Therapeutic Efficacy and Mechanism Study of Retinoid Acid on Immune Thrombocytopenia Patients

The recruitment status of this study is unknown because the information has not been verified recently.
Verified August 2012 by Peking University People's Hospital.
Recruitment status was  Active, not recruiting
Sponsor:
Collaborator:
Beijing Municipal Science & Technology Commission
Information provided by (Responsible Party):
Xiao-hui Zhang, Peking University People's Hospital
ClinicalTrials.gov Identifier:
NCT01667263
First received: August 15, 2012
Last updated: August 17, 2012
Last verified: August 2012
  Purpose

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.


Condition Intervention Phase
Autoimmune Thrombocytopenia
Drug: All-trance retinoid acid
Drug: Danazol
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Clinical Study on Therapeutic Efficacy and Mechanism of All-trance-retinoid Acid in Refractory Immune Thrombocytopenia Patients

Resource links provided by NLM:


Further study details as provided by Peking University People's Hospital:

Primary Outcome Measures:
  • Platelet count [ Time Frame: Every two weeks ] [ Designated as safety issue: Yes ]
    Platelet count and change in platelet count


Secondary Outcome Measures:
  • Megakaryocyte count [ Time Frame: Every two weeks ] [ Designated as safety issue: No ]
    Megakaryocyte count and change in megakaryocyte count

  • Megakaryocyte ploidy distribution [ Time Frame: Every two weeks ] [ Designated as safety issue: No ]
    Megakaryocyte ploidy distribution


Other Outcome Measures:
  • Bleeding episodes [ Time Frame: Every two weeks ] [ Designated as safety issue: Yes ]
    Number of bleeding episodes and bleeding site

  • Number of patients with adverse events [ Time Frame: Every two weeks ] [ Designated as safety issue: No ]

Estimated Enrollment: 80
Study Start Date: June 2011
Estimated Study Completion Date: June 2013
Estimated Primary Completion Date: June 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: All-trance retinoid acid &Danazol
Danazol 200mg tablet by mouth and all-trance retinoid acid 10mg tablet by mouth every 8 hours a day for 12 consecutive weeks.
Drug: All-trance retinoid acid
Other Name: Retinoid acid
Drug: Danazol
Other Names:
  • Danocrine
  • Cleregil
  • Danol
Active Comparator: Danazol
Danazol 200mg tablet by mouth every 8 hours a day for 12 consecutive weeks.
Drug: Danazol
Other Names:
  • Danocrine
  • Cleregil
  • Danol

Detailed 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.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   Yes
Criteria

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.
  Contacts and Locations
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Please refer to this study by its ClinicalTrials.gov identifier: NCT01667263

Locations
China, Beijing
Peking University People's Hospital, Peking University Insititute of Hematology
Beijing, Beijing, China, 100044
Sponsors and Collaborators
Peking University People's Hospital
Beijing Municipal Science & Technology Commission
Investigators
Principal Investigator: Xiao-hui Zhang, Professor Peking University People's Hospital, Peking University Insititute of Hematology
  More Information

Publications:
LIU Wen-bin, WANG Zhao-yue, CAO Li-juan, ZHAO Xiao-juan, ZHU Ming-qing, BAI Xia, RUAN Chang-geng.Therapeutic Effect and Mechanism of All-trans-retinoic Acid Treatment in Refractory Idiopathic Thrombocytopenic Purpura.Suzhou University Journal of Medical Science.2009;3 476-479.

Responsible Party: Xiao-hui Zhang, Professor, Peking University People's Hospital
ClinicalTrials.gov Identifier: NCT01667263     History of Changes
Other Study ID Numbers: U1111-1132-6877, Z111107058811024
Study First Received: August 15, 2012
Last Updated: August 17, 2012
Health Authority: China: Beijing Municipal Science and Technology Commission

Keywords provided by Peking University People's Hospital:
therapeutic efficacy
mechanism
all-trance retinoid acid
refractory immune thrombocytopenia

Additional relevant MeSH terms:
Thrombocytopenia
Purpura, Thrombocytopenic, Idiopathic
Blood Platelet Disorders
Hematologic Diseases
Purpura, Thrombocytopenic
Purpura
Blood Coagulation Disorders
Thrombotic Microangiopathies
Hemorrhagic Disorders
Autoimmune Diseases
Immune System Diseases
Hemorrhage
Pathologic Processes
Skin Manifestations
Signs and Symptoms
Danazol
Estrogen Antagonists
Estrogen Receptor Modulators
Hormone Antagonists
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs
Pharmacologic Actions

ClinicalTrials.gov processed this record on August 28, 2014