A Multicenter, Randomized, Double Blind, Placebo Controlled Study to Evaluate Safety and Efficacy of Orally Administered NUV001 Nutraceutical Supplement in Sickle Cell Disease Patients.
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 168
- 试验地点
- 12
- 主要终点
- Safety as measured by subject incident of treatment-emergent adverse events
研究概览
简要总结
This multicenter, randomized, double-blind, parallel-group, placebo-controlled pilot study evaluated the safety, tolerability, and exploratory efficacy of orally administered NUV001 in adult participants with sickle cell disease (HbSS or HbSβ0 genotypes). A total of 168 participants were randomized in a 1:1:1 ratio to receive NUV001 immediate-release (IR), NUV001 gastro-resistant (GR), or placebo, in addition to standard of care, for 90 days over 5 study visits. The primary objective was to assess safety and tolerability based on adverse events, clinical laboratory safety parameters, and vital signs. Exploratory secondary objectives evaluated hematologic, hemolysis, and patient-reported outcomes.
详细描述
This was a multicenter, randomized, double-blind, parallel-group, placebo-controlled pilot study conducted in adult participants with sickle cell disease.
Participants were randomized in a 1:1:1 ratio to one of three treatment groups:
- NUV001 immediate-release (IR)
- NUV001 gastro-resistant (GR)
- Matching placebo
All treatments were administered orally in addition to standard of care for 90 days.
The study was designed to evaluate safety and tolerability, including adverse events, clinical laboratory safety parameters, and vital signs. Exploratory objectives included evaluation of hematologic parameters, markers of hemolysis, and patient-reported outcomes related to pain and quality of life.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Men or women over 18 to 65 years, both inclusive.
- •Non-smokers.
- •BMI > 18 kg/m2
- •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).
- •Haemoglobin levels between 5.5 and 10.5 g/dl during Screening (for newly diagnosed or patients not on any treatment for SCD).
- •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.
- •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)
- •Patient or the patient's legally authorized representative has given written informed consent.
排除标准
- •Patients with known or suspected allergy to any ingredient of the food supplement
- •Patient having consumed vitamin or food supplements containing NAD+ precursors (niacin, tryptophan, nicotinamide, NMN, NR etc...) during the month before selection.
- •Patient has a significant medical condition that required hospitalization (other than sickle cell crisis) within two months of the screening visit.
- •Patient has prothrombin time INR > 2.
- •Patient has serum albumin less than 3.0 g/dl.
- •Patient has received any blood products within three months of the Screening visit.
- •Patients hospitalized for acute vaso-occlusive crisis within one month of the Screening visit.
- •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).
- •Patient with diagnosed cancer in the past 2 years.
- •Patients participating simultaneously in another clinical research protocol or having recently participated in another research for which the exclusion period has not been completed.
- •Pregnant, lactating or parturient women.
- •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.
- •Majors under legal protection or unable to express their consent.
- •People in an emergency situation unable to express their prior consent.
研究组 & 干预措施
Placebo
Participants received matching placebo for 90 days, in addition to standard of care.
干预措施: Placebo (Dietary Supplement)
结局指标
主要结局
Safety as measured by subject incident of treatment-emergent adverse events
时间窗: 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
时间窗: 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
时间窗: 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)
次要结局
- Change in hematocrit(Day 0, Day 30, Day 60, Day 90)
- Change in F-Hb content in RBCs(Day 0, Day 30, Day 60, Day 90)
- Change in RBC sickling(Day 0, Day 30, Day 60, Day 90)
- Change in reticulocyte level(Day 0, Day 30, Day 60, Day 90)
- Change in indirect bilirubin level(Day 0, Day 30, Day 60, Day 90)
- Change in pain perception(Day 0, Day 30, Day 60, Day 90)
- Change in the % of F-hemoglobin positive cells(Day 0, Day 30, Day 60, Day 90)
- ASCQ-Me Questionnaire (Adult Sickle Cell Quality of Life Measurement Information System)(Day 0, Day 30, Day 60, Day 90)
- Change in serum lactate dehydrogenase level(Day 0, Day 30, Day 60, Day 90)
- Pain relief assessment(Day 0, Day 30, Day 60, Day 90)