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

NCT05789355 | Active Not Recruiting

• 1 other identifier: Safety-DRNUV001- SCD
• 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 (12)

• 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.

  between Day 0 and Day 30

• 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.

  between Day 0 and Day 60

• 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.

  between Day 0 and Day 90

• 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.

  between Day 0 and Day 120

• 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)

  between Day 0 and Day 30

• 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)

  between Day 0 and Day 60

• 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)

  between Day 0 and Day 90

• 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)

  between Day 0 and Day 120

• 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)

  between Day 0 and Day 30

• 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)

  between Day 0 and Day 60

• 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)

  between Day 0 and Day 90

• 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)

  between Day 0 and Day 120

Secondary Outcomes (17)

• Change in Lactate dehydrogenase (LDH) levels

  Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.

• Change in Hemoglobin (HGB) levels

  Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.

• Change in Hematocrit (HCT)

  Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.

• Change in circulating level of red line cell precursors

  Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.

• Change in mean corpuscular volume (MCV)

  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:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

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

• Laurent LAGANIER

  LGD SARL

  STUDY DIRECTOR

• Estelle JEAN-MIGNARD

  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: Single-center, prospective, open-label pilot study.

Study Record Dates

• First Submitted: February 28, 2023
• First Posted: March 29, 2023
• Study Start: April 1, 2023
• Primary Completion: May 10, 2024
• Study Completion: June 30, 2024
• Last Updated: May 28, 2024

Record last verified: 2024-05

Data Sharing

• IPD Sharing: Will not share

Locations

FR (1)