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A study to test Taldefgropeb Alfa in patients with Spinal Muscular Atrophy.
EU CTIS ID: 2024-511852-42-00
What this study is testing
To evaluate the efficacy of taldefgrobep alfa in participants who are already taking a stable regimen of nusinersen and/or risdiplam and/or have a history of onasemnogene abeparvovec-xioi, compared to placebo, measured by change in the 32 item Motor Function Measure (MFM-32) total score between Baseline and Week 48.
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Spinal Muscular Atrophy confirmed by genetic diagnosis of 5q-autosomal recessive SMA as well as SMN2 copy number
- Ambulant or Non-ambulant
- Treated with an SMA disease-modifying therapy and anticipated to remain on that same treatment regimen dose throughout the trial including nusinersen, risdiplam and/or history of onasemnogene abeparvovec
You likely can't join if
- Cannot have previously taken anti-myostatin therapies
- Must weigh at least 15 kg
- Respiratory insufficiency, defined by the medical necessity for invasive or non-invasive ventilation for daytime treatment while awake (use overnight or during daytime naps is acceptable).
- History of Spinal Fusion within 6 months of Screening. MAGEC rod non-surgical adjustments are allowed during the study
- Presence of an implanted shunt for the drainage of CSF or an implanted central nervous system (CNS) catheter
The study team makes the final eligibility decision.
Where it's taking place
- United States
- United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
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, 18-64 years, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United States; United Kingdom. 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 EU CTIS; the study team decides eligibility.