Clinical trial NCT03075878: 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
Overall status: TERMINATED
Phase: PHASE1
PHASE2
Conditions: Warm Autoimmune Hemolytic Anemia
Lead sponsor: Alexion Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2017-03-09
Enrollment: 8
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

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.

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

Primary outcomes:
measure: Count Of Participants Reporting Treatment-emergent Adverse Events (TEAEs)
timeFrame: Day 0 (after first dose) through Day 112
Secondary outcomes:
measure: Maximum Serum Concentration (Cmax) On Day 0 And Day 28
timeFrame: Predose, 5 minutes, 2, 4, 6, 24, and 48 hours, and 5 days postdose
measure: Change From Baseline In Reticulocyte Count At Day 33
timeFrame: Baseline, Day 33
measure: Change From Baseline In Hemoglobin At Day 33
timeFrame: Baseline, Day 33
measure: Immunogenicity Of ALXN1830 At Day 112, As Assessed By Anti-ALXN1830 Antibody Level
timeFrame: Day 112
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Alexion Study Site
state: California
city: San Francisco
country: United States
facility: Alexion Study Site
state: California
city: Boston
country: United States
facility: Alexion Study Site
state: Massachusetts
city: Pittsfield
country: United States
facility: Alexion Study Site
state: Massachusetts
city: Rochester
country: United States
facility: Alexion Study Site
state: Minnesota
city: Cleveland
country: United States
facility: Alexion Study Site
state: Ohio
city: Philadelphia
country: United States
facility: Alexion Study Site
state: Pennsylvania
city: Pittsburgh
country: United States
facility: Alexion Study Site
state: Pennsylvania
city: Seattle
country: United States
facility: Alexion Study Site
state: Washington
city: Amman
country: Jordan
facility: Alexion Study Site
Study officials:
References: