Long-Term Follow-Up of TX200-TR101 (STEADFAST Long Term)

• Sponsor: Sangamo Therapeutics (Industry)
• Overall Status: Enrolling by invitation
• CT.gov ID: NCT05987527
• Collaborator: (none)
• Enrollment: 21
• Locations: 6
• Anticipated Duration (Months): 192.7
• Patients Per Site: 3.5
• Patients Per Site Per Month: 0

Study Details

Study Description

Brief Summary

This long-term follow-up study is being conducted to collect long-term (up to 15 years post-infusion) safety and tolerability data from subjects enrolled in studies evaluating TX200-TR101.

Condition or Disease	Intervention/Treatment	Phase
Kidney Transplant Rejection; End Stage Renal Disease	Diagnostic Test: Urine sample; Diagnostic Test: Blood sample; Other: Lifestyle questionnaire

Detailed Description

This is long- term follow up observational trial for patients who participated previously to a multicentre, first-in-human, open-label, single ascending dose, dose-ranging study of autologous, chimeric antigen receptor T regulatory cells (CAR-Treg) in HLA-A2 mismatched living donor kidney transplant recipients, with a control cohort of mismatched kidney transplant recipients of similar immunological risk.

The aim is for this observational long-term follow up study (TX200-KT03) is to collect long-term (up to 15 years post-infusion) safety and tolerability data from subjects enrolled in studies evaluating TX200-TR101. In addition, the long-term effects and durability of the clinical effect of TX200-TR101 in subjects who received TX200-TR101 will be assessed and compared with control participants in the interventional Study (TX200- KT02).

Study Design

• Study Type: Observational
• Anticipated Enrollment: 21 participants
• Observational Model: Case-Control
• Time Perspective: Prospective
• Official Title: 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
• Anticipated Study Start Date: Aug 15, 2023
• Anticipated Primary Completion Date: Sep 4, 2039
• Anticipated Study Completion Date: Sep 4, 2039

Arms and Interventions

Arm	Intervention/Treatment
Treatment group Subjects underwent a kidney transplant as per planned standard of care and were administered study drug TX200-TR101 in study TX200-KT02 post transplantation Assigned interventions - subjects who received TX200-TR101 in clinical study TX200-KT02	Diagnostic Test: Urine sample; Routine procedures; Diagnostic Test: Blood sample; Routine procedures; Other: Lifestyle questionnaire; Lifestyle questionnaire
Control group Subjects underwent a kidney transplant as per planned standard of care in study TX200-KT02 with no study drug administered, up to 6 subjects	Diagnostic Test: Urine sample; Routine procedures; Diagnostic Test: Blood sample; Routine procedures; Other: Lifestyle questionnaire; Lifestyle questionnaire

Outcome Measures

Primary Outcome Measures

1. Safety and Tolerability [Up to 15 years post infusion]

   Long term safety and tolerability of TX200-TR101 infusion evaluated by overall survival

2. Safety and Tolerability [Up to 15 years post infusion]

   Long term safety and tolerability of TX200-TR101 infusion evaluated by and incidence of serious adverse events (SAEs) according to CTCAE V5.0.

Secondary Outcome Measures

1. Long term graft related outcomes [Up to 15 years post infusion]

   Incidence of graft rejection according to the Banff classification criteria

2. Long term safety [Up to 15 years post infusion]

   Number of in-patient days in hospital

3. Long term safety [Up to 15 years post infusion]

   Incidence of Adverse Events related to TX20-TR101

4. Long term safety [Up to 15 years post infusion]

   incidence of Adverse Events of special interest as defined in the protocol

5. Long term safety [Up to 15 years post infusion]

   Incidence of anti-drug antibodies against HLA A2 CAR Tregs

6. Long term safety [Up to 15 years post infusion]

   Change in immunosuppression regime

7. Composite efficacy profile of TX200-TR101 [Up to 15 years post infusion]

   Incidence of graft loss due to rejection

8. Composite efficacy profile of TX200-TR101 [Up to 15 years post infusion]

   Death

9. Composite efficacy profile of TX200-TR101 [Up to 15 years post infusion]

   Incidence of chronic graft dysfunction measured by changes in estimated glomerular filtration rate

10. Impact on Quality of Life [Up to 15 years post infusion]

    SF-36 Questionnaire scaled with lower score meaning more disability

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years to 72 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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.

Exclusion Criteria:

• N/A

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	University Hospitals Leuven	Leuven		Belgium
2	Charité Universitätsmedizin	Berlin		Germany
3	University Medical Center Groningen	Groningen		Netherlands
4	Leiden University Medical Centre	Leiden		Netherlands
5	Erasmus MC, University Medical Center	Rotterdam		Netherlands
6	Oxford University Hospitals NHS Foundation Trust	Oxford		United Kingdom

Sponsors and Collaborators

• Sangamo Therapeutics

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Additional Information:

• Link to parent study STEADFAST, TX200-KT02

Publications

None provided.

• Responsible Party: Sangamo Therapeutics
• ClinicalTrials.gov Identifier: NCT05987527
• Other Study ID Numbers: TX200-KT03; 2022-002440-40
• First Posted: Aug 14, 2023
• Last Update Posted: Aug 14, 2023
• Last Verified: Aug 1, 2023

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No
• Keywords provided by Sangamo Therapeutics: Regulatory T-Cells; Genetically modified cells; Chimeric antigen receptor; Long term follow up; T-Regs; Cell Therapy
• Additional relevant MeSH terms: Kidney Failure, Chronic

Study Results

No Results Posted as of Aug 14, 2023