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NCT Number: NCT03827343

Retrospective Study of Immunotherapy Related Toxicities and Factors Impacting Outcomes in Children and Adults With Cancer

Immunotherapy is changing the landscape of cancer therapy. Particularly unique to immunotherapy is the toxicity profile, which differs from chemotherapy-based strategies and can be associated with inflammatory responses and/or autoimmune type reactions resulting from activation of the immune system. Referred to as immune related adverse events (irAEs), these adverse events may require systemic immunosuppression or have other consequences and present unique management challenges. Specific to CAR T-cell and other adoptive cell therapies is the constellation of symptoms referred to as cytokine release syndrome (CRS), which can range in severity from mild to severe, and can require both cytokine directed blockade and/or systemic immunosuppression to ameliorate the side effects. While side effects may be unique to each individual immunotherapy, and may also be patient specific, cumulative experience will help to inform toxicity profiles and improve management of side effects and overall outcomes.

Given the number of immunotherapeutic approaches at the NCI, the primary goal of this protocol is to facilitate retrospective chart review of various immunotherapy trials at the NCI used in the treatment of cancer to comprehensively study toxicity profiles. This study will not involve the use of specimens or participant contact. All data that is needed has already been collected on the individual treatment protocols and is available in CRIS records or protocol specific databases.

Data will only be collected from treatment protocols where the PI has given permission for use of the data on the trial the subject was enrolled on. This protocol will be amended to incorporate new research objectives and new protocols as necessary....

Active, Not Recruiting

This study is active but is not currently recruiting participants.

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Key information

Conditions

Macrophage Activation Syndrome Hemic and Lymphatic Diseases Histiocytosis Histiocytosis, Non-Langerhans-Cell Immune System Diseases Immunoproliferative Disorders Lymphatic Diseases Lymphohistiocytosis, Hemophagocytic Lymphoproliferative Disorders Primary Hemophagocytic Lymphohistiocytosis

Age range

1 month–120 year

Sex eligibility

All sexes

Study type

Observational

Primary location

National Cancer Institute (NCI)

Bethesda, Maryland, 20892, United States

About this study

Immunotherapy is changing the landscape of cancer therapy. Particularly unique to immunotherapy is the toxicity profile, which differs from chemotherapy-based strategies and can be associated with inflammatory responses and/or autoimmune type reactions resulting from activation of the immune system. Referred to as immune related adverse events (irAEs), these adverse events may require systemic immunosuppression or have other consequences and present unique management challenges. Specific to CAR T-cell and other adoptive cell therapies is the constellation of symptoms referred to as cytokine release syndrome (CRS), which can range in severity from mild to severe, and can require both cytokine directed blockade and/or systemic immunosuppression to ameliorate the side effects. While side effects may be unique to each individual immunotherapy, and may also be patient specific, cumulative experience will help to inform toxicity profiles and improve management of side effects and overall outcomes.

Given the number of immunotherapeutic approaches at the NCI, the primary goal of this protocol is to facilitate retrospective chart review of various immunotherapy trials at the NCI used in the treatment of cancer to comprehensively study toxicity profiles. This study will not involve the use of specimens or participant contact. All data that is needed has already been collected on the individual treatment protocols and is available in CRIS records or protocol specific databases. Data will only be collected from treatment protocols where the PI has given permission for use of the data on the trial the subject was enrolled on or from standard of care protocols. This protocol will be amended to incorporate new research objectives and new protocols as necessary.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

  • Retrospective chart review of various immunotherapy trials at the NCI used in the treatment of cancer to comprehensively study toxicity profiles. This study will not involve the use of specimens or participant contact. All data that is needed has already been collected on the individual treatment protocols and is available in CRIS records or protocol specific databases.

Treatment and study plan

Primary outcomes

  1. To develop a retrospective study to allow for comparison of immunotherapy related toxicity profiles and risk factors across a set of protocols in the NCI.

    Time frame: 2 years

    Summary of immunotherapy related toxicity profiles and risk factors across a set of protocols in the NCI.

Secondary outcomes

  1. Evaluate infectious complications and their risk factors in patients who receive CAR-T cell therapy for cancer

    Time frame: 2 years

    Summary of infectious complications and their risk factors in patients who receive CAR-T cell therapy for cancer

  2. Evaluate the incidence, risk factors for, and treatment of HLH/MAS (now renamed IEC-HS) in patients who receive CAR-T cell therapy.

    Time frame: 2 years

    Incidence and treatment of HLH/MAS (now renamed IEC-HS) in patients who receive CAR-T cell therapy

  3. Incidence and time to resolution

    Time frame: 2 years

    Incidence of and time to resolution of grade 3 and 4 cytopenias post-CAR T-cell therapy in those who achieve a complete remission.

  4. Overall and relapse free survival

    Time frame: 2 years

    Summary of overall and relapse free survival and transplant associated toxicities for patients undergoing HSCT following CAR-T cell therapy

  5. Incidence of end organ toxicities

    Time frame: 2 years

    Incidence of grade 3 and 4 end organ toxicities experienced within the first 30 days of CAR T-cell therapy and includes associations between pre-CAR organ function as well as post-CAR response to such toxicities as well as validate the CAR-Comorbidity Index as predictive model of CRS severity

  6. Evaluate response and toxicity profile of second CAR T-cell infusions

    Time frame: 2 years

    Summary of factors associated with response to second CAR T-cell infusion

  7. Evaluate impact of race/ethnicity and obesity on CAR T-cell outcomes

    Time frame: 2 years

    Summary of response and toxicity profiles in patients based on race/ethnicity and also in those who are obese compared to those who are not.

  8. Evaluate impact of cryopreservation on outcomes following CAR T-cell infusion

    Time frame: 2 years

    Summary of cryopreservation and patients' outcomes following CAR T-cell infusion

  9. Evaluate outcomes for patients with ALL and Down Syndrome following CAR T-cell therapy

    Time frame: 2 years

    Summary of outcomes of patients with ALL and Down Syndrome who have received CAR T-cell therapy

  10. Evaluate absolute lymphocyte count following lymphodepleting chemotherapy

    Time frame: 2 years

    Summary of absolute lymphocyte count across lymphodepleting regimens

  11. Evaluate relationship between clinical variable and apheresis and manufacturing products

    Time frame: 2 years

    Summary of clinical variables and apheresis and manufacturing products

  12. Evaluate incidence of hypertension, identify risk factors for development of hypertension, summarize medical management in CAR setting, and identify complications

    Time frame: 2 years

    Summary of incidence of hypertension, risk factors, medical management and complications

  13. Describe baseline demographics, prior treatment characteristics and outcomes based on patients who are referred to CAR T-cell program

    Time frame: 2 years

    Summary of demographics, prior treatment and outcomes for patients referred to CAR T-cell program

  14. Incidence of pre-infusion BCA and use of BCA as a prognostic marker for CAR T-cell associated toxicity and efficacy

    Time frame: 2 years

    Incidence of BCA pre-infusion and use as a prognostic marker for CAR T-cell associated toxicity and efficacy

  15. Presence and durability of CD72 expression in both B-ALL and normal B-ALL/hematogones

    Time frame: 2 years

    Summary of CD72 expression in both B-ALL and normal B-ALL/hematogones and evaluate use as prognostic marker for CAR T-cell associated toxicity and efficacy

  16. Evaluate interventions, documentation of care goals, and use of palliative care consultation or other symptom management

    Time frame: 2 years

    Summary of interventions, documentation of care goals, and use of palliative care consultation or other symptom management for patients treated with CAR T-cell therapy

  17. Evaluate long-term outcomes of survivors who received CAR T-cell therapy

    Time frame: 2 years

    Summary of long-term outcomes of survivors who received CAR T-cell therapy

  18. Evaluate cross compare responses and outcomes based on NGS MRD and FC MRD

    Time frame: 2 Years

    Summary of cross compare responses and outcomes based on NGS MRD and FC MRD

  19. Evaluate the role of manufacturing changes on CAR T-cell outcomes

    Time frame: 2 Years

    Summary of the role of manufacturing changes on CAR T-cell outcomes

  20. Evaluate the impact of clonal hematopoiesis on CAR T-cell outcomes

    Time frame: 2 Years

    Summary of the impact of clonal hematopoiesis on CAR T-cell outcomes

  21. Evaluate CAR T-cell related coagulopathies

    Time frame: 2 Years

    Summary of CAR T-cell related coagulopathies

  22. Evaluate the use of anti-cytokine therapy to treat toxicities after CAR T-cell infusion

    Time frame: 2 Years

    Summary of the use of anti-cytokine therapy to treat toxicities after CAR T-cell infusion

  23. Evaluate outcomes of CAR T-cells in patients with extramedullary disease

    Time frame: 2 Years

    Summary of outcomes of CAR T-cells in patients with extramedullary disease

  24. To evaluate outcomes of CAR T-cells based on time of infusion

    Time frame: 2 years

    Summary of outcomes of CAR T-cells based on time of infusion

Sponsors and collaborators

Lead sponsor

National Cancer Institute (NCI)

Nih

Registry information

Important dates

Study start
2019
Primary completion
2026
Study completion
2026
First posted
Feb 1, 2019
Registry last updated
Jul 28, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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