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 study about?

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.

1 initial administration

The study involves the administration of nusinersen, a medication used for treating spinal muscular atrophy. The medication is given as a solution for injection.

The administration is performed through a method called intrathecal use, which involves delivering the medication directly into the space around the spinal cord.

2 regular maintenance dosing

Participants receive a regular maintenance dose of nusinersen every four months. The dose is 12 milligrams (mg).

The interval between doses is four months, with a possible variation of up to two weeks.

3 comparison of administration methods

The study aims to compare how the body processes nusinersen when administered through the ThecaFlex DRx system versus a standard lumbar puncture.

The main focus is to determine the highest amount of nusinersen found in the blood after dosing and the amount present over the first 24 hours.

4 monitoring and data collection

Participants are monitored to measure the maximum observed concentration of nusinersen in the blood.

The study also measures the area under the plasma concentration-time curve from zero time to 24 hours after administration.

Who Can Join the Study?

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 Study?

There are no specific reasons listed that would prevent a patient from participating in this study.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
Fondazione Policlinico Universitario Agostino Gemelli IRCCS	Rome	Italy
Hospital Universitario Y Politecnico La Fe	Valencia	Spain
Osrodek Badan Klinicznych Przy Szpitalu Specjalistycznym Im. Ludwika Rydygiera W Krakowie Sp. z o.o.	Cracow	Poland

Other Sites

Site Name	City	Country
Instytut Centrum Zdrowia Matki Polki	Lodz	Poland
Hopital Beaujon	Clichy	France
Hospital Sant Joan De Deu Barcelona	Esplugues De Llobregat	Spain
Klinikum der Technischen Universitaet Muenchen (TUM Klinikum)	Munich	Germany
Cxdnmk Cqryovi Npil	Milan	Italy
Uawfulobxbtgbhkcvlemk Examn Ayz	Essen	Germany
Avkozqsysu Penqtbnh Hxgrekja Di Mndvwpciq	Marseille	France
Fucafcemz Pzhv Ln Inscfgwdsbnpx Bukoogmfo Doi Hxswvavs Ujehtjfasrguw Lz Pax	Madrid	Spain

Trial status

Country	Status	Recruitment Start
France	Not yet recruiting	31.03.2025
Germany	Not yet recruiting	31.03.2025
Italy	Not recruiting	31.03.2025
Poland	Not yet recruiting	31.03.2025
Spain	Recruiting	31.03.2025

Trial locations

Investigated drugs:

Nusinersen

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.

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 ID:

2024-514239-21-00

Protocol code:

295SM101

NCT ID:

NCT06555419

Trial Phase:

Human Pharmacology (Phase I) – Other

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Study on How Nusinersen is Processed in Adults and Children with Spinal Muscular Atrophy Using the ThecaFlex DRx System

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?