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.
| VIT-2763-SCD-202 Clinical Study Team | |
| +41 588 518 00 | |
| [email protected] |
18 Years - 60 Years
Phase 2
Interventional
All
Drug
Male or female subjects with confirmed diagnosis of SCD, including HbS/S or HbS/βT0 genotype.
Subjects who had at least 1 and no more than 10 vaso-occlusive crises (VOC) episodes reported within 12 months prior to screening.
Body weight ≥40 kg and ≤120 kg at screening and baseline.
Subjects on concomitant hydroxyurea must be on a stable dose (mg/kg) for ≥3 months prior to screening Visit V1
Female subjects of childbearing potential, must have negative pregnancy, must have stopped breastfeeding as of first dose, and must either commit to true abstinence from heterosexual contact or must be willing to use adequate contraceptive precautions.
Male subjects must practice true abstinence or agree to use a condom during sexual contact with a pregnant female or a female of childbearing potential
Hb level <6.0 g/dl or >10.4 g/dl for female participants and >11.0 g/dl for male participants, at screening Visit V1
Having received red blood cell (RBC) transfusion therapy within 4 weeks prior to screening, or ongoing or planned RBC transfusion therapy during the course of the study
Low levels of Ferritin or transferrin saturation or total iron-binding capacity at screening
Subjects being hospitalized for SCD-related events within 14 days before the screening visit
Chronic liver disease or history of liver cirrhosis, and/or high levels of alanine aminotransferase or aspartate aminotransferase at baseline
Low estimated glomerular filtration rate, and/or significant high urinary albumin/creatinine ratio at screening or on chronic dialysis.
Newly diagnosed folate deficiency anemia, which is considered clinically relevant by the Investigator at screening
Any history or clinically important finding of cardiac or pulmonary disorders
Family history of long-QT syndrome or sudden death without a preceding diagnosis of a condition that could be causative of sudden death
Clinically significant bacterial, fungal, parasitic, or viral infection which requires therapy. Note: A subject meeting this criterion should delay screening and/or enrolment for a minimum of 2 weeks, or if excluded can be re-screened at a later time point.
Concomitant use of certain hormonal contraceptives as defined in the study protocol, are not allowed within 4 weeks prior to screening and until 1 moth after the end of the study and the use of progesterone-only hormonal contraception as the sole measure to prevent pregnancy.
Pregnant or females currently breastfeeding.
History or known concomitant solid tumours and/or haematological malignancies unless resolved in the ≥2 past years, except for basal or squamous cell carcinoma of the skin, carcinoma in situ of the cervix or breast, incidental histologic finding of prostate cancer
Unable to take and absorb oral medications
Acute peptic stomach or duodenal ulcer in the previous 6 months before screening and/or healed after 3 months of treatment.
Uncontrolled hemorrhages