A Phase 2 interventional study of VIT-2763 60 mg QD and VIT-2763 60 mg BID in Beta-Thalassemia, sponsored by Vifor (International) Inc.. Withdrawn at 7 sites in 3 countries. Open to participants aged 18 Years to 65 Years. Per ClinicalTrials.gov, last updated 2022-11-10.
Sponsored by Vifor (International) Inc. (part of CSL Limited) · Phase 2, Interventional, and Treatment
The main purpose of this study is to evaluate the efficacy of 3 multiple doses of VIT-2763 as measured by the reduction in red blood cell (RBC) transfusion burden from Week 13 to Week 24, to identify the most efficacious and safe dose.
All patients giving written informed consent will undergo a 12-week screening period to determine eligibility for study entry. At Day 1, patients who meet the eligibility requirements will be randomized in a double-blind manner to 1 of 4 treatment groups: VIT-2763 60 mg (once daily), 60 mg (twice daily), or 120 mg (twice daily) or placebo.
The randomisation will be stratified (balanced allocation across treatment groups) according to β0/β0 genotype (yes/no).
The duration of the treatment with VIT-2763 or placebo is 24 weeks, after which patients will undergo a 12-week post-treatment follow-up period.
294 studies on the registry are indexed under beta-Thalassemia; 69 are open to participants now.
Browse beta-Thalassemia studies →Vifor (International) Inc. is the lead sponsor of 10 studies on the registry; none are open to participants now.
First submitted before Vifor (International) Inc. became part of CSL Limited.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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).
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
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.
The US National Cancer Institute’s scale of how severe an adverse event is — any unwanted medical event during a study, such as a symptom, an illness or an abnormal test result, whether or not a treatment caused it. Whether it did is judged separately.
“Side effects of earlier treatment resolved to grade 1 or less” means they have settled to mild or gone.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
CTCAE grade
The US National Cancer Institute’s scale of how severe an adverse event is — any unwanted medical event during a study, such as a symptom, an illness or an abnormal test result, whether or not a treatment caused it. Whether it did is judged separately.
“Side effects of earlier treatment resolved to grade 1 or less” means they have settled to mild or gone.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
VIT-2763 60 mg administered once daily
Drug: VIT-2763 60 mg QD
VIT-2763 60 mg administered twice daily
Drug: VIT-2763 60 mg BID
VIT-2763 120 mg administered twice daily
Drug: VIT-2763 120 mg BID
Placebo capsule administered twice daily
Drug: Placebo
Participants receive one VIT-2763 60 mg capsule plus one Placebo capsule orally in the morning, and 2 Placebo capsules orally in the evening, for 24 weeks.
Also known as: Vamifeport 60 mg QD
Participants receive one VIT-2763 60 mg capsule plus one Placebo capsule orally, twice daily for 24 weeks.
Also known as: Vamifeport 60 mg BID
Participants receive two VIT-2763 60 mg capsules orally, twice daily for 24 weeks.
Also known as: Vamifeport 120 mg BID
Participants receive two Placebo capsules matching VIT-2763 orally, twice daily for 24 weeks.
Proportion of patients achieving ≥33% reduction of RBC transfusions from baseline and a reduction of ≥2 units assessed consecutively from Week 13 to Week 24 compared to the baseline transfusion
Time frame: Week 13 to Week 24 comparing to Baseline (Day -83 to Day 1)
Change from baseline in RBC transfusions over Weeks 13 to 24 compared to the baseline RBC transfusion burden derived using the last 12 weeks prior to randomization.
Time frame: Week 13 to Week 24 comparing to Baseline (Day -83 to Day 1)
Proportion of patients achieving ≥50% reduction of RBC transfusions and ≥2 units assessed over any consecutive 12-week interval from Week 1 to 24.
Time frame: Any consecutive 12-week interval from Week 1 to Week 24 comparing to Baseline (Day -83 to Day 1)
Proportion of patients achieving ≥33% reduction of RBC transfusions and ≥2 units assessed over any consecutive 12-week interval from Week 1 to 24.
Time frame: Any consecutive 12-week interval from Week 1 to Week 24 comparing to Baseline (Day -83 to Day 1)
Mean change from baseline in Quality of Life (QoL) total score
Transfusion-dependent QoL Questionnaire (TranQuol): a disease-specific, validated, QoL measure developed for thalassemia patients. The adult version includes 36 questions grouped into 5 domains: physical health, emotional health, sexual health, family functioning, and school/career functioning. The total score ranges from 0 (worst) to 100 (best).
Time frame: Week 15 and Week 24 comparing to Baseline (Day 1)
Plan to share: No
No publications or documents are linked to this record.
Started Sep 2021 (actual). Primary completion is estimated for Jul 2023, 22 months after the start.
How these figures are worked out, and which studies are left out of them.
This study is withdrawn, as verified in Mar 2022. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: VIT-2763