NCT07459114

Brief 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. The trial is an open-label, dose escalation and expansion basket trial.

Trial Health

65
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
70

participants targeted

Target at P75+ for phase_1

Timeline
27mo left

Started May 2026

Typical duration for phase_1

Status
not yet recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

February 21, 2026

Completed
16 days until next milestone

First Posted

Study publicly available on registry

March 9, 2026

Completed
3 months until next milestone

Study Start

First participant enrolled

May 31, 2026

Expected
2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 31, 2028

3 months until next milestone

Study Completion

Last participant's last visit for all outcomes

August 30, 2028

Last Updated

March 9, 2026

Status Verified

March 1, 2026

Enrollment Period

2 years

First QC Date

February 21, 2026

Last Update Submit

March 6, 2026

Conditions

Thrombotic MicroangiopathiesITP Immune-Mediated Thrombocytopenia

Keywords

iTTPTMAThrombotic Microangiopathiesimmune-mediated thrombotic thrombocytic purplurathrombotic microangiopathyTGD001

Outcome Measures

Primary Outcomes (1)

  • 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 Outcomes (11)

  • Change from baseline in lactate dehydrogenase (LDH) and other tissue damage markers

    90 days

  • Change from baseline in platelet count

    90 days

  • Time to resolution of the thrombotic episode

    90 days

  • Change from baseline of disease-related signs and symptoms using CTCAE v5.0 criteria

    90 days

  • Duration of ICU stay and hospitalization

    90 days

  • +6 more secondary outcomes

Study Arms (7)

Dose Level 1

EXPERIMENTAL

TGD001 IV bolus administration

Drug: TGD001

Dose Level 2

EXPERIMENTAL

TGD001 IV bolus administration

Drug: TGD001

Dose Level 3

EXPERIMENTAL

TGD001 IV bolus administration

Drug: TGD001

Dose Level 4

EXPERIMENTAL

TGD001 IV bolus administration

Drug: TGD001

Basket 1

EXPERIMENTAL

TGD001 IV bolus administration

Drug: TGD001

Basket 2

EXPERIMENTAL

TGD001 IV bolus administration

Drug: TGD001

Basket 3

EXPERIMENTAL

TGD001 IV bolus administration

Drug: TGD001

Interventions

TGD001DRUG

IV bolus administration

Basket 1Basket 2Basket 3Dose Level 1Dose Level 2Dose Level 3Dose Level 4

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

MeSH Terms

Conditions

Thrombotic MicroangiopathiesPurpura, Thrombocytopenic, Idiopathic

Condition Hierarchy (Ancestors)

ThrombocytopeniaBlood Platelet DisordersHematologic DiseasesHemic and Lymphatic DiseasesCytopeniaPurpura, ThrombocytopenicPurpuraBlood Coagulation DisordersHemorrhagic DisordersAutoimmune DiseasesImmune System DiseasesHemorrhagePathologic ProcessesPathological Conditions, Signs and SymptomsSkin ManifestationsSigns and Symptoms

Study Officials

  • Marielle Klein Hesselink, MD

    TargED

    STUDY DIRECTOR

Central Study Contacts

Melinda Snyder

CONTACT

1 617 233 4057melinda.snyder@targedbio.com

Barbara van den Aarsen

CONTACT

31620161487barbara.van.den.aarsen@Targedbio.com

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SEQUENTIAL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 21, 2026

First Posted

March 9, 2026

Study Start (Estimated)

May 31, 2026

Primary Completion (Estimated)

May 31, 2028

Study Completion (Estimated)

August 30, 2028

Last Updated

March 9, 2026

Record last verified: 2026-03

Data Sharing

IPD Sharing
Will not share

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.