Overview

This trial is active, not recruiting.

Condition autoimmune thrombocytopenia
Treatments all-trance retinoid acid, danazol
Phase phase 2
Sponsor Peking University People's Hospital
Collaborator Beijing Municipal Science & Technology Commission
Start date June 2011
End date June 2013
Trial size 80 participants
Trial identifier NCT01667263, U1111-1132-6877, Z111107058811024

Summary

Firstly, on the basis of our primary research, the investigators are to further confirm the efficacy and safety of differentiation inducer---all-trance retinoid acid (ATRA) in the treatment of refractory immune thrombocytopenia (RITP). Secondly, from the prospective of induced differentiation, the investigators want to verify whether ATRA is a potential curative agent for RITP. Thirdly, the investigators are trying to explore the possible pathogenesis of RITP and the mechanism of ATRA in curing RITP.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety/efficacy study
Intervention model parallel assignment
Masking open label
Primary purpose treatment
Arm
(Experimental)
Danazol 200mg tablet by mouth and all-trance retinoid acid 10mg tablet by mouth every 8 hours a day for 12 consecutive weeks.
all-trance retinoid acid Retinoid acid
danazol Danocrine
(Active Comparator)
Danazol 200mg tablet by mouth every 8 hours a day for 12 consecutive weeks.
danazol Danocrine

Primary Outcomes

Measure
Platelet count
time frame: Every two weeks

Secondary Outcomes

Measure
Megakaryocyte count
time frame: Every two weeks
Megakaryocyte ploidy distribution
time frame: Every two weeks

Eligibility Criteria

Male or female participants at least 18 years old.

Inclusion Criteria: - Primary immune thrombocytopenia (ITP) confirmed by excluding other supervened causes of thrombocytopenia; - Failure to achieve at least response or loss of response after splenectomy; - Need of treatment(s)(including, but not limited to, low dose of corticosteroids) to minimize the risk of clinically significant bleeding; - 18 years older. Exclusion Criteria: - Secondary thrombocytopenia; - Complicated with sever cardio-cerebrovascular diseases; - Unable to do blood routine test for the sake of time, distance, economic issues or other reasons.

Additional Information

Official title Clinical Study on Therapeutic Efficacy and Mechanism of All-trance-retinoid Acid in Refractory Immune Thrombocytopenia Patients
Principal investigator Xiao-hui Zhang, Professor
Description Refractory immune thrombocytopenia (RITP) is a severe bleeding disorder with a high mortality rate of 10% to 30%, which is resulted from the toxicities associated with therapies, as well as from life-threatening bleedings. Up to now, treatments for RITP patients are still limited and no consensus has been reached about the standard treatment protocol, therefore, it remains a great problem and challenge for hematologists to think out ways to treat RITP, that is to say, new therapies should be explored to maintain a relatively stable and safe platelet counts with minimum toxicities, so as to improve patients' quality of life and reduce mortalities related to severe bleedings and therapies. Immune thrombocytopenia (ITP) is an autoimmune disorder ascribed not only to increased platelet (PLT) destruction, but also to reduced PLT production, which is appears to be related to impaired megakaryocyte maturation. All-trance retinoid acid (ATRA) belongs to a class of retinoids, which exerts immunomodulatory and differentiation inducing capacities, and has been studied in clinical trails about autoimmune disease and has been successfully applied in clinic to cure APL by inducing the differentiation of cancer cells. In this study, the investigators retrospectively reviewed the medical chart of 35 RITP patients who take ATRA as part of the combination therapy and have been regularly visiting professor Zhang's clinic, a professional clinic of bleeding and coagulation disorders from February 2008 to August 2012. The investigators made phone calls for some other detailed medically relevant information not recorded, including previous ITP history, bleeding episodes and etc.
Trial information was received from ClinicalTrials.gov and was last updated in August 2012.
Information provided to ClinicalTrials.gov by Peking University People's Hospital.