Health Condition 1: D570- Hb-SS disease with crisis
Conditions
Interventions
Intervention1: NUV001: NUV001 (1000 mg, daily in two administration orally for 90 days) Immediate
release IR gel capsule formulation
Intervention2: NUV001: NUV001 (1000 mg, daily in two administratio
Sponsors
LGD SARLNUVAMID
Eligibility
Inclusion 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
Exclusion criteria: 1.Patients with known or suspected allergy to any ingredient of the food supplement (NMN, Isomalt, Magnesium stearate etc.) 2.Patient having consumed vitamin or food supplements containing niacin, tryptophan, NMN or NR 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 1.Adverse events 2.Change routine laboratory parameters from Baseline to Final visit Complete Blood Count (Hemoglobin, Hematocrit, RBC count, Mean cell hemoglobin, Mean cell hemoglobin concentration, Mean cell volume, Platelet count, White blood cell count) Composite Metabolic Panel – Random blood sugar, Serum Calcium, Serum Electrolytes, Serum Protein, Serum Albumin, Serum Alkaline Phosphatase, Serum Bilirubin, Blood urea nitrogen and Serum Creatinine. Liver Function Tests – Aspartate Aminotransferase, Alanine Aminotransferase (Other Liver Function Tests are already covered under Composite Metabolic Panel). Kidney Function Tests – eGFR (Other Kidney Function Tests are already covered under Composite Metabolic Panel) 3.Vital Signs (Blood Pressure, Pulse Rate, Respiration Rate and Body temperature) Timepoint: Day 0, Day 30, Day 60, Day 90. | — |
Secondary
| Measure | Time frame |
|---|---|
| 1.Increase in the % of F-hemoglobin positive cells 2.Increased F-Hb content in RBCs 3.Reduced RBC sickling 4.Reduced reticulocyte level 5.Increased hematocrit 6.Pain reduction 7.Reduction of the vaso-occlusive crisis occurrence 8.Change in laboratory markers associated with hemolysis a.indirect bilirubin level, b.absolute reticulocyte count and percentage of reticulocytes c.Serum lactate dehydrogenase 9.Questionnaire on acute and/or chronic pain, energy level, usage of narcotics, and activity levels throughout the study.Timepoint: Day 0, Day 30, Day 60, Day 90. | — |
Countries
India
Contacts
Public ContactMr Sohal Pendse
ProRelix Services LLP
Outcome results
None listed