Natural History Study of the KSHV Inflammatory Cytokine Syndrome (KICS)

NCT ID: NCT01419561

Last Updated: 2025-12-24

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE2

Total Enrollment

140 participants

Study Classification

INTERVENTIONAL

Study Start Date

2011-09-08

Study Completion Date

2028-12-31

Brief Summary

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Background:

\- KSHV inflammatory cytokine syndrome (KICS) is a newly recognized disease caused by Kaposi sarcoma-associated herpesvirus (KSHV). This virus can cause cancer. People with KICS can have severe symptoms. They include fever, weight loss, and fluid in the legs or abdomen. People with KICS may also be at risk of getting other cancers associated with KSHV. These cancers include Kaposi sarcoma and lymphoma. Because KICS is a newly identified disease, more information is needed on how the disease works and what can be done to treat it.

Objectives:

\- To collect genetic and medical information from people with KSHV inflammatory cytokine syndrome.

Eligibility:

\- Individuals at least 18 years of age who have Kaposi sarcoma herpes virus and symptoms that resemble those caused by KICS.

Design:

* Participants will have regular study visits. The schedule will be determined by the study researchers.
* Participants will provide a complete medical history and have a full physical exam. Blood and urine samples will be collected as well.
* People with KICS that requires treatment may get new experimental treatments. These treatments may include antiviral drugs and chemotherapy drugs, depending on the nature of the disease.
* Participants will have imaging studies, such as chest x-rays and computed tomography scans, to study the tumors.
* Bone marrow and lymph node biopsies may be done to collect tissue samples for study.
* Participants who have Kaposi sarcoma will have photographs taken of their lesions.

Detailed Description

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Background

-Kaposi sarcoma-associated herpesvirus (KSHV) inflammatory cytokine syndrome (KICS) is a newly recognized syndrome caused by KSHV. It is characterized by severe inflammatory symptoms including fevers, wasting, cytopenias, hypoalbuminemia, and hyponatremia, associated in some cases with lymphadenopathy or effusions, without pathological evidence of MCD. Patients with KICS exhibit elevated KSHV viral loads and cytokine dysregulation, with elevations of IL-6, IL-10, and a KSHV-encoded IL-6 homolog, viral IL-6.

Objective

-Assessment of the natural history of KSHV inflammatory cytokine syndrome (KICS), including the spectrum of clinical, laboratory and radiographic abnormalities seen in affected participants.

Eligibility

* Adults of any HIV status with:

* At least two symptoms, laboratory or radiographic abnormalities which are at least possibly attributable to KICS (including fever, fatigue, cachexia, edema, respiratory or gastrointestinal symptoms, hematologic cytopenias, hypoalbuminemia, hyponatremia, lymphadenopathy, organomegaly, effusions)
* C-reactive protein \>3mg/L
* Evidence of KSHV infection or a risk exposure for KSHV infection
* No evidence of KSHV-associated multicentric Castleman disease
* Participants with these characteristics will be further evaluated to identify those whose clinical and laboratory features are consistent with the KICS working case definition to be followed in the natural history phase of the study.

Design

-This is a single center natural history study with a cohort of up to 120 evaluable participants. Participants who meet the criteria for KICS will go onto a natural history arm, which permits observation for KICS with or without other concurrent KSHV-associated disorders. Participants who require KS and/or primary effusion lymphoma (PEL) treatment along with a KICS diagnosis will receive rational therapies for these conditions or be treated for their KS and/or PEL on a separate protocol while still followed on this study.

Conditions

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KSHV Inflammatory Cytokine Syndrome (KICS) KSHV HHV-8

Keywords

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KSHV KICS HIV Cytokines HHV-8

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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1

Evaluation for Alternative Causes of KICS Symptoms (inactive)

Group Type NO_INTERVENTION

No interventions assigned to this group

2

Natural history/Observation arm (inactive)

Group Type NO_INTERVENTION

No interventions assigned to this group

3

High dose zidovudine + valganciclovir (inactive)

Group Type EXPERIMENTAL

Zidovudine

Intervention Type DRUG

Zidovudine 600 mg will be administered orally 4 times a day or i.v. at 300 mg every 6 hours for 14 days for cycle 1 and for 7 days (up to additional 7 days if ongoing symptoms) for following cycles.

Valganiclovir

Intervention Type DRUG

Valganciclovir (900mg) will be administered orally twice/day or Ganciclovir (5 mg/kg) will be administered i.v. over 1 hour for 14 days for cycle 1 and for 7 days (up to additional 7 days if ongoing symptoms) for following cycles.

4

Rituximab with or without liposomal doxorubicin (inactive)

Group Type EXPERIMENTAL

Liposomal Doxorubicin

Intervention Type DRUG

Liposomal doxorubicin (20 mg/m2) will be administered i.v. over 1 hour at day 1 of each cycle

Rituximab

Intervention Type DRUG

Rituximab (375 mg/m2) will be admnistered i.v. at 50 mg/hr up to 100 mg/hr at day 1 of the first cycle and at 100mg/hr up to 400 mg

/hr at day 1 of following cycles.

5

Standard and alternative rational therapies (inactive)

Group Type OTHER

Standard Therapies

Intervention Type OTHER

Standard of Care drugs

6

Natural history

Group Type OTHER

Cohort 1

Intervention Type OTHER

Participants who are infected with KSHV who meet criteria for inflammatory cytokine syndrome (KICS)

Interventions

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Zidovudine

Zidovudine 600 mg will be administered orally 4 times a day or i.v. at 300 mg every 6 hours for 14 days for cycle 1 and for 7 days (up to additional 7 days if ongoing symptoms) for following cycles.

Intervention Type DRUG

Liposomal Doxorubicin

Liposomal doxorubicin (20 mg/m2) will be administered i.v. over 1 hour at day 1 of each cycle

Intervention Type DRUG

Valganiclovir

Valganciclovir (900mg) will be administered orally twice/day or Ganciclovir (5 mg/kg) will be administered i.v. over 1 hour for 14 days for cycle 1 and for 7 days (up to additional 7 days if ongoing symptoms) for following cycles.

Intervention Type DRUG

Rituximab

Rituximab (375 mg/m2) will be admnistered i.v. at 50 mg/hr up to 100 mg/hr at day 1 of the first cycle and at 100mg/hr up to 400 mg

/hr at day 1 of following cycles.

Intervention Type DRUG

Standard Therapies

Standard of Care drugs

Intervention Type OTHER

Cohort 1

Participants who are infected with KSHV who meet criteria for inflammatory cytokine syndrome (KICS)

Intervention Type OTHER

Eligibility Criteria

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Inclusion Criteria

* Age greater than or equal to18 Years.
* Any HIV status.
* At least two manifestations drawn from at least two of the categories (clinical symptoms, laboratory abnormalities and/or radiographic abnormalities), which are at least possibly attributable to KICS and are not readily explicable from known medical conditions in the participant:
* Clinical symptoms (each at least grade 1 by CTCAE definitions)
* Fever (\>38 degrees C), chills or rigors
* Fatigue or lethargy
* Cachexia or edema
* Cough, dyspnea, airway hyperreactivity, or nasal inflammation
* Nausea, anorexia, abdominal pain or altered bowel habit
* Athralgia or myalgia
* Altered mental state
* Neuropathy with or without pain
* Laboratory abnormalities
* Anemia (hemoglobin\<12.0g/dL)
* Thrombocytopenia (platelets\<100,000 cells/microL)
* Leukopenia (white cell count\<4,000 cells/microL)
* Hypoalbuminemia (albumin\<3.5g/dL)
* Hyponatremia (sodium\<135mmol/L)
* Coagulopathy (PT or PTT \>1.5 times upper limit of normal)
* Radiographic Abnormalities
* Pathologic lymphadenopathy (at least five discrete nodes each \>1cm in their longest dimension)
* Splenomegaly (\>12 cm in the longest dimension)
* Hepatomegaly (\>17cm in the longest dimension)
* Body cavity effusions not caused by primary effusion lymphoma nor chylous effusions directly related to lymphatic infiltration by KS
* C-reactive protein (CRP) \>3mg/L.
* Exposure risk for KSHV infection (including being a first or second generation immigrant from an endemic area, or male-to-male sexual activity) or evidence of KSHV infection demonstrated by one of:

* Molecular evidence of KSHV in whole blood, or KSHV VL levels within circulating PBMCs as determined by the Whitby laboratory
* Immunohistochemical evidence of KSHV in tissues (for example by staining for LANA or vIL-6) confirmed in the Laboratory of Pathology (LP), CCR, NCI.
* Presence of KS or PEL (KSHV-associated malignancies), confirmed in the LP, CCR, NCI.
* Women of child-bearing potential (WOCBP) and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry, for the duration of study participation, and after treatment (if received), according to drug requirements. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately.

Exclusion Criteria

\- Biopsy proven KSHV-associated MCD, confirmed in the LP, CCR, NCI.

Note: In collaboration with LP, we have recently found that some participants with KICS but without a lymph node or splenic diagnosis of MCD have MCD-like cells in their effusions or circulating blood. This may in fact represent a newly recognized form of KSHV-MCD, but our analysis continues. While certain of these participants were historically included in this study, given this new understanding, they will not be entered on this protocol and removed if liquid MCD is diagnosed.

* Pregnancy
* Any abnormality that would be scored as NCI CTC Grade 4 toxicity that is unrelated to HIV, its treatment, or to KICS that would preclude the use of all of the study treatments or the ability to monitor the natural history of KICS untreated.
* Any condition or set of circumstances that in the opinion of the investigators would make participation in this study unsafe or otherwise inappropriate for a given individual.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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National Cancer Institute (NCI)

NIH

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Robert Yarchoan, M.D.

Role: PRINCIPAL_INVESTIGATOR

National Cancer Institute (NCI)

Locations

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National Institutes of Health Clinical Center

Bethesda, Maryland, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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Anaida Widell

Role: CONTACT

Phone: (240) 760-6074

Email: [email protected]

Robert Yarchoan, M.D.

Role: CONTACT

Phone: (240) 760-6075

Email: [email protected]

References

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Chang Y, Cesarman E, Pessin MS, Lee F, Culpepper J, Knowles DM, Moore PS. Identification of herpesvirus-like DNA sequences in AIDS-associated Kaposi's sarcoma. Science. 1994 Dec 16;266(5192):1865-9. doi: 10.1126/science.7997879.

Reference Type BACKGROUND
PMID: 7997879 (View on PubMed)

Moore PS, Gao SJ, Dominguez G, Cesarman E, Lungu O, Knowles DM, Garber R, Pellett PE, McGeoch DJ, Chang Y. Primary characterization of a herpesvirus agent associated with Kaposi's sarcomae. J Virol. 1996 Jan;70(1):549-58. doi: 10.1128/JVI.70.1.549-558.1996.

Reference Type BACKGROUND
PMID: 8523568 (View on PubMed)

Viejo-Borbolla A, Schulz TF. Kaposi's sarcoma-associated herpesvirus (KSHV/HHV8): key aspects of epidemiology and pathogenesis. AIDS Rev. 2003 Oct-Dec;5(4):222-9.

Reference Type BACKGROUND
PMID: 15012001 (View on PubMed)

Ramaswami R, Lurain K, Polizzotto MN, Widell A, Ekwede I, Tassi E, Rupert A, Marshall VA, Roth MJ, Filie AC, Pittaluga S, Jaffe ES, Wang HW, Whitby D, Uldrick TS, Yarchoan R. Characteristics and outcomes of Kaposi sarcoma herpesvirus-associated inflammatory cytokine syndrome. Blood Adv. 2025 Nov 25;9(22):5720-5731. doi: 10.1182/bloodadvances.2025016685.

Reference Type DERIVED
PMID: 40763275 (View on PubMed)

Lage SL, Ramaswami R, Rocco JM, Rupert A, Davis DA, Lurain K, Manion M, Whitby D, Yarchoan R, Sereti I. Inflammasome activation in patients with Kaposi sarcoma herpesvirus-associated diseases. Blood. 2024 Oct 3;144(14):1496-1507. doi: 10.1182/blood.2024024144.

Reference Type DERIVED
PMID: 38941593 (View on PubMed)

Related Links

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Other Identifiers

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11-C-0220

Identifier Type: -

Identifier Source: secondary_id

110220

Identifier Type: -

Identifier Source: org_study_id