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

NCT05791591 | Active Not Recruiting

• 2 other identifiers: LGD-NUV001-CT01-22LGD-CLI-006
• Study Type: interventional
• Target: 168
• Locations: 1 country
• Sites: 8

Brief Summary

A total of 170 patients male or female who are carrying SS or Sbeta0 versions of the beta globin gene will be included in the study. The subjects will be assigned with 1:1:1 ratio of either NUV001 Immediate release IR or NUV001 Gastro resistant GR or Placebo. The treatment duration of the study will be 90 days which has in total 5 visits. The primary end point of this study is to check the safety and tolerance of the orally administered nutraceutical supplement. This endpoint will be checked by assessing the Adverse events, Vital signs of the subject and the Change in hematological parameters from Baseline to Final visit.

Conditions

Sickle Cell Disease

Keywords

Hb-SS; Hb S/β0-Thal

Outcome Measures

Primary Outcomes (9)

• Safety as measured by subject incident of treatment-emergent adverse events

  Subject incidence of treatment-emergent adverse events

  between Day 0 and Day 30

• Safety as measured by subject incident of treatment-emergent adverse events

  Subject incidence of treatment-emergent adverse events

  between Day 0 and Day 60

• Safety as measured by subject incident of treatment-emergent adverse events

  Subject incidence of treatment-emergent adverse events

  between Day 0 and Day 90

• Safety as measured by subject incident of treatment-emergent clinically significant changes in clinical laboratory safety tests

  Subject incidence of treatment-emergent clinically significant changes in clinical laboratory safety tests (Complete Blood Count (absolute counts and %), Random blood glucose concentration and Serum concentrations in Calcium, Electrolytes, Protein, Albumin, Alkaline Phosphatase, Bilirubin, Blood urea nitrogen, Creatinine, Aspartate Aminotransferase, Alanine Aminotransferase)

  between Day 0 and Day 30

• Safety as measured by subject incident of treatment-emergent clinically significant changes in clinical laboratory safety tests

  Subject incidence of treatment-emergent clinically significant changes in clinical laboratory safety tests (Complete Blood Count (absolute counts and %), Random blood glucose concentration and Serum concentrations in Calcium, Electrolytes, Protein, Albumin, Alkaline Phosphatase, Bilirubin, Blood urea nitrogen, Creatinine, Aspartate Aminotransferase, Alanine Aminotransferase)

  between Day 0 and Day 60

• Safety as measured by subject incident of treatment-emergent clinically significant changes in clinical laboratory safety tests

  Subject incidence of treatment-emergent clinically significant changes in clinical laboratory safety tests (Complete Blood Count (absolute counts and %), Random blood glucose concentration and Serum concentrations in Calcium, Electrolytes, Protein, Albumin, Alkaline Phosphatase, Bilirubin, Blood urea nitrogen, Creatinine, Aspartate Aminotransferase, Alanine Aminotransferase)

  between Day 0 and Day 90

• Safety as measured by subject incident 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 in millimeters of mercury (mmHg), Pulse Rate in beats per minute (bpm), Respiration Rate in number of breaths per minute and Body temperature in Celsius)

  between Day 0 and Day 30

• Safety as measured by subject incident 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 in millimeters of mercury (mmHg), Pulse Rate in beats per minute (bpm), Respiration Rate in number of breaths per minute and Body temperature in Celsius)

  between Day 0 and Day 60

• Safety as measured by subject incident 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 in millimeters of mercury (mmHg), Pulse Rate in beats per minute (bpm), Respiration Rate in number of breaths per minute and Body temperature in Celsius)

  between Day 0 and Day 90

Secondary Outcomes (10)

• Change in the % of F-hemoglobin positive cells

  Day 0, Day 30, Day 60, Day 90

• Change in F-Hb content in RBCs

  Day 0, Day 30, Day 60, Day 90

• Change in RBC sickling

  Day 0, Day 30, Day 60, Day 90

• Change in hematocrit

  Day 0, Day 30, Day 60, Day 90

• Change in indirect bilirubin level

  Day 0, Day 30, Day 60, Day 90

Study Arms (3)

NUV001 - IR

EXPERIMENTAL

Sickle cell disease patients receiving NUV001 Immediate release gel capsule formulation

Dietary Supplement: NUV001 - IR

NUV001 - GR

EXPERIMENTAL

Sickle cell disease patients receiving NUV001 Gastro resistant gel capsule formulation

Dietary Supplement: NUV001 - GR

Placebo

PLACEBO COMPARATOR

Sickle cell disease patients receiving Placebo

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 - 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 - 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

Study Sites (8)

Aman Hospital and Research Center

Vadodara, Gujarat, Vadodara-390021, India

Location

Kingsway Hospital

Nagpur, Maharashtra, Nagpur-440001, 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 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: OTHER
• 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 15, 2023
• Primary Completion: October 31, 2024
• Study Completion: November 30, 2024
• Last Updated: May 28, 2024

Record last verified: 2024-05

Data Sharing

• IPD Sharing: Will not share

Locations

IN (8)