Skip to content
Clinical Trials – home

Rare diseases

Investigational molecules

Locations

Our offerNewsroom

Not recruitingRare disease

Long-term Safety Study of Onasemnogene Abeparvovec for Patients with Spinal Muscular Atrophy

Fast replyRegistered drug
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

What is this trial about?

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

This clinical trial focuses on the long-term follow-up of patients with Spinal Muscular Atrophy (SMA), a genetic disorder that affects the control of muscle movement. The study involves a treatment called OAV101, also known by its scientific name onasemnogene abeparvovec or its code name AVXS-101. This treatment is administered as a solution for infusion, which means it is given directly into the bloodstream through a vein. The purpose of the study is to assess the long-term safety of this treatment in patients who have previously participated in clinical trials involving OAV101.

Participants in this study will be monitored over an extended period to observe any serious side effects or specific adverse events that may arise from the treatment. The study aims to understand the long-term effects of OAV101 on patients with SMA, focusing on safety aspects. The trial will track the number and proportion of patients experiencing these events throughout the study duration, which can last up to 15 years.

In addition to safety, the study will also look at developmental milestones, which are key skills or abilities that children typically achieve as they grow. Researchers will assess how many participants reach these milestones and whether they maintain them over time. Changes in motor function will be measured using specific scales, such as the Hammersmith Functional Motor Scale – Expanded and the Revised Upper Limb Module. The study will also monitor vital signs and laboratory values to identify any significant changes that could be related to the treatment.

The research process

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

  1. Step 1

    Joining the study

    Participation in a previous OAV101 clinical trial is required.

    Written informed consent must be obtained before any assessment is performed.

    Patient, parent, or legal guardian must be willing and able to comply with study procedures.

  2. Step 2

    Treatment administration

    The treatment involves the administration of Zolgensma, which contains the active substance onasemnogene abeparvovec.

    The medication is provided as a solution for infusion and is administered through intravenous use.

  3. Step 3

    Monitoring and follow-up

    The primary objective is to assess long-term safety, focusing on treatment-emergent serious adverse events (SAEs) and adverse events of special interest (AESIs).

    The study will monitor the number and proportion of patients reporting these events over the entire duration of the study, which is up to 15 years.

  4. Step 4

    Developmental milestone assessment

    Participants will be evaluated for developmental milestones using the Developmental Milestone Checklist.

    The study will track the number and proportion of participants demonstrating each milestone and maintaining them over time.

  5. Step 5

    Functional and motor skills evaluation

    Changes from baseline in the Hammersmith Functional Motor Scale – Expanded (HFMSE) total score will be assessed.

    Changes from baseline in the Revised Upper Limb Module (RULM) total score will also be evaluated.

  6. Step 6

    Vital signs and laboratory assessments

    The study will monitor potentially clinically significant findings in vital signs and laboratory values.

    These findings will be summarized individually for each parameter.

Who can join the trial?

5 criteria

  • The patient must have previously participated in an OAV101 clinical trial.
  • Written informed consent must be obtained before any assessment is performed. This means the patient or their legal representative must sign a document agreeing to participate after being informed about the study.
  • The patient, or their parent/legal guardian, must be willing and able to follow the study procedures.
  • The study includes both male and female participants.
  • The study is open to individuals who may be considered part of a vulnerable population, which means they might need extra protection or care.

Who cannot join the trial?

9 criteria

  • Patients who have any other serious health conditions that could interfere with the study.
  • Patients who have had a severe allergic reaction to any of the ingredients in the study medication.
  • Patients who are currently participating in another clinical trial.
  • Patients who have received certain treatments for Spinal Muscular Atrophy (SMA) that might affect the study results.
  • Patients who are unable to follow the study procedures or attend the required visits.
  • Patients who have a history of certain infections that could affect their safety during the study.
  • Patients who are pregnant or breastfeeding, as the effects on the baby are not known.
  • Patients who have a history of drug or alcohol abuse that could interfere with the study.
  • Patients who have any condition that, in the opinion of the study doctor, makes them unsuitable for the study.
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.

We usually reply within a few days

Verified sites

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

Trial locations

Where you can join this trial

Countries are shaded by recruitment status. Click a recruiting country to ask about joining there.

Not recruiting
Not finding your country?

Not sure what to do next?

Joining a clinical trial can seem overwhelming. We guide you step by step, so you know exactly what to expect and how we support you through the process.

See the full process and FAQ

Investigated drugs

OAV101 is a medication being studied for its long-term safety in patients with Spinal Muscular Atrophy (SMA). It is administered either intravenously (IV) or intrathecally (IT). The study aims to monitor any serious adverse events or specific adverse events that may occur over time in patients who have received this treatment.

What is already known about the treatment

OAV101 – This medication is administered either intravenously (IV) or intrathecally (IT) and is currently being studied for its long-term safety in treating Spinal Muscular Atrophy (SMA). It is in the clinical trial phase, with ongoing research to evaluate its safety profile over an extended period. OAV101 is primarily indicated for patients with SMA, a genetic disorder affecting muscle strength and movement. The medication works by delivering a functional copy of the SMN1 gene, which is crucial for motor neuron survival, thereby addressing the underlying cause of SMA. It is classified as a gene therapy, aiming to provide a long-term therapeutic effect by correcting the 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. SMA is classified into different types based on the age of onset and the highest physical milestone achieved.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-511707-42-00Protocol codeCOAV101A12308Estimated enrolment173 patientsSponsorNovartis Pharma AG

sourced from the EU Clinical Trials Register and site verification

Want to learn more about this trial or check if you can participate?

Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.

ClinicalTrials.eu

Every clinical trial in Europe, in plain language.

Explore the full ecosystem of clinical research in Europe.

Search trials

Legal notice · Published by CTIN POLAND sp. z o.o., ul. rtm. Witolda Pileckiego 67/109, 02-781 Warsaw, Poland · KRS 0001111334 · REGON 528919042 · NIP 9512598637

© 2026 Clinical Trials EU – European Clinical Trials Information Network

GDPR compliance, ISO 9001 and ISO 27001 certified (LL-C Certification)

On this site, “treatment” means an investigational medicine being studied in a clinical trial. Its safety and efficacy for the use being studied have not yet been confirmed, some participants may receive a placebo or a comparator medicine, and taking part does not guarantee any health benefit. The decision to take part is made by the doctor at the research site. This site is for information only and does not replace medical advice.

This service is not affiliated with the European Commission, the EMA, or the official CTIS system. Most information comes from publicly available international clinical-trial registries, supplemented by data from academic sites, national regulators and commercial sponsors. On this site, “treatment” and “therapy” mean a medicine being tested in a clinical trial. Its safety and effectiveness in the use being studied are not yet confirmed, some participants may receive a placebo or a comparator, and taking part does not guarantee a health benefit. The doctor at the research site decides who can take part. This site provides information, not medical advice. Certain content and visual elements on this website have been generated or enhanced using artificial intelligence (AI).