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

Last updated: January 27, 2025
Sponsor: Vifor (International) Inc.
Overall Status: Completed

Phase

2

Condition

Sickle Cell Disease

Red Blood Cell Disorders

Treatment

Placebo BID

Placebo TID

VIT-2763 360 mg

Clinical Study ID

NCT04817670
VIT-2763-SCD-202
2020-005072-34
  • Ages 18-60
  • All Genders

Study 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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Male or female subjects with confirmed diagnosis of SCD, including only HbS/S orHbS/βT0 genotype.

  • Subjects who had at least 1 and no more than 10 vaso-occlusive crises (VOC) episodesreported 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 monthsprior to screening Visit V1

  • Female subjects of childbearing potential, must have negative pregnancy, must havestopped breastfeeding as of first dose, and must either commit to true abstinencefrom heterosexual contact or must be willing to use adequate contraceptiveprecautions.

  • Male subjects must practice true abstinence or agree to use a condom during sexualcontact with a pregnant female or a female of childbearing potential

Exclusion

Exclusion Criteria:

  • Hb level <6.0 g/dl or >10.4 g/dl for female participants and >11.0 g/dl for maleparticipants, at screening Visit V1

  • Having received red blood cell (RBC) transfusion therapy within 4 weeks prior toscreening, or ongoing or planned RBC transfusion therapy during the course of thestudy

  • Low levels of Ferritin or transferrin saturation or total iron-binding capacity atscreening

  • Subjects being hospitalized for SCD-related events within 14 days before thescreening visit

  • Chronic liver disease or history of liver cirrhosis, and/or high levels of alanineaminotransferase or aspartate aminotransferase at baseline

  • Low estimated glomerular filtration rate, and/or significant high urinaryalbumin/creatinine ratio at screening or on chronic dialysis.

  • Newly diagnosed folate deficiency anemia, which is considered clinically relevant bythe 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 ofa condition that could be causative of sudden death

  • Clinically significant bacterial, fungal, parasitic, or viral infection whichrequires therapy. Note: A subject meeting this criterion should delay screeningand/or enrolment for a minimum of 2 weeks, or if excluded can be re-screened at alater 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 lastadministration of the study drug and the use of progesterone-only hormonalcontraception as the sole measure to prevent pregnancy.

  • Pregnant or females currently breastfeeding.

  • History or known concomitant solid tumours and/or haematological malignancies unlessresolved in the ≥2 past years, except for basal or squamous cell carcinoma of theskin, carcinoma in situ of the cervix or breast, incidental histologic finding ofprostate cancer

  • Unable to take and absorb oral medications

  • Acute peptic stomach or duodenal ulcer in the previous 6 months before screeningand/or healed after 3 months of treatment.

  • Uncontrolled hemorrhages

Study Design

Total Participants: 25
Treatment Group(s): 5
Primary Treatment: Placebo BID
Phase: 2
Study Start date:
November 18, 2021
Estimated Completion Date:
March 07, 2024

Study 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.

Connect with a study center

  • Investigator Site 801

    Colombes, 92700
    France

    Site Not Available

  • Investigator Site 802

    Lyon, 690003
    France

    Site Not Available

  • Investigator Site 305

    Athens, 11527
    Greece

    Site Not Available

  • Investigator site 301

    Athens, GR-11527
    Greece

    Site Not Available

  • Investigator Site 302

    Patra,
    Greece

    Site Not Available

  • Investigator Site 101

    Baabda,
    Lebanon

    Site Not Available

  • Investigator Site 102

    Beirut,
    Lebanon

    Site Not Available

  • Investigator Site 103

    Tripoli,
    Lebanon

    Site Not Available

  • Investigator Site 606

    Liverpool, L9 7AL
    United Kingdom

    Site Not Available

  • Investigator Site 601

    London,
    United Kingdom

    Site Not Available

  • Investigator Site 603

    London, SE59RS
    United Kingdom

    Site Not Available

  • Investigator Site 605

    London,
    United Kingdom

    Site Not Available

  • Investigator Site 608

    London, W12 0HS
    United Kingdom

    Site Not Available

  • Investigator Site 607

    Manchester, M13 9WL
    United Kingdom

    Site Not Available

  • Investigator Site 709

    Birmingham, Alabama 35233-2110
    United States

    Site Not Available

  • Investigator Site 708

    Los Angeles, California 90027
    United States

    Site Not Available

  • Investigator Site 713

    Aurora, Colorado 80045
    United States

    Site Not Available

  • Investigator Site 714

    Washington, District of Columbia 20060
    United States

    Site Not Available

  • Investigator Site 706

    Hollywood, Florida 33023
    United States

    Site Not Available

  • Investigator Site 703

    Chicago, Illinois 60612
    United States

    Site Not Available

  • Investigator Site 701

    Greenville, North Carolina 27834
    United States

    Site Not Available

  • Investigator Site 711

    Charleston, South Carolina 29425
    United States

    Site Not Available

  • Investigator Site 702

    Milwaukee, Wisconsin 53226
    United States

    Site Not Available

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