Ignite Creation Date:
2024-10-26 @ 3:37 PM
Last Modification Date:
2024-10-26 @ 3:37 PM
Study NCT ID:
NCT06555419
Status:
NOT_YET_RECRUITING
Last Update Posted:
None
First Post:
2024-08-09
Brief Title:
A Study to Find Out How Nusinersen is Processed in the Body When Given Through the ThecaFlex DRx System in Adult and Pediatric Participants With Spinal Muscular Atrophy PIERRE-PK
Sponsor:
None
Organization:
None
Study Overview
Official Title:
An Open Label Single Cohort Study to Assess the Pharmacokinetic Profile of Nusinersen BIIB058 Administered Via the ThecaFlex DRx System PIERREPK
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-08
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:
PIERRE-PK
Brief Summary:
In this PIERRE-PK study researchers will learn how the body processes nusinersen when it is given through the ThecaFlex DRx System compared to when nusinersen is given by lumbar puncture LP The ThecaFlex DRx system is an investigational implantable medical device developed by Alcyone Therapeutics Inc It consists of a catheter which is a flexible tube connected to a port which is placed under the skin Alcyone Therapeutics Inc has an ongoing study called PIERRE to test the ThecaFlex DRx system Participants with spinal muscular atrophy SMA in the PIERRE study may be enrolled in the PIERRE-PK study
The main objective of the PIERRE-PK study is to learn how the body processes nusinersen when given by the ThecaFlex DRx system compared to a lumbar puncture The main questions researchers want to answer are
What is the highest amount of nusinersen found in the blood after dosing
How much nusinersen is found in the blood over the first 24 hours after dosing
The PIERRE-PK study will be done as follows
Participants will be screened to check if they can join the study The screening period will be up to 30 days for this study and may overlap with the PIERRE study
Participants will receive a dose of nusinersen by lumbar puncture
The ThecaFlex DRx system will be implanted after the lumbar puncture as part of the PIERRE study
Participants will receive a dose of nusinersen by the ThecaFlex DRx system as part of the PIERRE study
Researchers will take blood samples before and after each dose The last blood sample will be taken 24 hours after the dose
The total study duration for each participant in the PIERRE-PK study will be up to 5 months This period will overlap with the participants first 5 months in the PIERRE study
The main objective of the PIERRE-PK study is to learn how the body processes nusinersen when given by the ThecaFlex DRx system compared to a lumbar puncture The main questions researchers want to answer are
What is the highest amount of nusinersen found in the blood after dosing
How much nusinersen is found in the blood over the first 24 hours after dosing
The PIERRE-PK study will be done as follows
Participants will be screened to check if they can join the study The screening period will be up to 30 days for this study and may overlap with the PIERRE study
Participants will receive a dose of nusinersen by lumbar puncture
The ThecaFlex DRx system will be implanted after the lumbar puncture as part of the PIERRE study
Participants will receive a dose of nusinersen by the ThecaFlex DRx system as part of the PIERRE study
Researchers will take blood samples before and after each dose The last blood sample will be taken 24 hours after the dose
The total study duration for each participant in the PIERRE-PK study will be up to 5 months This period will overlap with the participants first 5 months in the PIERRE study
Detailed Description:
None
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