To Assess the Efficacy and Safety of RVT-1401 in the Treatment of Warm Autoimmune Hemolytic Anemia (ASCEND-WAIHA).

Stopped early · Phase 2

Conditions studied: Warm Autoimmune Hemolytic Anemia

In brief

This is a Phase 2 non-randomized, open-label study to investigate the efficacy, safety and tolerability of RVT-1401 in patients with Warm Autoimmune Hemolytic Anemia.

Key facts

Study ID: NCT04253236
Run by: Immunovant Sciences GmbH
People needed: 5
Starts: 2020-08-11
Expected to finish: 2021-04-01
Last updated by the study team: 2022-07-28

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

Male or female ≥ 18 years of age.
Diagnosis of primary or secondary WAIHA as documented by a positive direct antiglobulin test (DAT) specific for anti-IgG alone or anti-IgG plus C3d.
Secondary WAIHA may only include Stage 0 chronic lymphocytic leukemia (CLL) in which separate treatment is not indicated, nor anticipated to require active management for the duration of the study.
Have failed or not tolerated at least one prior WAIHA treatment regimen as per local standards (e.g., steroids, rituximab, azathioprine, cyclophosphamide, cyclosporine, mycophenolate mofetil (MMF), danazol, or vincristine). Failure is defined as worsening or refractory disease despite steroids and or immunosuppressants.
Participants with splenectomy ≥3 months from Day 1 who are up to date on vaccinations (based on age and local guidance) are allowed.
At Screening and Baseline, subject's hemoglobin level must be <10 g/dL and the subject must have documented symptoms related to anemia (e.g., weakness, dizziness, fatigue, shortness of breath, chest pain).
Subject's concurrent treatment for WAIHA may consist only of steroids (stable dose for at least two weeks prior to Day 1), immunosuppressant therapy (azathioprine, MMF, or cyclosporine) that has been at a stable dose for at least four weeks prior to Day 1, or erythropoietin (stable dose for at least 6 weeks prior to Day 1). [Note: starting doses of WAIHA therapy must be maintained throughout the study except in the case of a rescue medication as per local standards for safety. Steroid taper down to 10 mg/day will be allowed for participants who achieve response for at least 2 weeks.]
A female participant is eligible to participate if she is of:
Non-childbearing potential defined as pre-menopausal females with a documented bilateral tubal ligation, bilateral oophorectomy (removal of the ovaries) or hysterectomy; hysteroscopic sterilization, or postmenopausal defined as 12 months of spontaneous amenorrhea.
Child-bearing potential and agrees to use one of the contraception methods listed in the protocol for an appropriate period of time (as determined by the product label or Principal Investigator) prior to the start of dosing to sufficiently minimize the risk of pregnancy at that point. Female participants must agree to use contraception until 90 days after the last dose of study treatment.
Male participants must agree to use one of the contraception methods listed in the protocol. This criterion must be followed from the time of the first dose of study treatment until 90 days after the last dose of study treatment.
Willing and capable of giving written informed consent, which includes compliance with the requirements and restrictions listed in the consent form.
Other, more specific inclusion criteria are defined in the protocol.

You may not qualify if…

Participants with other types of AIHA (e.g., cold antibody AIHA, cold agglutinin syndrome, mixed type AIHA, or paroxysmal cold hemoglobinuria).
Participants requiring more than 2 units of RBC per week in the 2 weeks prior to Screening and Baseline.
Use of rituximab, any monoclonal antibody for immunomodulation, or proteasome inhibitor, within the past 3 months prior to Screening.
Immunoglobulins given by SC, IV (IVIG), or intramuscular route, or plasmapheresis/plasma exchange (PE) within 60 days before Screening.
Total IgG level <6 g/L (at Screening).
Absolute neutrophil count <1000 cells/mm3(at Screening).
Other, more specific exclusion criteria are defined in the protocol.

Where it is running

University of Iowa Hospitals & Clinics — Iowa City, Iowa, United States
Norton Cancer Institute — Louisville, Kentucky, United States
Massachusetts General Hospital Cancer Center - Hematology/Oncology — Boston, Massachusetts, United States
University of Michigan - Internal Medicine Division of Hematology/Oncology — Ann Arbor, Michigan, United States
Leo W. Jenkins Cancer Center — Greenville, North Carolina, United States
Ha'Emek Medical Center — Afula, Israel
Carmal MC — Haifa, Israel
Meir Medical Center — Kfar Saba, Israel
Gachon University Gil Medical Center — Incheon, South Korea
Seoul National University Bundang Hospital — Seongnam, South Korea
Korea University Anam Hospital — Seoul, South Korea
Seoul National University Hospital - Department of Internal Medicine — Seoul, South Korea
Asan Medical Center — Seoul, South Korea
Samsung Medical Center — Seoul, South Korea
Hospital Universitario Quirónsalud Madrid — Barcelona, Spain
Hospital Universitario Quirón — Madrid, Spain
Faculty of Medicine, Chulalongkorn University, King Chulalongkorn Memorial Hospital — Bangkok, Thailand
Faculty of Medicine, Chiang Mai University, Maharaj Nakorn Chiang Mai Hospital — Chiang Mai, Thailand
Faculty of Medicine, Prince of Songkla University,Songklanagarind Hospital — Hat Yai, Thailand
Faculty of Medicine, Khon Kaen University, Srinagarind Hospital — Khon Kaen, Thailand
Royal Cornwall Hospital — Truro, United Kingdom

Full record on ClinicalTrials.gov

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