Evaluation of D-Fi for the Treatment of Wounds Due to DEB
Evaluation of Dabocemagene Autoficel (D-Fi; FCX-007; Genetically Modified Autologous Human Dermal Fibroblasts) for the Treatment of Wounds Due to Dystropic Epidermolysis Bullosa
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
CCB-EB-304 is a multi-center, intra-patient randomized and controlled, open-label, rater-blinded Phase 3 study of D-Fi for the treatment of persistent non-healing and recurrent DEB wounds in approximately 32 subjects. Each subject will serve as his/her own control. Each subject's Target Wounds will be paired then randomized to receive D-Fi (Treatment Wound) or remain untreated (Control Wound). One Target Wound Pair will be identified for each subject.
Subjects will receive intradermal injections of D-Fi in each specified Treatment Wound in three or more treatment sessions. The first treatment session occurs at Day 1, the second at Week 8/Month 2 and the third at Week 16/Month 4. Additional treatment sessions may occur at Week 26/Month 6 and Week 32/Month 8 and Week 40/Month 10 when unclosed Treatment Wounds may be re-treated, and unclosed Control Wounds may be treated. Safety and efficacy assessments will occur at scheduled intervals through Week 48/Month 12, when the Treatment Period is completed, and is followed by an optional Open-Label Extension (OLE) treatment phase of an additional 96 weeks. A long-term safety follow-up period (through 15 years) commences for subjects who have received one or more D-Fi injections, either following the treatment phase or if the subject had opted-in, the OLE.
Study Type
Enrollment (Estimated)
Phase
- Phase 3
Contacts and Locations
Study Locations
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California
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Redwood City, California, United States, 94063
- Stanford University School of Medicine, Dermatology
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Santa Margarita, California, United States, 92688
- Mission Dermatology
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Colorado
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Aurora, Colorado, United States, 80045
- Children's Hospital Colorado
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Massachusetts
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Worcester, Massachusetts, United States, 01655
- University of Massachusetts
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-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
- Child
- Adult
- Older Adult
Accepts Healthy Volunteers
Description
Inclusion Criteria:
- Male or female ≥2 years of age at the Screening visit
- Clinical diagnosis of DEB with confirmation of COL7A1 genetic mutation.
Exclusion Criteria:
- Medical instability limiting ability to travel to the investigative site.
- Active infection with human immunodeficiency virus, hepatitis B or hepatitis C.
- The presence of clinically significant COL7 antibodies.
- Evidence of systemic infection.
- Known allergy to any of the constituents of the product.
- Female who is pregnant or breastfeeding.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
|---|---|
|
Experimental: D-Fi COL7A1 Genetically-Corrected Autologous Fibroblasts
Intra-subject randomized (paired wounds in each subject receive experimental treatment, D-Fi, or remain untreated).
One target wound pair will be identified for each subject.
Following pairing, target wounds will be randomly assigned as the treatment wound (D-Fi is administered) or control wound.
Subjects will receive intradermal injections of D-Fi in each specified treatment wound in two or more treatment sessions.
The first treatment session occurs at Day 1, the second at Week 8/Month 2 and the third at Week 16/Month 4. Additional treatment sessions may occur at Week 26/Month 6, Week 32/Month 8 and Week 40/Month 10 when unclosed treatment wounds may be re-treated, and unclosed control wounds may be treated.
|
D-Fi is composed of fibroblasts isolated from the subject's skin biopsies which are genetically corrected with the full length COL7A1 gene encoding for type VII collagen.
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
|---|---|
|
The difference in Complete (100%) Wound Closure of the Target Wound Pair at either Week 22 (Visit 6), and Week 24 (Visit 7) or at Week 24 (Visit 7) and Week 26 (Visit 8) as assessed by a Blinded Assessor.
Time Frame: Week 26
|
Week 26
|
Collaborators and Investigators
Sponsor
Collaborators
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Estimated)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Genetic Diseases, Inborn
- Connective Tissue Diseases
- Skin Diseases
- Congenital Abnormalities
- Skin Diseases, Genetic
- Skin Abnormalities
- Skin Diseases, Vesiculobullous
- Collagen Diseases
- Congenital, Hereditary, and Neonatal Diseases and Abnormalities
- Skin and Connective Tissue Diseases
- Epidermolysis Bullosa
- Epidermolysis Bullosa Dystrophica
Other Study ID Numbers
- CCB-EB-304
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
product manufactured in and exported from the U.S.
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