Centro Clinico Nemo
Milan, Italy
RecruitingRare disease
Study on How Nusinersen is Processed in Adults and Children with Spinal Muscular Atrophy Using the ThecaFlex DRx System
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What is this trial about?
A plain-language summary of the goals, design and what participants do
This clinical trial is focused on understanding how the body processes the medication nusinersen when it is given to people with Spinal Muscular Atrophy (SMA). SMA is a genetic condition that affects the muscles, making them weaker over time. The study will use a system called the ThecaFlex DRx™ to deliver nusinersen, which is also known by its code name BIIB058. This medication is typically given as a solution for injection.
The purpose of the study is to compare how nusinersen is processed in the body when delivered through the ThecaFlex DRx system versus the traditional method of a lumbar puncture, which is a procedure where a needle is inserted into the lower back to deliver medication. Researchers are particularly interested in finding out the highest amount of nusinersen that appears in the blood after it is given and how much of it is present in the blood during the first 24 hours after administration.
Participants in the study will continue their regular nusinersen treatment, which involves receiving a 12 mg dose every four months. The study will observe how the medication is absorbed and processed in the body, providing valuable information that could help improve treatment methods for those living with Spinal Muscular Atrophy.
The research process
The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.
Who can join the trial?
5 criteria
- The participant must have **Spinal Muscular Atrophy**, a condition that affects the muscles and nerves.
- The participant should be receiving regular maintenance doses of **nusinersen**, a medication used to treat Spinal Muscular Atrophy. This means they should be getting a 12 milligram dose every 4 months, with a possible variation of 2 weeks.
- The participant must be part of the **PIERRE study** to join this specific study.
- The study is open to both **male and female** participants.
- The study includes participants who are considered a **vulnerable population**, which means they might need extra protection or care.
Who cannot join the trial?
1 criterion
- There are no specific reasons listed that would prevent a patient from participating in this study.
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Verified sites
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Verified Verified Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Rome, ItalyResponsive Fundacion Para La Investigacion Biomedica Del Hospital Universitario La Paz
Madrid, Spain
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Investigated drugs
Nusinersen is a medication used to treat spinal muscular atrophy (SMA). In this clinical trial, researchers are studying how the body processes nusinersen when it is administered using a special delivery system called the ThecaFlex DRx system. The goal is to understand how much of the medication enters the bloodstream and how long it stays there after being given through this new method compared to the traditional method of a lumbar puncture.
What is already known about the treatment
Nusinersen – Nusinersen is administered via an injection into the spinal canal, specifically using the ThecaFlex DRx system. It is currently approved for use in treating Spinal Muscular Atrophy (SMA) and is well-documented in medical literature for this purpose. The main therapeutic indication for nusinersen is to improve motor function in individuals with SMA. At the molecular level, nusinersen works by increasing the production of a protein essential for motor neuron survival, which is deficient in SMA patients. It is classified as an antisense oligonucleotide, a type of medication that targets specific RNA molecules to modify protein production.
Investigated diseases
Spinal Muscular Atrophy – Spinal Muscular Atrophy (SMA) is a genetic disorder that affects the motor neurons in the spinal cord, leading to muscle weakness and atrophy. It is caused by a deficiency of a protein necessary for the survival of these neurons. As the disease progresses, individuals may experience difficulty with movement, swallowing, and breathing. The severity of symptoms can vary widely, with some individuals experiencing mild weakness and others facing significant physical challenges. SMA is typically diagnosed in infancy or early childhood, but it can also manifest later in life. The progression of the disease can lead to increased dependency on assistive devices for mobility and daily activities.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase ITrial ID2024-514239-21-00Protocol code295SM101Estimated enrolment58 patientsSponsorBiogen Idec Research Limited
sourced from the EU Clinical Trials Register and site verification
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