A Multicenter Investigation of Recombinant Human Thrombopoietin (rhTPO) Combining Cyclosporin A Versus Cyclosporin A in Management of Steroid-Resistant/Relapsed Immune Thrombocytopenia （ITP）

• ClinicalTrials.gov Identifier: NCT02203422
• Recruitment Status: Withdrawn
• First Posted: July 29, 2014
• Last Update Posted: April 20, 2016
• Sponsor: Shandong University
• Information provided by (Responsible Party): Ming Hou, Shandong University

Study Description

Brief Summary:

The project was undertaking by Qilu Hospital of Shandong University and other 5 well-known hospitals in China. In order to report the efficacy and safety of recombinant human thrombopoietin combining with cyclosporin A for the treatment of adults with refractory immune thrombocytopenia (ITP), compared to cyclosporin A monotherapy.

Condition or disease	Intervention/treatment	Phase
Immune Thrombocytopenia	Drug: Cyclosporin A; Drug: rhTPO	Phase 3

Detailed Description:

The investigators are undertaking a parallel group, multicenter, randomized controlled trial of 120 steroid-resistant/relapsed ITP adult patients from 6 medical centers in China. One part of the participants are randomly selected to receive recombinant human thrombopoietin (given subcutaneously at a dose of 300 Units/kg for 14 consecutive days, following with a flexible dosage depending on platelet count until the 29th day), combining with cyclosporin A (given orally at a dose of 1.5-2.0mg/kg twice daily for 3 consecutive months, adjusted to maintain serum levels between 200-400 ng/ml and tapered by 50 mg/d per week if patients achieved a complete response). The others are selected to receive cyclosporin A monotherapy (given orally at a dose of 1.5-2.0mg/kg twice daily for 3 consecutive months, adjusted to maintain serum levels between 200-400 ng/ml and tapered by 50 mg/d per week if patients achieved a complete response). Platelet count, bleeding and other symptoms were evaluated before and after treatment. Adverse events are also recorded throughout the study. In order to report the efficacy and safety of the combination therapy compared to cyclosporin A monotherapy for the treatment of adults with steroid-resistant/relapsed ITP.

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 0 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Double (Participant, Investigator)
• Primary Purpose: Treatment
• Official Title: Recombinant Human Thrombopoietin (rhTPO) Combining Cyclosporin A Versus Cyclosporin A in Management of Steroid-Resistant/Relapsed Immune Thrombocytopenia （ITP）
• Study Start Date: July 2014
• Estimated Primary Completion Date: July 2015

Arms and Interventions

Arm	Intervention/treatment
Experimental: combination treatment group; 60 enrolled patients are randomly picked up to take cyclosporin A in combination with rhTPO at the indicated dose.	Drug: Cyclosporin A; given orally at a dose of 1.5-2.0mg/kg twice daily for 3 consecutive months, adjusted to maintain serum levels between 200-400 ng/ml and tapered by 50 mg/d per week if patients achieved a complete response.; Drug: rhTPO; given subcutaneously at a dose of 300 Units/kg for 14 consecutive days, following with a flexible dosage depending on platelet count until the 29th day
Active Comparator: single treatment group; 60 enrolled patients are randomly picked up to take cyclosporin A at the indicated dose.	Drug: Cyclosporin A; given orally at a dose of 1.5-2.0mg/kg twice daily for 3 consecutive months, adjusted to maintain serum levels between 200-400 ng/ml and tapered by 50 mg/d per week if patients achieved a complete response.

Outcome Measures

Primary Outcome Measures :

1. Evaluation of platelet response (Complete Response) [ Time Frame: The time frame is up to 3 months per subject ]
   CR. A complete response (CR) was defined as a sustained (≥ 3 months) platelet count ≥ 100×10^9/L
2. Evaluation of platelet response (Response) [ Time Frame: The time frame is up to 3 months per subject ]
   R. A response (R) was defined as a sustained (≥ 3 months) platelet count ≥ 30×10^9/L without recurrence of thrombocytopenia.
3. Evaluation of platelet response (No Response) [ Time Frame: The time frame is up to 3 months per subject ]
   NR.No response (NR) was defined as platelet count < 30 × 10^9/L or a less than two fold increase in platelet count from baseline or the presence of bleeding. Platelet count must be measured on two occasions more than a day apart.
4. Evaluation of platelet response (relapses) [ Time Frame: The time frame is up to 3 months per subject ]
   A relapses was defined as platelet count falls below 30×10^9/L or bleeding accrues after achieving R or CR.

Secondary Outcome Measures :

1. The number and frequency of therapy associated adverse events The number and frequency of therapy associated adverse events The number and frequency of therapy associated adverse events The number and frequency of therapy associated adverse events [ Time Frame: up to 3 months per subject ]

Eligibility Criteria

• Ages Eligible for Study: 18 Years to 80 Years (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

Inclusion Criteria:

1. Meet the diagnostic criteria for immune thrombocytopenia.
2. Steroid-resistant/relapsed hospitalized patients, may be male or female, between the ages of 18-80 years.
3. To show a platelet count < 30×10^9/L, and with bleeding manifestations.
4. Eastern Cooperative Oncology Group（ECOG）performance status ≤ 2.
5. 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. Current HIV infection or hepatitis B virus or hepatitis C virus infections. 3.Severe medical condition (lung, hepatic or renal disorder) other than ITP. 4.Unstable or uncontrolled disease or condition related to or impacting cardiac function (e.g., unstable angina, congestive heart failure, uncontrolled hypertension or cardiac arrhythmia)

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

6.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.

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

Contacts and Locations

Locations

China, Shandong

Qilu Hospital, Shandong University

Jinan, Shandong, China, 250012

Sponsors and Collaborators

Shandong University

Investigators

• Principal Investigator: Ming Hou, Dr.; Shandong University

More Information

• Responsible Party: Ming Hou, Professor and Director, Shandong University
• ClinicalTrials.gov Identifier: NCT02203422
• Other Study ID Numbers: ITP-Cyclosporin A
• First Posted: July 29, 2014
• Last Update Posted: April 20, 2016
• Last Verified: July 2014

Keywords provided by Ming Hou, Shandong University:

Immune Thrombocytopenia; Recombinant Human Thrombopoietin; Cyclosporin A

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; Cyclosporine; Cyclosporins; Enzyme Inhibitors; Molecular Mechanisms of Pharmacological Action; Immunosuppressive Agents; Immunologic Factors; Physiological Effects of Drugs; Antifungal Agents; Anti-Infective Agents; Dermatologic Agents; Antirheumatic Agents; Calcineurin Inhibitors