A Pilot Study of Nicotinamide Riboside Supplementation in Allogeneic Hematopoietic Cell Transplantation
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- Percent of participants experiencing a CTCAE 5.0 grade 3 or higher related to the study drug
研究概览
简要总结
The purpose of this study is to find a safe and tolerable way to improve engraftment after transplant. Research studies have shown that adding nicotinamide riboside to donor cells has the potential to increase blood stem cell numbers and potentially decrease the time to engraftment. Also, nicotinamide riboside, TRU NIAGEN (the study drug) is a type of vitamin B supplement that the general public can get without a prescription and is well tolerated.
详细描述
This is a single-center, pilot feasibility study of NR supplementation in allogeneic (donor) HCT in which the study team is seeking to obtain preliminary data on any beneficial effect of increasing NAD+ levels in-vivo to facilitate engraftment. Engraftment is defined as the process during which transplanted stem cells begin to grow in the bone marrow and produce new white blood cells, red blood cells, and platelets. It takes about 14 to 24 days for donor cells to engraft after infusion, and the time between which blood counts fall to the time when they recover is a very critical period. Transfusions are frequently required to prevent bleeding and antibiotics are needed to prevent infections during this critical time period. Shortening the time of engraftment decreases these risks and can improve long-term health status.
The primary objectives of this study is to evaluate safety and tolerability of NR supplementation
The secondary objective of this study is to evaluate neutrophil and platelet recovery after HCT
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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •All disease indications for allogeneic hematopoietic cell transplantation except for myelofibrosis.
- •Subject must meet program eligibility criteria* for myeloablative conditioning alloHCT and plan to undergo myeloablative conditioning.
- •HLA-identical related donor or unrelated human donor source with bone marrow graft.
- •Subjects must provide a written informed consent.
排除标准
- •History of allergy or intolerance to NR precursor compounds, including niacin or nicotinamide
- •Pregnant or breastfeeding women are excluded from this study since allogeneic HCT is a strict contraindication.
- •Subjects with uncontrolled intercurrent illness or psychiatric illness/social situations that would limit compliance with study requirements.
研究组 & 干预措施
Nicotinamide riboside (NR)
干预措施: Nicotinamide riboside (NR) (Drug)
结局指标
主要结局
Percent of participants experiencing a CTCAE 5.0 grade 3 or higher related to the study drug
时间窗: day +21
Safety of NR in allogeneic HCT recipients as measured by percent of participants experiencing a CTCAE 5.0 grade 3 or higher that is related to the study drug
Percent of participants who receive >75% of scheduled doses.
时间窗: day +21
Tolerability of NR in allogeneic HCT recipients, defined as 8 or more of 15 participants (i.e. at least 50% of enrolled subjects) able to receive \>75% of their scheduled doses (overall rate 53%, 95% confidence intervals 33%-86%). This will include both patients who discontinue NR due to study drug related toxicity and patients who are not able to stay on NR due to transplant related toxicity that limits ability to take the drug
Percent of participants who receive >75% of scheduled doses.
时间窗: day +7
Tolerability of NR in allogeneic HCT recipients, defined as 8 or more of 15 participants (i.e. at least 50% of enrolled subjects) able to receive \>75% of their scheduled doses (overall rate 53%, 95% confidence intervals 33%-86%). This will include both patients who discontinue NR due to study drug related toxicity and patients who are not able to stay on NR due to transplant related toxicity that limits ability to take the drug
Percent of participants who receive >75% of scheduled doses.
时间窗: day +14
Tolerability of NR in allogeneic HCT recipients, defined as 8 or more of 15 participants (i.e. at least 50% of enrolled subjects) able to receive \>75% of their scheduled doses (overall rate 53%, 95% confidence intervals 33%-86%). This will include both patients who discontinue NR due to study drug related toxicity and patients who are not able to stay on NR due to transplant related toxicity that limits ability to take the drug
Percent of participants experiencing a CTCAE 5.0 grade 3 or higher related to the study drug
时间窗: day +7
Safety of NR in allogeneic HCT recipients as measured by percent of participants experiencing a CTCAE 5.0 grade 3 or higher that is related to the study drug
Percent of participants experiencing a CTCAE 5.0 grade 3 or higher related to the study drug
时间窗: day +14
Safety of NR in allogeneic HCT recipients as measured by percent of participants experiencing a CTCAE 5.0 grade 3 or higher that is related to the study drug
次要结局
- Median days to neutrophil recovery after HCT(Up to 100 days from start of treatment)
- Median days to platelet recovery after HCT(Up to 100 days from start of treatment)