A monocentric, prospective, open-label pilot study will evaluate the safety, tolerability, and plasma and urine residual rate parameters of oral NUV001 in adult patients with sickle cell disease (SCD) S homozygous genotype. The study is not yet recruiting.
The researchers plan to enroll 12 adult patients with SCD aged 18 to 60 years. There will be at least 2 patients who have not received hydroxyurea treatment and a maximum of 10 patients who have previously been treated with hydroxyurea. Those who have received the medication within 6 months of the screening visit must maintain continuous and unmodified therapy for the duration of the study.
Furthermore, female patients of childbearing potential must use an acceptable method of birth control, including hormonal contraceptives or an intrauterine device.
Patients who have consumed food supplements containing tryptophan, glutamine, or vitamin B3 during the month before selection will not be included in the study, as well as those with allergies to any ingredient of the food supplement.
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The exclusion criteria also cite significant cardiovascular or liver disease, renal or lung insufficiency or lymphopenia, significant medical conditions that required hospitalization within 2 months of the screening visit or hospitalization for acute vaso-occlusive crisis within 1 month of the screening visit, transfusions within 3 months of the screening visit, a diagnosis of cancer in the past 2 years, and serum albumin levels lower than 3.0 g/dL.
According to the study protocol, the participants will receive active supplementation with 1000 mg/day (4 × 250 mg tablet) of NUV001 for 3 months of follow-up. The patients will be observed for another month after the supplementation period.
The researchers will monitor the incidence of treatment-emergent adverse events, significant changes in clinical laboratory safety tests, and vital signs changes during the first 30, 60, 90, and 120 days as the primary outcome measures.
The secondary outcome measures will include changes in the levels of lactate dehydrogenase, hemoglobin, F-hemoglobin, hematocrit, and reticulocytes, as well as changes in the circulating level of red line cell precursors, circulating irreversibly sickle cells, mean corpuscular volume, mean corpuscular hemoglobin content, red blood cell sickling, nicotinamide adenine dinucleotide and β-nicotinamide mononucleotide concentration in whole blood, methyl-nicotinamide and nicotinamide concentration in plasma, nicotinamide and N-methyl-2-pyridone-5-carboxamide concentration in urine, and baseline changes in self-questionnaire quality of life results.
The study is sponsored by LGD and is estimated to be completed by December 31, 2023.
Effect of NUV001 supplementation in patients suffering from sickle cell disease (SCD). ClinicalTrials.gov. March 29, 2023. Accessed April 20, 2023.