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Apitegromab

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.

Table of Contents

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.[1][2]

Both studies are interventional, which means the research team gives the study treatment and then measures what happens next.[1][2]

The available trials include a Phase 3 study and a Phase 2 study, with planned enrollment of 236 and 52 participants, respectively.[1][2]

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.[1]

This study focuses on patients with type 2 and type 3 SMA, which are forms of the disease included in the trial summary.[1]

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.[1]

The primary outcome is the incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) by severity.[1]

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.[2]

This trial is authorised and plans to enroll 52 participants.[2]

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.[2]

The brief summary also says the study is evaluating motor function outcomes, which are the results related to movement and muscle control.[2]

Outcomes and endpoints

The Phase 3 study uses safety-related endpoints, especially TEAEs and SAEs, to understand long-term risk and tolerability.[1]

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.[2]

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.[2]

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.[1][2]

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.[1]

The Phase 2 study includes subjects younger than 2 years old with SMA.[2]

These eligibility details show that the trials are aimed at different age groups and different stages of the disease.[1][2]

Key terms explained

Authorised means the study has been approved to move forward.[1][2]

Enrollment is the planned number of participants in a trial.[1][2]

Serum is the liquid part of blood that researchers can test to measure treatment levels or other markers.[2]

Baseline means the starting point before treatment effects are measured.[2]

Raw score means the direct score from a test before it is adjusted or changed in any way.[2]

Trial IDPhaseCondition studiedStatusEnrollment
NCT05626855Phase 3Spinal Muscular Atrophy (type 2 and type 3)Authorised236
2024-520130-29-00Phase 2Spinal Muscular Atrophy in subjects less than 2 years oldAuthorised52

FAQ

  • What condition are the Apitegromab trials studying? The trials are studying spinal muscular atrophy, also called SMA. This is a genetic condition that causes muscle weakness and muscle wasting.
  • Who can take part in these trials? One trial includes patients with type 2 and type 3 SMA who completed earlier Apitegromab studies. Another trial includes subjects younger than 2 years old with SMA.
  • What phases are the Apitegromab trials? The available trials are in Phase 2 and Phase 3. Phase 2 studies often look at how the treatment works in the body and early signs of benefit, while Phase 3 studies focus more on safety and efficacy in a larger group.
  • What are the trials trying to measure? One trial is measuring long-term safety and tolerability, including treatment-emergent adverse events and serious adverse events. The other trial is measuring drug levels and motor function outcomes.
  • How many people are planned to join the studies? The Phase 3 study plans to enroll 236 participants, and the Phase 2 study plans to enroll 52 participants.

Ongoing Clinical Trials on Apitegromab

  • Study of Apitegromab Treatment in Children Under 2 Years Old with Spinal Muscular Atrophy (SMA)

    Recruiting

    2 1 1
    Investigated drugs:
    Belgium France Italy The Netherlands Spain

  • Study on Long-Term Safety and Effects of Apitegromab in Patients with Type 2 and Type 3 Spinal Muscular Atrophy Who Completed Previous Trials

    Not recruiting

    3 1 1
    Investigated drugs:
    Belgium France Germany Italy The Netherlands Poland +1

Glossary

  • Spinal Muscular Atrophy (SMA): A genetic condition that causes muscle weakness and muscle loss.
  • Type 2 SMA: A form of SMA included in the long-term Phase 3 study.
  • Type 3 SMA: Another form of SMA included in the long-term Phase 3 study.
  • Phase 2: A trial stage that often looks at how a treatment works in the body and early signs of benefit.
  • Phase 3: A later trial stage that usually studies safety and effectiveness in a larger group of people.
  • Interventional study: A study where participants receive a treatment so researchers can measure its effects.
  • Long-term safety: How safe a treatment appears when used over a longer period of time.
  • Tolerability: How well people can handle a treatment without major problems.
  • Treatment-emergent adverse event (TEAE): A health problem that starts or gets worse after treatment begins.
  • Serious adverse event (SAE): A severe health problem that may need urgent medical care or hospital treatment.
  • PK (pharmacokinetics): A way to study how a treatment moves through the body.
  • PD (pharmacodynamics): A way to study what a treatment does in the body.

References