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

NCT03075878 | Terminated Phase 1 Results DMC FDA Drug

• 1 other identifier: SYNT001-102
• Study Type: interventional
• Target: 8
• Locations: 2 countries
• Sites: 10

Brief Summary

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

Conditions

Warm Autoimmune Hemolytic Anemia

Outcome Measures

Primary Outcomes (1)

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

  A TEAE was defined as any adverse event 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" 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. All serious TEAEs were not considered related to the study drug.

  Day 0 (after first dose) through Day 112

Secondary Outcomes (4)

• Maximum Serum Concentration (Cmax) On Day 0 And Day 28

  Predose, 5 minutes, 2, 4, 6, 24, and 48 hours, and 5 days postdose

• Change From Baseline In Reticulocyte Count At Day 33

  Baseline, Day 33

• Change From Baseline In Hemoglobin At Day 33

  Baseline, Day 33

• Immunogenicity Of ALXN1830 At Day 112, As Assessed By Anti-ALXN1830 Antibody Level

  Day 112

Study Arms (2)

Cohort 1: ALXN1830

EXPERIMENTAL

SYNT001 Dose 1

Drug: ALXN1830

Cohort 2: ALXN1830

EXPERIMENTAL

SYNT001 Dose 2

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 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Alexion Pharmaceuticals, Inc.: lead

Study Sites (10)

Alexion Study Site

Los Angeles, California, 90033, United States

Location

Alexion Study Site

San Francisco, California, 94143, United States

Location

Alexion Study Site

Boston, Massachusetts, 02114, United States

Location

Alexion Study Site

Pittsfield, Massachusetts, 01201, United States

Location

Alexion Study Site

Rochester, Minnesota, 55905, United States

Location

Alexion Study Site

Cleveland, Ohio, 44106, United States

Location

Alexion Study Site

Philadelphia, Pennsylvania, 19104, United States

Location

Alexion Study Site

Pittsburgh, Pennsylvania, 15232, United States

Location

Alexion Study Site

Seattle, Washington, 98195, United States

Location

Alexion Study Site

Amman, 11941, Jordan

Location

MeSH Terms

Interventions

orilanolimab

Limitations and Caveats

The study was terminated after the safety, tolerability, PK, PD, and efficacy were characterized in the Warm Autoimmune Hemolytic Anemia participants in Cohort 1 (SYNT001 Dose 1).

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 3, 2017
• First Posted: March 9, 2017
• Study Start: January 10, 2018
• Primary Completion: April 15, 2019
• Study Completion: August 6, 2019
• Last Updated: May 13, 2020
• Results First Posted: May 13, 2020

Record last verified: 2020-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (9); JO (1)