Overview

This trial has been completed.

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 2012
End date July 2016
Trial size 95 participants
Trial identifier NCT01667263, U1111-1132-6877, Z111107058811024

Summary

Randomized, open-label, multicentre study to compare the efficacy and safety of ATRA plus danazol with danazol monotherapy in patients with corticosteroid-resistant/relapsed ITP.

United States No locations recruiting
Other countries No locations recruiting

Study Design

Allocation randomized
Intervention model parallel assignment
Primary purpose treatment
Masking no masking
Arm
(Experimental)
Danazol 400mg po and all-trance retinoid acid 10mg bid po
all-trance retinoid acid Retinoid acid
danazol Danocrine
(Active Comparator)
Danazol 400mg po
danazol Danocrine

Primary Outcomes

Measure
the sustained platelet response at the 6-month follow-up
time frame: From the start of study treatment (Day 1) up to the end of Month 6

Secondary Outcomes

Measure
overall response
time frame: From the start of study treatment (Day 1) up to the end of Month 6
primary response rate at 4 weeks
time frame: From the start of study treatment (Day 1) up to week 4 of treatment
primary response rate at 8 weeks
time frame: From the start of study treatment (Day 1) up to week 8 of treatment
time to response
time frame: From the start of study treatment (Day 1) up to the end of month 6
duration of response
time frame: From the start of study treatment (Day 1) up to the end of month 6
reduction in bleeding symptoms
time frame: From the start of study treatment (Day 1) up to the end of month 6
safety
time frame: From the start of study treatment (Day 1) up to the end of month 6

Eligibility Criteria

All participants at least 18 years old.

Inclusion Criteria

  • Primary immune thrombocytopenia (ITP) confirmed by excluding other supervened causes of thrombocytopenia;
  • Platelet count of less than 30×109/L at enrolment
  • Patients who did not achieve a sustained response to treatment with full-dose corticosteroids for a minimum duration of 4 weeks or who relapsed during steroid-tapering or after its discontinuation.
  • 18 years older.

Exclusion Criteria

  • Secondary immune thrombocytopenia (e.g., patients with HIV, HCV, Helicobacter pylori infection or patients with systemic lupus erythematosus)
  • congestive heart failure
  • severe arrhythmia
  • nursing or pregnant women
  • aspartate aminotransferase and alanine transaminase levels ≥ 3× the upper limit of the normal threshold criteria
  • creatinine or serum bilirubin levels each 1•5 times or more than the normal range
  • active or previous malignancy
  • Unable to do blood routine test for the sake of time, distance, economic issues or other reasons.

Additional Information

Official title The Combination of Oral All-trans Retinoic Acid and Danazol vs Danazol as Second-line Treatment in Adult Immune Thrombocytopenia: a Multicenter, Randomized, Open-label Trial
Principal investigator Xiao-Hui Zhang, Professor
Description Immune thrombocytopenia (ITP) is a severe bleeding disorder. Approximately 2/3 of patients achieve remission from first-line therapies. However, the underlying mechanism of corticosteroid-resistant or relapsed ITP is not well understood; thus, treatment remains a great challenge. All-trans retinoic acid (ATRA) has an immunomodulatory effect on haematopoiesis, making it a possible treatment option. A multicentre prospective study was performed in non-splenectomized ITP patients who were either resistant to a standard dose of corticosteroids or had relapsed. Patients were randomized to ATRA+danazol and danazol monotherapy group. Platelet count, bleeding and other symptoms were evaluated before and after treatment. Adverse events are also recorded throughout the study, in order to compare the efficacy and safety of ATRA plus danazol with danazol monotherapy in patients with corticosteroid-resistant/relapsed ITP.
Trial information was received from ClinicalTrials.gov and was last updated in February 2017.
Information provided to ClinicalTrials.gov by Peking University People's Hospital.