Clinical trial NCT04618328: A Study of ATRA in the Treatment of ITP
Official title: A Study of All-trans Retinoic Acid in the Treatment of Immune Thrombocytopenia
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
Conditions: Immune Thrombocytopenia
Lead sponsor: Peking University People's Hospital
Sponsor class: OTHER
Collaborators: name: Qilu Hospital of Shandong University
name: Beijing Hospital
name: Navy General Hospital, Beijing
First posted: 2020-11-05
Enrollment: 80
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: ATRA is used to treat ITP patients.
Primary purpose: TREATMENT

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

Detailed 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.

Eligibility criteria: 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.

Primary outcomes:
measure: Sustained response
timeFrame: 6 months
Secondary outcomes:
measure: complete response (CR)
timeFrame: day 14
measure: Response (R)
timeFrame: day 14
measure: Number of patients with bleeding
timeFrame: 6 months
measure: Number of patients with adverse events
timeFrame: 6 months
measure: Time to response
timeFrame: 6 months
measure: Duration of response (DOR)
timeFrame: 6 months
measure: loss of response
timeFrame: 6 months
Other outcomes:
Locations:
city: Beijing
country: China
facility: Peking University Institute of Hematology
state: Beijing
Study officials:
affiliation: Peking University People's Hospital, Peking University Insititute of Hematology
name: Xiaohui Zhang, Doctor
role: PRINCIPAL_INVESTIGATOR
References: