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

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What is this study about?

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.

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 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 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 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 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 pharmacokinetic analysis

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

Pharmacokinetic parameters are estimated using population modeling techniques.

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 Study?

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 Study?

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.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country	Status
Hospital Universitario Y Politecnico La Fe	Valencia	Spain
Katholieke Universiteit te Leuven	Leuven	Belgium

Other Sites

Site Name	City	Country
Fondazione Istituto Neurologico Nazionale Casimiro Mondino	Pavia	Italy
Hospital Sant Joan De Deu Barcelona	Esplugues De Llobregat	Spain
Dolnoslaski Szpital Specjalistyczny Im. T.Marciniaka-Centrum Medycyny Ratunkowej	Wroclaw	Poland
Universitair Medisch Centrum Utrecht	Utrecht	The Netherlands
Azienda Unita Sanitaria Locale Di Bologna	Bologna	Italy
Instytut Pomnik Centrum Zdrowia Dziecka	Warsaw	Poland
Medical Center – University Of Freiburg	Freiburg Im Breisgau	Germany
Uniwersytecki Szpital Kliniczny W Poznaniu	Poznan	Poland
Universitair Ziekenhuis Gent	Gent	Belgium
Ospedale Pediatrico Bambino Gesu’	Rome	Italy
Uqsmwrkelfbkqkmshyaud Ezuhg Aha	Essen	Germany
Fjtmavkf Naqgaimwo Bxym	Brno	Czechia
Cmekty Cpqfjod Nyar	Milan	Italy
Fjehuhmb nbzxykfbs Msldu a Hqxzygw	Prague	Czechia
Hcrjblim Upgjhcafkncud Dwxlipms	Donostia / San Sebastian	Spain
Kyhvhmsc drx Ueqlbrlbqmco Mkjdfzdu Ajz	Munich	Germany
Ueshanxeezzcyz Chosvbi Kyjicexrv	Gdansk	Poland
Udwvaqfxge Oq Agngfsk	Edegem	Belgium

Trial status

Country	Status	Recruitment Start
Belgium	Not recruiting	19.01.2023
Czechia	Not recruiting	19.01.2023
Germany	Not recruiting	19.01.2023
Italy	Not recruiting	19.01.2023
Poland	Not recruiting	19.01.2023
Spain	Not recruiting	19.01.2023
The Netherlands	Not recruiting	19.01.2023

Trial locations

Investigated drugs:

Taldefgrobep Alfa

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.

Investigated diseases:

Spinal muscular atrophy

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 ID:

2024-511852-42-00

Protocol code:

BHV2000-301

NCT ID:

NCT05337553

Trial Phase:

Therapeutic confirmatory (Phase III)

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

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?