Clinical trial NCT05791591: Safety and Efficacy of Orally Administered NUV001 Nutraceutical Supplement in Sickle Cell Disease Patients
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.
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: NA
Conditions: Sickle Cell Disease
Keywords: Hb-SS
Hb S/β0-Thal
Lead sponsor: LGD
Sponsor class: INDUSTRY
Collaborators: name: ProRelix Research
First posted: 2023-03-30
Enrollment: 168
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: OTHER

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.


Eligibility criteria: 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.

Primary outcomes:
measure: Safety as measured by subject incident of treatment-emergent adverse events
timeFrame: between Day 0 and Day 30
measure: Safety as measured by subject incident of treatment-emergent adverse events
timeFrame: between Day 0 and Day 60
measure: Safety as measured by subject incident of treatment-emergent adverse events
timeFrame: between Day 0 and Day 90
measure: Safety as measured by subject incident of treatment-emergent clinically significant changes in clinical laboratory safety tests
timeFrame: between Day 0 and Day 30
measure: Safety as measured by subject incident of treatment-emergent clinically significant changes in clinical laboratory safety tests
timeFrame: between Day 0 and Day 60
measure: Safety as measured by subject incident of treatment-emergent clinically significant changes in clinical laboratory safety tests
timeFrame: between Day 0 and Day 90
measure: Safety as measured by subject incident of treatment-emergent clinically significant changes in vital signs
timeFrame: between Day 0 and Day 30
measure: Safety as measured by subject incident of treatment-emergent clinically significant changes in vital signs
timeFrame: between Day 0 and Day 60
measure: Safety as measured by subject incident of treatment-emergent clinically significant changes in vital signs
timeFrame: between Day 0 and Day 90
Secondary outcomes:
measure: Change in the % of F-hemoglobin positive cells
timeFrame: Day 0, Day 30, Day 60, Day 90
measure: Change in F-Hb content in RBCs
timeFrame: Day 0, Day 30, Day 60, Day 90
measure: Change in RBC sickling
timeFrame: Day 0, Day 30, Day 60, Day 90
measure: Change in hematocrit
timeFrame: Day 0, Day 30, Day 60, Day 90
measure: Change in indirect bilirubin level
timeFrame: Day 0, Day 30, Day 60, Day 90
measure: Change in reticulocyte level
timeFrame: Day 0, Day 30, Day 60, Day 90
measure: Change in serum lactate dehydrogenase level
timeFrame: Day 0, Day 30, Day 60, Day 90
measure: ASCQ-Me Questionnaire (Adult Sickle Cell Quality of Life Measurement Information System)
timeFrame: Day 0, Day 30, Day 60, Day 90
measure: Change in pain perception
timeFrame: Day 0, Day 30, Day 60, Day 90
measure: Pain relief assessment
timeFrame: Day 0, Day 30, Day 60, Day 90
Other outcomes:
Locations:
city: Vadodara
country: India
facility: Aman Hospital and Research Center
state: Gujarat
city: Nagpur
country: India
facility: Kingsway Hospital
state: Maharashtra
city: Ahmedabad
country: India
facility: Sai Krupa Hospital & Research Centre
city: Hyderabad
country: India
facility: Thalassemia & Sickle Cell Society
city: Indore
country: India
facility: Index Medical College
city: Kolkata
country: India
facility: NRSMC Hospital
city: Nagpur
country: India
facility: Arihant Hospital
city: Nagpur
country: India
facility: Shalinitai Meghe Hospital & Research Centre
Study officials:
affiliation: LGD
name: Matthias Canault, PhD
role: STUDY_DIRECTOR
References: