Ignite Creation Date:
2024-10-26 @ 3:35 PM
Last Modification Date:
2024-10-26 @ 3:35 PM
Study NCT ID:
NCT06519552
Status:
RECRUITING
Last Update Posted:
None
First Post:
2024-06-23
Brief Title:
A Clinical Study Evaluating the Safety Tolerability and Initial Efficacy of JWK008 in Patients With Mucopolysaccharidosis Type I
Sponsor:
None
Organization:
None
Study Overview
Official Title:
A Clinical Study Evaluating the Safety Tolerability and Initial Efficacy of JWK008 Given by a Single Intravenous Infusion in Patients With Mucopolysaccharidosis Type I
Status:
RECRUITING
Status Verified Date:
2024-07
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This study is a single-center single-arm non-randomized open-label non controlled dose-escalation prospective clinical trial designed to assess the safety tolerability and preliminary efficacy of JWK008 injection in patients with MPS I
Detailed Description:
MPS I is a rare autosomal recessive disease caused by deficiency of the α-L-iduronidase IDUA gene which encodes a lysosomal enzyme required for degradation of glycosaminoglycans GAGsWhile currently available therapies enzyme replacement therapy ERT and hematopoietic stem cell transplantation HSCT provide clinical benefit over untreated disease progressionthey still have significant limitations ERT does not cross the blood-brain barrier and therefore does not treat the central nervous system CNS effects of the disease And HSCT although it can prevent cognitive decline in patients has a high mortality rate and morbidity The investigators have designed a novel IDUA fusion protein with the ability to cross the blood-brain barrier through the addition of the brain-targeting peptide Mtfp On this basis the investigators constructed an IDUA gene expression cassette for liver-targeted expression and used a highly efficient liver-specific promoter to make the IDUA gene specifically and efficiently expressed in liver tissue and the expressed protein can enter the central nervous system to exert therapeutic effects
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None