UROKINASE, CATALYTIC DOMAIN, FUSED WITH A SINGLE-CHAIN ANTIBODY AGAINST VON WILLEBRAND FACTOR for Immune-mediated thrombotic thrombocytopenic purpura | Thrombotic microangiopathy

Inclusion criteria

• {"criterion_text":"- Adult: age ≥18 at the time of signing the informed consent form (ICF)"}
• {"criterion_text":"- Willing and able to provide informed consent and sign the ICF"}
• {"criterion_text":"- Male or female of childbearing potential must refrain from sexual intercourse or must use a contraceptive method that is highly effective (with a failure rate of <1% per year) during the trial and for at least 90 days after the last dose of trial drug"}
• {"criterion_text":"- Symptomatic acute TMA episode"}
• {"criterion_text":"- Patient accessible to follow-up"}

Exclusion criteria

• {"criterion_text":"- Diagnosis other than TMA, which could account for the findings of thrombocytopenia and hemolytic anemia (e.g., disseminated intravascular coagulation [DIC], Evans syndrome)"}
• {"criterion_text":"- Pre-existing severe medical, neurological, or psychiatric disease that would significantly confound the endpoint evaluations (Investigator judgment)"}
• {"criterion_text":"- Known hypersensitivity to any of the trial treatments or their excipients or to drugs of similar chemical classes"}
• {"criterion_text":"- Participant participating in a trial involving an investigational drug or device within the last 30 days that would impact this trial"}
• {"criterion_text":"- Participants who cannot comply with trial protocol requirements and procedures"}
• {"criterion_text":"- Severe active infection indicated by sepsis (requirement for pressors with or without positive blood cultures)"}
• {"criterion_text":"- Pregnant or breastfeeding"}
• {"criterion_text":"- History of bleeding diathesis or evidence of active abnormal bleeding within the previous 30 days"}
• {"criterion_text":"- Active internal bleeding"}
• {"criterion_text":"- Indication of symptomatic intracranial hemorrhage (sICH)"}
• {"criterion_text":"- Severe, uncontrolled hypertension (systolic blood pressure >180 mmHg or diastolic blood pressure >110 mmHg)"}
• {"criterion_text":"- Disseminated malignancy or other co-morbid illness limiting life expectancy <3 months independent of the TMA disorder"}
• {"criterion_text":"- Recent major surgery (Investigator judgment)"}

Primary endpoints

• {"endpoint_text":"- Treatment emergent adverse events","definition_or_measurement_approach":""}

Secondary endpoints

• {"endpoint_text":"- Time to clinical response","definition_or_measurement_approach":"Time-to-event measure (time from intervention to clinical response) as stated"}
• {"endpoint_text":"- Change in platelet count from baseline","definition_or_measurement_approach":"Change from baseline platelet count"}
• {"endpoint_text":"- Time to recovery of platelets to ≥150×10E9/L","definition_or_measurement_approach":"Time-to-event: time until platelet count reaches ≥150×10^9/L"}
• {"endpoint_text":"- Change of organ damage markers from baseline","definition_or_measurement_approach":"Change from baseline in organ damage biomarkers"}
• {"endpoint_text":"- Time to recovery of LDH to ≤2 × ULN","definition_or_measurement_approach":"Time-to-event: time until LDH ≤2 × upper limit of normal"}
• {"endpoint_text":"- Assessment or improvement (improvement of ≥1 grade in CTCAE v5.0 scale) of disease-related signs and symptoms within 90 days post intervention (by number of unique participants and by total number of AEs)","definition_or_measurement_approach":"Improvement defined as ≥1 grade improvement on CTCAE v5.0 within 90 days post intervention; reported by number of participants and total AEs"}
• {"endpoint_text":"- Number of days in the intensive care unit and the number of days hospitalized","definition_or_measurement_approach":"Count of ICU days and total hospitalization days"}
• {"endpoint_text":"- All-cause and disease-specific mortality within 90 days post-intervention","definition_or_measurement_approach":"Mortality (all-cause and disease-specific) assessed up to 90 days post-intervention"}
• {"endpoint_text":"- Plasma concentrations of TGD001, PK parameters such as Cmax, Tmax, AUC, t1/2","definition_or_measurement_approach":"Pharmacokinetic parameters: plasma concentrations and derived PK parameters (Cmax, Tmax, AUC, t1/2)"}
• {"endpoint_text":"- Development of ADA","definition_or_measurement_approach":"Assessment of anti-drug antibodies (ADA) development"}

France

Earliest CTIS Part Ii Submission Date

26-01-2026

Latest Decision Or Authorization Date

11-03-2026

Processing Time Days

44

Number Of Sites

1

Number Of Participants

10

Germany

Earliest CTIS Part Ii Submission Date

18-02-2026

Latest Decision Or Authorization Date

06-03-2026

Processing Time Days

16

Number Of Sites

1

Number Of Participants

10

Italy

Earliest CTIS Part Ii Submission Date

19-11-2025

Latest Decision Or Authorization Date

10-03-2026

Processing Time Days

111

Number Of Sites

1

Number Of Participants

10

Spain

Earliest CTIS Part Ii Submission Date

07-01-2026

Latest Decision Or Authorization Date

09-03-2026

Processing Time Days

61

Number Of Sites

1

Number Of Participants

10

Primary sponsor

Full Name

TargED Biopharmaceuticals B.V.

Organisation Type

Pharmaceutical company

Country Of Registered Address

Netherlands

Contract research organisations

Name

Prime Vigilance

Responsibilities

sponsorDuties code 8, contact info provided (phone and email)

Third parties

• {"country":"United Kingdom","full_name":"Prime Vigilance","duties_or_roles":"sponsorDuties code 8","organisation_type":"Health care"}