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.

Study Overview

• Status: Completed
• Conditions: Sickle Cell Disease
• Intervention / Treatment: Dietary supplement: NUV001 - IR; Dietary supplement: NUV001 - GR; Dietary supplement: Placebo

Detailed Description

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.

• Study Type: Interventional
• Enrollment (Actual): 168
• Phase: Not Applicable

Contacts and Locations

Study Locations

• India
  • Ahmedabad, India
    • Sai Krupa Hospital & Research Centre
  • Ahmedabad, India, 380008
    • Shivam Hospital
  • Hyderabad, India
    • Thalassemia & Sickle Cell Society
  • Indore, India
    • Index Medical College
  • Kolkata, India
    • NRSMC Hospital
  • Nagpur, India
    • Shalinitai Meghe Hospital & Research Centre
  • Nagpur, India
    • Arihant Multispeciality Hospital
  • Gujarat
    • Vadodara, Gujarat, India, Vadodara-390021
      • Aman Hospital and Research Center
  • Maharashtra
    • Nagpur, Maharashtra, India, Nagpur-440001
      • Kingsway Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 65 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Double

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: NUV001 Immediate-Release (IR); 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; Daily supplementation with 1000 mg of NUV001 (in two administration orally) immediate release gel capsule formulation for 90 days in total.
Experimental: NUV001 Gastro-Resistant (GR); 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; Daily supplementation with 1000 mg of NUV001 (in two administration orally) gastro resistant gel capsule formulation for 90 days in total.
Placebo Comparator: Placebo; Participants received matching placebo for 90 days, in addition to standard of care.	Dietary supplement: Placebo; Placebo containing starch Powder (1000 mg, daily in two administration orally for 90 days).

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.
Change in reticulocyte count.		Baseline, Day 30, Day 60, Day 90.
Change in serum lactate dehydrogenase level.		Baseline, Day 30, Day 60, Day 90.
Change in ASCQ-Me Questionnaire (Adult Sickle Cell Quality of Life Measurement Information System)	Questionnaire on acute and/or chronic pain, energy level, usage of pain medications and activity levels.	Baseline, Day 30, Day 60, Day 90.
Change in pain intensity score.	Evaluation of pain intensity for each body location (using a numeric pain rating scale from 0, no pain to 10 worst possible pain)	Baseline, Day 30, Day 60, Day 90.
Change in pain relief score.	Evaluation and evaluation of pain relief (pain relief scale in percent from 0%, no relief to 100% complete relief)	Baseline, Day 30, Day 60, Day 90.
Occurrence of vaso-occlusive crises during the study		Day 0 to Day 90.

Collaborators and Investigators

• Sponsor: LGD
• Collaborators: Nuvamid SA; ProRelix Research
• Investigators: Study Director: Matthias CANAULT, PhD, LGD

Study record dates

Study Major Dates

• Study Start (Actual): April 16, 2023
• Primary Completion (Actual): July 3, 2024
• Study Completion (Actual): July 3, 2024

Study Registration Dates

• First Submitted: February 21, 2023
• First Submitted That Met QC Criteria: March 17, 2023
• First Posted (Actual): March 30, 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: Hb-SS; Hb S/β0-Thal
• 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: LGD-NUV001-CT01-22; LGD-CLI-006 (Other Identifier: LGD)

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