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

NCT03075904 | Terminated Phase 1 Results DMC FDA Drug

• 1 other identifier: SYNT001-103
• Study Type: interventional
• Target: 8
• Locations: 1 country
• Sites: 3

Brief Summary

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).

Conditions

Pemphigus; Pemphigus Vulgaris; Pemphigus Foliaceus

Outcome Measures

Primary Outcomes (1)

• Count Of Participants Reporting Treatment-emergent Adverse Events (TEAEs)

  A TEAE was defined as any adverse event (AE) that starts on or after the first dose of study drug or occurs prior to the first dose and worsens in severity on or after the first dose of study drug, during the Treatment Period and Follow-up Period. A TEAE was considered "serious" (Grade 3) if, in the view of either the investigator or sponsor, it resulted in any of the following outcomes: death, life-threatening adverse drug event, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect, or an event that may have jeopardized the participant and may have required medical or surgical intervention to prevent one of the previously listed outcomes. A summary of serious and all other non-serious adverse events, regardless of causality, is located in the Reported Adverse Events module.

  Day 1 (after first dose) through Day 112

Secondary Outcomes (4)

• Maximum Percent Reduction Of Mean Total Immunoglobulin G (IgG) Levels From Baseline

  Baseline through Day 112

• Maximum Percent Reduction In Mean Pemphigus Disease Area Index (PDAI) Total Activity Score From Baseline

  Baseline through Day 112

• Maximum Percent Reduction Of Mean Circulating Immune Complexes (CIC) Levels From Baseline

  Baseline through Day 112

• Maximum Percent Reduction Of Mean Anti-Desmoglein (Dsg) 1 And 3 Antibodies From Baseline

  Baseline through Day 112

Study Arms (2)

Cohort 1: ALXN1830

EXPERIMENTAL

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

Drug: ALXN1830

Cohort 2: ALXN1830

EXPERIMENTAL

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).

Drug: ALXN1830

Interventions

ALXN1830 DRUG

Administered via IV infusion.

Also known as: SYNT001

Cohort 1: ALXN1830; Cohort 2: ALXN1830

Eligibility Criteria

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

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Alexion Pharmaceuticals, Inc.: lead

Study Sites (3)

Alexion Study Site

Chapel Hill, North Carolina, 27516, United States

Location

Alexion Study Site

Durham, North Carolina, 27710, United States

Location

Alexion Study Site

Philadelphia, Pennsylvania, 19104, United States

Location

Related Publications (1)

• Werth VP, Culton DA, Concha JSS, Graydon JS, Blumberg LJ, Okawa J, Pyzik M, Blumberg RS, Hall RP 3rd. Safety, Tolerability, and Activity of ALXN1830 Targeting the Neonatal Fc Receptor in Chronic Pemphigus. J Invest Dermatol. 2021 Dec;141(12):2858-2865.e4. doi: 10.1016/j.jid.2021.04.031. Epub 2021 Jun 12.

  PMID: 34126109 DERIVED

MeSH Terms

Conditions

Pemphigus

Interventions

orilanolimab

Condition Hierarchy (Ancestors)

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

Results Point of Contact

• Title: Alexion Pharmaceuticals, Inc.
• Organization: Alexion Pharmaceuticals, Inc.

clinicaltrials@alexion.com

855-752-2356

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 6, 2017
• First Posted: March 9, 2017
• Study Start: July 18, 2017
• Primary Completion: January 16, 2019
• Study Completion: January 16, 2019
• Last Updated: February 5, 2020
• Results First Posted: February 5, 2020

Record last verified: 2020-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (3)