A Phase 2 interventional study of VIT-2763 120 mg and VIT-2763 360 mg in Sickle Cell Disease, sponsored by Vifor (International) Inc.. Completed at 22 sites in 5 countries. Open to participants aged 18 Years to 60 Years. Per ClinicalTrials.gov, last updated 2025-02-18.
Sponsored by Vifor (International) Inc. (part of CSL Limited) · Phase 2, Interventional, and Treatment
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.
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.
1,134 studies on the registry are indexed under Anemia, Sickle Cell; 238 are open to participants now.
This study's enrollment of 25 is below the median of 40 across 769 interventional studies indexed under Anemia, Sickle Cell.
Browse Anemia, Sickle Cell studies →Vifor (International) Inc. is the lead sponsor of 10 studies on the registry; none are open to participants now.
First submitted before Vifor (International) Inc. became part of CSL Limited.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GFR and eGFR (kidney filtering)
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
QT and QTc
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Participants receive VIT-2763 60 mg, twice a day during 8 weeks.
Drug: VIT-2763 120 mg
Participants receive VIT-2763 120 mg, twice a day during 8 weeks.
Drug: VIT-2763 240 mg
Participants receive VIT-2763 120 mg, three times a day during 8 weeks.
Drug: VIT-2763 360 mg
Participants receive a placebo, twice a day during 8 weeks.
Drug: Placebo BID
Participants receive a placebo, three times a day during 8 weeks.
Drug: Placebo TID
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
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
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
Participants receive 2 capsules of placebo in the morning and in the evening, for 8 weeks. Capsules are to be taken orally.
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.
Mean Change From Baseline in Haemolysis Marker (Indirect Bilirubin)
Mean change from baseline in haemolysis markers was measured by reduction of indirect bilirubin.
Time frame: Baseline and after 8 weeks of treatment
Mean Change From Baseline in Haemolysis Marker (Direct and Total Bilirubin)
Mean change from baseline in haemolysis markers was measured by direct and total bilirubin.
Time frame: Baseline and after 8 weeks of treatment
Mean Change From Baseline in Haemolysis Marker (Lactate Dehydrogenase)
Mean change from baseline in haemolysis markers was measured by lactate dehydrogenase.
Time frame: Baseline and after 8 weeks of treatment
Mean Change From Baseline in Haemolysis Marker (Potassium)
Mean change from baseline in haemolysis markers was measured by potassium.
Time frame: Baseline and after 8 weeks of treatment
Mean Change From Baseline in Haemolysis Marker (Hemoglobin and Haptoglobin)
Mean change from baseline in haemolysis markers was measured by hemoglobin and haptoglobin.
Time frame: 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
TEAEs were defined as adverse events (AEs) with an onset date later or on the same date as first investigational medicinal product (IMP) intake. The severity grading was determined according to the Common Terminology Criteria for AEs, where the Common Terminology Criteria grades relate to severity as follows: Grade 1: Mild, Grade 2: Moderate, Grade 3: Severe, Grade 4: Life-threatening and Grade 5: Death.
Time frame: From first dose of study drug up to 12 weeks
There were 22 sites initiated for this study in 5 countries (The United Kingdom, Lebanon, Greece, the United States, and France).
| Milestone | Cohort 1: VIT-2763 60 mg BID (120 mg/Day) | Cohort 2: VIT-2763 120 mg BID (240 mg/Day) | Cohort 3: VIT-2763 120 mg TID (360 mg/Day) | Cohort 4: Placebo | Cohort 2a: VIT-2763 30 mg BID (60 mg/Day) |
|---|---|---|---|---|---|
| Started | 6 | 6 | 6 | 6 | 1 |
| Intent-to-treat (itt) population | 6 | 6 | 6 | 6 | 0 |
| Completed | 5 | 6 | 5 | 6 | 1 |
| Not completed | 1 | 0 | 1 | 0 | 0 |
| Withdrew: Withdrawal by subject | 1 | 0 | 0 | 0 | 0 |
| Withdrew: Lost to follow-up | 0 | 0 | 1 | 0 | 0 |
Mean change from baseline in haemolysis markers was measured by reduction of indirect bilirubin.
| micromoles per liter (umol/L) | Cohort 1: VIT-2763 60 mg BID (120 mg/Day) | Cohort 2: VIT-2763 120 mg BID (240 mg/Day) | Cohort 3: VIT-2763 120 mg TID (360 mg/Day) | Cohort 4: Placebo |
|---|---|---|---|---|
| Baseline | 24.0 ± 13.51 | 56.4 ± 43.71 | 44.0 ± 24.48 | 31.2 ± 12.95 |
| Change at 8 weeks | -4.0 ± 4.69 | -5.8 ± 11.35 | -21.8 ± 11.62 | 0.6 ± 7.30 |
Mean change from baseline in haemolysis markers was measured by direct and total bilirubin.
| umol/L | Cohort 1: VIT-2763 60 mg BID (120 mg/Day) | Cohort 2: VIT-2763 120 mg BID (240 mg/Day) | Cohort 3: VIT-2763 120 mg TID (360 mg/Day) | Cohort 4: Placebo |
|---|---|---|---|---|
| Direct Bilirubin | -0.5 ± 1.29 | -2.0 ± 2.83 | -2.5 ± 7.05 | -0.4 ± 1.52 |
| Total Bilirubin | -4.2 ± 4.76 | -13.2 ± 16.57 | -26.0 ± 16.00 | -0.2 ± 7.08 |
Mean change from baseline in haemolysis markers was measured by lactate dehydrogenase.
| units per liter (U/L) | Cohort 1: VIT-2763 60 mg BID (120 mg/Day) | Cohort 2: VIT-2763 120 mg BID (240 mg/Day) | Cohort 3: VIT-2763 120 mg TID (360 mg/Day) | Cohort 4: Placebo |
|---|---|---|---|---|
| Mean Change From Baseline in Haemolysis Marker (Lactate Dehydrogenase) | -45.8 ± 47.56 | -24.0 ± 6.08 | 7.7 ± 197.50 | 47.8 ± 60.06 |
Mean change from baseline in haemolysis markers was measured by potassium.
| millimoles per liter (mmol/L) | Cohort 1: VIT-2763 60 mg BID (120 mg/Day) | Cohort 2: VIT-2763 120 mg BID (240 mg/Day) | Cohort 3: VIT-2763 120 mg TID (360 mg/Day) | Cohort 4: Placebo |
|---|---|---|---|---|
| Mean Change From Baseline in Haemolysis Marker (Potassium) | 0.08 ± 0.396 | 0.08 ± 0.192 | -0.22 ± 0.319 | -0.05 ± 0.295 |
Mean change from baseline in haemolysis markers was measured by hemoglobin and haptoglobin.
| grams per liter (g/L) | Cohort 1: VIT-2763 60 mg BID (120 mg/Day) | Cohort 2: VIT-2763 120 mg BID (240 mg/Day) | Cohort 3: VIT-2763 120 mg TID (360 mg/Day) | Cohort 4: Placebo |
|---|---|---|---|---|
| Hemoglobin | -3.400 ± 1.4748 | -2.067 ± 5.5142 | -1.575 ± 8.4017 | 2.733 ± 9.9933 |
| Haptoglobin | 0.102 ± 0.2281 | -0.012 ± 0.0286 | 0.528 ± 0.8312 | 0.000 ± 0.0000 |
TEAEs were defined as adverse events (AEs) with an onset date later or on the same date as first investigational medicinal product (IMP) intake. The severity grading was determined according to the Common Terminology Criteria for AEs, where the Common Terminology Criteria grades relate to severity as follows: Grade 1: Mild, Grade 2: Moderate, Grade 3: Severe, Grade 4: Life-threatening and Grade 5: Death.
| Participants | Cohort 1: VIT-2763 60 mg BID (120 mg/Day) | Cohort 2: VIT-2763 120 mg BID (240 mg/Day) | Cohort 3: VIT-2763 120 mg TID (360 mg/Day) | Cohort 4: Placebo | Cohort 2a: VIT-2763 30 mg BID (60 mg/Day) |
|---|---|---|---|---|---|
| Any TEAEs | 4 | 4 | 5 | 5 | 1 |
| TEAEs related to IMP | 0 | 0 | 0 | 1 | 0 |
| TEAEs with severity: Mild | 0 | 3 | 2 | 3 | 1 |
| TEAEs with severity: Moderate | 3 | 1 | 1 | 2 | 1 |
| TEAEs with severity: Severe | 1 | 0 | 2 | 0 | 0 |
| TEAEs with severity: Life threatening | 0 | 0 | 0 | 0 | 0 |
| TEAEs with severity: Death | 0 | 0 | 0 | 0 | 0 |
Collected over From the first dose of study drug up to 12 weeks. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Cohort 1: VIT-2763 60 mg BID (120 mg/Day) | 0/6 (0%) | 1/6 (16.7%) | 4/6 (66.7%) |
| Cohort 2: VIT-2763 120 mg BID (240 mg/Day) | 0/6 (0%) | 0/6 (0%) | 4/6 (66.7%) |
| Cohort 3: VIT-2763 120 mg TID (360 mg/Day) | 0/6 (0%) | 0/6 (0%) | 5/6 (83.3%) |
| Cohort 4: Placebo | 0/6 (0%) | 1/6 (16.7%) | 5/6 (83.3%) |
| Cohort 2a: VIT-2763 30 mg BID (60 mg/Day) | 0/1 (0%) | 1/1 (100%) | 1/1 (100%) |
| Event | Cohort 1: VIT-2763 60 mg BID (120 mg/Day) | Cohort 2: VIT-2763 120 mg BID (240 mg/Day) | Cohort 3: VIT-2763 120 mg TID (360 mg/Day) | Cohort 4: Placebo | Cohort 2a: VIT-2763 30 mg BID (60 mg/Day) |
|---|---|---|---|---|---|
| Sickle cell anaemia with crisisBlood and lymphatic system disorders | 0/6 | 0/6 | 0/6 | 1/6 | 1/1 |
| COVID-19Infections and infestations | 0/6 | 0/6 | 0/6 | 0/6 | 1/1 |
| AnaemiaBlood and lymphatic system disorders | 1/6 | 0/6 | 0/6 | 0/6 | 0/1 |
| Event | Cohort 1: VIT-2763 60 mg BID (120 mg/Day) | Cohort 2: VIT-2763 120 mg BID (240 mg/Day) | Cohort 3: VIT-2763 120 mg TID (360 mg/Day) | Cohort 4: Placebo | Cohort 2a: VIT-2763 30 mg BID (60 mg/Day) |
|---|---|---|---|---|---|
| Oropharyngeal painRespiratory, thoracic and mediastinal disorders | 0/6 | 1/6 | 0/6 | 0/6 | 1/1 |
| CoughRespiratory, thoracic and mediastinal disorders | 0/6 | 0/6 | 0/6 | 1/6 | 1/1 |
| Sickle cell anaemia with crisisBlood and lymphatic system disorders | 3/6 | 1/6 | 2/6 | 2/6 | 0/1 |
| ArthralgiaMusculoskeletal and connective tissue disorders | 1/6 | 2/6 | 0/6 | 0/6 | 0/1 |
| Back painMusculoskeletal and connective tissue disorders | 2/6 | 0/6 | 0/6 | 0/6 | 0/1 |
| MyalgiaMusculoskeletal and connective tissue disorders | 0/6 | 0/6 | 0/6 | 2/6 | 0/1 |
| HeadacheNervous system disorders | 1/6 | 2/6 | 0/6 | 0/6 | 0/1 |
| PyrexiaGeneral disorders | 0/6 | 0/6 | 0/6 | 2/6 | 0/1 |
| AnaemiaBlood and lymphatic system disorders | 0/6 | 1/6 | 0/6 | 0/6 | 0/1 |
| ThrombocytosisBlood and lymphatic system disorders | 0/6 | 0/6 | 1/6 | 0/6 | 0/1 |
This analysis was performed on the safety set. The safety set consisted of all randomized participants who had taken at least one dose of investigational medicinal product (IMP). Data for Cohort 2a are not reported here because of concerns regarding participant privacy as there was only 1 participant in the Cohort 2a arm.
| Age, Continuous(years) | Cohort 1: VIT-2763 60 mg BID (120 mg/Day) | Cohort 2: VIT-2763 120 mg BID (240 mg/Day) | Cohort 3: VIT-2763 120 mg TID (360 mg/Day) | Cohort 4: Placebo | Total |
|---|---|---|---|---|---|
| Mean | 30.2 ± 7.41 | 36.0 ± 11.87 | 29.3 ± 9.48 | 25.3 ± 7.17 | 30.2 ± 9.40 |
| Sex: Female, Male(Participants) | Cohort 1: VIT-2763 60 mg BID (120 mg/Day) | Cohort 2: VIT-2763 120 mg BID (240 mg/Day) | Cohort 3: VIT-2763 120 mg TID (360 mg/Day) | Cohort 4: Placebo | Total |
|---|---|---|---|---|---|
| Female | 4 | 3 | 3 | 3 | 13 |
| Male | 2 | 3 | 3 | 3 | 11 |
| Ethnicity (NIH/OMB)(Participants) | Cohort 1: VIT-2763 60 mg BID (120 mg/Day) | Cohort 2: VIT-2763 120 mg BID (240 mg/Day) | Cohort 3: VIT-2763 120 mg TID (360 mg/Day) | Cohort 4: Placebo | Total |
|---|---|---|---|---|---|
| Hispanic or Latino | 0 | 0 | 0 | 0 | 0 |
| Not Hispanic or Latino | 5 | 6 | 5 | 6 | 22 |
| Unknown or Not Reported | 1 | 0 | 1 | 0 | 2 |
| Race (NIH/OMB)(Participants) | Cohort 1: VIT-2763 60 mg BID (120 mg/Day) | Cohort 2: VIT-2763 120 mg BID (240 mg/Day) | Cohort 3: VIT-2763 120 mg TID (360 mg/Day) | Cohort 4: Placebo | Total |
|---|---|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 | 0 | 0 |
| Asian | 0 | 0 | 0 | 0 | 0 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 | 0 | 0 |
| Black or African American | 2 | 3 | 3 | 4 | 12 |
| White | 4 | 3 | 2 | 2 | 11 |
| More than one race | 0 | 0 | 0 | 0 | 0 |
| Unknown or Not Reported | 0 | 0 | 1 | 0 | 1 |
PDFs hosted by the registry, ClinicalTrials.gov.
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Studies of the drug tested here: VIT-2763