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

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

8 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-07-18

Study Completion Date

2019-01-16

Brief Summary

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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).

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Detailed Description

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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.

Conditions

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Pemphigus Pemphigus Vulgaris Pemphigus Foliaceus

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Cohort 1: ALXN1830

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

Group Type EXPERIMENTAL

ALXN1830

Intervention Type DRUG

Administered via IV infusion.

Cohort 2: ALXN1830

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).

Group Type EXPERIMENTAL

ALXN1830

Intervention Type DRUG

Administered via IV infusion.

Interventions

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ALXN1830

Administered via IV infusion.

Intervention Type DRUG

Other Intervention Names

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SYNT001

Eligibility Criteria

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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

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No