Clinical trial NCT00584935: Clinical Trial Evaluating Rituximab in Ocular Cicatricial Pemphigoid
Official title: Phase I/II Clinical Trial Evaluating Rituximab in Ocular Cicatricial Pemphigoid
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Ocular Cicatricial Pemphigoid
Keywords: Pemphigoid
Blistering Diseases
Blindness
Autoimmune
Rituximab
Lead sponsor: University of Alabama at Birmingham
Sponsor class: OTHER
Collaborators: name: Genentech, Inc.
name: Biogen
First posted: 2008-01-02
Enrollment: 3
Sex: ALL
Minimum age (years): 19
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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.

Eligibility 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.

Primary outcomes:
measure: Number of Participants With no Evidence of Further Scarring (Fosters Staging) at 16 Weeks
timeFrame: 16 weeks
measure: 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.
timeFrame: 16 weeks
Secondary outcomes:
measure: 1. Stability of Visual Acuity (Snellen's Test) at 16 Weeks
timeFrame: 16 weeks
measure: 2. Stability of Visual Acuity (Snellen's Test) at 24 Weeks
timeFrame: 24 weeks
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama at Birmingham
state: Alabama
Study officials:
affiliation: University of Alabama at Birmingham
name: Craig A Elmets
role: PRINCIPAL_INVESTIGATOR
References: