Muscular Atrophy, SpinalPhase 1PIERRE-PKNCT06555419
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)
A Phase 1 study testing Nusinersen and ThecaFlex DRx System in people with Muscular Atrophy, Spinal, run by Biogen at 21 sites in 7 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 3 years and older
- Conditions the study is for
- Muscular Atrophy, Spinal
- What is being tested?
- Nusinersen and ThecaFlex DRx System
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 78 participants
- Where
- 21 sitesin 7 countries
- Study dates
- 16 Jan 2025to 30 Sep 2027
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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 the LP-delivered study dose and the last nusinersen dose prior to study enrollment.
- Participants in the 28 mg group
- Participant must have completed 1 or 2 of the 50 mg loading doses as per the approved 50/28 mg dose regimen.
- Participant is on regular LP maintenance dosing of nusinersen 28 mg every 4 months, with 120 days ± 14 days between the LP-delivered study dose and the last nusinersen dose prior to study enrollment.
Who cannot join
- Ongoing participation or participation within 6 months or 5 half-lives of the agent (whichever is longer) of enrollment in other interventional clinical trials for the treatment of SMA (except for the PIERRE study or interventional clinical trials of myostatin inhibitors [e.g., apitegromab and taldefgrobep alfa]).
- 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 for intrathecal (IT) administration.
- Participant is pregnant, currently breastfeeding, or intending to become pregnant during the study.
- NOTE: Other protocol-defined Inclusion/Exclusion criteria may apply.
Trial locations and contacts
21 sites in 7 countries. Contact the study team or a site near you to ask about taking part.
Global Biogen Clinical Trial Center contact
21 sites in 7 countries
United States12 sites, 12 recruiting
- Recruiting
Boston Children's Hospital
Boston, Massachusetts, 02115
617-355-8036
Basil T Darras
- Recruiting
Stacey Hall Developmental Pediatrics
Charlottesville, Virginia, 22903
434-924-8184
Rebecca J Scharf
- Recruiting
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611-2605
312-227-3550
Nancy Kuntz
- Recruiting
Helen DeVos Children's Hospita
Grand Rapids, Michigan, 49503
616-391-2241
Jena Krueger
- Recruiting
Milton S. Hershey Medical Center | Pennsylvania State University_Hershey
Hershey, Pennsylvania, 17033
717-232-5443
Dustin Paul
- Recruiting
Texas Childrens Hospital Houston
Houston, Texas, 77030
832-822-1750
David Bauer
- Recruiting
Children's Hospital of the King's Daughters_Norfolk
Norfolk, Virginia, 23507
757-668-7000
Crystal Proud
- Recruiting
Children's Hospital of Orange County
Orange, California, 92868
714-509-3605
Julian Thomas
- Recruiting
Nemours Children's Hospital, Orlando
Orlando, Florida, 32827
407-650-7715
Abdul Hamid Omer
- Recruiting
Stanford University Medical Center | Department of Neurology_Palo Alto
Palo Alto, California, 94304
650-723-5152
John Day
- Recruiting
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
215-590-3174
Patrick Cahill
- Recruiting
St. Joseph's Hospital and Medical Center - Barrow Neurological Institute
Phoenix, Arizona, 85013
602-406-6262
Shafeeq Ladha
Italy2 sites
- Withdrawn
Fondazione Serena Onlus - Centro Clinico Nemo_Milano
Milan, 20162
- Withdrawn
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Roma, 168
Poland2 sites, 2 recruiting
- Recruiting
Szpital Specjalistyczny im. L.Rydygiera w Krakowie
Krakow, 31-826
48604565621
Ryszard Nowak
- Recruiting
Instytut Centrum Zdrowia Matki Polki
Lodz, 93-338
+48606989185
Przyslo Lukasz
Spain2 sites, 2 recruiting
- Recruiting
Hospital Universitario La Paz
Madrid, 28046
+34917277388
Maria Garcia Romero
- Recruiting
Hospital Universitari i Politecnic La Fe_Valencia
Valencia, 46026
34 961 24 55 42
Juan Francisco Vazquez Costa
France1 site, 1 recruiting
- Recruiting
Hôpital Raymond Poincaré
Garches, Hauts De Seine, 92380
+33147104684
Marta Gomez
Germany1 site, 1 recruiting
- Recruiting
Universitaetsklinikum Essen
Essen, 45147
49 201 7230
Tim Hagenacker
United Kingdom1 site, 1 recruiting
- Recruiting
Royal Hallamshire Hospital Neurology Department
Sheffield, South Yorkshire, S102JF
+441142713708
Chanma Hewadduma
About this study
Official title: An Open Label Study to Assess the Pharmacokinetic Profile of Nusinersen (BIIB058) Administered Via the ThecaFlex DRx™ System (PIERRE-PK)
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:
Read more
- 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.
Read the 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.
- Conditions
- Muscular Atrophy, Spinal
- Being tested
- Nusinersen, ThecaFlex DRx System
- Medical subject headings
- Muscular Atrophy, Spinal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NusinersenDrug
Also called BIIB058
Administered as specified in the treatment arm.
ThecaFlex DRx SystemDevice
Implanted as specified in the treatment arm.
| Study group | Role | What they receive |
|---|---|---|
| Nusinersen 12 mg Via LP and ThecaFlex DRx System | Experimental | 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. Receives: Drug: Nusinersen; Device: ThecaFlex DRx System |
| Nusinersen 28 mg Via LP and ThecaFlex DRx System | Experimental | Participants will receive a maintenance dose of nusinersen, 28 mg via LP in the PIERRE-PK study, followed by implantation of the ThecaFlex DRx System and the subsequent nusinersen 28 mg maintenance dose via the system in the PIERRE study. Receives: Drug: Nusinersen; Device: ThecaFlex DRx System |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum Observed Concentration (Cmax) of Nusinersen Delivered via Standard LP and via ThecaFlex DRx System
Measured: Pre-dose and at multiple time points post-dose over 4 months
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
Measured: Pre-dose and at multiple time points post-dose over 4 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Aug 2024
- Study start16 Jan 2025
- Primary completion30 Sep 2027 (estimated)
- Study completion30 Sep 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Muscular Atrophy, SpinalStudies of the same condition, open and under way.
- More trials run by BiogenThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 78 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Biogen
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Alcyone Therapeutics, Inc
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Biogen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06555419
- Sponsor's protocol ID
- 295SM101
- Other IDs
- 2024-514239-21-00
- Acronym
- PIERRE-PK
- Status last verified
- Sep 2026
- First submitted
- 9 Aug 2024
- Last updated
- 3 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/
Study at a glance
From the registry record, last updated by the sponsor on 3 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open Label Study to Assess the Pharmacokinetic Profile of Nusinersen (BIIB058) Administered Via the ThecaFlex DRx™ System (PIERRE-PK)
- ClinicalTrials.gov ID
- NCT06555419
- Acronym / sponsor ID
- PIERRE-PK / 295SM101
- Conditions
- Muscular Atrophy, Spinal
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 78 participants (estimated)
- Lead sponsor
- BiogenIndustry (pharma, biotech, devices)
- Collaborators
- Alcyone Therapeutics, Inc
- Study start
- 16 Jan 2025 (actual)
- Primary completion
- 30 Sep 2027 (estimated)
- Study completion
- 30 Sep 2027 (estimated)
- First posted
- 15 Aug 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Nusinersen 12 mg Via LP and ThecaFlex DRx System | Experimental | Drug: Nusinersen, Device: ThecaFlex DRx SystemParticipants 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 28 mg Via LP and ThecaFlex DRx System | Experimental | Drug: Nusinersen, Device: ThecaFlex DRx SystemParticipants will receive a maintenance dose of nusinersen, 28 mg via LP in the PIERRE-PK study, followed by implantation of the ThecaFlex DRx System and the subsequent nusinersen 28 mg maintenance dose via the system in the PIERRE study. |
Who may qualify
- Ages
- 3 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- 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 the LP-delivered study dose and the last nusinersen dose prior to study enrollment.
- Participants in the 28 mg group
Key exclusion criteria (6 listed)
- Ongoing participation or participation within 6 months or 5 half-lives of the agent (whichever is longer) of enrollment in other interventional clinical trials for the treatment of SMA (except for the PIERRE study or interventional clinical trials of myostatin inhibitors [e.g., apitegromab and taldefgrobep alfa]).
- 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 for intrathecal (IT) administration.
- Participant is pregnant, currently breastfeeding, or intending to become pregnant during the study.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| NusinersenAlso: BIIB058Administered as specified in the treatment arm. | Drug | Nusinersen 12 mg Via LP and ThecaFlex DRx System, Nusinersen 28 mg Via LP and ThecaFlex DRx System |
| ThecaFlex DRx SystemImplanted as specified in the treatment arm. | Device | Nusinersen 12 mg Via LP and ThecaFlex DRx System, Nusinersen 28 mg Via LP and ThecaFlex DRx System |
Primary outcome
Maximum Observed Concentration (Cmax) of Nusinersen Delivered via Standard LP and via ThecaFlex DRx System
Time frame: Pre-dose and at multiple time points post-dose over 4 months
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
Time frame: Pre-dose and at multiple time points post-dose over 4 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Sep 2026.
- Individual participant dataSharing planned: Yes. In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Global Biogen Clinical Trial Center contact
Medical Director study director
Biogen
21 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Nusinersen and ThecaFlex DRx System in people with Muscular Atrophy, Spinal, run by Biogen at 21 sites in 7 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 3 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 21 sites in United States, Italy, Poland, Spain, France and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact US Biogen Clinical Trial Center (866-633-4636, clinicaltrials@biogen.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Maximum Observed Concentration (Cmax) of Nusinersen Delivered via Standard LP and via ThecaFlex DRx System (time frame: Pre-dose and at multiple time points post-dose over 4 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Sep 2026.