In short
Clinical trials are studying UROKINASE, CATALYTIC DOMAIN, FUSED WITH A SINGLE-CHAIN ANTIBODY AGAINST VON WILLEBRAND FACTOR in people with acute ischemic stroke and in people with immune-mediated thrombotic thrombocytopenic purpura or other thrombotic microangiopathies. These early-phase trials mainly aim to evaluate safety, tolerability, and treatment-emergent side effects in carefully selected patient groups.
Key points
- Clinical trials of UROKINASE, CATALYTIC DOMAIN, FUSED WITH A SINGLE-CHAIN ANTIBODY AGAINST VON WILLEBRAND FACTOR are early Phase 1 studies. One trial is in people with acute ischemic stroke, including those who receive endovascular thrombectomy and those who do not receive standard clot-busting treatment. The other trial is in people with suspected or diagnosed acute immune-mediated thrombotic thrombocytopenic purpura and related thrombotic microangiopathy. The main goals are to assess safety, tolerability, and treatment-emergent adverse events. The stroke trial also measures symptomatic intracranial hemorrhage. Together, the trials plan to enroll 200 participants.
Trial overview
Two authorized interventional trials are studying UROKINASE, CATALYTIC DOMAIN, FUSED WITH A SINGLE-CHAIN ANTIBODY AGAINST VON WILLEBRAND FACTOR. Both are Phase 1 studies, which are early trials mainly designed to look at safety and tolerability.
Acute ischemic stroke study
The first trial is titled “Evaluation of Novel Thrombolytic TGD001 in Patients With Acute Ischemic Stroke” and is being done in people with acute ischemic stroke. The study is authorized, planned for 130 participants, and is given as an interventional study with intravenous treatment.
This study includes people whose stroke is supported by neuroimaging, meaning brain scans are used to confirm the diagnosis. It includes two groups: people who receive endovascular thrombectomy and people who do not receive endovascular thrombectomy or standard-of-care intravenous thrombolysis.
The main goal is to assess the safety and tolerability of single intravenous doses of TGD001 in this stroke setting. The primary outcomes are symptomatic intracranial hemorrhage and treatment-emergent adverse events.
Thrombotic microangiopathy study
The second trial is titled “TGD001 Treatment in Thrombotic Microangiopathies” and is also authorized and interventional. It studies people with immune-mediated thrombotic thrombocytopenic purpura and thrombotic microangiopathy.
This trial plans to enroll 70 participants and is focused on an acute episode, meaning a sudden active phase of illness. The brief summary says the study aims to assess the safety and tolerability of TGD001 in participants with suspicion or clinical diagnosis of an acute iTTP episode.
The primary outcome in this study is treatment-emergent adverse events. That means the researchers are watching for health problems that appear after treatment begins.
Who can participate
In the stroke trial, participants must have acute ischemic stroke and the diagnosis must be supported by imaging. The trial also separates participants based on whether they receive endovascular thrombectomy or not.
In the thrombotic microangiopathy trial, participants are people with suspicion or a clinical diagnosis of an acute immune-mediated thrombotic thrombocytopenic purpura episode. The condition list also includes thrombotic microangiopathy, showing that the study is focused on this disease group.
What the trials measure
The stroke trial uses two main endpoints, or main results the study wants to measure: symptomatic intracranial hemorrhage and treatment-emergent adverse events. These endpoints help researchers check both serious bleeding and overall safety.
The thrombotic microangiopathy trial uses treatment-emergent adverse events as its primary endpoint. This endpoint is a standard way to track whether new health problems happen after treatment starts.
Trial phase and study design
Both studies are in Phase 1, which is usually the first stage of testing in people. At this stage, the main focus is safety, tolerability, and early signs of how the treatment behaves in the target group.
Both trials are listed as interventional, meaning the researchers give a treatment and then observe the results. Together, the two studies plan to enroll 200 people.