Understanding how nusinersen is processed in the body using a new delivery system for spinal muscular atrophy

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This study investigates the pharmacokinetics of nusinersen when administered through the ThecaFlex DRx™ System compared to the traditional lumbar puncture method in patients with spinal muscular atrophy (SMA). The ThecaFlex DRx system is an investigational device designed to deliver medication directly to the spinal canal. Researchers aim to determine the peak concentration of nusinersen in the blood and its distribution over the first 24 hours post-administration. Participants must be enrolled in a related study to qualify for this investigation.

Why it matters

Who can join: the full rules

Key Inclusion Criteria:

\- 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.

Key Exclusion Criteria:

* 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.

Where it's running

• St. Joseph's Hospital and Medical Center - Barrow Neurological Institute
  Phoenix, Arizona, United States Recruiting
• Children's Hospital of Orange County
  Orange, California, United States Recruiting
• Stanford University Medical Center | Department of Neurology_Palo Alto
  Palo Alto, California, United States Recruiting
• Nemours Children's Hospital, Orlando
  Orlando, Florida, United States Recruiting
• Ann & Robert H. Lurie Children's Hospital of Chicago
  Chicago, Illinois, United States Recruiting
• Boston Children's Hospital
  Boston, Massachusetts, United States Recruiting

Study contacts

• Study coordinator: US Biogen Clinical Trial Center
• Email: clinicaltrials@biogen.com
• Phone: 866-633-4636

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Salanersen after early gene therapy in babies with SMA

  This study tests whether adding salanersen (BIIB115) after early gene therapy with Onasemnogene abeparvovec helps babies with SMA.

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• Salanersen given before symptoms to help movement in babies with genetic SMA

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  Recruiting at 14 sites Phase 3 Biogen · Chicago, Illinois and 13 other locations · NCT07221669

• Evaluating pregnancy outcomes in women with spinal muscular atrophy exposed to Spinraza

  This study is trying to see how pregnancy goes for women with spinal muscular atrophy who have used Spinraza, compared to women without the condition who haven't used the drug.

  Recruiting at 13 sites Biogen · Palo Alto, California and 13 other locations · NCT05789758

• Observing the natural history of spinal muscular atrophy

  This study is trying to understand how spinal muscular atrophy affects patients over time by looking at their experiences and health data from both treated and untreated individuals.

  Recruiting at 5 sites Fondazione Policlinico Universitario Agostino Gemelli IRCCS · Genova and 4 other locations · NCT05755451

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.