Not recruitingRare disease

Long-term Safety and Efficacy Study of Onasemnogene Abeparvovec in Patients with Spinal Muscular Atrophy

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial focuses on a condition called Spinal Muscular Atrophy (SMA), a genetic disorder that affects the muscles used for movement. The study is examining the long-term effects of a treatment known as AVXS-101, also referred to by its code name OAV101. This treatment is a type of gene therapy, which involves using a specially designed virus to deliver a healthy copy of a gene to the patient's cells. The goal of this therapy is to help improve muscle function in individuals with SMA.

The purpose of the study is to gather information on the safety and effectiveness of AVXS-101 over an extended period. Participants in this study have previously received AVXS-101 in earlier clinical trials. The study will monitor various aspects of the participants' health, including their ability to reach developmental milestones, changes in motor skills, and overall physical health. Researchers will also observe any significant changes in areas such as breathing, swallowing, and heart function.

Throughout the study, participants will undergo regular assessments to track their progress and any potential side effects. These assessments will help researchers understand how the treatment impacts the participants' health over time. The study aims to provide valuable insights into the long-term benefits and risks of using AVXS-101 for treating Spinal Muscular Atrophy.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Joining the study
Participation begins after receiving AVXS-101 gene replacement therapy in a previous clinical study.
The patient or their guardian must complete the informed consent process and agree to follow the study procedures and visit schedule.
Step 2
Treatment administration
The treatment involves the administration of Zolgensma, which contains the active substance onasemnogene abeparvovec.
This is given as a solution for infusion through an intravenous route.
Step 3
Monitoring and follow-up
The study aims to collect long-term safety and effectiveness data.
Patients will be monitored for developmental milestones and changes in various health assessments, such as motor skills, cognitive function, and physical health.
Step 4
Assessment of health changes
The study will track changes from baseline in several areas, including motor function, cognitive abilities, and physical health.
Specific assessments include the Hammersmith Functional Motor Scale, Bayley Scales of Infant and Toddler Development, and others.
Step 5
Evaluation of adverse events
The study will record any serious adverse events or adverse events of special interest that occur during the trial.
Participants will be monitored for any significant changes in health, such as the need for ventilatory or nutritional support.
Step 6
Study duration
The estimated end date for the study is May 21, 2034.
Participants will be involved in the study for the duration necessary to collect the required data.

Who can join the trial?

6 criteria

The patient must have Spinal Muscular Atrophy (SMA), a condition that affects the muscles and nerves.
The patient must have received AVXS-101 gene replacement therapy in a clinical study sponsored by Novartis Pharma AG. This therapy involves replacing a faulty gene with a healthy one to help treat SMA.
The patient, or their parent or legal guardian, must be willing and able to complete the informed consent process. This means they understand the study and agree to participate.
The patient, or their parent or legal guardian, must be able to follow the study procedures and attend scheduled visits.
Both male and female patients can participate.
The study includes patients who may be considered a vulnerable population, meaning they might need extra protection or care.

Who cannot join the trial?

7 criteria

Patients who have not been previously treated with AVXS-101 in a Novartis-sponsored clinical trial.
Patients who are not within the specified age range for the study.
Patients who do not have a confirmed diagnosis of Spinal Muscular Atrophy (SMA), a genetic condition that affects the muscles.
Patients who are unable to comply with the study requirements or follow-up visits.
Patients who have any other medical condition that the study doctors believe would make it unsafe for them to participate.
Patients who are pregnant or breastfeeding.
Patients who are participating in another clinical trial that could interfere with this study.

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Verified
Association Institut De Myologie
Paris, France
Verified
Centre Hospitalier Regional De La Citadelle
Liege, Belgium
Responsive
Universitair Ziekenhuis Gent
Gent, Belgium

Trial locations

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Investigated drugs

AVXS-101 (also known as OAV101) is a gene therapy used to treat patients with spinal muscular atrophy (SMA). This therapy works by delivering a functional copy of the SMN1 gene to the patient's cells. The SMN1 gene is responsible for producing a protein that is crucial for the survival of motor neurons, which are essential for muscle movement. By providing a working version of this gene, AVXS-101 aims to improve muscle function and slow the progression of SMA.

Onasemnogene Abeparvovec

What is already known about the treatment

AVXS-101 – This medication is administered as a one-time intravenous infusion. It is currently being studied in clinical trials for its long-term safety and effectiveness in treating Spinal Muscular Atrophy (SMA). AVXS-101 is designed to address the genetic root of SMA by delivering a functional copy of the SMN1 gene, which is crucial for motor neuron survival. It belongs to the class of gene therapy medications, aiming to provide a lasting therapeutic effect by correcting the underlying genetic defect.

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 the shoulders, hips, and back. SMA is caused by mutations in the SMN1 gene, which is responsible for producing a protein essential for motor neuron survival. The severity of the disease can vary, with some individuals experiencing symptoms in infancy, while others may not show signs until later in childhood or adulthood. As the disease progresses, individuals may experience difficulties with movement, swallowing, and breathing. The progression rate and severity can differ significantly among individuals with SMA.