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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03075904
• Date of registration: 06/03/2017
• Prospective Registration: Yes
• Primary sponsor: Alexion Pharmaceuticals
• Public title: A Safety and Dose-Finding Study of SYNT001 in Subjects With Pemphigus (Vulgaris or Foliaceus)
• Scientific title: A Phase 1B/2, Multicenter, Open-Label, Safety, and Dose-Finding Study of SYNT001 in Subjects With Pemphigus (Vulgaris or Foliaceus)
• Date of first enrolment: July 18, 2017
• Target sample size: 8
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT03075904
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Sequential Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1/Phase 2

Countries of recruitment

United States

Key inclusion & exclusion criteria

Inclusion Criteria:

Participants must have meet the following criteria to be included:

- Were willing and able to read, understand and sign an informed consent form

- Documented diagnosis of pemphigus vulgaris or foliaceus

- Were required to use medically acceptable contraception

Exclusion Criteria:

Participants meeting any of the following criteria were excluded:

- Were 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 human immunodeficiency virus (HIV) or hepatitis C antibody

- Positive for hepatitis B surface antigen

- IV immunoglobulin treatment 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

- Participant had any current medical condition that, in the opinion of the
Investigator, may have compromised their safety or compliance, preclude successful
conduct of the study, or interfere with interpretation of the results



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Pemphigus Vulgaris

Pemphigus

Pemphigus Foliaceus

Intervention(s)

Drug: ALXN1830

Primary Outcome(s)

Count Of Participants Reporting Treatment-emergent Adverse Events (TEAEs) [Time Frame: Day 1 (after first dose) through Day 112]

Secondary Outcome(s)

Maximum Percent Reduction Of Mean Anti-Desmoglein (Dsg) 1 And 3 Antibodies From Baseline [Time Frame: Baseline through Day 112]

Maximum Percent Reduction In Mean Pemphigus Disease Area Index (PDAI) Total Activity Score From Baseline [Time Frame: Baseline through Day 112]

Maximum Percent Reduction Of Mean Circulating Immune Complexes (CIC) Levels From Baseline [Time Frame: Baseline through Day 112]

Maximum Percent Reduction Of Mean Total Immunoglobulin G (IgG) Levels From Baseline [Time Frame: Baseline through Day 112]

Secondary ID(s)

SYNT001-103

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 05/02/2020
• URL: https://clinicaltrials.gov/ct2/show/results/NCT03075904