Safety, Tolerability, Pharmacokinetics (PK), Pharmacodyna... | NCT04364269

ID	Type	Description	Link
2019-002221-29	EudraCT Number

Status Module

Name	Class
Labcorp Corporation of America Holdings, Inc	INDUSTRY

Description Module

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
VIT-2763 Once a day (QD)	Experimental	Participants will be assigned to receive VIT-2763 once a day (QD) in a total daily dose of 60 mg or 120 mg depending on their body weight. The study medication (VIT-2763 and/or matching placebo) will be administered for all participants twice a day to maintain the blind.	Drug: VIT-2763 once a day (QD)
VIT-2763 Twice a day (BID)	Experimental	Participants will be assigned to receive VIT-2763 Twice a day (BID) in a total daily dose of 60 mg or 120 mg depending on their body weight.	Drug: VIT-2763 twice a day (BID)
Placebo	Placebo Comparator	Participants will be assigned to receive Placebo, Twice a day.	Drug: Placebo

Interventions

Name	Type	Description	Arm Group Labels	Other Names
VIT-2763 once a day (QD)	Drug	Participants will receive VIT-2763 QD at a dose of 60 mg if their body weight is between 40 kg to 59 kg or at a dose of 120 mg if their body weight is between 60 kg and 100 kg, during 12 weeks.

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)	Please note that in this section we are presenting just the overview of the adverse events experienced by the trial participants, in particular, the number of participants with at least one TEAE. Please refer to the detailed tables included on the Adverse Event Module for specifics.	From baseline to Week 16
Changes in the Systolic Blood Pressure (SBP) and Diastolic Blood Pressure (DBP)	Summary of the values by visit from baseline and changes from baseline by post-baseline visit.	From baseline to Week 12
Changes in the Heart Rate	Summary of the values by visit from baseline and changes from baseline by post-baseline visit.	From baseline to Week 12
Changes in 12-lead Electrocardiogram (ECG) Parameters	Values by visit from baseline and changes from baseline by post-baseline visit. The following ECG parameters were recorded: PR interval, QRS duration, QT interval, RR interval and QTcF interval. PR interval represents the time from the onset of the P wave to the start of the QRS complex. QRS duration represents the time required for a stimulus to spread through the ventricles (ventricular depolarization). QT interval represents the time from the start of the Q wave to the end of the T wave. RR interval represents the time from the onset of one R wave to the onset of the next one, one complete cardiac cycle. QT corrected for heart rate (QTc) interval reflects ventricular repolarization.	From baseline to Week 12

Secondary Outcomes

Measure	Description	Time Frame
Change From Baseline in Total Serum Iron	Assessment of total serum Iron from baseline over a 12-week period (absolute and change from baseline). For the serum iron parameter, the 'Baseline' was collected during the screening period within the biochemistry sample.	From baseline to Week 12
Change From Baseline in Serum Ferritin

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Documented diagnosis of NTDT, including a β-thalassemia intermedia-phenotype.
NTDT is defined as subjects having received less than 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 at east 14 days prior to randomisation).
Male and female adult NTDT subjects, 18-65 years of age inclusive (Cohort I only) at time of screening.
Male and female adolescent NTDT subjects, 12-17 years of age inclusive (Cohort II only) at time of screening.
Subjects must have a mean baseline hemoglobin (Hb) equal to or lower than 11 g/dl, based on at least 2 consecutive measurements with at least 1 week apart within 6 weeks prior to randomisation/baseline.

Exclusion Criteria:

Documented diagnosis of transfusion dependent thalassemia (TDT), including a beta-thalassemia major phenotype (including β0/β0, β+/β+, β0/β+ genotype), and mixed compound heterozygous for sickling phenotype variants such as Hb S/β- thalassemia, or transfusion dependent non-deletional Hb H disease (i.e., Hb constant spring) or Hb C disease.
Subjects on concomitant iron chelation therapy (ICT) or subjects on prior ICT when discontinued less than 4 weeks prior randomisation. If ICT was discontinued at least 4 weeks prior randomization the subject is eligible.
ICT naïve subjects or subjects who discontinued ICT therapy at least 6 months before the screening visit with serum ferritin lower than 150 ng/ml and/or documented liver iron concentration (LIC) equal to or lower than 1 mg/g liver dry weight assessed through magnetic resonance imaging (MRI), or subjects on prior ICT with serum ferritin lower than 300 ng/ml and/or documented LIC lower than 3 mg/g liver dry weight assessed through MRI.
Subjects with transferrin saturation (TSAT) less than 30%.
Subjects with documented LIC greater than 15 mg/g liver dry weight assessed through MRI, or a documented myocardial T2* less than 20 ms, if available per local practice and retrieved within 24 months prior to randomization.
Adult or adolescent subjects with body weight lower than 40.0 kg or greater than 100 kg at screening.
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.
Estimated glomerular filtration rate (eGFR) less than 30 ml/min/1.73 m2 (according to chronic kidney disease classification Stage 4 or higher), and/or significant albuminuria greater than 30 mg/mmol. eGFR should be estimated according to Chronic Kidney Disease Epidemiology Collaboration formula (CKI-EPI) in adults, and Schwartz formula in adolescents.
Newly diagnosed folate deficiency anemia and/or Vitamin B12 megaloblastic anemia. Subjects with known folate deficiency anemia and/or Vitamin B12 megaloblastic anemia who are on at least 12 weeks stable replacement therapy are eligible.
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.
Subjects with history of partial or total splenectomy within 6 months prior to screening.

Name	Affiliation	Role
Clinical Research Department	Vifor (International) Inc.	Study Director

Facility	Status	City	State	ZIP	Country	Contacts
Clinical Site #301		Athens	Attica	115 27	Greece
Clinical Site #302

PubMed Identifier	Type	Citation	Retractions
41291806	Derived	Kattamis A, Taher A, Viprakasit V, Levin C, Hermosilla R, Szecsody P, Richard F, Cappellini MD, Porter J. Safety and pharmacodynamics of the ferroportin inhibitor vamifeport in patients with non-transfusion-dependent beta-thalassemia: results from a randomized phase 2a study. Orphanet J Rare Dis. 2025 Nov 25;20(1):608. doi: 10.1186/s13023-025-04119-y.

Results Section

Participant Flow Module

Arm/Group Information

ID	Title	Description
FG000	VIT-2763 QD	Participants who received VIT-2763 once a day (QD) in a total daily dose of 60 mg or 120 mg, depending on their body weight, during 12 weeks. Participants received VIT-2763 QD at a dose of 60 mg if their body weight was between 40 kg to 59 kg or at a dose of 120 mg if their body weight was between 60 kg and 100 kg. The study medication (VIT-2763 and/or matching placebo) was administered to all participants twice a day to maintain the blind. QD = once a day

Baseline Characteristics Module

Outcome Measures Module

Adverse Events Module

More Info Module

Type	Includes Protocol	Includes SAP	Includes ICF	Document Label	Document Date	Document Uploaded Date	Document File Name
Prot	Yes	No	No	Study Protocol	Apr 12, 2021	Oct 14, 2022

Arm/Group Information

ID	Title	Description
BG000	VIT-2763 QD	Participants who received VIT-2763 once a day (QD) in a total daily dose of 60 mg or 120 mg, depending on their body weight, during 12 weeks. Participants received VIT-2763 QD at a dose of 60 mg if their body weight was between 40 kg to 59 kg or at a dose of 120 mg if their body weight was between 60 kg and 100 kg. The study medication (VIT-2763 and/or matching placebo) was administered to all participants twice a day to maintain the blind. QD = once a day
BG001	VIT-2763 BID	Participants who received VIT-2763 twice a day (BID) in a total daily dose of 60 mg or 120 mg, depending on their body weight, during 12 weeks. Participants received VIT-2763 BID at a dose of 60 mg if their body weight was between 40 kg to 59 kg or at a dose of 120 mg if their body weight was between 60 kg and 100 kg. BID = twice a day
BG002	Placebo	Participants who received twice a day hard capsules of placebo, during 12 weeks.
BG003	Total	Total of all reporting groups

Units	Counts
Participants	BG0009 BG00112 BG0024 BG00325

Type

Title

Description

Population Description

Reporting Status

Anticipated Posting Date

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Time Frame

Units Analyzed

Denominator Units Selected

Arm/Group Information

Denominators

Classes

Analyses

Primary

Number of Participants With Treatment-Emergent Adverse Events (TEAEs)

Please note that in this section we are presenting just the overview of the adverse events experienced by the trial participants, in particular, the number of participants with at least one TEAE. Please refer to the detailed tables included on the Adverse Event Module for specifics.

The safety set (SS) was defined as all randomized participants who had taken at least 1 dose of study medication. The participants in the SS were to be analyzed based on the treatment they received, regardless of randomization.

Posted

Count of Participants

Participants

From baseline to Week 16

ID	Title	Description
OG000	VIT-2763 QD	Participants who received VIT-2763 once a day (QD) in a total daily dose of 60 mg or 120 mg, depending on their body weight, during 12 weeks. Participants received VIT-2763 QD at a dose of 60 mg if their body weight was between 40 kg to 59 kg or at a dose of 120 mg if their body weight was between 60 kg and 100 kg. The study medication (VIT-2763 and/or matching placebo) was administered to all participants twice a day to maintain the blind. QD = once a day
OG001	VIT-2763 BID	Participants who received VIT-2763 twice a day (BID) in a total daily dose of 60 mg or 120 mg, depending on their body weight, during 12 weeks. Participants received VIT-2763 BID at a dose of 60 mg if their body weight was between 40 kg to 59 kg or at a dose of 120 mg if their body weight was between 60 kg and 100 kg. BID = twice a day
OG002	Placebo	Participants who received twice a day hard capsules of placebo, during 12 weeks.

Units	Counts
Participants	OG0009 OG00112 OG0024

Title

Denominators

Categories

Title	Measurements
	OG0006 OG0017 OG0023

Primary

Changes in the Systolic Blood Pressure (SBP) and Diastolic Blood Pressure (DBP)

Summary of the values by visit from baseline and changes from baseline by post-baseline visit.

Posted

Mean

Standard Deviation

millimeters of mercury (mmHg)

From baseline to Week 12

ID	Title	Description
OG000	VIT-2763 QD	Participants who received VIT-2763 once a day (QD) in a total daily dose of 60 mg or 120 mg, depending on their body weight, during 12 weeks. Participants received VIT-2763 QD at a dose of 60 mg if their body weight was between 40 kg to 59 kg or at a dose of 120 mg if their body weight was between 60 kg and 100 kg. The study medication (VIT-2763 and/or matching placebo) was administered to all participants twice a day to maintain the blind. QD = once a day
OG001	VIT-2763 BID	Participants who received VIT-2763 twice a day (BID) in a total daily dose of 60 mg or 120 mg, depending on their body weight, during 12 weeks. Participants received VIT-2763 BID at a dose of 60 mg if their body weight was between 40 kg to 59 kg or at a dose of 120 mg if their body weight was between 60 kg and 100 kg. BID = twice a day

Primary

Changes in the Heart Rate

Summary of the values by visit from baseline and changes from baseline by post-baseline visit.

Posted

Mean

Standard Deviation

Pulse Rate (bpm)

From baseline to Week 12

ID	Title	Description
OG000	VIT-2763 QD	Participants who received VIT-2763 once a day (QD) in a total daily dose of 60 mg or 120 mg, depending on their body weight, during 12 weeks. Participants received VIT-2763 QD at a dose of 60 mg if their body weight was between 40 kg to 59 kg or at a dose of 120 mg if their body weight was between 60 kg and 100 kg. The study medication (VIT-2763 and/or matching placebo) was administered to all participants twice a day to maintain the blind. QD = once a day
OG001	VIT-2763 BID	Participants who received VIT-2763 twice a day (BID) in a total daily dose of 60 mg or 120 mg, depending on their body weight, during 12 weeks. Participants received VIT-2763 BID at a dose of 60 mg if their body weight was between 40 kg to 59 kg or at a dose of 120 mg if their body weight was between 60 kg and 100 kg. BID = twice a day
OG002	Placebo

Primary

Changes in 12-lead Electrocardiogram (ECG) Parameters

Values by visit from baseline and changes from baseline by post-baseline visit. The following ECG parameters were recorded: PR interval, QRS duration, QT interval, RR interval and QTcF interval.

PR interval represents the time from the onset of the P wave to the start of the QRS complex. QRS duration represents the time required for a stimulus to spread through the ventricles (ventricular depolarization). QT interval represents the time from the start of the Q wave to the end of the T wave. RR interval represents the time from the onset of one R wave to the onset of the next one, one complete cardiac cycle. QT corrected for heart rate (QTc) interval reflects ventricular repolarization.

Posted

Mean

Standard Deviation

milliseconds (ms)

From baseline to Week 12

ID	Title	Description
OG000	VIT-2763 QD	Participants who received VIT-2763 once a day (QD) in a total daily dose of 60 mg or 120 mg, depending on their body weight, during 12 weeks. Participants received VIT-2763 QD at a dose of 60 mg if their body weight was between 40 kg to 59 kg or at a dose of 120 mg if their body weight was between 60 kg and 100 kg. The study medication (VIT-2763 and/or matching placebo) was administered to all participants twice a day to maintain the blind. QD = once a day
OG001	VIT-2763 BID

Secondary

Change From Baseline in Total Serum Iron

Assessment of total serum Iron from baseline over a 12-week period (absolute and change from baseline).

For the serum iron parameter, the 'Baseline' was collected during the screening period within the biochemistry sample.

The full analysis set (FAS) was defined as all participants who were randomized to treatment, received at least one dose of randomized treatment and had at least one post-baseline pharmacodynamic assessment. The FAS was created in accordance with the intent-to-treat principles. The participants in the FAS were to be analyzed based on the treatment that they were randomized to.

Posted

Mean

Standard Deviation

Micromoles per Litre (umol/L)

From baseline to Week 12

ID	Title	Description
OG000	VIT-2763 QD	Participants who received VIT-2763 once a day (QD) in a total daily dose of 60 mg or 120 mg, depending on their body weight, during 12 weeks. Participants received VIT-2763 QD at a dose of 60 mg if their body weight was between 40 kg to 59 kg or at a dose of 120 mg if their body weight was between 60 kg and 100 kg. The study medication (VIT-2763 and/or matching placebo) was administered to all participants twice a day to maintain the blind. QD = once a day
OG001	VIT-2763 BID	Participants who received VIT-2763 twice a day (BID) in a total daily dose of 60 mg or 120 mg, depending on their body weight, during 12 weeks. Participants received VIT-2763 BID at a dose of 60 mg if their body weight was between 40 kg to 59 kg or at a dose of 120 mg if their body weight was between 60 kg and 100 kg. BID = twice a day

Secondary

Change From Baseline in Serum Ferritin

Assessment of serum ferritin from baseline over a 12-week period (absolute and change from baseline).

For the serum ferritin parameter, the 'Baseline' was collected during the screening period within the biochemistry sample.

Posted

Mean

Standard Deviation

Microgrammes per Litre (ug/L)

From baseline to Week 12

ID	Title	Description
OG000	VIT-2763 QD	Participants who received VIT-2763 once a day (QD) in a total daily dose of 60 mg or 120 mg, depending on their body weight, during 12 weeks. Participants received VIT-2763 QD at a dose of 60 mg if their body weight was between 40 kg to 59 kg or at a dose of 120 mg if their body weight was between 60 kg and 100 kg. The study medication (VIT-2763 and/or matching placebo) was administered to all participants twice a day to maintain the blind. QD = once a day
OG001	VIT-2763 BID	Participants who received VIT-2763 twice a day (BID) in a total daily dose of 60 mg or 120 mg, depending on their body weight, during 12 weeks. Participants received VIT-2763 BID at a dose of 60 mg if their body weight was between 40 kg to 59 kg or at a dose of 120 mg if their body weight was between 60 kg and 100 kg. BID = twice a day

Secondary

Change From Baseline in Serum Transferrin

Assessment of serum transferrin from baseline over a 12-week period (absolute and change from baseline).

For the serum transferrin parameter, the "Baseline 2h post-dose" was defined as the value at Visit 3 2h post-dose.

Posted

Mean

Standard Deviation

Grammes per Litre (g/L)

From baseline to Week 12

ID	Title	Description
OG000	VIT-2763 QD	Participants who received VIT-2763 once a day (QD) in a total daily dose of 60 mg or 120 mg, depending on their body weight, during 12 weeks. Participants received VIT-2763 QD at a dose of 60 mg if their body weight was between 40 kg to 59 kg or at a dose of 120 mg if their body weight was between 60 kg and 100 kg. The study medication (VIT-2763 and/or matching placebo) was administered to all participants twice a day to maintain the blind. QD = once a day
OG001	VIT-2763 BID	Participants who received VIT-2763 twice a day (BID) in a total daily dose of 60 mg or 120 mg, depending on their body weight, during 12 weeks. Participants received VIT-2763 BID at a dose of 60 mg if their body weight was between 40 kg to 59 kg or at a dose of 120 mg if their body weight was between 60 kg and 100 kg. BID = twice a day

Secondary

Change From Baseline in Calculated Transferrin Saturation (TSAT) )

Assessment of TSAT from baseline over a 12-week period (absolute and change from baseline).

For the calculated transferrin saturation parameter, the 'Baseline' was collected during the screening period within the biochemistry sample.

Transferrin Saturation (TSAT) was calculated as Total Iron /Total Iron Binding Capacity (TIBC) X 100.

Posted

Mean

Standard Deviation

TSAT Percentage (%)

From baseline to Week 12

ID	Title	Description
OG000	VIT-2763 QD	Participants who received VIT-2763 once a day (QD) in a total daily dose of 60 mg or 120 mg, depending on their body weight, during 12 weeks. Participants received VIT-2763 QD at a dose of 60 mg if their body weight was between 40 kg to 59 kg or at a dose of 120 mg if their body weight was between 60 kg and 100 kg. The study medication (VIT-2763 and/or matching placebo) was administered to all participants twice a day to maintain the blind. QD = once a day
OG001	VIT-2763 BID	Participants who received VIT-2763 twice a day (BID) in a total daily dose of 60 mg or 120 mg, depending on their body weight, during 12 weeks. Participants received VIT-2763 BID at a dose of 60 mg if their body weight was between 40 kg to 59 kg or at a dose of 120 mg if their body weight was between 60 kg and 100 kg. BID = twice a day

Secondary

Pharmacokinetics Parameters - VIT-2763 Plasma Concentration Over Time

Sparse sampling for determination of VIT-2763 plasma concentration following multiple dosing was obtained from pre-dose trough to 3 hours or 4 hours post-dose at selected study visits. Pharmacokinetics parameters (Cmax, clearance, distribution volume, area under the curve (AUC) were not calculated for the study.

Posted

Mean

Standard Deviation

nanograms per milliliter (ng/mL)

Baseline, Week 4, Week 8 and Week 12

ID	Title	Description
OG000	VIT-2763 QD	Participants who received VIT-2763 once a day (QD) in a total daily dose of 60 mg or 120 mg, depending on their body weight, during 12 weeks. Participants received VIT-2763 QD at a dose of 60 mg if their body weight was between 40 kg to 59 kg or at a dose of 120 mg if their body weight was between 60 kg and 100 kg. The study medication (VIT-2763 and/or matching placebo) was administered to all participants twice a day to maintain the blind. QD = once a day
OG001	VIT-2763 BID	Participants who received VIT-2763 twice a day (BID) in a total daily dose of 60 mg or 120 mg, depending on their body weight, during 12 weeks. Participants received VIT-2763 BID at a dose of 60 mg if their body weight was between 40 kg to 59 kg or at a dose of 120 mg if their body weight was between 60 kg and 100 kg. BID = twice a day

Arm/Groups

ID	Title	Description	Deaths (At Risk)	Serious Events (At Risk)	Other Events (Affected)	Other Events (At Risk)
EG000	VIT-2763 QD	Participants who received VIT-2763 once a day (QD) in a total daily dose of 60 mg or 120 mg, depending on their body weight, during 12 weeks. Participants received VIT-2763 QD at a dose of 60 mg if their body weight was between 40 kg to 59 kg or at a dose of 120 mg if their body weight was between 60 kg and 100 kg. The study medication (VIT-2763 and/or matching placebo) was administered to all participants twice a day to maintain the blind. QD = once a day	9	9	6	9
EG001	VIT-2763 BID	Participants who received VIT-2763 twice a day (BID) in a total daily dose of 60 mg or 120 mg, depending on their body weight, during 12 weeks. Participants received VIT-2763 BID at a dose of 60 mg if their body weight was between 40 kg to 59 kg or at a dose of 120 mg if their body weight was between 60 kg and 100 kg. BID = twice a day	12	12	7	12
EG002	Placebo	Participants who received twice a day hard capsules of placebo, during 12 weeks.	4	4	3	4

Other Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Anaemia	Blood and lymphatic system disorders	MedDRA (23.0)	Systematic Assessment	EG0001 events1 affected9 at risk EG0011 events1 affected12 at risk EG0020 events0 affected4 at risk
Haemolysis	Blood and lymphatic system disorders	MedDRA (23.0)	Systematic Assessment	EG0001 events1 affected9 at risk EG0010 events0 affected12 at risk EG0020 events0 affected4 at risk
Angina pectoris	Cardiac disorders	MedDRA (23.0)	Systematic Assessment	EG0000 events0 affected9 at risk EG0011 events1 affected12 at risk EG0020 events0 affected4 at risk
Palpitations	Cardiac disorders	MedDRA (23.0)	Systematic Assessment	EG0001 events1 affected9 at risk EG0011 events1 affected12 at risk EG0020 events0 affected4 at risk
Hypoacusis	Ear and labyrinth disorders	MedDRA (23.0)	Systematic Assessment	EG0000 events0 affected9 at risk EG0010 events0 affected12 at risk EG0021 events1 affected4 at risk
Photopsia	Eye disorders	MedDRA (23.0)	Systematic Assessment	EG0001 events1 affected9 at risk EG0010 events0 affected12 at risk EG0020 events0 affected4 at risk
Vitreous floaters	Eye disorders	MedDRA (23.0)	Systematic Assessment	EG0001 events1 affected9 at risk EG0010 events0 affected12 at risk EG0020 events0 affected4 at risk
Abdominal pain	Gastrointestinal disorders	MedDRA (23.0)	Systematic Assessment	EG0001 events1 affected9 at risk EG0010 events0 affected12 at risk EG0020 events0 affected4 at risk
Dyspepsia	Gastrointestinal disorders	MedDRA (23.0)	Systematic Assessment	EG0000 events0 affected9 at risk EG0012 events2 affected12 at risk EG0020 events0 affected4 at risk
Faeces discoloured	Gastrointestinal disorders	MedDRA (23.0)	Systematic Assessment	EG0000 events0 affected9 at risk EG0011 events1 affected12 at risk EG0020 events0 affected4 at risk
Nausea	Gastrointestinal disorders	MedDRA (23.0)	Systematic Assessment	EG0000 events0 affected9 at risk EG0011 events1 affected12 at risk EG0020 events0 affected4 at risk
Vomiting	Gastrointestinal disorders	MedDRA (23.0)	Systematic Assessment	EG0000 events0 affected9 at risk EG0011 events1 affected12 at risk EG0020 events0 affected4 at risk
Fatigue	General disorders	MedDRA (23.0)	Systematic Assessment	EG0002 events2 affected9 at risk EG0012 events2 affected12 at risk EG0020 events0 affected4 at risk
Non-cardiac chest pain	General disorders	MedDRA (23.0)	Systematic Assessment	EG0000 events0 affected9 at risk EG0010 events0 affected12 at risk EG0021 events1 affected4 at risk
Pyrexia	General disorders	MedDRA (23.0)	Systematic Assessment	EG0000 events0 affected9 at risk EG0012 events2 affected12 at risk EG0020 events0 affected4 at risk
Jaundice	Hepatobiliary disorders	MedDRA (23.0)	Systematic Assessment	EG0001 events1 affected9 at risk EG0010 events0 affected12 at risk EG0020 events0 affected4 at risk
Rhinitis	Infections and infestations	MedDRA (23.0)	Systematic Assessment	EG0000 events0 affected9 at risk EG0011 events1 affected12 at risk EG0020 events0 affected4 at risk
Urinary tract infection	Infections and infestations	MedDRA (23.0)	Systematic Assessment	EG0001 events1 affected9 at risk EG0010 events0 affected12 at risk EG0020 events0 affected4 at risk
Alanine aminotransferase increased	Investigations	MedDRA (23.0)	Systematic Assessment	EG0000 events0 affected9 at risk EG0011 events1 affected12 at risk EG0020 events0 affected4 at risk
Aspartate aminotransferase increased	Investigations	MedDRA (23.0)	Systematic Assessment	EG0000 events0 affected9 at risk EG0011 events1 affected12 at risk EG0020 events0 affected4 at risk
Blood creatine phosphokinase increased	Investigations	MedDRA (23.0)	Systematic Assessment	EG0000 events0 affected9 at risk EG0011 events1 affected12 at risk EG0020 events0 affected4 at risk
Decreased appetite	Metabolism and nutrition disorders	MedDRA (23.0)	Systematic Assessment	EG0000 events0 affected9 at risk EG0011 events1 affected12 at risk EG0020 events0 affected4 at risk
Back pain	Musculoskeletal and connective tissue disorders	MedDRA (23.0)	Systematic Assessment	EG0000 events0 affected9 at risk EG0011 events1 affected12 at risk EG0020 events0 affected4 at risk
Bone pain	Musculoskeletal and connective tissue disorders	MedDRA (23.0)	Systematic Assessment	EG0000 events0 affected9 at risk EG0011 events1 affected12 at risk EG0020 events0 affected4 at risk
Headache	Nervous system disorders	MedDRA (23.0)	Systematic Assessment	EG0000 events0 affected9 at risk EG0011 events1 affected12 at risk EG0021 events1 affected4 at risk
Presyncope	Nervous system disorders	MedDRA (23.0)	Systematic Assessment	EG0000 events0 affected9 at risk EG0011 events1 affected12 at risk EG0020 events0 affected4 at risk
Pollakiuria	Renal and urinary disorders	MedDRA (23.0)	Systematic Assessment	EG0000 events0 affected9 at risk EG0011 events1 affected12 at risk EG0020 events0 affected4 at risk
Cough	Respiratory, thoracic and mediastinal disorders	MedDRA (23.0)	Systematic Assessment	EG0000 events0 affected9 at risk EG0011 events1 affected12 at risk EG0020 events0 affected4 at risk
Oropharyngeal pain	Respiratory, thoracic and mediastinal disorders	MedDRA (23.0)	Systematic Assessment	EG0000 events0 affected9 at risk EG0011 events1 affected12 at risk EG0020 events0 affected4 at risk
Respiratory symptom	Respiratory, thoracic and mediastinal disorders	MedDRA (23.0)	Systematic Assessment	EG0000 events0 affected9 at risk EG0010 events0 affected12 at risk EG0021 events1 affected4 at risk
Pruritus	Skin and subcutaneous tissue disorders	MedDRA (23.0)	Systematic Assessment	EG0000 events0 affected9 at risk EG0010 events0 affected12 at risk EG0021 events1 affected4 at risk
Skin ulcer	Skin and subcutaneous tissue disorders	MedDRA (23.0)	Systematic Assessment	EG0000 events0 affected9 at risk EG0011 events1 affected12 at risk EG0020 events0 affected4 at risk

ID	Term
D013789	Thalassemia
D000745	Anemia, Hemolytic, Congenital
D000743	Anemia, Hemolytic
D000740	Anemia
D006402	Hematologic Diseases
D006425	Hemic and Lymphatic Diseases
D006453	Hemoglobinopathies
D030342	Genetic Diseases, Inborn
D009358	Congenital, Hereditary, and Neonatal Diseases and Abnormalities