Ignite Creation Date:
2024-05-06 @ 11:48 AM
Last Modification Date:
2024-10-26 @ 12:50 PM
Study NCT ID:
NCT03605069
Status:
TERMINATED
Last Update Posted:
2021-08-25
First Post:
2018-06-25
Brief Title:
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
Sponsor:
Phoenicis Therapeutics
Organization:
Phoenicis Therapeutics
Study Overview
Official Title:
A First in Human Double-blind Randomized Intra-subject Placebo-controlled Multiple Dose Study of QR-313 Evaluating Safety Proof of Mechanism Preliminary Efficacy and Systemic Exposure in Subjects With DDEB or RDEB Due to Mutations in Exon 73 of the COL7A1 Gene
Status:
TERMINATED
Status Verified Date:
2021-08
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Low enrollment
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
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
Detailed Description:
This clinical trial will evaluate the safety and tolerability proof of mechanism systemic exposure and preliminary efficacy following topical application of QR-313 to subjects with confirmed DDEB or RDEB with one or more pathogenic mutations in exon 73 in the COL7A1 gene
Up to two Target Wound Areas TWAs per subject will be selected and randomized Each TWA will be treated with IMP for 8 weeks either QR-313 or matching placebo All subjects will continue to be followed up for 8 weeks post last dose
Subjects will be monitored through home visits and site visits An imaging system will be used to assess the target wound at all home and study site visits
QR-313 is a 21-nucleotide antisense oligonucleotide AON designed to hybridize to a specific sequence in the COL7A1 pre-messengerRNA pre-mRNA
Up to two Target Wound Areas TWAs per subject will be selected and randomized Each TWA will be treated with IMP for 8 weeks either QR-313 or matching placebo All subjects will continue to be followed up for 8 weeks post last dose
Subjects will be monitored through home visits and site visits An imaging system will be used to assess the target wound at all home and study site visits
QR-313 is a 21-nucleotide antisense oligonucleotide AON designed to hybridize to a specific sequence in the COL7A1 pre-messengerRNA pre-mRNA
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
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
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 2017-004806-17 | EUDRACT_NUMBER | None | None |