TGD001 Treatment in Thrombotic Microangiopathies

Overview

Sponsor-declared trial summary

Key facts

Sponsor

TargED Biopharmaceuticals B.V.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Hemic and Lymphatic Diseases [C15]

Decision date (initial)

2026-03-11

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety

To assess the safety and tolerability of TGD001 in participants with suspicion or clinical diagnosis of an acute iTTP episode

Secondary objectives 8

1. To assess the time to clinical response
2. To assess changes in platelet count
3. To assess changes in organ damage markers
4. To assess changes in clinical symptoms and organ function until Day 90
5. To assess the duration of ICU admission and hospitalization
6. To assess all-cause and disease-specific mortality until Day 90
7. To determine the PK characteristics of TGD001
8. To assess the immunogenicity of TGD001

Conditions and MedDRA coding

Immune-Mediated Thrombotic Thrombocytopenic Purpura

Regulatory references

Scientific advice from competent authorities

Federal Agency For Medicines And Health Products

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

1. Adult: age ≥18 at the time of signing the informed consent form (ICF)
2. Willing and able to provide informed consent and sign the ICF
3. 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
4. Symptomatic acute TMA episode
5. Patient accessible to follow-up

Exclusion criteria 13

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

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Treatment emergent adverse events

Secondary endpoints 10

1. Time to clinical response
2. Change in platelet count from baseline
3. Time to recovery of platelets to ≥150×10E9/L
4. Change of organ damage markers from baseline
5. Time to recovery of LDH to ≤2 × ULN
6. 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)
7. Number of days in the intensive care unit and the number of days hospitalized
8. All-cause and disease-specific mortality within 90 days post-intervention
9. Plasma concentrations of TGD001, PK parameters such as Cmax, Tmax, AUC, t1/2
10. Development of ADA

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

TargED Biopharmaceuticals B.V.

Sponsor organisation

TargED Biopharmaceuticals B.V.

Address

Princetonlaan 6

City

Utrecht

Postcode

3584 CB

Country

Netherlands

Scientific contact point

Organisation

TargED Biopharmaceuticals B.V.

Contact name

Marielle Hesselink

Public contact point

Organisation

TargED Biopharmaceuticals B.V.

Contact name

Marielle Hesselink

Third parties 1

Locations

4 EU/EEA countries · 4 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 22 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

1 submissions — initial application plus substantial / non-substantial modifications