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) SS GENOTYPE: A PILOT STUDY

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.

Study Overview

• Status: Completed
• Conditions: Sickle Cell Disease
• Intervention / Treatment: Dietary supplement: NUV001

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 If a subject is withdrawn from this study part, the subject may be replaced as necessary with another subject assigned to the same treatment at the discretion of the sponsor's team in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 food supplement in adult patients with SCD, 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: Interventional
• Enrollment (Actual): 12
• Phase: Not Applicable

Contacts and Locations

Study Locations

• France
  • Marseille, France, 13005
    • Aphm Hopital La Timone Adultes Sce Medecine Interne (Umap)

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 60 years (Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion criteria:

• Participants meeting the following criteria could be included:
• Male or female between 18 and 60 years old.

• Females of childbearing potential should be using one of the following acceptable methods of birth control:

  • Intrauterine Device in place for at least 60 days prior to the first dose of the study (Visit 1) 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 (Visit 1) throughout the study, and for 30 days after study completion.
• Patients whose weight is greater than 50 kg.
• Patients diagnosed with homozygous sickle cell anemia of SS genotype (documented by genotyping).
• Patients who have been treated with an anti-sickling agent (Siklos®) within six months of the screening visit (Visit 0) must maintain the therapy continuous and unmodified for at least six months with the intent to continue for the duration of the study.
• Patients who are available to attend on an outpatient basis for visits provided for in the protocol and can complete the data collection documents (and quality of life scale).
• Patients have given written informed consent.
• Patients with a health insurance coverage.

Exclusion criteria:

• Participants meeting the following criteria could not be included:
• Patients with known or suspected allergies to any ingredient of the food supplement (β-NMN, Isomalt, Magnesium stearate, microcrystalline cellulose).
• Patients who have consumed food supplements containing tryptophan, glutamine or vitamin B3 in various forms (nicotinic acid/niacin and nicotinamide) during the month before selection.
• Patients have a significant medical condition that required hospitalization (other than sickle cell crisis) within two months of the screening visit (Visit 0).
• Patients have serum albumin < 3.0 g/dl (< 30 g/L).
• Patients have been transfused and received any blood products within three months of the Screening Visit (Visit 0).
• Patients have been hospitalized for acute vaso-occlusive crisis within one month of the Screening Visit (Visit 0).
• Patient has clinically significant cardiovascular or liver disease, renal or lung insufficiency or lymphopenia (with clinically significant abnormal results on the screening bioassays: CBC, transaminases (AST, ALT, GGT, ALP), bilirubin, creatinine, CPK, ionogram, blood glucose, lipid profile).
• Patients with a diagnosed cancer in the past 2 years.
• Pregnant or lactating woman. Women of childbearing potential should have a negative serum or urine 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)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: NUV001; Daily supplementation with NUV001 1000 mg	Dietary supplement: NUV001; 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of adverse events and treatment-emergent adverse events through Day 120.	Participant incidence of AEs/TEAEs, including vaso-occlusive crises and SCD-related hospitalizations, from first dose through Day 120.	First dose through Day 120.
Clinically significant changes in laboratory safety parameters through Day 120.	Clinically significant changes from baseline through Day 120 in hematology, CRP, liver function tests, renal function tests, CPK, electrolytes, fasting glucose, and albumin.	Baseline through Day 120.
Clinically significant changes in vital signs through Day 120.	Clinically significant changes from baseline through Day 120 in systolic blood pressure, diastolic blood pressure, pulse rate, body temperature, and body weight if retained as part of tolerability assessment.	Baseline through Day 120.

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change from baseline in hematologic parameters through Day 120.	Change from baseline through Day 120 in Red Blood Cell count, Hemoglobin, Hematocrit, Mean Corpuscular Volume, Mean Corpuscular Hemoglobin, Mean Corpuscular Hemoglobin Concentration, and Reticulocyte count.	Baseline (Day 0), Day 15, Day 30, Day 60, Day 90, Day 120.
Change from baseline in markers of hemolysis through Day 120.	Change from baseline through Day 120 in Lactate DeHydrogenase and Bilirubin parameters as markers of hemolysis.	Baseline (Day 0), Day 15, Day 30, Day 60, Day 90, Day 120.
Change from baseline in Fetal Hemoglobin-related erythroid parameters through Day 120.	Change from baseline through Day 120 in percentage of F-cells, percentage of F-reticulocytes, and distribution of hemoglobin F expression within F-cells.	Baseline (Day 0), Day 15, Day 30, Day 60, Day 90, Day 120.
Change from baseline in sickling-related Red Blood Cell parameters through Day 120.	Change from baseline through Day 120 in percentage of irreversibly sickled cells and in vitro hypoxia-induced Red Blood Cell sickling.	Baseline (Day 0), Day 15, Day 30, Day 60, Day 90, Day 120.
Change from baseline in blood β-NMN and NAD+ concentrations through Day 120.	Change from baseline through Day 120 in whole blood β-Nicotinamide mononucleotide (β-NMN) and nicotinamide adenine dinucleotide (NAD+) concentrations.	Baseline (Day 0), Day 15, Day 30, Day 60, Day 90, Day 120.
Change from baseline in plasma NAD-related metabolites through Day 120.	Change from baseline through Day 120 in plasma nicotinamide (NAM) and 1-methylnicotinamide (MeNAM) concentrations.	Baseline (Day 0), Day 15, Day 30, Day 60, Day 90, Day 120.
Change from baseline in urinary NAD-related metabolites through Day 120.	Change from baseline through Day 120 in urinary 1-methylnicotinamide (MeNAM) and 2PY concentrations.	Baseline (Day 0), Day 15, Day 30, Day 60, Day 90, Day 120.
Change from baseline in health-related quality of life through Day 120.	Change from baseline through Day 120 in Short Form-36 (SF-36) questionnaire domain scores and summary scores.	Baseline (Day 0), Day 15, Day 30, Day 60, Day 90, and Day 120.
Change from baseline in pain outcomes through Day 120.	Change from baseline through Day 120 in pain severity and pain interference scores assessed using the Brief Pain Inventory questionnaire.	Baseline (Day 0), Day 15, Day 30, Day 60, Day 90, and Day 120
Change from baseline in analgesic use through Day 120.	Change from baseline through Day 120 in use of analgesic and opioid medications, assessed using recorded concomitant medication use and equianalgesic conversion where applicable.	Baseline (Day 0) through Day 120.

Collaborators and Investigators

• Sponsor: LGD
• Collaborators: Assistance Publique Hopitaux De Marseille; CEN Biotech; Etablissement Français du Sang
• Investigators: Study Director: Matthias CANAULT, PhD-HDR, LGD; Principal Investigator: Estelle JEAN, MD, Assistance Publique Hôpitaux Marseille

Study record dates

Study Major Dates

• Study Start (Actual): March 30, 2023
• Primary Completion (Actual): May 6, 2024
• Study Completion (Actual): May 6, 2024

Study Registration Dates

• First Submitted: February 28, 2023
• First Submitted That Met QC Criteria: March 28, 2023
• First Posted (Actual): March 29, 2023

Study Record Updates

• Last Update Posted (Actual): June 17, 2026
• Last Update Submitted That Met QC Criteria: June 15, 2026
• Last Verified: June 1, 2026

More Information

Terms related to this study

• Keywords: safety; tolerance; SS genotype of sickle cell disease
• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Hematologic Diseases; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hemoglobinopathies; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Hemic and Lymphatic Diseases; Anemia, Sickle Cell
• Other Study ID Numbers: Safety-DR-NUV001-SCD; 2022-A01570-43 (Other Identifier: ANSM)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No