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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT03075878
Recruitment Status : Terminated (Focus resources on a planned phase 2/3 study)
First Posted : March 9, 2017
Last Update Posted : September 23, 2019
Information provided by (Responsible Party):
Alexion Pharmaceuticals

Brief Summary:
This study is being conducted to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), immunogenicity, and effects on warm autoimmune hemolytic anemia (WAIHA) disease activity markers of intravenous (IV) SYNT001.

Condition or disease Intervention/treatment Phase
Anemia, Hemolytic, Autoimmune Drug: SYNT001 Phase 1 Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 8 participants
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 1b/2, Multicenter, Open-Label, Safety, Tolerability, and Activity Study of SYNT001 in Subjects With Warm Autoimmune Hemolytic Anemia (WAIHA)
Actual Study Start Date : January 10, 2018
Actual Primary Completion Date : August 19, 2019
Actual Study Completion Date : August 19, 2019

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Anemia

Arm Intervention/treatment
Experimental: Cohort 1
SYNT001 Dose 1
Drug: SYNT001
250mL IV infused over 1 hour

Experimental: Cohort 2
SYNT001 Dose 2
Drug: SYNT001
250mL IV infused over 1 hour

Primary Outcome Measures :
  1. Assessment of safety data and tolerability [ Time Frame: Day 0 - Day 112 or Day 0 - Day 140 ]
    counts and percentages of adverse events and serious adverse events, physical exams, vital signs, ECGs, and clinical laboratory data

Secondary Outcome Measures :
  1. Pharmacokinetics (serum concentration of SYNT001) [ Time Frame: Day 0, 1, 2, 5, 28, 29, 30, 33 or Day 0, 7, 14, 28, 84 ]
    serum concentration of SYNT001

  2. Hematocrit [ Time Frame: Day 0, 7, 14, 21, 28, 33, 42, 56, 84, 112 or Day 0, 7, 14, 28, 42, 56, 70, 84, 91, 140 ]
    disease activity marker

  3. Hemoglobin [ Time Frame: Day 0, 7, 14, 21, 28, 33, 42, 56, 84, 112 or Day 0, 7, 14, 28, 42, 56, 70, 84, 91, 140 ]
    disease activity marker

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

Subjects must meet the following criteria to be included:

  • Willing and able to read, understand and sign an informed consent form
  • Confirmed diagnosis by enrolling physician of WAIHA
  • Must use medically acceptable contraception

Exclusion Criteria:

Subjects meeting any of the following criteria are to be excluded:

  • Subject 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 HIV 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
  • IVIG treatment within 30 days of screening
  • Plasmapheresis or immunoadsorption within 30 days of screening
  • Subject has any current medical condition that, in the opinion of the Investigator, may compromise their safety or compliance, preclude successful conduct of the study, or interfere with interpretation of the results

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03075878

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United States, California
USC - Norris Comprehensive Cancer Center
Los Angeles, California, United States, 90033
University of California San Francisco - Outpatient Hematology Clinic/Infusion Center
San Francisco, California, United States, 94143
United States, Massachusetts
Massachusetts General Hospital - Cancer Center
Boston, Massachusetts, United States, 02114
Berkshire Medical Center, Cancer and Infusion Center
Pittsfield, Massachusetts, United States, 01201
United States, Minnesota
Mayo Clinic - Rochester
Rochester, Minnesota, United States, 55905
United States, Ohio
Cleveland Clinic Taussig Cancer Center
Cleveland, Ohio, United States, 44106
United States, Pennsylvania
Hospital of the University of Pennsylvania
Philadelphia, Pennsylvania, United States, 19104
UPMC Hillman Cancer Center
Pittsburgh, Pennsylvania, United States, 15232
United States, Washington
University of Washington Medical Center
Seattle, Washington, United States, 98195
Al-Essra Hospital
Amman, Jordan, 11941
Sponsors and Collaborators
Alexion Pharmaceuticals

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Responsible Party: Alexion Pharmaceuticals Identifier: NCT03075878     History of Changes
Other Study ID Numbers: SYNT001-102
First Posted: March 9, 2017    Key Record Dates
Last Update Posted: September 23, 2019
Last Verified: September 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Anemia, Hemolytic
Anemia, Hemolytic, Autoimmune
Hematologic Diseases
Pathologic Processes
Autoimmune Diseases
Immune System Diseases