Key Insights

Highlights

Treatment Research

6 treatments under study, led by Genetically Modified Epidermolysis Bullosa Selfassembled Skin Substitute Gmebsass

Research Leaders

12 sponsors active, led by CIBER Enfermedades raras

Related Conditions

Often studied with 7 related conditions

Clinical Risk Assessment

Based on trial outcomes

High Risk

Score: 90/100

Termination Rate

28.6%

2 terminated out of 7 trials

Success Rate

0.0%

-86.6% vs benchmark

Late-Stage Pipeline

14%

1 trials in Phase 3/4

Results Transparency

0%

1 of 0 completed with results

Key Signals

1 with results0

Data Visualizations

Phase Distribution

6Total
Phase 1/2 (5)
Phase 3 (1)

Trial Status

Unknown2
Recruiting2
Terminated2
Active Not Recruiting1

Clinical Trials (7)

Showing 7 of 7 trials
NCT07193134Phase 1/2Recruiting

GMEB-SASS: A Gene-Modified Skin Substitute for RDEB Treatment

NCT04186650Phase 1/2Active Not RecruitingPrimary

Ex Vivo Gene Therapy Clinical Trial for RDEB Using Genetically Corrected Autologous Skin Equivalent Grafts

NCT06563414Recruiting

A Natural History Study of Corneal Abrasions in Patients With Dystrophic Epidermolysis Bullosa (DEB)

NCT02810951Phase 1/2TerminatedPrimary

A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa (RDEB)

NCT03605069Phase 1/2TerminatedPrimary

A Double-blind, Randomized, Intra-subject Placebo-controlled, Multicenter, Multiple Dose Study, Evaluating Safety, Proof of Mechanism, Preliminary Efficacy and Systemic Exposure in Subjects With Confirmed DDEB or RDEB Diagnosis With One or More Pathogenic Mutations in Exon 73 in the COL7A1 Gene

NCT04153630Phase 1/2UnknownPrimary

Safety Study and Preliminary Efficacy of Infusion Haploidentical Mesenchymal Stem Cells Derived From Bone Marrow for Treating Recessive Dystrophic Epidermolysis Bullosa

NCT02286427Phase 3UnknownPrimary

A Comparative Study of the Healing of Chronic Ulcers of Recessive Epidermolysis Bullosa : Dressing vs Amniotic Membrane

Showing all 7 trials

Research Network

Common Treatments

  • Genetically Modified Epidermolysis Bullosa Selfassembled Skin Substitute Gmebsass1
  • Qr3131
  • Amniotic Membrane1
  • Col7a1sin Retroviral Vector Engineered Autologous Tissueengineered Skin1
  • Mesenchymal Stem Cells Derived From Bone Marrow Bmmscs1

Active Sponsors

  • CIBER Enfermedades raras1
  • Institut National de la Santé Et de la Recherche Médicale, France1
  • Instituto de Investigación Hospital Universitario La Paz1
  • Instituto de Salud Carlos III1
  • Assistance Publique - Hôpitaux de Paris1

Top Investigators

Related Conditions

  • Epidermolysis Bullosa Dystrophica1
  • Epidermolysis Bullosa Dystrophica Dominans1
  • Epidermolysis Bullosa Dystrophica Dominant1
  • Rdeb1
  • Recessive Dystrophic Epidermolysis Bullosa1

Activity Timeline