Dystrophic Epidermolysis BullosaPhase 3NCT04917874
A Long-term Treatment With B-VEC for Dystrophic Epidermolysis Bullosa
A Phase 3 study testing Open Label Topical Beremagene Geperpavec (B-VEC) in people with Dystrophic Epidermolysis Bullosa and DEB - Dystrophic Epidermolysis Bullosa, run by Krystal Biotech, Inc. at 6 sites in United States.
Trial summary
This is a 112-week (approximately two-year) open-label extension study of Beremagene Geperpavec (B-VEC), for participants aged 2 months and older, who have been diagnosed with Dystrophic Epidermolysis Bullosa (DEB). Participants will be dosed weekly with the topical B-VEC therapy. Read more about this study
- Who is it for?
- Ages 2 months and older
- Conditions the study is for
- Dystrophic Epidermolysis Bullosa, DEB - Dystrophic Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa
- What is being tested?
- Open Label Topical Beremagene Geperpavec (B-VEC)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 47 participants
- Where
- 6 sitesUnited States
- Study dates
- 25 May 2021to 31 Jul 2023
- Phase
- Phase 3
- Are there results?
- None posted in the registry
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 2 months and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Willing and able to give consent/assent
- Clinical diagnosis of epidermolysis bullosa
- Confirmation of diagnosis (either DDEB or RDEB) by genetic testing including COL7A1.
- Age: 2 months of age and older at the time of informed consent/assent
- Women of childbearing age must be willing to use reliable birth control method throughout the treatment duration and for 3 months following the last treatment application
Who cannot join
- Diseases or conditions that could interfere with the assessment of safety of the study treatment and compliance of the participant with study visits/procedures, as determined by the Investigator
- Pregnant or nursing women
- Active infection in the area that will undergo administration, that the Investigator believes will negatively impact the IP application
- Known allergy to any of the constituents of the product
- Concurrent skin transplant or mesh skin grafting; if the participant is currently having transplantation or grafting surgeries they must wait until the transplant has begun to heal and the graft has absorbed prior to initiating B-VEC therapy
- Participation in an interventional gene therapy clinical trial within the past three (3) months (not including B-VEC administration)
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
United States6 sites
Ascension Seton- Dell's Children Medical Group
Austin, Texas, 78723
Northwestern University
Chicago, Illinois, 60611
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Pediatric Skin Research
Coral Gables, Florida, 33146
Mission Dermatology Center
Rancho Santa Margarita, California, 92688
Stanford University
Redwood City, California, 94063
About this study
Official title: Open Label Treatment of Beremagene Geperpavec (B-VEC)
This is a 112-week (approximately two-year) open-label extension study of Beremagene Geperpavec (B-VEC), for participants aged 2 months and older, who have been diagnosed with Dystrophic Epidermolysis Bullosa (DEB). Participants will be dosed weekly with the topical B-VEC therapy. The primary endpoint will be to assess long term safety and tolerability of the topical gene therapy. The study is for those who participated in Phase 3 study, as well as, new participants who were unable to participate in the Phase 3 study, who meet all enrollment criteria.
- Conditions
- Dystrophic Epidermolysis Bullosa, DEB - Dystrophic Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa, Dominant Dystrophic Epidermolysis Bullosa
- Being tested
- Open Label Topical Beremagene Geperpavec (B-VEC)
- Keywords
- DEB, Krystal Biotech
- 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.
Open Label Topical Beremagene Geperpavec (B-VEC)Biological
Topical gel of non-integrating, replication-incompetent HSV-1 expressing the human collagen VII protein
| Study group | Role | What they receive |
|---|---|---|
| B-VEC | Experimental | Open label B-VEC topical treatment of DEB wounds. Receives: Biological: Open Label Topical Beremagene Geperpavec (B-VEC) |
What is being measured
The primary outcome is the main question the study is designed to answer.
To record safety outcomes of participants while on B-VEC.
Record reportable Adverse (AE) and Serious Adverse Events (SAEs) during the continued use of B-VEC to participants who have participated in and completed Krystal Biotech's Phase 3 Protocol (A Phase 3 Efficacy and Safety Study of Beremagene Geperpavec (B-VEC, previously KB103) for the Treatment of Dystrophic Epidermolysis Bullosa (DEB)), as an extension of use, upon study completion; as well as, for participants diagnosed with DEB who have not participated in the B-VEC Phase 3 trial.
Measured: Up to 112 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jun 2021
- Study start25 May 2021
- Primary completion31 Jul 2023
- Study completion31 Jul 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Dystrophic Epidermolysis BullosaStudies of the same condition, open and under way.
- More trials run by Krystal Biotech, Inc.The sponsor's other registered studies.
- 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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 47 participants (actual)
Sponsor and investigators
- Lead sponsor
- Krystal Biotech, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- David Chien, MD, Study director, Senior Vice President of Clinical Development
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04917874
- Sponsor's protocol ID
- B-VEC-EX-02
- Status last verified
- Apr 2024
- First submitted
- 2 Jun 2021
- Last updated
- 9 Apr 2024
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Marinkovich MP, Paller AS, Guide SV, Gonzalez ME, Lucky AW, Bagci IS, Agostini B, Fitzgerald K, Chen S, Chen H, Conner MM, Krishnan SM. Long-Term Safety and Tolerability of Beremagene Geperpavec-svdt (B-VEC) in an Open-Label Extension Study of Patients with Dystrophic Epidermolysis Bullosa. Am J Clin Dermatol. 2025 Jul;26(4):623-635. doi: 10.1007/s40257-025-00942-y. Epub 2025 Apr 12.
PubMed 40220208
Study at a glance
From the registry record, last updated by the sponsor on 9 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Open Label Treatment of Beremagene Geperpavec (B-VEC)
- ClinicalTrials.gov ID
- NCT04917874
- Acronym / sponsor ID
- B-VEC-EX-02
- Conditions
- Dystrophic Epidermolysis Bullosa, DEB - Dystrophic Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa, Dominant Dystrophic Epidermolysis Bullosa
- Keywords
- DEB, Krystal Biotech
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 47 participants (actual)
- Lead sponsor
- Krystal Biotech, Inc.Industry (pharma, biotech, devices)
- Study start
- 25 May 2021 (actual)
- Primary completion
- 31 Jul 2023 (actual)
- Study completion
- 31 Jul 2023 (actual)
- First posted
- 8 Jun 2021 (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.
This is a 112-week (approximately two-year) open-label extension study of Beremagene Geperpavec (B-VEC), for participants aged 2 months and older, who have been diagnosed with Dystrophic Epidermolysis Bullosa (DEB). Participants will be dosed weekly with the topical B-VEC therapy. The primary endpoint will be to assess long term safety and tolerability of the topical gene therapy. The study is for those who participated in Phase 3 study, as well as, new participants who were unable to participate in the Phase 3 study, who meet all enrollment criteria.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| B-VEC | Experimental | Biological: Open Label Topical Beremagene Geperpavec (B-VEC)Open label B-VEC topical treatment of DEB wounds. |
Who may qualify
- Ages
- 2 months and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Willing and able to give consent/assent
- Clinical diagnosis of epidermolysis bullosa
- Confirmation of diagnosis (either DDEB or RDEB) by genetic testing including COL7A1.
- Age: 2 months of age and older at the time of informed consent/assent
- Women of childbearing age must be willing to use reliable birth control method throughout the treatment duration and for 3 months following the last treatment application
Key exclusion criteria (6 listed)
- Diseases or conditions that could interfere with the assessment of safety of the study treatment and compliance of the participant with study visits/procedures, as determined by the Investigator
- Pregnant or nursing women
- Active infection in the area that will undergo administration, that the Investigator believes will negatively impact the IP application
- Known allergy to any of the constituents of the product
- Concurrent skin transplant or mesh skin grafting; if the participant is currently having transplantation or grafting surgeries they must wait until the transplant has begun to heal and the graft has absorbed prior to initiating B-VEC therapy
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 |
|---|---|---|
| Open Label Topical Beremagene Geperpavec (B-VEC)Topical gel of non-integrating, replication-incompetent HSV-1 expressing the human collagen VII protein | Biological | B-VEC |
Primary outcome
To record safety outcomes of participants while on B-VEC.
Time frame: Up to 112 weeks
Record reportable Adverse (AE) and Serious Adverse Events (SAEs) during the continued use of B-VEC to participants who have participated in and completed Krystal Biotech's Phase 3 Protocol (A Phase 3 Efficacy and Safety Study of Beremagene Geperpavec (B-VEC, previously KB103) for the Treatment of Dystrophic Epidermolysis Bullosa (DEB)), as an extension of use, upon study completion; as well as, for participants diagnosed with DEB who have not participated in the B-VEC Phase 3 trial.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Apr 2024.
- Derived from this study: PubMed 40220208Marinkovich MP, Paller AS, Guide SV, Gonzalez ME, Lucky AW, Bagci IS, Agostini B, Fitzgerald K, Chen S, Chen H, Conner MM, Krishnan SM. Long-Term Safety and Tolerability of Beremagene Geperpavec-svdt (B-VEC) in an Open-Label Extension Study of Patients with Dystrophic Epidermolysis Bullosa. Am J Clin Dermatol. 2025 Jul;26(4):623-635. doi: 10.1007/s40257-025-00942-y. Epub 2025 Apr 12.
Related trials for Dystrophic Epidermolysis Bullosa
RecruitingPhase 2
A Phase 2 Study of TCP-25 Gel in Patients With Epidermolysis Bullosa, STEP-study
Epidermolysis Bullosa (EB), Dystrophic Epidermolysis Bullosa
RecruitingPhase 2
A Study to Evaluate ENERGI-F703EB Cream in Epidermolysis Bullosa
Epidermolysis Bullosa (EB), Junctional Epidermolysis Bullosa
RecruitingObservational study
A Study to Assess the Incidence of Skin Cancers in Patients With Epidermolysis Bullosa Receiving Filsuvez
Epidermolysis Bullosa, Dystrophic, Epidermolysis Bullosa, Junctional
Not yet recruitingPhase 4
Phase 4 Master Protocol for Patients Prescribed Prademagene Zamikeracel for the Treatment of Wounds
Epidermolysis Bullosa (EB), Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Enrolling by invitationObservational study
A Long-Term Extension Study for Participants Previously Treated With EB-101 for the Treatment of RDEB
RDEB
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. This study is not recruiting at the moment.
David Chien, MD study director
Senior Vice President of Clinical Development
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Open Label Topical Beremagene Geperpavec (B-VEC) in people with Dystrophic Epidermolysis Bullosa and DEB - Dystrophic Epidermolysis Bullosa, run by Krystal Biotech, Inc. at 6 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 5 inclusion and 6 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: finished, as of the sponsor's last update on 9 Apr 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 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 phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To record safety outcomes of participants while on B-VEC. (time frame: Up to 112 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04917874, which also lists 1 linked publication.
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 9 Apr 2024.