Thrombotic MicroangiopathiesPhase 1/2NCT07459114
TGD001 Treatment in Thrombotic Microangiopathies
A Phase 1/2 study testing TGD001 in people with Thrombotic Microangiopathies and ITP Immune-Mediated Thrombocytopenia, run by TargED Biopharmaceuticals B.V..
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Thrombotic Microangiopathies, ITP Immune-Mediated Thrombocytopenia
- What is being tested?
- TGD001
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 70 participants
- Where
- No sites listed yet
- Study dates
- 31 Oct 2026to 30 Aug 2028
- Phase
- Phase 1/2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
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- 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
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: 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 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.
Read the detailed description
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.
- Being tested
- TGD001
- Keywords
- iTTP, TMA, Thrombotic Microangiopathies, immune-mediated thrombotic thrombocytic purplura, thrombotic microangiopathy, TGD001
- Medical subject headings
- Thrombotic Microangiopathies, Purpura, Thrombocytopenic, Idiopathic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TGD001Drug
IV bolus administration
| Study group | Role | What they receive |
|---|---|---|
| Dose Level 1 | Experimental | TGD001 IV bolus administration Receives: Drug: TGD001 |
| Dose Level 2 | Experimental | TGD001 IV bolus administration Receives: Drug: TGD001 |
| Dose Level 3 | Experimental | TGD001 IV bolus administration Receives: Drug: TGD001 |
| Dose Level 4 | Experimental | TGD001 IV bolus administration Receives: Drug: TGD001 |
| Basket 1 | Experimental | TGD001 IV bolus administration Receives: Drug: TGD001 |
| Basket 2 | Experimental | TGD001 IV bolus administration Receives: Drug: TGD001 |
| Basket 3 | Experimental | TGD001 IV bolus administration Receives: Drug: TGD001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 90 days
Secondary outcomes (11)
Change from baseline in lactate dehydrogenase (LDH) and other tissue damage markers
Measurement of LDH and other tissue damage markers
Measured: 90 days
Change from baseline in platelet count
Measurements of platelet count from baseline
Measured: 90 days
Time to resolution of the thrombotic episode
Measurement of platelet count and LDH levels
Measured: 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
Measured: 90 days
Duration of ICU stay and hospitalization
Number of days in the ICU and the number of days hospitalized
Measured: 90 days
Occurrence of all-cause and disease-specific mortality
All-cause and disease-specific mortality post-intervention
Measured: 90 days
Peak plasma concentration (Cmax) of TGD001
Plasma concentrations and Cmax of TGD001
Measured: 90 days
Area under the plasma concentration versus time curve (AUC) of TGD001
Plasma concentrations of and AUC of TGD001
Measured: 90 days
Time to maximum TGD001 plasma concentration
Plasma concentrations and Tmax of TGD001
Measured: 90 days
Plasma half-life (t1/2) of TGD001
Plasma concentrations and t1/2 of TGD001
Measured: 90 days
Number of participants with ADA
Development of ADA
Measured: 90 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Mar 2026
- Study start31 Oct 2026 (estimated)
- Primary completion31 May 2028 (estimated)
- Study completion30 Aug 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Thrombotic MicroangiopathiesStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 70 participants (estimated)
Sponsor and investigators
- Lead sponsor
- TargED Biopharmaceuticals B.V.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Marielle Klein Hesselink, MD, Study director, TargED
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07459114
- Sponsor's protocol ID
- TG1-CL-301
- Status last verified
- Sep 2026
- First submitted
- 21 Feb 2026
- Last updated
- 29 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- 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.