Dystrophic Epidermolysis BullosaNCT01768026
Prospective, Longitudinal Natural History Study in Dystrophic Epidermolysis Bullosa
An observational study of Dystrophic Epidermolysis Bullosa, run by Lotus Tissue Repair, Inc. at 1 site in United States.
Trial summary
The objective of this study is to characterize the extent and severity of disease in subjects with DEB and the progression of disease over a timeframe relevant to interventional studies. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Dystrophic Epidermolysis Bullosa
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- Feb 2013to Sep 2014
- 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 will be selected from clinical sites with interdisciplinary clinics for Dystrophic Epidermolysis Bullosa
Eligibility Criteria
- Subjects of any age (newborns included) may participate
- Subjects over 18 years of age and parent(s)/legal guardian(s) of subjects <18 years of age must provide written informed consent prior to participating in the study and informed assent will be obtained from minors at least 7 years of age
- Subjects must have a documented diagnosis of DEB based on clinical presentation and either skin biopsy results showing an absence or reduction in C7 or anchoring fibrils or genetic analysis showing a mutation in collagen, type VII, alpha 1 (Col7A1); alternatively, subjects must have a clinical diagnosis of DEB and a documented diagnosis of DEB (as above) in a first degree relative
- No experimental systemic therapy for DEB including, but not limited to, bone marrow transplantation, systemic immune suppression, or experimental therapies that involve live cells which have the potential for systemic spread such as gene transfer, stem cell infusions or other cell type injections
Trial locations and contacts
1 site in 1 country.
United States1 site
Stanford University School of Medicine
Palo Alto, California, 94304
About this study
Official title: A Prospective, Longitudinal Assessment of Disease Severity in Subjects With Dystrophic Epidermolysis Bullosa (DEB)
The objective of this study is to characterize the extent and severity of disease in subjects with DEB and the progression of disease over a timeframe relevant to interventional studies. The data from this study will be used to inform the study design and address statistical considerations of future treatment protocols.
Read the detailed description
This is a prospective, multicenter, multinational, longitudinal assessment of disease severity in subjects with DEB. Subjects with either dominant or recessive DEB (dominant dystrophic epidermolysis bullosa (DDEB) and recessive dystrophic epidermolysis bullosa (RDEB), respectively) will be assessed four times over a one year period: upon enrollment, and at 1 to 2 weeks and 6 and 12 months after enrollment. All subjects with either DDEB or RDEB that meet the study entry criteria will be offered participation in the study, provided they can be accommodated within the anticipated study timeline. In addition to their usual clinic assessment, subjects will have a quantitative evaluation of skin involvement and will be asked to fill out questionnaires that measure among other things disease severity, QOL, pain, pruritus, and medical and family histories.
- Conditions
- Dystrophic Epidermolysis Bullosa
- Keywords
- Dystrophic Epidermolysis Bullosa, Type VII Collagen, QOL Evaluation in Epidermolysis Bullosa, Instrument for Scoring Clinical Outcomes for Research of Epidermolysis Bullosa, Birmingham Epidermolysis Bullosa Severity Score
- 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.
| Study group | Role | What they receive |
|---|---|---|
| No treatment | Subjects diagnosed with Dystrophic Epidermolysis Bullosa |
What is being measured
The primary outcome is the main question the study is designed to answer.
Characterize the progression of disease severity in subjects with DEB over 6 - 12 months.
Disease severity and its impact on quality of life and function will be investigated over a one year period at the following timepoints: upon enrollment, and at 1 to 2 weeks and 6 and 12 months after enrollment.
Measured: One year period
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Jan 2013 (estimated)
- Study startFeb 2013
- Primary completionSep 2014 (estimated)
- Study completionSep 2014 (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
- Cohort
- Time perspective
- Prospective
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Lotus Tissue Repair, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Hal Landy, MD, Study director, Lotus Tissue Repair, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01768026
- Sponsor's protocol ID
- DEB-101-12
- Status last verified
- Feb 2013
- First submitted
- 10 Jan 2013
- Last updated
- 21 May 2013
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 May 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective, Longitudinal Assessment of Disease Severity in Subjects With Dystrophic Epidermolysis Bullosa (DEB)
- ClinicalTrials.gov ID
- NCT01768026
- Acronym / sponsor ID
- DEB-101-12
- Conditions
- Dystrophic Epidermolysis Bullosa
- Keywords
- Dystrophic Epidermolysis Bullosa, Type VII Collagen, QOL Evaluation in Epidermolysis Bullosa, Instrument for Scoring Clinical Outcomes for Research of Epidermolysis Bullosa, Birmingham Epidermolysis Bullosa Severity Score
- Study type
- Observational
- Recruitment status
- Withdrawn
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Lotus Tissue Repair, Inc.Industry (pharma, biotech, devices)
- Study start
- Feb 2013
- Primary completion
- Sep 2014 (estimated)
- Study completion
- Sep 2014 (estimated)
- First posted
- 15 Jan 2013 (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 objective of this study is to characterize the extent and severity of disease in subjects with DEB and the progression of disease over a timeframe relevant to interventional studies. The data from this study will be used to inform the study design and address statistical considerations of future treatment protocols.
Detailed description
This is a prospective, multicenter, multinational, longitudinal assessment of disease severity in subjects with DEB. Subjects with either dominant or recessive DEB (dominant dystrophic epidermolysis bullosa (DDEB) and recessive dystrophic epidermolysis bullosa (RDEB), respectively) will be assessed four times over a one year period: upon enrollment, and at 1 to 2 weeks and 6 and 12 months after enrollment. All subjects with either DDEB or RDEB that meet the study entry criteria will be offered participation in the study, provided they can be accommodated within the anticipated study timeline. In addition to their usual clinic assessment, subjects will have a quantitative evaluation of skin involvement and will be asked to fill out questionnaires that measure among other things disease severity, QOL, pain, pruritus, and medical and family histories.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| No treatment | – | –Subjects diagnosed with Dystrophic Epidermolysis Bullosa |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients will be selected from clinical sites with interdisciplinary clinics for Dystrophic Epidermolysis Bullosa
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
Characterize the progression of disease severity in subjects with DEB over 6 - 12 months.
Time frame: One year period
Disease severity and its impact on quality of life and function will be investigated over a one year period at the following timepoints: upon enrollment, and at 1 to 2 weeks and 6 and 12 months after enrollment.
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 May 2013.
- Dystrophic Epidermolysis Bullosa Research Association of America (DEBRA)Link provided by the sponsor.
- EB Care Patient Reported RegistryLink provided by the sponsor.
- Lotus Tissue Repair, Inc.Link provided by the sponsor.
- Dystrophic Epidermolysis Bullosa Research Association InternationalLink provided by the sponsor.
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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. This study is not recruiting at the moment.
Hal Landy, MD study director
Lotus Tissue Repair, Inc.
1 site 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, run by Lotus Tissue Repair, Inc. at 1 site in United States.
The full registered description is in the About this study section and the official record.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 21 May 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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?
The study was withdrawn before enrolling participants, so there are no results.
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?
Characterize the progression of disease severity in subjects with DEB over 6 - 12 months. (time frame: One year period). 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 21 May 2013.