At a glance
- What the study gets you
- Access to the study treatment being tested
- Type of study
- Interventional (receives a drug or procedure)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- Nusinersen (drug), ThecaFlex DRx System (device)
- How long the study runs
- Study runs about 32 months (dates as stated)
- About the drug or intervention
- Nusinersen — drug: Administered as specified in the treatment arm. · ThecaFlex DRx System — device: Implanted as specified in the treatment arm.
- Patient visit burden
- Not specified by the sponsor
- Type of study
- Testing a treatment
- Ages
- 3 Years and over
- Who
- All
- Number of participants
- 78
- Started
- 2025-01-16
- Last checked
- 2026-09
Plain English Summary
What is this study?
- • Testing a new treatment for muscular atrophy, spinal
- • Phase1 - 78 participants
- • 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)
Who can take part?
- • Ages 3 Years and over
- • Diagnosed with muscular atrophy, spinal
Where?
- • Sheffield - Royal Hallamshire Hospital Neurology Department
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
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 goal 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 join 1 of 2 groups based on their ongoing nusinersen treatment. One group will be receiving 12 mg doses of nusinersen. The other group will be receiving 28 mg doses of nusinersen. * Participants will first 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. This dose will be a regular maintenance dose that happens about 4 months after their first dose in this 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 approximately 5 months. This period will overlap with the participant's first 5 months in the PIERRE study.
More detail
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.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: 3 Years and over
- Who can join: All genders
Treatment history
Treatments you must have had:
- ✓ completed all 4 required 12 mg loading doses as per the approved 12 mg dose regimen
- ✓ completed 1 or 2 of the 50 mg loading doses as per the approved 50/28 mg dose regimen
What the study is looking for
- ✓\- Participants must be screened in the PIERRE study to be eligible for enrollment in the PIERRE PK study.
- ✓Participants in the 12 mg group
- ✓Participant must have completed all 4 required 12 mg loading doses as per the approved 12 mg dose regimen.
- ✓Participant is on regular LP maintenance dosing of nusinersen 12 mg every 4 months, with 120 days ± 14 days between...
- ✓Participants in the 28 mg group
Who cannot take part
- ✗Participant is naïve to nusinersen treatment.
- ✗Participant is receiving nusinersen at a dose other than 12 mg or 28 mg.
- ✗Participant has already undergone implantation of the ThecaFlex DRx system or any other implantable medical device...
- ✗Participant is pregnant, currently breastfeeding, or intending to become pregnant during the study.
- ✗NOTE: Other protocol-defined Inclusion/Exclusion criteria may apply.
See the full criteria
Where Is This Study? (1 UK site)
Royal Hallamshire Hospital Neurology Department
Sheffield S102JF, United Kingdom
How to Get in Touch
US Biogen Clinical Trial Center
Sponsor contactCONTACT
Global Biogen Clinical Trial Center
Sponsor contactCONTACT