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

Tracking Information

• First Submitted Date: December 26, 2007
• First Posted Date: January 2, 2008
• Results First Submitted Date: February 15, 2011
• Results First Posted Date: April 21, 2011
• Last Update Posted Date: February 7, 2018
• Study Start Date: January 2006
• Actual Primary Completion Date: January 2010 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: October 10, 2017)

• 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. 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 ]

Original Primary Outcome Measures (submitted: December 31, 2007)

• 1. No evidence of further scarring (Fosters Staging) at 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.

Current Secondary Outcome Measures (submitted: March 22, 2011)

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

Original Secondary Outcome Measures (submitted: December 31, 2007)

• 1. Stability of visual acuity (Snellen's Test) at 16 weeks
• 2. Stability of visual acuity (Snellen's Test) at 24 weeks
• 3. Decreased requirement for conventional medications at 16 weeks.

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Clinical Trial Evaluating Rituximab in Ocular Cicatricial Pemphigoid
• Official Title: Phase I/II Clinical Trial Evaluating Rituximab in Ocular Cicatricial Pemphigoid
• 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.
• Study Type: Interventional
• Study Phase: Phase 1; Phase 2
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Ocular Cicatricial Pemphigoid

Intervention

Drug: Rituximab

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

Study Arms

Experimental: Rituximab

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

Intervention: Drug: Rituximab

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: March 22, 2011): 3
• Original Estimated Enrollment (submitted: December 31, 2007): 5
• Actual Study Completion Date: May 2010
• Actual Primary Completion Date: January 2010 (Final data collection date for primary outcome measure)

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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 19 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT00584935
• Other Study ID Numbers: F060213003
• Has Data Monitoring Committee: No
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Current Responsible Party: Craig Elmets, University of Alabama at Birmingham
• Original Responsible Party: Not Provided
• Current Study Sponsor: University of Alabama at Birmingham
• Original Study Sponsor: Same as current

Collaborators

• Genentech, Inc.
• Biogen

Investigators

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

• PRS Account: University of Alabama at Birmingham
• Verification Date: January 2018