Clinical trial NCT02995707: The Effective and Safety of Thalidomide in NTDT
Official title: The Phase II Clinical Trials of Thalidomide in NTDT
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
Conditions: Thalassemia
Keywords: Thalidomide NTDT
Lead sponsor: 303rd Hospital of the People's Liberation Army
Sponsor class: OTHER
First posted: 2016-12-16
Enrollment: 30
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility criteria: 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.

Primary outcomes:
measure: The level of Hemoglobin
timeFrame: 18 months
Secondary outcomes:
measure: The effects of relative and absolute values of HbF
timeFrame: 18 months
Other outcomes:
Locations:
city: Nanjing
country: China
facility: NO.3 Hospital of the Chinese People's Liberation Army
state: Guangxi
Study officials:
affiliation: NO.3 Hospital of the Chinese People's Liberation Army
name: Yin X Lin, director
role: STUDY_DIRECTOR
References:
citation: Ren Q, Zhou YL, Wang L, Chen YS, Ma YN, Li PP, Yin XL. Clinical trial on the effects of thalidomide on hemoglobin synthesis in patients with moderate thalassemia intermedia. Ann Hematol. 2018 Oct;97(10):1933-1939. doi: 10.1007/s00277-018-3395-5. Epub 2018 Jun 22.