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CompletedNCT04364269VITHALUpdated Dec 14, 2023Results posted

Safety, Tolerability, Pharmacokinetics (PK), Pharmacodynamics (PD) and Preliminary Efficacy of VIT-2763 in β-thalassaemia

A Phase 2 interventional study of VIT-2763 once a day (QD) and VIT-2763 twice a day (BID) in Beta-Thalassemia and Non-transfusion-dependent Thalassemia, sponsored by Vifor (International) Inc.. Completed at 16 sites in 5 countries. Open to participants aged 12 Years to 65 Years. Per ClinicalTrials.gov, last updated 2023-12-14.

Sponsored by Vifor (International) Inc. (part of CSL Limited) · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
35
Allocation
Randomized
Ages
12 Years to 65 Years
Sex
All
01

Study summary

This is a randomised, double-blind, placebo-controlled parallel group trial to investigate the safety, tolerability and efficacy of multiple doses of VIT-2763 versus placebo in participants with non-transfusion-dependent Beta-thalassemia (NTDT).

Read the detailed description

The study includes a 12-week treatment period and a safety follow-up period of 4 weeks.

About 36 participants (adults and adolescents) are expected to take part in this study at a number of different institutions internationally.

Adult Participants (Cohort I) will be randomized to receive either VIT-2763 once daily (QD) or twice daily (BID) or placebo, at a dose of 120 mg or 60mg depending on their body weight. Following cohort I review, adolescent participants (Cohort II) will be randomized to the same study arms with the same interventions.

The study medication will be given as oral capsules, containing 60 mg of VIT-2763 or placebo.

02

Conditions studied

  • Beta-Thalassemia
  • Non-transfusion-dependent Thalassemia

Browse trials for

Keywords

  • Beta-Thalassemia
03

In context

beta-Thalassemia

294 studies on the registry are indexed under beta-Thalassemia; 69 are open to participants now.

This study's enrollment of 35 is below the median of 40 across 208 interventional studies indexed under beta-Thalassemia.

Browse beta-Thalassemia studies →

Lead sponsor

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.

Browse Vifor (International) Inc. studies →All CSL Limited studies →

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
12 Years to 65 Years
Sexes eligible
All
Accepts healthy volunteers
No

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.

Inclusion criteria

  • Documented diagnosis of NTDT, including a β-thalassemia intermedia-phenotype.
  • NTDT is defined as subjects having received less than 5 units of red blood cells (RBCs) during the 24-week period prior to randomisation/first drug administration of VIT-2763 or placebo (Day 1; 1 unit is defined as 200 to 350 ml of transfused packed RBCs and last RBC transfusion must have been received at east 14 days prior to randomisation).
  • Male and female adult NTDT subjects, 18-65 years of age inclusive (Cohort I only) at time of screening.
  • Male and female adolescent NTDT subjects, 12-17 years of age inclusive (Cohort II only) at time of screening.
  • Subjects must have a mean baseline (Hb) equal to or lower than 11 g/dl, based on at least 2 consecutive measurements with at least 1 week apart within 6 weeks prior to randomisation/baseline.

Exclusion criteria

Exclusion Criteria:

  • Documented diagnosis of transfusion dependent thalassemia (TDT), including a beta-thalassemia major phenotype (including β0/β0, β+/β+, β0/β+ genotype), and mixed compound heterozygous for sickling phenotype variants such as S/β- thalassemia, or transfusion dependent non-deletional Hb H disease (i.e., Hb constant spring) or Hb C disease.
  • Subjects on concomitant iron chelation therapy (ICT) or subjects on prior ICT when discontinued less than 4 weeks prior randomisation. If ICT was discontinued at least 4 weeks prior randomization the subject is eligible.
  • ICT naïve subjects or subjects who discontinued ICT therapy at least 6 months before the screening visit with serum ferritin lower than 150 ng/ml and/or documented liver iron concentration (LIC) equal to or lower than 1 mg/g liver dry weight assessed through magnetic resonance imaging (MRI), or subjects on prior ICT with serum ferritin lower than 300 ng/ml and/or documented LIC lower than 3 mg/g liver dry weight assessed through MRI.
  • Subjects with transferrin saturation (TSAT) less than 30%.
  • Subjects with documented LIC greater than 15 mg/g liver dry weight assessed through MRI, or a documented myocardial T2* less than 20 ms, if available per local practice and retrieved within 24 months prior to randomization.
  • Adult or adolescent subjects with body weight lower than 40.0 kg or greater than 100 kg at screening.
  • Chronic liver disease and/or (ALT), aspartate transaminase (AST) or gamma-glutamyl transpeptidase (GGT) above 3-fold the (ULN) range at screening.
  • Estimated (eGFR) less than 30 ml/min/1.73 m2 (according to chronic kidney disease classification Stage 4 or higher), and/or significant albuminuria greater than 30 mg/mmol. eGFR should be estimated according to Chronic Kidney Disease Epidemiology Collaboration formula (CKI-EPI) in adults, and Schwartz formula in adolescents.
  • Newly diagnosed folate deficiency anemia and/or Vitamin B12 megaloblastic anemia. Subjects with known folate deficiency anemia and/or Vitamin B12 megaloblastic anemia who are on at least 12 weeks stable replacement therapy are eligible.
  • Any history or finding of cardiac disorders, such as clinically relevant cardiac arrhythmia, cardiomyopathy, coronary disease, valve disorder, or heart failure according to classification 3-4.
  • Subjects with history of partial or total splenectomy within 6 months prior to screening.
Terms in these criteria, in plain language(6)

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.

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).

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.

ULN (upper limit of normal)

The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.

“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.

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.

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.

NYHA class (heart failure)

The New York Heart Association’s four classes of how much heart failure limits physical activity.

I
No limits: ordinary activity causes no undue tiredness or breathlessness.
II
Slight limits: comfortable at rest, ordinary activity causes symptoms.
III
Marked limits: comfortable at rest, less than ordinary activity causes symptoms.
IV
Symptoms even at rest; any activity makes them worse.

Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.

05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment
35 participants (actual)

Study arms

  • Experimental
    VIT-2763 Once a day (QD)

    Participants will be assigned to receive VIT-2763 once a day (QD) in a total daily dose of 60 mg or 120 mg depending on their body weight. The study medication (VIT-2763 and/or matching placebo) will be administered for all participants twice a day to maintain the blind.

    Drug: VIT-2763 once a day (QD)

  • Experimental
    VIT-2763 Twice a day (BID)

    Participants will be assigned to receive VIT-2763 Twice a day (BID) in a total daily dose of 60 mg or 120 mg depending on their body weight.

    Drug: VIT-2763 twice a day (BID)

  • Placebo comparator
    Placebo

    Participants will be assigned to receive Placebo, Twice a day.

    Drug: Placebo

Interventions

  • DrugVIT-2763 once a day (QD)

    Participants will receive VIT-2763 QD at a dose of 60 mg if their body weight is between 40 kg to 59 kg or at a dose of 120 mg if their body weight is between 60 kg and 100 kg, during 12 weeks.

  • DrugVIT-2763 twice a day (BID)

    Participants will receive VIT-2763 BID at a dose of 60 mg if their body weight is between 40 kg to 59 kg or at a dose of 120 mg if their body weight is between 60 kg and 100 kg, during 12 weeks.

  • DrugPlacebo

    Participants will receive hard capsules of Placebo, twice a day.

06

What researchers measure

Primary outcomes

  1. Number of Participants With Treatment-Emergent Adverse Events (TEAEs)

    Please note that in this section we are presenting just the overview of the adverse events experienced by the trial participants, in particular, the number of participants with at least one TEAE. Please refer to the detailed tables included on the Adverse Event Module for specifics.

    Time frame: From baseline to Week 16

  2. Changes in the Systolic Blood Pressure (SBP) and Diastolic Blood Pressure (DBP)

    Summary of the values by visit from baseline and changes from baseline by post-baseline visit.

    Time frame: From baseline to Week 12

  3. Changes in the Heart Rate

    Summary of the values by visit from baseline and changes from baseline by post-baseline visit.

    Time frame: From baseline to Week 12

  4. Changes in 12-lead Electrocardiogram (ECG) Parameters

    Values by visit from baseline and changes from baseline by post-baseline visit. The following ECG parameters were recorded: PR interval, QRS duration, QT interval, RR interval and QTcF interval. PR interval represents the time from the onset of the P wave to the start of the QRS complex. QRS duration represents the time required for a stimulus to spread through the ventricles (ventricular depolarization). QT interval represents the time from the start of the Q wave to the end of the T wave. RR interval represents the time from the onset of one R wave to the onset of the next one, one complete cardiac cycle. QT corrected for heart rate (QTc) interval reflects ventricular repolarization.

    Time frame: From baseline to Week 12

Secondary outcomes

  1. Change From Baseline in Total Serum Iron

    Assessment of total serum Iron from baseline over a 12-week period (absolute and change from baseline). For the serum iron parameter, the 'Baseline' was collected during the screening period within the biochemistry sample.

    Time frame: From baseline to Week 12

  2. Change From Baseline in Serum Ferritin

    Assessment of serum ferritin from baseline over a 12-week period (absolute and change from baseline). For the serum ferritin parameter, the 'Baseline' was collected during the screening period within the biochemistry sample.

    Time frame: From baseline to Week 12

  3. Change From Baseline in Serum Transferrin

    Assessment of serum transferrin from baseline over a 12-week period (absolute and change from baseline). For the serum transferrin parameter, the "Baseline 2h post-dose" was defined as the value at Visit 3 2h post-dose.

    Time frame: From baseline to Week 12

  4. Change From Baseline in Calculated Transferrin Saturation (TSAT) )

    Assessment of TSAT from baseline over a 12-week period (absolute and change from baseline). For the calculated transferrin saturation parameter, the 'Baseline' was collected during the screening period within the biochemistry sample. Transferrin Saturation (TSAT) was calculated as Total Iron /Total Iron Binding Capacity (TIBC) X 100.

    Time frame: From baseline to Week 12

  5. Pharmacokinetics Parameters - VIT-2763 Plasma Concentration Over Time

    Sparse sampling for determination of VIT-2763 plasma concentration following multiple dosing was obtained from pre-dose trough to 3 hours or 4 hours post-dose at selected study visits. Pharmacokinetics parameters (Cmax, clearance, distribution volume, area under the curve (AUC) were not calculated for the study.

    Time frame: Baseline, Week 4, Week 8 and Week 12

07

Results

Posted Dec 14, 2023

Participant flow

Participant flow — Overall Study
MilestoneVIT-2763 QDVIT-2763 BIDPlacebo
Started9124
Completed8114
Not completed110
Withdrew: Adverse event100
Withdrew: Withdrawal by subject010

Outcome measures

PrimaryNumber of Participants With Treatment-Emergent Adverse Events (TEAEs)

Please note that in this section we are presenting just the overview of the adverse events experienced by the trial participants, in particular, the number of participants with at least one TEAE. Please refer to the detailed tables included on the Adverse Event Module for specifics.

Time frame:
From baseline to Week 16
Reported as:
Count of participants · Participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
ParticipantsVIT-2763 QDVIT-2763 BIDPlacebo
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)673
PrimaryChanges in the Systolic Blood Pressure (SBP) and Diastolic Blood Pressure (DBP)

Summary of the values by visit from baseline and changes from baseline by post-baseline visit.

Time frame:
From baseline to Week 12
Reported as:
Mean · millimeters of mercury (mmHg)
Changes in the Systolic Blood Pressure (SBP) and Diastolic Blood Pressure (DBP)
millimeters of mercury (mmHg)VIT-2763 QDVIT-2763 BIDPlacebo
SBP Baseline114.2 ± 11.62114.7 ± 13.88114.8 ± 12.97
SBP Week 1115.0 ± 8.35116.7 ± 10.65104.0 ± 8.98
SBP Change from Baseline to Week 10.8 ± 8.942.0 ± 9.72-10.8 ± 10.90
SBP Week 2113.9 ± 9.41118.3 ± 14.85114.8 ± 15.06
SBP Change from Baseline to Week 2-0.3 ± 6.603.6 ± 8.780.0 ± 2.83
SBP Week 4112.0 ± 11.20113.9 ± 12.41104.8 ± 6.85
SBP Change from Baseline to Week 4-2.2 ± 8.77-0.8 ± 8.51-10.0 ± 14.72
SBP Week 8114.9 ± 7.41113.8 ± 10.54109.8 ± 12.61
SBP Change from Baseline to Week 80.7 ± 11.110.9 ± 9.63-5.0 ± 1.63
SBP Week 12113.4 ± 11.02112.8 ± 11.31107.5 ± 10.08
SBP Change from Baseline to Week 12-0.8 ± 13.02-0.1 ± 10.85-7.3 ± 3.40
DBP Baseline65.94 ± 10.08865.00 ± 10.18966.50 ± 7.853
DBP Week 166.44 ± 6.71069.33 ± 9.92161.75 ± 4.856
DBP Change from Baseline to Week 10.50 ± 4.8864.33 ± 6.344-4.75 ± 6.185
DBP Week 265.89 ± 9.54568.83 ± 11.53564.00 ± 6.055
DBP Change from Baseline to Week 2-0.06 ± 5.3533.83 ± 8.726-2.50 ± 3.000
DBP Week 465.22 ± 11.04362.33 ± 9.06962.75 ± 3.775
DBP Change from Baseline to Week 4-0.72 ± 9.398-2.67 ± 9.921-3.75 ± 5.058
DBP Week 863.44 ± 6.57967.18 ± 9.17466.75 ± 7.500
DBP Change from Baseline to Week 8-2.50 ± 8.2163.27 ± 8.4390.25 ± 7.848
DBP Week 1263.67 ± 7.69767.64 ± 9.65762.50 ± 3.317
DBP Change from Baseline to Week 12-2.28 ± 10.1403.73 ± 6.310-4.00 ± 6.880
PrimaryChanges in the Heart Rate

Summary of the values by visit from baseline and changes from baseline by post-baseline visit.

Time frame:
From baseline to Week 12
Reported as:
Mean · Pulse Rate (bpm)
Changes in the Heart Rate
Pulse Rate (bpm)VIT-2763 QDVIT-2763 BIDPlacebo
Baseline82.00 ± 12.13577.71 ± 10.20873.75 ± 5.560
Week 182.33 ± 11.69480.33 ± 9.87570.75 ± 6.449
Change from Baseline to Week 10.33 ± 6.0422.63 ± 5.859-3.00 ± 5.715
Week 283.56 ± 12.85675.83 ± 9.95375.00 ± 8.756
Change from Baseline to Week 21.56 ± 10.442-1.88 ± 8.2631.25 ± 3.775
Week 482.33 ± 14.41477.92 ± 9.83073.25 ± 3.775
Change from Baseline to Week 40.33 ± 8.8180.21 ± 5.541-0.50 ± 1.915
Week 883.22 ± 11.32280.27 ± 10.99272.50 ± 5.196
Change from Baseline to Week 81.22 ± 6.6482.68 ± 9.040-1.25 ± 7.411
Week 1281.44 ± 9.00279.91 ± 8.73474.25 ± 4.193
Change from Baseline to Week 12-0.56 ± 8.7192.32 ± 9.4190.50 ± 6.137
PrimaryChanges in 12-lead Electrocardiogram (ECG) Parameters

Values by visit from baseline and changes from baseline by post-baseline visit. The following ECG parameters were recorded: PR interval, QRS duration, QT interval, RR interval and QTcF interval. PR interval represents the time from the onset of the P wave to the start of the QRS complex. QRS duration represents the time required for a stimulus to spread through the ventricles (ventricular depolarization). QT interval represents the time from the start of the Q wave to the end of the T wave. RR interval represents the time from the onset of one R wave to the onset of the next one, one complete cardiac cycle. QT corrected for heart rate (QTc) interval reflects ventricular repolarization.

Time frame:
From baseline to Week 12
Reported as:
Mean · milliseconds (ms)
Changes in 12-lead Electrocardiogram (ECG) Parameters
milliseconds (ms)VIT-2763 QDVIT-2763 BIDPlacebo
PR interval - Baseline158.3 ± 25.14147.6 ± 20.05175.8 ± 38.92
PR interval - Baseline 2h post-dose161.2 ± 21.28155.1 ± 17.98175.0 ± 14.07
PR interval - Change from Baseline to Baseline 2h post-dose2.9 ± 25.786.3 ± 16.45-0.8 ± 38.59
PR interval - Week 1159.2 ± 22.90153.0 ± 14.12189.5 ± 18.72
PR interval - Change from Baseline to Week 10.9 ± 16.594.7 ± 12.7113.8 ± 25.67
PR interval - Week 2167.6 ± 26.49153.0 ± 17.87185.5 ± 4.43
PR interval - Change from Baseline to Week 29.2 ± 27.645.5 ± 13.569.8 ± 37.56
PR interval - Week 4154.0 ± 26.49154.2 ± 21.19185.0 ± 7.39
PR interval - Change from Baseline to Week 4-4.3 ± 18.858.1 ± 15.489.3 ± 33.42
PR interval - Week 8161.2 ± 19.34153.5 ± 20.61185.8 ± 10.14
PR interval - Change from Baseline to Week 82.9 ± 17.057.1 ± 12.6610.0 ± 32.86
PR interval - Week 12160.6 ± 19.55156.6 ± 14.47182.8 ± 11.98
PR interval - Change from Baseline to Week 122.2 ± 19.5011.5 ± 14.257.0 ± 36.09
QRS duration - Baseline94.0 ± 18.7388.8 ± 19.17119.3 ± 50.12
QRS duration - Baseline 2h post-dose94.3 ± 20.2195.8 ± 10.9685.0 ± 11.60
QRS duration - Change from Baseline to Baseline 2h post-dose0.3 ± 4.827.0 ± 14.39-34.3 ± 55.27
QRS duration - Week 198.4 ± 23.1696.1 ± 11.2798.0 ± 16.57
QRS duration - Change from Baseline to Week 14.4 ± 14.757.3 ± 13.45-21.3 ± 65.76
QRS duration - Week 294.0 ± 29.0196.3 ± 14.1391.3 ± 12.09
QRS duration - Change from Baseline to Week 20.0 ± 23.047.6 ± 17.96-28.0 ± 61.36
QRS duration - Week 492.3 ± 23.3292.6 ± 15.1194.3 ± 8.10
QRS duration - Change from Baseline to Week 4-1.7 ± 11.166.0 ± 16.76-25.0 ± 54.02
QRS duration - Week 891.0 ± 24.7294.9 ± 18.2793.3 ± 10.81
QRS duration - Change from Baseline to Week 8-3.0 ± 15.997.4 ± 28.52-26.0 ± 60.02
QRS duration - Week 1294.3 ± 27.3184.8 ± 18.6691.8 ± 10.59
QRS duration - Change from Baseline to Week 120.3 ± 20.64-2.7 ± 23.70-27.5 ± 60.69
QT interval - Baseline371.7 ± 76.41374.2 ± 21.78420.8 ± 48.75
QT interval - Baseline 2h post-dose385.7 ± 50.89376.8 ± 22.25379.5 ± 30.17
QT interval - Change from Baseline to Baseline 2h post-dose14.0 ± 61.002.6 ± 21.49-41.3 ± 68.30
QT interval - Week 1394.1 ± 47.36374.8 ± 33.12381.5 ± 32.18
QT interval - Change from Baseline to Week 122.4 ± 57.200.7 ± 28.96-39.3 ± 71.63
QT interval - Week 2384.0 ± 55.52367.0 ± 24.68378.3 ± 27.89
QT interval - Change from Baseline to Week 212.3 ± 76.14-7.2 ± 27.61-42.5 ± 61.87
QT interval - Week 4370.1 ± 67.13373.6 ± 21.22378.0 ± 36.51
QT interval - Change from Baseline to Week 4-1.6 ± 23.070.9 ± 24.94-42.8 ± 61.50
QT interval - Week 8389.7 ± 39.20379.4 ± 25.08381.8 ± 37.99
QT interval - Change from Baseline to Week 818.0 ± 69.604.6 ± 22.59-39.0 ± 64.59
QT interval - Week 12380.9 ± 59.97364.2 ± 36.22388.3 ± 42.85
QT interval - Change from Baseline to Week 129.2 ± 33.53-10.5 ± 33.69-32.5 ± 72.06
RR interval - Baseline789.4 ± 128.89801.4 ± 95.56877.5 ± 114.65
RR interval - Baseline 2h post-dose781.2 ± 155.21805.5 ± 119.36812.5 ± 124.38
RR interval - Change from Baseline to Baseline 2h post-dose-8.2 ± 87.784.1 ± 112.91-65.0 ± 196.09
RR interval - Week 1793.2 ± 105.35864.9 ± 149.33826.5 ± 96.57
RR interval - Change from Baseline to Week 13.8 ± 82.8263.5 ± 137.59-51.0 ± 98.80
RR interval - Week 2757.3 ± 120.25830.1 ± 138.34833.8 ± 16.01
RR interval - Change from Baseline to Week 2-32.1 ± 146.7028.7 ± 117.34-43.8 ± 103.92
RR interval - Week 4783.7 ± 110.55829.8 ± 154.72825.8 ± 96.08
RR interval - Change from Baseline to Week 4-5.8 ± 62.1134.1 ± 137.64-51.8 ± 111.05
RR interval - Week 8765.0 ± 91.52810.1 ± 138.16844.8 ± 135.47
RR interval - Change from Baseline to Week 8-24.4 ± 116.4217.2 ± 108.39-32.8 ± 152.45
RR interval - Week 12780.7 ± 140.88812.9 ± 93.89878.3 ± 133.70
RR interval - Change from Baseline to Week 12-8.8 ± 115.9520.0 ± 73.460.8 ± 172.81
QTcF interval - Baseline403.3 ± 80.48400.5 ± 22.76416.8 ± 17.46
QTcF interval - Baseline 2h post-dose419.3 ± 36.58410.1 ± 25.34407.3 ± 13.05
QTcF interval - Change from Baseline to Baseline 2h post-dose16.0 ± 66.519.6 ± 22.60-9.5 ± 6.76
QTcF interval - Week 1425.7 ± 37.25397.3 ± 30.44406.8 ± 26.02
QTcF interval - Change from Baseline to Week 122.3 ± 65.53-3.3 ± 24.92-10.0 ± 20.38
QTcF interval - Week 2420.8 ± 42.28393.3 ± 28.90402.0 ± 30.30
QTcF interval - Change from Baseline to Week 217.4 ± 77.84-7.2 ± 27.52-14.8 ± 14.52
QTcF interval - Week 4401.3 ± 64.21401.8 ± 27.41402.8 ± 23.63
QTcF interval - Change from Baseline to Week 4-2.0 ± 24.742.1 ± 24.41-14.0 ± 7.07
QTcF interval - Week 8426.0 ± 30.12399.6 ± 27.17404.3 ± 19.86
QTcF interval - Change from Baseline to Week 822.7 ± 80.360.3 ± 26.86-12.5 ± 4.51
QTcF interval - Week 12414.1 ± 53.40391.4 ± 42.91405.3 ± 25.71
QTcF interval - Change from Baseline to Week 1210.8 ± 37.94-8.0 ± 37.96-11.5 ± 10.63
SecondaryChange From Baseline in Total Serum Iron

Assessment of total serum Iron from baseline over a 12-week period (absolute and change from baseline). For the serum iron parameter, the 'Baseline' was collected during the screening period within the biochemistry sample.

Time frame:
From baseline to Week 12
Reported as:
Mean · Micromoles per Litre (umol/L)
Change From Baseline in Total Serum Iron
Micromoles per Litre (umol/L)VIT-2763 QDVIT-2763 BIDPlacebo
Baseline23.81 ± 13.43828.42 ± 9.79630.88 ± 13.329
Week 112.54 ± 9.15911.38 ± 7.69328.20 ± 17.919
Change from Baselines to Week 1-11.27 ± 7.158-17.03 ± 9.611-2.68 ± 12.365
Week 211.71 ± 8.62212.99 ± 8.97832.48 ± 15.541
Change from Baseline to Week 2-9.74 ± 4.707-15.43 ± 12.7841.60 ± 3.996
Week 413.90 ± 12.22212.85 ± 7.58331.08 ± 13.721
Change from Baseline to Week 4-9.33 ± 4.659-14.03 ± 8.4320.20 ± 2.902
Week 811.35 ± 7.82211.55 ± 9.65126.70 ± 12.040
Change from Baseline to Week 8-11.88 ± 9.627-17.05 ± 11.936-0.37 ± 3.550
Week 1213.72 ± 11.03111.62 ± 6.44227.10 ± 12.856
Change from Baseline to Week 12-10.54 ± 6.782-16.22 ± 10.8920.03 ± 2.701
SecondaryChange From Baseline in Serum Ferritin

Assessment of serum ferritin from baseline over a 12-week period (absolute and change from baseline). For the serum ferritin parameter, the 'Baseline' was collected during the screening period within the biochemistry sample.

Time frame:
From baseline to Week 12
Reported as:
Mean · Microgrammes per Litre (ug/L)
Change From Baseline in Serum Ferritin
Microgrammes per Litre (ug/L)VIT-2763 QDVIT-2763 BIDPlacebo
Baseline403.14 ± 226.3151133.23 ± 2119.115440.03 ± 321.453
Week 1416.76 ± 220.567471.53 ± 209.800445.80 ± 228.244
Change from Baseline to Week 1-10.14 ± 31.50940.89 ± 164.4665.77 ± 93.946
Week 2441.34 ± 269.599500.79 ± 230.076411.35 ± 207.477
Change from Baseline to Week 23.29 ± 60.57923.80 ± 149.147-49.80 ± 76.867
Week 4476.60 ± 282.457585.71 ± 311.224389.83 ± 240.553
Change from Baseline to Week 41.05 ± 61.63889.98 ± 249.397-50.20 ± 80.958
Week 8463.67 ± 206.646508.56 ± 211.366340.23 ± 165.356
Change from Baseline to Week 8-11.88 ± 43.06352.84 ± 157.952-9.45 ± 20.577
Week 12465.72 ± 227.378927.42 ± 1478.471261.70 ± 53.033
Change from Baseline to Week 12-15.84 ± 66.208-139.23 ± 626.3807.25 ± 57.205
SecondaryChange From Baseline in Serum Transferrin

Assessment of serum transferrin from baseline over a 12-week period (absolute and change from baseline). For the serum transferrin parameter, the "Baseline 2h post-dose" was defined as the value at Visit 3 2h post-dose.

Time frame:
From baseline to Week 12
Reported as:
Mean · Grammes per Litre (g/L)
Change From Baseline in Serum Transferrin
Grammes per Litre (g/L)VIT-2763 QDVIT-2763 BIDPlacebo
Baseline 2h post-dose1.601 ± 0.43771.673 ± 0.31771.655 ± 0.3003
Week 11.610 ± 0.45571.822 ± 0.39031.683 ± 0.2210
Change from Baseline to Week 10.009 ± 0.11840.149 ± 0.13340.028 ± 0.2419
Week 21.601 ± 0.50431.759 ± 0.35691.655 ± 0.2408
Change from Baseline to Week 2-0.014 ± 0.15160.087 ± 0.14290.000 ± 0.1846
Week 41.708 ± 0.26721.705 ± 0.34961.745 ± 0.3756
Change from Baseline to Week 40.053 ± 0.20140.054 ± 0.15350.090 ± 0.2740
Week 81.640 ± 0.27451.725 ± 0.31941.553 ± 0.2836
Change from Baseline to Week 8-0.015 ± 0.06750.093 ± 0.1618-0.120 ± 0.0854
Week 121.706 ± 0.19541.656 ± 0.29741.610 ± 0.4784
Change from Baseline to Week 12-0.040 ± 0.11160.100 ± 0.1044-0.063 ± 0.1266
SecondaryChange From Baseline in Calculated Transferrin Saturation (TSAT) )

Assessment of TSAT from baseline over a 12-week period (absolute and change from baseline). For the calculated transferrin saturation parameter, the 'Baseline' was collected during the screening period within the biochemistry sample. Transferrin Saturation (TSAT) was calculated as Total Iron /Total Iron Binding Capacity (TIBC) X 100.

Time frame:
From baseline to Week 12
Reported as:
Mean · TSAT Percentage (%)
Change From Baseline in Calculated Transferrin Saturation (TSAT) )
TSAT Percentage (%)VIT-2763 QDVIT-2763 BIDPlacebo
Baseline69.3 ± 31.1679.0 ± 24.0583.3 ± 33.50
Week 136.8 ± 24.4432.2 ± 21.4456.5 ± 30.56
Change from Baselone to Week 1-32.6 ± 19.55-46.8 ± 22.55-26.8 ± 28.77
Week 236.0 ± 22.8135.8 ± 24.1371.5 ± 33.91
Change from Baseline to Week 2-29.5 ± 14.57-43.2 ± 33.23-11.8 ± 23.50
Week 441.0 ± 32.2738.4 ± 24.8582.5 ± 35.00
Change from Baseline to Week 4-24.0 ± 18.21-38.7 ± 27.57-0.8 ± 1.50
Week 832.3 ± 17.4833.5 ± 27.0877.3 ± 39.26
Change from Baseline to Week 8-32.7 ± 25.80-47.9 ± 29.70-0.3 ± 0.58
Week 1236.0 ± 25.9334.8 ± 17.7676.3 ± 40.99
Change from Baseline to Week 12-27.6 ± 20.37-46.8 ± 23.61-1.3 ± 2.31
SecondaryPharmacokinetics Parameters - VIT-2763 Plasma Concentration Over Time

Sparse sampling for determination of VIT-2763 plasma concentration following multiple dosing was obtained from pre-dose trough to 3 hours or 4 hours post-dose at selected study visits. Pharmacokinetics parameters (Cmax, clearance, distribution volume, area under the curve (AUC) were not calculated for the study.

Time frame:
Baseline, Week 4, Week 8 and Week 12
Reported as:
Mean · nanograms per milliliter (ng/mL)
Pharmacokinetics Parameters - VIT-2763 Plasma Concentration Over Time
nanograms per milliliter (ng/mL)VIT-2763 QDVIT-2763 BID
Visit 3 1 hour post-dose796.5 ± 508.2609.6 ± 455.2
Visit 3 4 hours post-dose222.3 ± 73.1411.0 ± 269.5
Visit 6 pre-dose17.2 ± 10.2114.4 ± 78.2
Visit 6 1 hour post-dose986.2 ± 796.7483.5 ± 247.3
Visit 6 3 hours post-dose578.2 ± 494.5544.3 ± 337.5
Visit 7 pre-dose15.5 ± 6.6111.0 ± 61.9
Visit 7 1 hour post-dose751.7 ± 682.3692.2 ± 470.2
Visit 7 4 hours post-dose295.5 ± 209.5384.3 ± 200.9
Visit 8 pre-dose361.5 ± 579.2136.0 ± 76.8
Visit 8 1 hour post-dose894.1 ± 670.3650.6 ± 474.5
Visit 8 3 hours post-dose684.0 ± 405.5569.9 ± 343.1

Adverse events

Collected over up to 20 weeks (all visits/phone calls through study completion are considered). Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
VIT-2763 QD0/9 (0%)0/9 (0%)6/9 (66.7%)
VIT-2763 BID0/12 (0%)0/12 (0%)7/12 (58.3%)
Placebo0/4 (0%)0/4 (0%)3/4 (75%)
Most frequent other events
Showing 10 of 32
Most frequent other events
EventVIT-2763 QDVIT-2763 BIDPlacebo
HypoacusisEar and labyrinth disorders0/90/121/4
Non-cardiac chest painGeneral disorders0/90/121/4
HeadacheNervous system disorders0/91/121/4
Respiratory symptomRespiratory, thoracic and mediastinal disorders0/90/121/4
PruritusSkin and subcutaneous tissue disorders0/90/121/4
FatigueGeneral disorders2/92/120/4
DyspepsiaGastrointestinal disorders0/92/120/4
PyrexiaGeneral disorders0/92/120/4
AnaemiaBlood and lymphatic system disorders1/91/120/4
HaemolysisBlood and lymphatic system disorders1/90/120/4

Baseline characteristics

Age, Categorical
Age, Categorical(Participants)VIT-2763 QDVIT-2763 BIDPlaceboTotal
<=18 years0000
Between 18 and 65 years912425
>=65 years0000
Sex: Female, Male
Sex: Female, Male(Participants)VIT-2763 QDVIT-2763 BIDPlaceboTotal
Female6219
Male310316
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)VIT-2763 QDVIT-2763 BIDPlaceboTotal
Hispanic or Latino0000
Not Hispanic or Latino911424
Unknown or Not Reported0101
Race (NIH/OMB)
Race (NIH/OMB)(Participants)VIT-2763 QDVIT-2763 BIDPlaceboTotal
American Indian or Alaska Native0000
Asian5319
Native Hawaiian or Other Pacific Islander0000
Black or African American0000
White49316
More than one race0000
Unknown or Not Reported0000
Region of Enrollment
Region of Enrollment(participants)VIT-2763 QDVIT-2763 BIDPlaceboTotal
Greece3317
Lebanon1203
Italy0303
Israel0123
Thailand5319
08

Study locations

16 sites
  • Clinical Site #301
    Athens, Attiki 115 27, Greece
  • Clinical Site #302
    Rio, 265 04, Greece
  • Clinical Site #303
    Thessaloniki, 54642, Greece
  • Clinical Site #401
    Afula, 18411, Israel
  • Clinical Site #402
    Haifa, 34362, Israel
  • Clinical Site #403
    Petah Tikva, 49100, Israel
  • Clinical Site #201
    Milan, MI 20122, Italy
  • Clinical Site #203
    Napoli, 80131, Italy
  • Clinical Site #206
    Napoli, 80138, Italy
  • Clinical Site #207
    Orbassano, 10043, Italy
  • Clinical Site #205
    Palermo, 90146, Italy
  • Clinical Site #202
    Verona, 37134, Italy
  • Clinical Site #101
    Beirut, 11-0236b, Lebanon
  • Clinical Site #501
    Bangkok, 10700, Thailand
  • Clinical Site #502
    Chiang Mai, 50200, Thailand
  • Clinical Site #503
    Phitsanulok, 65000, Thailand
09

References and documents

Study documents

PDFs hosted by the registry, ClinicalTrials.gov.

Individual participant data

Plan to share: No

10

Timeline

Tracking since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reachedSimilar studies: half by the dot; the band is the middle halfHalf of similar studies reached it by the dot; the band is the middle half, from a quarter to three quarters

Ran from Jun 2020 to primary completion in Oct 2021, 16 months.

Results were first posted in Dec 2023, 2 years 2 months after primary completion.

Of 29 phase 2 interventional studies under beta-Thalassemia, half reached primary completion within 32 months of starting (the median), counting those still running; a quarter within 21 and three quarters within 57. This study's 16 months is below that.

How these figures are worked out, and which studies are left out of them.

Registered
Apr 1, 2020
First posted
Apr 28, 2020
StartActual
Jun 11, 2020
Primary completionActual
Oct 11, 2021
CompletionActual
Nov 3, 2021
Results first posted
Dec 14, 2023
Last update
Dec 14, 2023
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on Dec 14, 2023, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT04364269
Lead sponsor
Vifor (International) Inc.
Collaborators
Labcorp Corporation of America Holdings, Inc
Responsible party
Sponsor
First posted
Apr 28, 2020
Start date
Jun 11, 2020
Primary completion
Oct 11, 2021
Completion
Nov 3, 2021
Results posted
Dec 14, 2023
Last update
Dec 14, 2023

Study contacts

Clinical Research Department
study director · Vifor (International) Inc.

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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This study is completed, as verified in Dec 2023. You cannot join it, but the record below documents what was studied.

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Studies of the drug tested here: VIT-2763

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