A Safety Study of SYNT001 in Participants With Warm Autoimmune Hemolytic Anemia (WAIHA)

Alexion Pharmaceuticals

Status and phase: Terminated; Phase 2; Phase 1

Conditions: Warm Autoimmune Hemolytic Anemia

Treatments: Drug: ALXN1830

Study type: Interventional

Funder types: Industry

Identifiers

NCT03075878

SYNT001-102

Details and patient eligibility

About

This main study objective was to evaluate the safety and tolerability of intravenous (IV) SYNT001 (ALXN1830) in participants with WAIHA.

Full description

This study planned to evaluate 2 cohorts: Cohort 1, up to 8 participants to receive IV doses of ALXN1830 (SYNT001 Dose 1); Cohort 2, up to 12 participants to receive IV doses of ALXN1830 (SYNT001 Dose 2).

This study was terminated after the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy were characterized in participants with WAIHA in Cohort 1 (SYNT001 Dose 1), before any participants were enrolled in Cohort 2.

Enrollment: 8 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

Participants had to meet the following criteria to be included:

• Willing and able to read, understand, and sign an informed consent form
• Confirmed diagnosis of WAIHA by enrolling physician
• Must have used medically acceptable contraception

Exclusion criteria

Participants who met any of the following criteria were excluded:

• Participant 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 or hepatitis C antibody
• Positive for hepatitis B surface antigen
• Any exposure to an investigational drug or device within the 30 days prior to screening
• Intravenous immunoglobulin treatment within 30 days of 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, precluded successful conduct of the study, or interfered 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:

SYNT001 Dose 1

Treatment:

Drug: ALXN1830

Cohort 2: ALXN1830

Experimental group

Description:

SYNT001 Dose 2

Treatment:

Drug: ALXN1830