The Effective and Safety of Thalidomide in NTDT

Study Overview
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Brief Summary
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This is an exploratory experiment, aims to explore the effective and safety of thalidomide in the treatment of NTDT to improve the hemoglobin level, improve the quality of life, reduce blood transfusion, so as to avoid the adverse reactions caused by transfusion.15 30 patients will be enrolled, including type α 5 13 cases, type β 10 17 cases.

Detailed Description
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The project is a single arm research of thalidomide in NTDT,patients volunteered to participate in this trial and met the following inclusion criteria will be enrolled: age from 18 65, Eastern Cooperative Oncology Group (ECOG) performance status score of 2 or less. Subjects should take thalidomide 50mg per day for 12 weeks. During this time, hepatic and renal function, hematologic function, electrocardiogram and the adverse reactions were closely observed. According to the protocol, the blood routine, peripheral blood reticulocyte count, nucleated red blood cell count, hemoglobin electrophoresis and other indicators were evaluated in screening-period visit, 4-weeks visit, 8-weeks visit and 12-weeks visit.

Official Title
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The Phase II Clinical Trials of Thalidomide in NTDT

Conditions
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Thalassemia

Intervention / Treatment
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* Drug: Thalidomide


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients meeting all of the following criteria will be considered for admission to the trial: Diagnosis of NTDT; Ages 18-65 years; ECOG: 0 2 scores; Sign an informed consent agreeing to the clinical trial participation. Exclusion Criteria: Patients presenting with any of the following criteria will not be included in the trial: Patients received hydroxycarbamide, Yisui Shengxue Granule in three months; Women during Pregnancy, breastfeeding or those of childbearing age who do not want to take contraceptive measures; Patients had comorbidities like severe heart or lung diseases, liver dysfunction, cerebrovascular, cardiovascular, liver, kidney, tumor or other serious primary diseases; Patients Allergic to the drug ingredients; Patients with any Mental problem; Patients had Participated in other drug clinical trials in the past 1 month; Patients had a history of venous or arterial thrombosis; In certain circumstances that the researchers determined it was not suitable for the research.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 65 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: thalidomide thalassemia<br>thalidomide:50mg/d p.o | Drug: Thalidomide<br>* thalidomide:50mg/d p.o at bedtime<br>* Other names: fǎn yìng tíng;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| The level of Hemoglobin | All participants will complete the treatment for 18 months, the hemoglobin levels will be observed during the treatment. | 18 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| The effects of relative and absolute values of HbF | The highest hemoglobin value will be observed,the value increased more than 20g/L defined as the marked effect, increased to 10 20g/L defined as effective, otherwise invalid. | 18 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Thalidomide NTDT