Active not recruitingPHASE3INTERVENTIONAL

A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy

This study is testing a new medicine, taldefgrobep alfa, for children and young adults aged 4 to 21 who have Spinal Muscular Atrophy (SMA). It aims to find out if this new medicine, when given alongside their current SMA treatment (like nusinersen, risdiplam, or if they've had onasemnogene abeparvovec), can safely help improve muscle strength and movement even more. The researchers believe that taldefgrobep alfa works by blocking a protein called myostatin, which normally limits muscle growth. By blocking it, they hope to help people with SMA build more muscle. This study compares the new medicine to a dummy treatment (placebo) to understand its effects better.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Biohaven Pharmaceuticals, Inc.

Enrolment target

269

Start

06 Jul 2022

Estimated completion

01 Dec 2027

Spinal Muscular Atrophy Neuromuscular Diseases SMA

What is this study about?

This study is about a new medicine called taldefgrobep alfa. It's being tested for people living with Spinal Muscular Atrophy, also known as SMA. SMA is a condition that affects muscles, making them weaker over time. There are already some good treatments available for SMA that help by increasing a specific protein in the body. This new medicine is designed to work in a different way, making it a possible addition to existing treatments.

Our muscles have a natural 'brake' on their growth, controlled by a protein called myostatin. Taldefgrobep alfa works by blocking this myostatin protein. The idea is that by removing this 'brake', muscles might be able to grow bigger and stronger. In earlier studies, this medicine was well tolerated, meaning people didn't seem to have significant problems with it. The hope is that by combining this new approach with existing SMA medicines, people with SMA could see even greater improvements in their movement and overall muscle function.

This study will compare taldefgrobep alfa with a 'placebo', which is a dummy treatment that looks just like the real medicine but contains no active ingredient. This is a common and important way for researchers to truly understand if a new medicine is effective and safe. Comparing it to a placebo helps ensure that any improvements seen are genuinely due to the study medicine and not just other factors.

Key takeaways

This study tests a new medicine, taldefgrobep alfa, for SMA.
It's for people aged 4-21 who are already on other SMA treatments.
The medicine aims to boost muscle growth by blocking a protein called myostatin.
Participants will receive either the new medicine or a dummy treatment (placebo).
The goal is to see if it can further improve muscle strength and movement.
The study also checks how safe the new medicine is.

Who may be eligible?

To be part of this study, you would typically need to meet certain conditions. First, you must have a confirmed diagnosis of Spinal Muscular Atrophy (SMA) that's been checked by genetic tests, and you should be between 4 and 21 years old. It doesn't matter if you can walk or not; both ambulant and non-ambulant individuals can be considered. A key requirement is that you are already on a stable dose of an existing SMA treatment, such as nusinersen, risdiplam, or have previously received onasemnogene abeparvovec, and you expect to continue with that treatment throughout the study.

There are also some reasons why someone might not be able to join. For example, if you have already tried other medicines that block myostatin, you wouldn't be eligible for this study. Your weight must be at least 15 kilograms. Also, if you need a machine to help you breathe during the day while you're awake, you wouldn't be able to participate (though using a breathing machine overnight or for naps is usually fine). If you've had spinal fusion surgery in the last six months, or if you have certain types of implanted medical devices in your brain or spine, you would also be excluded.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Do you have a genetically confirmed diagnosis of Spinal Muscular Atrophy (SMA)?
Are you between 4 and 21 years old?
Are you currently taking or have you previously received an approved SMA treatment (like nusinersen, risdiplam, or onasemnogene abeparvovec)?
Do you weigh at least 15 kilograms (around 2 stone 5 lbs)?
Do you not need a breathing machine to help you breathe during the daytime while you're awake (using one overnight or for naps is okay)?
Have you not received any other 'anti-myostatin' treatments in the past?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you would likely have a series of visits to the study clinic. These visits would involve various assessments to check your muscle strength, movement, and overall health. You would receive either the study medicine, taldefgrobep alfa, or a placebo (a dummy treatment) through injections. The study team would closely monitor your health and any changes you experience throughout the trial. They'll also check your blood or other samples from time to time. The exact number of visits, tests, and the total length of your involvement would be fully explained by the study team before you make any decisions.

Potential risks and benefits

Taking part in any clinical study has potential benefits and potential risks. A possible benefit of being in this study is that you might receive a new medicine, taldefgrobep alfa, which could potentially improve your muscle strength and movement. However, there's no guarantee, and you might receive the placebo group, which would not contain the active medicine. As with any medicine, there could be side effects or risks associated with taldefgrobep alfa, which would be fully explained to you by the study team. You always have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (53)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Phoenix Children's
Verified postcode
Phoenix, United States
UCSD & Rady Children's
Verified postcode
La Jolla, United States
Children's Hospital of Los Angeles
Verified postcode
Los Angeles, United States
UCSF Benioff Children's Hospital, Medical Center
Verified postcode
San Francisco, United States
Children's Hospital Colorado
Verified postcode
Aurora, United States
UF Health, Shands Hospital
Verified postcode
Gainesville, United States
Rare Disease Research
Verified postcode
Atlanta, United States
Northwestern University - Feinberg School of Medicine - Ann & Robert H. Lurie Children's Hospital of Chicago
Verified postcode
Chicago, United States
Indiana University -Riley Research
Verified postcode
Indianapolis, United States
University of Iowa
Verified postcode
Iowa City, United States
University of Kansas Medical Center
Verified postcode
Fairway, United States
Boston Children's Hospital - Harvard
Verified postcode
Boston, United States

Common questions

What is Spinal Muscular Atrophy (SMA)?

SMA is a condition that causes muscles to become weaker over time due to a problem with specific nerve cells.

What does 'adjunctive therapy' mean?

It means the new medicine, taldefgrobep alfa, would be given alongside your current SMA treatment, not instead of it.

What is a 'placebo'?

A placebo is a fake medicine that looks like the real thing but has no active ingredients. It helps researchers compare the effects of the active medicine.

What is myostatin?

Myostatin is a natural protein in your body that slows down muscle growth. This new medicine aims to block it.

Will I know if I'm getting the real medicine or the placebo?

No, typically in these types of studies, neither you nor your doctor will know who is getting the active medicine or the placebo. This is to ensure the study results are as fair and accurate as possible.