Safety and Efficacy of Orally Administered NUV001 Nutraceutical Supplement in Sickle Cell Disease Patients
Study Details
Study Description
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
N/A |
Study Design
Arms and Interventions
| 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)
|
Outcome Measures
Primary Outcome Measures
- Safety as measured by subject incident of treatment-emergent adverse events [between Day 0 and Day 30]
Subject incidence of treatment-emergent adverse events
- Safety as measured by subject incident of treatment-emergent adverse events [between Day 0 and Day 60]
Subject incidence of treatment-emergent adverse events
- Safety as measured by subject incident of treatment-emergent adverse events [between Day 0 and Day 90]
Subject incidence of treatment-emergent adverse events
- Safety as measured by subject incident of treatment-emergent clinically significant changes in clinical laboratory safety tests [between Day 0 and Day 30]
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)
- Safety as measured by subject incident of treatment-emergent clinically significant changes in clinical laboratory safety tests [between Day 0 and Day 60]
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)
- Safety as measured by subject incident of treatment-emergent clinically significant changes in clinical laboratory safety tests [between Day 0 and Day 90]
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)
- Safety as measured by subject incident of treatment-emergent clinically significant changes in vital signs [between Day 0 and Day 30]
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)
- Safety as measured by subject incident of treatment-emergent clinically significant changes in vital signs [between Day 0 and Day 60]
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)
- Safety as measured by subject incident of treatment-emergent clinically significant changes in vital signs [between Day 0 and Day 90]
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)
Secondary Outcome Measures
- 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]
% of total hemoglobin measured by HP-LC
- Change in RBC sickling [Day 0, Day 30, Day 60, Day 90]
% of circulating irreversibly sickled cells
- Change in hematocrit [Day 0, Day 30, Day 60, Day 90]
% of RBC in blood
- Change in indirect bilirubin level [Day 0, Day 30, Day 60, Day 90]
Indirect bilirubin level expressed in mg/dL
- Change in reticulocyte level [Day 0, Day 30, Day 60, Day 90]
reticulocytes count expressed in percentage of red blood cells
- Change in serum lactate dehydrogenase level [Day 0, Day 30, Day 60, Day 90]
Serum lactate dehydrogenase expressed in international units per liter (IU/L)
- ASCQ-Me Questionnaire (Adult Sickle Cell Quality of Life Measurement Information System) [Day 0, Day 30, Day 60, Day 90]
Questionnaire on acute and/or chronic pain, energy level, usage of pain medications and activity levels
- Change in pain perception [Day 0, Day 30, Day 60, Day 90]
Evaluation of pain intensity for each body location (using a numeric pain rating scale from 0, no pain to 10 worst possible pain)
- Pain relief assessment [Day 0, Day 30, Day 60, Day 90]
Evaluation and evaluation of pain relief (pain relief scale in percent from 0%, no relief to 100% complete relief)
Eligibility Criteria
Criteria
Inclusion Criteria:
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Men or women over 18 to 65 years, both inclusive.
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Non-smokers.
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BMI > 18 kg/m2
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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).
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Haemoglobin levels between 5.5 and 10.5 g/dl during Screening (for newly diagnosed or patients not on any treatment for SCD).
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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.
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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)
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Patient or the patient's legally authorized representative has given written informed consent.
Exclusion Criteria:
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Patients with known or suspected allergy to any ingredient of the food supplement
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Patient having consumed vitamin or food supplements containing NAD+ precursors (niacin, tryptophan, nicotinamide, NMN, NR etc...) during the month before selection.
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Patient has a significant medical condition that required hospitalization (other than sickle cell crisis) within two months of the screening visit.
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Patient has prothrombin time INR > 2.0.
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Patient has serum albumin less than 3.0 g/dl.
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Patient has received any blood products within three months of the Screening visit.
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Patients hospitalized for acute vaso-occlusive crisis within one month of the Screening visit.
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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).
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Patient with diagnosed cancer in the past 2 years.
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Patients participating simultaneously in another clinical research protocol or having recently participated in another research for which the exclusion period has not been completed.
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Pregnant, lactating or parturient women.
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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.
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Majors under legal protection or unable to express their consent.
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People in an emergency situation unable to express their prior consent.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Aman Hospital and Research Center | Vadodara | Gujarat | India | Vadodara-390021 |
| 2 | Kingsway Hospital | Nagpur | Maharashtra | India | Nagpur-440001 |
| 3 | Shree Samarth Hospital | Pune | Maharashtra | India | Pune-411011 |
Sponsors and Collaborators
- LGD
- ProRelix Services LLP
Investigators
- Study Director: Matthias Canault, PhD, LGD
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- LGD-NUV001-CT01-22
- LGD-CLI-006