A Study of ATRA in the Treatment of ITP

• ClinicalTrials.gov Identifier: NCT04618328
• Recruitment Status: Not yet recruiting
• First Posted: November 5, 2020
• Last Update Posted: November 5, 2020
• Sponsor: Peking University People's Hospital
• Collaborators: Qilu Hospital of Shandong University; Beijing Hospital; The Sixth Medical Center of PLA General Hospital
• Information provided by (Responsible Party): Xiao Hui Zhang, Peking University People's Hospital

Study Description

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)

Condition or disease	Intervention/treatment	Phase
Immune Thrombocytopenia	Drug: all trans retinoic acid	Phase 2

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.

Study Design

• Study Type: Interventional (Clinical Trial)
• Estimated Enrollment: 80 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Intervention Model Description: ATRA is used to treat ITP patients.
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: A Study of All-trans Retinoic Acid in the Treatment of Immune Thrombocytopenia
• Estimated Study Start Date: November 2020
• Estimated Primary Completion Date: October 2021
• Estimated Study Completion Date: October 2021

Arms and Interventions

Arm	Intervention/treatment
Experimental: ATRA treatment group; ATRA is given at a daily dose of 10 mg twice daily orally for 12 weeks	Drug: all trans retinoic acid; ATRA is given concomitantly at a daily dose of 10 mg twice daily orally for 12 weeks; Other Name: ATRA

Outcome Measures

Primary Outcome Measures :

1. Sustained response [ Time Frame: 6 months ]
   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

Secondary Outcome Measures :

1. complete response (CR) [ Time Frame: day 14 ]
   complete response (CR) was defined as platelet count more than 100,000 per cubic millimeter and absence of bleeding.
2. Response (R) [ Time Frame: day 14 ]
   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.
3. Number of patients with bleeding [ Time Frame: 6 months ]
   Number of patients with bleeding complication ( WHO bleeding score).
4. Number of patients with adverse events [ Time Frame: 6 months ]
   Number of patients with adverse events
5. Time to response [ Time Frame: 6 months ]
   The time from starting treatment to time of achievement of CR or R
6. Duration of response (DOR) [ Time Frame: 6 months ]
   Duration of response at 6-month follow up.
7. loss of response [ Time Frame: 6 months ]
   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)

Eligibility Criteria

• Ages Eligible for Study: 18 Years to 70 Years (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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.

Contacts and Locations

Contacts

Contact: Xiaohui Zhang, Doctor; +8613522338836; zhangxh100@sina.com

Locations

China, Beijing

Peking University Institute of Hematology

Beijing, Beijing, China, 100044

Sponsors and Collaborators

Peking University People's Hospital

Qilu Hospital of Shandong University

Beijing Hospital

The Sixth Medical Center of PLA General Hospital

Investigators

• Principal Investigator: Xiaohui Zhang, Doctor; Peking University People's Hospital, Peking University Insititute of Hematology

More Information

• Responsible Party: Xiao Hui Zhang, Vice president of Peking Univeristy Institute of Hematology, Peking University People's Hospital
• ClinicalTrials.gov Identifier: NCT04618328
• Other Study ID Numbers: 5000 offer
• First Posted: November 5, 2020
• Last Update Posted: November 5, 2020
• Last Verified: October 2020

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:

Thrombocytopenia; Purpura, Thrombocytopenic, Idiopathic; Blood Platelet Disorders; Hematologic Diseases; Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Thrombotic Microangiopathies; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Skin Manifestations; Tretinoin; Antineoplastic Agents; Keratolytic Agents; Dermatologic Agents