Tocilizumab for KSHV-Associated Multicentric Castleman Disease
Primary Purpose
Castleman Disease, Multicentric Castleman Disease, Giant Lymph Node Hyperplasia
Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Zidovudine
Tocilizumab
Valganciclovir (VGC)
Sponsored by
About this trial
This is an interventional treatment trial for Castleman Disease focused on measuring Kaposi Sarcoma Herpesvirus, Human Immunodeficiency Virus, Tocilizumab, Multicentric Castleman Disease, Interleukin-6, Castleman Disease, Acquired Immunodeficiency Syndrome (AIDS), Human Herpesvirus-8
Eligibility Criteria
- INCLUSION CRITERIA:
- Pathologically confirmed Kaposi sarcoma (KS)-associated herpes virus multi-centric Castleman disease (KSHV-MCD)
- Age greater than or equal to 18
- At least one clinical symptom probably or definitely attributed to KSHV-MCD
- Intermittent or persistent fever for at least 1 week (>38 degrees C)
- Fatigue (Common Terminology Criteria for Adverse Events (CTCAE) Grade 2 or greater)
- Gastrointestinal symptoms [includes nausea and anorexia] (CTCAE Grade 1 or greater)
- Respiratory symptoms [includes cough and airway hyperreactivity]
(CTCAE Grade 1 or greater)
- At least one laboratory abnormality probably or definitely attributed to KSHVMCD
- Anemia (Hgb [men] </=12.5 gm/dL, Hgb [women] </= 11 gm/dL)
- Thrombocytopenia (</=130,000/mm(3))
- Hypoalbuminemia (<3.4 g/dl)
- Elevated C-reactive protein (CRP) (CRP > 3 mg/L)] probably or definitely attributable to KSHV-MCD
- No life- or organ-threatening manifestations of MCD
- Eastern Cooperative Oncology Group (ECOG) performance status less than or equal to 2
- Human Immunodeficiency Virus (HIV)-infected patients should be receiving or willing to initiate an effective combination antiretroviral therapy (cART) regimen
- Willingness to complete tuberculosis evaluation and start prophylactic antituberculosis therapy as soon as is medically feasible if patients have a reactive tuberculin skin test and have not completed an adequate course of prevented anti-tuberculosis therapy, following American Thoracic Society/ Centers for Disease Control recommended guidelines:
http://www.cdc.gov/mmwr/preview/mmwrhtml/mm5231a4.htm
- Ability to understand and willingness to give informed consent
- Women of child bearing potential must agree to use birth control for the duration of the study
EXCLUSION CRITERIA:
- Uncontrolled bacterial, mycobacterial, or fungal infection
- Uncontrolled intercurrent illness including, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements or ability to receive therapy.
- Pregnant or lactating women
- Any abnormality that would be scored as National Cancer Institute (NCI) Common Toxicity Criteria (CTC) Grade 3 toxicity that is unrelated to HIV, its treatment, or to MCD that would preclude protocol treatment. Exceptions include:
- Lymphopenia
- Direct manifestations of Kaposi sarcoma or MCD
- Direct manifestation of HIV (i.e. low cluster of differentiation 4 (CD4) count)
- Direct manifestation of HIV therapy (i.e. Hyperbilirubinemia associated with protease inhibitors)
- Asymptomatic hyperuricemia
- Hypophosphatemia
- Elevated creatine kinase (CK) attributed to exercise
- Past or present history of malignant tumors other than Kaposi sarcoma unless one of the following:
- Complete remission for greater than or equal to 1 year from completion of therapy
- Completely resected basal cell carcinoma
- In situ squamous cell carcinoma of the cervix or anus
- Patients with concurrent Kaposi sarcoma requiring immediate cytotoxic chemotherapy
- History of tocilizumab therapy within prior three months
- History of rituximab or bevacizumab therapy within three months
- History of greater than or equal to 2 allergic reaction or any grade anaphylactic reaction during prior administration of tocilizumab
Sites / Locations
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Tocilizumab
Arm Description
Tocilizumab 8 mg/kg on Day 1 of a 14 day cycle a maximum of 6 cycles. If indicated, zidovudine (AZT) and valganciclovir (VGC) will be administered concurrently with tocilizumab, with day 1 of the cycle being the day tocilizumab is administered.
Outcomes
Primary Outcome Measures
Percentage of Participants With an Overall Clinical Benefit Response
Overall clinical benefit response is defined as Complete Response (CR): Full resolution of all clinical symptoms and laboratory abnormalities (whether or not these are indicator abnormalities) probably or definitely attributable to MCD, lasting at least 3 weeks; and Partial Response (PR): At least 50% of the abnormalities probably or definitely attributed to KSHV-MCD must improve by the minimum amounts specified to attain PR. Only abnormalities present in a specific patient at baseline may count toward the achievement of a PR (e.g. if six of indicator abnormalities are present at baseline, at least three must meet the specified criteria to be considered a PR) assessed using a modified Kaposi sarcoma herpes virus-associated multicentric Castleman disease (KSHV- MCD) Clinical Benefit Response Criteria and the National Cancer Institute (NCI) KSHV-MCD criteria.
Secondary Outcome Measures
Percentage of Participants With a Clinical Response
Clinical response is defined as a Complete Response (CR): Full resolution of all signs and symptoms attributable to MCD, lasting 1 cycle (3-4 weeks depending on regimen; Symptom Free Disease (SFD:) Full resolution of all signs and symptoms attributable to MCD, not yet lasting 1 cycle (3-4 weeks depending on regimen); and Partial Response (PR): Improvement in at least 50% of signs and symptoms by at least 1 grade (NCI-Common Terminology Criteria for Adverse Events (CTCAE v4), with no increased MCD related increases, lasting 1 cycle (3-4 weeks depending on regimen) assessed by the National Cancer Institute Kaposi sarcoma herpes virus-associated multicentric Castleman disease (NCI KSHV-MCD) Response Criteria.
Percentage of Participants With a Biochemical Response
A biochemical response is defined as a Complete Response (CR): Normalization of abnormalities attributed to MCD in the following labs: Complete blood count (CBC), Chem 20, C-Reactive protein (CRP), lasting 1 cycle (3-4 weeks depending on regimen); Partial Response (PR): 50% improvement in all labs abnormalities attributable to MCD, lasting 1 cycle (3-4 weeks depending on regimen), and was assessed by the National Cancer Institute (NCI) Kaposi sarcoma herpes virus-associated multicentric Castleman Disease (KSHV-MCD) Response Criteria.
Percentage of Participants With a Radiographic Response
A radiographic response is defined as a Complete Response (CR): Normalization of all lymph nodes to <1.5 cm in greatest transverse dimension, decrease to < 1 cm of lymph nodes 1.1-1.5 cm at baseline (or 75% decrease in the sum of products of diameters (SPD)), Spleen < 12 cm greatest dimension, no pleural effusions; Complete Response unconfirmed (CRu): Residual lymph node mass >1.5 cm or splenomegaly > 12 cm that has decrease by >75% and does not change over one year; and Partial Response (PR): For lymph nodes, >50% decrease in SPD of 6 dominant nodes, for spleen 50% decrease in longest transverse dimension assessed by the National Cancer Institute (NCI) Kaposi sarcoma herpes virus-associated multicentric Castleman Disease (KSHV-MCD) Response Criteria.
Percentage of Participants With Kaposi Sarcoma Responses Per the Acquired Immunodeficiency Syndrome (AIDS) Clinical Trials Group (ACTG) Response Criteria
The ACTG Criteria is defined as a Complete Response (CR), Partial Response (PR), Stable Disease (SD), Progressive Disease (PD), and Symptom Free Disease (SFD). The terms Improved (I), Stable (S), Mixed (M) Response, and Worse (W) are also used to further describe participants who have SD or PR.
Changes in Plasma Exposure of Ritonavir in Response to Tocilizumab and Zidovudine (AZT) Metabolized by Cytochrome P450 (CYP450)
Cumulative plasma exposure of Ritonavir will be evaluated.
Changes in Plasma Exposure of Lopinavir in Response to Tocilizumab and AZT Metabolized by Cytochrome P450 (CYP450)
Cumulative plasma exposure of Lopinavir will be evaluated.
Changes in Plasma Exposure of Atazanavir in Response to Tocilizumab and Zidovudine (AZT) Metabolized by Cytochrome P450 (CYP450)
Cumulative plasma exposure of Atazanavir will be evaluated.
Changes in Plasma Exposure of Efavirenz in Response to Tocilizumab and Zidovudine (AZT) Metabolized by Cytochrome P450 (CYP450)
Cumulative plasma exposure of Efavirenz will be evaluated.
Percentage of Participants With Grade 3 or Greater Serious Adverse Events
Here is the percentage of participants with Grade 3 or greater serious adverse events assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0). A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life-threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned. Grade 3 is severe or medically significant, Grade 4 is life threatening, and Grade 5 is death.
Percentage of Participants With <Grade 3 Non- Serious Adverse Events
Here is the percentage of participants with <Grade 3 non-serious adverse events assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0). A non-serious adverse event is any untoward medical occurrence. Grade 1 is mild and Grade 2 is moderate.
Number of Participants With Serious and Non-serious Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v4.0)
Here is the count of participants with serious and non-serious adverse events assessed by the Common Terminology Criteria for Adverse Events (CTCAE v4.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life-threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned.
Effect of Tocilizumab on the Pharmacokinetics (PK) of Antiretroviral Agents
Evaluate the effect of tocilizumab on the pharmacokinetics of antiretroviral agents that are cytochrome P3A4 (CYP3A4) substrates in patients with symptomatic Kaposi sarcoma herpes virus-associated multicentric Castleman Disease (KSHV-MCD).
Percentage of Participants Progression-free Survival at 4 Months
Progression-free survival is defined as participants who progress or die by 4 months after the start of treatment with tocilizumab and tocilizumab /AZT/VGC.
Percentage of Participants With Overall Survival 4 Months After Treatment With Tocilizumab and Tocilizumab /Zidovudine (AZT)/Valganciclovir (VGC)
Overall survival is defined as the percentage of participants alive at 4 months after the start of treatment with tocilizumab and tocilizumab /AZT/VGC.
Full Information
NCT ID
NCT01441063
First Posted
September 24, 2011
Last Updated
October 8, 2020
Sponsor
National Cancer Institute (NCI)
1. Study Identification
Unique Protocol Identification Number
NCT01441063
Brief Title
Tocilizumab for KSHV-Associated Multicentric Castleman Disease
Official Title
Pilot Study of Tocilizumab in Patients With Symptomatic Kaposi Sarcoma Herpesvirus (KSHV) - Associated Multicentric Castleman Disease
Study Type
Interventional
2. Study Status
Record Verification Date
October 2020
Overall Recruitment Status
Completed
Study Start Date
September 13, 2011 (Actual)
Primary Completion Date
June 6, 2018 (Actual)
Study Completion Date
October 5, 2020 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
National Cancer Institute (NCI)
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
Background:
- Kaposi's sarcoma-associated herpes virus (KSHV)-associated multicentric Castleman disease (KSHV-MCD) is caused by a herpes virus known as KSHV. This disease can also cause several other cancers, including Kaposi sarcoma. People with KSHV-MCD often have symptoms like fever, weight and muscle loss, and fluid in the legs or abdomen. Tocilizumab may be able to block the chemicals in the body that cause KSHV-MCD symptoms. Researchers want to test this drug and other anti-virus drugs to find the best combination of drugs to treat KSHV-MCD.
Objectives:
- To test the effectiveness of tocilizumab with and without other anti-virus drugs for KSHV-MCD.
Eligibility:
- People at least 18 years of age who have KSHV-MCD and have certain symptoms and blood abnormalities caused by their KSHV-MCD.
Design:
Participants will be screened with a medical history and physical exam. They will also have blood tests, and a skin biopsy.
Participants will have tocilizumab injections every 2 weeks for up to 12 weeks. They will provide daily blood samples for the first 3 days of treatment.
After the sixth dose, participants will be monitored for 4 weeks to check for possible side effects.
Those whose KSHV-MCD does not improve or worsens during the study may have tocilizumab combined with two other anti-virus drugs, zidovudine and valganciclovir. These drugs are pills that will be taken four times a day for 5 days out of every 2 weeks.
Blood, urine, and saliva samples will be collected throughout the study.
Detailed Description
BACKGROUND:
Kaposi sarcoma herpesvirus-associated multicentric Castleman disease (KSHVMCD) is a rare lymphoproliferative disorder that develops predominantly in human immunodeficiency deficiency virus (HIV) infected patients. Patients often have symptoms from interleukin-6 (IL-6), KSHV encoded viral IL-6 (vIL-6), and other cytokines
Goals of therapy include rapid resolution symptoms and elimination of reservoirs of KSHV-infected plasmablasts.
Tocilizumab is a humanized anti-IL-6 receptor (gp80) antibody with activity against MCD unrelated to KSHV (KSHV-negative MCD). While tocilizumab does not directly affect vIL-6 signaling or other KSHV driven pathologic processes, IL-6 overproduction plays a major role in symptoms in KSHV-MCD, and blocking IL-6 may be sufficient to treat this disorder by blocking autocrine and paracrine stimulation. Combination with zidovudine (AZT) and valganciclovir (VGC), agents that target KSHV replication, have virus-activated cytotoxic activity, and are active in KSHV-MCD may be useful and necessary in some patients.
OBJECTIVES:
Primary objective: Estimate clinical benefit of tocilizumab 8mg/kg every 2 weeks for up to 12 weeks in patients with symptomatic KSHV-MCD using a modified KSHVMCD Clinical Benefit Response Criteria
Secondary objectives:
Estimate best clinical, biochemical, radiographic, and overall responses in patients with KSHV-MCD treated for up to 12 weeks with tocilizumab 8mg/kg every 2 weeks using the prior National Cancer Institute (NCI) KSHV-MCD Response Criteria.
In patients with inadequate response to tocilizumab monotherapy: explore preliminarily the activity of tocilizumab 8mg/kg every 2 weeks, combined with AZT 600 mg orally q6 hours and VGC 900 mg orally q12 hours on days 1-5 of a 14-day cycle
Evaluate safety and tolerability of tocilizumab alone and combined with AZT/VGC
Evaluate the effect of tocilizumab on the pharmacokinetics of antiretroviral agents that are Cytochrome P450 3A4 (CYP3A4) substrates in patients with symptomatic KSHV-MCD
Evaluate progression-free and overall survival of patients treated with tocilizumab and tocilizumab/AZT/VGC
Evaluate of effect of tocilizumab on KS
Eligibility
Pathologically confirmed KSHV-associated MCD
Age greater than or equal to 18
At least one clinical symptom and at least one laboratory attributable to KSHV-MCD
Eastern Cooperative Oncology Group (ECOG) performance status less than or equal to 2
No life- or organ-threatening manifestations of MCD
Patients requiring therapy for rheumatoid arthritis will be excluded
HIV-infected patients must agree to continue or start combination antiretroviral therapy
DESIGN:
Open label, single center pilot study. Eligible patients receive tocilizumab 8 mg/kg every 2 weeks for up to 12 weeks. In addition, patients requiring treatment intensification also receive AZT 600 mg orally q6 hours and VGC 900 mg orally q12 hours on days 1-5 of a 14-day cycle.
Sample size 17: two stage phase II design, alpha equals beta equals 0.10, ruling out <20% KSHV-MCD Clinical Benefit Partial Response or better with tocilizumab and targeting a >50% KSHV-MCD Clinical Benefit Partial Response or better requires 10 in the first stage. 0-2 of 10 major response: stop accrual, 3+/10: accrual to 17 total.
Responses evaluated by KSHV-MCD Clinical Benefit Response Criteria and NCI KSHV-MCD criteria under prospective evaluation.
Safety and tolerability evaluated using current Common Terminology Criteria for Adverse Events (CTCAE).
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Castleman Disease, Multicentric Castleman Disease, Giant Lymph Node Hyperplasia
Keywords
Kaposi Sarcoma Herpesvirus, Human Immunodeficiency Virus, Tocilizumab, Multicentric Castleman Disease, Interleukin-6, Castleman Disease, Acquired Immunodeficiency Syndrome (AIDS), Human Herpesvirus-8
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
8 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Tocilizumab
Arm Type
Experimental
Arm Description
Tocilizumab 8 mg/kg on Day 1 of a 14 day cycle a maximum of 6 cycles. If indicated, zidovudine (AZT) and valganciclovir (VGC) will be administered concurrently with tocilizumab, with day 1 of the cycle being the day tocilizumab is administered.
Intervention Type
Drug
Intervention Name(s)
Zidovudine
Other Intervention Name(s)
Retrovir
Intervention Description
Zidovudine (AZT) 600 mg orally q6 hours (every 6 hours)
Intervention Type
Drug
Intervention Name(s)
Tocilizumab
Other Intervention Name(s)
Actemra
Intervention Description
Tocilizumab 8mg/kg every 2 weeks
Intervention Type
Drug
Intervention Name(s)
Valganciclovir (VGC)
Other Intervention Name(s)
Valcyte
Intervention Description
Valganciclovir (VGC) 900 mg orally q12 hours (every 12 hours) on days 1-5 of a 14-day cycle.
Primary Outcome Measure Information:
Title
Percentage of Participants With an Overall Clinical Benefit Response
Description
Overall clinical benefit response is defined as Complete Response (CR): Full resolution of all clinical symptoms and laboratory abnormalities (whether or not these are indicator abnormalities) probably or definitely attributable to MCD, lasting at least 3 weeks; and Partial Response (PR): At least 50% of the abnormalities probably or definitely attributed to KSHV-MCD must improve by the minimum amounts specified to attain PR. Only abnormalities present in a specific patient at baseline may count toward the achievement of a PR (e.g. if six of indicator abnormalities are present at baseline, at least three must meet the specified criteria to be considered a PR) assessed using a modified Kaposi sarcoma herpes virus-associated multicentric Castleman disease (KSHV- MCD) Clinical Benefit Response Criteria and the National Cancer Institute (NCI) KSHV-MCD criteria.
Time Frame
every 2 weeks for up to 12 weeks
Secondary Outcome Measure Information:
Title
Percentage of Participants With a Clinical Response
Description
Clinical response is defined as a Complete Response (CR): Full resolution of all signs and symptoms attributable to MCD, lasting 1 cycle (3-4 weeks depending on regimen; Symptom Free Disease (SFD:) Full resolution of all signs and symptoms attributable to MCD, not yet lasting 1 cycle (3-4 weeks depending on regimen); and Partial Response (PR): Improvement in at least 50% of signs and symptoms by at least 1 grade (NCI-Common Terminology Criteria for Adverse Events (CTCAE v4), with no increased MCD related increases, lasting 1 cycle (3-4 weeks depending on regimen) assessed by the National Cancer Institute Kaposi sarcoma herpes virus-associated multicentric Castleman disease (NCI KSHV-MCD) Response Criteria.
Time Frame
up to 12 weeks
Title
Percentage of Participants With a Biochemical Response
Description
A biochemical response is defined as a Complete Response (CR): Normalization of abnormalities attributed to MCD in the following labs: Complete blood count (CBC), Chem 20, C-Reactive protein (CRP), lasting 1 cycle (3-4 weeks depending on regimen); Partial Response (PR): 50% improvement in all labs abnormalities attributable to MCD, lasting 1 cycle (3-4 weeks depending on regimen), and was assessed by the National Cancer Institute (NCI) Kaposi sarcoma herpes virus-associated multicentric Castleman Disease (KSHV-MCD) Response Criteria.
Time Frame
up to 12 weeks
Title
Percentage of Participants With a Radiographic Response
Description
A radiographic response is defined as a Complete Response (CR): Normalization of all lymph nodes to <1.5 cm in greatest transverse dimension, decrease to < 1 cm of lymph nodes 1.1-1.5 cm at baseline (or 75% decrease in the sum of products of diameters (SPD)), Spleen < 12 cm greatest dimension, no pleural effusions; Complete Response unconfirmed (CRu): Residual lymph node mass >1.5 cm or splenomegaly > 12 cm that has decrease by >75% and does not change over one year; and Partial Response (PR): For lymph nodes, >50% decrease in SPD of 6 dominant nodes, for spleen 50% decrease in longest transverse dimension assessed by the National Cancer Institute (NCI) Kaposi sarcoma herpes virus-associated multicentric Castleman Disease (KSHV-MCD) Response Criteria.
Time Frame
up to 12 weeks
Title
Percentage of Participants With Kaposi Sarcoma Responses Per the Acquired Immunodeficiency Syndrome (AIDS) Clinical Trials Group (ACTG) Response Criteria
Description
The ACTG Criteria is defined as a Complete Response (CR), Partial Response (PR), Stable Disease (SD), Progressive Disease (PD), and Symptom Free Disease (SFD). The terms Improved (I), Stable (S), Mixed (M) Response, and Worse (W) are also used to further describe participants who have SD or PR.
Time Frame
Baseline, week 7, and at off study visit, approximately 2 weeks following last study treatment for those with KS, up to 14 weeks.
Title
Changes in Plasma Exposure of Ritonavir in Response to Tocilizumab and Zidovudine (AZT) Metabolized by Cytochrome P450 (CYP450)
Description
Cumulative plasma exposure of Ritonavir will be evaluated.
Time Frame
Cycle 1 Day 1: pre Tocilizumab, and 15 minutes, 24 hrs and 48 hrs post drug; Cycle 2-6: pre Tocilizumab dose and 15 minutes post drug dose.
Title
Changes in Plasma Exposure of Lopinavir in Response to Tocilizumab and AZT Metabolized by Cytochrome P450 (CYP450)
Description
Cumulative plasma exposure of Lopinavir will be evaluated.
Time Frame
Cycle 1 Day 1: pre Tocilizumab, and 15 minutes, 24 hrs and 48 hrs post drug; Cycle 2-6: pre Tocilizumab dose and 15 minutes post drug dose.
Title
Changes in Plasma Exposure of Atazanavir in Response to Tocilizumab and Zidovudine (AZT) Metabolized by Cytochrome P450 (CYP450)
Description
Cumulative plasma exposure of Atazanavir will be evaluated.
Time Frame
Cycle 1 Day 1: pre Tocilizumab, and 15 minutes, 24 hrs and 48 hrs post drug; Cycle 2-6: pre Tocilizumab dose and 15 minutes post drug dose.
Title
Changes in Plasma Exposure of Efavirenz in Response to Tocilizumab and Zidovudine (AZT) Metabolized by Cytochrome P450 (CYP450)
Description
Cumulative plasma exposure of Efavirenz will be evaluated.
Time Frame
Cycle 1 Day 1: pre Tocilizumab, and 15 minutes, 24 hrs and 48 hrs post drug; Cycle 2-6: pre Tocilizumab dose and 15 minutes post drug dose.
Title
Percentage of Participants With Grade 3 or Greater Serious Adverse Events
Description
Here is the percentage of participants with Grade 3 or greater serious adverse events assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0). A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life-threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned. Grade 3 is severe or medically significant, Grade 4 is life threatening, and Grade 5 is death.
Time Frame
each cycle, up to 6 years, 8 months and 24 days.
Title
Percentage of Participants With <Grade 3 Non- Serious Adverse Events
Description
Here is the percentage of participants with <Grade 3 non-serious adverse events assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0). A non-serious adverse event is any untoward medical occurrence. Grade 1 is mild and Grade 2 is moderate.
Time Frame
each cycle, up to 6 years, 8 months and 24 days.
Title
Number of Participants With Serious and Non-serious Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v4.0)
Description
Here is the count of participants with serious and non-serious adverse events assessed by the Common Terminology Criteria for Adverse Events (CTCAE v4.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life-threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned.
Time Frame
Date treatment consent signed to date off study, approximately 6 years, 8 months and 24 days.
Title
Effect of Tocilizumab on the Pharmacokinetics (PK) of Antiretroviral Agents
Description
Evaluate the effect of tocilizumab on the pharmacokinetics of antiretroviral agents that are cytochrome P3A4 (CYP3A4) substrates in patients with symptomatic Kaposi sarcoma herpes virus-associated multicentric Castleman Disease (KSHV-MCD).
Time Frame
Cycle 1, Day 1 and Cycle 2-6 Day 1
Title
Percentage of Participants Progression-free Survival at 4 Months
Description
Progression-free survival is defined as participants who progress or die by 4 months after the start of treatment with tocilizumab and tocilizumab /AZT/VGC.
Time Frame
4 months
Title
Percentage of Participants With Overall Survival 4 Months After Treatment With Tocilizumab and Tocilizumab /Zidovudine (AZT)/Valganciclovir (VGC)
Description
Overall survival is defined as the percentage of participants alive at 4 months after the start of treatment with tocilizumab and tocilizumab /AZT/VGC.
Time Frame
4 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
INCLUSION CRITERIA:
Pathologically confirmed Kaposi sarcoma (KS)-associated herpes virus multi-centric Castleman disease (KSHV-MCD)
Age greater than or equal to 18
At least one clinical symptom probably or definitely attributed to KSHV-MCD
Intermittent or persistent fever for at least 1 week (>38 degrees C)
Fatigue (Common Terminology Criteria for Adverse Events (CTCAE) Grade 2 or greater)
Gastrointestinal symptoms [includes nausea and anorexia] (CTCAE Grade 1 or greater)
Respiratory symptoms [includes cough and airway hyperreactivity]
(CTCAE Grade 1 or greater)
At least one laboratory abnormality probably or definitely attributed to KSHVMCD
Anemia (Hgb [men] </=12.5 gm/dL, Hgb [women] </= 11 gm/dL)
Thrombocytopenia (</=130,000/mm(3))
Hypoalbuminemia (<3.4 g/dl)
Elevated C-reactive protein (CRP) (CRP > 3 mg/L)] probably or definitely attributable to KSHV-MCD
No life- or organ-threatening manifestations of MCD
Eastern Cooperative Oncology Group (ECOG) performance status less than or equal to 2
Human Immunodeficiency Virus (HIV)-infected patients should be receiving or willing to initiate an effective combination antiretroviral therapy (cART) regimen
Willingness to complete tuberculosis evaluation and start prophylactic antituberculosis therapy as soon as is medically feasible if patients have a reactive tuberculin skin test and have not completed an adequate course of prevented anti-tuberculosis therapy, following American Thoracic Society/ Centers for Disease Control recommended guidelines:
http://www.cdc.gov/mmwr/preview/mmwrhtml/mm5231a4.htm
Ability to understand and willingness to give informed consent
Women of child bearing potential must agree to use birth control for the duration of the study
EXCLUSION CRITERIA:
Uncontrolled bacterial, mycobacterial, or fungal infection
Uncontrolled intercurrent illness including, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements or ability to receive therapy.
Pregnant or lactating women
Any abnormality that would be scored as National Cancer Institute (NCI) Common Toxicity Criteria (CTC) Grade 3 toxicity that is unrelated to HIV, its treatment, or to MCD that would preclude protocol treatment. Exceptions include:
Lymphopenia
Direct manifestations of Kaposi sarcoma or MCD
Direct manifestation of HIV (i.e. low cluster of differentiation 4 (CD4) count)
Direct manifestation of HIV therapy (i.e. Hyperbilirubinemia associated with protease inhibitors)
Asymptomatic hyperuricemia
Hypophosphatemia
Elevated creatine kinase (CK) attributed to exercise
Past or present history of malignant tumors other than Kaposi sarcoma unless one of the following:
Complete remission for greater than or equal to 1 year from completion of therapy
Completely resected basal cell carcinoma
In situ squamous cell carcinoma of the cervix or anus
Patients with concurrent Kaposi sarcoma requiring immediate cytotoxic chemotherapy
History of tocilizumab therapy within prior three months
History of rituximab or bevacizumab therapy within three months
History of greater than or equal to 2 allergic reaction or any grade anaphylactic reaction during prior administration of tocilizumab
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Robert Yarchoan, M.D.
Organizational Affiliation
National Cancer Institute (NCI)
Official's Role
Principal Investigator
Facility Information:
Facility Name
National Institutes of Health Clinical Center, 9000 Rockville Pike
City
Bethesda
State/Province
Maryland
ZIP/Postal Code
20892
Country
United States
12. IPD Sharing Statement
Plan to Share IPD
No
Citations:
PubMed Identifier
25331113
Citation
Uldrick TS, Polizzotto MN, Aleman K, Wyvill KM, Marshall V, Whitby D, Wang V, Pittaluga S, O'Mahony D, Steinberg SM, Little RF, Yarchoan R. Rituximab plus liposomal doxorubicin in HIV-infected patients with KSHV-associated multicentric Castleman disease. Blood. 2014 Dec 4;124(24):3544-52. doi: 10.1182/blood-2014-07-586800. Epub 2014 Oct 20.
Results Reference
background
PubMed Identifier
20583924
Citation
Uldrick TS, Wang V, O'Mahony D, Aleman K, Wyvill KM, Marshall V, Steinberg SM, Pittaluga S, Maric I, Whitby D, Tosato G, Little RF, Yarchoan R. An interleukin-6-related systemic inflammatory syndrome in patients co-infected with Kaposi sarcoma-associated herpesvirus and HIV but without Multicentric Castleman disease. Clin Infect Dis. 2010 Aug 1;51(3):350-8. doi: 10.1086/654798.
Results Reference
background
PubMed Identifier
2384605
Citation
Brandt SJ, Bodine DM, Dunbar CE, Nienhuis AW. Dysregulated interleukin 6 expression produces a syndrome resembling Castleman's disease in mice. J Clin Invest. 1990 Aug;86(2):592-9. doi: 10.1172/JCI114749.
Results Reference
background
PubMed Identifier
32276276
Citation
Ramaswami R, Lurain K, Peer CJ, Serquina A, Wang V, Widell A, Goncalves P, Steinberg SM, Marshall V, George J, Figg WD, Whitby D, Ziegelbauer J, Uldrick TS, Yarchoan R. Tocilizumab in patients with symptomatic Kaposi sarcoma herpesvirus-associated multicentric Castleman disease. Blood. 2020 Jun 18;135(25):2316-2319. doi: 10.1182/blood.2019004602. No abstract available.
Results Reference
result
Links:
URL
https://clinicalstudies.info.nih.gov/cgi/detail.cgi?B_2011-C-0233.html
Description
NIH Clinical Center Detailed Web Page
Learn more about this trial
Tocilizumab for KSHV-Associated Multicentric Castleman Disease
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