Phase I/II Safety and Dose Escalation Trial of the Omega-3 Fatty Acids Docosahexaenoic Acid (DHA) and Eicosapentaenoic Acid (EPA) in Children and Young Adults With Sickle Cell Disease (SCD)
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 发起方
- 入组人数
- 4
- 试验地点
- 1
- 主要终点
- Determine Whether 6 Months of Supplementation With SCD-Omegatex™ Will Reduce Thermal Sensitivity by Quantitative Sensory Testing to Below Pre-treatment Levels
研究概览
简要总结
The purpose of this study is to determine the safety of a new formulation of the omega-3 fatty acids Docosahexaenoic Acid (DHA) and Eicosapentaenoic Acid (EPA) and to assess whether it decreases inflammation and inflammatory pain in children and young adults with Sickle Cell Disease.
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
- 8 Years 至 25 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subjects who meet all of the following criteria are eligible for enrollment into the study:
- •Participant has signed the informed consent/assent with parent signing informed consent as age appropriate.
- •Established diagnosis of HbSS, HbSC or HbSβo Thalassemia
- •History of ≥1 vasoocclusive events (managed at home and/or in hospital) in preceding 12 months.
- •Regular compliance with comprehensive care.
- •Aged 8 years or greater and less than 26 years.
- •At enrollment, subject should be in his/her baseline steady state and not in the midst of any acute complication due to SCD. Must be at least 2 weeks from infection or vasoocclusive crisis at time of screening labs
排除标准
- •Baseline hemoglobin levels <5.5 gm/dL.
- •Inability to swallow capsules
- •Poor compliance with previous treatment regimens.
- •Hepatic dysfunction
- •Renal dysfunction
- •PT and/or PTT ≥ 20% outside of normal
- •Allergy to fish, shell fish or soy
- •Triglyceride levels <80mg/dL.
- •Pregnancy.
- •Chronic Transfusion Therapy.
- •Transfusion within the last 30 days.
- •Treatment with any investigational drug or regular fish oil supplementations in last 60 days.
- •Currently receiving another investigational agent, or on such an agent with the last 60 days.
- •Dosage changes in preceding 3 months if on hydroxyurea
- •Diagnosed bleeding disorder or patient on concomitant anti-coagulation.
- •Conditional or abnormal result on most recent transcranial doppler or history of stroke.
- •Other active chronic illness that could adversely affect subjects performance
- •Children in Care
- •Platelet count less than 100,000
研究组 & 干预措施
SCD-Omegatex™
single arm
干预措施: SCD-Omegatex™ (Drug)
结局指标
主要结局
Determine Whether 6 Months of Supplementation With SCD-Omegatex™ Will Reduce Thermal Sensitivity by Quantitative Sensory Testing to Below Pre-treatment Levels
时间窗: 6 months
No results are available from any outcome measures as study was stopped early due to manufacturing issues with study drug and will not be reopened.
Clinical Safety, in a Dose Escalation Trial of SCD-Omegatex™ as Evidenced by an Absence of Adverse Events.
时间窗: 6 months with continuous monitoring
No results are available from any outcome measures as study was stopped early due to manufacturing issues with study drug and will not be reopened.
次要结局
- Number of Days With Pain Measured by iPad Daily Report Pain Calendar(8 months)
- Changes in Individual Thermal Sensitivity Thresholds by QST(8 months)
- Health-associated Quality of Life(6 months)
研究者
Robin E. Miller
Physician
Nemours Children's Clinic