Actively Recruiting
A Multi-Institution Study of TGFb2 Imprinted, Ex Vivo Expanded Universal Donor NK Cell Infusions as Adoptive Immunotherapy in Combination With Gemcitabine and Docetaxel in Patients With Relapsed or Refractory Pediatric Bone and Soft Tissue
Led by Nationwide Children's Hospital · Updated on 2026-01-28
50
Participants Needed
22
Research Sites
52 weeks
Total Duration
On this page
Sponsors
N
Nationwide Children's Hospital
Lead Sponsor
N
National Pediatric Cancer Foundation
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating whether adding infusions of a special type of immune cells called natural killer (NK) cells to the chemotherapy regimen gemcitabine and docetaxel (GEM/DOX) can improve outcomes for children and young adults with sarcomas that have come back or did not respond to previous treatments. This study aims to determine the safety and effectiveness of this combined treatment in relapsed or refractory pediatric sarcomas, including osteosarcoma, Ewing sarcoma, rhabdomyosarcoma, and other soft tissue sarcomas. The study is a multi-center, phase 1 and 2 trial with ongoing safety and toxicity analysis conducted by Nationwide Children's Hospital. Participants receive up to 8 cycles of treatment, each lasting 21 days. Each cycle includes intravenous gemcitabine on days 1 and 8, docetaxel on day 8, oral dexamethasone on days 7 to 9 to prevent side effects, pegfilgrastim on day 9 to help white blood cells recover, and intravenous TGF-beta imprinted (TGFb2i), ex vivo expanded universal donor NK cells on day 12. Tumor response is checked after cycles 2, 4, 6, and 8. The study carefully monitors for toxicities related to the combined treatment and tracks how well the NK cells persist in the body and relate to clinical outcomes. During the trial, participants undergo regular assessments including tumor measurements using the RECIST 1.1 criteria every two cycles, evaluation of treatment-related side effects, and laboratory tests to monitor blood counts and organ function. Safety and disease progression are followed for 3 to 5 years. The study also evaluates six-month progression-free survival to understand how well the treatment controls the cancer. Overall, participants will be closely monitored throughout the study and during long-term follow-up to assess the treatment's effects and safety.
CONDITIONS
Brief Title
A Multi-Institution Study of TGFβ Imprinted, Ex Vivo Expanded Universal Donor NK Cell Infusions as Adoptive Immunotherapy in Combination With Gemcitabine and Docetaxel in Patients With Relapsed or Refractory Pediatric Bone and Soft Tissue
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients aged 2 to 40 years with relapsed or refractory osteosarcoma, Ewing sarcoma, rhabdomyosarcoma, or non-rhabdomyosarcoma soft tissue sarcoma
- Measurable disease using RECIST 1.1 criteria
- Received between one and four prior cytotoxic systemic treatments for relapse sarcoma
- Local control with surgery or radiation considered per standard care before enrollment
- No myelosuppressive chemotherapy within 14 days prior to protocol therapy
- At least 2 weeks since local palliative radiation or 4 weeks since other radiation therapy
- At least 6 weeks since hematopoietic cell transplant
- At least 7 days or five half-lives since biologic anti-neoplastic agents
- At least 3 weeks since prior monoclonal antibody therapy
- Prior gemcitabine or docetaxel allowed if given at least 6 months before enrollment without severe allergic reactions or high-grade side effects
- Karnofsky score 160 for patients 16 years or older; Lansky score 160 for patients under 16
- Normal organ and marrow function within 7 days of starting therapy
- Neuropathy grade 2 or less at enrollment
- Well-controlled seizure disorders except diazepam use
- Use of adequate contraception for women of childbearing potential and men during and after study participation
- Ability and willingness to provide informed consent or assent
You will not qualify if you...
- Receiving other investigational agents
- Receiving other cancer treatments outside study drugs
- History of allergic reactions to docetaxel, gemcitabine, or pegfilgrastim
- Prior cellular therapies such as CAR-T or other manufactured cell products
- Bone marrow only disease
- Certain intermediate or malignant Grade 2 or 3 soft tissue tumors as defined by WHO classification
- Tumors near critical structures where swelling may cause severe symptoms
- Central nervous system metastatic disease
- Use of systemic corticosteroids outside supportive dexamethasone except for life-threatening conditions
- Use of strong CYP3A4 inducers, diazepam, or other chemotherapeutic agents besides study drugs
- Uncontrolled active infections or psychiatric/social conditions limiting compliance
- Pregnant or breastfeeding women
- HIV-positive patients on combination antiretroviral therapy
- Inability to comply with safety monitoring requirements
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 8 cycles of 21 days each
Participants receive up to 8 cycles of treatment every 21 days, including gemcitabine, docetaxel, supportive dexamethasone and pegfilgrastim, and universal donor TGFβ imprinted ex vivo expanded NK cells.
Visits during each 21-day cycle for treatment administration and assessments
Duration - 3 to 5 years
Participants are monitored for treatment response and safety outcomes for 3 to 5 years after completing treatment.
Assessments every two cycles during treatment and periodic follow-up visits post-treatment
Trial Site Locations
Total: 22 locations
1
University of Alabama
South Birmingham, Alabama, United States, 35233
Actively Recruiting
2
Phoenix Children's Hospital
Phoenix, Arizona, United States, 85016
Actively Recruiting
3
Arkansas Children's Hospital
Little Rock, Arkansas, United States, 72202
Actively Recruiting
4
Children's Hospital of Los Angeles
Los Angeles, California, United States, 90027
Actively Recruiting
5
Stanford University
Palo Alto, California, United States, 94304
Actively Recruiting
6
University of Florida
Gainesville, Florida, United States, 32610
Actively Recruiting
7
Nemours Jacksonville
Jacksonville, Florida, United States, 32207
Actively Recruiting
8
University of Miami
Miami, Florida, United States, 33136
Actively Recruiting
9
Johns Hopkins All Children's Hospital
St. Petersburg, Florida, United States, 33701
Actively Recruiting
10
Washington University/St Louis Childrens
St Louis, Missouri, United States, 63110
Actively Recruiting
11
Roswell Park Comprehensive Cancer Center
Buffalo, New York, United States, 14263
Actively Recruiting
12
Montefiore Medical Center
The Bronx, New York, United States, 10467
Actively Recruiting
13
University of North Carolina
Chapel Hill, North Carolina, United States, 27599
Actively Recruiting
14
Levine Cancer Institute
Charlotte, North Carolina, United States, 28203
Actively Recruiting
15
Duke Children's Hospital/Duke Health
Durham, North Carolina, United States, 27710
Actively Recruiting
16
Cleveland Clinic
Cleveland, Ohio, United States, 44195
Actively Recruiting
17
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
Actively Recruiting
18
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
19
Vanderbilt University Medical Center
Nashville, Tennessee, United States, 37232
Actively Recruiting
20
UT Southwestern
Dallas, Texas, United States, 75390
Actively Recruiting
21
University of Texas MD Anderson Cancer Center
Houston, Texas, United States, 77030
Actively Recruiting
22
Primary Children's Hospital
Salt Lake City, Utah, United States, 84113
Actively Recruiting
Research Team
J
Jessica Crimella, BSN, RN, CCRP
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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