Safety and Efficacy of Orally Administered NUV001 Nutraceutical Supplement in Sickle Cell Disease Patients

A Multicenter, Randomized, Double Blind, Placebo Controlled Study to Evaluate Safety and Efficacy of Orally Administered NUV001 Nutraceutical Supplement in Sickle Cell Disease Patients.

This multicenter, randomized, double-blind, parallel-group, placebo-controlled pilot study evaluated the safety, tolerability, and exploratory efficacy of orally administered NUV001 in adult participants with sickle cell disease (HbSS or HbSβ0 genotypes). A total of 168 participants were randomized in a 1:1:1 ratio to receive NUV001 immediate-release (IR), NUV001 gastro-resistant (GR), or placebo, in addition to standard of care, for 90 days over 5 study visits. The primary objective was to assess safety and tolerability based on adverse events, clinical laboratory safety parameters, and vital signs. Exploratory secondary objectives evaluated hematologic, hemolysis, and patient-reported outcomes.

This was a multicenter, randomized, double-blind, parallel-group, placebo-controlled pilot study conducted in adult participants with sickle cell disease.

Participants were randomized in a 1:1:1 ratio to one of three treatment groups:

1. NUV001 immediate-release (IR)
2. NUV001 gastro-resistant (GR)
3. Matching placebo

All treatments were administered orally in addition to standard of care for 90 days.

The study was designed to evaluate safety and tolerability, including adverse events, clinical laboratory safety parameters, and vital signs. Exploratory objectives included evaluation of hematologic parameters, markers of hemolysis, and patient-reported outcomes related to pain and quality of life.

The study was conducted as a pilot, feasibility, and design-informing clinical study.

Inclusion Criteria:

1. Men or women over 18 to 65 years, both inclusive.
2. Non-smokers.
3. BMI > 18 kg/m2
4. Patients diagnosed with sickle cell disease (documented by haemoglobin electrophoresis) and carrying SS or Sbeta0 versions of the beta globin gene (documented by genotyping, known through medical history).
5. Haemoglobin levels between 5.5 and 10.5 g/dl during Screening (for newly diagnosed or patients not on any treatment for SCD).
6. If the patient has been treated with an anti-sickling agent within three months of the Screening visit, the therapy must have been continuous for at least three months with the intent to continue for the duration of the study.
7. Available to attend on an outpatient basis for visits provided for in the protocol and able to complete the data collection documents (compliance and quality of life scale)
8. Patient or the patient's legally authorized representative has given written informed consent.

Exclusion Criteria:

1. Patients with known or suspected allergy to any ingredient of the food supplement
2. Patient having consumed vitamin or food supplements containing NAD+ precursors (niacin, tryptophan, nicotinamide, NMN, NR etc...) during the month before selection.
3. Patient has a significant medical condition that required hospitalization (other than sickle cell crisis) within two months of the screening visit.
4. Patient has prothrombin time INR > 2.0.
5. Patient has serum albumin less than 3.0 g/dl.
6. Patient has received any blood products within three months of the Screening visit.
7. Patients hospitalized for acute vaso-occlusive crisis within one month of the Screening visit.
8. Patient has clinically significant, cardiovascular or liver disease or renal insufficiency or lymphopenia , evident in medical history (with clinically significant abnormal results on the Screening bioassays for eg.: Complete blood count, Aspartate transaminases, Alanine transaminases, Gamma glutamyl transferase, Alkaline Phosphatase, Bilirubin, Creatinine, Creatinine Phosphokinase, Blood Glucose, HbA1c, Lipid Profile).
9. Patient with diagnosed cancer in the past 2 years.
10. Patients participating simultaneously in another clinical research protocol or having recently participated in another research for which the exclusion period has not been completed.
11. Pregnant, lactating or parturient women.
12. Persons deprived of their liberty by a judicial or administrative decision, hospitalized without consent or admitted to a health or social establishment for purposes other than that of research.
13. Majors under legal protection or unable to express their consent.
14. People in an emergency situation unable to express their prior consent.