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Not yet recruiting NCT07459114

TGD001 Treatment in Thrombotic Microangiopathies

Phase I / Phase II Interventional Thrombotic Microangiopathies ITP Immune-Mediated Thrombocytopenia

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: TGD001.
Who it may be relevant to
Registry conditions: Thrombotic Microangiopathies, ITP Immune-Mediated Thrombocytopenia. Basic parameters: from 18 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Center list to be confirmed — check the primary protocol.
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

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

Overview

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.

Interventions

  • Drug TGD001
    IV bolus administration

Primary outcome measures

  • Incidence of treatment emergent adverse events (safety and tolerability of TGD001) [Time frame: 90 days]
Secondary outcome measures (11)
  • 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]

Eligibility criteria

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

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Non-randomized
Model
Sequential
Masking
Open label
Primary purpose
Treatment

Study locations

Center list to be confirmed — check the primary protocol.

Identifiers

NCT: NCT07459114 · TG1-CL-301

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗