A study to evaluate the safety and effectiveness of urokinase, catalytic domain, fused with a single-chain antibody against von Willebrand factor in patients with immune-mediated thrombotic thrombocytopenic purpura.

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What is this study about?

This study focuses on diseases known as Thrombotic Microangiopathies, a group of conditions where small blood vessels become damaged and blocked by tiny blood clots. One specific type of this condition is Immune-Mediated Thrombocytopenic Purpura, a rare disorder where the body’s immune system mistakenly attacks and destroys platelets, which are the cells responsible for helping blood clot. The research aims to evaluate the safety and effectiveness of an experimental drug called TGD001. This medication is a specialized protein designed to target certain factors in the blood that contribute to the formation of these harmful clots.

The treatment involving TGD001 is administered through an intravenous method, which means it is delivered directly into a vein using a solution for injection. During the study, participants will receive the medication to observe how the body reacts and to see if it helps manage the symptoms of the disease. The process involves monitoring how the drug moves through the body and checking for any side effects or changes in blood cell levels over time.

Who Can Join the Study?

You must be an adult, which means you are 18 years of age or older when you sign the study agreement.
You must be willing and able to understand the study details and sign the informed consent form, which is a document that explains everything about the study before you agree to join.
If you are a man or a woman who could become pregnant, you must avoid sexual intercourse or use a highly effective method of birth control (one that has a failure rate of less than 1% per year) during the study and for at least 90 days after your last dose of the study medicine.
You must be experiencing symptoms of an acute TMA episode, which is a sudden and serious condition involving thrombotic microangiopathy, a medical issue where small blood clots form in tiny blood vessels throughout the body.
You must be able to attend all necessary follow-up appointments to monitor your health during the study.

Who Cannot Join the Study?

Having a different medical condition that explains low blood platelets or hemolytic anemia (a condition where red blood cells are destroyed more quickly than normal) instead of thrombotic microangiopathy (a group of disorders that cause blood clots in small blood vessels).
Having other serious medical, brain-related, or mental health conditions that might make it difficult for doctors to accurately measure the study results.
Having a known hypersensitivity (an allergic reaction) to the study drugs, any of the ingredients used to make them, or drugs that are very similar to them.
Having taken part in another study involving an experimental drug or medical device within the last 30 days that could interfere with this study.
Being unable to follow the specific rules and steps required for this study.
Having a severe, active infection known as sepsis (a life-threatening reaction to an infection that requires medicine to help raise blood pressure).
Being pregnant or currently breastfeeding.
A history of bleeding diathesis (a tendency to bleed more easily or for a longer time than normal) or showing signs of unusual bleeding in the last 30 days.
Having active bleeding inside the body.
Showing signs of symptomatic intracranial hemorrhage (bleeding inside the skull that causes symptoms like headache or confusion).
Having very high, uncontrolled hypertension (high blood pressure), defined as a top number higher than 180 or a bottom number higher than 110.
Having cancer that has spread throughout the body or other serious illnesses that would limit life expectancy to less than 3 months.
Having had a major surgery recently, as decided by the study doctor.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country	Status
Hospital Universitario Y Politecnico La Fe	Valencia	Spain

Other Sites

Site Name	City	Country
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico	Milan	Italy
Unexaaexgf Hoiuwiki Cdrmbjd	Cologne	Germany
Atirztfipf Puvogxzh Hnasnysn Do Ppfms	Paris	France

Trial status

Country	Status	Recruitment Start
France	Not yet recruiting	01.03.2026
Germany	Not yet recruiting	01.03.2026
Italy	Not yet recruiting	01.03.2026
Spain	Not yet recruiting	01.03.2026

Trial locations

Investigated drugs:

UROKINASE, CATALYTIC DOMAIN, FUSED WITH A SINGLE-CHAIN ANTIBODY AGAINST VON WILLEBRAND FACTOR

TGD001 is a medication being tested to see if it is safe and effective for people with certain blood disorders. It works by targeting the processes in the body that cause abnormal blood clots to form in small blood vessels.

Investigated diseases:

Immune-Mediated Thrombotic Thrombocytopenic Purpura – This is a rare blood disorder where the body’s immune system mistakenly attacks an enzyme needed to break down blood clots. This process leads to the formation of small blood clots throughout the body’s blood vessels. As these clots form, they consume a large number of platelets, which are the cells responsible for clotting. This results in a significant decrease in the overall platelet count in the bloodstream. The condition typically progresses through the rapid development of these micro-clots and a subsequent drop in platelets.

Thrombotic microangiopathy – This term describes a group of conditions characterized by the formation of tiny blood clots within small blood vessels. These small clots can obstruct blood flow and cause damage to various tissues and organs. The condition involves a decrease in platelet counts and the presence of damaged red blood cells in the blood. It can progress by affecting different organ systems depending on where the vessel blockage occurs. The underlying mechanism involves various ways the body’s clotting and blood cell processes are disrupted.

Trial ID:

2025-523802-34-00

Protocol code:

TG1-CL-301

Trial Phase:

Human Pharmacology (Phase I) – Other

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A study to evaluate the safety and effectiveness of urokinase, catalytic domain, fused with a single-chain antibody against von Willebrand factor in patients with immune-mediated thrombotic thrombocytopenic purpura.

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?