Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

A Safety and Dose-Finding Study of SYNT001 in Subjects With Pemphigus (Vulgaris or Foliaceus)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03075904
Recruitment Status : Terminated (Study objectives achieved)
First Posted : March 9, 2017
Last Update Posted : October 3, 2019
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals

Brief Summary:
This is a multicenter, open-label safety study to determine the dose regimen of SYNT001 administered intravenously in subjects with pemphigus (vulgaris or foliaceus).

Condition or disease Intervention/treatment Phase
Pemphigus Pemphigus Vulgaris Pemphigus Foliaceus Drug: SYNT001 Phase 1 Phase 2

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 8 participants
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 1b/2, Multicenter, Open-Label, Safety, and Dose-Finding Study of SYNT001 in Subjects With Pemphigus (Vulgaris or Foliaceus)
Actual Study Start Date : August 1, 2017
Actual Primary Completion Date : January 16, 2019
Actual Study Completion Date : January 16, 2019

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Pemphigus

Arm Intervention/treatment
Experimental: Cohort 1
SYNT001 Dose 1 (weekly)
Drug: SYNT001
IV 250 mL over 1 hour

Experimental: Cohort 2
SYNT001 Dose 2 (weekly and/or every other week)
Drug: SYNT001
IV 250 mL over 1 hour




Primary Outcome Measures :
  1. Assessment of safety data (counts and percentages of adverse events) [ Time Frame: Day 0 - Day 112 or Day 0 - Day 140 ]
    counts and percentages of adverse events


Secondary Outcome Measures :
  1. Pharmacokinetics (serum concentration of SYNT001) [ Time Frame: Day 0 - Day 33 or Day 0 - Day 84 ]
    serum concentration of SYNT001

  2. Pemphigus Disease Area Index (PDAI) [ Time Frame: Screening to Day 112 or Screening to Day 140 ]
    disease activity marker



Information from the National Library of Medicine

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, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

Subjects must meet the following criteria to be included:

  • Willing and able to read, understand and sign an informed consent form
  • Documented diagnosis of pemphigus vulgaris or foliaceus
  • Must use medically acceptable contraception

Exclusion Criteria:

Subjects meeting any of the following criteria are to be excluded:

  • Subject unable or unwilling to comply with the protocol
  • Active non-hematologic malignancy or history of non-hematologic malignancy in the 3 years prior to screening (exclusive of non-melanoma skin cancer and cervical cancer in situ)
  • Positive for HIV or hepatitis C antibody
  • Positive for hepatitis B surface antigen
  • IVIG use within 30 days of screening
  • Any exposure to an investigational drug or device within the 30 days prior to screening
  • Plasmapheresis or immunoadsorption within 30 days of screening
  • Subject has any current medical condition that, in the opinion of the Investigator, may compromise their safety or compliance, preclude successful conduct of the study, or interfere with interpretation of the results

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03075904


Locations
Layout table for location information
United States, California
Alexion Study Site
Palo Alto, California, United States, 94304
United States, Georgia
Alexion Study Site
Atlanta, Georgia, United States, 30322
United States, New York
Alexion Study Site
Buffalo, New York, United States, 14203
United States, North Carolina
Alexion Study Site
Chapel Hill, North Carolina, United States, 27516
Alexion Study Site
Durham, North Carolina, United States, 27710
United States, Ohio
Alexion Study Site
Cleveland, Ohio, United States, 44106
United States, Pennsylvania
Alexion Study Site
Philadelphia, Pennsylvania, United States, 19104
Sponsors and Collaborators
Alexion Pharmaceuticals

Layout table for additonal information
Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03075904     History of Changes
Other Study ID Numbers: SYNT001-103
First Posted: March 9, 2017    Key Record Dates
Last Update Posted: October 3, 2019
Last Verified: October 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
Layout table for MeSH terms
Pemphigus
Skin Diseases, Vesiculobullous
Skin Diseases
Autoimmune Diseases
Immune System Diseases