An Open Label Randomized Controlled Trial to Evaluate the Efficacy and Safety of HYDROXYUREA in Management of Beta Thalassemia Patients in Karachi Pakistan
试验速览
- 阶段
- 2 期
- 发起方
- 入组人数
- 100
- 试验地点
- 1
- 主要终点
- No. of participants who become responders or partial responders
研究概览
简要总结
Objectives
Primary objectives:
- To determine the efficacy of Hydroxyurea in the study participants.
- Hypothesis: The study will result in either maintenance or rise in hemoglobin as compared to the control treatment.
Secondary objectives:
- To determine the compliance of Hydroxyurea in study participants.
- To determine the safety of Hydroxyurea in the study participants. Design and Outcomes
An open label randomized controlled trial to test the efficacy and safety of Hydroxyurea on beta thalassemia major patients. It is a six months study. Findings of physical examination, vital sign variables, laboratory variables and ultrasound at baseline, during and end of the study will be listed. Schedule of intervention is mentioned in section 6.1. later in the protocol.
Interventions and Duration Hydroxyurea will be given to the participants in intervention arm along with the standard treatment if thalassemia (blood transfusion and iron chelation therapy) and the control arm will receive the standard treatment (blood transfusion and iron chelation therapy) only. Each participant will be followed up for 6 months after initiating the intervention. Intervention will be given for 6 months or until the participant withdraws from the study or due to any reason, the investigator stops the intervention.
Sample Size and Population This pilot study will be done on 100 patients initially. Stratified randomization will be done on the basis of presence of Xmn polymorphism. And the study population will be assigned to intervention or control arm randomly through a computer software (randomizer.org).
详细描述
Study Enrollment Procedures
- The study participants will be examined on the screening visit to assess their eligibility to participate. Each participant will consent in writing (Appendix I) to the screening process before the start of the examination and laboratory or radiology investigations. Screening log will be maintained to record this information.
- Parental consent (for minors age group < 18 years) will be obtained from a legal guardian accompanying the participant.
- The research unit will produce a computer generated sets of random allocations stratified on the basis of presence or absence of Xmn in advance of the start of the study. They will be then sealed in consecutively numbered opaque envelopes. The study participants will be randomly assigned to the investigation arm or control arm accordingly.
Storage and Accountability
All study drugs must be kept in a secure place under adequate storage conditions - protected from moisture and light and under required temperature (in refrigerator if required). Records of dispensing and returns will be maintained by the trial site (Omair Sana Foundation) in form of counts of tablets dispensed or returned. The subject must return all unused study medication for each treatment period to the trial center.
Statistical Analysis plan Intention to treat analysis Each participant will be analyzed in the group he was assigned to (investigational arm and control arm) at the time of study initiation rather than what he /she will take (drug orstandard treatment). Data will be entered and analyzed through statistical software SPSS version 17. Descriptive and inferential statistics will be analyzed. Descriptive statistics will be presented as means, standard deviations and ninety five percent confidence levels of the means for continuous variables and frequency and percentages for categorical variable.
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
- None
入排标准
- 年龄范围
- 6 Months 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients with homozygous beta thalassemia major diagnosed on the basis of genetic mutation
- •Beta thalassemia intermedia (patients carrying homozygous, heterozygous or compound heterozygous beta thalassemia genes) (HbS, HbE)
- •Those patients who do not have baseline HbE suggestive of beta thalassemia, genetic mutations will be performed to confirm the diagnosis.
- •Age: 6 months and onwards
- •Gender: Either
- •Able to understand study procedures and to comply with them for the entire length of the study.
- •Provide written informed consent if aged 18 years and above and if minor that is below 18 years (Parental consent will be taken)
排除标准
- •Chronic liver disease, renal failure, history of stroke
- •Participants who have developed immune hemolytic anemia
- •Spleenomegaly (liver and spleen >5 cm below coastal margin
- •If allergic or sensitive to Hydroxyurea or its ingredients
- •Patients on immunosuppressants including Azathiopine or any other drug causing bone marrow suppression, Hepatitis C treatment that may cause red cell suppression and NSAIDS
- •Inability or unwillingness of individual or legal guardian/representative to give written informed consent.
研究组 & 干预措施
Hydroxyurea arm
This arm will be given investigational drug that is Hydroxyurea .This intervention will be given along with the standard treatment that is blood transfusion and iron chelation.
干预措施: Hydroxyurea (Drug)
结局指标
主要结局
No. of participants who become responders or partial responders
时间窗: 6 months
The responders will be those who become transfusion independent (in those who were transfusion dependent before Hydroxyurea) or those who maintain or show 1-2 g/dl increase in Hb from baseline and partial responders will be those whose transfusion requirement decreases by atleast 50% as compared to baseline.
次要结局
- The compliance of Hydroxyurea in study participants.(6 months)
- Number of participants with abnormal/deranged laboratory values(6 months)
- No. of participants with severe adverse events related to the intervention(6 months)
研究者
Dr.Saqib Hussain Ansari
Principal Investigator
Omair Sana Foundation