Trial Outcomes & Findings for Rituximab in the Treatment of Patients With Bullous Pemphigoid (NCT NCT00286325)

Results Overview

The primary safety endpoint is the occurrence of treatment emergent adverse events including infections, infusion reactions and disease progression. These were determined by clinical evaluation and laboratory questions. Disease progression is defined as development of new blisters despite therapy. These are reported as the number of participants with a study related SAE.

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

8 participants

Primary outcome timeframe

1 year

Results posted on

2013-04-11

Participant Flow

Patients with bullous pemphigoid were recruited in dermatology clinics from 2005 - 2009.

Patient were screened for appropriate diagnosis, disease activity, prednisone dosage before entry into the study

Participant milestones

Participant milestones
Measure	Treated Open label study subjects all treated with rituximab
Overall Study STARTED	8
Overall Study COMPLETED	8
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Rituximab in the Treatment of Patients With Bullous Pemphigoid

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Treated n=8 Participants Open label study subjects all treated with rituximab
Age, Categorical Between 18 and 65 years	5 Participants n=5 Participants
Age, Categorical >=65 years	3 Participants n=5 Participants
Age Continuous	61 years STANDARD_DEVIATION 9.7 • n=5 Participants
Sex: Female, Male Female	6 Participants n=5 Participants
Sex: Female, Male Male	2 Participants n=5 Participants
Region of Enrollment United States	8 participants n=5 Participants
Age, Categorical <=18 years	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: 1 year

Population: The safety analysis was performed on all subjects

The primary safety endpoint is the occurrence of treatment emergent adverse events including infections, infusion reactions and disease progression. These were determined by clinical evaluation and laboratory questions. Disease progression is defined as development of new blisters despite therapy. These are reported as the number of participants with a study related SAE.

Outcome measures

Outcome measures
Measure	Treated n=8 Participants Open label study subjects all treated with rituximab
Primary Safety Endpoint Treatment emergent adverse events	0 participants
Primary Safety Endpoint Infusion reactions	0 participants
Primary Safety Endpoint Disease Progression	1 participants

SECONDARY outcome

Timeframe: 1 year

Population: Excluded subject with diagnosis of epidermolysis bullosa acquisita , made after study entry.

The first study visit in which patient reported and was confirmed to have no new blister or lesion formation .

Outcome measures

Outcome measures
Measure	Treated n=7 Participants Open label study subjects all treated with rituximab
Number of Days to Cessation of New Blister	57 Days Interval 13.0 to 163.0

SECONDARY outcome

Timeframe: 24 weeks

Population: Excluded subject with epidermolysis bullosa acquisita diagnosed after study entry.

Subject systemic corticosteroid dosage at week 24 was 25% of starting dose or 10 mg/day of prednisone or less

Outcome measures

Outcome measures
Measure	Treated n=7 Participants Open label study subjects all treated with rituximab
Systemic Corticosteroid Dose of 25% of Starting Dose or 10 mg/Day by Week 24	7 participants

SECONDARY outcome

Timeframe: Week 0 and at 24 weeks

Population: Excluded subject with diagnosis of epidermolysis bullosa acquisita made after study entry, excluded one subject with no circulating antibodies measured at either time point.

IgG antibodies against BP 180 measured in units (by ELISA) for each participant at week 0 compared to value at week 24,

Outcome measures

Outcome measures
Measure	Treated n=6 Participants Open label study subjects all treated with rituximab
IgG Anti Bullous Pemphigoid (BP) 180 Measured in Units by ELISA at Week 24.	40.5 Elisa Units Interval 6.0 to 72.0

SECONDARY outcome

Timeframe: Week 0 and at 24 weeks

Population: excluded subject with epidermolysis bullosa acquisita diagnosed after study entry

Peripheral blood B cell number at week 24 compared to B cell number at week 0

Outcome measures

Outcome measures
Measure	Treated n=7 Participants Open label study subjects all treated with rituximab
B Cell Number at Week 24	3.99 B cells per microliter Interval 1.41 to 137.6

Adverse Events

Treated

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Dr. Russell P Hall

Duke University Medical Center

Phone: 919-684-3110

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place