Clinical trials: RDEB
Epidermolysis Bullosa (EB), Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Phase 4 Master Protocol for Patients Prescribed Prademagene Zamikeracel for the Treatment of Wounds
The Master Protocol includes 3 studies (Study A, Study B, and Study C) that will evaluate pz-cel and related processes in the post-marketing setting.
- Conditions
- Epidermolysis Bullosa (EB), Recessive Dystrophic Epidermolysis Bullosa (RDEB), Dystrophic Epidermolysis Bullosa (DEB)
- Trial name
- NCT07717736
- Testing
- Non-conforming pz-cel surgical application to RDEB wounds, Additional biopsies, Pz-cel surgical application to DEB wounds
- Volunteers
- 100 people
- Sponsor
- Abeona Therapeutics, Inc
Epidermolysis Bullosa Dystrophica, Recessive
A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa (RDEB)
The purpose of this study is to evaluate the safety of FCX-007, evaluate Type VII collagen (COL7) expression and the presence of anchoring fibrils and to analyze wound healing as a result of FCX-007 administration in subjects…
- Trial name
- NCT02810951
- Testing
- FCX-007
- Where
- 2 sites in United States
- Volunteers
- 6 people
- Sponsor
- Castle Creek Biosciences, LLC.
Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa
A Phase 3b Study for the Treatment of Dystrophic Epidermolysis Bullosa (DEB) in New and Previously EB-101 Treated Patients
To evaluate and further characterize the safety of EB-101 (LZRSE-Col7A1 gene-corrected keratinocyte sheets with type VII collagen [C7] expression) for the treatment of large, chronic DEB wounds in new and previously EB-101…
- Conditions
- Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa, RDEB
- Trial name
- NCT05725018
- Testing
- EB-101 Surgical application of RDEB wounds
- Where
- 2 sites in United States
- Volunteers
- 12 people
- Sponsor
- Abeona Therapeutics, Inc
RDEB
A Long-Term Extension Study for Participants Previously Treated With EB-101 for the Treatment of RDEB
A Long-Term Extension Study for Participants Previously Treated with EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)
- Trial name
- NCT05708677
- Testing
- EB-101
- Where
- 2 sites in United States
- Volunteers
- 22 people
- Sponsor
- Abeona Therapeutics, Inc
Recessive Dystrophic Epidermolysis Bullosa
A Long-Term Safety Follow-up Registry for Patients Who Received Treatment With Prademagene Zamikeracel for Recessive Dystrophic Epidermolysis Bullosa (RDEB) in the Post-Marketing Setting
The goal of this observational study is to evaluate the long-term safety and wound healing durability (up to 15 years) for participants with Recessive Dystrophic Epidermolysis Bullosa (RDEB) who were treated with pz-cel.
- Trial name
- NCT07700966
- Testing
- Prademagene Zamikeracel
- Where
- 4 sites in United States
- Volunteers
- 250 people
- Sponsor
- Abeona Therapeutics, Inc
Epidermolysis Bullosa Dystrophica, Epidermolysis Bullosa
Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa
This trial will create a skin graft, which the investigators call "LEAES," using the patient's own skin cells that have been genetically engineered in the lab to express a missing protein called type VII collagen.
- Trial name
- NCT01263379
- Testing
- LZRSE-Col7A1 Engineered Autologous Epidermal Sheets
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Abeona Therapeutics, Inc
Dystrophic Epidermolysis Bullosa
A Phase I/II Study of KB103, a Topical HSV1-COL7, on DEB Patients
This study was conducted to assess the safety and efficacy of topical Beremagene Geperpavec (KB103, HSV1-COL7) on DEB patients.
- Trial name
- GEM-1 (NCT03536143)
- Testing
- Topical beremagene geperpavec
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Krystal Biotech, Inc.
Recessive Dystrophic Epidermolysis Bullosa
A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa
The purpose of this study is to determine whether administration of FCX-007 in addition to standard of care improves wound healing as compared to standard of care alone (control) in children, adolescents, and adults with…
- Trial name
- DEFI-RDEB (NCT04213261)
- Testing
- FCX-007 (dabocemagene autoficel; see below for FCX-007 description)
- Where
- 5 sites in United States
- Volunteers
- 6 people
- Sponsor
- Castle Creek Biosciences, LLC.
Recessive Dystrophic Epidermolysis Bullosa
Clinical Trial to Assess Safety and Efficacy of Autologous Cultured Epidermal Grafts Containing Epidermal Stem Cells Genetically Modified in Patients With RDEB.
Prospective open-label, uncontrolled clinical study to assess the safety and efficacy of autologous cultured epidermal grafts containing epidermal stem cells genetically modified with the aid of a gamma-retroviral vector carrying…
- Trial name
- HOLOGENE7 (NCT02984085)
- Testing
- Genetically corrected cultured epidermal autograft (ATMP)
- Where
- Austria
- Volunteers
- 3 people
- Sponsor
- Holostem s.r.l.
Dystrophic Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa
Long-Term Follow-up Protocol
The main objective of this prospective, observational, long-term follow-up (LTFU) study is to evaluate the long-term safety profile of the gene therapy products evaluated by Krystal Biotech, Inc.
- Conditions
- Dystrophic Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa, Dominant Dystrophic Epidermolysis Bullosa
- Trial name
- NCT04917887
- Where
- 3 sites in United States
- Volunteers
- 50 people
- Sponsor
- Krystal Biotech, Inc.
Dystrophic Epidermolysis Bullosa, DEB - Dystrophic Epidermolysis Bullosa
A Study Comparing KB803 and Matched Placebo in Patients With Dystrophic Epidermolysis Bullosa
KB803-EYE-01 is a Phase 3 double-blind, randomized, placebo-controlled, crossover study to evaluate the safety and efficacy of KB803 versus matched placebo in pediatric and adult subjects with recurrent corneal abrasions due to…
- Conditions
- Dystrophic Epidermolysis Bullosa, DEB - Dystrophic Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa, Dominant Dystrophic Epidermolysis Bullosa
- Trial name
- IOLITE (NCT07016750)
- Testing
- KB803
- Where
- 5 sites in United States
- Volunteers
- 16 people
- Sponsor
- Krystal Biotech, Inc.
Dystrophic Epidermolysis Bullosa
MSC EVs in Dystrophic Epidermolysis Bullosa
INVESTIGATIONAL PRODUCT: AGLE-102 is an allogeneic extracellular vesicle (EV) product derived from normal donor mesenchymal stem cells (MSCs).
- Trial name
- NCT04173650
- Testing
- AGLE-102
- Where
- 3 sites in United States
- Volunteers
- 8 people
- Sponsor
- Aegle Therapeutics
Recessive Dystrophic Epidermolysis Bullosa
A Phase 1/2 Trial of PTR-01 in Adult Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Protocol PTR-01-001 is a Phase 1/2 study of PTR-01. The study is divided into an up to 4-week Screening Period, a 10-week Treatment Period and an 8-week Follow-up Period.
- Trial name
- PTR-01-001 (NCT03752905)
- Testing
- PTR-01, Normal saline
- Where
- 3 sites in United States
- Volunteers
- 12 people
- Sponsor
- Phoenix Tissue Repair, Inc.
Dystrophic Epidermolysis Bullosa
Study of PTW-002 in Patients With Dominant or Recessive Dystrophic Epidermolysis Bullosa Due to Mutation(s) in Exon 73 of the COL7A1 Gene
A double-blind, randomized, intra-patient placebo- controlled, multiple dose study of PTW-002 evaluating safety, proof of mechanism, preliminary efficacy, and systemic exposure in patients with Dominant Dystrophic Epidermolysis…
- Trial name
- NCT05529134
- Testing
- PTW-002 10 mg/g gel
- Where
- 3 sites in United States
- Volunteers
- 8 people
- Sponsor
- Phoenicis Therapeutics
Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa
Phase 3, Open-label Clinical Trial of EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)
The purpose of this trial is to evaluate safety and efficacy of surgical application of EB-101 (autologous, gene-corrected keratinocyte sheets) as a treatment of recessive dystrophic epidermolysis bullosa (RDEB).
- Trial name
- NCT04227106
- Testing
- EB-101
- Where
- 2 sites in United States
- Volunteers
- 11 people
- Sponsor
- Abeona Therapeutics, Inc
Dystrophic Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa
Ph 3 Efficacy and Safety of B-VEC for the Treatment of DEB
To determine whether administration of topical B-VEC improves wound healing as compared to placebo, and to evaluate durability, repeat dosing (Primary Endpoint) and further obtain safety and tolerability data.
- Conditions
- Dystrophic Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa, Dominant Dystrophic Epidermolysis Bullosa
- Trial name
- GEM-3 (NCT04491604)
- Testing
- Topical Beremagene Geperpavec
- Where
- 3 sites in United States
- Volunteers
- 31 people
- Sponsor
- Krystal Biotech, Inc.
Recessive Dystrophic Epidermolysis Bullosa
A Study of PTR-01 in Recessive Dystrophic Epidermolysis Bullosa
Protocol PTR-01-002 is a 3-part Phase 2, open-label study of PTR-01. While new patients will be enrolled, priority will be given to patients that satisfactorily completed study PTR-01-001.
- Trial name
- NCT04599881
- Testing
- PTR-01
- Where
- 2 sites in United States
- Volunteers
- 6 people
- Sponsor
- Phoenix Tissue Repair, Inc.
Recessive Dystrophic Epidermolysis Bullosa
Extension Study to PTR-01-002 (A Study in Recessive Dystrophic Epidermolysis Bullosa (RDEB) Patients Previously Treated With PTR-01)
A sub-set of patients who participated in PTR-01-002 will be enrolled in an open-label study, if they meet the study eligibility criteria.
- Trial name
- NCT05143190
- Testing
- PTR-01
- Where
- 2 sites in United States
- Volunteers
- 2 people
- Sponsor
- Phoenix Tissue Repair, Inc.
Recessive Dystrophic Epidermolysis Bullosa
Allogeneic ABCB5-positive Stem Cells for Treatment of Epidermolysis Bullosa
The aim of this clinical trial is to investigate the efficacy (by monitoring overall improvement of EB symptoms) and safety (by monitoring adverse events) of three doses of allo-APZ2-EB administered intravenously to patients with…
- Trial name
- NCT03529877
- Testing
- allo-APZ2-EB
- Where
- 6 sites in United States, Austria and 3 more
- Volunteers
- 16 people
- Sponsor
- RHEACELL GmbH & Co. KG
Dystrophic Epidermolysis Bullosa, DEB - Dystrophic Epidermolysis Bullosa
A Long-term Treatment With B-VEC for Dystrophic Epidermolysis Bullosa
This is a 112-week (approximately two-year) open-label extension study of Beremagene Geperpavec (B-VEC), for participants aged 2 months and older, who have been diagnosed with Dystrophic Epidermolysis Bullosa (DEB).
- Conditions
- Dystrophic Epidermolysis Bullosa, DEB - Dystrophic Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa, Dominant Dystrophic Epidermolysis Bullosa
- Trial name
- NCT04917874
- Testing
- Open Label Topical Beremagene Geperpavec (B-VEC)
- Where
- 6 sites in United States
- Volunteers
- 47 people
- Sponsor
- Krystal Biotech, Inc.