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Completed

NCT Number: NCT01667263

The Combination of ATRA and Danazol as Second-line Treatment in Adult Immune Thrombocytopenia

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.

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Key information

Conditions

Autoimmune Thrombocytopenia Autoimmune Diseases Blood Coagulation Disorders Blood Platelet Disorders Cytopenia Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhage Hemorrhagic Disorders Immune System Diseases Pathologic Processes Pathological Conditions, Signs and Symptoms Purpura Purpura, Thrombocytopenic Purpura, Thrombocytopenic, Idiopathic Signs and Symptoms Skin Manifestations Thrombocytopenia Thrombotic Microangiopathies

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Beijing Hospital, Ministry of Health, Beijing, Beijing Municipality, China

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

All-trans retinoic acid

Drug

Other names: Retinoid acid

Danazol

Drug

Other names: Danocrine, Cleregil, Danol

Primary outcomes

  1. the sustained platelet response at the 12-month follow-up

    Time frame: From the start of study treatment (Day 1) up to the end of Month 12

    The number of participants (responders) with platelet count >=30x10^9/L and at least a 2-fold increase in the baseline count (PR) or a platelet count >=100x10^9/L (CR) and the absence of bleeding, without rescue medication at 12-month follow-up.

Secondary outcomes

  1. overall response

    Time frame: From the start of study treatment (Day 1) up to the end of Month 12

    The number of participants with platelet count >=30×10^9/L at least once and at least a doubling of the baseline platelet count without the administration of any other platelet increasing therapy

  2. primary response rate at 4 weeks

    Time frame: From the start of study treatment (Day 1) up to week 4 of treatment

    The number of participants with platelet count >=30×10^9/L and at least a doubling of the baseline platelet count without the administration of any other platelet increasing therapy at week 4 of treatment

  3. primary response rate at 8 weeks

    Time frame: From the start of study treatment (Day 1) up to week 8 of treatment

    The number of participants with platelet count >=30×10^9/L and at least a doubling of the baseline platelet count without the administration of any other platelet increasing therapy at week 8 of treatment

  4. time to response

    Time frame: From the start of study treatment (Day 1) up to the end of month 12

    Time to response was defined as the time from starting treatment to the time to achieve the response.

  5. duration of response

    Time frame: From the start of study treatment (Day 1) up to the end of month 12

    Duration of response was measured from the achievement of response to the loss of response.

  6. reduction in bleeding symptoms

    Time frame: From the start of study treatment (Day 1) up to the end of month 12

    Changes of bleeding after treatment. Bleeding was defined in accordance with the WHO bleeding scale (0, no bleeding; 1, petechiae; 2, mild blood loss; 3, gross blood loss; and 4, debilitating blood loss).

  7. safety

    Time frame: From the start of study treatment (Day 1) up to the end of follow-up

    All patients were assessed for safety every week during the first 8 weeks of treatment, and at 2-week intervals thereafter. Adverse events were scaled according to Common Terminology Criteria for Adverse Events (CTCAE) version 5.0

Sponsors and collaborators

Lead sponsor

Peking University People's Hospital

Other

Collaborators

  • Beijing Hospital
  • Beijing Municipal Science & Technology Commission
  • Beijing Tongren Hospital
  • Navy General Hospital, Beijing
  • Qilu Hospital of Shandong University

Registry information

Official study 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

Important dates

Study start
2012
Primary completion
2016
Study completion
2017
First posted
Aug 17, 2012
Registry last updated
Sep 7, 2017

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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