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Study on Higher Doses of Nusinersen for Patients with Spinal Muscular Atrophy Who Previously Participated in a Nusinersen Study

Verified siteInvestigationalNo placebo
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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 studying a condition known as Spinal Muscular Atrophy (SMA). SMA is a genetic disease that affects the muscles, leading to weakness and movement difficulties. The study involves a treatment called Nusinersen, which is also known by the code name BIIB058. Nusinersen is a type of medication that is injected into the body to help manage the symptoms of SMA. It is designed to target a specific gene related to the disease, aiming to improve muscle function and strength.

The purpose of this study is to evaluate the long-term safety and how well patients tolerate higher doses of Nusinersen. Participants in this study have previously taken part in another study involving Nusinersen. During the course of the study, participants will receive Nusinersen injections and will be monitored for any side effects or changes in their health. The study will also track various health parameters, such as growth and neurological function, over a period of time.

Participants will be observed for any adverse events, which are unexpected medical issues that may arise during the study. The study will also look at changes in laboratory tests, heart function, and other vital signs. The goal is to ensure that the treatment is safe and to understand its effects on the body over an extended period. This study is an important step in understanding how Nusinersen can help people with SMA and improve their quality of life.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial assessment

    Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes verifying completion of the Day 302 visit in the previous study (232SM203).

  2. Step 2

    Medication administration

    The medication nusinersen is administered as a solution for injection through a procedure called intrathecal use, which involves delivering the medication directly into the spinal canal.

    Two dosages are used: SPINRAZA 28 mg and SPINRAZA 50 mg. The specific dosage and frequency are determined by the study protocol.

  3. Step 3

    Monitoring and follow-up

    Participants are monitored for adverse events and changes in health parameters over a period of up to Day 1921.

    Regular assessments include checking growth parameters, clinical laboratory results, electrocardiograms (ECG), and vital signs.

    Neurological examinations are conducted to track changes from baseline, with specific assessments for participants aged ≤2 years and those older than 2 years.

  4. Step 4

    Evaluation of outcomes

    The study evaluates the number of new motor milestones achieved, the use and duration of respiratory support, and overall survival.

    Changes in scores from various neuromuscular tests, such as the CHOP INTEND and HINE, are measured.

    The study also tracks changes in plasma levels of neurofilament, a marker of nerve cell damage.

  5. Step 5

    Completion of study

    The study is estimated to conclude by August 15, 2026.

    Final assessments are conducted to evaluate the long-term safety and tolerability of the higher doses of nusinersen.

Who can join the trial?

5 criteria

  • The patient must have completed the Day 302 visit in a previous study called 232SM203.
  • The patient must have a condition called Spinal Muscular Atrophy, which is a genetic disorder that affects the muscles.
  • The patient can be of any gender, meaning both males and females are eligible.
  • The patient must be within the age range of 2 to 3 years old.
  • The study includes participants who may be considered a vulnerable population, which means they might need extra care or protection.

Who cannot join the trial?

7 criteria

  • Participants who have not previously taken part in study 232SM203 (NCT04089566) cannot join this study.
  • Individuals who have any medical condition that the study doctors believe would make it unsafe for them to participate.
  • Participants who are unable to follow the study procedures or attend the required visits.
  • Individuals who are currently taking part in another clinical trial or have taken part in another trial recently.
  • Participants who have had a serious allergic reaction to any of the study medications or similar medications in the past.
  • Individuals who are pregnant or breastfeeding cannot participate in the study.
  • Participants who have any other condition that the study doctors believe would interfere with the study results.
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Investigated drugs

Nusinersen is a medication used to treat spinal muscular atrophy (SMA). In this study, it is given in higher doses to see if it is safe and well-tolerated over a long period. Nusinersen is administered directly into the spinal fluid, which helps it reach the central nervous system where it can help improve muscle function and slow the progression of SMA.

What is already known about the treatment

Nusinersen – Nusinersen is administered through an injection into the spinal canal, a method known as intrathecal administration. 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 genetic material to modify protein production.

Investigated diseases

Spinal Muscular Atrophy – Spinal Muscular Atrophy (SMA) is a genetic disorder characterized by the progressive loss of motor neurons, which are nerve cells in the spinal cord that control muscle movement. This leads to muscle weakness and atrophy, primarily affecting the muscles closest to the center of the body, such as those in the shoulders, hips, and back. The severity of SMA can vary, with some individuals experiencing significant physical limitations, while others may have milder symptoms. Over time, individuals with SMA may experience difficulties with activities such as walking, sitting, and even breathing. The progression of the disease can differ greatly among individuals, with some experiencing rapid decline and others having a more gradual progression. SMA is considered a rare disease and is often diagnosed in infancy or early childhood.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2023-505637-27-00Protocol code232SM302Estimated enrolment106 patientsSponsorBiogen Idec Research Limited

sourced from the EU Clinical Trials Register and site verification

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