Beta-ThalassemiaPhase 2NCT04938635
Efficacy and Safety Study of Multiple Doses of VIT-2763 in Adults With Transfusion-dependent Beta-thalassemia
A Phase 2 study testing VIT-2763 60 mg QD, VIT-2763 60 mg BID and VIT-2763 120 mg BID in people with Beta-Thalassemia, run by Vifor (International) Inc. at 7 sites in 3 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Beta-Thalassemia
- What is being tested?
- VIT-2763 60 mg QD, VIT-2763 60 mg BID and VIT-2763 120 mg BID
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- 7 sitesin 3 countries
- Study dates
- 20 Sep 2021to Jul 2023
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Body weight ≥40.0 kg and ≤100 kg at screening
- Documented diagnosis of beta-thalassemia or Hb E/beta-thalassemia
- Red blood cell (RBC) transfusion dependence, defined as at least 6 RBC units in the 24 weeks prior to randomization and no transfusion-free period for ≥35 days during that period
- Ability to understand the requirements of the study and provide written informed consent
Who cannot join
- Documented diagnosis of Hb S/beta-thalassemia, alpha-thalassemia, or delta beta (δβ)-thalassemia, or hereditary persistence of foetal Hb.
- History of partial or total splenectomy within 4 months prior to screening.
- History of myocardial iron overload
- Chronic liver disease or history of liver cirrhosis
- Clinically relevant renal disease
- History or clinically important finding of cardiac disorders
Show all 12Show fewer
- History of clinically significant lung disease
- Uncontrolled hypertension (> Grade 1 according to NCI CTCAE current version)
- Unable to take and absorb oral medications.
- Pregnancy or breastfeeding
- History of drug or alcohol abuse within 2 years prior to screening
- History or concomitant solid tumors and/or hematological malignancies unless resolved in the ≥5 past years.
Trial locations and contacts
7 sites in 3 countries.
7 sites in 3 countries
Bulgaria3 sites
Investigational site #802
Plovdiv
Investigational site #801
Sofia
Investigational site #804
Stara Zagora
Israel3 sites
Investigator Site 404
Jerusalem
Investigator Site 406
Petah Tikva
Investigator Site 405
Safed
United States1 site
Investigator site #710
Whittier, California, 90603-2137
About this study
Official title: A Phase 2b, Double-blind, Randomised, Placebo-controlled, Multicentre Study to Assess the Efficacy and Safety of VIT-2763 Multiple Doses in Adults With Transfusion-dependent Beta-thalassaemia
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.
Read the detailed description
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.
- Conditions
- Beta-Thalassemia
- Being tested
- VIT-2763 60 mg QD, VIT-2763 60 mg BID, VIT-2763 120 mg BID, Placebo
- Keywords
- Beta-Thalassemia, Transfusion-dependent 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 60 mg QDDrug
Also called Vamifeport 60 mg QD
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.
VIT-2763 60 mg BIDDrug
Also called Vamifeport 60 mg BID
Participants receive one VIT-2763 60 mg capsule plus one Placebo capsule orally, twice daily for 24 weeks.
VIT-2763 120 mg BIDDrug
Also called Vamifeport 120 mg BID
Participants receive two VIT-2763 60 mg capsules orally, twice daily for 24 weeks.
PlaceboDrug
Participants receive two Placebo capsules matching VIT-2763 orally, twice daily for 24 weeks.
| Study group | Role | What they receive |
|---|---|---|
| VIT-2763 60 mg QD | Experimental | VIT-2763 60 mg administered once daily Receives: Drug: VIT-2763 60 mg QD |
| VIT-2763 60 mg BID | Experimental | VIT-2763 60 mg administered twice daily Receives: Drug: VIT-2763 60 mg BID |
| VIT-2763 120 mg BID | Experimental | VIT-2763 120 mg administered twice daily Receives: Drug: VIT-2763 120 mg BID |
| Placebo | Placebo comparator | Placebo capsule administered twice daily Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: Week 13 to Week 24 comparing to Baseline (Day -83 to Day 1)
Secondary outcomes (4)
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.
Measured: 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.
Measured: 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.
Measured: 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).
Measured: Week 15 and Week 24 comparing to Baseline (Day 1)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Jun 2021
- Study start20 Sep 2021
- Primary completionJul 2023 (estimated)
- Study completionJul 2023 (estimated)
Why it stopped: Strategic reasons
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vifor (International) Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Labcorp Corporation of America Holdings, Inc
- Responsible party
- Sponsor
- Investigators
- Peter Szecsödy, Study director, Vifor (International) Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04938635
- Sponsor's protocol ID
- VIT-2763-THAL-203
- Other IDs
- 2021-001639-23
- Status last verified
- Mar 2022
- First submitted
- 16 Jun 2021
- Last updated
- 10 Nov 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Nov 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2b, Double-blind, Randomised, Placebo-controlled, Multicentre Study to Assess the Efficacy and Safety of VIT-2763 Multiple Doses in Adults With Transfusion-dependent Beta-thalassaemia
- ClinicalTrials.gov ID
- NCT04938635
- Acronym / sponsor ID
- VIT-2763-THAL-203
- Conditions
- Beta-Thalassemia
- Keywords
- Beta-Thalassemia, Transfusion-dependent Thalassemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Strategic reasons
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Vifor (International) Inc.Industry (pharma, biotech, devices)
- Collaborators
- Labcorp Corporation of America Holdings, Inc
- Study start
- 20 Sep 2021 (actual)
- Primary completion
- Jul 2023 (estimated)
- Study completion
- Jul 2023 (estimated)
- First posted
- 24 Jun 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| VIT-2763 60 mg QD | Experimental | Drug: VIT-2763 60 mg QDVIT-2763 60 mg administered once daily |
| VIT-2763 60 mg BID | Experimental | Drug: VIT-2763 60 mg BIDVIT-2763 60 mg administered twice daily |
| VIT-2763 120 mg BID | Experimental | Drug: VIT-2763 120 mg BIDVIT-2763 120 mg administered twice daily |
| Placebo | Placebo comparator | Drug: PlaceboPlacebo capsule administered twice daily |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Body weight ≥40.0 kg and ≤100 kg at screening
- Documented diagnosis of beta-thalassemia or Hb E/beta-thalassemia
- Red blood cell (RBC) transfusion dependence, defined as at least 6 RBC units in the 24 weeks prior to randomization and no transfusion-free period for ≥35 days during that period
- Ability to understand the requirements of the study and provide written informed consent
Key exclusion criteria (12 listed)
- Documented diagnosis of Hb S/beta-thalassemia, alpha-thalassemia, or delta beta (δβ)-thalassemia, or hereditary persistence of foetal Hb.
- History of partial or total splenectomy within 4 months prior to screening.
- History of myocardial iron overload
- Chronic liver disease or history of liver cirrhosis
- Clinically relevant renal disease
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 60 mg QDAlso: Vamifeport 60 mg QDParticipants 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. | Drug | VIT-2763 60 mg QD |
| VIT-2763 60 mg BIDAlso: Vamifeport 60 mg BIDParticipants receive one VIT-2763 60 mg capsule plus one Placebo capsule orally, twice daily for 24 weeks. | Drug | VIT-2763 60 mg BID |
| VIT-2763 120 mg BIDAlso: Vamifeport 120 mg BIDParticipants receive two VIT-2763 60 mg capsules orally, twice daily for 24 weeks. | Drug | VIT-2763 120 mg BID |
| PlaceboParticipants receive two Placebo capsules matching VIT-2763 orally, twice daily for 24 weeks. | Drug | Placebo |
Primary outcome
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)
4 secondary outcomes
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
Time frame: Week 15 and Week 24 comparing to Baseline (Day 1)
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Nov 2022.
- Individual participant dataSharing planned: No
Related trials for Beta-Thalassemia
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
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.
Peter Szecsödy study director
Vifor (International) Inc.
7 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing VIT-2763 60 mg QD, VIT-2763 60 mg BID and VIT-2763 120 mg BID in people with Beta-Thalassemia, run by Vifor (International) Inc. at 7 sites in 3 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 4 inclusion and 12 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: withdrawn, as of the sponsor's last update on 10 Nov 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in Bulgaria, Israel, United States. 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?
The study was withdrawn before enrolling participants, so there are no results.
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?
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)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 10 Nov 2022.