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

Warm Autoimmune Hemolytic Anemia Clinical Trial

Official title:

A Phase 1B/2, Multicenter, Open-Label, Safety, Tolerability, and Activity Study of SYNT001 in Patients With Warm Autoimmune Hemolytic Anemia (WAIHA)

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT03075878
• Other study ID #: SYNT001-102
• Status: Terminated
• Phase: Phase 1/Phase 2
• Start date: January 10, 2018
• Est. completion date: August 6, 2019

Study information

• Verified date: May 2020
• Source: Alexion Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 8
• Est. completion date: August 6, 2019
• Est. primary completion date: April 15, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Anemia
• Anemia, Hemolytic
• Anemia, Hemolytic, Autoimmune
• Hemolysis
• Warm Autoimmune Hemolytic Anemia

Intervention

Drug:
• ALXN1830
Administered via IV infusion.

Locations

Country	Name	City	State
Jordan	Alexion Study Site	Amman
United States	Alexion Study Site	Boston	Massachusetts
United States	Alexion Study Site	Cleveland	Ohio
United States	Alexion Study Site	Los Angeles	California
United States	Alexion Study Site	Philadelphia	Pennsylvania
United States	Alexion Study Site	Pittsburgh	Pennsylvania
United States	Alexion Study Site	Pittsfield	Massachusetts
United States	Alexion Study Site	Rochester	Minnesota
United States	Alexion Study Site	San Francisco	California
United States	Alexion Study Site	Seattle	Washington

Sponsors (1)

Lead Sponsor	Collaborator
Alexion Pharmaceuticals

Countries where clinical trial is conducted

United States,  Jordan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Maximum Serum Concentration (Cmax) On Day 0 And Day 28	The Cmax was determined directly from the concentration-time profile. Starting on Days 0 and 28, serum samples were collected just prior to the start of study drug infusion (predose), at 5 minutes, at 2, 4, 6, 24, and 48 hours, and at 5 days postdose after the end-of-study drug infusion. Results are reported in micrograms/milliliter (ug/mL).	Predose, 5 minutes, 2, 4, 6, 24, and 48 hours, and 5 days postdose
Secondary	Change From Baseline In Reticulocyte Count At Day 33	Reticulocyte count was measured as a PD biomarker. Pharmacodynamic samples were collected for analyses throughout the study prior to infusion of study drug for Cohort 1. Measurements for PD biomarkers were derived from the laboratory results. Results are reported in cells/liter (cells*10^12/L). A decrease in reticulocyte count indicated a potential improvement in condition.	Baseline, Day 33
Secondary	Change From Baseline In Hemoglobin At Day 33	Hemoglobin was measured as a PD biomarker. Pharmacodynamic samples were collected for analyses throughout the study prior to infusion of study drug for Cohort 1. Measurements for PD biomarkers were derived from the laboratory results. Results are reported in grams/deciliter (g/dL). An increase in hemoglobin indicated a potential improvement in condition.	Baseline, Day 33
Secondary	Immunogenicity Of ALXN1830 At Day 112, As Assessed By Anti-ALXN1830 Antibody Level	Immunogenicity analyses are reported for Day 112. Testing was carried out to detect binding antidrug antibodies by anti-ALXN1830 antibody level. Results are reported as the reciprocal of the titer where they cross the study cut point.	Day 112