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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03075878
• Date of registration: 03/03/2017
• Prospective Registration: Yes
• Primary sponsor: Alexion Pharmaceuticals
• Public title: A Safety Study of SYNT001 in Participants With Warm Autoimmune Hemolytic Anemia (WAIHA)
• Scientific title: A Phase 1B/2, Multicenter, Open-Label, Safety, Tolerability, and Activity Study of SYNT001 in Patients With Warm Autoimmune Hemolytic Anemia (WAIHA)
• Date of first enrolment: January 10, 2018
• Target sample size: 8
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT03075878
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Sequential Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1/Phase 2

Countries of recruitment

Jordan, United States

Key inclusion & exclusion 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



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Warm Autoimmune Hemolytic Anemia

Intervention(s)

Drug: ALXN1830

Primary Outcome(s)

Count Of Participants Reporting Treatment-emergent Adverse Events (TEAEs) [Time Frame: Day 0 (after first dose) through Day 112]

Secondary Outcome(s)

Change From Baseline In Hemoglobin At Day 33 [Time Frame: Baseline, Day 33]

Maximum Serum Concentration (Cmax) On Day 0 And Day 28 [Time Frame: Predose, 5 minutes, 2, 4, 6, 24, and 48 hours, and 5 days postdose]

Immunogenicity Of ALXN1830 At Day 112, As Assessed By Anti-ALXN1830 Antibody Level [Time Frame: Day 112]

Change From Baseline In Reticulocyte Count At Day 33 [Time Frame: Baseline, Day 33]

Secondary ID(s)

SYNT001-102

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 13/05/2020
• URL: https://clinicaltrials.gov/ct2/show/results/NCT03075878