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

NCT04817670 | Completed Phase 2 Results DMC FDA Drug

• 2 other identifiers: VIT-2763-SCD-2022020-005072-34
• Study Type: interventional
• Target: 25
• Locations: 5 countries
• Sites: 22

Brief 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.

Conditions

Sickle Cell Disease

Keywords

anemia; sickle cell disease; vamifeport

Outcome Measures

Primary Outcomes (1)

• Mean Change From Baseline in Haemolysis Marker (Indirect Bilirubin)

  Mean change from baseline in haemolysis markers was measured by reduction of indirect bilirubin.

  Baseline and after 8 weeks of treatment

Secondary Outcomes (5)

• Mean Change From Baseline in Haemolysis Marker (Direct and Total Bilirubin)

  Baseline and after 8 weeks of treatment

• Mean Change From Baseline in Haemolysis Marker (Lactate Dehydrogenase)

  Baseline and after 8 weeks of treatment

• Mean Change From Baseline in Haemolysis Marker (Potassium)

  Baseline and after 8 weeks of treatment

• Mean Change From Baseline in Haemolysis Marker (Hemoglobin and Haptoglobin)

  Baseline and after 8 weeks of treatment

• Number of Participants With Treatment-emergent Adverse Events (TEAEs), TEAEs Related to IMP and by Severity of TEAEs

  From first dose of study drug up to 12 weeks

Study Arms (5)

Cohort 1

EXPERIMENTAL

Participants receive VIT-2763 60 mg, twice a day during 8 weeks.

Drug: VIT-2763 120 mg

Cohort 2

EXPERIMENTAL

Participants receive VIT-2763 120 mg, twice a day during 8 weeks.

Drug: VIT-2763 240 mg

Cohort 3

EXPERIMENTAL

Participants receive VIT-2763 120 mg, three times a day during 8 weeks.

Drug: VIT-2763 360 mg

Cohort 4a

PLACEBO COMPARATOR

Participants receive a placebo, twice a day during 8 weeks.

Drug: Placebo BID

Cohort 4b

PLACEBO COMPARATOR

Participants receive a placebo, three times a day during 8 weeks.

Drug: Placebo TID

Interventions

VIT-2763 120 mg DRUG

Participants receive 2 capsules of VIT-2763 30 mg in the morning and in the evening, for 8 weeks. Capsules are to be taken orally.

Also known as: Vamifeport

Cohort 1

VIT-2763 360 mg DRUG

Participants receive 2 capsules of VIT-2763 60 mg in the morning, in the afternoon and in the evening for 8 weeks. Capsules are to be taken orally.

Also known as: Vamifeport

Cohort 3

VIT-2763 240 mg DRUG

Participants receive 2 capsules of VIT-2763 60 mg in the morning and in the evening, for 8 weeks. Capsules are to be taken orally.

Also known as: Vamifeport

Cohort 2

Placebo BID DRUG

Participants receive 2 capsules of placebo in the morning and in the evening, for 8 weeks. Capsules are to be taken orally.

Cohort 4a

Placebo TID DRUG

Participants receive 2 capsules of Placebo in the morning, in the afternoon and in the evening, for 8 weeks. Capsules are to be taken orally.

Cohort 4b

Eligibility Criteria

• Age: 18 Years - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

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.

Sponsors & Collaborators

• Vifor (International) Inc.: lead
• Fortrea: collaborator

Study Sites (22)

Investigator Site 709

Birmingham, Alabama, 35233-2110, United States

Location

Investigator Site 708

Los Angeles, California, 90027, United States

Location

Investigator Site 713

Aurora, Colorado, 80045, United States

Location

Investigator Site 706

Hollywood, Florida, 33023, United States

Location

Investigator Site 703

Chicago, Illinois, 60612, United States

Location

Investigator Site 701

Greenville, North Carolina, 27834, United States

Location

Investigator Site 711

Charleston, South Carolina, 29425, United States

Location

Investigator Site 702

Milwaukee, Wisconsin, 53226, United States

Location

Investigator Site 801

Colombes, 92700, France

Location

Investigator Site 802

Lyon, 690003, France

Location

Investigator Site 305

Athens, 11527, Greece

Location

Investigator site 301

Athens, GR-11527, Greece

Location

Investigator Site 302

Pátrai, Greece

Location

Investigator Site 101

Baabda, Lebanon

Location

Investigator Site 102

Beirut, Lebanon

Location

Investigator Site 103

Tripoli, Lebanon

Location

Investigator Site 606

Liverpool, L9 7AL, United Kingdom

Location

Investigator Site 603

London, SE59RS, United Kingdom

Location

Investigator Site 608

London, W12 0HS, United Kingdom

Location

Investigator Site 601

London, United Kingdom

Location

Investigator Site 605

London, United Kingdom

Location

Investigator Site 607

Manchester, M13 9WL, United Kingdom

Location

Related Publications (2)

• Nyffenegger N, Zennadi R, Kalleda N, Flace A, Ingoglia G, Buzzi RM, Doucerain C, Buehler PW, Schaer DJ, Durrenberger F, Manolova V. The oral ferroportin inhibitor vamifeport improves hemodynamics in a mouse model of sickle cell disease. Blood. 2022 Aug 18;140(7):769-781. doi: 10.1182/blood.2021014716.

  PMID: 35714304 BACKGROUND

• Parrow NL, Doherty JM, Conrey A, Thein SL, Fleming RE. Relationships Between Markers of Iron Status and Hematological Parameters in Patients With Sickle Cell Disease. Adv Hematol. 2024 Dec 3;2024:9872440. doi: 10.1155/ah/9872440. eCollection 2024.

  PMID: 39659429 DERIVED

Related Links

• Related Info

MeSH Terms

Conditions

Anemia, Sickle Cell; Anemia

Interventions

VIT-2763

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Results Point of Contact

• Title: Study Director
• Organization: CSL Behring

clinicaltrials@cslbehring.com

+1 610-878-4000

Study Officials

• Ricardo Hermosilla, PhD

  Vifor (International) Inc.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 23, 2021
• First Posted: March 26, 2021
• Study Start: November 18, 2021
• Primary Completion: March 7, 2024
• Study Completion: March 7, 2024
• Last Updated: February 18, 2025
• Results First Posted: February 18, 2025

Record last verified: 2025-01

Data Sharing

• IPD Sharing: Will not share

Locations

LE (3); GB (6); US (8); GR (3); FR (2)