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

NCT05791591 | Completed

• 2 other identifiers: LGD-NUV001-CT01-22LGD-CLI-006
• Study Type: interventional
• Target: 168
• Locations: 1 country
• Sites: 9

Brief Summary

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.

Conditions

Sickle Cell Disease

Keywords

Hb-SS; Hb S/β0-Thal

Outcome Measures

Primary Outcomes (3)

• Incidence of adverse events through Day 90.

  Subject incidence of treatment-emergent adverse events through the treatment period.

  Baseline to Day 90

• Change from baseline in hematologic and biochemical safety parameters at Day 90.

  Change from baseline to Day 90 in complete blood count, blood glucose, calcium, electrolytes, total protein, albumin, alkaline phosphatase, bilirubin, blood urea nitrogen, creatinine, AST, ALT, and estimated glomerular filtration rate (eGFR).

  Baseline and Day 90

• Change from baseline in vital signs at Day 90.

  Change from baseline to Day 90 in systolic and diastolic blood pressure, pulse rate, respiration rate, and body temperature.

  Baseline and Day 90.

Secondary Outcomes (11)

• Change in percentage of HbF-positive cells.

  Baseline, Day 30, Day 60, Day 90.

• Change in HbF content in Red Blood Cells.

  Baseline, Day 30, Day 60, Day 90.

• Change in percentage of circulating irreversibly sickled cells.

  Baseline, Day 30, Day 60, Day 90.

• Change in hematocrit.

  Baseline, Day 30, Day 60, Day 90.

• Change in indirect bilirubin level.

  Baseline, Day 30, Day 60, Day 90.

Study Arms (3)

NUV001 Immediate-Release (IR)

EXPERIMENTAL

Participants received oral NUV001 immediate-release formulation at a total daily dose of 1000 mg for 90 days, in addition to standard of care.

Dietary Supplement: NUV001 - IR

NUV001 Gastro-Resistant (GR)

EXPERIMENTAL

Participants received oral NUV001 gastro-resistant formulation at a total daily dose of 1000 mg for 90 days, in addition to standard of care.

Dietary Supplement: NUV001 - GR

Placebo

PLACEBO COMPARATOR

Participants received matching placebo for 90 days, in addition to standard of care.

Dietary Supplement: Placebo

Interventions

NUV001 - IR DIETARY_SUPPLEMENT

Daily supplementation with 1000 mg of NUV001 (in two administration orally) immediate release gel capsule formulation for 90 days in total.

NUV001 Immediate-Release (IR)

NUV001 - GR DIETARY_SUPPLEMENT

Daily supplementation with 1000 mg of NUV001 (in two administration orally) gastro resistant gel capsule formulation for 90 days in total.

NUV001 Gastro-Resistant (GR)

Placebo DIETARY_SUPPLEMENT

Placebo containing starch Powder (1000 mg, daily in two administration orally for 90 days).

Placebo

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Men or women over 18 to 65 years, both inclusive.
• Non-smokers.
• BMI \> 18 kg/m2
• 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).
• Haemoglobin levels between 5.5 and 10.5 g/dl during Screening (for newly diagnosed or patients not on any treatment for SCD).
• 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.
• 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)
• Patient or the patient's legally authorized representative has given written informed consent.

You may not qualify if:

• Patients with known or suspected allergy to any ingredient of the food supplement
• Patient having consumed vitamin or food supplements containing NAD+ precursors (niacin, tryptophan, nicotinamide, NMN, NR etc...) 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 prothrombin time INR \> 2.0.
• Patient has serum albumin less than 3.0 g/dl.
• Patient has received any blood products within three months of the Screening visit.
• Patients hospitalized for acute vaso-occlusive crisis within one month of the Screening visit.
• 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).
• Patient with diagnosed cancer in the past 2 years.
• Pregnant, lactating or parturient women.
• 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.
• Majors under legal protection or unable to express their consent.
• People in an emergency situation unable to express their prior consent.

Sponsors & Collaborators

• LGD: lead
• ProRelix Research: collaborator
• Nuvamid SA: collaborator

Study Sites (9)

Aman Hospital and Research Center

Vadodara, Gujarat, Vadodara-390021, India

Location

Kingsway Hospital

Nagpur, Maharashtra, Nagpur-440001, India

Location

Shivam Hospital

Ahmedabad, 380008, India

Location

Sai Krupa Hospital & Research Centre

Ahmedabad, India

Location

Thalassemia & Sickle Cell Society

Hyderabad, India

Location

Index Medical College

Indore, India

Location

NRSMC Hospital

Kolkata, India

Location

Arihant Multispeciality Hospital

Nagpur, India

Location

Shalinitai Meghe Hospital & Research Centre

Nagpur, India

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

  LGD

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 21, 2023
• First Posted: March 30, 2023
• Study Start: April 16, 2023
• Primary Completion: July 3, 2024
• Study Completion: July 3, 2024
• Last Updated: June 17, 2026

Record last verified: 2026-06

Data Sharing

• IPD Sharing: Will not share

Locations

IN (9)