Dystrophic Epidermolysis BullosaNCT04917887
Long-Term Follow-up Protocol
An observational study of Dystrophic Epidermolysis Bullosa and Recessive Dystrophic Epidermolysis Bullosa, run by Krystal Biotech, Inc. at 3 sites in United States.
Trial summary
The main objective of this prospective, observational, long-term follow-up (LTFU) study is to evaluate the long-term safety profile of the gene therapy products evaluated by Krystal Biotech, Inc. which have a shared backbone of HSV-1, in participants who received at least one dose of investigational product (IP). Read more about this study
- Who is it for?
- Ages 6 months and older
- Conditions the study is for
- Dystrophic Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa, Dominant Dystrophic Epidermolysis Bullosa
- How many people
- 50 participants
- Where
- 3 sitesUnited States
- Study dates
- 25 May 2021to 25 May 2028
- Phase
- Observational
- 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
Study population: Participants who received at least one treatment of gene therapy from a Krystal Biotech, Inc. sponsored study, will be asked to roll-over to this LTFU protocol upon either premature discontinuation from, or completion of a prior parent treatment protocol. However, if a participant enrolls in a different Sponsor gene therapy trial during the LTFU phase this participant will be dropped out from the study.
Who may be able to join
- All adult and pediatric participants who received at least one gene therapy treatment in a previous Krystal Biotech, Inc. sponsored study, and have discontinued or completed the parent treatment protocol (which do not have the LTFU embedded into the parent protocol), as applicable.
- Consent/Assent must be obtained by the participants (and, parental/legal representative, when applicable) prior to any study-related data being collected.
- Participant is willing and able to adhere to the protocol requirements.
Who cannot join
- Participants who enroll into a non-Krystal Biotech, Inc. interventional gene therapy clinical trial will be excluded from the study.
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
Pediatric Skin Research
Coral Gables, Florida, 33146
Edilmary Urdaneta
Mercedes Gonzalez, MD
- Recruiting
Mission Dermatology Center
Rancho Santa Margarita, California, 92688
Guida Pinto, 949-858-3376 ext. 108, gpinto.missionderm@gmail.com
Shireen Guide, MD
- Recruiting
Stanford University
Redwood City, California, 94063
Sinem Bagci, 650-484-6878, isbagci@stanford.edu
Peter Marinkovich, MD
About this study
Official title: A Prospective, Observational, Long-Term Follow-Up (LTFU) Study to Evaluate the Long-Term Safety of the Krystal Biotech, Inc. Gene Therapy Backbone Products Using HSV-1 Backbone
The main objective of this prospective, observational, long-term follow-up (LTFU) study is to evaluate the long-term safety profile of the gene therapy products evaluated by Krystal Biotech, Inc. which have a shared backbone of HSV-1, in participants who received at least one dose of investigational product (IP).
Read the detailed description
Due to the guidelines for gene therapy for vectors which may undergo latency/ reactivation, Krystal Biotech, Inc. has established a Long-Term Follow-up Protocol which will identify and mitigate the long-term risks to the participants receiving their gene therapy products and capture delayed adverse events in participants, annually, for 5-years following the last study visit. Additionally, it will allow the Sponsor to further understand the persistence of the gene therapy products used in their parent treatment protocols.
- Conditions
- Dystrophic Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa, Dominant Dystrophic Epidermolysis Bullosa
- Medical subject headings
- Epidermolysis Bullosa Dystrophica
What is being measured
The primary outcome is the main question the study is designed to answer.
Serious Adverse Events (AE)
Incidence of delayed Adverse Events suspected to be related to a Krystal Biotech HSV-1 vector backbone investigational product.
A Serious Adverse Event (SAE) is any untoward medical occurrence (whether considered to be related to gene therapy or not) that:
- Is fatal
- Is life-threatening (places the participant at immediate risk of death)
- Requires inpatient hospitalization or prolongation of existing hospitalization
- Is a persistent or significant disability/incapacity, or
- Is a congenital abnormality/birth defect
Measured: 5 years
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 completion25 May 2028 (estimated)
- Study completion25 May 2028 (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
- Observational
- Observational model
- Other
- Time perspective
- Prospective
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Krystal Biotech, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04917887
- Sponsor's protocol ID
- KRYS-LTFU-01
- Status last verified
- Jun 2021
- First submitted
- 2 Jun 2021
- Last updated
- 8 Jun 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Jun 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective, Observational, Long-Term Follow-Up (LTFU) Study to Evaluate the Long-Term Safety of the Krystal Biotech, Inc. Gene Therapy Backbone Products Using HSV-1 Backbone
- ClinicalTrials.gov ID
- NCT04917887
- Acronym / sponsor ID
- KRYS-LTFU-01
- Conditions
- Dystrophic Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa, Dominant Dystrophic Epidermolysis Bullosa
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Krystal Biotech, Inc.Industry (pharma, biotech, devices)
- Study start
- 25 May 2021 (actual)
- Primary completion
- 25 May 2028 (estimated)
- Study completion
- 25 May 2028 (estimated)
- 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.
The main objective of this prospective, observational, long-term follow-up (LTFU) study is to evaluate the long-term safety profile of the gene therapy products evaluated by Krystal Biotech, Inc. which have a shared backbone of HSV-1, in participants who received at least one dose of investigational product (IP).
Detailed description
Due to the guidelines for gene therapy for vectors which may undergo latency/ reactivation, Krystal Biotech, Inc. has established a Long-Term Follow-up Protocol which will identify and mitigate the long-term risks to the participants receiving their gene therapy products and capture delayed adverse events in participants, annually, for 5-years following the last study visit. Additionally, it will allow the Sponsor to further understand the persistence of the gene therapy products used in their parent treatment protocols.
Study design
- Observational model
- Other
- Time perspective
- Prospective
Who may qualify
- Ages
- 6 months and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Participants who received at least one treatment of gene therapy from a Krystal Biotech, Inc. sponsored study, will be asked to roll-over to this LTFU protocol upon either premature discontinuation from, or completion of a prior parent treatment protocol. However, if a participant enrolls in a different Sponsor gene therapy trial during the LTFU phase this participant will be dropped out from the study.
Key inclusion criteria (3 listed)
- All adult and pediatric participants who received at least one gene therapy treatment in a previous Krystal Biotech, Inc. sponsored study, and have discontinued or completed the parent treatment protocol (which do not have the LTFU embedded into the parent protocol), as applicable.
- Consent/Assent must be obtained by the participants (and, parental/legal representative, when applicable) prior to any study-related data being collected.
- Participant is willing and able to adhere to the protocol requirements.
Key exclusion criteria (1 listed)
- Participants who enroll into a non-Krystal Biotech, Inc. interventional gene therapy clinical trial will be excluded from the study.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Serious Adverse Events (AE)
Time frame: 5 years
Incidence of delayed Adverse Events suspected to be related to a Krystal Biotech HSV-1 vector backbone investigational product. A Serious Adverse Event (SAE) is any untoward medical occurrence (whether considered to be related to gene therapy or not) that: * Is fatal * Is life-threatening (places the participant at immediate risk of death) * Requires inpatient hospitalization or prolongation of existing hospitalization * Is a persistent or significant disability/incapacity, or * Is a congenital abnormality/birth defect
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 8 Jun 2021.
- Individual participant dataSharing planned: No
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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.
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Dystrophic Epidermolysis Bullosa and Recessive Dystrophic Epidermolysis Bullosa, run by Krystal Biotech, Inc. 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 3 inclusion and 1 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 8 Jun 2021. 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.
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 Brittani Agostini (4125865830, bagostini@krystalbio.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Serious Adverse Events (AE) (time frame: 5 years). 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 8 Jun 2021.