Actively Recruiting

Phase 1
Age: 18Years +
All Genders
ID05473910

A Controlled Multi-Arm Phase 1 Umbrella Study Evaluating the Safety and Feasibility of T-Cell Receptor Engineered Donor T-Cells Targeting HA1 (TSC-100) or HA2 (TSC-101) in HLA-A0201 Positive Patients Undergoing Allogeneic Peripheral Blood Stem Cell Transplantation

Led by TScan Therapeutics, Inc. · Updated on 2026-04-08

75

Participants Needed

15

Research Sites

N/A

Total Duration

On this page

AI-Summary

What this Trial Is About

Researchers are evaluating the safety and feasibility of two genetically engineered donor-derived T cell therapies, TSC-100 and TSC-101, in patients with acute myeloid leukemia (AML), myelodysplastic syndromes (MDS), or acute lymphoblastic leukemia (ALL) who are undergoing allogeneic peripheral blood stem cell transplantation. This Phase 1, multi-center, open-label umbrella study is designed to assess these treatments combined with the standard of care, compared to the standard of care alone, following hematopoietic cell transplantation (HCT) from haploidentical donors or matched unrelated donors (MUD or MMUD). The study focuses on patients positive for HLA A*02:01 and aims to determine the optimally tolerated dose and preliminary efficacy of these T cell therapies. Participants receive either TSC-100 or TSC-101 based on their HLA type and minor antigen positivity, or standard of care alone. The treatments are administered intravenously alongside standard reduced intensity conditioning regimens such as fludarabine with cyclophosphamide and total body irradiation, or other institutional protocols. Dose escalation follows an interval 3+3 design to evaluate safety and tolerability, with up to two doses given. Once the recommended phase 2 dose is identified, additional patients may be enrolled at that dose level. Supportive care and infectious prophylaxis are provided per institutional guidelines. During the study, participants will undergo safety evaluations including monitoring for dose-limiting toxicities and adverse events over a two-year follow-up period. Researchers will assess disease-free survival, relapse rates, overall survival, and immune responses to the therapies at various intervals up to two years. Participants must consent to mandatory procedures such as bone marrow biopsies and blood sampling for research and agree to long-term follow-up for up to 15 years if receiving the investigational T cell infusion. The study involves careful monitoring and comparison of outcomes between treatment and control groups to evaluate the investigational therapies' potential.

CONDITIONS

Brief Title

A Study of TSC-100 and TSC-101 in AML, ALL and MDS in Patients Undergoing Allogeneic Peripheral Blood Stem Transplantation

Who Can Participate

Age: 18Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Male or female aged 18 years or older at the time of signing the informed consent
  • Eastern Cooperative Oncology Group (ECOG) performance status of 2 or less at screening
  • Use of contraception as per local regulations during the study and for at least 12 months after last dose
  • Male participants must agree to use highly effective contraception during and for 12 months after treatment and avoid sperm donation
  • Female participants must not be pregnant or breastfeeding and either not be of childbearing potential or agree to contraceptive guidance during and 12 months after treatment
  • Preparing to undergo allogeneic hematopoietic cell transplantation for AML, MDS, or ALL
  • Treatment arm participants must be HLA-A*02:01 positive; control arm participants may have any HLA type
  • Possess HA-1 positive genotype to be eligible for TSC-100 or HA-2 positive genotype to be eligible for TSC-101
  • Have a haploidentical donor, mismatched unrelated donor (MMUD), or matched unrelated donor (MUD) meeting donor criteria
  • Clinically indicated for haploidentical donor, MMUD, or MUD transplantation, reduced intensity conditioning, and peripheral blood stem cell transplantation
  • Meet organ function requirements per institutional standards
  • Capable of giving signed informed consent including compliance with study procedures
  • Agree to mandatory study procedures including bone marrow biopsy and blood sampling
  • Agree to long-term follow-up for up to 15 years after initial treatment if receiving investigational T cell infusion
Not Eligible

You will not qualify if you...

  • Medical or psychological conditions making the participant unsuitable for cell therapy including uncontrolled malignancy or active central nervous system disease
  • Participants with donor-specific HLA antibodies considered high enough to require desensitization and no alternative donors
  • Participants positive for HLA-A*02:07 who meet criteria for TSC-101
  • Evidence of clinically significant infection or uncontrolled viral reactivation of CMV, EBV, Adenovirus, BK virus, or human herpesvirus 6
  • Active cardiac disease including recent angina, significant arrhythmias, recent myocardial infarction, or uncontrolled heart failure
  • Prior allogeneic hematopoietic cell transplantation
  • History of hypersensitivity to murine proteins
  • Enrollment in another trial with a novel investigational agent
  • Use of anti-thymocyte globulin, alemtuzumab, or other T-cell depleting agents from Day -14 through end of study
  • Donors positive for any HLA-A02 allele except HA-1 negative for TSC-100 or any HLA-A02 allele for TSC-101
  • Donors testing positive for HIV, HTLV, hepatitis B or C, syphilis, West Nile virus, or other specified infections
  • Related donors residing outside the USA unless screening and procedures can be done at the treatment site

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

1 visit (in-person)

Treatment

Duration - Up to 40 days for dose limiting toxicity evaluation; treatment may include repeated dosing as determined by safety review committee

Participants receive either TSC-100 or TSC-101 combined with standard of care, or standard of care alone, including reduced intensity conditioning, hematopoietic cell infusion, and acute graft-versus-host disease prophylaxis.

Multiple visits during and shortly after treatment for dosing and safety evaluations

Follow-up

Duration - Up to 2 years

Participants are monitored for safety, adverse events, disease-free survival, relapse rates, overall survival, and immunogenicity for up to 2 years after treatment.

Regular visits for safety and efficacy assessments over 2 years

Trial Site Locations

Total: 15 locations

1

City of Hope

Duarte, California, United States, 91010

Actively Recruiting

2

Yale

New Haven, Connecticut, United States, 06510

Actively Recruiting

3

Memorial Healthcare System

Hollywood, Florida, United States, 33021

Actively Recruiting

4

Northside Hospital

Atlanta, Georgia, United States, 30342

Actively Recruiting

5

John Hopkins University

Baltimore, Maryland, United States, 21287

Actively Recruiting

6

Mass General Hospital

Boston, Massachusetts, United States, 02114

Actively Recruiting

7

Karmanos Cancer Institute

Detroit, Michigan, United States, 48201

Actively Recruiting

8

Hackensack University Medical Center

Hackensack, New Jersey, United States, 07601

Actively Recruiting

9

Columbia University

New York, New York, United States, 10027

Actively Recruiting

10

Mount Sinai

New York, New York, United States, 10029-6696

Actively Recruiting

11

University North Carolina

Chapel Hill, North Carolina, United States, 27599

Actively Recruiting

12

UPenn

Philadelphia, Pennsylvania, United States, 19104

Actively Recruiting

13

Baylor University Medical Center

Dallas, Texas, United States, 75246

Actively Recruiting

14

MD Anderson

Houston, Texas, United States, 77030

Actively Recruiting

15

Froedert and Medical College of Wisconsin

Milwaukee, Wisconsin, United States, 53226

Actively Recruiting

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Research Team

J

Jim Murray

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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