Beta-ThalassemiaPhase 2VITHALNCT04364269

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

A Phase 2 study testing VIT-2763 once a day (QD) and VIT-2763 twice a day (BID) in people with Beta-Thalassemia and Non-transfusion-dependent Thalassemia, run by Vifor (International) Inc. at 16 sites in 5 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial 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 more about this study

Who is it for?: Ages 12 to 65 years
Conditions the study is for: Beta-Thalassemia, Non-transfusion-dependent Thalassemia
What is being tested?: VIT-2763 once a day (QD) and VIT-2763 twice a day (BID)
Type of treatment: Pill or oral medicine
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 35 participants
Where: 16 sitesin 5 countries
Study dates: 11 Jun 2020to 3 Nov 2021
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 12 to 65 years
All sexes
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

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 hemoglobin (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.

Who cannot join

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 Hb 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.

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Chronic liver disease and/or alanine transaminase (ALT), aspartate transaminase (AST) or gamma-glutamyl transpeptidase (GGT) above 3-fold the upper limit of normal (ULN) range at screening.
Estimated glomerular filtration rate (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 clinically important finding of cardiac disorders, such as clinically relevant cardiac arrhythmia, cardiomyopathy, coronary disease, valve disorder, or heart failure according to New York Heart Association classification 3-4.
Subjects with history of partial or total splenectomy within 6 months prior to screening.

Trial locations and contacts

16 sites in 5 countries.

Country

City, postcode or hospital

16 sites in 5 countries

Italy6 sites

Clinical Site #201
Milan, MI, 20122
Clinical Site #203
Naples, 80131
Clinical Site #206
Naples, 80138
Clinical Site #207
Orbassano, 10043
Clinical Site #205
Palermo, 90146
Clinical Site #202
Verona, 37134

Other trials in Italy

Greece3 sites

Clinical Site #301
Athens, Attica, 115 27
Clinical Site #302
Rio, 265 04
Clinical Site #303
Thessaloniki, 54642

Other trials in Greece

Israel3 sites

Clinical Site #401
Afula, 18411
Clinical Site #402
Haifa, 34362
Clinical Site #403
Petah Tikva, 49100

Other trials in Israel

Thailand3 sites

Clinical Site #501
Bangkok, 10700
Clinical Site #502
Chiang Mai, 50200
Clinical Site #503
Phitsanulok, 65000

Other trials in Thailand

Lebanon1 site

Clinical Site #101
Beirut, 11-0236b

Other trials in Lebanon

About this study

Official title: A Phase 2a, Double-blind, Randomised, Placebo-controlled, Parallel Group, Multicentre Study on Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of Multiple Doses of VIT-2763 in Subjects With Non-transfusion Dependent β-thalassaemia

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.

Conditions: Beta-Thalassemia, Non-transfusion-dependent Thalassemia
Being tested: VIT-2763 once a day (QD), VIT-2763 twice a day (BID), Placebo
Keywords: Beta-Thalassemia
Medical subject headings: beta-Thalassemia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

VIT-2763 once a day (QD)Drug
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.
VIT-2763 twice a day (BID)Drug
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.
PlaceboDrug
Participants will receive hard capsules of Placebo, twice a day.

Study groups in NCT04364269
Study group	Role	What they receive
VIT-2763 Once a day (QD)	Experimental	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. Receives: Drug: VIT-2763 once a day (QD)
VIT-2763 Twice a day (BID)	Experimental	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. Receives: Drug: VIT-2763 twice a day (BID)
Placebo	Placebo comparator	Participants will be assigned to receive Placebo, Twice a day. Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

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.
Measured: From baseline to Week 16
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.
Measured: From baseline to Week 12
Changes in the Heart Rate
Summary of the values by visit from baseline and changes from baseline by post-baseline visit.
Measured: From baseline to Week 12
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.
Measured: From baseline to Week 12

Secondary outcomes (5)

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.
Measured: From baseline to Week 12
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.
Measured: From baseline to Week 12
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.
Measured: From baseline to Week 12
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.
Measured: From baseline to Week 12
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.
Measured: Baseline, Week 4, Week 8 and Week 12

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

11 Jun 20203 Nov 2021

First posted28 Apr 2020
Study start11 Jun 2020
Primary completion11 Oct 2021
Study completion3 Nov 2021

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (1)Papers the sponsor has linked to this study.
Other trials for Beta-ThalassemiaStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Model description: Multiple dose, multicenter, double-blind, placebo-controlled parallel group study in adult and adolescent male and female subjects with non-transfusion dependent thalassemia (NTDT)
Masking: Quadruple blind
Who is masked: Participant, Care provider, Investigator, Outcomes assessor
Enrolment: 35 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT04364269
Sponsor's protocol ID: VIT-2763-THAL-201
Other IDs: 2019-002221-29, 2019
Acronym: VITHAL
Status last verified: Dec 2023
First submitted: 1 Apr 2020
Last updated: 14 Dec 2023
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
FDA-regulated drug: No
FDA-regulated device: No
Individual data sharing: No

Publications

Kattamis A, Taher A, Viprakasit V, Levin C, Hermosilla R, Szecsody P, Richard F, Cappellini MD, Porter J. Safety and pharmacodynamics of the ferroportin inhibitor vamifeport in patients with non-transfusion-dependent beta-thalassemia: results from a randomized phase 2a study. Orphanet J Rare Dis. 2025 Nov 25;20(1):608. doi: 10.1186/s13023-025-04119-y.
PubMed 41291806

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

Active, not recruiting
Evaluation the Safety and Efficacy of KL003 Cell Injection in the Treatment of Transfusion-dependent β-thalassemia.
Transfusion-dependent Beta-Thalassemia
RecruitingObservational study
Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy
Transfusion-dependent Beta-Thalassemia, Gene Therapy
Not yet recruiting
Gene Therapy of Beta Thalassemia Using a Self-inactivating Lentiviral Vector
Beta-Thalassemia
RecruitingPhase 1/2
β-Thalassemia Treatment With KL003 Cell Injection
Transfusion-dependent Beta-Thalassemia
Active, not recruitingPhase 3
A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)
Non-Transfusion-dependent Alpha-Thalassemia, Non-Transfusion-dependent Beta-Thalassemia

All Beta-Thalassemia trials

Study at a glance

From the registry record, last updated by the sponsor on 14 Dec 2023. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase 2a, Double-blind, Randomised, Placebo-controlled, Parallel Group, Multicentre Study on Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of Multiple Doses of VIT-2763 in Subjects With Non-transfusion Dependent β-thalassaemia
ClinicalTrials.gov ID: NCT04364269
Acronym / sponsor ID: VITHAL / VIT-2763-THAL-201
Conditions: Beta-Thalassemia, Non-transfusion-dependent Thalassemia
Keywords: Beta-Thalassemia
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 35 participants (actual)
Lead sponsor: Vifor (International) Inc.Industry (pharma, biotech, devices)
Collaborators: Labcorp Corporation of America Holdings, Inc
Study start: 11 Jun 2020 (actual)
Primary completion: 11 Oct 2021 (actual)
Study completion: 3 Nov 2021 (actual)
First posted: 28 Apr 2020 (actual)
Results: Posted 14 Dec 2023

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

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.

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

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind (participant, care provider, investigator, outcomes assessor)
Number of arms: 3
Model description: Multiple dose, multicenter, double-blind, placebo-controlled parallel group study in adult and adolescent male and female subjects with non-transfusion dependent thalassemia (NTDT)

Arm	Type	Receives
VIT-2763 Once a day (QD)	Experimental	Drug: 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.
VIT-2763 Twice a day (BID)	Experimental	Drug: 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.
Placebo	Placebo comparator	Drug: PlaceboParticipants will be assigned to receive Placebo, Twice a day.

Who may qualify

Ages: 12 to 65 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (5 listed)

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 hemoglobin (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.

Key exclusion criteria (11 listed)

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 Hb 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.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
VIT-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.	Drug	VIT-2763 Once a day (QD)
VIT-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.	Drug	VIT-2763 Twice a day (BID)
PlaceboParticipants will receive hard capsules of Placebo, twice a day.	Drug	Placebo

Primary outcome

Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Time frame: From baseline to Week 16
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.
Changes in the Systolic Blood Pressure (SBP) and Diastolic Blood Pressure (DBP)
Time frame: From baseline to Week 12
Summary of the values by visit from baseline and changes from baseline by post-baseline visit.
Changes in the Heart Rate
Time frame: From baseline to Week 12
Summary of the values by visit from baseline and changes from baseline by post-baseline visit.
Changes in 12-lead Electrocardiogram (ECG) Parameters
Time frame: From baseline to Week 12
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.

5 secondary outcomes

Change From Baseline in Total Serum Iron
Time frame: From baseline to Week 12
Change From Baseline in Serum Ferritin
Time frame: From baseline to Week 12
Change From Baseline in Serum Transferrin
Time frame: From baseline to Week 12
Change From Baseline in Calculated Transferrin Saturation (TSAT) )
Time frame: From baseline to Week 12
Pharmacokinetics Parameters - VIT-2763 Plasma Concentration Over Time
Time frame: Baseline, Week 4, Week 8 and Week 12

Results and safety

Results posted to the registry on 14 Dec 2023, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
VIT-2763 QD	9	8
VIT-2763 BID	12	11
Placebo	4	4

Primary outcome: Number of Participants With Treatment-Emergent Adverse Events (TEAEs)

Time frame: From baseline to Week 16. Count of participants. Unit: Participants

Measure	VIT-2763 QD n = 9	VIT-2763 BID n = 12	Placebo n = 4
Value	6	7	3

Population analysed: The safety set (SS) was defined as all randomized participants who had taken at least 1 dose of study medication. The participants in the SS were to be analyzed based on the treatment they received, regardless of randomization.

Primary outcome: Changes in the Systolic Blood Pressure (SBP) and Diastolic Blood Pressure (DBP)

Time frame: From baseline to Week 12. Mean (Standard Deviation). Unit: millimeters of mercury (mmHg)

Measure	VIT-2763 QD n = 9	VIT-2763 BID n = 12	Placebo n = 4
SBP Baseline	114.2 (11.62)	114.7 (13.88)	114.8 (12.97)
SBP Week 1	115.0 (8.35)	116.7 (10.65)	104.0 (8.98)
SBP Change from Baseline to Week 1	0.8 (8.94)	2.0 (9.72)	-10.8 (10.90)
SBP Week 2	113.9 (9.41)	118.3 (14.85)	114.8 (15.06)
SBP Change from Baseline to Week 2	-0.3 (6.60)	3.6 (8.78)	0.0 (2.83)
SBP Week 4	112.0 (11.20)	113.9 (12.41)	104.8 (6.85)
SBP Change from Baseline to Week 4	-2.2 (8.77)	-0.8 (8.51)	-10.0 (14.72)
SBP Week 8	114.9 (7.41)	113.8 (10.54)	109.8 (12.61)

Primary outcome: Changes in the Heart Rate

Time frame: From baseline to Week 12. Mean (Standard Deviation). Unit: Pulse Rate (bpm)

Measure	VIT-2763 QD n = 9	VIT-2763 BID n = 12	Placebo n = 4
Baseline	82.00 (12.135)	77.71 (10.208)	73.75 (5.560)
Week 1	82.33 (11.694)	80.33 (9.875)	70.75 (6.449)
Change from Baseline to Week 1	0.33 (6.042)	2.63 (5.859)	-3.00 (5.715)
Week 2	83.56 (12.856)	75.83 (9.953)	75.00 (8.756)
Change from Baseline to Week 2	1.56 (10.442)	-1.88 (8.263)	1.25 (3.775)
Week 4	82.33 (14.414)	77.92 (9.830)	73.25 (3.775)
Change from Baseline to Week 4	0.33 (8.818)	0.21 (5.541)	-0.50 (1.915)
Week 8	83.22 (11.322)	80.27 (10.992)	72.50 (5.196)

Primary outcome: Changes in 12-lead Electrocardiogram (ECG) Parameters

Time frame: From baseline to Week 12. Mean (Standard Deviation). Unit: milliseconds (ms)

Measure	VIT-2763 QD n = 9	VIT-2763 BID n = 12	Placebo n = 4
PR interval - Baseline	158.3 (25.14)	147.6 (20.05)	175.8 (38.92)
PR interval - Baseline 2h post-dose	161.2 (21.28)	155.1 (17.98)	175.0 (14.07)
PR interval - Change from Baseline to Baseline 2h post-dose	2.9 (25.78)	6.3 (16.45)	-0.8 (38.59)
PR interval - Week 1	159.2 (22.90)	153.0 (14.12)	189.5 (18.72)
PR interval - Change from Baseline to Week 1	0.9 (16.59)	4.7 (12.71)	13.8 (25.67)
PR interval - Week 2	167.6 (26.49)	153.0 (17.87)	185.5 (4.43)
PR interval - Change from Baseline to Week 2	9.2 (27.64)	5.5 (13.56)	9.8 (37.56)
PR interval - Week 4	154.0 (26.49)	154.2 (21.19)	185.0 (7.39)

5 secondary outcomes are reported in the full results.

Adverse events

Time frame: up to 20 weeks (all visits/phone calls through study completion are considered). Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
VIT-2763 QD	0 of 9 (0%)	6 of 9 (66.7%)	0 of 9
VIT-2763 BID	0 of 12 (0%)	7 of 12 (58.3%)	0 of 12
Placebo	0 of 4 (0%)	3 of 4 (75%)	0 of 4

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 14 Dec 2023.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 41291806Kattamis A, Taher A, Viprakasit V, Levin C, Hermosilla R, Szecsody P, Richard F, Cappellini MD, Porter J. Safety and pharmacodynamics of the ferroportin inhibitor vamifeport in patients with non-transfusion-dependent beta-thalassemia: results from a randomized phase 2a study. Orphanet J Rare Dis. 2025 Nov 25;20(1):608. doi: 10.1186/s13023-025-04119-y.
Individual participant dataSharing planned: No

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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Clinical Research Department study director

Vifor (International) Inc.

16 sites in 5 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 5 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 14 Dec 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 16 sites in Italy, Greece, Israel, Thailand, Lebanon. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 14 Dec 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of Participants With Treatment-Emergent Adverse Events (TEAEs) (time frame: From baseline to Week 16). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04364269, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Dec 2023.