- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01768026
Prospective, Longitudinal Natural History Study in Dystrophic Epidermolysis Bullosa
May 20, 2013 updated by: Lotus Tissue Repair, Inc.
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.
Study Overview
Status
Withdrawn
Conditions
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 Type
Observational
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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California
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Palo Alto, California, United States, 94304
- Stanford University School of Medicine
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
- Child
- Adult
- Older Adult
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Sampling Method
Non-Probability Sample
Study Population
Patients will be selected from clinical sites with interdisciplinary clinics for Dystrophic Epidermolysis Bullosa
Description
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
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Observational Models: Cohort
- Time Perspectives: Prospective
Cohorts and Interventions
Group / Cohort |
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No treatment
Subjects diagnosed with Dystrophic Epidermolysis Bullosa
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Characterize the progression of disease severity in subjects with DEB over 6 - 12 months.
Time Frame: One year period
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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.
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One year period
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Study Director: Hal Landy, MD, Lotus Tissue Repair, Inc.
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start
February 1, 2013
Primary Completion (Anticipated)
September 1, 2014
Study Completion (Anticipated)
September 1, 2014
Study Registration Dates
First Submitted
January 10, 2013
First Submitted That Met QC Criteria
January 11, 2013
First Posted (Estimate)
January 15, 2013
Study Record Updates
Last Update Posted (Estimate)
May 21, 2013
Last Update Submitted That Met QC Criteria
May 20, 2013
Last Verified
February 1, 2013
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- DEB-101-12
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Dystrophic Epidermolysis Bullosa
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Krystal Biotech, Inc.CompletedDystrophic Epidermolysis Bullosa | Recessive Dystrophic Epidermolysis Bullosa | Dominant Dystrophic Epidermolysis Bullosa | DEB - Dystrophic Epidermolysis BullosaUnited States
-
Krystal Biotech, Inc.CompletedDystrophic Epidermolysis Bullosa | Recessive Dystrophic Epidermolysis Bullosa | Dominant Dystrophic Epidermolysis BullosaUnited States
-
Krystal Biotech, Inc.RecruitingDystrophic Epidermolysis Bullosa | Recessive Dystrophic Epidermolysis Bullosa | Dominant Dystrophic Epidermolysis BullosaUnited States
-
Castle Creek Biosciences, LLC.TerminatedEpidermolysis Bullosa Dystrophica, RecessiveUnited States
-
Instituto de Investigación Hospital Universitario...Instituto de Salud Carlos III; Universidad Carlos III Madrid (TERMeG); St John... and other collaboratorsUnknownEpidermolysis Bullosa Dystrophica, RecessiveSpain
-
Phoenicis TherapeuticsNot yet recruitingDystrophic Epidermolysis BullosaUnited States
-
Thomas Jefferson UniversityOnconova Therapeutics, Inc.RecruitingRecessive Dystrophic Epidermolysis BullosaUnited States
-
Castle Creek Biosciences, LLC.Active, not recruitingRecessive Dystrophic Epidermolysis BullosaUnited States
-
Phoenix Tissue Repair, Inc.Phoenix Tissue Repair, a BridgeBio companyCompletedRecessive Dystrophic Epidermolysis BullosaUnited States
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RHEACELL GmbH & Co. KGFGK Clinical Research GmbH; Ticeba GmbH; Granzer Regulatory Consulting & ServicesCompletedRecessive Dystrophic Epidermolysis BullosaUnited States, Austria, France, Germany, United Kingdom