Ignite Creation Date:
2024-05-06 @ 8:23 PM
Last Modification Date:
2024-10-26 @ 3:26 PM
Study NCT ID:
NCT06362447
Status:
NOT_YET_RECRUITING
Last Update Posted:
2024-04-12
First Post:
2024-04-02
Brief Title:
Efficacy of Injectable Gentamicin in Hereditary Ichthyosis
Sponsor:
University Hospital Toulouse
Organization:
University Hospital Toulouse
Study Overview
Official Title:
Phase 2 Study Evaluating the Efficacy of Injectable Gentamicin in Hereditary Ichthyosis
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-08
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
GENTIC
Brief Summary:
This study will evaluate the efficacy and safety of intravenous gentamicin in congenital ichthyosis due to a non-sens mutation The primary objective is the severity of scales and erythema at the third month compared to baseline Secondary objectives will include the importance of itching trans epidermal water loss cutaneous expression of the targeted protein the security of the drug and patients satisfaction
Detailed Description:
Congenital ichthyoses represent a group of diseases characterized by disabling cutaneous anomalies scales and inconstant erythema often associated with extra cutaneous anomalies that may be severe The treatment is non curative and symptomatic including local treatments ie emollients Oral retinoids may be helpful in moderate to severe forms There is a huge need for novel therapies ideally targeting the molecular defect Gentamicin may be a novel therapeutic option for congenital ichthyosis
Apart its antimicrobial effect gentamicin can achieve stop codon readthrough and produce full-length protein
In this study gentamicin 10 mgkg will be administrated once weekly for 3 months The study will include monthly visits a follow-up visit 3 months after the stopping the drug and an end-of-study visit 3 months after the follow-up visit Kidney and hearing functions will be assessed regularly
Apart its antimicrobial effect gentamicin can achieve stop codon readthrough and produce full-length protein
In this study gentamicin 10 mgkg will be administrated once weekly for 3 months The study will include monthly visits a follow-up visit 3 months after the stopping the drug and an end-of-study visit 3 months after the follow-up visit Kidney and hearing functions will be assessed regularly
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None