Therapeutic Efficacy and Mechanism Study of Retinoid Acid on Immune Thrombocytopenia Patients
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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 |
- Platelet count [ Time Frame: Every two weeks ] [ Designated as safety issue: Yes ]Platelet count and change in platelet count
- 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
- 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:
|
|
Active Comparator: Danazol
Danazol 200mg tablet by mouth every 8 hours a day for 12 consecutive weeks.
|
Drug: Danazol
Other Names:
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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 |
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| China, Beijing | |
| Peking University People's Hospital, Peking University Insititute of Hematology | |
| Beijing, Beijing, China, 100044 | |
| Principal Investigator: | Xiao-hui Zhang, Professor | Peking University People's Hospital, Peking University Insititute of Hematology |
More Information
Publications:
| 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 May 19, 2013