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Effect of NUV001 Supplementation in Patients Suffering From Sickle Cell Disease (SCD)

Primary Purpose

Sickle Cell Disease

Status
Recruiting
Phase
Not Applicable
Locations
France
Study Type
Interventional
Intervention
NUV001
Sponsored by
LGD
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Sickle Cell Disease focused on measuring safety, tolerance, SS genotype of sickle cell disease

Eligibility Criteria

18 Years - 60 Years (Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria: Man or woman comprised between 18 and 60 years old. Patients diagnosed with homozygous sickle cell anemia of SS genotype (documented by genotyping). Females of childbearing potential should be using one of the following acceptable methods of birth control: Intrauterine Device (IUD) in place for at least 60 days prior to the first dose of the study throughout the study and for 30 days after completion of the study. Hormonal contraceptives for at least 90 days prior to the first dose of the study throughout the study, and for 30 days after study completion. Patients whose weight is greater than 50 kg. Patient that has been treated with an anti-sickling agent (Hydroxyurea) within six months of the screening visit, must maintain the therapy continuous and unmodified for at least six months with the intent to continue for the duration of the study. Patient available to attend on an outpatient basis for visits provided for in the protocol and able to complete the data collection documents (and quality of life scale). Patient has given written informed consent. Patient with health insurance scheme Exclusion Criteria: Patient with known or suspected allergy to any ingredient of the food supplement . Patient having consumed food supplements containing tryptophan, glutamine or vitamin B3 in its various forms (nicotinic acid/niacin and nicotinamide) during the month before selection Patient has a significant medical condition that required hospitalization (other than sickle cell crisis) within two months of the screening visit. Patient has serum albumin < 3.0 g/dl. Patient has been transfused and received any blood products within three months of the Screening Visit. Patient has been hospitalized for acute vaso-occlusive crisis within one months of the Screening Visit. Patient has clinically significant, cardiovascular or liver disease, renal or lung insufficiency or lymphopenia (with clinically significant abnormal results on the screening bioassays: complete blood count, transaminases (ASAT, ALAT, GGT, ALP), bilirubin, creatinine, CPK, Ionogram, blood glucose, lipid profile). Patients with diagnosed cancer in the past 2 years Pregnant or lactating woman. Woman of childbearing potential should have a negative (serum or urinary TBD) pregnancy test at screening and a negative urine pregnancy test at inclusion prior to administration of the Study Product.

Sites / Locations

  • Aphm Hopital La Timone Adultes Sce Medecine Interne (Umap)Recruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

NUV001

Arm Description

Daily supplementation with NUV001 1000 mg

Outcomes

Primary Outcome Measures

Incidence of treatment-emergent adverse events
Subject incidence of treatment-emergent Adverse events (AEs) and Severe adverse events. Including Hospitalization days and Vaso-occlusive crisis occurrences.
Incidence of treatment-emergent adverse events
Subject incidence of treatment-emergent Adverse events (AEs) and Severe adverse events. Including Hospitalization days and Vaso-occlusive crisis occurrences.
Incidence of treatment-emergent adverse events
Subject incidence of treatment-emergent Adverse events (AEs) and Severe adverse events. Including Hospitalization days and Vaso-occlusive crisis occurrences.
Incidence of treatment-emergent adverse events
Subject incidence of treatment-emergent Adverse events (AEs) and Severe adverse events. Including Hospitalization days and Vaso-occlusive crisis occurrences.
Incidence of treatment-emergent clinically significant changes in clinical laboratory safety tests
Subject incidence of treatment-emergent clinically significant changes in clinical laboratory safety tests (Blood formulation, Inflammation parameters, Conjugated and free Bilirubin, alanine and aspartate aminotransferases (ASAT, ALAT),gamma-glutamyltransferase (GGT) and alkaline phosphatase (ALP), Creatine phosphokinase (CPK), Creatine, Urea, Creatinuria, Proteinuria, creatinine clearance)Ionogram Na/K/Cl, fasting blood glucose, albumine)
Incidence of treatment-emergent clinically significant changes in clinical laboratory safety tests
Subject incidence of treatment-emergent clinically significant changes in clinical laboratory safety tests (Blood formulation, Inflammation parameters, Conjugated and free Bilirubin, alanine and aspartate aminotransferases (ASAT, ALAT),gamma-glutamyltransferase (GGT) and alkaline phosphatase (ALP), Creatine phosphokinase (CPK), Creatine, Urea, Creatinuria, Proteinuria, creatinine clearance)Ionogram Na/K/Cl, fasting blood glucose, albumine)
Incidence of treatment-emergent clinically significant changes in clinical laboratory safety tests
Subject incidence of treatment-emergent clinically significant changes in clinical laboratory safety tests (Blood formulation, Inflammation parameters, Conjugated and free Bilirubin, alanine and aspartate aminotransferases (ASAT, ALAT),gamma-glutamyltransferase (GGT) and alkaline phosphatase (ALP), Creatine phosphokinase (CPK), Creatine, Urea, Creatinuria, Proteinuria, creatinine clearance)Ionogram Na/K/Cl, fasting blood glucose, albumine)
Incidence of treatment-emergent clinically significant changes in clinical laboratory safety tests
Subject incidence of treatment-emergent clinically significant changes in clinical laboratory safety tests (Blood formulation, Inflammation parameters, Conjugated and free Bilirubin, alanine and aspartate aminotransferases (ASAT, ALAT),gamma-glutamyltransferase (GGT) and alkaline phosphatase (ALP), Creatine phosphokinase (CPK), Creatine, Urea, Creatinuria, Proteinuria, creatinine clearance)Ionogram Na/K/Cl, fasting blood glucose, albumine)
Incidence of treatment-emergent clinically significant changes in Vital Signs
Subject incidence of treatment-emergent clinically significant changes in vital signs (systolic and diastolic blood Pressure, Pulse Rate and Body temperature)
Incidence of treatment-emergent clinically significant changes in Vital Signs
Subject incidence of treatment-emergent clinically significant changes in vital signs (systolic and diastolic blood Pressure, Pulse Rate and Body temperature)
Incidence of treatment-emergent clinically significant changes in Vital Signs
Subject incidence of treatment-emergent clinically significant changes in vital signs (systolic and diastolic blood Pressure, Pulse Rate and Body temperature)
Incidence of treatment-emergent clinically significant changes in Vital Signs
Subject incidence of treatment-emergent clinically significant changes in vital signs (systolic and diastolic blood Pressure, Pulse Rate and Body temperature)

Secondary Outcome Measures

Change in Lactate dehydrogenase (LDH) levels
Lactate dehydrogenase (LDH) levels evolutions from baseline
Change in Hemoglobin (HGB) levels
Hemoglobin (HGB) levels evolutions from baseline.
Change in Hematocrit (HCT)
Hematocrit (HCT) evolutions from baseline.
Change in circulating level of red line cell precursors
circulating level of red line cell precursors (basophilic, polychromic and orthochromatic / reticulocytes erythroid cells) evolutions from baseline measured by flow cytometry
Change in mean corpuscular volume (MCV)
mean corpuscular volume (MCV) evolutions from baseline measured
Change in mean corpuscular hemoglobin content (MCHT)
mean corpuscular hemoglobin content (MCHT) evolutions from baseline measured
Change in reticulocyte level
reticulocytes count expressed in percentage of red blood cells
F-Hemoglobin
F-Hemoglobin (HbF) level, % of F-cells, Distribution of HbF within F-cells (% of high and low F-cells) evolutions from baseline.
Circulating irreversibly sickle cells (ISCs)
Evolution from baseline of the percent of circulating irreversibly sickle cells (ISCs)
Red blood cells (RBC) sickling
Evolution from baseline of in vitro Red blood cells (RBC) sickling under hypoxia
Nicotinamide adenine dinucleotide (NAD)+ concentration in whole blood
Evolution from baseline of NAD+ concentration in whole blood
β-Nicotinamide mononucleotide (NMN) concentration in whole blood
Evolution from baseline of NMN concentration in whole blood
Methyl-Nicotinamide (Me-NAM) concentration in plasma
Evolution from baseline of Me-NAM concentration in plasma
Nicotinamide (NAM) concentration in plasma
Evolution from baseline of NAM concentration in plasma
Nicotinamide (NAM) concentration in urine
Evolution from baseline of NAM concentration in urine
N-Methyl-2-pyridone-5-carboxamide (2PY) concentration in urine
Evolution from baseline of 2PY concentration in urine
Change from baseline of self-questionnaire quality of life 36-Item Short Form Survey (SF-36) version 1
Evolutions from baseline in General activity, mood, walking ability, normal work (including housework), relations with other people, sleep, enjoyment of life as measured by the 36-Item Short Form Survey (SF-36) version 1 questionnaire. SF-36 scores range from 0 (worst) to 100 (best)

Full Information

First Posted
February 28, 2023
Last Updated
April 21, 2023
Sponsor
LGD
Collaborators
CEN Biotech, Assistance Publique Hopitaux De Marseille, Etablissement Français du Sang
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1. Study Identification

Unique Protocol Identification Number
NCT05789355
Brief Title
Effect of NUV001 Supplementation in Patients Suffering From Sickle Cell Disease (SCD)
Official Title
Effect of NUV001 Supplementation for 120 Days in Patients Suffering From Sickle Cell Disease (SCD) S Homozygous (SS) Genotype: A Pilot Study
Study Type
Interventional

2. Study Status

Record Verification Date
April 2023
Overall Recruitment Status
Recruiting
Study Start Date
April 1, 2023 (Actual)
Primary Completion Date
November 10, 2023 (Anticipated)
Study Completion Date
December 31, 2023 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
LGD
Collaborators
CEN Biotech, Assistance Publique Hopitaux De Marseille, Etablissement Français du Sang

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

Brief Summary
This is a pilot study of daily dosing of NUV001 as a dietary supplement in 12 sickle cell disease patients with 3 months of follow-up plus 1 month after supplementation.The present study is designed to evaluate, first, the safety and tolerability parameters as well as to measure the plasma and urinary residues of daily oral doses of NUV001. Secondly, the study will evaluate the impact of NUV001 on biological parameters and quality of life of patients.
Detailed Description
This is a monocentric, prospective, open label pilot study designed for 12 adult patients suffering of Sickle Cell disease (SCD) SS genotype each 12 receiving the active supplementation of NUV001, 1000mg/day (4 x 250 mg tablet) for 3 months of follow-up plus 1 month after supplementation. A stratification according to the medical treatment is planned. At least 2 patients suffering of SCD SS genotype without hydroxyurea treatment and maximum 10 patients suffering of SCD SS genotype in association with hydroxyurea treatment. If a subject is withdrawn from this study part, the subject may be replaced as necessary with another subject assigned to the same treatment at the discretion of the sponsor's team in consultation with the investigator. The current study is designed to assess in the first part, the safety, tolerability, plasma, and urine residual rate parameters of daily oral doses of NUV001 as dietary supplement n adult patients suffering of sickle cell disease SS genotype. In a second part, the study will assess the pharmacological impact of NUV001 on biological parameters and the quality of life in patient suffering of sickle cell disease SS genotype.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Sickle Cell Disease
Keywords
safety, tolerance, SS genotype of sickle cell disease

7. Study Design

Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Model Description
Single-center, prospective, open-label pilot study.
Masking
None (Open Label)
Allocation
N/A
Enrollment
12 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
NUV001
Arm Type
Experimental
Arm Description
Daily supplementation with NUV001 1000 mg
Intervention Type
Dietary Supplement
Intervention Name(s)
NUV001
Intervention Description
Daily supplementation with NUV001 at 1000 mg (4 tablets of 250 mg each) for 90 days with a prolonged follow-up of 1 month (30 days) after stopping the supplementation
Primary Outcome Measure Information:
Title
Incidence of treatment-emergent adverse events
Description
Subject incidence of treatment-emergent Adverse events (AEs) and Severe adverse events. Including Hospitalization days and Vaso-occlusive crisis occurrences.
Time Frame
between Day 0 and Day 30
Title
Incidence of treatment-emergent adverse events
Description
Subject incidence of treatment-emergent Adverse events (AEs) and Severe adverse events. Including Hospitalization days and Vaso-occlusive crisis occurrences.
Time Frame
between Day 0 and Day 60
Title
Incidence of treatment-emergent adverse events
Description
Subject incidence of treatment-emergent Adverse events (AEs) and Severe adverse events. Including Hospitalization days and Vaso-occlusive crisis occurrences.
Time Frame
between Day 0 and Day 90
Title
Incidence of treatment-emergent adverse events
Description
Subject incidence of treatment-emergent Adverse events (AEs) and Severe adverse events. Including Hospitalization days and Vaso-occlusive crisis occurrences.
Time Frame
between Day 0 and Day 120
Title
Incidence of treatment-emergent clinically significant changes in clinical laboratory safety tests
Description
Subject incidence of treatment-emergent clinically significant changes in clinical laboratory safety tests (Blood formulation, Inflammation parameters, Conjugated and free Bilirubin, alanine and aspartate aminotransferases (ASAT, ALAT),gamma-glutamyltransferase (GGT) and alkaline phosphatase (ALP), Creatine phosphokinase (CPK), Creatine, Urea, Creatinuria, Proteinuria, creatinine clearance)Ionogram Na/K/Cl, fasting blood glucose, albumine)
Time Frame
between Day 0 and Day 30
Title
Incidence of treatment-emergent clinically significant changes in clinical laboratory safety tests
Description
Subject incidence of treatment-emergent clinically significant changes in clinical laboratory safety tests (Blood formulation, Inflammation parameters, Conjugated and free Bilirubin, alanine and aspartate aminotransferases (ASAT, ALAT),gamma-glutamyltransferase (GGT) and alkaline phosphatase (ALP), Creatine phosphokinase (CPK), Creatine, Urea, Creatinuria, Proteinuria, creatinine clearance)Ionogram Na/K/Cl, fasting blood glucose, albumine)
Time Frame
between Day 0 and Day 60
Title
Incidence of treatment-emergent clinically significant changes in clinical laboratory safety tests
Description
Subject incidence of treatment-emergent clinically significant changes in clinical laboratory safety tests (Blood formulation, Inflammation parameters, Conjugated and free Bilirubin, alanine and aspartate aminotransferases (ASAT, ALAT),gamma-glutamyltransferase (GGT) and alkaline phosphatase (ALP), Creatine phosphokinase (CPK), Creatine, Urea, Creatinuria, Proteinuria, creatinine clearance)Ionogram Na/K/Cl, fasting blood glucose, albumine)
Time Frame
between Day 0 and Day 90
Title
Incidence of treatment-emergent clinically significant changes in clinical laboratory safety tests
Description
Subject incidence of treatment-emergent clinically significant changes in clinical laboratory safety tests (Blood formulation, Inflammation parameters, Conjugated and free Bilirubin, alanine and aspartate aminotransferases (ASAT, ALAT),gamma-glutamyltransferase (GGT) and alkaline phosphatase (ALP), Creatine phosphokinase (CPK), Creatine, Urea, Creatinuria, Proteinuria, creatinine clearance)Ionogram Na/K/Cl, fasting blood glucose, albumine)
Time Frame
between Day 0 and Day 120
Title
Incidence of treatment-emergent clinically significant changes in Vital Signs
Description
Subject incidence of treatment-emergent clinically significant changes in vital signs (systolic and diastolic blood Pressure, Pulse Rate and Body temperature)
Time Frame
between Day 0 and Day 30
Title
Incidence of treatment-emergent clinically significant changes in Vital Signs
Description
Subject incidence of treatment-emergent clinically significant changes in vital signs (systolic and diastolic blood Pressure, Pulse Rate and Body temperature)
Time Frame
between Day 0 and Day 60
Title
Incidence of treatment-emergent clinically significant changes in Vital Signs
Description
Subject incidence of treatment-emergent clinically significant changes in vital signs (systolic and diastolic blood Pressure, Pulse Rate and Body temperature)
Time Frame
between Day 0 and Day 90
Title
Incidence of treatment-emergent clinically significant changes in Vital Signs
Description
Subject incidence of treatment-emergent clinically significant changes in vital signs (systolic and diastolic blood Pressure, Pulse Rate and Body temperature)
Time Frame
between Day 0 and Day 120
Secondary Outcome Measure Information:
Title
Change in Lactate dehydrogenase (LDH) levels
Description
Lactate dehydrogenase (LDH) levels evolutions from baseline
Time Frame
Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.
Title
Change in Hemoglobin (HGB) levels
Description
Hemoglobin (HGB) levels evolutions from baseline.
Time Frame
Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.
Title
Change in Hematocrit (HCT)
Description
Hematocrit (HCT) evolutions from baseline.
Time Frame
Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.
Title
Change in circulating level of red line cell precursors
Description
circulating level of red line cell precursors (basophilic, polychromic and orthochromatic / reticulocytes erythroid cells) evolutions from baseline measured by flow cytometry
Time Frame
Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.
Title
Change in mean corpuscular volume (MCV)
Description
mean corpuscular volume (MCV) evolutions from baseline measured
Time Frame
Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.
Title
Change in mean corpuscular hemoglobin content (MCHT)
Description
mean corpuscular hemoglobin content (MCHT) evolutions from baseline measured
Time Frame
Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.
Title
Change in reticulocyte level
Description
reticulocytes count expressed in percentage of red blood cells
Time Frame
Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.
Title
F-Hemoglobin
Description
F-Hemoglobin (HbF) level, % of F-cells, Distribution of HbF within F-cells (% of high and low F-cells) evolutions from baseline.
Time Frame
Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.
Title
Circulating irreversibly sickle cells (ISCs)
Description
Evolution from baseline of the percent of circulating irreversibly sickle cells (ISCs)
Time Frame
Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.
Title
Red blood cells (RBC) sickling
Description
Evolution from baseline of in vitro Red blood cells (RBC) sickling under hypoxia
Time Frame
Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.
Title
Nicotinamide adenine dinucleotide (NAD)+ concentration in whole blood
Description
Evolution from baseline of NAD+ concentration in whole blood
Time Frame
Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.
Title
β-Nicotinamide mononucleotide (NMN) concentration in whole blood
Description
Evolution from baseline of NMN concentration in whole blood
Time Frame
Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.
Title
Methyl-Nicotinamide (Me-NAM) concentration in plasma
Description
Evolution from baseline of Me-NAM concentration in plasma
Time Frame
Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.
Title
Nicotinamide (NAM) concentration in plasma
Description
Evolution from baseline of NAM concentration in plasma
Time Frame
Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.
Title
Nicotinamide (NAM) concentration in urine
Description
Evolution from baseline of NAM concentration in urine
Time Frame
Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.
Title
N-Methyl-2-pyridone-5-carboxamide (2PY) concentration in urine
Description
Evolution from baseline of 2PY concentration in urine
Time Frame
Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.
Title
Change from baseline of self-questionnaire quality of life 36-Item Short Form Survey (SF-36) version 1
Description
Evolutions from baseline in General activity, mood, walking ability, normal work (including housework), relations with other people, sleep, enjoyment of life as measured by the 36-Item Short Form Survey (SF-36) version 1 questionnaire. SF-36 scores range from 0 (worst) to 100 (best)
Time Frame
Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
60 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Man or woman comprised between 18 and 60 years old. Patients diagnosed with homozygous sickle cell anemia of SS genotype (documented by genotyping). Females of childbearing potential should be using one of the following acceptable methods of birth control: Intrauterine Device (IUD) in place for at least 60 days prior to the first dose of the study throughout the study and for 30 days after completion of the study. Hormonal contraceptives for at least 90 days prior to the first dose of the study throughout the study, and for 30 days after study completion. Patients whose weight is greater than 50 kg. Patient that has been treated with an anti-sickling agent (Hydroxyurea) within six months of the screening visit, must maintain the therapy continuous and unmodified for at least six months with the intent to continue for the duration of the study. Patient available to attend on an outpatient basis for visits provided for in the protocol and able to complete the data collection documents (and quality of life scale). Patient has given written informed consent. Patient with health insurance scheme Exclusion Criteria: Patient with known or suspected allergy to any ingredient of the food supplement . Patient having consumed food supplements containing tryptophan, glutamine or vitamin B3 in its various forms (nicotinic acid/niacin and nicotinamide) during the month before selection Patient has a significant medical condition that required hospitalization (other than sickle cell crisis) within two months of the screening visit. Patient has serum albumin < 3.0 g/dl. Patient has been transfused and received any blood products within three months of the Screening Visit. Patient has been hospitalized for acute vaso-occlusive crisis within one months of the Screening Visit. Patient has clinically significant, cardiovascular or liver disease, renal or lung insufficiency or lymphopenia (with clinically significant abnormal results on the screening bioassays: complete blood count, transaminases (ASAT, ALAT, GGT, ALP), bilirubin, creatinine, CPK, Ionogram, blood glucose, lipid profile). Patients with diagnosed cancer in the past 2 years Pregnant or lactating woman. Woman of childbearing potential should have a negative (serum or urinary TBD) pregnancy test at screening and a negative urine pregnancy test at inclusion prior to administration of the Study Product.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Amira NAMSI, PhD
Phone
+33 (0)6 08 15 03 01
Email
amira.namsi@groupecen.com
First Name & Middle Initial & Last Name or Official Title & Degree
Florent HERPIN
Phone
+33 (0)3 80 68 05 06
Email
florent.herpin@groupecen.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Laurent LAGANIER
Organizational Affiliation
LGD SARL
Official's Role
Study Director
First Name & Middle Initial & Last Name & Degree
Estelle JEAN-MIGNARD
Organizational Affiliation
Assistance Publique Hopitaux Marseille
Official's Role
Principal Investigator
Facility Information:
Facility Name
Aphm Hopital La Timone Adultes Sce Medecine Interne (Umap)
City
Marseille
ZIP/Postal Code
13005
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Estelle JEAN-MIGNARD, MD
Phone
+33 (0)4 91 38 80 93
Email
Estelle.JEAN@ap-hm.fr
First Name & Middle Initial & Last Name & Degree
Imane AGOUTI
Email
Imane.AGOUTI@ap-hm.fr
First Name & Middle Initial & Last Name & Degree
Estelle JEAN-MIGNARD, MD

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

Effect of NUV001 Supplementation in Patients Suffering From Sickle Cell Disease (SCD)

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