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

NCT ID: NCT01667263

Last Updated: 2017-09-07

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 130 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2012-06-01
• Study Completion Date: 2017-02-01

Brief 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.

Detailed 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.

Conditions

Autoimmune Thrombocytopenia

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

All-trans retinoic acid ＆Danazol

Danazol 400mg po and ATRA 10mg bid po

Group Type: EXPERIMENTAL

All-trans retinoic acid

Intervention Type: DRUG

Danazol

Intervention Type: DRUG

Danazol

Danazol 400mg po

Group Type: ACTIVE_COMPARATOR

Danazol

Intervention Type: DRUG

Interventions

All-trans retinoic acid

Intervention Type: DRUG

Danazol

Intervention Type: DRUG

Other Intervention Names

Retinoid acid; Danocrine; Cleregil; Danol

Eligibility Criteria

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.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Beijing Municipal Science & Technology Commission

OTHER

Sponsor Role: collaborator

Beijing Hospital

OTHER_GOV

Sponsor Role: collaborator

Qilu Hospital of Shandong University

OTHER

Sponsor Role: collaborator

Navy General Hospital, Beijing

OTHER

Sponsor Role: collaborator

Beijing Tongren Hospital

OTHER

Sponsor Role: collaborator

Peking University People's Hospital

OTHER

Sponsor Role: lead

Responsible Party

Xiao-hui Zhang

Professor

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Xiao-Hui Zhang, Professor

Role: PRINCIPAL_INVESTIGATOR

Peking University People's Hospital, Peking University Insititute of Hematology

Locations

Beijing Hospital, Ministry of Health

Beijing, Beijing Municipality, China

Peking University People's Hospital, Peking University Insititute of Hematology

Beijing, Beijing Municipality, China

Beijing Tongren Hospital

Beijing, Beijing Municipality, China

PLA Navy General Hospital

Beijing, Beijing Municipality, China

Qilu Hospital, Shandong University

Jinan, Shandong, China

Countries

China

References

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Reference Type: BACKGROUND

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Reference Type: BACKGROUND

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Reference Type: DERIVED

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Other Identifiers

Z111107058811024

Identifier Type: OTHER_GRANT

Identifier Source: secondary_id

U1111-1132-6877

Identifier Type: -

Identifier Source: org_study_id