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
- Are there results?
- Not yet: the study is under way
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
Show all 12Show fewer
- 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.
Study at a glance
From the registry record, last updated by the sponsor on 29 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT07459114
- Acronym / sponsor ID
- TG1-CL-301
- Keywords
- iTTP, TMA, Thrombotic Microangiopathies, immune-mediated thrombotic thrombocytic purplura, thrombotic microangiopathy, TGD001
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 70 participants (estimated)
- Lead sponsor
- TargED Biopharmaceuticals B.V.Industry (pharma, biotech, devices)
- Study start
- 31 Oct 2026 (estimated)
- Primary completion
- 31 May 2028 (estimated)
- Study completion
- 30 Aug 2028 (estimated)
- First posted
- 9 Mar 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 7
| Arm | Type | Receives |
|---|---|---|
| Dose Level 1 | Experimental | Drug: TGD001TGD001 IV bolus administration |
| Dose Level 2 | Experimental | Drug: TGD001TGD001 IV bolus administration |
| Dose Level 3 | Experimental | Drug: TGD001TGD001 IV bolus administration |
| Dose Level 4 | Experimental | Drug: TGD001TGD001 IV bolus administration |
| Basket 1 | Experimental | Drug: TGD001TGD001 IV bolus administration |
| Basket 2 | Experimental | Drug: TGD001TGD001 IV bolus administration |
| Basket 3 | Experimental | Drug: TGD001TGD001 IV bolus administration |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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 (12 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TGD001IV bolus administration | Drug | Basket 1, Basket 2, Basket 3, Dose Level 1, Dose Level 2, Dose Level 3, Dose Level 4 |
Primary outcome
Incidence of treatment emergent adverse events (safety and tolerability of TGD001)
Time frame: 90 days
Measurement of treatment emergent adverse events using the Common Terminology Criteria for Adverse Events (CTCAE) criteria
11 secondary outcomes
Change from baseline in lactate dehydrogenase (LDH) and other tissue damage markers
Time frame: 90 days
Change from baseline in platelet count
Time frame: 90 days
Time to resolution of the thrombotic episode
Time frame: 90 days
Change from baseline of disease-related signs and symptoms using CTCAE v5.0 criteria
Time frame: 90 days
Duration of ICU stay and hospitalization
Time frame: 90 days
Occurrence of all-cause and disease-specific mortality
Time frame: 90 days
Peak plasma concentration (Cmax) of TGD001
Time frame: 90 days
Area under the plasma concentration versus time curve (AUC) of TGD001
Time frame: 90 days
Time to maximum TGD001 plasma concentration
Time frame: 90 days
Plasma half-life (t1/2) of TGD001
Time frame: 90 days
Number of participants with ADA
Time frame: 90 days
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Sep 2026.
- Individual participant dataSharing planned: 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Marielle Klein Hesselink, MD study director
TargED
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing TGD001 in people with Thrombotic Microangiopathies and ITP Immune-Mediated Thrombocytopenia, run by TargED Biopharmaceuticals B.V..
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 29 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Melinda Snyder (1 617 233 4057, melinda.snyder@targedbio.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of treatment emergent adverse events (safety and tolerability of TGD001) (time frame: 90 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Sep 2026.