Clinical trials: Dystrophic Epidermolysis Bullosa
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
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.
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
Epidermolysis Bullosa, Junctional, Epidermolysis Bullosa Dystrophica
Safety and Efficacy of Apligraf in Nonhealing Wounds of Subjects With Junctional or Dystrophic Epidermolysis Bullosa (EB)
The purpose of this study is to evaluate the use of Apligraf for the treatment of nonhealing wounds in subjects with dystrophic or junctional epidermolysis bullosa.
- Trial name
- NCT00587223
- Testing
- Apligraf, Standard dressing regimen
- Where
- 5 sites in United States
- Volunteers
- 1 people
- Sponsor
- Organogenesis
Dystrophic Epidermolysis Bullosa
Evaluation of D-Fi for the Treatment of Wounds Due to DEB
The purpose of this study is to determine whether administration of D-Fi in addition to standard of care improves wound healing as compared to standard of care alone (control) in children, adolescents, and adults with Dystrophic…
- Trial name
- NCT06892639
- Testing
- D-Fi
- Where
- 4 sites in United States
- Volunteers
- 32 people
- Sponsor
- Castle Creek Biosciences, LLC.
Epidermolysis Bullosa, Junctional Epidermolysis Bullosa
A Study to Learn About the Safety and Efficacy of the Drug Oleogel-S10 in Japanese Patients With Epidermolysis Bullosa
The goal of this clinical trial is to learn if Oleogel-S10 gel works to treat skin wounds from two types of inherited epidermolysis bullosa (EB): junctional EB (JEB) or dystrophic EB (DEB) in the Japanese population.
- Conditions
- Epidermolysis Bullosa, Junctional Epidermolysis Bullosa, Epidermolysis Bullosa, Dystrophic, Epidermolysis Bullosa, Junctional
- Trial name
- REVIVE (NCT06917690)
- Testing
- Oleogel-S10
- Where
- 6 sites in Japan
- Volunteers
- 6 people
- Sponsor
- Chiesi Farmaceutici S.p.A.
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
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
ESSENCE Study: Efficacy and Safety of SD-101 Cream in Participants With Epidermolysis Bullosa
The aim was to assess the efficacy and safety of SD-101-6.0 cream versus Placebo (SD-101-0.0) cream in the treatment of skin lesions in participants with Epidermolysis Bullosa.
- Trial name
- NCT02384460
- Testing
- SD-101-6.0 cream
- Where
- 38 sites in United States, Australia and 11 more
- Volunteers
- 169 people
- Sponsor
- Scioderm, 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 (EB), Epidermolysis Bullosa Simplex
To Evaluate the Pharmacokinetic of Diacerein and Rhein After Maximum Use in Patients With Epidermolysis Bullosa (EB)
A pharmacokinetic (PK) study in 16-20 EB subjects to be allocated to two cohorts. Cohort 1 to include 8-10 subjects (ages 12 yrs and older); Cohort 2 to include 8-10 subjects (ages 6 months-11 yrs, inclusive).
- Conditions
- Epidermolysis Bullosa (EB), Epidermolysis Bullosa Simplex, Dystrophic Epidermolysis Bullosa, Junctional Epidermolysis Bullosa
- Trial name
- NCT03472287
- Testing
- Diacerein 1% Ointment
- Where
- 3 sites in United States
- Volunteers
- 11 people
- Sponsor
- Castle Creek Pharmaceuticals, LLC
Blister, Wound of Skin
Safety, Tolerability, and Pharmacokinetics of Ascending Topical Doses of TCP-25 Applied to Epidermal Suction Blister Wounds, Non-Healing Leg Ulcers and Patients With Dystrophic Epidermolysis Bullosa.
This is a three-part, Phase I, first-in-human study designed to evaluate the safety, tolerability, and potential systemic exposure of multiple topical doses of TCP-25.
- Conditions
- Blister, Wound of Skin, Epidermolysis Bullosa, Varicose Ulcer of Lower Limb
- Trial name
- NCT05378997
- Where
- Sweden
- Volunteers
- 35 people, healthy volunteers welcome
- Sponsor
- Xinnate AB
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
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.
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 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
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
Epidermolysis Bullosa
Study of Effectiveness and Safety of SD-101 in Participants With Epidermolysis Bullosa
The purpose of this study was to assess whether the topical use of SD-101 cream (3% or 6%) was effective in treating wounds in participants with Simplex, Recessive Dystrophic, or Junctional non-Herlitz Epidermolysis Bullosa (EB).
- Trial name
- NCT02014376
- Testing
- SD-101 dermal cream (3%), SD-101 Dermal Cream (6%), Vehicle (SD-101 0%)
- Where
- 7 sites in United States
- Volunteers
- 48 people
- Sponsor
- Scioderm, 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.
Epidermolysis Bullosa
Open-Label Extension Study to Evaluate the Safety of SD-101 Cream in Participants With Epidermolysis Bullosa
The purpose of this study was to assess the continued safety of topical use of SD-101 cream (6%) in participants with Epidermolysis Bullosa (EB). Funding Source: FDA Office of Orphan Products Development
- Trial name
- NCT02090283
- Testing
- SD-101 dermal cream (6%)
- Where
- 7 sites in United States
- Volunteers
- 42 people
- Sponsor
- Scioderm, Inc.