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

N/AActive Not RecruitingNCT05791591

LGD168 enrolled

Overview

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 Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

OTHER

Masking

DOUBLE

Enrollment

168

Conditions

Sickle Cell Disease

Interventions

Eligibility

Sex: ALLMin age: 18 YearsMax age: 65 Years

Medical Language ↔ Plain English

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.

Locations (8)

Aman Hospital and Research Center

Vadodara, Gujarat, India

Kingsway Hospital

Nagpur, Maharashtra, India

Sai Krupa Hospital & Research Centre

Ahmedabad, India

Thalassemia & Sickle Cell Society

Hyderabad, India

Index Medical College

Indore, India

NRSMC Hospital

Kolkata, India

Arihant Hospital

Nagpur, India

Shalinitai Meghe Hospital & Research Centre

Nagpur, India

Outcomes

Primary Outcomes

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

Subject incidence of treatment-emergent adverse events

Time frame: between Day 0 and Day 30

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

Subject incidence of treatment-emergent adverse events

Time frame: between Day 0 and Day 60

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

Subject incidence of treatment-emergent adverse events

Time frame: 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)

Time frame: between Day 0 and Day 30

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

Time frame: between Day 0 and Day 60

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

Time frame: 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)

Time frame: between Day 0 and Day 30

Safety as measured by subject incident of treatment-emergent clinically significant changes in vital signs

Time frame: between Day 0 and Day 60

Safety as measured by subject incident of treatment-emergent clinically significant changes in vital signs

Time frame: between Day 0 and Day 90