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The Effective and Safety of Thalidomide in NTDT

Phase 2
Conditions
Thalassemia
Interventions
Registration Number
NCT02995707
Lead Sponsor
Xiao-Lin Yin
Brief Summary

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

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.

Recruitment & Eligibility

Status
UNKNOWN
Sex
All
Target Recruitment
30
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.

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
thalidomide thalassemiaThalidomidethalidomide:50mg/d p.o
Primary Outcome Measures
NameTimeMethod
The level of Hemoglobin18 months

All participants will complete the treatment for 18 months, the hemoglobin levels will be observed during the treatment.

Secondary Outcome Measures
NameTimeMethod
The effects of relative and absolute values of HbF18 months

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.

Trial Locations

Locations (1)

NO.3 Hospital of the Chinese People's Liberation Army

🇨🇳

Nanjing, Guangxi, China

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