Ignite Creation Date:
2024-05-06 @ 8:03 PM
Last Modification Date:
2024-10-26 @ 3:19 PM
Study NCT ID:
NCT06238908
Status:
NOT_YET_RECRUITING
Last Update Posted:
2024-02-02
First Post:
2024-01-26
Brief Title:
Safety and Efficacy Study of NGGT003 in Hemophilia A Patients
Sponsor:
Institute of Hematology Blood Diseases Hospital China
Organization:
Institute of Hematology Blood Diseases Hospital China
Study Overview
Official Title:
Clinical Study on the Safety and Efficacy of an Intravenous Infusion of NGGT003 in the Treatment of Hemophilia A
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-01
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:
None
Brief Summary:
This is an early phase 1 open-label single-center dose-escalation pilot trial to evaluate the safety and efficacy of an intravenous infusion of NGGT003 in hemophilia A patients NGGT003 uses adeno-associated virus AAV as a vector carrying a liver specific promoter and codon optimized human FVIII gene B domain deletion mutant hFVIII BDD and expresses human FVIII protein in the liver through intravenous injection
Detailed Description:
Hemophilia A HA is an X-linked recessive genetic disease caused by mutations in the FVIII gene on the X chromosome leading to abnormal coagulation function In the male population the incidence rate of hemophilia A was about 15000 and female patients with hemophilia A were extremely rare Type A hemophilia patients mainly exhibit a tendency for bleeding with a wide range of bleeding sites and frequent recurrence which can form hematoma and joint deformation This is an early phase 1 open-label single-center dose-escalation pilot trial to evaluate the safety and efficacy of a single intravenous infusion of NGGT003 in hemophilia A patients 4-6 subjects will be enrolled and divided into 3 groups according to the principle of dose escalation respectively administered intravenous infusion of NGGT003 at low dose 4e11vgkg medium dose 1e12vgkg and high dose 25e12vgkg All subjects will undergo 52 weeks of treatment observation and further 260 weeks of long-term follow-up
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