NCT04364269

Safety, Tolerability, Pharmacokinetics (PK), Pharmacodynamics (PD) and Preliminary Efficacy of VIT-2763 in β-thalassaemia

A Phase 2a, Double-blind, Randomised, Placebo-controlled, Parallel Group, Multicentre Study on Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of Multiple Doses of VIT-2763 in Subjects With Non-transfusion Dependent β-thalassaemia

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 35 (actual)
• Sponsor: Vifor (International) Inc. · Industry
• Sex: All
• Age: 12 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

This is a randomised, double-blind, placebo-controlled parallel group trial to investigate the safety, tolerability and efficacy of multiple doses of VIT-2763 versus placebo in participants with non-transfusion-dependent Beta-thalassemia (NTDT).

Detailed description

The study includes a 12-week treatment period and a safety follow-up period of 4 weeks. About 36 participants (adults and adolescents) are expected to take part in this study at a number of different institutions internationally. Adult Participants (Cohort I) will be randomized to receive either VIT-2763 once daily (QD) or twice daily (BID) or placebo, at a dose of 120 mg or 60mg depending on their body weight. Following cohort I review, adolescent participants (Cohort II) will be randomized to the same study arms with the same interventions. The study medication will be given as oral capsules, containing 60 mg of VIT-2763 or placebo.

Conditions

• Beta-Thalassemia
• Non-transfusion-dependent Thalassemia

Interventions

Type	Name	Description
DRUG	VIT-2763 once a day (QD)	Participants will receive VIT-2763 QD at a dose of 60 mg if their body weight is between 40 kg to 59 kg or at a dose of 120 mg if their body weight is between 60 kg and 100 kg, during 12 weeks.
DRUG	VIT-2763 twice a day (BID)	Participants will receive VIT-2763 BID at a dose of 60 mg if their body weight is between 40 kg to 59 kg or at a dose of 120 mg if their body weight is between 60 kg and 100 kg, during 12 weeks.
DRUG	Placebo	Participants will receive hard capsules of Placebo, twice a day.

Timeline

• Start date: 2020-06-11
• Primary completion: 2021-10-11
• Completion: 2021-11-03
• First posted: 2020-04-28
• Last updated: 2023-12-14
• Results posted: 2023-12-14

Locations

16 sites across 5 countries: Greece, Israel, Italy, Lebanon, Thailand