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

NCT05789355 | Completed

• 2 other identifiers: Safety-DR-NUV001-SCD2022-A01570-43
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

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.

Conditions

Sickle Cell Disease

Keywords

safety; tolerance; SS genotype of sickle cell disease

Outcome Measures

Primary Outcomes (3)

• 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 Outcomes (10)

• Change from baseline in hematologic parameters through Day 120.

  Baseline (Day 0), Day 15, Day 30, Day 60, Day 90, Day 120.

• Change from baseline in markers of hemolysis through Day 120.

  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.

  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.

  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.

  Baseline (Day 0), Day 15, Day 30, Day 60, Day 90, Day 120.

Study Arms (1)

NUV001

EXPERIMENTAL

Daily supplementation with NUV001 1000 mg

Dietary Supplement: NUV001

Interventions

NUV001 DIETARY_SUPPLEMENT

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

NUV001

Eligibility Criteria

• Age: 18 Years - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• LGD: lead
• Assistance Publique Hopitaux De Marseille: collaborator
• Etablissement Français du Sang: collaborator
• CEN Biotech: collaborator

Study Sites (1)

Aphm Hopital La Timone Adultes Sce Medecine Interne (Umap)

Marseille, 13005, France

Location

MeSH Terms

Conditions

Anemia, Sickle Cell

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Matthias CANAULT, PhD-HDR

  LGD

  STUDY DIRECTOR

• Estelle JEAN, MD

  Assistance Publique Hopitaux Marseille

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Prospective single-arm, open-label and monocentric pilot study.

Study Record Dates

• First Submitted: February 28, 2023
• First Posted: March 29, 2023
• Study Start: March 30, 2023
• Primary Completion: May 6, 2024
• Study Completion: May 6, 2024
• Last Updated: June 17, 2026

Record last verified: 2026-06

Data Sharing

• IPD Sharing: Will not share

Locations

FR (1)