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.

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.

Study Overview

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

• Study Type: Interventional
• Enrollment (Estimated): 170
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Aditi Vaidya; Phone Number: +91 8602571013; Email: a.vaidya@prorelixresearch.com

Study Locations

• India
  • Ahmedabad, India
    • Recruiting
    • Sai Krupa Hospital & Research Centre
    • Contact: Manish Hathila, MD
  • Hyderabad, India
    • Recruiting
    • Thalassemia & Sickle Cell Society
    • Contact: Suman Jain, MD
  • Indore, India
    • Recruiting
    • Index Medical College
    • Contact: Sudhir Mourya, MD
  • Kolkata, India
    • Recruiting
    • NRSMC Hospital
    • Contact: Kanti Dolai Tuphan, MD
  • Nagpur, India
    • Recruiting
    • Arihant Hospital
    • Contact: Shweta Bhandarkar, MD
  • Nagpur, India
    • Recruiting
    • Shalinitai Meghe Hospital & Research Centre
    • Contact: Ankita Kapse, MD
  • Gujarat
    • Vadodara, Gujarat, India, Vadodara-390021
      • Recruiting
      • Aman Hospital and Research Center
      • Contact: Aman Khanna, MD; Phone Number: 9904402122; Email: amankhanna1974@gmail.com
  • Maharashtra
    • Nagpur, Maharashtra, India, Nagpur-440001
      • Recruiting
      • Kingsway Hospital
      • Contact: Riya Ballikar, MD
    • Pune, Maharashtra, India, Pune-411011
      • Withdrawn
      • Shree Samarth 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: Other
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Double

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: NUV001 - IR; Sickle cell disease patients receiving NUV001 Immediate release gel capsule formulation	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 - GR; Sickle cell disease patients receiving NUV001 Gastro resistant gel capsule formulation	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; Sickle cell disease patients receiving Placebo	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
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in the % of F-hemoglobin positive cells		Day 0, Day 30, Day 60, Day 90
Change in F-Hb content in RBCs	% of total hemoglobin measured by HP-LC	Day 0, Day 30, Day 60, Day 90
Change in RBC sickling	% of circulating irreversibly sickled cells	Day 0, Day 30, Day 60, Day 90
Change in hematocrit	% of RBC in blood	Day 0, Day 30, Day 60, Day 90
Change in indirect bilirubin level	Indirect bilirubin level expressed in mg/dL	Day 0, Day 30, Day 60, Day 90
Change in reticulocyte level	reticulocytes count expressed in percentage of red blood cells	Day 0, Day 30, Day 60, Day 90
Change in serum lactate dehydrogenase level	Serum lactate dehydrogenase expressed in international units per liter (IU/L)	Day 0, Day 30, Day 60, Day 90
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	Day 0, Day 30, Day 60, Day 90
Change in pain perception	Evaluation of pain intensity for each body location (using a numeric pain rating scale from 0, no pain to 10 worst possible pain)	Day 0, Day 30, Day 60, Day 90
Pain relief assessment	Evaluation and evaluation of pain relief (pain relief scale in percent from 0%, no relief to 100% complete relief)	Day 0, Day 30, Day 60, Day 90

Collaborators and Investigators

• Sponsor: LGD
• Collaborators: ProRelix Services LLP
• Investigators: Study Director: Matthias Canault, PhD, LGD

Study record dates

Study Major Dates

• Study Start (Actual): April 15, 2023
• Primary Completion (Estimated): May 31, 2024
• Study Completion (Estimated): May 31, 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 (Estimated): January 17, 2024
• Last Update Submitted That Met QC Criteria: January 15, 2024
• Last Verified: January 1, 2024

More Information

Terms related to this study

• Keywords: Hb-SS; Hb S/β0-Thal
• Additional Relevant MeSH Terms: Hematologic Diseases; Genetic Diseases, Inborn; Anemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; 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