Skip to content
Clinical Trials – home

Rare diseases

Investigational molecules

Locations

Our offerNewsroom

Not recruitingRare disease

Study of Taldefgrobep Alfa for Patients with Spinal Muscular Atrophy on Stable Nusinersen and/or Risdiplam Regimen or with Onasemnogene Abeparvovec-xioi History

Verified siteInvestigational
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 is focused on studying Spinal Muscular Atrophy (SMA), a genetic condition that affects the muscles used for movement. The study will test a treatment called Taldefgrobep Alfa, which is given as an injection. The purpose of the study is to evaluate how effective and safe this treatment is for people with SMA, whether they can walk or not.

Participants in the study may already be taking other SMA treatments like nusinersen, risdiplam, or have a history of using onasemnogene abeparvovec-xioi. The study will compare the effects of Taldefgrobep Alfa to a placebo over a period of 48 weeks. During this time, changes in muscle function will be measured to see if the treatment helps improve movement abilities.

The study will also monitor the safety of Taldefgrobep Alfa by checking for any side effects or changes in body composition, such as muscle and bone health. Participants will receive regular assessments to ensure the treatment is well-tolerated. The trial includes an open-label extension, meaning that after the initial study period, all participants may have the opportunity to receive Taldefgrobep Alfa if they choose to continue in the study.

The research process

The trial runs in 7 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, participation is confirmed for individuals with a genetic diagnosis of spinal muscular atrophy (SMA).

    Participants must be either ambulant or non-ambulant and already receiving a stable treatment regimen with nusinersen, risdiplam, or have a history of onasemnogene abeparvovec.

  2. Step 2

    Initial assessment

    An initial assessment is conducted to establish a baseline for the Motor Function Measure (MFM-32) score.

    This assessment helps in evaluating the efficacy of the treatment over the course of the study.

  3. Step 3

    Treatment administration

    Participants receive either taldefgrobep alfa or a placebo, administered as a solution for injection in a pre-filled syringe.

    The treatment is given according to the study's schedule, which spans up to 48 weeks.

  4. Step 4

    Ongoing assessments

    Regular assessments are conducted to monitor changes in motor function, using the MFM-32 score, at various intervals up to week 48.

    Additional assessments include the Revised Upper Limb Module (RULM) and the Hammersmith Functional Motor Scale Expanded (RHS).

  5. Step 5

    Safety and tolerability monitoring

    Safety and tolerability are evaluated through various measures, including changes in lean body mass and bone mineral density.

    Monitoring includes assessments of injection acceptability and tracking any adverse events or lab abnormalities.

  6. Step 6

    Pharmacokinetic analysis

    Blood samples are collected to measure the trough plasma concentrations of taldefgrobep alfa.

    Pharmacokinetic parameters are estimated using population modeling techniques.

  7. Step 7

    Completion of study

    The study concludes with a final assessment at week 48 to determine the change from baseline in the MFM-32 score.

    Participants may have the option to continue in an open-label extension phase, where all receive the active treatment.

Who can join the trial?

6 criteria

  • The patient must have **Spinal Muscular Atrophy (SMA)**, which is confirmed by a genetic test showing a specific type called **5q-autosomal recessive SMA** and the number of copies of a gene called **SMN2**.
  • The patient can either be able to walk (ambulant) or not able to walk (non-ambulant).
  • The patient must be currently receiving a treatment for SMA that changes the course of the disease. This includes medications like **nusinersen**, **risdiplam**, or having a history of receiving a treatment called **onasemnogene abeparvovec**. The patient should continue with the same treatment dose during the trial.
  • The patient must be between the ages of 2 and 3 years old.
  • Both male and female patients can participate.
  • The trial includes individuals who may be considered vulnerable, meaning they might need extra care or protection.

Who cannot join the trial?

4 criteria

  • Participants who are not already taking a stable regimen of medications like nusinersen, risdiplam, or have not had a history of treatment with onasemnogene abeparvovec-xioi.
  • Participants who are not within the specified age range for the study.
  • Participants who do not meet the gender requirements for the study.
  • Participants who are considered part of a vulnerable population that the study is not designed to include.
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

  • Taldefgrobep Alfa

    is a medication being studied for its potential to improve motor function in people with Spinal Muscular Atrophy (SMA). It is being tested to see if it can help both those who can walk and those who cannot. The study aims to determine if this medication can enhance the physical abilities of participants over a period of time.

  • Nusinersen

    is a medication that is already used to treat Spinal Muscular Atrophy. It works by helping to increase the production of a protein that is important for the health of motor neurons, which are the nerve cells that control muscle movement. Participants in the trial may already be taking this medication as part of their treatment plan.

  • Risdiplam

    is another medication used in the treatment of Spinal Muscular Atrophy. It helps to increase the levels of a protein that is crucial for muscle function and survival. This medication is taken orally and is part of the standard care for some participants in the trial.

  • Onasemnogene Abeparvovec-xioi

    is a gene therapy used to treat Spinal Muscular Atrophy. It delivers a copy of a gene that is missing or not working properly in people with SMA. This therapy is typically given as a one-time infusion and is part of the medical history of some participants in the trial.

What is already known about the treatment

Taldefgrobep Alfa – This medication is administered through injection and is currently being studied in clinical trials for its effectiveness in treating Spinal Muscular Atrophy (SMA). It is not yet widely available in medical practice, as it is still under investigation. The main therapeutic indication for Taldefgrobep Alfa is to improve motor function in individuals with SMA. At the molecular level, it works by inhibiting myostatin, a protein that limits muscle growth, thereby potentially enhancing muscle strength and function. It is classified pharmacologically as a myostatin inhibitor.

Investigated diseases

Spinal Muscular Atrophy – Spinal Muscular Atrophy (SMA) is a genetic disorder characterized by weakness and wasting of the muscles used for movement. It is caused by a loss of specialized nerve cells, called motor neurons, in the spinal cord and brainstem. This leads to progressive muscle weakness and atrophy, primarily affecting the proximal muscles, which are closer to the center of the body. SMA can vary in severity, with some individuals experiencing difficulty with basic movements such as sitting and walking. The progression of muscle weakness can lead to complications with breathing and swallowing. The disease is typically diagnosed in infancy or early childhood, but milder forms can present later in life.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-511852-42-00Protocol codeBHV2000-301Estimated enrolment180 patientsSponsorBiohaven Pharmaceuticals Inc.

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.