubic or perineum region are excluded from being index wounds.
• The subject has clinical evidence of an active infection at the wound site.
• Current malignancy (including skin cancer), a life expectancy of \< 2 years, or severe cardiopulmonary disease that restricts ambulation to the clinical facility.
• History of coagulopathy.
• Chronic use of systemic steroids or immunosuppressive agents.
• Allergy to human albumin, streptomycin, or penicillin.
• The subject is likely to be a recipient of tissue or organ transplantation.
• Current history of alcohol or substance abuse or has a history of alcohol or substance abuse that required treatment within the previous 12 months.
• History of poor compliance or unreliability.
• Females who are pregnant, nursing, or planning a pregnancy during their participation in the study.

Sponsors & Collaborators

• Aegle Therapeutics: lead

Study Sites (3)

Phoenix Children's Hospital

Phoenix, Arizona, 85016, United States

RECRUITING

USC /Norris Comprehensive Cancer Center University of Southern California

Los Angeles, California, 90033, United States

RECRUITING

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

MeSH Terms

Conditions

Epidermolysis Bullosa Dystrophica

Condition Hierarchy (Ancestors)

Epidermolysis Bullosa; Skin Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Genetic Diseases, Inborn; Collagen Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases; Skin Diseases, Vesiculobullous

Study Officials

• David T Woodley, MD

  University of Southern California Dept of Dermatology

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Mei Chen, Ph.D.

CONTACT

323-865-0621; chenm@usc.edu

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 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.

Study Record Dates

• First Submitted: November 18, 2019
• First Posted: November 22, 2019
• Study Start: August 13, 2024
• Primary Completion: September 1, 2025
• Study Completion: March 1, 2026
• Last Updated: June 25, 2025

Record last verified: 2025-06

Locations

US (3)