An observational study in Macrophage Activation Syndrome and Primary Hemophagocytic Lymphohistiocytosis, sponsored by National Cancer Institute (NCI). Active, not recruiting at 1 site in United States. Open to participants aged 1 Month to 120 Years. Per ClinicalTrials.gov, last updated 2026-10-02.
Sponsored by National Cancer Institute (NCI) · Observational
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....
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.
99 studies on the registry are indexed under Lymphohistiocytosis, Hemophagocytic; 36 are open to participants now.
This study's enrollment of 500 is above the median of 128 across 29 observational studies indexed under Lymphohistiocytosis, Hemophagocytic.
Browse Lymphohistiocytosis, Hemophagocytic studies →National Cancer Institute (NCI) is the lead sponsor of 3,506 studies on the registry; 334 are open to participants now.
Of its 402 completed or terminated interventional studies of FDA-regulated products, 365 (91%) have results posted.
Counted across the registry records on this site, refreshed daily.
Children and adults with cancer enrolled on immunotherapy treatment protocols in the NCI.
Retrospective chart review of children and adults with cancer enrolled on immunotherapy treatment protocols in the NCI.
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.
Summary of immunotherapy related toxicity profiles and risk factors across a set of protocols in the NCI.
Time frame: 2 years
Evaluate infectious complications and their risk factors in patients who receive CAR-T cell therapy for cancer
Summary of infectious complications and their risk factors in patients who receive CAR-T cell therapy for cancer
Time frame: 2 years
Evaluate the incidence, risk factors for, and treatment of HLH/MAS (now renamed IEC-HS) in patients who receive CAR-T cell therapy.
Incidence and treatment of HLH/MAS (now renamed IEC-HS) in patients who receive CAR-T cell therapy
Time frame: 2 years
Incidence and time to resolution
Incidence of and time to resolution of grade 3 and 4 cytopenias post-CAR T-cell therapy in those who achieve a complete remission.
Time frame: 2 years
Overall and relapse free survival
Summary of overall and relapse free survival and transplant associated toxicities for patients undergoing HSCT following CAR-T cell therapy
Time frame: 2 years
Incidence of end organ toxicities
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
Time frame: 2 years
Evaluate response and toxicity profile of second CAR T-cell infusions
Summary of factors associated with response to second CAR T-cell infusion
Time frame: 2 years
Evaluate impact of race/ethnicity and obesity on CAR T-cell outcomes
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.
Time frame: 2 years
Evaluate impact of cryopreservation on outcomes following CAR T-cell infusion
Summary of cryopreservation and patients' outcomes following CAR T-cell infusion
Time frame: 2 years
Evaluate outcomes for patients with ALL and Down Syndrome following CAR T-cell therapy
Summary of outcomes of patients with ALL and Down Syndrome who have received CAR T-cell therapy
Time frame: 2 years
Evaluate absolute lymphocyte count following lymphodepleting chemotherapy
Summary of absolute lymphocyte count across lymphodepleting regimens
Time frame: 2 years
Evaluate relationship between clinical variable and apheresis and manufacturing products
Summary of clinical variables and apheresis and manufacturing products
Time frame: 2 years
Evaluate incidence of hypertension, identify risk factors for development of hypertension, summarize medical management in CAR setting, and identify complications
Summary of incidence of hypertension, risk factors, medical management and complications
Time frame: 2 years
Describe baseline demographics, prior treatment characteristics and outcomes based on patients who are referred to CAR T-cell program
Summary of demographics, prior treatment and outcomes for patients referred to CAR T-cell program
Time frame: 2 years
Incidence of pre-infusion BCA and use of BCA as a prognostic marker for CAR T-cell associated toxicity and efficacy
Incidence of BCA pre-infusion and use as a prognostic marker for CAR T-cell associated toxicity and efficacy
Time frame: 2 years
Presence and durability of CD72 expression in both B-ALL and normal B-ALL/hematogones
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
Time frame: 2 years
Evaluate interventions, documentation of care goals, and use of palliative care consultation or other symptom management
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
Time frame: 2 years
Evaluate long-term outcomes of survivors who received CAR T-cell therapy
Summary of long-term outcomes of survivors who received CAR T-cell therapy
Time frame: 2 years
Evaluate cross compare responses and outcomes based on NGS MRD and FC MRD
Summary of cross compare responses and outcomes based on NGS MRD and FC MRD
Time frame: 2 Years
Evaluate the role of manufacturing changes on CAR T-cell outcomes
Summary of the role of manufacturing changes on CAR T-cell outcomes
Time frame: 2 Years
Evaluate the impact of clonal hematopoiesis on CAR T-cell outcomes
Summary of the impact of clonal hematopoiesis on CAR T-cell outcomes
Time frame: 2 Years
Evaluate CAR T-cell related coagulopathies
Summary of CAR T-cell related coagulopathies
Time frame: 2 Years
Evaluate the use of anti-cytokine therapy to treat toxicities after CAR T-cell infusion
Summary of the use of anti-cytokine therapy to treat toxicities after CAR T-cell infusion
Time frame: 2 Years
Evaluate outcomes of CAR T-cells in patients with extramedullary disease
Summary of outcomes of CAR T-cells in patients with extramedullary disease
Time frame: 2 Years
To evaluate outcomes of CAR T-cells based on time of infusion
Summary of outcomes of CAR T-cells based on time of infusion
Time frame: 2 years
Plan to share: Yes — All IPD recorded in the medical record will be shared with intramural investigators upon request.
Supporting information: Study protocol, Sap, Icf
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
This study is active, not recruiting, as verified in Jun 2026. You cannot join it, but the record below documents what was studied.
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Macrophage Activation Syndrome
National Cancer Institute (NCI)