AuthorisedPhase I and Phase II (Integrated)- OtherInterventional

An Adaptive Dose Escalation and Expansion Basket Trial to Explore the Safety, Pharmacology, and Clinical Activity of TGD001 in Immune-Mediated Thrombotic Thrombocytopenic Purpura (iTTP) and Other Thrombotic Microangiopathies

This research study is investigating a new medication called TGD001. We are looking for people with a rare blood condition called Immune-Mediated Thrombotic Thrombocytopenic Purpura (iTTP) or similar blood disorders where tiny blood clots form. The main goal of this study is to find out if TGD001 is safe when given to people and to understand how it behaves in the body. We also want to see if it helps improve the condition and reduces symptoms. This is an early-stage study, combining Phase I and Phase II, which means we are carefully testing different doses to find the most effective and safest amount. Your safety and well-being are our top priorities throughout this research.

At a glance

Status

Authorised

Phase

Phase I and Phase II (Integrated)- Other

Sponsor

TargED Biopharmaceuticals B.V.

Enrolment target

Start

06 Mar 2026

Immune-Mediated Thrombotic Thrombocytopenic Purpura Thrombotic microangiopathy

What is this study about?

This study is about a new medicine called TGD001, which is being tested for a serious but rare blood condition named Immune-Mediated Thrombotic Thrombocytopenic Purpura, often shortened to iTTP. People with iTTP have issues with their blood clotting system, leading to tiny clots that can block blood vessels and cause problems in organs like the brain or kidneys. The study is also looking at other similar conditions where these small clots form across the body.

The main purpose of this research is to see if TGD001 is safe for people to take. We will be carefully watching for any side effects. We also want to understand how the medicine works in the body and how it is processed. Another important aim is to see if TGD001 can help people get better by improving their symptoms and preventing further complications related to their condition.

This is an early stage of testing, called a Phase I and Phase II study. This means we are starting with a small number of people and gradually increasing the dose of TGD001 to find the safest and most effective amount. If successful, this new medicine could offer a new treatment option for people living with these challenging blood disorders.

Key takeaways

This study is testing a new medicine (TGD001) for a rare blood condition called iTTP.
The main goals are to check the medicine's safety and see how it works in the body.
It's an early-stage study, aiming to find the best dose.
Participants must be 18 or older and have iTTP or a similar blood disorder.
You will have regular check-ups and blood tests if you join.
You can stop participating at any time.

Who may be eligible?

To join this study, you must be at least 18 years old. Both men and women can take part. The study is specifically for people who have been diagnosed with Immune-Mediated Thrombotic Thrombocytopenic Purpura (iTTP) or similar blood disorders that cause tiny blood clots.

The research team will need to check your medical history and conduct some tests to make sure that taking part in the study is right for you and won't put you at unnecessary risk. They will explain all the specific requirements in more detail.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you at least 18 years old?
Have you been diagnosed with Immune-Mediated Thrombotic Thrombocytopenic Purpura (iTTP)?
Have you been diagnosed with a similar blood disorder involving tiny blood clots?
Are you able to attend regular clinic visits and follow study procedures?

Answer every question to see your result.

What does participation involve?

If you decide to join this study, you will be given the new medicine, TGD001, and followed closely by the study team. You will have regular visits to the clinic where doctors and nurses will check your health, ask about any symptoms you're having, and look for any side effects. These visits will likely include blood tests to check your blood cell counts, how your organs are functioning, and to measure levels of the study drug in your body.

The length of your participation will depend on how you respond to the medicine and what the study doctors think is best for you. There will be an initial period of treatment, followed by continued monitoring to see how you are doing after stopping the medicine. The study doctors will provide a detailed schedule of visits and tests so you know exactly what to expect from beginning to end.

Potential risks and benefits

Taking part in a clinical trial has potential benefits and risks. You might benefit from receiving a new treatment that isn't yet widely available, and it could improve your condition. However, there's always a chance the new medicine might not help you, or it could cause side effects that we don't fully know about yet. The study team will monitor you very closely for any problems. Remember, you can decide to leave the study at any time, for any reason, without it affecting your usual medical care.

Locations (4)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
Germany
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Unverified
France
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Unverified
Italy
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Unverified
Spain

Common questions

What is iTTP?

iTTP (Immune-Mediated Thrombotic Thrombocytopenic Purpura) is a rare but serious blood disorder where your body's immune system mistakenly attacks healthy cells, leading to tiny blood clots forming in small blood vessels throughout the body.

What is the new medicine TGD001?

TGD001 is an investigational new medicine being tested in this study. We want to understand how it works, if it's safe, and if it can help people with iTTP and similar conditions.

What does 'Phase I and Phase II' mean?

This means it's an early-stage study, combining two steps. Phase I looks mainly at safety and finding the right dose, while Phase II starts to look more at how well the medicine works.

Will I get a placebo (dummy treatment)?

The information provided doesn't say if a placebo is used. The study team will be able to tell you if there are different treatment groups and what they involve.

How long will I be in the study?

The exact length of your participation will be explained by the study team. It typically involves receiving the drug followed by a period of careful follow-up to see how you're doing.