TGD001 Treatment in Thrombotic Microangiopathies

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.

Study Overview

• Status: Not yet recruiting
• Conditions: Thrombotic Microangiopathies; ITP Immune-Mediated Thrombocytopenia
• Intervention / Treatment: Drug: TGD001

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 Type: Interventional
• Enrollment (Estimated): 70
• Phase: Phase 2; Phase 1

Contacts and Locations

• Study Contact: Name: Melinda Snyder; Phone Number: 1 617 233 4057; Email: melinda.snyder@targedbio.com
• Study Contact Backup: Name: Barbara van den Aarsen; Phone Number: 31620161487; Email: barbara.van.den.aarsen@Targedbio.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

7

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Dose Level 1; TGD001 IV bolus administration	Drug: TGD001; IV bolus administration
Experimental: Dose Level 2; TGD001 IV bolus administration	Drug: TGD001; IV bolus administration
Experimental: Dose Level 3; TGD001 IV bolus administration	Drug: TGD001; IV bolus administration
Experimental: Dose Level 4; TGD001 IV bolus administration	Drug: TGD001; IV bolus administration
Experimental: Basket 1; TGD001 IV bolus administration	Drug: TGD001; IV bolus administration
Experimental: Basket 2; TGD001 IV bolus administration	Drug: TGD001; IV bolus administration
Experimental: Basket 3; TGD001 IV bolus administration	Drug: TGD001; IV bolus administration

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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	90 days

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change from baseline in lactate dehydrogenase (LDH) and other tissue damage markers	Measurement of LDH and other tissue damage markers	90 days
Change from baseline in platelet count	Measurements of platelet count from baseline	90 days
Time to resolution of the thrombotic episode	Measurement of platelet count and LDH levels	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	90 days
Duration of ICU stay and hospitalization	Number of days in the ICU and the number of days hospitalized	90 days
Occurrence of all-cause and disease-specific mortality	All-cause and disease-specific mortality post-intervention	90 days
Peak plasma concentration (Cmax) of TGD001	Plasma concentrations and Cmax of TGD001	90 days
Area under the plasma concentration versus time curve (AUC) of TGD001	Plasma concentrations of and AUC of TGD001	90 days
Time to maximum TGD001 plasma concentration	Plasma concentrations and Tmax of TGD001	90 days
Plasma half-life (t1/2) of TGD001	Plasma concentrations and t1/2 of TGD001	90 days
Number of participants with ADA	Development of ADA	90 days

Collaborators and Investigators

• Sponsor: TargED Biopharmaceuticals B.V.
• Investigators: Study Director: Marielle Klein Hesselink, MD, TargED

Study record dates

Study Major Dates

• Study Start (Estimated): May 31, 2026
• Primary Completion (Estimated): May 31, 2028
• Study Completion (Estimated): August 30, 2028

Study Registration Dates

• First Submitted: February 21, 2026
• First Submitted That Met QC Criteria: March 6, 2026
• First Posted (Actual): March 9, 2026

Study Record Updates

• Last Update Posted (Actual): March 9, 2026
• Last Update Submitted That Met QC Criteria: March 6, 2026
• Last Verified: March 1, 2026

More Information

Terms related to this study

• Keywords: Thrombotic Microangiopathies; TMA; thrombotic microangiopathy; iTTP; immune-mediated thrombotic thrombocytic purplura; TGD001
• Additional Relevant MeSH Terms: Cytopenia; Pathologic Processes; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Skin Manifestations; Hematologic Diseases; Blood Coagulation Disorders; Hemorrhagic Disorders; Blood Platelet Disorders; Purpura, Thrombocytopenic; Purpura; Thrombocytopenia; Pathological Conditions, Signs and Symptoms; Signs and Symptoms; Hemic and Lymphatic Diseases; Purpura, Thrombocytopenic, Idiopathic; Thrombotic Microangiopathies
• Other Study ID Numbers: TG1-CL-301

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: 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.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No