VIT-2763-SCD-202
Research type
Research Study
Full title
A phase 2a, double-blind, randomized, placebo-controlled, ascending dose and maintenance dose, efficacy, and safety study of multiple doses of VIT-2763 in subjects with sickle cell disease.
IRAS ID
294381
Contact name
Banu Kaya
Contact email
Sponsor organisation
Vifor (International) Inc.
Eudract number
2020-005072-34
Duration of Study in the UK
1 years, 1 months, 11 days
Research summary
This study is a Phase 2a, multiple dose, double-blind, randomised, placebo-controlled, ascending dose and maintenance dose study in Sickle Cell Disease patients. The primary objective of this study is to explore the effect of VIT-2763 (a drug inhibiting the iron transporter protein in the body called ferroportin (FPN)) This action might reduce the concentration of diseased hemoglobin preventing the destruction of red blood cells. The study shall be funded by the sponsor, Vifor (International) Inc..
This study shall be conducted at 10-15 sites across 4 countries and shall include 25 participants aged between 18-50 at the time of screening. Study participation is expected to last a maximum of 14 weeks, and this includes a non-treatment screening period of up to 2 weeks, a treatment period of 8 weeks and a 4 week safety follow up period.
VIT-2763 will be administered daily during 2 parts (A and B) of 4 weeks each. Participants shall be randomly assigned into cohorts using a central validated procedure and provided with specific doses as per protocol. At randomisation/baseline in the Part A, 25 subjects with SCD will be randomly and evenly assigned into 4 VIT-2763 dose groups to receive either 30 mg or 60 mg VIT-2763 and 1 placebo group. During Part B, subjects in Cohort 1a (VIT-2763 30 mg) and Cohort 2a (VIT-2763 60 mg) will remain on the same VIT-2763 dose as in Part A; whereas, subjects in Cohort 1b and Cohort 2b will continue with an increased dose of VIT-2763, 60 mg and 120 mg, respectively. Subjects randomised to placebo during Part A (Cohort 3) will continue unchanged during Part B.
REC name
East of England - Essex Research Ethics Committee
REC reference
21/EE/0092
Date of REC Opinion
19 May 2021
REC opinion
Further Information Favourable Opinion