Ocular Cicatricial Pemphigoid
Conditions
Keywords
Pemphigoid, Blistering Diseases, Blindness, Autoimmune, Rituximab
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.
Interventions
DRUGRituximab
The Rituximab dose is 1000mg (1gm) given as an IV infusion every two weeks for 2 doses (days 1 and 15).
Sponsors
University of Alabama at Birmingham
Genentech, Inc.
Biogen
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
19 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With no Evidence of Further Scarring (Fosters Staging) at 16 Weeks | 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. 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
| Measure | Time frame |
|---|---|
| 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 |
Countries
United States
Contacts
PRINCIPAL_INVESTIGATORCraig A Elmets
University of Alabama at Birmingham
Participant flow
Recruitment details
Three patients were recruited from the investigator's practice.
Pre-assignment details
Subjects must meet inclusion/exclusion criteria.
Participants by arm
| Arm | Count |
|---|---|
| Rituximab | 3 |
| Total | 3 |
Baseline characteristics
| Characteristic | Rituximab |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 1 Participants |
| Age, Categorical Between 18 and 65 years | 2 Participants |
| Age, Continuous | 61 years STANDARD_DEVIATION 28 |
| Region of Enrollment United States | 3 participants |
| Sex: Female, Male Female | 2 Participants |
| Sex: Female, Male Male | 1 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 3 |
| other Total, other adverse events | 0 / 3 |
| serious Total, serious adverse events | 0 / 3 |
Outcome results
Primary
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
Population: The data for this Outcome was not collected and due to the length of time, the records have been destroyed.
Primary
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
Time frame: 16 weeks
Population: The data for this Outcome was not collected and due to the length of time, the records have been destroyed.
Secondary
1. Stability of Visual Acuity (Snellen's Test) at 16 Weeks
Time frame: 16 weeks
Population: The data for this Outcome was not collected and due to the length of time, the records have been destroyed.
Secondary
2. Stability of Visual Acuity (Snellen's Test) at 24 Weeks
Time frame: 24 weeks
Population: The data for this Outcome was not collected and due to the length of time, the records have been destroyed.