CompletedNCT04817670ViSionSerenityUpdated Feb 18, 2025Results posted

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

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. · Phase 2, Interventional, and Treatment

Phase: Phase 2
Study type: Interventional
Enrollment: 25
Allocation: Randomized
Ages: 18 Years to 60 Years
Sex: All

Study 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.

Read the 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.

Conditions studied

Sickle Cell Disease

Browse trials for

Anemia, Sickle Cell 1,102

Keywords

anemia
sickle cell disease
vamifeport

Who can participate

Ages eligible: 18 Years to 60 Years
Sexes eligible: All
Accepts healthy volunteers: No

Inclusion criteria

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

Exclusion criteria

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 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.
Uncontrolled hemorrhages

Study design

Phase: Phase 2
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel assignment
Masking: Double (Participant, Investigator)
Enrollment: 25 participants (actual)

Study arms

Experimental
Cohort 1
Participants receive VIT-2763 60 mg, twice a day during 8 weeks.
Drug: VIT-2763 120 mg
Experimental
Cohort 2
Participants receive VIT-2763 120 mg, twice a day during 8 weeks.
Drug: VIT-2763 240 mg
Experimental
Cohort 3
Participants receive VIT-2763 120 mg, three times a day during 8 weeks.
Drug: VIT-2763 360 mg
Placebo comparator
Cohort 4a
Participants receive a placebo, twice a day during 8 weeks.
Drug: Placebo BID
Placebo comparator
Cohort 4b
Participants receive a placebo, three times a day during 8 weeks.
Drug: Placebo TID

Interventions

DrugVIT-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.
Also known as: Vamifeport
DrugVIT-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.
Also known as: Vamifeport
DrugVIT-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.
Also known as: Vamifeport
DrugPlacebo BID
Participants receive 2 capsules of placebo in the morning and in the evening, for 8 weeks. Capsules are to be taken orally.
DrugPlacebo 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.

What researchers measure

Primary outcomes

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

Secondary outcomes

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

Results

Posted Feb 18, 2025

Participant flow

There were 22 sites initiated for this study in 5 countries (The United Kingdom, Lebanon, Greece, the United States, and France).

Participant flow — Overall Study
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

Outcome measures

PrimaryMean 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
Reported as:: Mean · micromoles per liter (umol/L)

Mean Change From Baseline in Haemolysis Marker (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

SecondaryMean 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
Reported as:: Mean · umol/L

Mean Change From Baseline in Haemolysis Marker (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

SecondaryMean 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
Reported as:: Mean · units per liter (U/L)

Mean Change From Baseline in Haemolysis Marker (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

SecondaryMean 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
Reported as:: Mean · millimoles per liter (mmol/L)

Mean Change From Baseline in Haemolysis Marker (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

SecondaryMean 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
Reported as:: Mean · grams per liter (g/L)

Mean Change From Baseline in Haemolysis Marker (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

SecondaryNumber 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
Reported as:: Count of participants · Participants

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

Adverse events

Collected over From the first dose of study drug up to 12 weeks. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
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%)

Most frequent serious events

Most frequent serious events
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

Most frequent other events

Showing 10 of 31

Most frequent other events
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

Baseline characteristics

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
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
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)
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)
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

Study locations

22 sites

Investigator Site 709
Birmingham, Alabama 35233-2110, United States
Investigator Site 708
Los Angeles, California 90027, United States
Investigator Site 713
Aurora, Colorado 80045, United States
Investigator Site 706
Hollywood, Florida 33023, United States
Investigator Site 703
Chicago, Illinois 60612, United States
Investigator Site 701
Greenville, North Carolina 27834, United States
Investigator Site 711
Charleston, South Carolina 29425, United States
Investigator Site 702
Milwaukee, Wisconsin 53226, United States
Investigator Site 801
Colombes, 92700, France
Investigator Site 802
Lyon, 690003, France
Investigator Site 305
Athens, 11527, Greece
Investigator site 301
Athens, GR-11527, Greece
Investigator Site 302
Patra, Greece
Investigator Site 101
Baabda, Lebanon
Investigator Site 102
Beirut, Lebanon
Investigator Site 103
Tripoli, Lebanon
Investigator Site 606
Liverpool, L9 7AL, United Kingdom
Investigator Site 603
London, SE59RS, United Kingdom
Investigator Site 608
London, W12 0HS, United Kingdom
Investigator Site 601
London, United Kingdom
Investigator Site 605
London, United Kingdom
Investigator Site 607
Manchester, M13 9WL, United Kingdom

References and documents

Publications

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. PubMed 35714304 ↗

Study documents

Study protocol · Mar 16, 2023
Statistical analysis plan · Apr 26, 2024

Documents are hosted by the registry — open the source record to download them.

Individual participant data

Plan to share: No

Registry details

Key details

Study ID: NCT04817670
Lead sponsor: Vifor (International) Inc.
Collaborators: Fortrea
Responsible party: Sponsor
First posted: Mar 26, 2021
Start date: Nov 18, 2021
Primary completion: Mar 7, 2024
Completion: Mar 7, 2024
Results posted: Feb 18, 2025
Last update: Feb 18, 2025

Study contacts

Ricardo Hermosilla, PhD

study director · Vifor (International) Inc.

Oversight

Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No

View the source record on ClinicalTrials.gov ↗

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