In short
DNL952 is an investigational substance being studied in a clinical trial for adults with late-onset Pompe disease (LOPD). The trial is a Phase 1/2 study with 32 participants and is currently authorised to begin. It is given as an intravenous infusion, and the main goal is to measure its safety and tolerability in people with LOPD.
Key points
- DNL952 is an investigational drug, not yet approved as a medicine.
- It is being tested in a Phase 1/2 clinical trial.
- The trial focuses on adults with late-onset Pompe disease (LOPD).
- It is given as an intravenous infusion.
- The study will include 32 participants.
- The main goal is to assess safety and tolerability, including infusion-related reactions.
What is DNL952?
DNL952 is an investigational substance, meaning it has not been approved as a medicine. It is being developed for the treatment of late-onset Pompe disease (LOPD), a rare genetic condition that causes muscle weakness. The trial record does not state its chemical class or whether it is a small molecule or a protein-based substance.
How does DNL952 work?
The trial record does not describe the mechanism of action of DNL952. No information is provided about its molecular target or how it might affect the body in people with late-onset Pompe disease.
Which conditions is it studied for?
- Late-onset Pompe disease (LOPD) — a rare inherited disorder in which a sugar called glycogen builds up in muscles, leading to progressive weakness and breathing problems.
How is DNL952 given?
In the clinical trial, DNL952 is given as an intravenous infusion. This means the drug is delivered directly into a vein over a period of time.
What do the clinical trials show?
One clinical trial of DNL952 is registered in the EU Clinical Trials Information System (CTIS). It is a Phase 1/2 study, which means it combines the first tests in humans (Phase 1) with early tests of how well the drug may work (Phase 2). The trial is currently authorised, meaning it has received approval to begin but may not yet have started enrolling participants.
What is the trial measuring?
The main outcomes of the trial are safety-related. The study will measure the incidence, severity, and seriousness of treatment-emergent adverse events (TEAEs), which are any unwanted medical events that appear after treatment starts. It will also measure the incidence and severity of infusion-related reactions (IRRs), which are side effects that can occur during or shortly after the infusion.
Who can take part?
The trial is open to adult participants with late-onset Pompe disease. The planned enrollment is 32 participants. The record does not provide further details about specific inclusion or exclusion criteria.
What side effects are known?
No side effects of DNL952 are described in the trial record. Because the trial is in its early stages, the safety profile of the drug is not yet known. The study itself is designed to identify side effects, including infusion-related reactions.