Trial Outcomes & Findings for Initial Study of Rituximab to Treat Primary Biliary Cirrhosis (NCT NCT00364819)

NCT ID: NCT00364819

Last Updated: 2017-07-02

Results Overview

• Recruitment status: COMPLETED
• Study phase: PHASE1/PHASE2
• Target enrollment: 6 participants
• Primary outcome timeframe: 52 weeks
• Results posted on: 2017-07-02

Participant Flow

Participant milestones

Measure	Open Label Study Arm Primary Biliary Cirrhosis rituximab: rituximab 1000 mg IV day 1 and 15, given over 5 - 6 hours
Overall Study STARTED	6
Overall Study COMPLETED	6
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Initial Study of Rituximab to Treat Primary Biliary Cirrhosis

Baseline characteristics by cohort

Measure	Open Label Study Arm n=6 Participants Primary Biliary Cirrhosis rituximab: rituximab 1000 mg IV day 1 and 15, given over 5 - 6 hours
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	6 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	6 Participants n=5 Participants
Sex: Female, Male Male	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	3 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	3 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	6 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Anti-mitochondrial Antibody Anti-mitochondrial Antibody Positive	6 Participants n=5 Participants
Anti-mitochondrial Antibody Anti-mitochondrial Antibody Negative	0 Participants n=5 Participants
Liver Biopsy Stage Ludwig Stage I	1 Participants n=5 Participants
Liver Biopsy Stage Ludwig Stage II	4 Participants n=5 Participants
Liver Biopsy Stage Ludwig Stage III	1 Participants n=5 Participants
Liver Biopsy Stage Ludwig Stage IV	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: 52 weeks

Outcome measures

Measure	Open Label Study Arm n=6 Participants Primary Biliary Cirrhosis rituximab: rituximab 1000 mg IV day 1 and 15, given over 5 - 6 hours
Number of Participants With Adverse Events Serious Adverse Events	1 Participants
Number of Participants With Adverse Events Non-Serious Adverse Events	3 Participants
Number of Participants With Adverse Events No Events	2 Participants

SECONDARY outcome

Timeframe: 52 Weeks

The difference in serum immunoglobulin G from Baseline to Week 52

Outcome measures

Measure	Open Label Study Arm n=6 Participants Primary Biliary Cirrhosis rituximab: rituximab 1000 mg IV day 1 and 15, given over 5 - 6 hours
Change in Serum Immunoglobulin G	167 mg/dL Standard Deviation 450

SECONDARY outcome

Timeframe: 52 Weeks

Population: 1 subject with missing data.

The difference in serum immunoglobulin A from Baseline to Week 52

Outcome measures

Measure	Open Label Study Arm n=5 Participants Primary Biliary Cirrhosis rituximab: rituximab 1000 mg IV day 1 and 15, given over 5 - 6 hours
Change in Serum Immunoglobulin A	36.8 mg/dL Standard Deviation 89.7

SECONDARY outcome

Timeframe: 52 Weeks

The difference in serum immunoglobulin M from Baseline to Week 52

Outcome measures

Measure	Open Label Study Arm n=6 Participants Primary Biliary Cirrhosis rituximab: rituximab 1000 mg IV day 1 and 15, given over 5 - 6 hours
Change in Serum Immunoglobulin M	121 mg/dL Standard Deviation 74.5

SECONDARY outcome

Timeframe: 52 Weeks

The difference in serum alkaline phosphatase from Baseline to Week 52

Outcome measures

Measure	Open Label Study Arm n=6 Participants Primary Biliary Cirrhosis rituximab: rituximab 1000 mg IV day 1 and 15, given over 5 - 6 hours
Change in Serum Alkaline Phosphatase	55.8 U/L Standard Deviation 151.5

Adverse Events

Open Label Study Arm

Serious events: 1 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events

Measure	Open Label Study Arm n=6 participants at risk Primary Biliary Cirrhosis rituximab: rituximab 1000 mg IV day 1 and 15, given over 5 - 6 hours
Infections and infestations Shingles	16.7% 1/6 • Number of events 1 • 52 weeks after initial infusion of rituximab

Other adverse events

Measure	Open Label Study Arm n=6 participants at risk Primary Biliary Cirrhosis rituximab: rituximab 1000 mg IV day 1 and 15, given over 5 - 6 hours
Immune system disorders Lymphopenia	16.7% 1/6 • Number of events 1 • 52 weeks after initial infusion of rituximab
Infections and infestations Upper respiratory infection	16.7% 1/6 • Number of events 1 • 52 weeks after initial infusion of rituximab
Skin and subcutaneous tissue disorders Dry skin	16.7% 1/6 • Number of events 1 • 52 weeks after initial infusion of rituximab
Skin and subcutaneous tissue disorders Hyperpigmentation	16.7% 1/6 • Number of events 1 • 52 weeks after initial infusion of rituximab

Additional Information

Christopher L. Bowlus, MD

University of California Davis

Phone: 916-734-8986

Email: clbowlus@ucdavis.edu

Results disclosure agreements

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