The Effective and Safety of Thalidomide in TI

The Multi-center Clinical Trials of Thalidomide in TI

This is an exploratory experiment, aims to explore the effective and safety of thalidomide in the treatment of TI to improve the hemoglobin level, improve the quality of life, reduce blood transfusion, so as to avoid the adverse reactions caused by transfusion,60~100 patients will be enrolled.

Study Overview

• Status: Unknown
• Conditions: Thalassemia
• Intervention / Treatment: Drug: Thalidomide

Detailed Description

The project is a single arm research of thalidomide in TI,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. 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,1-month visit, 3-months visit , 6-months visit ,9-months visit, 12-months visit and 15-months visit.

• Study Type: Interventional
• Enrollment (Anticipated): 100
• Phase: Phase 2

Contacts and Locations

Study Locations

• China
  • Guangxi
    • Nanjing, Guangxi, China, 530021
      • Recruiting
      • NO.3 Hospital of the Chinese People's Liberation Army

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 65 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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;
  • If not blood transfusion,the level of HB<90g/dl, or blood transfusion to maintain the HB;
  • 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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: thalidomide thalassemia; thalidomide:50mg/d p.o	Drug: Thalidomide; thalidomide:50mg/d p.o at bedtime; Other Names: fǎn yìng tíng

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The Effective Rate of Patients	All participants will complete the treatment for 24 months, the hemoglobin value will be observed during the treatment,the value increased more than 20g/L defined as the marked effect,increased to 10~20g/L defined as effective, otherwise invalid.	24 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The Marked Improvement Rate of Patients	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.	24 months

Collaborators and Investigators

• Sponsor: Xiao-Lin Yin

Publications and helpful links

General Publications

• 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.

Study record dates

Study Major Dates

• Study Start (Actual): May 2, 2017
• Primary Completion (Anticipated): February 2, 2019
• Study Completion (Anticipated): May 2, 2019

Study Registration Dates

• First Submitted: June 8, 2017
• First Submitted That Met QC Criteria: June 11, 2017
• First Posted (Actual): June 14, 2017

Study Record Updates

• Last Update Posted (Actual): June 14, 2017
• Last Update Submitted That Met QC Criteria: June 12, 2017
• Last Verified: June 1, 2017

More Information

Terms related to this study

• Keywords: Thalidomide TI
• Additional Relevant MeSH Terms: Hematologic Diseases; Genetic Diseases, Inborn; Anemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; Thalassemia; Physiological Effects of Drugs; Anti-Infective Agents; Antineoplastic Agents; Immunosuppressive Agents; Immunologic Factors; Angiogenesis Inhibitors; Angiogenesis Modulating Agents; Growth Substances; Growth Inhibitors; Anti-Bacterial Agents; Leprostatic Agents; Thalidomide
• Other Study ID Numbers: 303 PLA

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Twenty four months later after the last visit，summery report will be shared with other researchers through database

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No