A Study to Assess the Safety, Tolerability, Efficacy, Pharmacokinetics, and Immunogenicity of Intravenous Administration of ARGX-119 in Pediatric Participants Aged 5 to Less Than 18 Years With Spinal Muscular Atrophy
This study is investigating a new medicine, ARGX-119, for children and teenagers aged 5 to 17 who have Spinal Muscular Atrophy (SMA). The main goals are to find the most suitable dose of ARGX-119, understand its safety, and see how effective it is in improving muscle weakness. Participants will continue their usual SMA treatment while in the study. For the first 24 weeks, some will receive ARGX-119 and others a dummy medicine (placebo). After this, everyone will receive ARGX-119 for up to two years. The researchers hope this medicine might help strengthen muscles by improving signals between nerves and muscles.
At a glance
What is this study about?
Spinal Muscular Atrophy (SMA) is a condition that causes muscle weakness and can make it harder to move around. While there are already some good treatments for SMA, doctors are always looking for new ways to help patients even more. This particular study is for children and teenagers with SMA aged 5 to 17 years old.
The study is exploring a new medicine called ARGX-119. The main idea behind ARGX-119 is to help improve how well the signals travel from your nerves to your muscles. In SMA, these signals can be faulty, leading to muscle weakness and tiredness. By boosting these signals, researchers hope ARGX-119 might help make muscles stronger and reduce tiredness, which could improve how children feel and their ability to do daily activities.
This is a 'Phase 2' study, which means it’s designed to find the best dose of the medicine and get a clearer idea of how safe and effective it is. It's an important step before potentially wider use. All participants will continue to receive their usual SMA treatments during the study. The researchers will be carefully monitoring everyone to understand how ARGX-119 works in the body and if it triggers any immune responses.
Key takeaways
- This study is for children aged 5-17 with SMA, already on existing SMA treatments.
- It tests a new medicine, ARGX-119, to see if it improves muscle strength and reduces tiredness.
- Participants will receive either ARGX-119 or a dummy medicine for 24 weeks, then everyone receives ARGX-119.
- The medicine is given through a drip (IV).
- Regular clinic visits and tests will monitor safety and how well the medicine works.
- Participation lasts up to about 2.5 years, and you can withdraw at any time.
Who may be eligible?
To be part of this study, children and teenagers need to be between 5 and 17 years old. They must have a confirmed diagnosis of SMA (specifically '5q-SMA'). Importantly, they should already be receiving stable treatment for their SMA, such as nusinersen or risdiplam, or have previously been treated with onasemnogene abeparvovec. They also need to be able to walk at least 50 metres without walking aids.
Some things might mean a child can't take part. For example, if they have another serious medical condition that might make it unsafe or make it hard to tell if the new medicine is working. Children who've had major surgery recently (within 3 months, unless it was for spinal fusion), or who plan to have major surgery during the study, can’t join.
Also, if they have very severe scoliosis (a curved spine) or stiff joints, or if they needed breathing support during the day (not just overnight or for naps), they wouldn't be able to participate. Having certain past treatments, like 'antimyostatin therapies' in the last six months, would also mean they can't join this study.
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.
- Is my child aged between 5 and 17 years old?
- Does my child have a confirmed diagnosis of '5q-SMA'?
- Is my child currently on a stable SMA treatment (like nusinersen or risdiplam) or has they had previous gene therapy?
- Can my child walk at least 50 metres (about 160 feet) without using walking aids?
- Does my child NOT use breathing support during the daytime (breathing support only at night or for naps is fine)?
- Has my child NOT had certain other treatments (like antimyostatin therapies) in the last six months?
What does participation involve?
If you or your child decide to take part in this study, it involves several steps and visits. For the first 24 weeks (about 6 months), participants will be randomly assigned to receive either the study medicine, ARGX-119, or a 'placebo' (a dummy medicine that looks the same but contains no active drug). Neither you, your child, nor the study team will know who is getting which treatment during this time – this is called 'double-blinded'. Both treatments will be given through a drip into a vein (intravenously).
After these first 24 weeks, everyone who completes this part of the study will then receive ARGX-119 for up to 100 weeks (which is about two years). During the study, there will be regular visits to the clinic for assessments. These will include check-ups, blood tests, and tests to measure muscle strength and how well your child can move. The total time a child could be in the study is about 2.5 years.
Potential risks and benefits
Locations (24)
- Arkansas Children's HospitalVerified postcodeLittle Rock, United States· Recruiting
- Rady Childrens HospitalVerified postcodeSan Diego, United States· Not yet recruiting
- Stanford University Medical CenterVerified postcodeStanford, United States· Not yet recruiting
- Connecticut Children's Medical CenterVerified postcodeHartford, United States· Recruiting
- Rare Disease Research FL LLCVerified postcodeKissimmee, United States· Recruiting
- Ann & Robert H. Lurie Children's Hospital of ChicagoVerified postcodeChicago, United States· Recruiting
- University of Iowa Stead Family Children's HospitalVerified postcodeIowa City, United States· Recruiting
- The Johns Hopkins HospitalVerified postcodeBaltimore, United States· Not yet recruiting
- Boston Children's HospitalVerified postcodeBoston, United States· Not yet recruiting
- The Curators of the University of Missouri on behalf of University of Missouri Health CareVerified postcodeColumbia, United States· Not yet recruiting
- Columbia University Herbert Irving Comprehensive Cancer CenterVerified postcodeNew York, United States· Not yet recruiting
- Rare Disease Research NC, LLCVerified postcodeHillsborough, United States· Recruiting
Common questions
What is Spinal Muscular Atrophy (SMA)?
SMA is a genetic condition that affects the nerves that control muscle movement, leading to muscle weakness over time.
What is ARGX-119?
ARGX-119 is a new investigational medicine being tested to see if it can help improve muscle strength in children with SMA.
What is a 'placebo'?
A placebo is a dummy medicine that looks exactly like the study drug but contains no active ingredients. It helps researchers compare the real drug's effects.
Will my child still get their usual SMA treatment?
Yes, all participants in this study will continue to receive their existing, stable SMA treatments alongside the study medicine or placebo.
How long will the study last?
Participants will be in the study for a total of up to about two and a half years.
How to find out more
Sabine Coppieters, MD
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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