A Study to Estimate Safety and Efficacy of Bay 43-9006 in the Treatment of Hepatocellular Carcinoma

This study has been completed.

Study NCT00044512 Information provided by Bayer

First Received: August 30, 2002 Last Updated: June 19, 2009 History of Changes

Study Type:	Interventional
Study Design:	Non-Randomized, Open Label, Uncontrolled, Single Group Assignment
Condition:	Carcinoma, Hepatocellular
Intervention:	Drug: Nexavar (Sorafenib, BAY43-9006)

Participant Flow

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Recruitment Details

Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
Only subjects with measurable, histologically or cytologically documented hepatocellur carcinoma (HCC) which was inoperable or who had refused surgery could participate in this study.

Pre-Assignment Details

Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Of 147 enrolled patients, 137 received treatment. 10 patients failed screening; reasons were: target lesions identified at baseline (3), liver function tests too high for inclusion (2), prior systemic anticancer treatment (2), creatinine too high for inclusion (1), platelets too low for inclusion (1), diagnosis of HCC not confirmed (1)

Reporting Groups

	Description
BAY 43-9006 400 mg b.i.d.	Sorafenib 400 mg administered b.i.d.

Participant Flow for 2 periods

Period: Treatment

	BAY 43-9006 400 mg b.i.d.
STARTED	137
COMPLETED	137
NOT COMPLETED	0

Period: Follow Up

	BAY 43-9006 400 mg b.i.d.
STARTED	137
COMPLETED	135
NOT COMPLETED	2
Lost to Follow-up	2

Baseline Characteristics

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Reporting Groups

	Description
BAY 43-9006 400 mg b.i.d.	Sorafenib 400 mg administered b.i.d.

Baseline Measures

	BAY 43-9006 400 mg b.i.d.
Number of Participants [units: participants]	137
Age [units: participants]
<=18 years	0
Between 18 and 65 years	84
>=65 years	53
Gender [units: participants]
Female	40
Male	97
Child Pugh Status^[1] [units: participants]
Status A	98
Status B	38
Missing	1
Eastern Cooperative Group performance status (ECOG PS) at study entry^[2] [units: participants]
Grade 0	68
Grade 1	69
Grade 2	0
Grade 3	0
Grade 4	0
Stage of Disease at study entry (TNM Classification)^[3] [units: participants]
Stage 1	0
Stage 2	4
Stage 3	42
Stage 4	91

[1]	The Child-Pugh score is used to assess the prognosis of chronic liver disease, mainly cirrhosis. One of the inclusion criterion for this study was a cirrhotic status of Child Pugh class was A or B. Child Pugh class C were excluded.
[2]	Eastern Cooperative Oncology Group (ECOG) Performance Status (PS) is a scoring system used to quantify cancer patients' general well-being. One of the inclusion criteria for this study was an ECOG PS of 0 or 1.
[3]	TNM (Tumor Nodes, Metastases) is a classification system used for solid tumors. One of the inclusion criterion for this study was inoperable tumor (T2 to T4, any N, M0 or M1 as defined by the tumor nodes metastasis [TNM] classification).

Outcome Measures

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1. Primary:

Objective Response Rate [ Until 30 days after termination of active therapy ]

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2. Secondary:

Duration of Response [ Time from first dose of study drug until disease progression ]

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Measure Type	Secondary
Measure Title	Duration of Response
Measure Description	Duration of response was calculated from the first date of receiving study drug until documented progressive disease (PD) in subjects who exhibited an objective response.
Time Frame	Time from first dose of study drug until disease progression
Safety Issue	No

Population Description

Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
Intention to Treat (ITT) analyses were performed on all treated subjects and for subgroups baseline Child Pugh status (A vs B), ECOG PS (0 vs 1), TNM stage at study entry (II/III vs IV), hepatitis B and C status (positive vs negative). The 3 subjects are censored at time of evaluation. The Median is not estimable so the reported number is biased.

Reporting Groups

	Description
BAY 43-9006 400 mg b.i.d.	Sorafenib 400 mg administered b.i.d.

Measured Values

	BAY 43-9006 400 mg b.i.d.
Number of Participants Analyzed [units: participants]	3
Duration of Response [units: days] Median ( Full Range )	374 ( 369 to 443 )

No statistical analysis provided for Duration of Response

3. Secondary:

Time to Response [ Until objective response occurs ]

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4. Secondary:

Time to Progression [ Until progression occurs ]

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5. Secondary:

Duration of Stable Disease [ Until documented PD or response. ]

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6. Secondary:

Time to Minor Response [ Until MR was first documented ]

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7. Secondary:

Duration of Minor Response [ Time from MR to PD ]

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8. Secondary:

Overall Survival [ Start of treatment to death ]

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Serious Adverse Events

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Other Adverse Events

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More Information

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Certain Agreements:

Principal Investigators are NOT employed by the organization sponsoring the study.

There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

The agreement is:

	The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
	The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
	Other disclosure agreement that restricts the right of the PI to discuss or publish trial results after the trial is completed. Restriction Description: Agreement between Sponsor and PI should be reached before publication

Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
Subjects had advanced disease and were heavily pretreated. National Cancer Institute-Common Toxicity Criteria (NCI-CTC) was translated to Medical Dictionary for Regulatory Activities (MedDRA) for System Organ Class (SOC) only.

Results Point of Contact:

Name/Title: Therapeutic Area Head
Organization: BAYER
e-mail: clinical-trials-contact@bayerhealthcare.com

No publications provided

Responsible Party:	Bayer HealtCare Pharmaceuticals ( Therapeutic Area Head )
Study ID Numbers:	10874
Study First Received:	August 30, 2002
Results First Received:	February 20, 2009
Last Updated:	June 19, 2009
ClinicalTrials.gov Identifier:	NCT00044512 History of Changes
Health Authority:	United States: Food and Drug Administration