First Clinical Trial with activated regulatory T-cells in patients early after haematopoietic stem cell transplantation (HSCT)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

ActiTrexx GmbH

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Neoplasms [C04]

Trial duration

13 Dec 2023 → 14 Jan 2026

Decision date (initial)

2024-10-02

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Research Private equity · Federal Ministery of Education

External identifiers

EU CT number

2024-516599-14-00

EudraCT number

2021-006490-32

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Prophylaxis, Others, Safety, Dose response

To assess the safety and tolerability of ATreg

Secondary objectives 5

1. To evaluate the feasibility of generating ATreg
2. To assess the frequency and severity of acute GvHD in ATreg treated patients within the first 100 days after HSCT (day 90 after ATreg application)
3. To assess HSCT engraftment after ATreg application (frequency of graft failure)
4. To determine the non- relapse mortality in ATreg treated patients
5. To evaluate long term follow up after ATreg application

Conditions and MedDRA coding

Leukaemia

Regulatory references

Scientific advice from competent authorities

Paul-Ehrlich-Institut

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 7

1. Adults aged >= 18years, any gender (m/f/d)
2. Willing and able to provide written informed consent
3. Healthy 3rd party donor available
4. Meets all criteria for a HSCT and the HSCT (source either peripheral blood or bone marrow, any HLA status, any AB0 blood group status) is planned
5. Patients with HLA compatible HSCT (10/10 or 9/10 matched donor)
6. Hematologic malignancy such as AML, MDS, ALL, MM, MPN, lymphoma (B-NHL and Hodgkin lymphoma) that needs HSCT
7. Has a life expectancy of >6 weeks

Exclusion criteria 13

1. Patients after haploidentical HSCT
2. Is a pregnant or nursing woman. Sexually active women with childbearing potential as well as sexually active male patients who are unwilling to use an effective form of contraception during participation in the study from time of inclusion until 2 months after ATreg therapy
3. Is not willing to comply with all clinical study procedures
4. Has any other known medical condition which is considered to be clinically significant, which in the opinion of the Investigator would affect study participation, the performance or interpretation of study assessments or make the patient incapable to understand the nature, scope, and possible consequences of the study
5. Has had any other malignancy within the last 3 years
6. Is housed in an institution under a court or official order
7. Has a severe uncontrolled infection (Investigator decision)
8. Has a HIV infection or an active HBV or HCV infection
9. Has participated in another investigational study within 14 days of inclusion
10. Has been treated with prednisolone at >2 mg/kg/day (or equivalent dosing of alternative glucocorticosteroids) at time of ATreg infusion
11. Has a clinical manifestation of acute GvHD (any grade)
12. Has known contraindications to the protocol specified treatments/medication
13. Has known allergy/hypersensitivity to any component of the study product

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Type, incidence, and severity of ATreg-related Serious Adverse Events (SAEs) according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI)-(CTCAE) version 5.0 2017 within 24 h, 3 days and 7 days as well as 14 days after ATreg application

Secondary endpoints 9

1. Incidence of failed ATreg preparations as characterised in the release criteria
2. Incidence and severity of acute GvHD (Grades II-IV) in ATreg treated patients at days 28, 56 and 90 after ATreg application (day 100 after HSCT)
3. Incidence of severe infections at day 14 after ATreg application
4. Incidence of relapse at days 28, 56 and 90 after ATreg application (day 100 after HSCT)
5. Change from baseline in neutrophils, platelets, and haemoglobin at day 14 after ATreg application to assess engraftment
6. Incidence of mortality not based on relapse at day 90 after ATreg application (day 100 after HSCT)
7. Frequency and severity of GvHD at 6 months after ATreg application
8. Incidence of relapse of the underlying hematologic malignancy at 6 months after ATreg application
9. Number of systemic infections requiring systemic antibacterial, antiviral or antifungal treatment at 6 months after ATreg application

Investigational products

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

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

ActiTrexx GmbH

Sponsor organisation

ActiTrexx GmbH

Address

Langenbeckstrasse 1, Oberstadt Oberstadt

City

Mainz

Postcode

55131

Country

Germany

Scientific contact point

Organisation

ActiTrexx GmbH

Contact name

Management ActiTrexx

Public contact point

Organisation

ActiTrexx GmbH

Contact name

Management ActiTrexx

Locations

1 EU/EEA country · 4 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

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

Documents 9 files

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

Application history

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