Actively Recruiting

Phase 1
Age: 18Years - 75Years
All Genders
NCT04678401

IS-free Treg HaploHCT

Led by Dana-Farber Cancer Institute · Updated on 2025-10-20

30

Participants Needed

1

Research Sites

450 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

This research study is evaluating the safety and efficacy of the IS-free Treg-cell graft-engineered haplo transplant method in people with relapsed/refractory and Ultra-high risk acute myeloid leukemia (AML) and/or myelodysplastic syndromes (MDS) receiving a haploidentical donor allogeneic hematopoietic stem cell transplant (HSCT). The names of the study interventions involved in this study are: * Radiation-Total Myeloid and Lymphoid Irradiation (TMLI) * Chemotherapy (Fludarabine, Thiotepa, Cyclophosphamide plus Mesna) * Infusion of haplo Treg-enriched donor cells (experimental therapy) * Infusion of unmodified haplo donor T cells (includes cancer-fighting T effector cells) * Infusion of haplo donor CD34+ Peripheral Blood Stem Cells

CONDITIONS

Official Title

IS-free Treg HaploHCT

Who Can Participate

Age: 18Years - 75Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Histologically confirmed relapsed or refractory AML with \u22655% blasts in bone marrow or myelodysplastic syndrome EB-2 with 10% or more blasts in bone marrow
  • Ultra high-risk AML or MDS with mutated TP53 as defined by 2022 International Consensus Classification
  • Ultra high-risk AML or MDS with multi-hit or complex karyotype mutated TP53 with response and low blast counts
  • Available haploidentical HLA-matched related donor aged 18 to 65 years
  • Age 18 to 65 years for cohorts A and B; age 18 to 75 years for cohort C
  • ECOG performance status of 0, 1, or 2
  • Adequate lung, heart, liver, and kidney function as specified
  • Agreement to use contraception during the study and after completion if applicable
  • Ability to understand and sign informed consent
Not Eligible

You will not qualify if you...

  • Cytotoxic chemotherapy or radiotherapy within 2 weeks prior to study (4 weeks for certain drugs)
  • Residual severe side effects from prior cancer therapy (Grade >1), except alopecia
  • Prior therapies or conditions increasing risk of hepatic veno-occlusive disease
  • Currently receiving other investigational agents within 21 days prior to study
  • Extramedullary disease in immune privileged sites such as CNS, testes, or eyes
  • Recent myocardial infarction (within 2 years)
  • Venous thromboembolism within 1 year unless cleared
  • Recent stroke or transient ischemic attack within 1 year
  • Recent serious gastrointestinal bleeding or ulcers within 6 months
  • History of thrombotic microangiopathy or related syndromes
  • History of severe reactions to iron infusions or murine antibody products
  • Known donor-specific antibodies requiring treatment
  • Unable to stop medications affecting liver enzymes during conditioning
  • Uncontrolled infections
  • Prior hematopoietic or solid organ transplantation
  • Medical conditions preventing use of myeloablative radiation
  • HIV infection on combination antiretroviral therapy
  • Hepatitis B or C infection
  • Psychiatric or social conditions limiting study compliance
  • Pregnancy or breastfeeding
  • History of other malignancies unless disease-free for 5 years or specific exceptions

AI-Screening

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Trial Site Locations

Total: 1 location

1

Dana Farber Cancer Institute

Boston, Massachusetts, United States, 02115

Actively Recruiting

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Research Team

J

John Koreth, MBBS, DPhil

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NON_RANDOMIZED

Model

PARALLEL

Primary Purpose

TREATMENT

Number of Arms

3

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