Long-Term Follow-Up of Patients who have received an Autologous Antigen-Specific Chimeric Antigen Receptor T Regulatory Cell Therapy (CAR- Treg therapy, TX200-TR101) in a prior clinical study.

Long-Term Follow-Up of Patients who have received an Autologous Antigen-Specific Chimeric Antigen Receptor T Regulatory Cell Therapy (CAR- Treg therapy, TX200-TR101) in a prior clinical study. - Long-Term Follow-Up of TX200-TR101 (STEADFAST Long Term)

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2022-002440-40-DE

Enrollment

Registered

2022-12-20

Start date

2023-03-31

Completion date

Unknown

Last updated

2024-05-13

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Prevention of immune mediated allograft rejection in patients with end-stage renal disease (ESRD) who are tissue typed as HLA-A*02 negative and have received a kidney transplant from an HLA-A*02 positive living donor. MedDRA version: 21.1 Level: LLT Classification code 10050436 Term: Prophylaxis against renal transplant rejection System Organ Class: 10042613 - Surgical and medical procedures

Interventions

Product Name: Autologous HLA-A2 Chimeric Antigen Receptor T regulatory cells Product Code: TX200-TR101 Pharmaceutical Form: Dispersion for infusion INN or Proposed INN: Not yet assigned Current Spons

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. Subjects who enrolled in the Phase I/IIa study TX200-KT02, received a transplanted kidney and have either completed or withdrawn from that study. 2. Willing and able to provide written informed consent (IC) in accordance with local regulations and governing Independent Ethics Committee (IEC)/Institutional Review Board (IRB) requirements prior to any procedure or evaluation performed specifically for the sole purpose of the study. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 10 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 5

Exclusion criteria

Exclusion criteria: 1. Subjects/persons committed to an institution following an administrative or judicial order.

Design outcomes

Primary

Measure	Time frame
Main Objective: To evaluate the long-term safety and tolerability of TX200-TR101 up to 15 years post-TX200-TR101 infusion/baseline. ;Secondary Objective: To evaluate the effect of TX200-TR101 on long-term graft-related outcomes up to 15 years post-TX200-TR101 infusion/baseline. To evaluate the effect of TX200-TR101 on long-term safety outcomes up to 15 years post-TX200-TR101 infusion/baseline. To evaluate the composite efficacy profile of TX200-TR101 up to 15 years post-TX200-TR101 infusion/baseline. To evaluate quality of life over time up to 15 years post-TX200-TR101 infusion/baseline. To evaluate the long-term persistence of CAR-Tregs and impact in the periphery up to 15 years post-TX200-TR101 infusion/baseline (if available).;Primary end point(s): From the day of TX200-TR101 infusion through to 15 years post-TX200-TR101 infusion/baseline: • Overall survival • Incidence and grade of Serious Adverse Events (SAEs).;Timepoint(s) of evaluation of this end point: From the day of TX200-TR101 infusion through to 15 years post-TX200-TR101 infusion/baseline	—

Measure

Time frame

Main Objective: To evaluate the long-term safety and tolerability of TX200-TR101 up to 15 years post-TX200-TR101 infusion/baseline. ;Secondary Objective: To evaluate the effect of TX200-TR101 on long-term graft-related outcomes up to 15 years post-TX200-TR101 infusion/baseline. To evaluate the effect of TX200-TR101 on long-term safety outcomes up to 15 years post-TX200-TR101 infusion/baseline. To evaluate the composite efficacy profile of TX200-TR101 up to 15 years post-TX200-TR101 infusion/baseline. To evaluate quality of life over time up to 15 years post-TX200-TR101 infusion/baseline. To evaluate the long-term persistence of CAR-Tregs and impact in the periphery up to 15 years post-TX200-TR101 infusion/baseline (if available).;Primary end point(s): From the day of TX200-TR101 infusion through to 15 years post-TX200-TR101 infusion/baseline: • Overall survival • Incidence and grade of Serious Adverse Events (SAEs).;Timepoint(s) of evaluation of this end point: From the day of TX200-TR101 infusion through to 15 years post-TX200-TR101 infusion/baseline

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Secondary

Measure	Time frame
Secondary end point(s): • Incidence of immune-mediated rejection in terms of BCAR episodes according to the Banff criteria (including type, severity and timing) • Incidence of graft loss due to rejection • Incidence and severity of chronic graft dysfunction, as measured by eGFR • Incidence and (semi-quantitative) intensity of de novo donor-specific anti-HLA antibodies (DSA). • Number of in-patient days in hospital • Incidence of any Adverse Events (AEs)considered related to TX200-TR101 according to the investigator • Incidence of Adverse Events of Special Interest (AESI), including: o Infections requiring medical intervention, where severity is Grade 2 or greater. Any infection that is suspected or confirmed opportunistic in nature; new-onset diabetes mellitus (NODM), new or aggravation of hypertension, new malignancies, or new (up-to 8 years post-infusion) dyslipidaemia requiring medical intervention • Change in immunosuppression regimen to include: o Target levels in blood for each immunosuppressant. o Number and name of drugs given to achieve intended level of immunosuppression. o Any incidence of rescue medication given to avoid potential imminent rejection episode. • Incidence of anti-drug antibodies against HLA-A2 CAR-Tregs. o Death, or; o Incidence of immune-mediated rejection in terms of BCAR episodes according to the Banff criteria (including type, severity and timing), or; o Incidence of graft loss due to rejection, or; o Incidence and severity of chronic graft dysfunction, as measured by eGFR Absolute value and change from baseline in SF-36v2® Levels (absolute values) and changes from baseline (i.e. Pre- TX200-TR101 infusion/baseline) in biomarkers in blood relating to the presence of HLA-A2 CAR-Tregs;Timepoint(s) of evaluation of this end point: From the day of TX200-TR101 infusion through to 15 years post-TX200-TR101 infusion/baseline	—

Measure

Time frame

Secondary end point(s): • Incidence of immune-mediated rejection in terms of BCAR episodes according to the Banff criteria (including type, severity and timing) • Incidence of graft loss due to rejection • Incidence and severity of chronic graft dysfunction, as measured by eGFR • Incidence and (semi-quantitative) intensity of de novo donor-specific anti-HLA antibodies (DSA). • Number of in-patient days in hospital • Incidence of any Adverse Events (AEs)considered related to TX200-TR101 according to the investigator • Incidence of Adverse Events of Special Interest (AESI), including: o Infections requiring medical intervention, where severity is Grade 2 or greater. Any infection that is suspected or confirmed opportunistic in nature; new-onset diabetes mellitus (NODM), new or aggravation of hypertension, new malignancies, or new (up-to 8 years post-infusion) dyslipidaemia requiring medical intervention • Change in immunosuppression regimen to include: o Target levels in blood for each immunosuppressant. o Number and name of drugs given to achieve intended level of immunosuppression. o Any incidence of rescue medication given to avoid potential imminent rejection episode. • Incidence of anti-drug antibodies against HLA-A2 CAR-Tregs. o Death, or; o Incidence of immune-mediated rejection in terms of BCAR episodes according to the Banff criteria (including type, severity and timing), or; o Incidence of graft loss due to rejection, or; o Incidence and severity of chronic graft dysfunction, as measured by eGFR Absolute value and change from baseline in SF-36v2® Levels (absolute values) and changes from baseline (i.e. Pre- TX200-TR101 infusion/baseline) in biomarkers in blood relating to the presence of HLA-A2 CAR-Tregs;Timepoint(s) of evaluation of this end point: From the day of TX200-TR101 infusion through to 15 years post-TX200-TR101 infusion/baseline

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Countries

Belgium, Germany, Netherlands, United Kingdom

Contacts

Public ContactTX200-KT03 Information Desk

Sangamo Therapeutics France SAS

smb-tx200-kt03@sangamo.com

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026