A Phase 1/2 interventional study of TGD001 in Thrombotic Microangiopathies and ITP Immune-Mediated Thrombocytopenia, sponsored by TargED Biopharmaceuticals B.V.. Not yet recruiting. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-09-29.
Sponsored by TargED Biopharmaceuticals B.V. · Phase 1/2, Interventional, and Treatment
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.
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.
52 studies on the registry are indexed under Thrombotic Microangiopathies; 21 are open to participants now.
This study's planned enrollment of 70 is above the median of 40 across 24 interventional studies indexed under Thrombotic Microangiopathies.
Browse Thrombotic Microangiopathies studies →This is the only study on the registry with TargED Biopharmaceuticals B.V. as lead sponsor.
Counted across the registry records on this site, refreshed daily.
Key Inclusion Criteria:
Key Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
After a transplant from a donor, the donor’s immune cells attack the recipient’s body, often the skin, gut or liver.
The study doctor’s estimate of how long someone is likely to live. Criteria use it so that people can stay in the study long enough for it to answer its question.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GVHD (graft-versus-host disease)
After a transplant from a donor, the donor’s immune cells attack the recipient’s body, often the skin, gut or liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Life expectancy
The study doctor’s estimate of how long someone is likely to live. Criteria use it so that people can stay in the study long enough for it to answer its question.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
TGD001 IV bolus administration
Drug: TGD001
TGD001 IV bolus administration
Drug: TGD001
TGD001 IV bolus administration
Drug: TGD001
TGD001 IV bolus administration
Drug: TGD001
TGD001 IV bolus administration
Drug: TGD001
TGD001 IV bolus administration
Drug: TGD001
TGD001 IV bolus administration
Drug: TGD001
IV bolus administration
Incidence of treatment emergent adverse events (safety and tolerability of TGD001)
Measurement of treatment emergent adverse events using the Common Terminology Criteria for Adverse Events (CTCAE) criteria
Time frame: 90 days
Change from baseline in lactate dehydrogenase (LDH) and other tissue damage markers
Measurement of LDH and other tissue damage markers
Time frame: 90 days
Change from baseline in platelet count
Measurements of platelet count from baseline
Time frame: 90 days
Time to resolution of the thrombotic episode
Measurement of platelet count and LDH levels
Time frame: 90 days
Change from baseline of disease-related signs and symptoms using CTCAE v5.0 criteria
Assessment or improvement of disease related signs and symptoms
Time frame: 90 days
Duration of ICU stay and hospitalization
Number of days in the ICU and the number of days hospitalized
Time frame: 90 days
Occurrence of all-cause and disease-specific mortality
All-cause and disease-specific mortality post-intervention
Time frame: 90 days
Peak plasma concentration (Cmax) of TGD001
Plasma concentrations and Cmax of TGD001
Time frame: 90 days
Area under the plasma concentration versus time curve (AUC) of TGD001
Plasma concentrations of and AUC of TGD001
Time frame: 90 days
Time to maximum TGD001 plasma concentration
Plasma concentrations and Tmax of TGD001
Time frame: 90 days
Plasma half-life (t1/2) of TGD001
Plasma concentrations and t1/2 of TGD001
Time frame: 90 days
Number of participants with ADA
Development of ADA
Time frame: 90 days
No study locations are listed for this record.
Plan to share: No — IPD will not be shared due to the small sample size in this early phase clinical trial in a rare patient population. Such a small sample size would pose a significant risk of re-identification of study participants.
No publications or documents are linked to this record.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
This study is not yet recruiting, as verified in Sep 2026. You cannot join it, but the record below documents what was studied.
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Thrombotic Microangiopathies →