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

Sponsor

Vifor (International) Inc. (Industry)

Overall Status

Active, not recruiting

CT.gov ID

NCT04817670

Collaborator

Labcorp Drug Development, Inc. (Industry)

Enrollment

Locations

Arms

26.7

Anticipated Duration (Months)

2.7

Patients Per Site

0.1

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Sickle Cell Disease	Drug: VIT-2763 120 mg Drug: VIT-2763 360 mg Drug: VIT-2763 240 mg Drug: Placebo BID Drug: Placebo TID	Phase 2

Detailed 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.

Study Design

Study Type:

Interventional

Anticipated Enrollment :

24 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

Double (Participant, Investigator)

Primary Purpose:

Treatment

Official Title:

A Phase 2a, Double-blind, Randomised, Placebo-controlled, Efficacy, and Safety Study of Multiple Doses of VIT-2763 in Subjects With Sickle Cell Disease

Actual Study Start Date :

Jun 9, 2021

Anticipated Primary Completion Date :

Aug 1, 2023

Anticipated Study Completion Date :

Sep 1, 2023

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Cohort 1 Participants receive VIT-2763 60 mg, twice a day during 8 weeks.	Drug: VIT-2763 120 mg 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. Other Names: Vamifeport
Experimental: Cohort 2 Participants receive VIT-2763 120 mg, twice a day during 8 weeks.	Drug: VIT-2763 240 mg 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. Other Names: Vamifeport
Experimental: Cohort 3 Participants receive VIT-2763 120 mg, three times a day during 8 weeks.	Drug: VIT-2763 360 mg 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. Other Names: Vamifeport
Placebo Comparator: Cohort 4a Participants receive a placebo, twice a day during 8 weeks.	Drug: Placebo BID Participants receive 2 capsules of placebo in the morning and in the evening, for 8 weeks. Capsules are to be taken orally.
Placebo Comparator: Cohort 4b Participants receive a placebo, three times a day during 8 weeks.	Drug: Placebo TID 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.

Outcome Measures

Primary Outcome Measures

Mean change from baseline in haemolysis markers [8 weeks of treatment]
Measured by reduction of indirect bilirubin (umol/L)(calculated)

Secondary Outcome Measures

Mean change from baseline in haemolysis markers [8 weeks of treatment]
Measured by direct and total bilirubin (umol/L)
Mean change from baseline in haemolysis markers [8 weeks of treatment]
Measured by lactate dehydrogenase (LDH) (IU/L)
Mean change from baseline in haemolysis markers [8 weeks of treatment]
Measured by potassium (mmol/L)
Mean change from baseline in haemolysis markers [8 weeks of treatment]
Measured by Hemoglobin (Hb) level (g/L)
Mean change from baseline in haemolysis markers [8 weeks of treatment]
Measured by free haptoglobin (umol/L)
Frequency and severity of reported or observed adverse events (AEs) [8 weeks of treatment]
By system organ class (SOC) and preferred terms (PTs) using Medical Dictionary for Regulatory Activities (MedDRA) coded terms, indicating seriousness criteria and relatedness

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years to 60 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

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

Exclusion Criteria:

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Investigator Site 709	Birmingham	Alabama	United States	35233-2110
2	Investigator Site 706	Hollywood	Florida	United States	33023
3	Investigator Site 701	Greenville	North Carolina	United States	27834
4	Investigator Site 302	Patra		Greece
5	Investigator Site 101	Baabda		Lebanon
6	Investigator Site 102	Beirut		Lebanon
7	Investigator Site 103	Tripoli		Lebanon
8	Investigator Site 603	London		United Kingdom	SE59RS
9	Investigator Site 601	London		United Kingdom