Ignite Creation Date:
2024-10-26 @ 3:41 PM
Last Modification Date:
2024-10-26 @ 3:41 PM
Study NCT ID:
NCT06611436
Status:
RECRUITING
Last Update Posted:
None
First Post:
2024-09-18
Brief Title:
BeCoMe-9 a Clinical Study of BE-101 for the Treatment of Adults with Moderately Severe or Severe Hemophilia B
Sponsor:
None
Organization:
None
Study Overview
Official Title:
BeCoMe-9 a Phase 12 Dose Escalation and Expansion Study of BE-101 for the Treatment of Adults with Moderately Severe or Severe Hemophilia B
Status:
RECRUITING
Status Verified Date:
2024-10
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:
The BeCoMe-9 Study BE-101-01 is a Phase 12 first in human multi-center open-label dose-escalation study to evaluate the safety and clinical activity of a single intravenous IV dose of BE-101 in adults with moderately severe or severe Hemophilia B Once infused BE-101 is designed to engraft and continuously secrete FIX into the circulation to restore clinically meaningful levels of active FIX BE-101 is an autologous persons own cells B Cell Medicine BCM which uses CRISPRCas9 gene editing to precisely insert human FIX gene into those cells
Detailed Description:
The study includes 2 distinct parts Part 1 and Part 2 In Part 1 an ascending-dose design will be utilized to enable evaluation of increasing doses in a stepwise manner The objective for this dose escalation is to identify the dose of BE-101 required to achieve desired FIX activity 28 days after infusion Upon identification of a safe and efficacious dose in Part 1 an expansion phase Part 2 will initiate The initial cohort in the Part 2 expansion Part 2a phase will include up to 6 adult participants to further characterize the safety and activity of BE-101 at the selected dose Additional cohorts for adolescents and redosing for participants in Part 1 of the study will occur following data availability of Part 1
Up to 24 participants will be enrolled across Part 1 up to 18 and Part 2a up to 6 Consented participants will complete a screening period to assess eligibility and upon enrollment will undergo leukapheresis collection to support BE-101 manufacturing Following administration participants will be monitored for safety and clinical activity The total duration of study participation is approximately 52 weeks post IV administration of BE-101
Up to 24 participants will be enrolled across Part 1 up to 18 and Part 2a up to 6 Consented participants will complete a screening period to assess eligibility and upon enrollment will undergo leukapheresis collection to support BE-101 manufacturing Following administration participants will be monitored for safety and clinical activity The total duration of study participation is approximately 52 weeks post IV administration of BE-101
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