Who may be eligible for the TGD001 Treatment in Thrombotic Microangiopathies trial?

Key Inclusion Criteria: * 18 years old or older * Willing to sign an informed consent form * Willing to refrain from sexual intercourse or must use a contraceptive method that is highly effective for 90 days after the last dose of TGD001 * Symptomatic acute TMA episode * Accessible to follow-up Key Exclusion Criteria: * Diagnosis other than TMA, which could account for the findings of thrombocytopenia and hemolytic anemia * Diagnosis of Shiga-toxin induced HUS * Known bone marrow/graft failure * Diagnosis of ongoing severe, uncontrolled Graft versus Host Disease * Received therapeutic plasma exchange (PEX) within 7 days prior to screening * Use of an anticoagulant and/or thrombolytics * Active internal bleeding * Any major bleeding episode within the past 30 days, or diagnosis of chronic bleeding conditions * Known gastrointestinal ulcer * Severe, uncontrolled hypertension, renal impairment requiring dialysis, or liver impairment, or active infection indicated by sepsis * Life expectancy less than 3 months independent of TMA disorder * Pregnant or breastfeeding

ClinicalTrials.gov record NCT07459114

TGD001 Treatment in Thrombotic Microangiopathies

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Jul 27, 2026

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

About this page. This page shows the study's official ClinicalTrials.gov record ↗, with added help to make it easier to understand. We explain medical terms, highlight key details in plain language, and suggest questions you may want to ask the study team.

The record is periodically copied from ClinicalTrials.gov and may lag behind changes. AI summaries are checked by software, not a clinician.Read moreShow less

The official record is written by the study sponsor and published on ClinicalTrials.gov. It may not always reflect the latest changes. Before making any decisions, please confirm important information with the study team and your own healthcare team.

Where you see "AI summary", AI was used to create a plain-language explanation and software checked it against the source. It has not been reviewed by a clinician, so it may contain mistakes or leave out important details.

StatusNot yet recruitingThe study has been registered but has not started enrolling. Sites and contacts may not be listed yet.

Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.

PhasePhase 1/2A study that combines safety and dose-finding (phase 1) with a first look at whether the treatment has an effect (phase 2).

Age18 years old and older

SexAll

AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This is an open-label Phase 1/2 trial testing TGD001 given by IV bolus alongside standard care for people with suspected or clinically diagnosed acute immune thrombotic thrombocytopenic purpura (iTTP) or other acute thrombotic microangiopathies (TMAs). Part A is dose escalation to identify tolerated dose(s) and pharmacologic response. Part B is a basket-style expansion where cohorts of participants with iTTP and other TMAs receive single or repeat doses; baskets are planned up to ~20 participants and may be combined or expanded up to about 60 in Part B.

What it is studying. The study is testing the safety and tolerability of TGD001, its pharmacology (plasma levels and related measures), and whether specific dose(s) and regimens produce clinical effects on blood markers, platelet counts, and resolution of thrombotic episodes in iTTP and other TMAs.

What the researchers hope to learn. To identify tolerated dose(s) and dosing regimen(s) of TGD001; to characterize safety and treatment-emergent adverse events; to measure pharmacokinetics (Cmax, AUC, Tmax, half-life) and immunogenicity (anti-drug antibodies); and to assess effects on LDH, platelet count, time to thrombotic episode resolution, ICU/hospital stay, and mortality.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

This is a Phase 1/2, prospective, adaptive design trial of TGD001 in participants with suspicion or clinical diagnosis of acute immune thrombotic thrombocytopenic purpura (iTTP) episodes and participants with suspicion or clinical diagnosis of an acute Thrombotic Microangiopathy (TMA) episode. The trial is an open-label, dose escalation and expansion basket trial.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug: TGD001

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

Inclusion criteria

18 years old or older
Willing to sign an informed consent form
Willing to refrain from sexual intercourse or must use a contraceptive method that is highly effective for 90 days after the last dose of TGD001
Symptomatic acute TMA episode
Accessible to follow-up
Key

Exclusion criteria

Diagnosis other than TMA, which could account for the findings of thrombocytopenia and hemolytic anemia
Diagnosis of Shiga-toxin induced HUS
Known bone marrow/graft failure
Diagnosis of ongoing severe, uncontrolled Graft versus Host Disease
Received therapeutic plasma exchange (PEX) within 7 days prior to screening
Use of an anticoagulant and/or thrombolytics
Active internal bleeding
Any major bleeding episode within the past 30 days, or diagnosis of chronic bleeding conditions
Known gastrointestinal ulcer
Severe, uncontrolled hypertension, renal impairment requiring dialysis, or liver impairment, or active infection indicated by sepsis
Life expectancy less than 3 months independent of TMA disorder
Pregnant or breastfeeding

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Detailed description from the registry

In Part A, dose escalation of TGD001 is conducted in participants with suspicion or clinical diagnosis of immune thrombotic thrombocytopenic purpura (iTTP) in conjunction with their respective standard of care to identify a tolerated dose(s) with a pharmacological/clinical response to be evaluated in Part B. Once a recommended dose of TGD001 is established in Part A, the dose expansion/basket part of the trial will commence. In Part B, "basket" cohorts of participants with iTTP and other Thrombotic Microangiopathies (TMAs) will receive single or repeat doses of TGD001 in conjunction with their respective standard of care to determine the TGD001 dose and treatment regimen with clinical effect in reducing the initial thrombus burden. Baskets will include up to 20 participants each and may be combined based on emerging safety and efficacy findings. Baskets may be further expanded with the TGD001 dose(s) and regimen(s) that displayed the best clinical response as assessed by the Data Safety Management Committee up to a total of approximately 60 participants in Part B.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants receive TGD001 by IV bolus (single or repeat doses) alongside their usual standard of care in a dose‑escalation phase followed by dose‑expansion "basket" cohorts. The study includes safety monitoring and testing over a 90‑day period, with blood tests to track platelet count, LDH and other tissue‑damage markers, measurements of drug levels (pharmacokinetics) and checks for immune responses to the drug. Hospitalization and ICU duration are recorded as outcomes. All participants must sign informed consent and follow the stated contraception requirements.

How the study is structured. An open-label, non-randomized Phase 1/2 trial with adaptive, sequential dose escalation (Part A) followed by a basket-style dose expansion (Part B). Participants receive TGD001 by IV bolus together with their usual standard of care. Part B enrolls disease-specific cohorts (baskets) that get single or repeat doses; cohorts start around 20 participants and can be combined or expanded based on safety and response. No masking (participants and researchers know the treatment).

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

This is an early-phase study, mainly about safety and dose. What is already known about this treatment?
The registry says recruitment has not started. When is it expected to open, and can I register interest now?
No sites are listed on the registry yet. Where will the study take place?
Which of the eligibility criteria would need to be checked with tests before I could join?

More on thrombotic thrombocytopenic purpura

Clinical trials18 trials listed for thrombotic thrombocytopenic purpuraEvery trial the registry lists for thrombotic thrombocytopenic purpura, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in thrombotic thrombocytopenic purpura researchLatest monthly update: September 2026. Includes: Caplacizumab use. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start: Oct 31, 2026 (estimated)
Primary completion: May 31, 2028 (estimated)
Study completion: Aug 30, 2028 (estimated)
Target enrollment: 70 participants (estimated)
Last updated on registry: Sep 29, 2026
Data verified: Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor: TargED Biopharmaceuticals B.V.

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Melinda Snyder 📞 1 617 233 4057 ✉ melinda.snyder@targedbio.com
Barbara van den Aarsen 📞 31620161487 ✉ barbara.van.den.aarsen@Targedbio.com

Participating sites

Study locations have not been posted on ClinicalTrials.gov yet. This study is not yet recruiting, so sites may be added when recruitment begins. The central study contact above may be able to tell you about planned sites.

Terms used on this page · explanations by MedicalUpdates.ai

open-label: No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
dose escalation: Groups of participants receive increasing doses of a treatment, one group after another, to find a dose that can be given safely.
informed consent form: The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
follow-up: The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.
prior to screening: The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
Life expectancy: Some studies require that, in the investigator's judgement, a participant is likely to live long enough to complete the study period. It is a study requirement, not a prediction about any individual.
Key Inclusion Criteria: The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Key Exclusion Criteria: The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
standard of care: Treatment generally considered appropriate for a condition in a particular clinical setting. It can vary by patient, place and over time. In many studies the new treatment is added to, or compared with, standard of care.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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