Clinical Trial Evaluating Rituximab in Ocular Cicatricial Pemphigoid

• ClinicalTrials.gov Identifier: NCT00584935
• Recruitment Status: Completed
• First Posted: January 2, 2008
• Results First Posted: April 21, 2011
• Last Update Posted: February 7, 2018
• Sponsor: University of Alabama at Birmingham
• Collaborators: Genentech, Inc.; Biogen
• Information provided by (Responsible Party): Craig Elmets, University of Alabama at Birmingham

Study Description

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.

Condition or disease	Intervention/treatment	Phase
Ocular Cicatricial Pemphigoid	Drug: Rituximab	Phase 1; Phase 2

Detailed Description:

Cicatricial pemphigoid is an autoimmune blistering disease which can affect the skin, mucous membranes, and, in a small subset of patients, the eyes. Progressive ocular disease can lead to irreversible damage and blindness. Conventional treatments have included high dose systemic steroids, dapsone, and immunosuppressive agents such as azathioprine, methotrexate, cyclophosphamide, and mycophenolate mofetil. However, there are a subgroup of patients who fail to respond to these treatments, develop intolerable side effects, or have contraindications to their use. Patients may also develop resistance to these conventional treatment modalities. For these reasons, alternative treatment modalities are needed. Rituximab has been very effective in the treatment of other autoimmune disorders. It has recently been shown to be effective in the treatment of another autoimmune blistering disorder known as pemphigus. We thus propose that Rituximab will be effective in the treatment of cicatricial pemphigoid.

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 3 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Phase I/II Clinical Trial Evaluating Rituximab in Ocular Cicatricial Pemphigoid
• Study Start Date: January 2006
• Actual Primary Completion Date: January 2010
• Actual Study Completion Date: May 2010

Arms and Interventions

Arm	Intervention/treatment
Experimental: Rituximab; The Rituximab dose is 1000 mg (1gm) given as an IV infusion every two weeks for 2 doses (Days 1 and 15).	Drug: Rituximab; The Rituximab dose is 1000mg (1gm) given as an IV infusion every two weeks for 2 doses (days 1 and 15).

Outcome Measures

Primary Outcome Measures :

1. Number of Participants With no Evidence of Further Scarring (Fosters Staging) at 16 Weeks [ Time Frame: 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

2. 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. [ Time Frame: 16 weeks ]

Secondary Outcome Measures :

1. 1. Stability of Visual Acuity (Snellen's Test) at 16 Weeks [ Time Frame: 16 weeks ]
2. 2. Stability of Visual Acuity (Snellen's Test) at 24 Weeks [ Time Frame: 24 weeks ]

Eligibility Criteria

• Ages Eligible for Study: 19 Years and older (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

Inclusion Criteria:

1. 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

2. 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
3. Adults age 19 and older
4. Adequate renal function as indicated by serum creatinine levels less than 1.5

Exclusion Criteria:

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

Contacts and Locations

Locations

United States, Alabama

University of Alabama at Birmingham

Birmingham, Alabama, United States, 35294

Sponsors and Collaborators

University of Alabama at Birmingham

Genentech, Inc.

Biogen

Investigators

• Principal Investigator: Craig A Elmets; University of Alabama at Birmingham

More Information

• Responsible Party: Craig Elmets, Principal Investigator, University of Alabama at Birmingham
• ClinicalTrials.gov Identifier: NCT00584935
• Other Study ID Numbers: F060213003
• First Posted: January 2, 2008
• Results First Posted: April 21, 2011
• Last Update Posted: February 7, 2018
• Last Verified: January 2018

Keywords provided by Craig Elmets, University of Alabama at Birmingham:

Pemphigoid; Blistering Diseases; Blindness; Autoimmune; Rituximab

Additional relevant MeSH terms:

Pemphigoid, Benign Mucous Membrane; Pemphigoid, Bullous; Skin Diseases, Vesiculobullous; Skin Diseases; Autoimmune Diseases; Immune System Diseases; Conjunctival Diseases; Eye Diseases; Rituximab; Antineoplastic Agents, Immunological; Antineoplastic Agents; Immunologic Factors; Physiological Effects of Drugs; Antirheumatic Agents