- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT05789355
Effect of NUV001 Supplementation in Patients Suffering From Sickle Cell Disease (SCD)
Effect of NUV001 Supplementation for 120 Days in Patients Suffering From Sickle Cell Disease (SCD) S Homozygous (SS) Genotype: A Pilot Study
Study Overview
Status
Conditions
Intervention / Treatment
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 If a subject is withdrawn from this study part, the subject may be replaced as necessary with another subject assigned to the same treatment at the discretion of the sponsor's team in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.
Study Type
Enrollment (Estimated)
Phase
- Not Applicable
Contacts and Locations
Study Contact
- Name: Amira NAMSI, PhD
- Phone Number: +33 (0)6 08 15 03 01
- Email: amira.namsi@groupecen.com
Study Contact Backup
- Name: Florent HERPIN
- Phone Number: +33 (0)3 80 68 05 06
- Email: florent.herpin@groupecen.com
Study Locations
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Marseille, France, 13005
- Recruiting
- Aphm Hopital La Timone Adultes Sce Medecine Interne (Umap)
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Principal Investigator:
- Estelle JEAN-MIGNARD, MD
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Contact:
- Estelle JEAN-MIGNARD, MD
- Phone Number: +33 (0)4 91 38 80 93
- Email: Estelle.JEAN@ap-hm.fr
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Contact:
- Imane AGOUTI
- Email: Imane.AGOUTI@ap-hm.fr
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Description
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.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: NUV001
Daily supplementation with NUV001 1000 mg
|
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
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Incidence of treatment-emergent adverse events
Time Frame: between Day 0 and Day 30
|
Subject incidence of treatment-emergent Adverse events (AEs) and Severe adverse events.
Including Hospitalization days and Vaso-occlusive crisis occurrences.
|
between Day 0 and Day 30
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Incidence of treatment-emergent adverse events
Time Frame: between Day 0 and Day 60
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Subject incidence of treatment-emergent Adverse events (AEs) and Severe adverse events.
Including Hospitalization days and Vaso-occlusive crisis occurrences.
|
between Day 0 and Day 60
|
Incidence of treatment-emergent adverse events
Time Frame: between Day 0 and Day 90
|
Subject incidence of treatment-emergent Adverse events (AEs) and Severe adverse events.
Including Hospitalization days and Vaso-occlusive crisis occurrences.
|
between Day 0 and Day 90
|
Incidence of treatment-emergent adverse events
Time Frame: between Day 0 and Day 120
|
Subject incidence of treatment-emergent Adverse events (AEs) and Severe adverse events.
Including Hospitalization days and Vaso-occlusive crisis occurrences.
|
between Day 0 and Day 120
|
Incidence of treatment-emergent clinically significant changes in clinical laboratory safety tests
Time Frame: between Day 0 and Day 30
|
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)
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between Day 0 and Day 30
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Incidence of treatment-emergent clinically significant changes in clinical laboratory safety tests
Time Frame: between Day 0 and Day 60
|
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)
|
between Day 0 and Day 60
|
Incidence of treatment-emergent clinically significant changes in clinical laboratory safety tests
Time Frame: between Day 0 and Day 90
|
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)
|
between Day 0 and Day 90
|
Incidence of treatment-emergent clinically significant changes in clinical laboratory safety tests
Time Frame: between Day 0 and Day 120
|
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)
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between Day 0 and Day 120
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Incidence of treatment-emergent clinically significant changes in Vital Signs
Time Frame: between Day 0 and Day 30
|
Subject incidence of treatment-emergent clinically significant changes in vital signs (systolic and diastolic blood Pressure, Pulse Rate and Body temperature)
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between Day 0 and Day 30
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Incidence of treatment-emergent clinically significant changes in Vital Signs
Time Frame: between Day 0 and Day 60
|
Subject incidence of treatment-emergent clinically significant changes in vital signs (systolic and diastolic blood Pressure, Pulse Rate and Body temperature)
|
between Day 0 and Day 60
|
Incidence of treatment-emergent clinically significant changes in Vital Signs
Time Frame: between Day 0 and Day 90
|
Subject incidence of treatment-emergent clinically significant changes in vital signs (systolic and diastolic blood Pressure, Pulse Rate and Body temperature)
|
between Day 0 and Day 90
|
Incidence of treatment-emergent clinically significant changes in Vital Signs
Time Frame: between Day 0 and Day 120
|
Subject incidence of treatment-emergent clinically significant changes in vital signs (systolic and diastolic blood Pressure, Pulse Rate and Body temperature)
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between Day 0 and Day 120
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Change in Lactate dehydrogenase (LDH) levels
Time Frame: Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.
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Lactate dehydrogenase (LDH) levels evolutions from baseline
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Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.
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Change in Hemoglobin (HGB) levels
Time Frame: Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.
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Hemoglobin (HGB) levels evolutions from baseline.
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Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.
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Change in Hematocrit (HCT)
Time Frame: Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.
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Hematocrit (HCT) evolutions from baseline.
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Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.
|
Change in circulating level of red line cell precursors
Time Frame: Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.
|
circulating level of red line cell precursors (basophilic, polychromic and orthochromatic / reticulocytes erythroid cells) evolutions from baseline measured by flow cytometry
|
Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.
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Change in mean corpuscular volume (MCV)
Time Frame: Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.
|
mean corpuscular volume (MCV) evolutions from baseline measured
|
Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.
|
Change in mean corpuscular hemoglobin content (MCHT)
Time Frame: Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.
|
mean corpuscular hemoglobin content (MCHT) evolutions from baseline measured
|
Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.
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Change in reticulocyte level
Time Frame: Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.
|
reticulocytes count expressed in percentage of red blood cells
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Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.
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F-Hemoglobin
Time Frame: Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.
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F-Hemoglobin (HbF) level, % of F-cells, Distribution of HbF within F-cells (% of high and low F-cells) evolutions from baseline.
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Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.
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Circulating irreversibly sickle cells (ISCs)
Time Frame: Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.
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Evolution from baseline of the percent of circulating irreversibly sickle cells (ISCs)
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Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.
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Red blood cells (RBC) sickling
Time Frame: Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.
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Evolution from baseline of in vitro Red blood cells (RBC) sickling under hypoxia
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Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.
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Nicotinamide adenine dinucleotide (NAD)+ concentration in whole blood
Time Frame: Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.
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Evolution from baseline of NAD+ concentration in whole blood
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Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.
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β-Nicotinamide mononucleotide (NMN) concentration in whole blood
Time Frame: Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.
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Evolution from baseline of NMN concentration in whole blood
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Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.
|
Methyl-Nicotinamide (Me-NAM) concentration in plasma
Time Frame: Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.
|
Evolution from baseline of Me-NAM concentration in plasma
|
Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.
|
Nicotinamide (NAM) concentration in plasma
Time Frame: Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.
|
Evolution from baseline of NAM concentration in plasma
|
Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.
|
Nicotinamide (NAM) concentration in urine
Time Frame: Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.
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Evolution from baseline of NAM concentration in urine
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Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.
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N-Methyl-2-pyridone-5-carboxamide (2PY) concentration in urine
Time Frame: Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.
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Evolution from baseline of 2PY concentration in urine
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Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.
|
Change from baseline of self-questionnaire quality of life 36-Item Short Form Survey (SF-36) version 1
Time Frame: Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.
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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)
|
Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.
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Collaborators and Investigators
Sponsor
Investigators
- Study Director: Laurent LAGANIER, LGD SARL
- Principal Investigator: Estelle JEAN-MIGNARD, ASSISTANCE PUBLIQUE HOPITAUX MARSEILLE
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Estimated)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- Safety-DRNUV001- SCD
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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