Clinical trials: Epidermolysis Bullosa
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
Short Term Observational Study in DEB Patients
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 name
- NCT02178969
- Where
- 10 sites in United States, Australia and 5 more
- Volunteers
- 29 people
- Sponsor
- Shire
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.
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
Generalized Epidermolysis Bullosa Simplex
An International, Multicenter, Randomized, Double-Blind, Parallel Group, Vehicle-Controlled, Phase 2/3 Study With Open-Label Extension Evaluating the Efficacy and Safety of Diacerein 1% Ointment for the Treatment of Generalized Epidermolysis Bullosa Simplex (EBS)
The proposed Phase 2/3 trial with double-blind and open-label extension phases is an international, multicenter study designed to assess the efficacy and safety of diacerein 1% ointment in patients with generalized EBS.
- Trial name
- EBShield (NCT06073132)
- Testing
- AC-203, Vehicle
- Where
- 30 sites in United States, Australia and 16 more
- Volunteers
- 80 people
- Sponsor
- TWi Biotechnology, Inc.
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.
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.
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.
Epidermolysis Bullosa Simplex, Epidermolysis Bullosa, Junctional
INM-755 (Cannabinol) Cream for Treatment of Epidermolysis Bullosa
The purpose of this study is to evaluate the safety of INM-755 (cannabinol) cream and obtain preliminary evidence of efficacy in treating symptoms and healing wounds over a 28-day period in patients with epidermolysis bullosa…
- Conditions
- Epidermolysis Bullosa Simplex, Epidermolysis Bullosa, Junctional, Epidermolysis Bullosa Dystrophica, Kindler Syndrome
- Trial name
- NCT04908215
- Testing
- INM-755 (cannabinol) cream, Vehicle Cream
- Where
- 7 sites in France, Germany and 3 more
- Volunteers
- 19 people
- Sponsor
- InMed Pharmaceuticals Inc.
Dystrophic Epidermolysis Bullosa
A Pilot Study of HP802-247 in Dystrophic Epidermolysis Bullosa
This is a pilot study designed to see if HP802-247, an investigational treatment with living human skin cells, helps to heal blisters or wounds in subjects with Dystrophic Epidermolysis Bullosa (DEB).
- Trial name
- NCT01528306
- Testing
- HP802-247
- Sponsor
- Healthpoint
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.
Epidermolysis Bullosa
Survey to Identify Burdens and Unmet Needs of Patients With Epidermolysis Bullosa
This survey intends to collect information on key aspects of life with epidermolysis bullosa (EB), including diagnostic journey, treatment, management, daily living challenges, and overall psycho-social, socio-economic, academic…
- Trial name
- NCT03158662
- Testing
- Survey
- Where
- United States
- Volunteers
- 150 people
- Sponsor
- Amicus Therapeutics
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.
Epidermolysis Bullosa
Open Label Extension Study to Evaluate the Long-term Safety of Zorblisa (SD-101-6.0) in Patients With Epidermolysis Bullosa
The study aimed to assess the long-term safety of topical use of Zorblisa (SD-101-6.0) in participants with Epidermolysis Bullosa (EB).
- Trial name
- NCT02670330
- Testing
- SD-101-6.0 cream
- Where
- 30 sites in United States, Australia and 10 more
- Volunteers
- 152 people
- Sponsor
- Scioderm, Inc.
Epidermolysis Bullosa
A Study of the Efficacy and Safety of ABH001 in the Treatment of Patients With Epidermolysis Bullosa Who Have Wounds That Are Not Healing
The purpose of this study is to evaluate the efficacy and safety of ABH001 in the treatment of patients with epidermolysis bullosa who have wounds that are not healing.
- Trial name
- NCT01749306
- Testing
- ABH001, Control wound treatment
- Where
- 14 sites in United States, Austria and 6 more
- Volunteers
- 1 people
- Sponsor
- Shire Regenerative Medicine, Inc.
Epidermolysis Bullosa Simplex
A Phase II, Placebo Controlled, Clinical Trial of Topical TolaSure Targeting Aggregated Mutant Keratin in Epidermolysis Bullosa Simplex
This Phase II clinical study will assess the efficacy, safety and tolerability of topical TolaSure Gel in adults and pediatric patients (2 years of age and older) diagnosed with localized, intermediate, or severe epidermolysis…
- Trial name
- (TAMES) (NCT07027345)
- Testing
- 5% TolaSure Topical Gel
- Where
- 2 sites in United States
- Volunteers
- 40 people
- Sponsor
- BioMendics, LLC
Epidermolysis Bullosa
Allogeneic ABCB5-positive Dermal Mesenchymal Stromal Cells for Treatment of Epidermolysis Bullosa (Phase III, Cross-over)
The aim of this clinical trial is to investigate the safety and efficacy of allo-APZ2-OTS administered intravenously to subjects with recessive dystrophic epidermolysis bullosa (RDEB) compared to placebo.
- Trial name
- NCT05464381
- Testing
- allo-APZ2-OTS
- Where
- 16 sites in Argentina, Austria and 10 more
- Volunteers
- 91 people
- Sponsor
- RHEACELL GmbH & Co. KG
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
Epidermolysis Bullosa Simplex
Safety and Efficacy of Topical TolaSure Targeting Aggregated Mutant Keratin in Severe Epidermolysis Bullosa Simplex
TolaSure is a topical gel for the promotion of accelerated wound healing. This Phase I study will assess the safety, tolerability, and clinical effects of TolaSure when applied to wounded skin areas of patients diagnosed with…
- Trial name
- TAMES (NCT05062070)
- Testing
- TolaSure Topical Gel, Vehicle Topical Gel
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- BioMendics, LLC