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

Sickle Cell Disease Clinical Trial

Official title:

A Multicenter, Randomized, Double Blind, Placebo Controlled Study to Evaluate Safety and Efficacy of Orally Administered NUV001 Nutraceutical Supplement in Sickle Cell Disease Patients.

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT05791591
• Other study ID #: LGD-NUV001-CT01-22
• Secondary ID: LGD-CLI-006
• Status: Active, not recruiting
• Phase: N/A
• Start date: April 15, 2023
• Est. completion date: November 30, 2024

Study information

• Verified date: May 2024
• Source: LGD
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 168
• Est. completion date: November 30, 2024
• Est. primary completion date: October 31, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 65 Years

Eligibility

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.

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• Sickle Cell Disease

Intervention

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
• NUV001 - GR
Daily supplementation with 1000 mg of NUV001 (in two administration orally) gastro resistant gel capsule formulation for 90 days in total
• Placebo
Placebo containing starch Powder (1000 mg, daily in two administration orally for 90 days)

Locations

Country	Name	City	State
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	Kingsway Hospital	Nagpur	Maharashtra
India	Shalinitai Meghe Hospital & Research Centre	Nagpur
India	Aman Hospital and Research Center	Vadodara	Gujarat

Sponsors (2)

Lead Sponsor	Collaborator
LGD	ProRelix Services LLP

Country where clinical trial is conducted

India, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety as measured by subject incident of treatment-emergent adverse events	Subject incidence of treatment-emergent adverse events	between Day 0 and Day 30
Primary	Safety as measured by subject incident of treatment-emergent adverse events	Subject incidence of treatment-emergent adverse events	between Day 0 and Day 60
Primary	Safety as measured by subject incident of treatment-emergent adverse events	Subject incidence of treatment-emergent adverse events	between Day 0 and Day 90
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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	Change in the % of F-hemoglobin positive cells		Day 0, Day 30, Day 60, Day 90
Secondary	Change in F-Hb content in RBCs	% of total hemoglobin measured by HP-LC	Day 0, Day 30, Day 60, Day 90
Secondary	Change in RBC sickling	% of circulating irreversibly sickled cells	Day 0, Day 30, Day 60, Day 90
Secondary	Change in hematocrit	% of RBC in blood	Day 0, Day 30, Day 60, Day 90
Secondary	Change in indirect bilirubin level	Indirect bilirubin level expressed in mg/dL	Day 0, Day 30, Day 60, Day 90
Secondary	Change in reticulocyte level	reticulocytes count expressed in percentage of red blood cells	Day 0, Day 30, Day 60, Day 90
Secondary	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
Secondary	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
Secondary	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
Secondary	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