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Tests treatment safety and results for Spinal Muscular Atrophy (SMA)
Official title 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
ClinicalTrials.gov ID: NCT07287982
What this study is testing
What is ARGX-119 IV?
ARGX-119 IV is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for spinal muscular atrophy (sma).
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This study aims to find the correct dose of ARGX-119 for children with SMA. The study will also look at how safe the study drug is, how well it works, how it moves through the body, and how the immune system responds to it.
- Phase 2: a mid-size study of how well it works
- You might receive a placebo (an inactive treatment) instead of the study drug. You may not know which one you got.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 5 to 17
You may be able to join if
- Is aged ≥5 to \<18 years when completing the informed consent process, defined as providing informed assent according to local regulations and having...
- requirements.
- Has documented historical genetic diagnosis of 5q-SMA.
- Currently receiving a stable SMA treatment regimen (nusinersen or risdiplam) and/or have a history of onasemnogene abeparvovec treatment
- Must be able to walk at least 50 meters without walking aids in the 6MWT at screening
You likely can't join if
- Known medical condition that would interfere with an accurate assessment of SMA, confound the results of the study, or put the participant at undue...
- Recent major surgery, except spinal fusion, within 3 months of screening or intends to have major surgery during the study
- Current or previous administration of antimyostatin therapies in the past 6 months
- Severe scoliosis (defined as curvature \>40°) and/or contractures at screening. o History of spinal fusion within 6 months before screening or...
- Respiratory insufficiency, defined by the medical necessity for invasive or noninvasive ventilation for daytime treatment while awake. Ventilation...
The study team makes the final eligibility decision.
Where it's taking place
- Little Rock, Arkansas, United States
- San Diego, California, United States
- Stanford, California, United States
- Hartford, Connecticut, United States
- Kissimmee, Florida, United States
- Chicago, Illinois, United States
- Iowa City, Iowa, United States
- Baltimore, Maryland, United States
- Boston, Massachusetts, United States
- Columbia, Missouri, United States
- New York, New York, United States
- Hillsborough, North Carolina, United States
- Philadelphia, Pennsylvania, United States
- Memphis, Tennessee, United States
- Flower Mound, Texas, United States
- Houston, Texas, United States
- Norfolk, Virginia, United States
Compensation & support
Compensation mentioned.
ClinicalTrials.gov doesn't provide a reliable structured field for payment or travel support - confirm details with the study team.
Questions & answers
Do participants get paid in this trial?
This study's listing includes signals that participants may be compensated or receive a stipend. Amounts vary and are set by the study team - confirm the details with them.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 5 years to 17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Little Rock, Arkansas, United States; San Diego, California, United States; Stanford, California, United States; Hartford, Connecticut, United States; Kissimmee, Florida, United States; Chicago, Illinois, United States and 11 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.