Dystrophic Epidermolysis BullosaPhase 1/2NCT04173650
MSC EVs in Dystrophic Epidermolysis Bullosa
A Phase 1/2 study testing AGLE-102 in people with Dystrophic Epidermolysis Bullosa, run by Aegle Therapeutics at 3 sites in United States.
Trial 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). Read more about this study
- Who is it for?
- Ages 6 months and older
- Conditions the study is for
- Dystrophic Epidermolysis Bullosa
- What is being tested?
- AGLE-102
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 3 sitesUnited States
- Study dates
- 13 Aug 2024to Mar 2026
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 months and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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.
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- 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.
- Concurrent participation in another investigational drug, biologic or device study that could confound study data.
Trial locations and contacts
3 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States3 sites, 3 recruiting
- Recruiting
USC /Norris Comprehensive Cancer Center University of Southern California
Los Angeles, California, 90033
Mei Chen, PhD, 323-865-0621, chenm@usc.edu
David Woodley, MD
- Recruiting
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Griffin S Hogrogian, MSEd, 215-590-1930, stocktonhg@chop.edu
Marissa Perman, MD
- Recruiting
Phoenix Children's Hospital
Phoenix, Arizona, 85016
Kellie Badger, BS, RN, 602-933-2053, kbadger@phoenixchildres.com
Harper N Price, MD, FAAD, FAAP
About this study
Official title: Mesenchymal Stem Cell Extracellular Vesicles for the Treatment of Recessive Dystrophic Epidermolysis Bullosa Wounds
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.
Read the 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.
- Conditions
- Dystrophic Epidermolysis Bullosa
- Being tested
- AGLE-102
- Keywords
- Exosomes, Extracellular Vesicles
- Medical subject headings
- Epidermolysis Bullosa Dystrophica
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AGLE-102Drug
Also called AGLE 102, Control
Extracellular vesicles from bone marrow derived mesenchymal stem cells - up to six topical administrations
| Study group | Role | What they receive |
|---|---|---|
| AGLE 102 | Experimental | Treatment arm Receives: Drug: AGLE-102 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Treatment-emergent Adverse Events
Incidence of adverse events after single and repeat administrations of EVs
Measured: 22 weeks
Wound healing improvement
Change in wound surface area relative to baseline compared with standard of care control
Measured: 10 weeks
Secondary outcomes (5)
Complete wound closure efficacy
Incidence of complete wound closure versus standard of care alone within 10 weeks
Measured: 10 weeks
Time from baseline to complete wound closure
Time to wound closure versus standard of care alone
Measured: 22 weeks
Scar quality improvement
Change from baseline in Patient and Observer Scar Assessment Scales. The min value is 1 and the max value is 10. The min value is normal skin and the max value is worst scar imaginable.
Measured: 22 weeks
Pain relief efficacy
Change from baseline in patient reported pain using the Wong-Baker Faces Pain rating scale. The minimum value is value is 0 (no pain) and the maximum value is 10 (hurts worst).
Measured: 22 weeks
Quality of Life Improvement
Change from baseline in patient reported Quality of Life scale. The minimum value is 0 (would does not affect you at all) and the maximum value is 5 (wound has maximum impact) on care, daily function, appearance, and concerns regarding others' reactions to the wound.
Measured: 22 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Nov 2019
- Study start13 Aug 2024
- Primary completionSep 2025 (estimated)
- Study completionMar 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Dystrophic Epidermolysis BullosaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Model description
- 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.
- Masking
- None (open label)
- Enrolment
- 8 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Aegle Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- David T Woodley, MD, Principal investigator, University of Southern California Dept of Dermatology
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04173650
- Sponsor's protocol ID
- EB IND
- Status last verified
- Jun 2025
- First submitted
- 18 Nov 2019
- Last updated
- 25 Jun 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Jun 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Mesenchymal Stem Cell Extracellular Vesicles for the Treatment of Recessive Dystrophic Epidermolysis Bullosa Wounds
- ClinicalTrials.gov ID
- NCT04173650
- Acronym / sponsor ID
- EB IND
- Conditions
- Dystrophic Epidermolysis Bullosa
- Keywords
- Exosomes, Extracellular Vesicles
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 8 participants (estimated)
- Lead sponsor
- Aegle TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 13 Aug 2024 (actual)
- Primary completion
- Sep 2025 (estimated)
- Study completion
- Mar 2026 (estimated)
- First posted
- 22 Nov 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- 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.
| Arm | Type | Receives |
|---|---|---|
| AGLE 102 | Experimental | Drug: AGLE-102Treatment arm |
Who may qualify
- Ages
- 6 months and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (8 listed)
- 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.
Key exclusion criteria (16 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AGLE-102Also: AGLE 102, ControlExtracellular vesicles from bone marrow derived mesenchymal stem cells - up to six topical administrations | Drug | AGLE 102 |
Primary outcome
Treatment-emergent Adverse Events
Time frame: 22 weeks
Incidence of adverse events after single and repeat administrations of EVs
Wound healing improvement
Time frame: 10 weeks
Change in wound surface area relative to baseline compared with standard of care control
5 secondary outcomes
Complete wound closure efficacy
Time frame: 10 weeks
Time from baseline to complete wound closure
Time frame: 22 weeks
Scar quality improvement
Time frame: 22 weeks
Pain relief efficacy
Time frame: 22 weeks
Quality of Life Improvement
Time frame: 22 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Jun 2025.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
David T Woodley, MD principal investigator
University of Southern California Dept of Dermatology
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing AGLE-102 in people with Dystrophic Epidermolysis Bullosa, run by Aegle Therapeutics at 3 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 25 Jun 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Mei Chen, Ph.D. (323-865-0621, chenm@usc.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Treatment-emergent Adverse Events (time frame: 22 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Jun 2025.