In short
Clinical trials are investigating Apitegromab in people with spinal muscular atrophy (SMA), a genetic condition that causes muscle weakness and muscle loss. These studies are looking at long-term safety, tolerability, and possible benefits, and they include children under 2 years old as well as patients with type 2 and type 3 SMA.
Key points
- Clinical trials of Apitegromab are being done in people with spinal muscular atrophy (SMA). One Phase 3 study is looking at long-term safety and tolerability in patients with type 2 and type 3 SMA who finished earlier Apitegromab trials. Another Phase 2 study is in children younger than 2 years old with SMA and is measuring drug levels, body handling of the treatment, and motor function outcomes. The trials are interventional, meaning participants receive the study treatment. Together, they focus on safety, how the treatment behaves in the body, and possible effects on movement and muscle function.
Trial overview
The clinical trials listed for Apitegromab are studying people with spinal muscular atrophy (SMA), a genetic condition that causes muscle weakness and atrophy, which means muscles get smaller.
Both studies are interventional, which means the research team gives the study treatment and then measures what happens next.
The available trials include a Phase 3 study and a Phase 2 study, with planned enrollment of 236 and 52 participants, respectively.
Phase 3 long-term study in type 2 and type 3 SMA
NCT05626855 is a Phase 3 study that is authorised and is designed to evaluate the long-term safety and efficacy of Apitegromab in patients with SMA who completed previous Apitegromab trials.
This study focuses on patients with type 2 and type 3 SMA, which are forms of the disease included in the trial summary.
The brief summary says the study aims to evaluate long-term safety and tolerability, so the trial is watching how well patients handle the treatment over time.
The primary outcome is the incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) by severity.
Phase 2 study in children under 2 years old
The second study, 2024-520130-29-00, is a Phase 2 trial in subjects who are less than 2 years old and have SMA.
This trial is authorised and plans to enroll 52 participants.
The study is looking at how Apitegromab works in very young children, including both pharmacokinetics (PK), which means how the body handles the treatment, and pharmacodynamics (PD), which means what the treatment does in the body.
The brief summary also says the study is evaluating motor function outcomes, which are the results related to movement and muscle control.
Outcomes and endpoints
The Phase 3 study uses safety-related endpoints, especially TEAEs and SAEs, to understand long-term risk and tolerability.
The Phase 2 study includes several endpoints, such as population PK model-based measures like CL, Vd, Vc, and Vp, as well as Apitegromab and total latent myostatin concentrations in serum.
It also measures change from baseline in the raw score of the BSID-4 GMS at 48 weeks, which is a motor assessment used to track changes in movement skills over time.
In simple terms, these outcomes help researchers see whether the treatment stays safe, how it behaves in the body, and whether it may help with motor function.
Who may participate
The Phase 3 study includes patients with SMA who have completed previous Apitegromab trials, and it specifically mentions type 2 and type 3 SMA.
The Phase 2 study includes subjects younger than 2 years old with SMA.
These eligibility details show that the trials are aimed at different age groups and different stages of the disease.
Key terms explained
Authorised means the study has been approved to move forward.
Enrollment is the planned number of participants in a trial.
Serum is the liquid part of blood that researchers can test to measure treatment levels or other markers.
Baseline means the starting point before treatment effects are measured.
Raw score means the direct score from a test before it is adjusted or changed in any way.