Rare Diseases Clinical Trial
Official title:
Evaluating the Therapeutic Efficacy of Filgrastim in Severe Bullous Drug Eruptions (Lyell and Stevens-Johnson Syndromes)
Toxic epidermal necrolysis (TEN) including Stevens Johnson (SJS) and Lyell syndromes represent the most severe drug eruptions. It is an allergic disorder caused by cytotoxic T lymphocytes, specific of drugs, responsible for the destruction of keratinocytes by apoptosis. Regulatory T cell (CD25 high CD4+), normally responsible for controlling the activation of cytotoxic T lymphocytes, have altered function. Despite the progress made in the pathophysiological understanding of TEN, there is currently no effective treatment. The main symptom is bullous and skin peeling > 10% giving the appearance of great burns. The death rate is estimated between 30 and 40% due to visceral inflammatory injuries and bacterial superinfection. The risk of mortality is estimated during the initial treatment by calculating the SCORTEN (mortality>10% if SCORTEN>2, mortality>90% if SCORTEN>5). The morbidity is also very important (92% at 1 year), especially ophthalmologic with high risk of blindness... The therapeutic potential of G-CSF (Granulocyte-Colony Stimulating Factor) in TEN is supported by several observations. The G-CSF promotes skin healing. This has been shown in human burns, with a significant reduction in healing time under G-CSF. The mechanisms associate the growth factor effect on keratinocytes, macrophages stimulation and metalloprotease activity allowing tissue remodeling limiting sequels onset. Otherwise, healing altered in deficient G-CSF mice is corrected by the growth factor injection. The G-CSF is an immunomodulator whose activities appear to justify use in TEN : - Polarization of immune response to Th2 non-cytotoxic (anti Th1), - Preferential differentiation of naive LT (T lymphocytes) in regulator LT (CD25 high CD4+) and mobilization of regulator LT of the spinal cord to altered tissues. The G-CSF was used in a few cases of TEN with great efficacy. No data is available concerning sequels of SJS/TEN in treated patients. This clinical trial program, by providing proof of the efficacy of filgrastim in SJS/TEN, should allow progress in care of this serious toxics diseases. In the future, it could thus reduce the significant morbidity of these syndromes with a high rate of sequelae.
n/a
Status | Clinical Trial | Phase | |
---|---|---|---|
Recruiting |
NCT05794217 -
A Multi-Site Leukopak Repository Providing Annotated Biospecimens for Approved Investigator-Directed Biomedical Research Initiatives
|
||
Completed |
NCT03680365 -
Your Voice; Impact of Duchenne Muscular Dystrophy (DMD) on the Lives of Families
|
||
Completed |
NCT03290469 -
NICUSeq: A Trial to Evaluate the Clinical Utility of Human Whole Genome Sequencing (WGS) Compared to Standard of Care in Acute Care Neonates and Infants
|
N/A | |
Not yet recruiting |
NCT05955794 -
Vocal Pattern Assessment as a New Key to Identifying Rare Syndromes
|
N/A | |
Recruiting |
NCT04429750 -
Intact Cord Resuscitation in CDH
|
N/A | |
Not yet recruiting |
NCT04152876 -
Functional Genomics of Rare Genetic Diseases: Realization of Innovative Tools With High Diagnostic Power
|
||
Recruiting |
NCT03683966 -
MigALastat Therapy Adherence Among FABRY Patients: A Prospective Multicentral Observational Study
|
||
Not yet recruiting |
NCT04319796 -
European Registry on Rare Neurological Diseases
|
||
Completed |
NCT02736565 -
Pbi-shRNAâ„¢ EWS/FLI1 Type 1 LPX in Subjects With Advanced Ewing's Sarcoma
|
Phase 1 | |
Completed |
NCT05070988 -
Oral Health Related Quality of Life of Patients With Rare Diseases: a Qualitative Approach
|
||
Completed |
NCT03563677 -
Dual Guidance Structure for Evaluation of Patients With Unclear Diagnosis in Centers for Rare Diseases
|
N/A | |
Recruiting |
NCT05499091 -
Functional Study to Indentify Genetic Etiology of Rare Diseases - ORIGIN
|
N/A | |
Completed |
NCT03954652 -
Whole Genome Trio Sequencing as a Standard Routine Test in Patients With Rare Diseases - "GENOME FIRST APPROACH"
|
N/A | |
Recruiting |
NCT05703893 -
Investigation of the Neurovegetative Pattern in Patients With Thoracic Aortic Aneurysms (TAA)
|
||
Enrolling by invitation |
NCT04703179 -
Rare and Undiagnosed Disease Research Biorepository
|
||
Recruiting |
NCT04024774 -
Diagnostic Research in Patients With Rare Diseases -Solving the Unsolved Rare Diseases
|
||
Recruiting |
NCT06343558 -
Gait and Balance Impairment in Rare and Very Rare Neurological Diseases
|
||
Not yet recruiting |
NCT06412718 -
Validation of Human Drugs Target of Repurposed Drugs and Novel Therapies
|
||
Enrolling by invitation |
NCT04654000 -
Rheopheresis as Adjuvant Treatment of Calciphylaxis
|
N/A | |
Recruiting |
NCT03491280 -
Diagnostic Research in Patients With Rare Diseases - Solving the Unsolved Rare Diseases
|