NCT04817670

Study to Assess Efficacy and Safety of VIT-2763 (Vamifeport) in Subjects With Sickle Cell Disease

A Phase 2a, Double-blind, Randomised, Placebo-controlled, Efficacy, and Safety Study of Multiple Doses of VIT-2763 in Subjects With Sickle Cell Disease (ViSionSerenity)

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 25 (actual)
• Sponsor: Vifor (International) Inc. · Industry
• Sex: All
• Age: 18 Years – 60 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to investigate the effect of VIT-2763 on markers of hemolysis (breakdown in red blood cells) in sickle cell disease (SCD). The safety, tolerability and clinical beneficial effects of VIT-2763 for the treatment of SCD are also explored.

Detailed description

At randomization/baseline, participants are randomized into 3 VIT-2763 dose groups to receive either 60 mg twice daily (BID) (Cohort 1), or 120 mg BID (Cohort 2), or 120 mg 3 times daily (TID) (Cohort 3) and 2 placebo groups (BID, Cohort 4a or TID, Cohort 4b). The expected duration of patient participation is a maximum of 16 weeks, including a non-treatment screening period of up to 4 weeks, and 8-week treatment period, and a 4-week safety follow-up period.

Conditions

• Sickle Cell Disease

Interventions

Type	Name	Description
DRUG	VIT-2763 120 mg	Participants receive 2 capsules of VIT-2763 30 mg in the morning and in the evening, for 8 weeks. Capsules are to be taken orally.
DRUG	VIT-2763 360 mg	Participants receive 2 capsules of VIT-2763 60 mg in the morning, in the afternoon and in the evening for 8 weeks. Capsules are to be taken orally.
DRUG	VIT-2763 240 mg	Participants receive 2 capsules of VIT-2763 60 mg in the morning and in the evening, for 8 weeks. Capsules are to be taken orally.
DRUG	Placebo BID	Participants receive 2 capsules of placebo in the morning and in the evening, for 8 weeks. Capsules are to be taken orally.
DRUG	Placebo TID	Participants receive 2 capsules of Placebo in the morning, in the afternoon and in the evening, for 8 weeks. Capsules are to be taken orally.

Timeline

• Start date: 2021-11-18
• Primary completion: 2024-03-07
• Completion: 2024-03-07
• First posted: 2021-03-26
• Last updated: 2025-02-18
• Results posted: 2025-02-18

Locations

22 sites across 5 countries: United States, France, Greece, Lebanon, United Kingdom

Regulatory

• FDA-regulated drug study