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Trial details
A study to evaluate if VIT-2763 may be beneficial in the treatment of Nontransfusion Dependent Beta-thalassaemia.
Current status:
Approved
|
Date registered:
02/04/2020
|
Date last updated:
09/03/2020
Trial version(s)
History: 02/10/2019
Current: 02/10/2019
Click here to view the tips and fields' descriptions
Main Information
Primary registry identifying number
LBCTR2020021295
Protocol number
VIT-2763-THAL-201
MOH registration number
Trial already registered with the MoPH
Study registered at the country of origin
No.Prevalence of the disease is low in the country of origin Switzerland
Type of registration
Prospective
Date of registration in national regulatory agency
Primary sponsor
Vifor (International) Inc.
Primary sponsor: Country of origin
Switzerland
Public title
A study to evaluate if VIT-2763 may be beneficial in the treatment of Nontransfusion Dependent Beta-thalassaemia.
Acronym
Scientific title
A Phase 2a, Double-blind, Randomised, Placebo-controlled, Parallel Group, Multicentre Study on Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of Multiple Doses of VIT-2763 in Subjects with Non-transfusion Dependent Beta-thalassaemia
Acronym
Brief summary of the study: English
This is a Phase 2a, Double-blind, Randomised, Placebo-controlled, Parallel Group,Multicentre Study. The primary objective is to assess the safety and tolerability of VIT-2763 versus placebo in adult and adolescent NTDT subjects over a 12-week treatment period.The secondary objectives are to assess Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of Multiple Doses of VIT-2763.
Brief summary of the study: Arabic
هذه الدراسة في المرحلة 2 أ ، مزدوجة التعمية ، والعشوائية ، بالمقارنة مع دواء وهمي ، المجموعة الموازية ، دراسة متعددة المراكز. الهدف الأساسي هو تقييم سلامة التحمل و VIT-2763 مقابل الدواء الوهمي في موضوعات NTDT للبالغين والمراهقين على مدى فترة علاج مدتها 12 أسبوعًا. الأهداف الثانوية هي تقييم الحرائك الدوائية والديناميكا الدوائية والفعالية الأولية لجرعات متعددة من VIT-276
Health conditions/problem studied: Specify
Chronic anemia due to ineffective erythropoiesis (IE) in subjects with β-thalassaemia
Interventions: Specify
The study will commence with enrolment and treatment of adult NTDT subjects(Cohort I). Adult subjects will be randomised in an 8:8:4 ratio to receive either VIT-2763 once daily (QD) or twice daily (BID) or placebo, at a dose of 120 mg for subjects with a body weight ≥60 kg or at dose of 60 mg for subjects with a body weight <60 kg.Following Cohort I review, enrolment of adolescent NTDT subjects into Cohort II. Adolescent subjects will be randomised in a 4:4:2 ratio to receive either VIT-2763 QD or BID or placebo, at a dose of 120 mg for subjects with a body weight ≥60 kg or at a dose of 60 mg for subjects with a body weight <60 kg
Key inclusion and exclusion criteria: Inclusion criteria
1. Documented diagnosis of NTDT, including a β-thalassaemia intermedia-phenotype. 2. NTDT is defined as subjects having received <5 units of red blood cells (RBCs) during the 24-week period prior to randomisation/first drug administration of VIT-2763 or placebo (Day 1; 1 unit is defined as 200 to 350 ml of transfused packed RBCs and last RBC transfusion must have been received ≥14 days prior to randomisation). Note: Subjects who are supposed to receive RBC transfusions after randomisation in the Investigator’s opinion, and according to local practise, and having received at least 1 dose of VIT-2763, may be considered to stay on study treatment for safety reasons, and in case there are no tolerability concerns. Subjects will be censored for secondary efficacy. 3. Male and female adult NTDT subjects, 18-65 years of age inclusive (Cohort I only) at time of screening. 4. Male and female adolescent NTDT subjects, 12-17 years of age inclusive (Cohort II only) at time of screening. 5. Subjects must have a mean baseline Hb ≤11 g/dl, based on 2 consecutive measurements ≥1 week apart within 6 weeks prior to randomisation/baseline, and obtained Hb values show less than 10% relative difference (and equal or less than 1.0 g/dl absolute change between the highest and lowest value) between at least 2 measurements. Note: If there is 1 retrospective Hb value available for the subject at maximum of 2 weeks prior to screening (Day -28), the Hb value can be taken into consideration. A subject not meeting this criterion would be excluded but can be rescreened at maximum 2 times at a later time point.
Key inclusion and exclusion criteria: Gender
Both
Key inclusion and exclusion criteria: Age minimum
12
Key inclusion and exclusion criteria: Age maximum
65
Key inclusion and exclusion criteria: Exclusion criteria
1. Documented diagnosis of transfusion dependent thalassaemia (TDT), including a beta-thalassaemia major phenotype (including β0/β0, β+/β+, β0/β+ genotype), and mixed compound heterozygous for sickling phenotype variants such as Hb S/β-thalassaemia, or transfusion dependent non-deletional Hb H disease (i.e., Hb constant spring) or Hb C disease. 2. Subjects on concomitant iron chelation therapy (ICT) or subjects on prior ICT when discontinued less than 4 weeks prior randomisation. Note: If ICT was discontinued ≥4 weeks prior randomisation the subject is eligible. 3. ICT naïve subjects with serum ferritin <150 ng/ml and documented LIC ≤1 mg/g liver dry weight assessed through MRI, or subjects on prior ICT with serum ferritin <300 ng/ml and documented LIC <3 mg/g liver dry weight assessed through MRI. 4. Subjects with TSAT <30%. 5. Subjects with documented LIC >15 mg/g liver dry weight assessed through MRI, or a documented myocardial T2-star (T2*) <20 ms. 6. Adult or adolescent subjects with body weight <40.0 kg or >100 kg at screening and/or randomisation. 7. Chronic liver disease and/or alanine transaminase (ALT), aspartate transaminase (AST) or gamma-glutamyl transpeptidase (GGT) above 3-fold the upper limit of normal (ULN) range at screening. Note: A subject fulfilling this criterion will be excluded but can be rescreened at a later time point (in order to fulfil eligibility, ≥2 values within ≥1 week should be assessed and be within eligibility limits). 8. Estimated glomerular filtration rate (eGFR) <30 ml/min/1.73 m2 (according to chronic kidney disease classification Stage 4 or higher), and/or significant albuminuria >30 mg/mmol. eGFR should be estimated according to Cockcroft-Gault. 9. Newly diagnosed folate deficiency anaemia and/or Vitamin B12 megaloblastic anaemia. Subjects with known folate deficiency anaemia and/or Vitamin B12 megaloblastic anaemia who are on ≥12 weeks stable replacement therapy are eligible. Note: A subject fulfilling this criterion will be excluded but can be rescreened at a later time point. 10. Any history or clinically important finding of cardiac disorders, such as clinically relevant cardiac arrhythmia, cardiomyopathy, coronary disease, valve disorder, or heart failure according to New York Heart Association classification 3-4. 11. Subjects with partial or total splenectomy.
Type of Study
Type
Interventional
Type of intervention
Pharmaceutical
Trial scope
Other
Trial scope: Specify scope
safety, efficacy, pharmacokinetics, pharmacodynamics, dose response.
Study design: Allocation
Randomized controlled trial
Study design: Masking
Blinded (masking used)
Study design: Control
Placebo
Study phase
2
Study design: Purpose
Treatment
Study design: Assignment
Parallel
IMP has market authorization
No
Name of IMP
VIT-2763
Type of IMP
Others
Type of IMP: Specify
chemical
Pharmaceutical class
VIT-2763 is a Ferroportin (FPN) inhibitor and hepcidin-mimetic.
Therapeutic indication
Iron loading anaemias and thalassaemia
Therapeutic benefit
Administration of VIT-2763 may results in improvement of anaemia and amelioration of ineffective erythropoiesis in NTD beta-thalassemia patients, as it was already shown in nonclinical disease models. This improvement in ineffective erythropoiesis may result in a clinical benefit for NTD β-thalassemia subjects, by improving the symptomatology of the chronic anemia and the complications of the extramedullary hematopoiesis.
Biospecimen retention
Samples without DNA
Biospecimen description
Urine will be taken for urinalysis (pH, protein, glucose, ketone, blood, spot urine for assessment of protein/creatinine and albumin/creatinine ratio) and urine drug and alcohol screen. blood samples for haematology and clinical chemistry and coagulation
Target sample size
36
Actual enrollment target size
Date of first enrollment: Type
Anticipated
Date of first enrollment: Date
11/05/2020
Date of study closure: Type
Anticipated
Date of study closure: Date
11/05/2021
Recruitment status
Pending
Date of completion
IPD sharing statement plan
No
IPD sharing statement description
Not applicable
Additional data URL
none
Summary Results
Secondary Identifying Numbers
Full name of issuing authority
Secondary identifying number
EMA
2019-002221-29
Sources of Monetary or Material Support
Name
Vifor (International) Inc.
Secondary Sponsors
Name
Not Applicable
Contact for Public/Scientific Queries
Contact type
First name
Last name
Address
Country
Telephone
Email
Affiliation
Public
Aziz
Zoghbi
MCT-CRO, Berytech Technology and Health, 5th Floor Damascus Road, Beirut, Lebanon
Lebanon
009611612 500
zog_az@mctcro. com
Regional Manager
Scientific
Ali
Taher
Chronic Care Center, Hazmieh, Lebanon
Lebanon
009613755 669
ataher@aub.edu. lb
PI
Centers/Hospitals Involved in the Study
Center/Hospital name
Name of principles investigator
Principles investigator speciality
Ethical approval
Chronic Care Center
Dr.Ali Taher
Professor of Medicine, Hematology & Oncology
Approved
Ethics Review
Ethics approval obtained
Approval date
Contact name
Contact email
Contact phone
Chronic Care Center
30/01/2020
Michelle Abi Saad
cccmas@chroniccare.org.lb
05-455101
Countries of Recruitment
Name
Lebanon
Health Conditions or Problems Studied
Condition
Code
Keyword
NTDT Thalassemia
Thalassaemia (D56)
thalassemia
Interventions
Intervention
Description
Keyword
VIT-2763 60 mg capsules
Adult subjects will be randomised in an 8:8:4 ratio to receive either VIT-2763 QD or BID or placebo at a dose of 120 mg for subjects with a body weight ≥60 kg or at a dose of 60 mg for subjects with a body weight <60 kg.
cohort 1/2
Primary Outcomes
Name
Time points
Measure
Reported or observed adverse events (AEs)
last study contact Visit 9/Week 16.
by SOC and PT MedDRA coded term, by severity and relation to study product in each treatment group.
Reported or observed serious adverse events (SAEs)
4 weeks (28+-4 days) following the last study drug administration.
by SOC and PT MedDRA coded term, by severity and relation to study product in each treatment group
Changes in vital signs
screening Visit V1 and on Visits V3 to V8. Vital signs should be performed at V3 to V8 before IMP dosing, after a resting period of at least 5 minutes.
Blood pressure and pulse rate
Changes in clinical laboratory safety tests
over 12 week treatment
haematology, serum biochemistry, coagulation, and urinalysis
12-Lead ECG
over 12 week treatment
ventricular rate, PR interval, QRS duration, QT interval and QTcF
Physical examination
Screening Visit V1 (i.e., Day -28 to -1) and on Visit V3 (Day 1), and V8 (Day 84)
general appearance, head (eyes, ears, nose and throat), cardiovascular, respiratory, abdominal, musculoskeletal, neurological, lymph nodes, and skin.
Key Secondary Outcomes
Name
Time points
Measure
Assessment of iron parameters
from baseline over a 12-week period
total serum iron, serum ferritin, serum transferrin, calculated transferrin saturation (TSAT
PK parameters
from pre-dose trough to 3 hours or 4 hours post-dose at selected study visits
Cmax, clearance, distribution volume, area under the curve (AUC)
Trial Results
Summary results in Lebanon
Study results globally
Date of posting of results summaries
Date of first journal publication of results
Results URL link
Baseline characteristics
Participant flow
Adverse events
Outcome measures
URL to protocol files
Link(s) to publications related to the study
Changes History
Change
Date
Download as PDF
Save a PDF copy of the summary of the trial