NCT02178969

Brief 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.

Trial Health

93
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
29

participants targeted

Target at below P25 for all trials

Timeline
Completed

Started Jun 2014

Shorter than P25 for all trials

Geographic Reach
6 countries

9 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

June 4, 2014

Completed
20 days until next milestone

Study Start

First participant enrolled

June 24, 2014

Completed
7 days until next milestone

First Posted

Study publicly available on registry

July 1, 2014

Completed
8 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 23, 2015

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

February 23, 2015

Completed
Last Updated

March 17, 2021

Status Verified

March 1, 2021

Enrollment Period

8 months

First QC Date

June 4, 2014

Last Update Submit

March 15, 2021

Conditions

Dystrophic Epidermolysis Bullosa

Outcome Measures

Primary Outcomes (2)

  • Wound Surface Area (WSA) of patient- and investigator-selected wounds

    Over 4 weeks

  • Wound Surface Area (WSA) as a percentage of Body Surface Area (BSA) in patients with DEB

    Over 4 weeks

Secondary Outcomes (1)

  • The number, severity and relationship to study procedures of adverse events (AEs) and serious AEs (SAEs)

    Over 4 weeks

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Patients with DEB.

You may qualify if:

  • 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.

You may not qualify if:

  • Patient has squamous cell carcinoma with evidence of locally invasive disease or distant metastases.
  • Patient is pregnant.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (10)

Ann and Robert H Lurie Childrens Hospital of Chicago

Chicago, Illinois, 60611, United States

Location

Columbia University Medical Center

New York, New York, 10032, United States

Location

Cincinnati Childrens Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Premier Specialists Pty Ltd

Kogarah, 2217, Australia

Location

Salzburger Landeskliniken

Salzburg, 5020, Austria

Location

Fakultni nemocnice Brno

Brno, 613 00, Czechia

Location

Service de Génétique et INSERM UMR 1163, Laboratoire de Génétique des maladies cutanées - Institut Imagine

Paris, France

Location

Universitätsklinikum Freiburg

Freiburg im Breisgau, 79104, Germany

Location

Ospedale Pediatrico Bambino Gesù

Roma, 00165, Italy

Location

Istituto Dermopatico Dell'immacolata IRCCS

Rome, 00167, Italy

Location

Related Publications (1)

  • 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.

    PMID: 35964087DERIVED

Related Links

Biospecimen

Retention: SAMPLES WITH DNA

Optional genetic test for DEB diagnosis

MeSH Terms

Conditions

Epidermolysis Bullosa Dystrophica

Condition Hierarchy (Ancestors)

Epidermolysis BullosaSkin AbnormalitiesCongenital AbnormalitiesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesSkin Diseases, GeneticGenetic Diseases, InbornCollagen DiseasesConnective Tissue DiseasesSkin and Connective Tissue DiseasesSkin DiseasesSkin Diseases, Vesiculobullous

Study Officials

  • Study Director

    Takeda

    STUDY DIRECTOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 4, 2014

First Posted

July 1, 2014

Study Start

June 24, 2014

Primary Completion

February 23, 2015

Study Completion

February 23, 2015

Last Updated

March 17, 2021

Record last verified: 2021-03

Locations

AU(1)IT(2)FR(1)DE(1)CZ(1)US(3)