Ignite Creation Date:
2024-05-06 @ 8:25 PM
Last Modification Date:
2024-10-26 @ 3:27 PM
Study NCT ID:
NCT06379789
Status:
RECRUITING
Last Update Posted:
2024-04-23
First Post:
2024-04-18
Brief Title:
A Trial to Learn if REGV131-LNP1265 is Safe and Works to Help the Body Make Clotting Factor in Pediatric Adolescent and Adult Patients With Hemophilia B
Sponsor:
Regeneron Pharmaceuticals
Organization:
Regeneron Pharmaceuticals
Study Overview
Official Title:
A Two-Part Open-Label Study of REGV131-LNP1265 A CRISPRCas9 Based Coagulation Factor IX Gene Insertion Therapy in Participants With Hemophilia B
Status:
RECRUITING
Status Verified Date:
2024-09
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:
BEYOND-9
Brief Summary:
Participants in this study have a genetic mutation specifically in the coagulation blood clotting Factor 9 gene that causes severe or moderately severe hemophilia B This study is researching an experimental gene insertion therapy the adding of a gene into your DNA called REGV131-LNP1265 also called the study drug Gene insertion therapy aims to teach the body how to produce clotting factor long-term without the need for factor replacement therapy
The main aim of this study is to find a safe and well-tolerated dose of the study drug by checking the side effects that may happen from taking it
The study is looking at several other research questions including
How much study drug is in the blood at different times
Whether the body makes antibodies against parts of the study drug which could make the drug less effective or could lead to side effects Antibodies are proteins produced by the bodys immune system in response to a foreign substance
Whether the body makes antibodies against the clotting factor replacement therapy
How quality of life is affected by hemophilia B and if it changes after taking study drug
How joint health is affected by hemophilia B and if it changes after taking study drug
How often visits are required for the emergency room urgent care center physicians office hospital telephone or online are required as a result of bleeding events and if the frequency changes after taking study drug
How often factor replacement therapy is needed both on a regular basis for prevention of bleeding and as needed to treat bleeding events and it if changes after taking study drug
Whether there is a difference in 2 different methods for measuring Factor 9 activity in the blood
The main aim of this study is to find a safe and well-tolerated dose of the study drug by checking the side effects that may happen from taking it
The study is looking at several other research questions including
How much study drug is in the blood at different times
Whether the body makes antibodies against parts of the study drug which could make the drug less effective or could lead to side effects Antibodies are proteins produced by the bodys immune system in response to a foreign substance
Whether the body makes antibodies against the clotting factor replacement therapy
How quality of life is affected by hemophilia B and if it changes after taking study drug
How joint health is affected by hemophilia B and if it changes after taking study drug
How often visits are required for the emergency room urgent care center physicians office hospital telephone or online are required as a result of bleeding events and if the frequency changes after taking study drug
How often factor replacement therapy is needed both on a regular basis for prevention of bleeding and as needed to treat bleeding events and it if changes after taking study drug
Whether there is a difference in 2 different methods for measuring Factor 9 activity in the blood
Detailed Description:
The study will be conducted with a 2-part adaptive design with enrollment of patients into sequential parts of the study
Part 1 Dose Escalation and Dose Confirmation in adult patients 18 years of age
Dose Escalation Cohorts to determine the recommended dose for expansion RDE of REGV131-LNP1265
Dose Confirmation Cohort to gain further confidence in safety tolerability and Coagulation Factor IX FIX functional activity data at the RDE
Part 2 Dose Expansion at the RDE
Part 2A Adult patients 18 years of age RDE of REGV131-LNP1265 as determined in Part 1
Part 2B Adolescent patients 18 and 12 years of age will be administered weight-adjusted RDE
Part 2C Adolescent and Pediatric patients 2 to 12 years may be enrolled in an age staggered sequential manner first participants aged 6 to 12 years and then participants 2 to 6 years of age and will receive a weight-adjusted RDE
Part 1 Dose Escalation and Dose Confirmation in adult patients 18 years of age
Dose Escalation Cohorts to determine the recommended dose for expansion RDE of REGV131-LNP1265
Dose Confirmation Cohort to gain further confidence in safety tolerability and Coagulation Factor IX FIX functional activity data at the RDE
Part 2 Dose Expansion at the RDE
Part 2A Adult patients 18 years of age RDE of REGV131-LNP1265 as determined in Part 1
Part 2B Adolescent patients 18 and 12 years of age will be administered weight-adjusted RDE
Part 2C Adolescent and Pediatric patients 2 to 12 years may be enrolled in an age staggered sequential manner first participants aged 6 to 12 years and then participants 2 to 6 years of age and will receive a weight-adjusted RDE
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 |
|---|---|---|---|
| 2023-507260-40-00 | REGISTRY | EU CTR Number | None |