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

Completed · Phase 2 · Has a placebo group

Conditions studied: Sickle Cell Disease

In brief

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.

Key facts

Study ID

NCT04817670

Run by

Vifor (International) Inc.

People needed

25

Starts

2021-11-18

Expected to finish

2024-03-07

Last updated by the study team

2025-02-18

Who can join

Age: 18 and older, up to 60. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Male or female subjects with confirmed diagnosis of SCD, including only 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

You may not qualify if…

• 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 week after the last administration of the study drug 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

Where it is running

• Investigator Site 709 — Birmingham, Alabama, United States
• Investigator Site 708 — Los Angeles, California, United States
• Investigator Site 713 — Aurora, Colorado, United States
• Investigator Site 706 — Hollywood, Florida, United States
• Investigator Site 703 — Chicago, Illinois, United States
• Investigator Site 701 — Greenville, North Carolina, United States
• Investigator Site 711 — Charleston, South Carolina, United States
• Investigator Site 702 — Milwaukee, Wisconsin, United States
• Investigator Site 801 — Colombes, France
• Investigator Site 802 — Lyon, France
• Investigator Site 305 — Athens, Greece
• Investigator site 301 — Athens, Greece
• Investigator Site 302 — Pátrai, Greece
• Investigator Site 101 — Baabda, Lebanon
• Investigator Site 102 — Beirut, Lebanon
• Investigator Site 103 — Tripoli, Lebanon
• Investigator Site 606 — Liverpool, United Kingdom
• Investigator Site 603 — London, United Kingdom
• Investigator Site 608 — London, United Kingdom
• Investigator Site 601 — London, United Kingdom
• Investigator Site 605 — London, United Kingdom
• Investigator Site 607 — Manchester, United Kingdom

Full record on ClinicalTrials.gov

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