Trial Outcomes & Findings for The Effect of Sorafenib on Portal Pressure (NCT NCT01714609)

NCT ID: NCT01714609

Last Updated: 2020-12-29

Results Overview

Number of participants with a decrease in HPVG that was \> 10% of baseline

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 10 participants
• Primary outcome timeframe: Three Months
• Results posted on: 2020-12-29

Participant Flow

Participant milestones

Measure	Placebo Subjects randomized to placebo will take two tablets of placebo by mouth twice daily. Placebo: Placebo Comparator: Placebo	Sorafenib Subjects randomized to Sorafenib will take Sorafenib 400 mg by mouth twice daily. Sorafenib: Sorafenib, 400 mg twice daily
Overall Study STARTED	5	5
Overall Study COMPLETED	4	5
Overall Study NOT COMPLETED	1	0

Reasons for withdrawal

Measure	Placebo Subjects randomized to placebo will take two tablets of placebo by mouth twice daily. Placebo: Placebo Comparator: Placebo	Sorafenib Subjects randomized to Sorafenib will take Sorafenib 400 mg by mouth twice daily. Sorafenib: Sorafenib, 400 mg twice daily
Overall Study Protocol Violation	1	0

Baseline Characteristics

The Effect of Sorafenib on Portal Pressure

Baseline characteristics by cohort

Measure	Placebo n=4 Participants Subjects randomized to placebo will take two tablets of placebo by mouth twice daily. Placebo: Placebo Comparator: Placebo	Sorafenib n=5 Participants Subjects randomized to Sorafenib will take Sorafenib 400 mg by mouth twice daily. Sorafenib: Sorafenib, 400 mg twice daily	Total n=9 Participants Total of all reporting groups
Age, Continuous	55.98 years STANDARD_DEVIATION 4.7 • n=5 Participants	58.78 years STANDARD_DEVIATION 4.25 • n=7 Participants	57.38 years STANDARD_DEVIATION 4.48 • n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Sex: Female, Male Male	4 Participants n=5 Participants	5 Participants n=7 Participants	9 Participants n=5 Participants
Region of Enrollment United States	4 participants n=5 Participants	5 participants n=7 Participants	9 participants n=5 Participants

PRIMARY outcome

Timeframe: Three Months

Number of participants with a decrease in HPVG that was > 10% of baseline

Outcome measures

Measure	Placebo n=4 Participants Subjects randomized to placebo will take two tablets of placebo by mouth twice daily. Placebo: Placebo Comparator: Placebo	Sorafenib n=5 Participants Subjects randomized to Sorafenib will take Sorafenib 400 mg by mouth twice daily. Sorafenib: Sorafenib, 400 mg twice daily
Patients With Change in HVPG From Baseline	1 participants	1 participants

Adverse Events

Placebo

Serious events: 1 serious events

Other events: 5 other events

Deaths: 0 deaths

Sorafenib

Serious events: 0 serious events

Other events: 5 other events

Deaths: 0 deaths

Serious adverse events

Measure	Placebo n=5 participants at risk Subjects randomized to placebo will take two tablets of placebo by mouth twice daily. Placebo: Placebo Comparator: Placebo	Sorafenib n=5 participants at risk Subjects randomized to Sorafenib will take Sorafenib 400 mg by mouth twice daily. Sorafenib: Sorafenib, 400 mg twice daily
General disorders Fever	20.0% 1/5 Adverse events were collected at the organ class level, unless specified	0.00% 0/5 Adverse events were collected at the organ class level, unless specified

Other adverse events

Measure	Placebo n=5 participants at risk Subjects randomized to placebo will take two tablets of placebo by mouth twice daily. Placebo: Placebo Comparator: Placebo	Sorafenib n=5 participants at risk Subjects randomized to Sorafenib will take Sorafenib 400 mg by mouth twice daily. Sorafenib: Sorafenib, 400 mg twice daily
Gastrointestinal disorders gastrointestinal disorders	100.0% 5/5 Adverse events were collected at the organ class level, unless specified	100.0% 5/5 Adverse events were collected at the organ class level, unless specified
General disorders General Disorders	80.0% 4/5 Adverse events were collected at the organ class level, unless specified	80.0% 4/5 Adverse events were collected at the organ class level, unless specified
Skin and subcutaneous tissue disorders Skin and subcutaneous tissue disorders	80.0% 4/5 Adverse events were collected at the organ class level, unless specified	100.0% 5/5 Adverse events were collected at the organ class level, unless specified
Metabolism and nutrition disorders Metabolism and nutrition disorders	60.0% 3/5 Adverse events were collected at the organ class level, unless specified	40.0% 2/5 Adverse events were collected at the organ class level, unless specified
Nervous system disorders Nervous System Disorders	60.0% 3/5 Adverse events were collected at the organ class level, unless specified	80.0% 4/5 Adverse events were collected at the organ class level, unless specified
Musculoskeletal and connective tissue disorders Muskoloskeletal and connective tissue	40.0% 2/5 Adverse events were collected at the organ class level, unless specified	20.0% 1/5 Adverse events were collected at the organ class level, unless specified
Psychiatric disorders Psychiatric disorders	40.0% 2/5 Adverse events were collected at the organ class level, unless specified	20.0% 1/5 Adverse events were collected at the organ class level, unless specified
Respiratory, thoracic and mediastinal disorders Respitory, thoracic and medistinal	40.0% 2/5 Adverse events were collected at the organ class level, unless specified	40.0% 2/5 Adverse events were collected at the organ class level, unless specified
Vascular disorders Vascular disorders	40.0% 2/5 Adverse events were collected at the organ class level, unless specified	0.00% 0/5 Adverse events were collected at the organ class level, unless specified
Hepatobiliary disorders Alanine aminostranferase	20.0% 1/5 Adverse events were collected at the organ class level, unless specified	0.00% 0/5 Adverse events were collected at the organ class level, unless specified
Hepatobiliary disorders Blood Bilirubin Increase	20.0% 1/5 Adverse events were collected at the organ class level, unless specified	0.00% 0/5 Adverse events were collected at the organ class level, unless specified
Ear and labyrinth disorders Ear and labyrinth disorders	20.0% 1/5 Adverse events were collected at the organ class level, unless specified	0.00% 0/5 Adverse events were collected at the organ class level, unless specified
General disorders INR increased	20.0% 1/5 Adverse events were collected at the organ class level, unless specified	0.00% 0/5 Adverse events were collected at the organ class level, unless specified
Infections and infestations Infections and infestations	20.0% 1/5 Adverse events were collected at the organ class level, unless specified	20.0% 1/5 Adverse events were collected at the organ class level, unless specified
General disorders Platelet Count Decrease	20.0% 1/5 Adverse events were collected at the organ class level, unless specified	0.00% 0/5 Adverse events were collected at the organ class level, unless specified
Reproductive system and breast disorders Reproductive system and breast disorders	20.0% 1/5 Adverse events were collected at the organ class level, unless specified	0.00% 0/5 Adverse events were collected at the organ class level, unless specified
Metabolism and nutrition disorders Weight Loss	20.0% 1/5 Adverse events were collected at the organ class level, unless specified	20.0% 1/5 Adverse events were collected at the organ class level, unless specified
Metabolism and nutrition disorders Weight Gain	0.00% 0/5 Adverse events were collected at the organ class level, unless specified	20.0% 1/5 Adverse events were collected at the organ class level, unless specified

Additional Information

Dr. Garcia-Tsao

Yale University

Phone: (203) 737-6063

Email: guadalupe.garcia-tsao@yale.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place