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Use of Infliximab for the Treatment of Pemphigus Vulgaris

This study has been completed.
Sponsor:
Collaborator:
Autoimmunity Centers of Excellence
Information provided by (Responsible Party):
National Institute of Allergy and Infectious Diseases (NIAID)
ClinicalTrials.gov Identifier:
NCT00283712
First received: January 26, 2006
Last updated: January 31, 2013
Last verified: January 2013
History of Changes
Results First Received: December 21, 2012  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Double Blind (Subject, Caregiver);   Primary Purpose: Treatment
Condition: Pemphigus
Interventions: Drug: Infliximab
Drug: Prednisone
Other: Placebo

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
Four study centers in the United States enrolled 20 subjects with pemphigus vulgaris (PV) who met entry criteria between August 2005 and December 2010.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Each participant signed an informed consent before undergoing any screening procedures to assess eligibility. Refer to the Eligibility Section for further details.

Reporting Groups
  Description
Experimental: Infliximab (Remicade, Revellex) Participants were randomized to receive intravenous infusions of infliximab (5mg/kg reconstituted in 10 mL of Sterile Water for Injection, USP ) at Weeks 0, 2, 6, and 14 over a time period of no less than two hours in a blinded (masked) fashion. Refer to section titled, "Detailed Description" for additional treatment information.
Placebo Comparator: Placebo Participants were randomized to receive intravenous infusions of placebo (5 mg/kg comprised of a white lyophilized powder reconstituted in 10 mL of Sterile Water for Injection, USP) at Weeks 0, 2, 6, and 14 over a time period of no less than two hours in a blinded (masked) fashion. Refer to section titled, "Detailed Description" for additional treatment information.

Participant Flow:   Overall Study
    Experimental: Infliximab (Remicade, Revellex)     Placebo Comparator: Placebo  
STARTED     10     10  
Intent-to-treat Sample     10     10  
Safety Sample     10     10  
Per-Protocol Sample     6     7  
COMPLETED     10     9 [1]
NOT COMPLETED     0     1  
Death                 0                 1  
[1] One subject in placebo arm died prior to study completion.



  Baseline Characteristics
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Reporting Groups
  Description
Experimental: Infliximab (Remicade, Revellex) Participants were randomized to receive intravenous infusions of infliximab (5mg/kg reconstituted in 10 mL of Sterile Water for Injection, USP ) at Weeks 0, 2, 6, and 14 over a time period of no less than two hours in a blinded (masked) fashion. Refer to section titled, "Detailed Description" for additional treatment information.
Placebo Comparator: Placebo Participants were randomized to receive intravenous infusions of placebo (5 mg/kg comprised of a white lyophilized powder reconstituted in 10 mL of Sterile Water for Injection, USP) at Weeks 0, 2, 6, and 14 over a time period of no less than two hours in a blinded (masked) fashion. Refer to section titled, "Detailed Description" for additional treatment information.
Total Total of all reporting groups

Baseline Measures
    Experimental: Infliximab (Remicade, Revellex)     Placebo Comparator: Placebo     Total  
Number of Participants  
[units: participants]
 		  10     10     20  
Age  
[units: participants]
 		     
<=18 years     0     0     0  
Between 18 and 65 years     10     8     18  
>=65 years     0     2     2  
Age  
[units: years]
Mean ± Standard Deviation 		  47.3  ± 15.2     51.1  ± 13.9     49.2  ± 14.3  
Gender  
[units: participants]
 		     
Female     4     4     8  
Male     6     6     12  
Region of Enrollment  
[units: participants]
 		     
United States     10     10     20  
PDAI Index Total Score [1]
[units: Total Activity Score]
Mean ± Standard Deviation 		  23.0  ± 14.6     20.8  ± 10.7     21.9  ± 12.4  
Pemphigus Vulgaris Disease Activity Score: Mucosal [2]
[units: Participants]
 		     
No oral ulcers     1     2     3  
1 to 5 lesions, small ulcers     6     2     8  
6 to 10 lesions, small ulcers     2     5     7  
>10 lesions or extension erosions     1     1     2  
Pemphigus Vulgaris Disease Activity Score: Cutaneous [2]
[units: Participants]
 		     
No blisters or erosions     3     3     6  
<20 blisters or <1 to 3% BSA     2     4     6  
20 to 40 blisters or 3 to 10% BSA     4     2     6  
>40 blisters or >10% BSA     1     1     2  
Pemphigus Vulgaris Disease Activity Score: Other Organ System [2]
[units: Participants]
 		     
No eye, esophageal, laryngeal involvement     9     10     19  
Any eye, esophageal, laryngeal involvement     1     0     1  
[1] The Pemphigus Disease Area Index (PDAI) was developed by the International Pemphigus Committee and measures both activity of and damage due to pemphigus on the skin, scalp, and mucous membranes. Total scores can range from 0 to a possible 263 maximum score, with 250 points representing disease activity (120 points for skin activity, 10 points for scalp activity, and 120 points for mucosal activity) and 13 points representing disease damage. Higher scores reflect worse disease.
[2] The Pemphigus Vulgaris Disease Activity(PVDA)score was used to grade a participant’s baseline disease activity using the SAGE II computerized burn mapping system, which calculated the total body surface area(BSA)involved. Scores were based on the number of lesions and blisters present as well as total body surface area involved. Three types of disease activity were assessed: mucosal, cutaneous, and other organ system. A higher score indicates more severe disease activity. For each of the 3 types of disease activity, the number of participants who met the disease activity condition is shown.



  Outcome Measures
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1.  Primary:   Participant Response to Treatment at Week 18   [ Time Frame: Baseline to Week 18 ]
  Show Outcome Measure 1

2.  Primary:   Treatment-Related Adverse Events >= Grade 3 On or Before Week 18   [ Time Frame: Baseline to Week 18 ]
  Show Outcome Measure 2

3.  Secondary:   Participant Response to Treatment at Week 18   [ Time Frame: Baseline to Week 18 ]
  Show Outcome Measure 3

4.  Secondary:   Participant Modified Response Status at Week 18   [ Time Frame: Baseline to Week 18 ]
  Show Outcome Measure 4

5.  Secondary:   Participant Time to Cessation of New Blisters   [ Time Frame: Baseline to Week 26 ]
  Show Outcome Measure 5

6.  Secondary:   Time to 80% Lesion Healing   [ Time Frame: Baseline to Week 26 ]
  Show Outcome Measure 6

7.  Secondary:   Total Prednisone Dosage Required for Participants to Achieve Cessation of New Blisters   [ Time Frame: Baseline to Week 26 ]
  Show Outcome Measure 7

8.  Secondary:   Total Prednisone Dosage Required for Participants to Achieve 80% Healing of Existing Erosions   [ Time Frame: Baseline to Week 26 ]
  Show Outcome Measure 8

9.  Secondary:   Participant Health Related Quality of Life (Medical Outcome Study Short Form 36) Score Changes From Baseline to Week 18   [ Time Frame: Baseline to Week 18 ]
  Show Outcome Measure 9

10.  Secondary:   Participant Dermatology-Related Quality of Life Changes From Baseline to Week 18   [ Time Frame: Baseline to Week 18 ]
  Show Outcome Measure 10

11.  Secondary:   Participant Duration of Clinical Response   [ Time Frame: Baseline to Week 26 ]
  Show Outcome Measure 11

12.  Secondary:   Participants Who Experienced Severe Infusion Reactions   [ Time Frame: Baseline to Week 26 ]
  Show Outcome Measure 12

13.  Secondary:   Participants Who Experienced Severe Infectious Complications   [ Time Frame: Baseline to Week 26 ]
  Show Outcome Measure 13

14.  Secondary:   Adverse Events Resulting in Treatment Discontinuation   [ Time Frame: Baseline to Week 26 ]
  Show Outcome Measure 14

15.  Secondary:   Participant Pemphigus Vulgaris Disease Activity Score   [ Time Frame: Baseline to Week 26 ]
  Show Outcome Measure 15


  Serious Adverse Events
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  Other Adverse Events
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  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.


Limitations and Caveats
Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.  


Results Point of Contact:  
Name/Title: Associate Director, Clinical Research Program
Organization: DAIT/NIAID
phone: 301-594-7669
e-mail: DAITClinicalTrialsGov@niaid.nih.gov


Publications:


Responsible Party: National Institute of Allergy and Infectious Diseases (NIAID)
ClinicalTrials.gov Identifier: NCT00283712     History of Changes
Other Study ID Numbers: DAIT APV01
Study First Received: January 26, 2006
Results First Received: December 21, 2012
Last Updated: January 31, 2013
Health Authority: United States: Food and Drug Administration