Trial Outcomes & Findings for Nelfinavir Mesylate in Treating Patients With Kaposi Sarcoma (NCT NCT03077451)

Last Updated: 2025-12-17

Results Overview

The binomial proportion and its exact 95% confidence interval will be used to estimate the overall response rate in the study. Overall response is defined as Complete response or partial response. Response and progression will be evaluated in this study using the AIDS Clinical Trials Groups (ACTG) response criteria for Kaposi sarcoma. Complete response (CR) is defined as the absence of any detectable residual disease, including tumor-associated edema, persisting for at least 4 weeks. Partial response (PR) is defined as no new lesions (skin or oral), or new visceral sites of involvement (or the appearance or worsening of tumor-associated edema or effusions); 50% or greater decrease in the number of all previously existing lesions lasting for at least 4 weeks; OR Complete flattening of at least 50% of all previously raised lesions; OR 50% decrease in the sum of the products of the largest perpendicular diameters of the marker lesions.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

36 participants

Primary outcome timeframe

Baseline up to 16 weeks

Results posted on

2025-12-17

Participant Flow

Participant milestones

Participant milestones
Measure	Treatment (Nelfinavir Mesylate) DOSE NELFINAVIR MESYLATE: Patients receive standard dose nelfinavir mesylate PO BID for 4 weeks in the absence of PD. Patients with PD at 4 weeks proceed to high-dose nelfinavir. At week 8, if there is SD or PR, patients advance to high-dose nelfinavir mesylate. Patients discontinue standard dose nelfinavir mesylate 4 weeks after documentation of CR. HIGH DOSE NELFINAVIR MESYLATE: Patients with PD continue to receive high-dose nelfinavir mesylate PO BID for 4 more weeks. If there is PD documented after 4 weeks at the high dose level, nelfinavir is discontinued. If there is SD or PR, patients continue receiving nelfinavir mesylate for 16 weeks. If there is CR, patients discontinue high-dose nelfinavir mesylate 4 weeks after documentation of CR. Laboratory Biomarker Analysis: Correlative studies Nelfinavir Mesylate: Given PO
Overall Study STARTED	36
Overall Study COMPLETED	20
Overall Study NOT COMPLETED	16

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Nelfinavir Mesylate in Treating Patients With Kaposi Sarcoma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Treatment (Nelfinavir Mesylate) n=36 Participants DOSE NELFINAVIR MESYLATE: Patients receive standard dose nelfinavir mesylate PO BID for 4 weeks in the absence of PD. Patients with PD at 4 weeks proceed to high-dose nelfinavir. At week 8, if there is SD or PR, patients advance to high-dose nelfinavir mesylate. Patients discontinue standard dose nelfinavir mesylate 4 weeks after documentation of CR. HIGH DOSE NELFINAVIR MESYLATE: Patients with PD continue to receive high-dose nelfinavir mesylate PO BID for 4 more weeks. If there is PD documented after 4 weeks at the high dose level, nelfinavir is discontinued. If there is SD or PR, patients continue receiving nelfinavir mesylate for 16 weeks. If there is CR, patients discontinue high-dose nelfinavir mesylate 4 weeks after documentation of CR. Laboratory Biomarker Analysis: Correlative studies Nelfinavir Mesylate: Given PO
Age, Continuous	52 years n=6 Participants
Sex: Female, Male Female	2 Participants n=6 Participants
Sex: Female, Male Male	34 Participants n=6 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	8 Participants n=6 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	27 Participants n=6 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	1 Participants n=6 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=6 Participants
Race (NIH/OMB) Asian	0 Participants n=6 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=6 Participants
Race (NIH/OMB) Black or African American	12 Participants n=6 Participants
Race (NIH/OMB) White	21 Participants n=6 Participants
Race (NIH/OMB) More than one race	0 Participants n=6 Participants
Race (NIH/OMB) Unknown or Not Reported	3 Participants n=6 Participants
Race/Ethnicity, Customized Non-Hispanic White	15 Participants n=6 Participants
Race/Ethnicity, Customized Non-Hispanic Black	11 Participants n=6 Participants
Race/Ethnicity, Customized Hispanic	8 Participants n=6 Participants
Race/Ethnicity, Customized Unknown	2 Participants n=6 Participants
HIV status HIV positive	30 Participants n=6 Participants
HIV status HIV negative	6 Participants n=6 Participants

PRIMARY outcome

Timeframe: Baseline up to 16 weeks

Population: Among 30 HIV-positive patients, there are 25 HIV-positive who were treated with high dose.

Outcome measures

Outcome measures
Measure	Treatment (Nelfinavir Mesylate) n=25 Participants DOSE NELFINAVIR MESYLATE: Patients receive standard dose nelfinavir mesylate PO BID for 4 weeks in the absence of PD. Patients with PD at 4 weeks proceed to high-dose nelfinavir. At week 8, if there is SD or PR, patients advance to high-dose nelfinavir mesylate. Patients discontinue standard dose nelfinavir mesylate 4 weeks after documentation of CR. HIGH DOSE NELFINAVIR MESYLATE: Patients with PD continue to receive high-dose nelfinavir mesylate PO BID for 4 more weeks. If there is PD documented after 4 weeks at the high dose level, nelfinavir is discontinued. If there is SD or PR, patients continue receiving nelfinavir mesylate for 16 weeks. If there is CR, patients discontinue high-dose nelfinavir mesylate 4 weeks after documentation of CR. Laboratory Biomarker Analysis: Correlative studies Nelfinavir Mesylate: Given PO	Standard Dose Treatment (Nelfinavir Mesylate) This study is not parallel arm study. Nelfinavir dose will be administrated in the sequential manner according to the response. AE summary is based on participants who received standard dose. There were totally 36 participants received standard dose initally. All patients will received standard dose of nelfinavir (1250 mg BID) for at least 4 weeks. If there is complete response (CR) at any time at the standard dose, nelfinavir will be discontinued 4 weeks after documentation of CR.
Efficacy of Therapeutic Escalation of Standard Dose Nelfinavir, Followed by High Dose Nelfinavir, for Treatment of Kaposi Sarcoma Lesions for HIV Positive Participants	32 percentage of overall response patients Interval 14.9 to 53.5	—

PRIMARY outcome

Timeframe: Baseline up to 16 weeks

Population: There are 6 HIV-negative who were treated with high dose.

The binomial proportion and its exact 95% confidence interval will be used to estimate the response rate in the study. Response rate will be estimated using the binomial proportion and it's exact 95% confidence interval for HIV negative participants.

Outcome measures

Outcome measures
Measure	Treatment (Nelfinavir Mesylate) n=6 Participants DOSE NELFINAVIR MESYLATE: Patients receive standard dose nelfinavir mesylate PO BID for 4 weeks in the absence of PD. Patients with PD at 4 weeks proceed to high-dose nelfinavir. At week 8, if there is SD or PR, patients advance to high-dose nelfinavir mesylate. Patients discontinue standard dose nelfinavir mesylate 4 weeks after documentation of CR. HIGH DOSE NELFINAVIR MESYLATE: Patients with PD continue to receive high-dose nelfinavir mesylate PO BID for 4 more weeks. If there is PD documented after 4 weeks at the high dose level, nelfinavir is discontinued. If there is SD or PR, patients continue receiving nelfinavir mesylate for 16 weeks. If there is CR, patients discontinue high-dose nelfinavir mesylate 4 weeks after documentation of CR. Laboratory Biomarker Analysis: Correlative studies Nelfinavir Mesylate: Given PO	Standard Dose Treatment (Nelfinavir Mesylate) This study is not parallel arm study. Nelfinavir dose will be administrated in the sequential manner according to the response. AE summary is based on participants who received standard dose. There were totally 36 participants received standard dose initally. All patients will received standard dose of nelfinavir (1250 mg BID) for at least 4 weeks. If there is complete response (CR) at any time at the standard dose, nelfinavir will be discontinued 4 weeks after documentation of CR.
Efficacy of Therapeutic Escalation of Standard Dose Nelfinavir, Followed by High Dose Nelfinavir, for Treatment of Kaposi Sarcoma Lesions for HIV Negative Participants	16.7 percentage of overall response patients Interval 0.4 to 64.1	—

SECONDARY outcome

Timeframe: Baseline to up to 16 weeks

Population: High dose of nelfinavir after standard dose of nelfinavir in the treatment of KS in participant who are HIV-positive. Overall Number of Participants Analyzed signifies all participants received high does of nelfinavir assessed for adverse events. Number Analyzed represents the number of participants affected with the total number of observed adverse events reported.

Frequency of adverse events and their severity will be tabulated to evaluate tolerance of high dose nelfinavir in the treatment of KS in participant who are HIV-positive

Outcome measures

Outcome measures
Measure	Treatment (Nelfinavir Mesylate) n=25 Participants DOSE NELFINAVIR MESYLATE: Patients receive standard dose nelfinavir mesylate PO BID for 4 weeks in the absence of PD. Patients with PD at 4 weeks proceed to high-dose nelfinavir. At week 8, if there is SD or PR, patients advance to high-dose nelfinavir mesylate. Patients discontinue standard dose nelfinavir mesylate 4 weeks after documentation of CR. HIGH DOSE NELFINAVIR MESYLATE: Patients with PD continue to receive high-dose nelfinavir mesylate PO BID for 4 more weeks. If there is PD documented after 4 weeks at the high dose level, nelfinavir is discontinued. If there is SD or PR, patients continue receiving nelfinavir mesylate for 16 weeks. If there is CR, patients discontinue high-dose nelfinavir mesylate 4 weeks after documentation of CR. Laboratory Biomarker Analysis: Correlative studies Nelfinavir Mesylate: Given PO	Standard Dose Treatment (Nelfinavir Mesylate) This study is not parallel arm study. Nelfinavir dose will be administrated in the sequential manner according to the response. AE summary is based on participants who received standard dose. There were totally 36 participants received standard dose initally. All patients will received standard dose of nelfinavir (1250 mg BID) for at least 4 weeks. If there is complete response (CR) at any time at the standard dose, nelfinavir will be discontinued 4 weeks after documentation of CR.
Frequency and Severity of Adverse Events in HIV-positive Participants as Assessed by CTCAE v4.0 Hyperglycemia	3 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-positive Participants as Assessed by CTCAE v4.0 Hypertriglyceridemia	5 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-positive Participants as Assessed by CTCAE v4.0 Arthralgia	2 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-positive Participants as Assessed by CTCAE v4.0 Back Pain	2 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-positive Participants as Assessed by CTCAE v4.0 Musculoskeletal and Connective Tissue DIsorder	2 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-positive Participants as Assessed by CTCAE v4.0 Myalgia	2 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-positive Participants as Assessed by CTCAE v4.0 Pain in Extremity	5 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-positive Participants as Assessed by CTCAE v4.0 Tumor Pain	2 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-positive Participants as Assessed by CTCAE v4.0 Dizziness	2 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-positive Participants as Assessed by CTCAE v4.0 Headache	2 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-positive Participants as Assessed by CTCAE v4.0 Insomnia	2 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-positive Participants as Assessed by CTCAE v4.0 Cough	6 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-positive Participants as Assessed by CTCAE v4.0 Nasal Congestion	3 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-positive Participants as Assessed by CTCAE v4.0 Respiratory, Thoracic and Medastinal Disorders	3 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-positive Participants as Assessed by CTCAE v4.0 Sore Throat	4 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-positive Participants as Assessed by CTCAE v4.0 Dry Skin	2 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-positive Participants as Assessed by CTCAE v4.0 Pruritus	3 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-positive Participants as Assessed by CTCAE v4.0 Rash Maculo-papular	2 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-positive Participants as Assessed by CTCAE v4.0 Skin and Subcutaneous Tissue Disorders	4 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-positive Participants as Assessed by CTCAE v4.0 Hypertension	13 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-positive Participants as Assessed by CTCAE v4.0 Anemia	7 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-positive Participants as Assessed by CTCAE v4.0 Edema Limbs	6 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-positive Participants as Assessed by CTCAE v4.0 Blood and Lymphatic System Disorder	2 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-positive Participants as Assessed by CTCAE v4.0 Abdominal Pain	3 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-positive Participants as Assessed by CTCAE v4.0 Constipation	2 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-positive Participants as Assessed by CTCAE v4.0 Diarrhea	42 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-positive Participants as Assessed by CTCAE v4.0 Nausea	7 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-positive Participants as Assessed by CTCAE v4.0 Vomiting	2 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-positive Participants as Assessed by CTCAE v4.0 Fever	3 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-positive Participants as Assessed by CTCAE v4.0 General Disorders and Administration Site Conditions	7 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-positive Participants as Assessed by CTCAE v4.0 Pain	6 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-positive Participants as Assessed by CTCAE v4.0 Herpes Simplex reactivation	3 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-positive Participants as Assessed by CTCAE v4.0 Infections and Infestations	6 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-positive Participants as Assessed by CTCAE v4.0 Injury, Poisoning and Procedural Complications	4 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-positive Participants as Assessed by CTCAE v4.0 Neutrophil Count Decreased	5 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-positive Participants as Assessed by CTCAE v4.0 White Blood Cell Decreased	3 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-positive Participants as Assessed by CTCAE v4.0 Anorexia	2 Number of adverse events	—

SECONDARY outcome

Timeframe: Baseline to up to 16 weeks

Population: High dose of Nelfinavir after standard dose among HIV-negative participants. Overall Number of Participants Analyzed signifies all participants received high does of nelfinavir assessed for adverse events. Number Analyzed represents the number of participants affected with the total number of observed adverse events reported.

Frequency of adverse events and their severity will be tabulated to evaluate tolerance of high dose nelfinavir in the treatment of KS in participant who are HIV-negative

Outcome measures

Outcome measures
Measure	Treatment (Nelfinavir Mesylate) n=6 Participants DOSE NELFINAVIR MESYLATE: Patients receive standard dose nelfinavir mesylate PO BID for 4 weeks in the absence of PD. Patients with PD at 4 weeks proceed to high-dose nelfinavir. At week 8, if there is SD or PR, patients advance to high-dose nelfinavir mesylate. Patients discontinue standard dose nelfinavir mesylate 4 weeks after documentation of CR. HIGH DOSE NELFINAVIR MESYLATE: Patients with PD continue to receive high-dose nelfinavir mesylate PO BID for 4 more weeks. If there is PD documented after 4 weeks at the high dose level, nelfinavir is discontinued. If there is SD or PR, patients continue receiving nelfinavir mesylate for 16 weeks. If there is CR, patients discontinue high-dose nelfinavir mesylate 4 weeks after documentation of CR. Laboratory Biomarker Analysis: Correlative studies Nelfinavir Mesylate: Given PO	Standard Dose Treatment (Nelfinavir Mesylate) This study is not parallel arm study. Nelfinavir dose will be administrated in the sequential manner according to the response. AE summary is based on participants who received standard dose. There were totally 36 participants received standard dose initally. All patients will received standard dose of nelfinavir (1250 mg BID) for at least 4 weeks. If there is complete response (CR) at any time at the standard dose, nelfinavir will be discontinued 4 weeks after documentation of CR.
Frequency and Severity of Adverse Events in HIV-negative Participants as Assessed by CTCAE v4.0 Anemia	1 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-negative Participants as Assessed by CTCAE v4.0 Diarrhea	9 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-negative Participants as Assessed by CTCAE v4.0 Flatulence	1 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-negative Participants as Assessed by CTCAE v4.0 Nausea	1 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-negative Participants as Assessed by CTCAE v4.0 Chills	1 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-negative Participants as Assessed by CTCAE v4.0 Fatigue	1 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-negative Participants as Assessed by CTCAE v4.0 Localized Edema	1 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-negative Participants as Assessed by CTCAE v4.0 Malaise	1 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-negative Participants as Assessed by CTCAE v4.0 Alanine Amino Transferase Increased	1 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-negative Participants as Assessed by CTCAE v4.0 Aspartate Amino Transferase Increased	2 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-negative Participants as Assessed by CTCAE v4.0 Blood Bilirubin Increased	1 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-negative Participants as Assessed by CTCAE v4.0 Creatinine Increased	1 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-negative Participants as Assessed by CTCAE v4.0 Investigations	6 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-negative Participants as Assessed by CTCAE v4.0 Anorexia	1 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-negative Participants as Assessed by CTCAE v4.0 Hyperglycemia	1 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-negative Participants as Assessed by CTCAE v4.0 Hyperkalemia	1 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-negative Participants as Assessed by CTCAE v4.0 Hypertriglyceridemia	3 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-negative Participants as Assessed by CTCAE v4.0 Hypoalbuminemia	1 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-negative Participants as Assessed by CTCAE v4.0 Hypocalcemia	1 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-negative Participants as Assessed by CTCAE v4.0 Hypoglycemia	1 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-negative Participants as Assessed by CTCAE v4.0 Hypokalemia	5 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-negative Participants as Assessed by CTCAE v4.0 Hyponatremia	1 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-negative Participants as Assessed by CTCAE v4.0 Hypophosphatemia	1 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-negative Participants as Assessed by CTCAE v4.0 Generalized Muscle Weakness	1 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-negative Participants as Assessed by CTCAE v4.0 Dizziness	2 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-negative Participants as Assessed by CTCAE v4.0 Headache	2 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-negative Participants as Assessed by CTCAE v4.0 Memory Impairment	1 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-negative Participants as Assessed by CTCAE v4.0 Insomnia	1 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-negative Participants as Assessed by CTCAE v4.0 Cough	1 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-negative Participants as Assessed by CTCAE v4.0 Dyspnea	1 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-negative Participants as Assessed by CTCAE v4.0 Sore Throat	1 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-negative Participants as Assessed by CTCAE v4.0 Hyperhidrosis	1 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-negative Participants as Assessed by CTCAE v4.0 Skin and Subcutaneous Tissue Disorders	3 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-negative Participants as Assessed by CTCAE v4.0 Hypertension	1 Number of adverse events	—
Frequency and Severity of Adverse Events in HIV-negative Participants as Assessed by CTCAE v4.0 Fall	1 Number of adverse events	—

SECONDARY outcome

Timeframe: Baseline to up to 16 weeks

Population: LANA and K8.1 were reported using percentage of cells with median and IQR.

Descriptive statistics will be used for changes in the relative abundance of all latent and lytic viral mRNA relative to cellular mRNA by expression array, and changes in the percentage of cells staining for K8.1 (lytic) and LANA (latent) antigens by immunohistochemistry. Generalized estimating equations will be used to evaluate these changes over time. Normalizing transformations will be used as needed.

Outcome measures

Outcome measures
Measure	Treatment (Nelfinavir Mesylate) n=36 Participants DOSE NELFINAVIR MESYLATE: Patients receive standard dose nelfinavir mesylate PO BID for 4 weeks in the absence of PD. Patients with PD at 4 weeks proceed to high-dose nelfinavir. At week 8, if there is SD or PR, patients advance to high-dose nelfinavir mesylate. Patients discontinue standard dose nelfinavir mesylate 4 weeks after documentation of CR. HIGH DOSE NELFINAVIR MESYLATE: Patients with PD continue to receive high-dose nelfinavir mesylate PO BID for 4 more weeks. If there is PD documented after 4 weeks at the high dose level, nelfinavir is discontinued. If there is SD or PR, patients continue receiving nelfinavir mesylate for 16 weeks. If there is CR, patients discontinue high-dose nelfinavir mesylate 4 weeks after documentation of CR. Laboratory Biomarker Analysis: Correlative studies Nelfinavir Mesylate: Given PO	Standard Dose Treatment (Nelfinavir Mesylate) This study is not parallel arm study. Nelfinavir dose will be administrated in the sequential manner according to the response. AE summary is based on participants who received standard dose. There were totally 36 participants received standard dose initally. All patients will received standard dose of nelfinavir (1250 mg BID) for at least 4 weeks. If there is complete response (CR) at any time at the standard dose, nelfinavir will be discontinued 4 weeks after documentation of CR.
Assess the Effect of Nelfinavir on KSHV Gene Expression in Tumor Tissue LANA (change)	0 percentage of cells Interval -5.0 to 0.0	—
Assess the Effect of Nelfinavir on KSHV Gene Expression in Tumor Tissue K8.1 at baseline	3 percentage of cells Interval 1.0 to 5.0	—
Assess the Effect of Nelfinavir on KSHV Gene Expression in Tumor Tissue LANA at baseline	10 percentage of cells Interval 3.0 to 20.0	—
Assess the Effect of Nelfinavir on KSHV Gene Expression in Tumor Tissue LANA at Day 70	7.5 percentage of cells Interval 5.0 to 20.0	—

SECONDARY outcome

Timeframe: Baseline to up to 16 weeks

Population: Participants who received study treatment. Area under the curve (AUC) of M8 measurements was reported by tumor response and adverse event grade using median and inter-quartile range (IQR).

Trough nelfinavir/M8 concentrations will be summarized using descriptive statistics and 95% confidence intervals. This outcome will also be displayed graphically as a trend in drug levels over time. The relationship between dose and drug exposure of nelfinavir/M8 and the pharmacodynamic (PD) effects will be determined using Pearson's correlation coefficient (r2) or appropriate non-parametric statistics for dichotomous and categorical variables (e.g., Mann-Whitney U-test or Kruskal-Wallis analysis of variance by ranks).

Outcome measures

Outcome measures
Measure	Treatment (Nelfinavir Mesylate) n=31 Participants DOSE NELFINAVIR MESYLATE: Patients receive standard dose nelfinavir mesylate PO BID for 4 weeks in the absence of PD. Patients with PD at 4 weeks proceed to high-dose nelfinavir. At week 8, if there is SD or PR, patients advance to high-dose nelfinavir mesylate. Patients discontinue standard dose nelfinavir mesylate 4 weeks after documentation of CR. HIGH DOSE NELFINAVIR MESYLATE: Patients with PD continue to receive high-dose nelfinavir mesylate PO BID for 4 more weeks. If there is PD documented after 4 weeks at the high dose level, nelfinavir is discontinued. If there is SD or PR, patients continue receiving nelfinavir mesylate for 16 weeks. If there is CR, patients discontinue high-dose nelfinavir mesylate 4 weeks after documentation of CR. Laboratory Biomarker Analysis: Correlative studies Nelfinavir Mesylate: Given PO	Standard Dose Treatment (Nelfinavir Mesylate) n=5 Participants This study is not parallel arm study. Nelfinavir dose will be administrated in the sequential manner according to the response. AE summary is based on participants who received standard dose. There were totally 36 participants received standard dose initally. All patients will received standard dose of nelfinavir (1250 mg BID) for at least 4 weeks. If there is complete response (CR) at any time at the standard dose, nelfinavir will be discontinued 4 weeks after documentation of CR.
Correlate Nelfinavir and the Primary Active Metabolite, M8, Concentrations With Tumor Response, Antiviral Response, and Adverse Effects in Participants With KS. AE maximum grade 1/2 among HIV positive	30280 ng*cycle/mL Interval 11151.0 to 45568.0	0 ng*cycle/mL Interval 0.0 to 2513.0
Correlate Nelfinavir and the Primary Active Metabolite, M8, Concentrations With Tumor Response, Antiviral Response, and Adverse Effects in Participants With KS. AE maximum grade 1/2 among HIV negative	92552 ng*cycle/mL Interval 49480.0 to 135624.0	—
Correlate Nelfinavir and the Primary Active Metabolite, M8, Concentrations With Tumor Response, Antiviral Response, and Adverse Effects in Participants With KS. Responders among HIV positive	52000 ng*cycle/mL Interval 23252.0 to 70075.0	—
Correlate Nelfinavir and the Primary Active Metabolite, M8, Concentrations With Tumor Response, Antiviral Response, and Adverse Effects in Participants With KS. Non-responder among HIV positive	28202 ng*cycle/mL Interval 8726.0 to 35372.0	0 ng*cycle/mL Interval 0.0 to 2513.0
Correlate Nelfinavir and the Primary Active Metabolite, M8, Concentrations With Tumor Response, Antiviral Response, and Adverse Effects in Participants With KS. Non-responder among HIV negative	80706 ng*cycle/mL Interval 6084.0 to 160927.0	—
Correlate Nelfinavir and the Primary Active Metabolite, M8, Concentrations With Tumor Response, Antiviral Response, and Adverse Effects in Participants With KS. AE maximum grade 3+ among HIV positive	31402 ng*cycle/mL Interval 19605.0 to 69630.0	—
Correlate Nelfinavir and the Primary Active Metabolite, M8, Concentrations With Tumor Response, Antiviral Response, and Adverse Effects in Participants With KS. AE maximum grade 3+ among HIV negative	80058 ng*cycle/mL Interval 42586.0 to 1175314.0	—

SECONDARY outcome

Timeframe: Up to 16 weeks

Population: Participants who received study treatment

Pre-treatment oral samples and oral samples during treatment will be collected from each participant. The sampling frequency is irrelevant as long as the quantity is sufficient. With 36 participants, a decrease in the proportion of participants with detectable KSHV DNA from 50% at baseline to 1% on treatment can be detected at the one-sided 0.05 significance level with 0.99 power using McNemar's test.

Outcome measures

Outcome measures
Measure	Treatment (Nelfinavir Mesylate) n=36 Participants DOSE NELFINAVIR MESYLATE: Patients receive standard dose nelfinavir mesylate PO BID for 4 weeks in the absence of PD. Patients with PD at 4 weeks proceed to high-dose nelfinavir. At week 8, if there is SD or PR, patients advance to high-dose nelfinavir mesylate. Patients discontinue standard dose nelfinavir mesylate 4 weeks after documentation of CR. HIGH DOSE NELFINAVIR MESYLATE: Patients with PD continue to receive high-dose nelfinavir mesylate PO BID for 4 more weeks. If there is PD documented after 4 weeks at the high dose level, nelfinavir is discontinued. If there is SD or PR, patients continue receiving nelfinavir mesylate for 16 weeks. If there is CR, patients discontinue high-dose nelfinavir mesylate 4 weeks after documentation of CR. Laboratory Biomarker Analysis: Correlative studies Nelfinavir Mesylate: Given PO	Standard Dose Treatment (Nelfinavir Mesylate) This study is not parallel arm study. Nelfinavir dose will be administrated in the sequential manner according to the response. AE summary is based on participants who received standard dose. There were totally 36 participants received standard dose initally. All patients will received standard dose of nelfinavir (1250 mg BID) for at least 4 weeks. If there is complete response (CR) at any time at the standard dose, nelfinavir will be discontinued 4 weeks after documentation of CR.
Assess the Effect of Nelfinavir on KSHV Copy Number in Saliva Positive swab result	14 Participants	—
Assess the Effect of Nelfinavir on KSHV Copy Number in Saliva Negative swab result	20 Participants	—
Assess the Effect of Nelfinavir on KSHV Copy Number in Saliva Unavailable swab result	2 Participants	—

Adverse Events

High Dose Treatment After Receiving the Standard Dose (Nelfinavir Mesylate)

Serious events: 1 serious events

Other events: 28 other events

Deaths: 0 deaths

Standard Dose Treatment (Nelfinavir Mesylate)

Serious events: 0 serious events

Other events: 5 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	High Dose Treatment After Receiving the Standard Dose (Nelfinavir Mesylate) n=31 participants at risk This study is not parallel arm study. Nelfinavir dose will be administrated in the sequential manner according to the response. AE summary is based on participants who received high dose. There were totally 31 participants received high dose after receiving standard dose. All patients will received standard dose of nelfinavir (1250 mg BID) for at least 4 weeks. If after 4 weeks there is progressive disease (PD), the participant will advance to the high dose nelfinavir (3125 mg BID). If by 8 weeks there is stable disease (SD) or partial response (PR) at the standard dose, the participant will advance to high dose of nelfinavir. The high dose nelfinavir treatment will be continued for at least 4 weeks. If there is progressive disease (PD) documented after 4 weeks at the high dose level, nelfinavir will be discontinued. If there is stable disease (SD) or partial response (PR) at the high dose level, high dose nelfinavir will be continued for up to 16 weeks. If there is a complete response (CR), high dose treatment will be discontinued 4 weeks after documentation of complete response.	Standard Dose Treatment (Nelfinavir Mesylate) n=36 participants at risk This study is not parallel arm study. Nelfinavir dose will be administrated in the sequential manner according to the response. AE summary is based on participants who received standard dose. There were totally 36 participants received standard dose initally. All patients will received standard dose of nelfinavir (1250 mg BID) for at least 4 weeks. If there is complete response (CR) at any time at the standard dose, nelfinavir will be discontinued 4 weeks after documentation of CR.
Gastrointestinal disorders Abdominal Pain	3.2% 1/31 • Number of events 1 • 8 weeks (4 weeks for standard dose and 4 weeks for high dose); Among 36 participants, 31 participants received the high dose of nelfinavir after receiving the standard dose of nelfinavir. Mortality report was based on all 36 participants. For mortality report in high dose was based on 31 participants which is a subset of 36 participants. Adverse Event and Serious Adverse Event reports were based on 36 participants treated with standard dose levels of nelfinavir and 31 participants treated with high dose after standard dose.	0.00% 0/36 • 8 weeks (4 weeks for standard dose and 4 weeks for high dose); Among 36 participants, 31 participants received the high dose of nelfinavir after receiving the standard dose of nelfinavir. Mortality report was based on all 36 participants. For mortality report in high dose was based on 31 participants which is a subset of 36 participants. Adverse Event and Serious Adverse Event reports were based on 36 participants treated with standard dose levels of nelfinavir and 31 participants treated with high dose after standard dose.
Gastrointestinal disorders Nausea	3.2% 1/31 • Number of events 1 • 8 weeks (4 weeks for standard dose and 4 weeks for high dose); Among 36 participants, 31 participants received the high dose of nelfinavir after receiving the standard dose of nelfinavir. Mortality report was based on all 36 participants. For mortality report in high dose was based on 31 participants which is a subset of 36 participants. Adverse Event and Serious Adverse Event reports were based on 36 participants treated with standard dose levels of nelfinavir and 31 participants treated with high dose after standard dose.	0.00% 0/36 • 8 weeks (4 weeks for standard dose and 4 weeks for high dose); Among 36 participants, 31 participants received the high dose of nelfinavir after receiving the standard dose of nelfinavir. Mortality report was based on all 36 participants. For mortality report in high dose was based on 31 participants which is a subset of 36 participants. Adverse Event and Serious Adverse Event reports were based on 36 participants treated with standard dose levels of nelfinavir and 31 participants treated with high dose after standard dose.
Gastrointestinal disorders Vomiting	3.2% 1/31 • Number of events 1 • 8 weeks (4 weeks for standard dose and 4 weeks for high dose); Among 36 participants, 31 participants received the high dose of nelfinavir after receiving the standard dose of nelfinavir. Mortality report was based on all 36 participants. For mortality report in high dose was based on 31 participants which is a subset of 36 participants. Adverse Event and Serious Adverse Event reports were based on 36 participants treated with standard dose levels of nelfinavir and 31 participants treated with high dose after standard dose.	0.00% 0/36 • 8 weeks (4 weeks for standard dose and 4 weeks for high dose); Among 36 participants, 31 participants received the high dose of nelfinavir after receiving the standard dose of nelfinavir. Mortality report was based on all 36 participants. For mortality report in high dose was based on 31 participants which is a subset of 36 participants. Adverse Event and Serious Adverse Event reports were based on 36 participants treated with standard dose levels of nelfinavir and 31 participants treated with high dose after standard dose.
Hepatobiliary disorders Cholecystitis	3.2% 1/31 • Number of events 1 • 8 weeks (4 weeks for standard dose and 4 weeks for high dose); Among 36 participants, 31 participants received the high dose of nelfinavir after receiving the standard dose of nelfinavir. Mortality report was based on all 36 participants. For mortality report in high dose was based on 31 participants which is a subset of 36 participants. Adverse Event and Serious Adverse Event reports were based on 36 participants treated with standard dose levels of nelfinavir and 31 participants treated with high dose after standard dose.	0.00% 0/36 • 8 weeks (4 weeks for standard dose and 4 weeks for high dose); Among 36 participants, 31 participants received the high dose of nelfinavir after receiving the standard dose of nelfinavir. Mortality report was based on all 36 participants. For mortality report in high dose was based on 31 participants which is a subset of 36 participants. Adverse Event and Serious Adverse Event reports were based on 36 participants treated with standard dose levels of nelfinavir and 31 participants treated with high dose after standard dose.

Other adverse events

Additional Information

Deukwoo Kwon

Statistical and Data Analysis Center, AIDS Malignancy Consortium

Phone: (713) 500-7964

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place