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

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Primary Purpose

Status

Terminated

Phase

Phase 1

Locations

International

Study Type

Interventional

Intervention

ALXN1830

About this trial

This is an interventional treatment trial for Warm Autoimmune Hemolytic Anemia

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept 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

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Arm Label

Cohort 1: ALXN1830

Cohort 2: ALXN1830

Arm Description

SYNT001 Dose 1

SYNT001 Dose 2

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

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).

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.

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.

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.

Full Information

NCT ID

NCT03075878

First Posted

March 3, 2017

Last Updated

May 1, 2020

Sponsor

Alexion

1. Study Identification

Unique Protocol Identification Number

NCT03075878

Brief Title

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

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

May 2020

Overall Recruitment Status

Terminated

Why Stopped

Focus resources on a planned phase 2/3 study

Study Start Date

January 10, 2018 (Actual)

Primary Completion Date

April 15, 2019 (Actual)

Study Completion Date

August 6, 2019 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Alexion

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

No

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

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

Detailed 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Warm Autoimmune Hemolytic Anemia

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1, Phase 2

Interventional Study Model

Sequential Assignment

Masking

None (Open Label)

Allocation

Non-Randomized

Enrollment

8 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Cohort 1: ALXN1830

Arm Type

Experimental

Arm Description

SYNT001 Dose 1

Arm Title

Cohort 2: ALXN1830

Arm Type

Experimental

Arm Description

SYNT001 Dose 2

Intervention Type

Drug

Intervention Name(s)

ALXN1830

Other Intervention Name(s)

SYNT001

Intervention Description

Administered via IV infusion.

Primary Outcome Measure Information:

Title

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

Description

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.

Time Frame

Day 0 (after first dose) through Day 112

Secondary Outcome Measure Information:

Title

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

Description

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).

Time Frame

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

Title

Change From Baseline In Reticulocyte Count At Day 33

Description

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.

Time Frame

Baseline, Day 33

Title

Change From Baseline In Hemoglobin At Day 33

Description

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.

Time Frame

Baseline, Day 33

Title

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

Description

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.

Time Frame

Day 112

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

No

Eligibility Criteria

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

Facility Information:

Facility Name

Alexion Study Site

City

Los Angeles

State/Province

California

ZIP/Postal Code

90033

Country

United States

Facility Name

Alexion Study Site

City

San Francisco

State/Province

California

ZIP/Postal Code

94143

Country

United States

Facility Name

Alexion Study Site

City

Boston

State/Province

Massachusetts

ZIP/Postal Code

02114

Country

United States

Facility Name

Alexion Study Site

City

Pittsfield

State/Province

Massachusetts

ZIP/Postal Code

01201

Country

United States

Facility Name

Alexion Study Site

City

Rochester

State/Province

Minnesota

ZIP/Postal Code

55905

Country

United States

Facility Name

Alexion Study Site

City

Cleveland

State/Province

Ohio

ZIP/Postal Code

44106

Country

United States

Facility Name

Alexion Study Site

City

Philadelphia

State/Province

Pennsylvania

ZIP/Postal Code

19104

Country

United States

Facility Name

Alexion Study Site

City

Pittsburgh

State/Province

Pennsylvania

ZIP/Postal Code

15232

Country

United States

Facility Name

Alexion Study Site

City

Seattle

State/Province

Washington

ZIP/Postal Code

98195

Country

United States

Facility Name

Alexion Study Site

City

Amman

ZIP/Postal Code

11941

Country

Jordan

12. IPD Sharing Statement

Plan to Share IPD

No

Warm Autoimmune Hemolytic Anemia

About this trial

Eligibility Criteria

Sites / Locations

Arms of the Study

Arm 1

Arm 2

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

1. Study Identification

2. Study Status

3. Sponsor/Collaborators

4. Oversight

5. Study Description

6. Conditions and Keywords

7. Study Design

8. Arms, Groups, and Interventions

10. Eligibility

12. IPD Sharing Statement

Learn more about this trial