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

NCT04253236 | Terminated Phase 2 Results FDA Drug

• 1 other identifier: RVT-1401-2003
• Study Type: interventional
• Target: 5
• Locations: 6 countries
• Sites: 21

Brief Summary

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.

Conditions

Warm Autoimmune Hemolytic Anemia

Outcome Measures

Primary Outcomes (2)

• Number of Responders at Week 13

  Responders were defined as the participants with level of hemoglobin (Hb) \>=10 grams per deciliter (g/dL) with at least a \>=2 g/dL increase from Baseline without rescue therapy or blood transfusions in the previous two weeks.

  Week 13

• Number of Participants With Any Treatment-emergent Adverse Event (TEAE), Serious AE (SAE), Treatment-related Adverse Event (AE), and Death

  AEs were defined as any untoward medical occurrence in a participant or clinical investigation participant, temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. Clinically significant changes determined by the Investigator such as vital signs, Electrocardiograms (ECGs), and clinical laboratory values were also reported as AEs. TEAEs were defined as AEs that either started on or after the date of the first dose of study drug. SAEs were defined as any untoward medical occurrence that, at any dose: resulted in death, was life-threatening, required hospitalization or prolongation of existing hospitalization, resulted in disability/incapacity, was a congenital anomaly/birth defect, or was an important medical event that may have jeopardized the participant or may have required medical or surgical intervention to prevent one of the other outcomes listed in the definition.

  Up to Week 20

Secondary Outcomes (8)

• Time to Response

  Up to Week 13

• Time to Achieving Hb Levels in the Normal Range

  Up to Week 13

• Number of Participants With Change in Functional Assessment of Chronic Illness Therapy-fatigue (FACIT-F) Score

  Up to Week 13

• Number of Participants With Change in Medical Research Council (MRC) Breathlessness Scale

  Up to Week 13

• Number of Participants With Change in Euro Quality-5 Dimension-3 Level (EQ-5D-3L) Score

  Up to Week 20

Study Arms (2)

Cohort 1

EXPERIMENTAL

Dosing Regimen A - 680 mg weekly for 12 weeks via once weekly subcutaneous (SC) injections

Drug: RVT-1401 680 mg/weekly

Cohort 2

EXPERIMENTAL

Dosing Regimen B - 340 mg weekly for 12 weeks via once weekly subcutaneous (SC) injections

Drug: RVT-1401 340 mg/weekly

Interventions

RVT-1401 680 mg/weekly DRUG

Non-randomized subjects will receive subcutaneous injection of 680 mg weekly for 12 weeks of RVT-1401

Cohort 1

RVT-1401 340 mg/weekly DRUG

Non-randomized subjects will receive subcutaneous injection of 340 mg weekly for 12 weeks of RVT-1401

Cohort 2

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

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.

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).

Sponsors & Collaborators

• Immunovant Sciences GmbH: lead

Study Sites (21)

University of Iowa Hospitals & Clinics

Iowa City, Iowa, 52242, United States

Location

Norton Cancer Institute

Louisville, Kentucky, 40202, United States

Location

Massachusetts General Hospital Cancer Center - Hematology/Oncology

Boston, Massachusetts, 02114, United States

Location

University of Michigan - Internal Medicine Division of Hematology/Oncology

Ann Arbor, Michigan, 48109, United States

Location

Leo W. Jenkins Cancer Center

Greenville, North Carolina, 27834, United States

Location

Ha'Emek Medical Center

Afula, 1834111, Israel

Location

Carmal MC

Haifa, 3436212, Israel

Location

Meir Medical Center

Kfar Saba, 4428164, Israel

Location

Gachon University Gil Medical Center

Incheon, 21565, South Korea

Location

Seoul National University Bundang Hospital

Seongnam, 13620, South Korea

Location

Korea University Anam Hospital

Seoul, 02841, South Korea

Location

Seoul National University Hospital - Department of Internal Medicine

Seoul, 03080, South Korea

Location

Asan Medical Center

Seoul, 05-505, South Korea

Location

Samsung Medical Center

Seoul, 06351, South Korea

Location

Hospital Universitario Quirónsalud Madrid

Barcelona, 08035, Spain

Location

Hospital Universitario Quirón

Madrid, 28223, Spain

Location

Faculty of Medicine, Chulalongkorn University, King Chulalongkorn Memorial Hospital

Bangkok, 10330, Thailand

Location

Faculty of Medicine, Chiang Mai University, Maharaj Nakorn Chiang Mai Hospital

Chiang Mai, 50200, Thailand

Location

Faculty of Medicine, Prince of Songkla University,Songklanagarind Hospital

Hat Yai, 90110, Thailand

Location

Faculty of Medicine, Khon Kaen University, Srinagarind Hospital

Khon Kaen, 40002, Thailand

Location

Royal Cornwall Hospital

Truro, TR1 3LJ, United Kingdom

Location

Results Point of Contact

• Title: Central Study Contact
• Organization: Immunovant, Inc

clinicaltrials@immunovant.com

1-800-797-0414

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 21, 2020
• First Posted: February 5, 2020
• Study Start: August 11, 2020
• Primary Completion: April 1, 2021
• Study Completion: April 1, 2021
• Last Updated: July 28, 2022
• Results First Posted: July 28, 2022

Record last verified: 2022-07

Locations

ES (2); KR (6); US (5); IL (3); TH (4); GB (1)