Sickle Cell DiseasePhase 2ViSionSerenityNCT04817670
Study to Assess Efficacy and Safety of VIT-2763 (Vamifeport) in Subjects With Sickle Cell Disease
A Phase 2 study testing VIT-2763 120 mg, VIT-2763 360 mg and VIT-2763 240 mg in people with Sickle Cell Disease, run by Vifor (International) Inc. at 22 sites in 5 countries.
Trial 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 more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- VIT-2763 120 mg, VIT-2763 360 mg and VIT-2763 240 mg
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 25 participants
- Where
- 22 sitesin 5 countries
- Study dates
- 18 Nov 2021to 7 Mar 2024
- 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 18 to 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- 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
Who cannot join
- 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.
Show all 16Show fewer
- 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
Trial locations and contacts
22 sites in 5 countries.
22 sites in 5 countries
United States8 sites
Investigator Site 713
Aurora, Colorado, 80045
Investigator Site 709
Birmingham, Alabama, 35233-2110
Investigator Site 711
Charleston, South Carolina, 29425
Investigator Site 703
Chicago, Illinois, 60612
Investigator Site 701
Greenville, North Carolina, 27834
Investigator Site 706
Hollywood, Florida, 33023
Investigator Site 708
Los Angeles, California, 90027
Investigator Site 702
Milwaukee, Wisconsin, 53226
United Kingdom6 sites
Investigator Site 606
Liverpool, L9 7AL
Investigator Site 603
London, SE59RS
Investigator Site 608
London, W12 0HS
Investigator Site 601
London
Investigator Site 605
London
Investigator Site 607
Manchester, M13 9WL
Greece3 sites
Investigator Site 305
Athens, 11527
Investigator site 301
Athens, GR-11527
Investigator Site 302
Pátrai
Lebanon3 sites
Investigator Site 101
Baabda
Investigator Site 102
Beirut
Investigator Site 103
Tripoli
France2 sites
Investigator Site 801
Colombes, 92700
Investigator Site 802
Lyon, 690003
About this study
Official title: A Phase 2a, Double-blind, Randomised, Placebo-controlled, Efficacy, and Safety Study of Multiple Doses of VIT-2763 in Subjects With Sickle Cell Disease (ViSionSerenity)
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
- Sickle Cell Disease
- Being tested
- VIT-2763 120 mg, VIT-2763 360 mg, VIT-2763 240 mg, Placebo BID, Placebo TID
- Keywords
- anemia, sickle cell disease, vamifeport
- Medical subject headings
- Anemia, Sickle Cell, Anemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VIT-2763 120 mgDrug
Also called Vamifeport
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.
VIT-2763 360 mgDrug
Also called 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.
VIT-2763 240 mgDrug
Also called 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.
Placebo BIDDrug
Participants receive 2 capsules of placebo in the morning and in the evening, for 8 weeks.
Capsules are to be taken orally.
Placebo TIDDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Experimental | Participants receive VIT-2763 60 mg, twice a day during 8 weeks. Receives: Drug: VIT-2763 120 mg |
| Cohort 2 | Experimental | Participants receive VIT-2763 120 mg, twice a day during 8 weeks. Receives: Drug: VIT-2763 240 mg |
| Cohort 3 | Experimental | Participants receive VIT-2763 120 mg, three times a day during 8 weeks. Receives: Drug: VIT-2763 360 mg |
| Cohort 4a | Placebo comparator | Participants receive a placebo, twice a day during 8 weeks. Receives: Drug: Placebo BID |
| Cohort 4b | Placebo comparator | Participants receive a placebo, three times a day during 8 weeks. Receives: Drug: Placebo TID |
What is being measured
The primary outcome is the main question the study is designed to answer.
Mean Change From Baseline in Haemolysis Marker (Indirect Bilirubin)
Mean change from baseline in haemolysis markers was measured by reduction of indirect bilirubin.
Measured: Baseline and after 8 weeks of treatment
Secondary outcomes (5)
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: From first dose of study drug up to 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Mar 2021
- Study start18 Nov 2021
- Primary completion7 Mar 2024
- Study completion7 Mar 2024
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 (2)Papers the sponsor has linked to this study.
- Other trials for Sickle Cell DiseaseStudies 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
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 25 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vifor (International) Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Fortrea
- Responsible party
- Sponsor
- Investigators
- Ricardo Hermosilla, PhD, Study director, Vifor (International) Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04817670
- Sponsor's protocol ID
- VIT-2763-SCD-202
- Other IDs
- 2020-005072-34
- Acronym
- ViSionSerenity
- Status last verified
- Jan 2025
- First submitted
- 23 Mar 2021
- Last updated
- 18 Feb 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
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 35714304Parrow NL, Doherty JM, Conrey A, Thein SL, Fleming RE. Relationships Between Markers of Iron Status and Hematological Parameters in Patients With Sickle Cell Disease. Adv Hematol. 2024 Dec 3;2024:9872440. doi: 10.1155/ah/9872440. eCollection 2024.
PubMed 39659429
Study at a glance
From the registry record, last updated by the sponsor on 18 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2a, Double-blind, Randomised, Placebo-controlled, Efficacy, and Safety Study of Multiple Doses of VIT-2763 in Subjects With Sickle Cell Disease (ViSionSerenity)
- ClinicalTrials.gov ID
- NCT04817670
- Acronym / sponsor ID
- ViSionSerenity / VIT-2763-SCD-202
- Conditions
- Sickle Cell Disease
- Keywords
- anemia, sickle cell disease, vamifeport
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 25 participants (actual)
- Lead sponsor
- Vifor (International) Inc.Industry (pharma, biotech, devices)
- Collaborators
- Fortrea
- Study start
- 18 Nov 2021 (actual)
- Primary completion
- 7 Mar 2024 (actual)
- Study completion
- 7 Mar 2024 (actual)
- First posted
- 26 Mar 2021 (actual)
- Results
- Posted 18 Feb 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
At randomization/baseline, participants are randomized into 3 VIT-2763 dose groups to receive either 60 mg twice daily (BID) (Cohort 1), or 120 mg BID (Cohort 2), or 120 mg 3 times daily (TID) (Cohort 3) and 2 placebo groups (BID, Cohort 4a or TID, Cohort 4b).
The expected duration of patient participation is a maximum of 16 weeks, including a non-treatment screening period of up to 4 weeks, and 8-week treatment period, and a 4-week safety follow-up period.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | Experimental | Drug: VIT-2763 120 mgParticipants receive VIT-2763 60 mg, twice a day during 8 weeks. |
| Cohort 2 | Experimental | Drug: VIT-2763 240 mgParticipants receive VIT-2763 120 mg, twice a day during 8 weeks. |
| Cohort 3 | Experimental | Drug: VIT-2763 360 mgParticipants receive VIT-2763 120 mg, three times a day during 8 weeks. |
| Cohort 4a | Placebo comparator | Drug: Placebo BIDParticipants receive a placebo, twice a day during 8 weeks. |
| Cohort 4b | Placebo comparator | Drug: Placebo TIDParticipants receive a placebo, three times a day during 8 weeks. |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (16 listed)
- 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
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 120 mgAlso: VamifeportParticipants receive 2 capsules of VIT-2763 30 mg in the morning and in the evening, for 8 weeks. Capsules are to be taken orally. | Drug | Cohort 1 |
| VIT-2763 360 mgAlso: VamifeportParticipants 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. | Drug | Cohort 3 |
| VIT-2763 240 mgAlso: VamifeportParticipants receive 2 capsules of VIT-2763 60 mg in the morning and in the evening, for 8 weeks. Capsules are to be taken orally. | Drug | Cohort 2 |
| Placebo BIDParticipants receive 2 capsules of placebo in the morning and in the evening, for 8 weeks. Capsules are to be taken orally. | Drug | Cohort 4a |
| Placebo TIDParticipants receive 2 capsules of Placebo in the morning, in the afternoon and in the evening, for 8 weeks. Capsules are to be taken orally. | Drug | Cohort 4b |
Primary outcome
Mean Change From Baseline in Haemolysis Marker (Indirect Bilirubin)
Time frame: Baseline and after 8 weeks of treatment
Mean change from baseline in haemolysis markers was measured by reduction of indirect bilirubin.
5 secondary outcomes
Mean Change From Baseline in Haemolysis Marker (Direct and Total Bilirubin)
Time frame: Baseline and after 8 weeks of treatment
Mean Change From Baseline in Haemolysis Marker (Lactate Dehydrogenase)
Time frame: Baseline and after 8 weeks of treatment
Mean Change From Baseline in Haemolysis Marker (Potassium)
Time frame: Baseline and after 8 weeks of treatment
Mean Change From Baseline in Haemolysis Marker (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
Time frame: From first dose of study drug up to 12 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Feb 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 35714304Nyffenegger 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.
- Derived from this study: PubMed 39659429Parrow NL, Doherty JM, Conrey A, Thein SL, Fleming RE. Relationships Between Markers of Iron Status and Hematological Parameters in Patients With Sickle Cell Disease. Adv Hematol. 2024 Dec 3;2024:9872440. doi: 10.1155/ah/9872440. eCollection 2024.
- Related InfoLink provided by the sponsor.
- Individual participant dataSharing planned: No
Related trials for Sickle Cell Disease
Active, not recruitingPhase 1/2
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Long-term Mitapivat Dosing in Subjects With Stable Sickle Cell Disease: An Extension of a Phase I Pilot Study of Mitapivat
Sickle Cell Disease, Hemolytic Anemia
RecruitingObservational study
HPC Offered for PRESERVE Expansion
Bone Marrow Transplant (BMT)
RecruitingPhase 1
Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease
Sickle Cell Disorders, Sickle Cell Disease
RecruitingPhase 4
Clinical and Biomarker Effects of Depot Medroxyprogesterone Acetate in Females With Sickle Cell Disease
Sickle Cell Disease (SCD), Vaso-Occlusive Pain Episode in Sickle Cell Disease
Not yet recruitingPhase 1
Buprenorphine for the Management of Pain in Children With Sickle Cell Disease
Sickle Cell Disease (SCD), Sickle Cell Disease Vaso-occlusive Crisis
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.
Ricardo Hermosilla, PhD study director
Vifor (International) Inc.
22 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing VIT-2763 120 mg, VIT-2763 360 mg and VIT-2763 240 mg in people with Sickle Cell Disease, run by Vifor (International) Inc. at 22 sites in 5 countries.
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 6 inclusion and 16 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 18 Feb 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 22 sites in United States, United Kingdom, Greece, Lebanon, France. 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 18 Feb 2025). 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?
Mean Change From Baseline in Haemolysis Marker (Indirect Bilirubin) (time frame: Baseline and after 8 weeks of treatment). 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 NCT04817670, which also lists 2 linked publications.
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 18 Feb 2025.