Use of KC706 for the Treatment of Pemphigus Vulgaris

This study has been completed.
Sponsor:
Information provided by:
Kemia, Inc
ClinicalTrials.gov Identifier:
NCT00606749
First received: January 22, 2008
Last updated: June 18, 2008
Last verified: June 2008
  Purpose

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.


Condition Intervention Phase
Pemphigus Vulgaris
Drug: KC706
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase 2 Open-Label Uncontrolled Pilot Study of KC706 in Patients With Stable, Active Pemphigus Vulgaris

Resource links provided by NLM:


Further study details as provided by Kemia, Inc:

Primary Outcome Measures:
  • 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. [ Time Frame: 16 weeks ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Evaluate the safety of KC706 in patients with PV and to assess plasma levels of KC706 with once daily dosing. [ Time Frame: 16 weeks ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 20
Study Start Date: November 2007
Study Completion Date: June 2008
Primary Completion Date: June 2008 (Final data collection date for primary outcome measure)
Intervention Details:
    Drug: KC706
    300 mg once daily (QD) for 12 weeks.
Detailed Description:

The present study is designed to follow-up on pre-clinical observations that administration of KC706 is associated with prevention of the development of lesions in a mouse model of PV. Patients participating in this study will be those with active disease in spite of ongoing treatment with corticosteroids and/or immunosuppressive agents. The dose chosen for this clinical study is 300 mg once daily. The primary assessment of interest will be pemphigus lesion status during dosing with KC706.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 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,
    • 1 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).

Exclusion Criteria:

  • 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,
  • Current or recent history (within 12 months of screening) of drug or substance abuse, including alcohol;
  • Known or suspected pregnancy; nursing mothers;
  • Clinically significant abnormality on the screening physical examination performed at the Baseline Visit, laboratory testing, vital signs or electrocardiogram suggestive of significant unstable medical condition other than the disease under study;
  • Condition which, in the opinion of the Investigator, could interfere with participation in the study or would put the patient at unacceptable risk;
  • History of noncompliance with medical regimens.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00606749

Locations
United States, Pennsylvania
Victoria Werth, MD
Philadelphia, Pennsylvania, United States, 19104
Sponsors and Collaborators
Kemia, Inc
Investigators
Principal Investigator: 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 Hospital Case 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
  More Information

No publications provided

Responsible Party: Michael R Hodges, MD, Kemia,Inc
ClinicalTrials.gov Identifier: NCT00606749     History of Changes
Other Study ID Numbers: KC706-C08
Study First Received: January 22, 2008
Last Updated: June 18, 2008
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Pemphigus
Skin Diseases, Vesiculobullous
Skin Diseases
Autoimmune Diseases
Immune System Diseases

ClinicalTrials.gov processed this record on July 31, 2014