Dystrophic Epidermolysis BullosaNCT02178969
Short Term Observational Study in DEB Patients
An observational study of Dystrophic Epidermolysis Bullosa, run by Shire at 10 sites in 7 countries.
Trial summary
The purpose of this study is to better understand disease extent and to identify appropriate methodologies to evaluate (dystrophic epidermolysis bullosa) DEB in a quantitative and qualitative manner. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Dystrophic Epidermolysis Bullosa
- How many people
- 29 participants
- Where
- 10 sitesin 7 countries
- Study dates
- 24 Jun 2014to 23 Feb 2015
- Phase
- Observational
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients with DEB.
Who may be able to join
- Written informed consent is provided. Patients 18 years of age and older, and parent(s)/legal guardian(s) of patients younger than 18 years of age, must provide written informed consent prior to participating in the study; additionally, informed assent will be obtained from patients younger than 18 years of age as specified by local requirements.
- Patient must have a documented diagnosis of DEB based on genetic analysis showing a mutation in COL7A1 or, in the opinion of the investigator, a confirmed diagnosis of DEB based on histologic criteria (antigen mapping or electron microscopy).
- Patient must have at least 5 wounds that are suitable for imaging, in the opinion of the investigator, at the time of enrollment.
- Patient is willing and able to undergo the protocol-specified procedures.
Who cannot join
- Patient has used or is currently using experimental treatment for DEB including, but not limited to, bone marrow transplantation, systemic immune suppression, or experimental procedures that involve live cells, with potential for systemic spread such as gene transfer, stem cell infusions, or other cell type injections such that, in the opinion of the investigator, inclusion poses an unacceptable risk to the patient or interpretation of these study data.
- Patient has squamous cell carcinoma with evidence of locally invasive disease or distant metastases.
- Patient is pregnant.
Trial locations and contacts
10 sites in 7 countries.
10 sites in 7 countries
United States3 sites
Ann and Robert H Lurie Childrens Hospital of Chicago
Chicago, Illinois, 60611
Cincinnati Childrens Hospital Medical Center
Cincinnati, Ohio, 45229
Columbia University Medical Center
New York, New York, 10032
Italy2 sites
Ospedale Pediatrico Bambino Gesù
Roma, 00165
Istituto Dermopatico Dell'immacolata IRCCS
Rome, 00167
France1 site
Service de Génétique et INSERM UMR 1163, Laboratoire de Génétique des maladies cutanées - Institut Imagine
Paris
About this study
Official title: A Prospective Short-term Study to Evaluate Methodologies for the Assessment of Disease Extent, Impact, and Wound Evolution in Patients With Dystrophic Epidermolysis Bullosa (DEB)
The purpose of this study is to better understand disease extent and to identify appropriate methodologies to evaluate (dystrophic epidermolysis bullosa) DEB in a quantitative and qualitative manner.
- Conditions
- 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.
Wound Surface Area (WSA) of patient- and investigator-selected wounds
Measured: Over 4 weeks
Wound Surface Area (WSA) as a percentage of Body Surface Area (BSA) in patients with DEB
Measured: Over 4 weeks
Secondary outcomes (1)
The number, severity and relationship to study procedures of adverse events (AEs) and serious AEs (SAEs)
Measured: Over 4 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Jul 2014 (estimated)
- Study start24 Jun 2014
- Primary completion23 Feb 2015
- Study completion23 Feb 2015
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.
- 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
- Cohort
- Time perspective
- Prospective
- Enrolment
- 29 participants (actual)
Sponsor and investigators
- Lead sponsor
- Shire
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02178969
- Sponsor's protocol ID
- SHP-608-002
- Status last verified
- Mar 2021
- First submitted
- 4 Jun 2014
- Last updated
- 17 Mar 2021
- Results posted
- No
- Data monitoring committee
- No
Publications
Paller AS, Pope E, Rudin D, Malyala A, Ramsdell D, Johnson R, Landy H, Murrell DF; DEB Investigators. A prospective short-term study to evaluate methodologies for the assessment of disease extent, impact, and wound evolution in patients with dystrophic epidermolysis bullosa. Orphanet J Rare Dis. 2022 Aug 13;17(1):314. doi: 10.1186/s13023-022-02461-z.
PubMed 35964087
Study at a glance
From the registry record, last updated by the sponsor on 17 Mar 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective Short-term Study to Evaluate Methodologies for the Assessment of Disease Extent, Impact, and Wound Evolution in Patients With Dystrophic Epidermolysis Bullosa (DEB)
- ClinicalTrials.gov ID
- NCT02178969
- Acronym / sponsor ID
- SHP-608-002
- Conditions
- Dystrophic Epidermolysis Bullosa
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 29 participants (actual)
- Lead sponsor
- ShireIndustry (pharma, biotech, devices)
- Study start
- 24 Jun 2014 (actual)
- Primary completion
- 23 Feb 2015 (actual)
- Study completion
- 23 Feb 2015 (actual)
- First posted
- 1 Jul 2014 (estimated)
- 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 purpose of this study is to better understand disease extent and to identify appropriate methodologies to evaluate (dystrophic epidermolysis bullosa) DEB in a quantitative and qualitative manner.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients with DEB.
Key inclusion criteria (4 listed)
- Written informed consent is provided. Patients 18 years of age and older, and parent(s)/legal guardian(s) of patients younger than 18 years of age, must provide written informed consent prior to participating in the study; additionally, informed assent will be obtained from patients younger than 18 years of age as specified by local requirements.
- Patient must have a documented diagnosis of DEB based on genetic analysis showing a mutation in COL7A1 or, in the opinion of the investigator, a confirmed diagnosis of DEB based on histologic criteria (antigen mapping or electron microscopy).
- Patient must have at least 5 wounds that are suitable for imaging, in the opinion of the investigator, at the time of enrollment.
- Patient is willing and able to undergo the protocol-specified procedures.
Key exclusion criteria (3 listed)
- Patient has used or is currently using experimental treatment for DEB including, but not limited to, bone marrow transplantation, systemic immune suppression, or experimental procedures that involve live cells, with potential for systemic spread such as gene transfer, stem cell infusions, or other cell type injections such that, in the opinion of the investigator, inclusion poses an unacceptable risk to the patient or interpretation of these study data.
- Patient has squamous cell carcinoma with evidence of locally invasive disease or distant metastases.
- Patient is pregnant.
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
Wound Surface Area (WSA) of patient- and investigator-selected wounds
Time frame: Over 4 weeks
Wound Surface Area (WSA) as a percentage of Body Surface Area (BSA) in patients with DEB
Time frame: Over 4 weeks
1 secondary outcome
The number, severity and relationship to study procedures of adverse events (AEs) and serious AEs (SAEs)
Time frame: Over 4 weeks
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 17 Mar 2021.
- Derived from this study: PubMed 35964087Paller AS, Pope E, Rudin D, Malyala A, Ramsdell D, Johnson R, Landy H, Murrell DF; DEB Investigators. A prospective short-term study to evaluate methodologies for the assessment of disease extent, impact, and wound evolution in patients with dystrophic epidermolysis bullosa. Orphanet J Rare Dis. 2022 Aug 13;17(1):314. doi: 10.1186/s13023-022-02461-z.
- To obtain more information on the study, click here/on this link.Link provided by the sponsor.
Related trials for Dystrophic Epidermolysis Bullosa
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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.
Study Director study director
Takeda
10 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Dystrophic Epidermolysis Bullosa, run by Shire at 10 sites in 7 countries.
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 4 inclusion and 3 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 17 Mar 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in United States, Italy, Australia, Austria, Czechia and 2 more countries. 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?
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?
Wound Surface Area (WSA) of patient- and investigator-selected wounds (time frame: Over 4 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?
On the ClinicalTrials.gov record for NCT02178969, 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 17 Mar 2021.