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)
- Conditions
- Spinal Muscular Atrophy
- Interventions
- Device: ThecaFlex DRx System
- Registration Number
- 2024-514239-21-00
- Lead Sponsor
- Biogen Idec Research Limited
- Brief Summary
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?
- Detailed Description
The primary objective of this study is to assess the Pharmacokinetic (PK) profile of nusinersen delivered via standard LP and via the ThecaFlex DRx System in participants with SMA.
Recruitment & Eligibility
- Status
- Authorised, recruiting
- Sex
- Not specified
- Target Recruitment
- 26
Participant is on regular maintenance dosing of nusinersen (12 milligrams [mg] dose) every 4 months, with 4 months (± 2 weeks) between the LP-delivered study dose and the last nusinersen dose prior to study enrollment.
Participants must be enrolled in the PIERRE study to be eligible for enrolment in the PIERRE PK study.
Ongoing participation or participation within 6 months or 5 half-lives of the agent (whichever is longer) of enrolment in other interventional clinical trials for the treatment of SMA (except for the PIERRE study).
Participant is naïve to nusinersen treatment.
Participant is receiving nusinersen at a dose other than 12 mg.
Participant has already undergone implantation of the ThecaFlex DRx system.
Participant is pregnant, currently breastfeeding, or intending to become pregnant during the study. NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Nusinersen Via LP and ThecaFlex DRx System Nusinersen Participants will receive a maintenance dose of nusinersen, 12 milligrams (mg) via LP in the PIERRE-PK study, followed by implantation of the ThecaFlex DRx System and the subsequent nusinersen 12 mg maintenance dose via the system in the PIERRE study. Nusinersen Via LP and ThecaFlex DRx System ThecaFlex DRx System Participants will receive a maintenance dose of nusinersen, 12 milligrams (mg) via LP in the PIERRE-PK study, followed by implantation of the ThecaFlex DRx System and the subsequent nusinersen 12 mg maintenance dose via the system in the PIERRE study.
- Primary Outcome Measures
Name Time Method Maximum Observed Concentration (Cmax) of Nusinersen Delivered via Standard LP and via ThecaFlex DRx System Maximum Observed Concentration (Cmax) of Nusinersen Delivered via Standard LP and via ThecaFlex DRx System
Area Under the Plasma Concentration-Time Curve From Zero Time to 24 Hours After Intrathecal Administration (AUC0-24h) of Nusinersen Delivered via Standard LP and via ThecaFlex DRx System Area Under the Plasma Concentration-Time Curve From Zero Time to 24 Hours After Intrathecal Administration (AUC0-24h) of Nusinersen Delivered via Standard LP and via ThecaFlex DRx System
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (9)
Centre Hospitalier Regional De Marseille
🇫🇷Marseille, France
Assistance Publique Hopitaux De Paris
🇫🇷Garches, France
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
🇮🇹Rome, Italy
Centro Clinico Nemo
🇮🇹Milan, Italy
Hospital Universitario Y Politecnico La Fe
🇪🇸Valencia, Spain
Hospital Universitario La Paz
🇪🇸Madrid, Spain
Szpital Specjalistyczny Im. Ludwika Rydygiera W Krakowie Sp. z o.o.
🇵🇱Cracow, Poland
Instytut Centrum Zdrowia Matki Polki
🇵🇱Lodz, Poland
Universitaetsklinikum Essen AöR
🇩🇪Essen, Germany
Centre Hospitalier Regional De Marseille🇫🇷Marseille, FranceCécile HalbertSite contact+33491385605cecile.halbert@ap-hm.fr