NCT00902044

Brief Summary

Patients have a type of cancer called sarcoma. Because there is no standard treatment for the patients cancer at this time or because the currently used treatments do not work fully in all cases, patients are being asked to volunteer to take part in a gene transfer research study using special immune cells. This research study combines two different ways of fighting disease: antibodies and T cells. Antibodies are proteins that protect the body from diseases caused by germs or toxic substances. They work by binding those germs or substances, which stops them from growing or exerting their toxic effects. T cells, also called T lymphocytes, are special infection-fighting blood cells that can kill other cells, including tumor cells or cells that are infected with germs. Both antibodies and T cells have been used to treat patients with cancers: they both have shown promise, but have not been strong enough to cure most patients. We have found from previous research that we can put a new gene into T cells that will make them recognize cancer cells and kill them. We now want to see if we can put a new gene in these cells that will let the T cells recognize and kill sarcoma cells. The new gene that we will put in makes an antibody specific for HER2 (Human Epidermal Growth Factor Receptor 2) that binds to sarcoma cells. In addition it contains CD28, which stimulated T cells and make them last longer. In other clinical studies using T cells, some investigators found that giving chemotherapy before the T cell infusion can improve the amount of time the T cells stay in the body and therefore the effect the T cells can have. Giving chemotherapy before a T cell infusion is called lymphodepletion since the chemotherapy is specifically chosen to decrease the number of lymphocytes in the body. Decreasing the number of patient's lymphocytes first should allow the T cells we infuse to expand and stay longer in your body, and potentially kill cancer cells more effectively. We will use fludarabine or the combination of cyclophosphamide and fludarabine as the chemotherapy agents for lymphodepletion. Cyclophosphamide and fludarabine are the chemotherapy agents most commonly used for lymphodepletion in immunotherapy clinical trials. The purpose of this study is to find the largest safe dose of chimeric T cells, and to see whether this therapy might help patients with sarcoma. Another purpose is to see if it is safe to give HER2-CD28 T cells after lymphodepleting chemotherapy.

Trial Health

75
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
36

participants targeted

Target at P50-P75 for phase_1

Timeline
75mo left

Started Feb 2010

Longer than P75 for phase_1

Geographic Reach
1 country

2 active sites

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress73%
Feb 2010Jul 2032

First Submitted

Initial submission to the registry

May 13, 2009

Completed
1 day until next milestone

First Posted

Study publicly available on registry

May 14, 2009

Completed
9 months until next milestone

Study Start

First participant enrolled

February 11, 2010

Completed
9.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 6, 2019

Completed
12.6 years until next milestone

Study Completion

Last participant's last visit for all outcomes

July 1, 2032

Expected
Last Updated

January 26, 2026

Status Verified

January 1, 2026

Enrollment Period

9.8 years

First QC Date

May 13, 2009

Last Update Submit

January 23, 2026

Conditions

Keywords

Refractory SarcomaMetastatic SarcomaSarcomaHER2-positiveGene TherapyHER2-specific T cells

Outcome Measures

Primary Outcomes (1)

  • Number of patients with dose limiting toxicity after one injection of HER2-specific T cells

    To determine the safety of one intravenous injection of autologous T cells expressing HER2-specific chimeric antigen receptor (CAR) in patients with advanced HER2-positive sarcoma. To determine the safety of one intravenous injection of 1x10\^8/m\^2 autologous T cells after lymphodepleting chemotherapy.

    6 weeks

Secondary Outcomes (2)

  • Frequency of HER2-specific T cells pre and post injection

    15 years

  • Change in tumor size from pre to post injection

    6 weeks

Study Arms (4)

Autologous HER2-specific T cells

EXPERIMENTAL

THIS ARM IS CLOSED Dose Level 1: 1x10\^4 cells/m2 Dose Level 2: 3x10\^4 cells/m2 Dose Level 3: 1x10\^5 cells/m2 (NOT BEING USED) Dose Level 4: 3x10\^5 cells/m2 (NOT BEING USED) Dose Level 5: 1x10\^6 cells/m2 Dose Level 6: 3x10\^6 cells/m2 Dose Level 7: 1x10\^7 cells/m2 Dose Level 8: 3x10\^7 cells/m2 Dose Level 9: 1x10\^8 cells/m2

Genetic: Autologous HER2-specific T cells

HER2-specific T cells+fludarabine

EXPERIMENTAL

Autologous HER2-specific T cells+fludarabine: Dose Level 9A: fludarabine followed by 1x10\^8 cells/m\^2

Genetic: Autologous HER2-specific T cellsDrug: Fludarabine

HER2-specific T cells+fludarab.+cycloph.

EXPERIMENTAL

Autologous HER2-specific T cells+fludarabine+cyclophosphamide: Dose Level 9B: fludarabine + cyclophosphamide followed by 1x10\^8 cells/m\^2

Genetic: Autologous HER2-specific T cellsDrug: FludarabineDrug: Cyclophosphamide

CAR Positive cells

EXPERIMENTAL

Dose Level 9C: fludarabine + cyclophosphamide followed by 1x10\^8 cells/m\^2 CAR positive cells/m\^2

Genetic: Autologous HER2-specific T cellsDrug: FludarabineDrug: CyclophosphamideGenetic: Autologous CAR Positive T cells

Interventions

Cyclophosphamide will be administered for 2 days. Fludarabine and cyclophosphamide will be given for 2 days, followed by fludarabine alone for the next 3 days, followed by 2 days of rest, before the T cells will be administered. Cyclophosphamide Dose: 30 mg/kg/day IV over 1 hour (with Mesna and IV hydration) Fludarabine Dose: \>10 kg: 25 mg/m2/day; \<10 kg: 1 mg/kg/day IV over 30 minutes

Also known as: Cytoxan
CAR Positive cellsHER2-specific T cells+fludarab.+cycloph.

Patient will receive one intravenous injection of autologous CAR T cells at dose level 9C. Further CAR T-cell dose escalation at dose level 9C will be done using the lymphodepletion schema as in dose level 9B.

CAR Positive cells

Each patient will receive one intravenous injection of autologous HER2-specific T cells at one of the dose levels. If the patient has stable disease or a reduction in the size of the tumor they can receive additional doses of HER2-specific T cells at 6 to 12 weeks intervals-each of which will consist of the same cell number as their HER2-specific T-cell injection. For the first two subsequent HER2-specific T-cell infusions, patients will be able to receive additional lymphodepleting chemotherapy according to their dose levels.

Autologous HER2-specific T cellsCAR Positive cellsHER2-specific T cells+fludarab.+cycloph.HER2-specific T cells+fludarabine

Fludarabine will be administered for 5 days prior to the T cells The dose: \>10 kg: 25 mg/m2/day; \<10 kg: 1 mg/kg/day IV over 30 minutes

Also known as: Fludara
CAR Positive cellsHER2-specific T cells+fludarab.+cycloph.HER2-specific T cells+fludarabine

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Procurement Eligibility:
  • Diagnosis of refractory HER2-positive sarcoma or metastatic HER2-positive osteosarcoma.
  • Karnofsky/Lansky score of 50 or greater
  • Informed consent explained to, understood by and signed by patient/guardian. Patient/guardian given copy of informed consent.
  • Treatment Eligibility:
  • Diagnosis of refractory HER2-positive sarcoma or metastatic HER2-positive sarcoma with disease progression after receiving at least one prior systemic therapy.
  • Recovered from acute toxic effects of all prior cytotoxic chemotherapy at least 4 weeks before entering this study. PD1/PDL1 inhibitors will be allowed to continue during treatment if medically indicated.
  • Normal ECHO (Left ventricular ejection fraction (LVEF) has to be within normal, institutional limits)
  • Life expectancy 6 weeks or greater
  • Karnofsky/Lansky score of 50 or greater
  • Bilirubin 3x or less, AST 3x or less, Serum creatinine 2x upper limit of normal or less, Hgb 7.0 g/dl or greater, WBC greater than 2,000/ul, ANC greater than 1,000/ul, platelets greater than 100,000/ul. Creatinine clearance is needed for patients with creatinine greater than 1.5 times upper limit of normal.
  • Pulse oximetry of 90% or greater on room air
  • Sexually active patients must be willing to utilize one of the more effective birth control methods for 6 months after the CTL infusion. Male partner should use a condom
  • Available autologous transduced T lymphocytes with 15% or more expression of HER2 CAR as determined by flow-cytometry and killing of HER2-positive targets 20 % or greater in cytotoxicity assay.
  • Chest radiograph for baseline evaluation of lungs
  • +1 more criteria

You may not qualify if:

  • At time of Procurement:
  • Known HIV positivity
  • Severe previous toxicity from cyclophosphamide or fludarabine
  • At time of Treatment:
  • Severe intercurrent infection
  • Known HIV positivity
  • Pregnant or lactating
  • History of hypersensitivity reactions to murine protein-containing products
  • Severe previous toxicity from cyclophosphamide or fludarabine

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Houston Methodist Hospital

Houston, Texas, 77030, United States

Location

Texas Children's Hospital

Houston, Texas, 77030, United States

Location

Related Publications (2)

  • Hegde M, Navai S, DeRenzo C, Joseph SK, Sanber K, Wu M, Gad AZ, Janeway KA, Campbell M, Mullikin D, Nawas Z, Robertson C, Mathew PR, Zhang H, Mehta B, Bhat RR, Major A, Shree A, Gerken C, Kalra M, Chakraborty R, Thakkar SG, Dakhova O, Salsman VS, Grilley B, Lapteva N, Gee A, Dotti G, Bao R, Salem AH, Wang T, Brenner MK, Heslop HE, Wels WS, Hicks MJ, Gottschalk S, Ahmed N. Autologous HER2-specific CAR T cells after lymphodepletion for advanced sarcoma: a phase 1 trial. Nat Cancer. 2024 Jun;5(6):880-894. doi: 10.1038/s43018-024-00749-6. Epub 2024 Apr 24.

  • Hegde M, Joseph SK, Pashankar F, DeRenzo C, Sanber K, Navai S, Byrd TT, Hicks J, Xu ML, Gerken C, Kalra M, Robertson C, Zhang H, Shree A, Mehta B, Dakhova O, Salsman VS, Grilley B, Gee A, Dotti G, Heslop HE, Brenner MK, Wels WS, Gottschalk S, Ahmed N. Tumor response and endogenous immune reactivity after administration of HER2 CAR T cells in a child with metastatic rhabdomyosarcoma. Nat Commun. 2020 Jul 15;11(1):3549. doi: 10.1038/s41467-020-17175-8.

MeSH Terms

Conditions

Sarcoma

Interventions

fludarabinefludarabine phosphateCyclophosphamide

Condition Hierarchy (Ancestors)

Neoplasms, Connective and Soft TissueNeoplasms by Histologic TypeNeoplasms

Intervention Hierarchy (Ancestors)

Phosphoramide MustardsNitrogen Mustard CompoundsMustard CompoundsHydrocarbons, HalogenatedHydrocarbonsOrganic ChemicalsPhosphoramidesOrganophosphorus Compounds

Study Officials

  • Nabil M Ahmed, MD

    Baylor College of Medicine - Texas Children's Hospital

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Associate Professor, Pediatric Hematology Oncology, Center for Cell and Gene Therapy

Study Record Dates

First Submitted

May 13, 2009

First Posted

May 14, 2009

Study Start

February 11, 2010

Primary Completion

December 6, 2019

Study Completion (Estimated)

July 1, 2032

Last Updated

January 26, 2026

Record last verified: 2026-01

Locations