Efficacy and Safety Study of Multiple Doses of VIT-2763 in Adults With Transfusion-dependent Beta-thalassemia

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

WITHDRAWN

Clinical Phase

PHASE2

Study Classification

INTERVENTIONAL

Study Start Date

2021-09-20

Study Completion Date

2023-07-31

Brief Summary

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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.

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Detailed Description

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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

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Beta-Thalassemia

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators

Study Groups

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VIT-2763 60 mg QD

VIT-2763 60 mg administered once daily

Group Type EXPERIMENTAL

VIT-2763 60 mg QD

Intervention Type DRUG

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 BID

VIT-2763 60 mg administered twice daily

Group Type EXPERIMENTAL

VIT-2763 60 mg BID

Intervention Type DRUG

Participants receive one VIT-2763 60 mg capsule plus one Placebo capsule orally, twice daily for 24 weeks.

VIT-2763 120 mg BID

VIT-2763 120 mg administered twice daily

Group Type EXPERIMENTAL

VIT-2763 120 mg BID

Intervention Type DRUG

Participants receive two VIT-2763 60 mg capsules orally, twice daily for 24 weeks.

Placebo

Placebo capsule administered twice daily

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Participants receive two Placebo capsules matching VIT-2763 orally, twice daily for 24 weeks.

Interventions

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VIT-2763 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.

Intervention Type DRUG

VIT-2763 60 mg BID

Participants receive one VIT-2763 60 mg capsule plus one Placebo capsule orally, twice daily for 24 weeks.

Intervention Type DRUG

VIT-2763 120 mg BID

Participants receive two VIT-2763 60 mg capsules orally, twice daily for 24 weeks.

Intervention Type DRUG

Placebo

Participants receive two Placebo capsules matching VIT-2763 orally, twice daily for 24 weeks.

Intervention Type DRUG

Other Intervention Names

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Vamifeport 60 mg QD Vamifeport 60 mg BID Vamifeport 120 mg BID

Eligibility Criteria

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Inclusion Criteria

* 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

Exclusion Criteria

* 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
* 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.

Minimum Eligible Age

18 Years

Maximum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No