Clinical Trial Evaluating Rituximab in Ocular Cicatricial Pemphigoid

NCT00584935 | Completed Phase 1 Results

• 2 other identifiers: F060213003UAB
• Study Type: interventional
• Target: 3
• Locations: 1 country
• Sites: 1

Brief Summary

Cicatricial pemphigoid is an autoimmune blistering disease which affects the skin, mucous membranes, and, in a small subset of patients, the eye. Progressive ocular disease can lead to irreversible damage and blindness. Conventional treatments include systemic steroids, dapsone, and immunosuppressive agents. These treatments, however, are not successful with all patients. Rituximab has been very effective in the treatment of other autoimmune disorders, and has recently been shown to be effective for autoimmune blistering pemphigus. We propose that it will also be effective in the treatment of cicatricial pemphigoid.

Conditions

Ocular Cicatricial Pemphigoid

Keywords

Pemphigoid; Blistering Diseases; Blindness; Autoimmune; Rituximab

Outcome Measures

Primary Outcomes (2)

• Number of Participants With no Evidence of Further Scarring (Fosters Staging) at 16 Weeks

  Stages Characteristics I Subconjunctival scarring and fibrosis II Fornix foreshortening (a-d describes % loss of inferior fornix depth) 1. 0-25% 2. 25-50% 3. 50-75% 4. 75-100% III Presence of symblepharon and number (n) (a-d describes % of horizontal involvement by symblepharons and n is the number of symblepharons countable) a. 0-25% b. 25-50% c. 50-75% d. 75-100% IV Ankyloblepharon, frozen globe

  16 weeks

• 2. The Proportion of Patients That Experience a Grade 3, Grade 4, or Grade 5 Toxicity Based Reaction on the NCI-CTC System at the Time of Their Infusions and During Follow-up Visits.

  16 weeks

Secondary Outcomes (2)

• 1. Stability of Visual Acuity (Snellen's Test) at 16 Weeks

  16 weeks

• 2. Stability of Visual Acuity (Snellen's Test) at 24 Weeks

  24 weeks

Study Arms (1)

Rituximab

EXPERIMENTAL

The Rituximab dose is 1000 mg (1gm) given as an IV infusion every two weeks for 2 doses (Days 1 and 15).

Drug: Rituximab

Interventions

Rituximab DRUG

The Rituximab dose is 1000mg (1gm) given as an IV infusion every two weeks for 2 doses (days 1 and 15).

Rituximab

Eligibility Criteria

• Age: 19 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Clinical diagnosis of ocular cicatricial pemphigoid (symptoms of conjunctivitis, irritation, burning, increased lacrimation, photophobia, dryness of the eyes along with conjunctival inflammation, trichiasis, and scarring
• One of the following:
• Failed response to the use of one or more conventional treatments for a minimum of 10 weeks; or
• Minimal conventional medication doses, with a significant adverse effects, contradiction to use, or progressive disease despite treatment
• Adults age 19 and older
• Adequate renal function as indicated by serum creatinine levels less than 1.5

You may not qualify if:

• known hypersensitivity to rituximab or its components
• Age less than 19 years
• Any other condition deemed by the investigator to be a significant hazard to the subject if the investigational therapy were initiated.

Sponsors & Collaborators

• Genentech, Inc.: collaborator
• Biogen: collaborator
• University of Alabama at Birmingham: lead

Study Sites (1)

University of Alabama at Birmingham

Birmingham, Alabama, 35294, United States

Location

MeSH Terms

Conditions

Pemphigoid, Benign Mucous Membrane; Pemphigoid, Bullous; Blindness

Interventions

Rituximab

Condition Hierarchy (Ancestors)

Conjunctival Diseases; Eye Diseases; Skin Diseases, Vesiculobullous; Skin Diseases; Skin and Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases; Vision Disorders; Sensation Disorders; Neurologic Manifestations; Nervous System Diseases; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Antibodies, Monoclonal, Murine-Derived; Antibodies, Monoclonal; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins

Limitations and Caveats

Genentech closed trial before site was able to enroll 5 patients.

Results Point of Contact

• Title: Wendy Cantrell, CRNP
• Organization: UAB Department of Dermatology

wcantrell@uabmc.edu

205-502-9960

Study Officials

• Craig A Elmets

  University of Alabama at Birmingham

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: December 26, 2007
• First Posted: January 2, 2008
• Study Start: January 1, 2006
• Primary Completion: January 1, 2010
• Study Completion: January 1, 2026
• Last Updated: May 4, 2026
• Results First Posted: April 21, 2011

Record last verified: 2026-04

Locations

US (1)