Use of KC706 for the Treatment of Pemphigus Vulgaris

NCT00606749 | Completed Phase 2

• 1 other identifier: KC706-C08
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to determine whether KC706 is effective in the prevention and healing of blisters in patients with pemphigus vulgaris, while the patient remains on stable doses of corticosteroids and/or immunosuppressants.

Conditions

Pemphigus Vulgaris

Outcome Measures

Primary Outcomes (1)

• To evaluate the ability of KC706 to prevent the appearance of new lesions and heal existing lesions, while maintaining stable doses of corticosteroids and/or immunosuppressants in patients with pemphigus vulgaris.

  16 weeks

Secondary Outcomes (1)

• Evaluate the safety of KC706 in patients with PV and to assess plasma levels of KC706 with once daily dosing.

  16 weeks

Interventions

KC706 DRUG

300 mg once daily (QD) for 12 weeks.

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• At least 18 years of age;
• Diagnosis of pemphigus vulgaris
• Patients must be taking and require either corticosteroid therapy or immunosuppressive therapy or both;
• Immunosuppressive therapy, if any,should have been administered at a stable dose for at least 60 days prior to the Baseline Visit and be well-tolerated, without the expectation that there will be a need to increase that dose during the next 30 days;
• Corticosteroids, if any, should have been administered at a stable dose for at least 30 days prior to the Baseline Visit without expectation that there will be a need to increase that dose during the next 30 days;
• Patients should have active PV skin, scalp or mucosal lesions that meet at least one of the following criteria at the Baseline Visit:
• \> 3 new lesions/week every week in the previous 3 weeks (skin, scalp, and/or mucosal), with healing occurring at a rate to match the appearance of new lesions; or
• At least 3 active, established lesions with a Pemphigus Lesion Score of at least 2; skin or scalp lesions must be ≥ 5mm in diameter to qualify; there is no size requirement for mucosal lesions; or,
• large active established skin, scalp, or mucosal lesion \> 10 mm;
• Accessibility to veins suitable for venipuncture;
• Patients must be cooperative, able to read, understand and give informed consent, and able to adhere to the study visit schedule and protocol requirements; and,
• Patients must be willing to follow adequate contraceptive measures during the study (both sexes).

You may not qualify if:

• Any history of opportunistic infections within 3 months prior to receiving study drug;
• Infection with HIV;
• Past or present diagnosis of hepatitis confirmed by serology or elevated hepatic enzymes;
• History of alcoholic liver disease or cirrhosis;
• Clinically significant concurrent medical disease or laboratory abnormalities evidenced by one or more of the following:
• Hepatobiliary AST or ALT ≥ 1.5 × upper limit of normal (ULN);alkaline phosphatase ≥ 1.5 × ULN; or, total bilirubin \> 90% of the ULN;
• Renal serum creatinine \> 1.5 mg/dL; or, significant proteinuria \> 2+ on urinary dip test;
• Hematologic hemoglobin \< 11 mg/dL; leukocytes \< 3.5 × 109/L; neutrophils \< 1.5 × 109/L; or, platelets \< 100 × 109/L;
• Presence or history of malignancy;
• Uncontrolled diabetes (defined as diabetes requiring hospitalization or emergency care in the 3 months prior to first dose of study drug);
• History or suspicion of Gilbert's syndrome;
• Significant blood loss (\> 500 mL) within 28 days prior to receipt of study drug;
• Use of an investigational drug within 30 days of screening, or longer if that drug is expected to have long-acting effects (e.g., modulation of B-cell activity);
• Use of Rituximab within the past 6 months;
• Use of intravenous IgG within the past 3 months,

Sponsors & Collaborators

• Kemia, Inc: lead

Study Sites (1)

Victoria Werth, MD

Philadelphia, Pennsylvania, 19104, United States

Location

MeSH Terms

Conditions

Pemphigus

Condition Hierarchy (Ancestors)

Skin Diseases, Vesiculobullous; Skin Diseases; Skin and Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases

Study Officials

• Victoria Werth, MD

  University of Pennsylvania

  PRINCIPAL INVESTIGATOR

• Bruce Strober, MD

  NYU MEDICAL CENTER

  PRINCIPAL INVESTIGATOR

• Francisco Kerdel, MD

  Florida Academic Dermatology Centers

  PRINCIPAL INVESTIGATOR

• Michael Kolodney, MD

  University of California, Los Angeles

  PRINCIPAL INVESTIGATOR

• Neil Korman, MD

  University Hospitals Cleveland Medical Center

  PRINCIPAL INVESTIGATOR

• Amit Pandya, MD

  University of Texas Southwestern Medical Center

  PRINCIPAL INVESTIGATOR

• David Rubenstein, MD, PhD

  University of North Carolina, Chapel Hill

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY

Study Record Dates

• First Submitted: January 22, 2008
• First Posted: February 5, 2008
• Study Start: November 1, 2007
• Primary Completion: June 1, 2008
• Study Completion: June 1, 2008
• Last Updated: June 19, 2008

Record last verified: 2008-06

Locations

US (1)