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

Alexion Pharmaceuticals

Status and phase: Terminated; Phase 2; Phase 1

Conditions: Pemphigus Foliaceus; Pemphigus Vulgaris; Pemphigus

Treatments: Drug: ALXN1830

Study type: Interventional

Funder types: Industry

Identifiers

NCT03075904

SYNT001-103

Details and patient eligibility

About

This was a multicenter, open-label safety study to determine the dose regimen of SYNT001 (ALXN1830) administered intravenously in participants with pemphigus (vulgaris or foliaceus).

Full description

This study planned to evaluate 2 cohorts: up to 8 participants to receive 5 weekly intravenous (IV) doses of ALXN1830 at 10 milligram/kilogram (mg/kg) (Cohort 1) and up to 12 participants to receive 3 x 30 mg/kg weekly doses of ALXN1830 IV (loading) followed by 5 x 10 mg/kg doses of ALXN1830 IV every other week or 10 weekly doses of ALXN1830 IV (maintenance) (Cohort 2).

This study was terminated after the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy were characterized in participants with pemphigus at a single dose level (10 mg/kg) in Cohort 1, before any participants were enrolled in Cohort 2.

The study consisted of 3 periods: Screening, Treatment, and Follow-Up.

Enrollment: 8 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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8 participants in 2 patient groups

Cohort 1: ALXN1830

Experimental group

Description:

Participants received 5 doses of ALXN1830 10 mg/kg administered weekly.

Treatment:

Drug: ALXN1830

Cohort 2: ALXN1830

Experimental group

Description:

Participants were to receive 3 doses of ALXN1830 30 mg/kg administered weekly (loading) followed by 5 doses of ALXN1830 10 mg/kg administered every other week or 10 weekly doses of ALXN1830 IV (maintenance).

Treatment:

Drug: ALXN1830