NCT04253236

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

A Phase 2, Multicenter, Non-Randomized, Open-Label Study of RVT-1401 for the Treatment of Patients With Warm Autoimmune Hemolytic Anemia

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 5 (actual)
• Sponsor: Immunovant Sciences GmbH · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Detailed description

This is a Phase 2, non-randomized, sequential, open-label study to investigate the safety, tolerability, PK, PD, and efficacy of RVT-1401 (680 mg/weekly and 340 mg/weekly) in patients with Warm Autoimmune Hemolytic Anemia that is worsening or refractory in spite of therapy with steroids and or immunosuppressants or worsening with steroid or immunosuppressant taper. Two cohorts of participants will be enrolled in a non-randomized sequential approach. Participants will be enrolled into Cohort 1 (680 mg/weekly) first followed by Cohort 2 (340 mg/weekly). Following the initial dose at the Baseline Visit (Week 1, Day 1), study visits will occur weekly throughout the treatment period. Following the final dose at Week 12, visits will occur weekly through Week 14 and then at Week 16 and Week 20. Safety, PK, PD, and clinical assessments will be collected throughout the study. Each participant will participate in the study for up to approximately 24 weeks: up to a 4-week screening period, a 12-week treatment period, and an 8-week follow up period.

Conditions

• Warm Autoimmune Hemolytic Anemia

Interventions

Type	Name	Description
DRUG	RVT-1401 680 mg/weekly	Non-randomized subjects will receive subcutaneous injection of 680 mg weekly for 12 weeks of RVT-1401
DRUG	RVT-1401 340 mg/weekly	Non-randomized subjects will receive subcutaneous injection of 340 mg weekly for 12 weeks of RVT-1401

Timeline

• Start date: 2020-08-11
• Primary completion: 2021-04-01
• Completion: 2021-04-01
• First posted: 2020-02-05
• Last updated: 2022-07-28
• Results posted: 2022-07-28

Locations

21 sites across 6 countries: United States, Israel, South Korea, Spain, Thailand, United Kingdom

Regulatory

• FDA-regulated drug study