A Study of ATRA in the Treatment of ITP

Immune Thrombocytopenia Clinical Trial

Official title:

A Study of All-trans Retinoic Acid in the Treatment of Immune Thrombocytopenia

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT04618328
• Other study ID #: 5000 offer
• Status: Not yet recruiting
• Phase: Phase 2
• Start date: November 2020
• Est. completion date: October 2021

Study information

• Verified date: October 2020
• Source: Peking University People's Hospital
• Contact: Xiaohui Zhang, Doctor
• Phone: +8613522338836
• Email: zhangxh100[@]sina.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

An open-label, multicenter study to compare the efficacy and safety of ATRA for the treatment of adults with primary immune thrombocytopenia (ITP)

Description:

The investigators are undertaking an open-label, single-arm, multicenter trial of adults patients with ITP in China. Patients with treatmant naive ITP were given ATRA treatment. Platelet count, bleeding and other symptoms were evaluated before and after treatment. Adverse events are also recorded throughout the study.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 80
• Est. completion date: October 2021
• Est. primary completion date: October 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 70 Years

Eligibility

Inclusion Criteria:

1. Confirmed newly-diagnosed, treatment-naive ITP;

2. Platelet counts <30×109/L ;

3. Platelet counts < 50×109/L and significant bleeding symptoms (WHO bleeding scale 2 or above);

4. Willing and able to sign written informed consent.

Exclusion Criteria:

1. Received chemotherapy or anticoagulants or other drugs affecting the platelet counts within 3 months before the screening visit;

2. Received first-line and second-line ITP-specific treatments (eg, steriods, cyclophosphamide, 6-mercaptopurine, vincristine, vinblastine, etc) within 3 months before the screening visit;

3. Received high-dose steroids or IVIG in the 3 weeks prior to the start of the study.

4. Current HIV infection or hepatitis B virus or hepatitis C virus infections;

5. Severe medical condition (lung, hepatic or renal disorder) other than chronic ITP. Unstable or uncontrolled disease or condition related to or impacting cardiac function (e.g., unstable angina, congestive heart failure, uncontrolled hypertension or cardiac arrhythmia);

6. Female patients who are nursing or pregnant, who may be pregnant, or who contemplate pregnancy during the study period;

7. Have a known diagnosis of other autoimmune diseases, established in the medical history and laboratory findings with positive results for the determination of antinuclear antibodies, anti-cardiolipin antibodies, lupus anticoagulant or direct Coombs test;

8. Patients who are deemed unsuitable for the study by the investigator.

Related Conditions & MeSH terms

• Immune Thrombocytopenia
• Purpura, Thrombocytopenic, Idiopathic
• Thrombocytopenia

Intervention

Drug:
• all trans retinoic acid
ATRA is given concomitantly at a daily dose of 10 mg twice daily orally for 12 weeks

Locations

Country	Name	City	State
China	Peking University Institute of Hematology	Beijing	Beijing

Sponsors (4)

Lead Sponsor	Collaborator
Peking University People's Hospital	Beijing Hospital, Qilu Hospital of Shandong University, The Sixth Medical Center of PLA General Hospital

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Sustained response	The maintenance of platelet count = 30 x 10^9/L, at least 2-fold increase of the baseline count, the absence of bleeding, and no need for rescue medication at the 6-month follow-up	6 months
Secondary	complete response (CR)	complete response (CR) was defined as platelet count more than 100,000 per cubic millimeter and absence of bleeding.	day 14
Secondary	Response (R)	Response (R) as platelet count more than 30,000 per cubic millimeter and at least 2-fold increase of the baseline count and absence of bleeding.	day 14
Secondary	Number of patients with bleeding	Number of patients with bleeding complication ( WHO bleeding score).	6 months
Secondary	Number of patients with adverse events	Number of patients with adverse events	6 months
Secondary	Time to response	The time from starting treatment to time of achievement of CR or R	6 months
Secondary	Duration of response (DOR)	Duration of response at 6-month follow up.	6 months
Secondary	loss of response	Platelet counts below 100 x 109/L or bleeding (from CR) or platelet counts below 30 x 109/L, less than 2-fold increase of baseline platelet count or bleeding (from R)	6 months