EFFECT OF NUV001 SUPPLEMENTATION FOR 120 DAYS IN PATIENTS SUFFERING FROM SICKLE CELL DISEASE (SCD) SS GENOTYPE: A PILOT STUDY
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 12
- 试验地点
- 2
- 主要终点
- Incidence of treatment-emergent clinically significant changes in clinical laboratory safety tests
研究概览
简要总结
This is a pilot study of daily dosing of NUV001 as a dietary supplement in 12 sickle cell disease patients with 3 months of follow-up plus 1 month after supplementation.The present study is designed to evaluate, first, the safety and tolerability parameters as well as to measure the plasma and urinary residues of daily oral doses of NUV001. Secondly, the study will evaluate the impact of NUV001 on biological parameters and quality of life of patients.
详细描述
This is a monocentric, prospective, open-label pilot study designed for 12 adult patients suffering of Sickle Cell disease (SCD) SS genotype each 12 receiving the active supplementation of NUV001, 1000mg/day (4 x 250 mg tablet) for 3 months of follow-up plus 1 month after supplementation. A stratification according to the medical treatment is planned. At least 2 patients suffering of SCD SS genotype without hydroxyurea treatment and maximum 10 patients suffering of SCD SS genotype in association with hydroxyurea treatment. If a subject is withdrawn from this study part, the subject may be replaced as necessary with another subject assigned to the same treatment at the discretion of the sponsor's team in consultation with the investigator.
The current study is designed to assess in the first part, the safety, tolerability, plasma, and urine residual rate parameters of daily oral doses of NUV001 as food supplement in adult patients with SCD, SS genotype. In a second part, the study will assess the pharmacological impact of NUV001 on biological parameters and the quality of life in patient suffering of sickle cell disease SS genotype.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 60 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants meeting the following criteria could be included:
- •Male or female between 18 and 60 years old.
- •Females of childbearing potential should be using one of the following acceptable methods of birth control:
- •Intrauterine Device in place for at least 60 days prior to the first dose of the study (Visit 1) throughout the study and for 30 days after completion of the study.
- •Hormonal contraceptives for at least 90 days prior to the first dose of the study (Visit 1) throughout the study, and for 30 days after study completion.
- •Patients whose weight is greater than 50 kg.
- •Patients diagnosed with homozygous sickle cell anemia of SS genotype (documented by genotyping).
- •Patients who have been treated with an anti-sickling agent (Siklos®) within six months of the screening visit (Visit 0) must maintain the therapy continuous and unmodified for at least six months with the intent to continue for the duration of the study.
- •Patients who are available to attend on an outpatient basis for visits provided for in the protocol and can complete the data collection documents (and quality of life scale).
- •Patients have given written informed consent.
- •Patients with a health insurance coverage.
排除标准
- •Participants meeting the following criteria could not be included:
- •Patients with known or suspected allergies to any ingredient of the food supplement (β-NMN, Isomalt, Magnesium stearate, microcrystalline cellulose).
- •Patients who have consumed food supplements containing tryptophan, glutamine or vitamin B3 in various forms (nicotinic acid/niacin and nicotinamide) during the month before selection.
- •Patients have a significant medical condition that required hospitalization (other than sickle cell crisis) within two months of the screening visit (Visit 0).
- •Patients have serum albumin < 3.0 g/dl (< 30 g/L).
- •Patients have been transfused and received any blood products within three months of the Screening Visit (Visit 0).
- •Patients have been hospitalized for acute vaso-occlusive crisis within one month of the Screening Visit (Visit 0).
- •Patient has clinically significant cardiovascular or liver disease, renal or lung insufficiency or lymphopenia (with clinically significant abnormal results on the screening bioassays: CBC, transaminases (AST, ALT, GGT, ALP), bilirubin, creatinine, CPK, ionogram, blood glucose, lipid profile).
- •Patients with a diagnosed cancer in the past 2 years.
- •Pregnant or lactating woman. Women of childbearing potential should have a negative serum or urine pregnancy test at screening and a negative urine pregnancy test at inclusion prior to administration of the study product.
研究组 & 干预措施
NUV001
Daily supplementation with NUV001 1000 mg
干预措施: NUV001 (Dietary Supplement)
结局指标
主要结局
Incidence of treatment-emergent clinically significant changes in clinical laboratory safety tests
时间窗: between Day 0 and Day 120
Subject incidence of treatment-emergent clinically significant changes in clinical laboratory safety tests (Blood formulation, Inflammation parameters, Conjugated and free Bilirubin, alanine and aspartate aminotransferases (ASAT, ALAT),gamma-glutamyltransferase (GGT) and alkaline phosphatase (ALP), Creatine phosphokinase (CPK), Creatine, Urea, Creatinuria, Proteinuria, creatinine clearance)Ionogram Na/K/Cl, fasting blood glucose, albumine)
Incidence of treatment-emergent adverse events
时间窗: between Day 0 and Day 120
Subject incidence of treatment-emergent Adverse events (AEs) and Severe adverse events. Including Hospitalization days and Vaso-occlusive crisis occurrences.
Incidence of treatment-emergent clinically significant changes in Vital Signs
时间窗: between Day 0 and Day 120
Subject incidence of treatment-emergent clinically significant changes in vital signs (systolic and diastolic blood Pressure, Pulse Rate and Body temperature)
次要结局
- Nicotinamide adenine dinucleotide (NAD)+ concentration in whole blood(Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.)
- F-Hemoglobin(Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.)
- Circulating irreversibly sickle cells (ISCs)(Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.)
- Red blood cells (RBC) sickling(Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.)
- Change in reticulocyte level(Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.)
- Methyl-Nicotinamide (Me-NAM) concentration in plasma(Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.)
- Change in Lactate dehydrogenase (LDH) levels(Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.)
- Change in Hemoglobin (HGB) levels(Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.)
- Change in circulating level of red line cell precursors(Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.)
- β-Nicotinamide mononucleotide (NMN) concentration in whole blood(Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.)
- Change in mean corpuscular volume (MCV)(Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.)
- Change in mean corpuscular hemoglobin content (MCHT)(Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.)
- Change in Hematocrit (HCT)(Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.)
- Nicotinamide (NAM) concentration in plasma(Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.)
- Change from baseline of self-questionnaire quality of life 36-Item Short Form Survey (SF-36) version 1(Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.)
- Nicotinamide (NAM) concentration in urine(Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.)
- N-Methyl-2-pyridone-5-carboxamide (2PY) concentration in urine(Day 0, Day 15, Day 30, Day 60, Day 90, Day 120.)