In short
Clinical trials are investigating GRNA-001 in people with Primary Hyperoxaluria Type 1 (PH1). The study is looking at safety and tolerability, and it is in an early phase trial with 23 participants. This article explains what the trial is studying and who it is for.
Key points
- Clinical trials are investigating GRNA-001 in Primary Hyperoxaluria Type 1 (PH1). The available study is an early Phase 1/2 trial with 23 participants. The main goal is to assess safety and tolerability. Researchers are also measuring treatment-emergent adverse events and serious adverse events. The trial is interventional, meaning participants receive the study treatment as part of the research. The data provided show one authorised study focused on people with PH1.
Trial overview
The available clinical trial for GRNA-001 is NCT06839235, a Phase 1/2 interventional study in Primary Hyperoxaluria Type 1 (PH1). The study status is Authorised, and the planned enrollment is 23 participants.
Condition and population studied
This trial is focused on people with Primary Hyperoxaluria Type 1 (PH1), which is the only condition listed in the source data. The data provided do not list more detailed inclusion or exclusion rules, so the main target population we can confirm is participants with PH1.
Trial phase and design
The study is a Phase 1/2 trial, which means it is in an early stage of clinical research. Phase 1 studies mainly look at safety, while Phase 2 studies begin to gather more information about how the treatment may perform in the disease being studied.
The trial is described as interventional, meaning the researchers give the study treatment to participants and then measure the results. The intervention listed is GRNA-001, and the trial title identifies the study as a Phase 1/2 study in PH1.
Outcomes being measured
The main outcome is the incidence and severity of treatment-emergent adverse events (TEAEs), including TEAEs related to GRNA-001 and serious adverse events (SAEs). In simple terms, researchers are counting health problems that start after treatment begins and checking how serious they are.
This focus shows that the study is designed first to understand safety and tolerability, which is typical for an early phase trial. The brief summary also states that the purpose is to evaluate the safety and tolerability of GRNA-001 in participants with PH1.
What this means for patients
For patients, this trial means researchers are still learning how GRNA-001 behaves in people with PH1 and whether it can be used safely in this group. Because the study is small, with 23 planned participants, it is meant to collect early research information rather than give final answers about treatment benefit.
The source data do not provide details about visits, length of follow-up, or exact eligibility criteria, so those points cannot be confirmed from the trial record provided. Based on the available information, the key topic is safety testing of GRNA-001 in a specific rare kidney-related condition, PH1.