Donor Regulatory T-cells for cGVHD in Patients Who do Not Obtain Complete Remission With Ruxolitinib

NCT05095649 | Recruiting Phase 2

• 1 other identifier: Treg4GVHD
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

Brief Summary

Phase II clinical trial to assess the efficacy of donor regulatory enriched T cells in steroid-refractory chronic graft versus host disease patients who did not obtain complete remission under treatment with ruxolitinib

Conditions

Chronic Graft vs Host Disease

Keywords

Ruxolitinib; T cells

Outcome Measures

Primary Outcomes (3)

• Number of Participants with overall response rate.

  Obtain ≥65% the overall response rate at 6 months after infusion

  6 months post-infusion

• Number of Participants with overall response rate.

  Obtain ≥75% the overall response rate at 1 year after infusion

  1 year post-infusion

• Survival

  Number of patients who survive after Regulatory T-cell enriched infusion

  1 year after Regulatory T-cell enriched infusion

Secondary Outcomes (14)

• Disease evaluation through Symptoms of the disease

  Screening, weeks 1, 2, 4, 6, 12 and months 6, 9 and 12 months after infusion

• Disease evaluation through measurement of quality of life

  Screening, weeks 1, 2, 4, 6, 12 and months 6, 9 and 12 months after infusion

• Disease evaluation through Symptoms of the disease

  Screening, weeks 1, 2, 4, 6, 12 and months 6, 9 and 12 months after infusion

• Immunosuppressive requirements.

  Screening, month1, months 3, 6, and 12 after infusion

• Free survival

  1 year after infusion.

Study Arms (1)

Regulatory T-cell enriched infusion

EXPERIMENTAL

The doses of Regulatory T-cell enriched infusion will be 2x10\^6 cells/kg

Biological: Regulatory T-cell enriched infusion

Interventions

Regulatory T-cell enriched infusion BIOLOGICAL

Enrichment of cluster of differentiation 25hi regulatory T cells from cluster of differentiation antigen 8 and/or cluster of differentiation antigen19 pre-depleted leukapheresis products.

Regulatory T-cell enriched infusion

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Recipient of allogeneic hematopoietic stem cell transplantation
• Participants must have steroid-refractory cGVHD and had obtained any response other than progression after at least 12 weeks of treatment with ruxolitinib. Steroid-refractory cGVHD is defined as having persistent signs and symptoms of cGVHD despite the use of prednisone at ≥ 0.25 mg/kg/day (or 0.5 mg/kg every other day) for at least 4 weeks (or equivalent dosing of alternate glucocorticoids) without complete resolution of signs and symptoms.
• Stable dose of glucocorticoids for 4 weeks prior to enrollment.
• No addition or subtraction of other immunosuppressive medications (e.g., calcineurin-inhibitors, sirolimus, mycophenolate-mofetil) for 4 weeks prior to enrollment. The dose of immunosuppressive medicines may be adjusted based on the therapeutic range of that drug.
• No age limit. In the case of children participating in the study, the informed consent will be signed by a parents or legal guardians.
• Eastern Cooperative Oncology Group scale performance status 0-2
• Participants must have adequate organ function
• Women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately.
• Ability to understand and the willingness to sign a written informed consent document

You may not qualify if:

• Ongoing prednisone requirement \>1 mg/kg/day (or equivalent).
• Concurrent use of calcineurin-inhibitor plus sirolimus (either agent alone is acceptable).
• History of active thrombotic microangiopathy, hemolytic-uremic syndrome or thrombotic thrombocytopenic purpura in the last 6 months.
• New immunosuppressive medication in the 4 weeks prior to enrollment.
• Extra-corporeal Photopheresis or rituximab therapy in the 4 weeks prior to enrollment.
• Post-transplant exposure to T-cell or interleukin-2 targeted medication within 100 days prior to enrollment.
• Donor lymphocyte infusion within 100 days prior to enrollment.
• Active malignant relapse.
• Active uncontrolled infection.
• Organ transplant (allograft) recipient.
• HIV-positive individuals on combination antiretroviral therapy are ineligible.
• Individuals with active uncontrolled hepatitis B or C are ineligible as they are at high risk of lethal treatment-related hepatotoxicity after hematopoietic stem cell transplant.
• Other investigational drugs within 4 weeks prior to enrollment, unless cleared by the Principal Investigator.
• Pregnant women are excluded from this study.

Sponsors & Collaborators

• Fundación Pública Andaluza para la gestión de la Investigación en Sevilla: lead

Study Sites (1)

José Antonio Pérez Simón

Seville, 41011, Spain

RECRUITING

Study Officials

• José Antonio Pérez-Simón, M.D. Ph.D

  Department of Hematology, Hospital Universitario Virgen del Rocío, Sevilla.

  PRINCIPAL INVESTIGATOR

Central Study Contacts

José Antonio Pérez-Simón, M.D. Ph.D

CONTACT

955013414; josea.perez.simon.sspa@juntadeandalucia.es

Clara M. Rosso, M.D. Ph.D

CONTACT

955013414; claram.rosso.sspa@juntadeandalucia.es

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: The doses of Treg-enriched cells will be 2x10\^6 cells/kg

Study Record Dates

• First Submitted: May 26, 2021
• First Posted: October 27, 2021
• Study Start: March 24, 2022
• Primary Completion: August 15, 2025
• Study Completion: February 15, 2026
• Last Updated: March 26, 2024

Record last verified: 2024-03

Data Sharing

• IPD Sharing: Will not share

Locations

ES (1)