Comprehensive Evaluation of Two Second-Line Therapeutic Approaches for Immune Thrombocytopenia (ITP) – a Pragmatic Randomized Controlled Trial - Holistic Study

A vizsgálat adatai

Ki végzi

Ostfold Hospital Trust

Azonosító

EU 2023-505573-32-00

A regiszteradat frissítve

2026. szeptember 15.

Eredeti leírás

To compare in a randomized clinical trial (RCT) the efficacy of the oral TPO-RA, Avatrombopag, to Rituximab, in adult ITP patients who fail to achieve adequate response (does not respond, relapse or cannot discontinue corticosteroids after 6 week from initiation) to a short course of corticosteroids.A teljes eredeti leírásKevesebb

To compare in a randomized clinical trial (RCT) the efficacy of the oral TPO-RA, Avatrombopag, to Rituximab, in adult ITP patients who fail to achieve adequate response (does not respond, relapse or cannot discontinue corticosteroids after 6 week from initiation) to a short course of corticosteroids.

Bevonási feltételek: Male or female aged ≥18 years; Diagnosis of primary ITP of less than one-year duration and having a platelet count of < 30 x109/L measured within two weeks prior to inclusion with failure to achieve response or relapse after at least one cycle of dexamethasone (20-40 mg daily for 4 days) or prednisone /prednisolone (1 mg/kg for at least two weeks). Shorter courses or lower doses are allowed if discontinued or modified due to side effects; Failure to achieve response or relapse after at least one cycle of dexamethasone (20-40 mg daily for 4 days) or prednisone /prednisolone (1 mg/kg for at least two weeks). Shorter courses or lower doses are allowed if discontinued or modified due to side effects. Ongoing treatment with corticosteroids is allowed at the time of inclusion; Clinical need for subsequent platelet elevating therapy assessed by the physician in charge; Signed and dated written informed consent.

Kizáró feltételek: Females of child-bearing potential refusing to follow effective contraceptive methods (as described in SmPC) for at least 12 months following the last administration of Rituximab or during treatment with Avatrombopag; Concomitant autoimmune hemolytic anemia; Known allergy, sensitivity or contraindication to Rituximab or Avatrombopag; Any contraindication according to the SmPC of Rituximab (e.g. active serious infections (e.g. tuberculosis, opportunistic infections, sepsis) and serious heart failure (NYHAC IV) or serious uncontrolled heart disease) or of Avatrombopag (e.g. cirrhosis and/or severe hepatic impairment).”; Presence of active malignancy unless deemed cured by adequate treatment. Participants with the following neoplastic conditions can be included: a. Monoclonal gammopathy of undetermined significance (MGUS) or monoclonal B lymphocytosis of undetermined significance (MBUS). b. Basal/squamous cell carcinoma of the skin c. Carcinoma in situ of the cervix d. Carcinoma in situ of the breast e. Incidental histological finding of prostate cancer (TNM stage T1a or T1b); Patients with history of poor compliance or history of alcohol/drug abuse or excessive alcohol beverage consumption that would interfere with the ability to comply with the study protocol, or any disease that might interfere with the ability to comply with the study protocol or give informed consent; Secondary ITP: ITP secondary to lymphoma or chronic lymphocytic leukemia; ITP secondary to the following autoimmune disorders: Systemic Lupus Erythematosus, or Antiphospholipid Syndrome; ITP secondary to Common Variable Immune Deficiency; ITP secondary the following viral infections: Human Immunodeficiency Virus or Hepatitis C Virus; Active hepatitis B virus (positive HBsAg). Patients with HBsAg negative and HBV core antibody positive (HBcAb) should accept to receive entecavir (Baraclude) for 12 months if they will be allocated to Rituximab. Monthly HBV DNA monitoring will be required while on treatment and for the 6 months after the last dose of the study drug; Presence of any serious comorbidity where the condition may worsen by the study drugs; Previous treatment for ITP with: Rituximab, other immune suppressants than corticosteroids e.g. mycophenolate mofetil, azathioprine, cyclosporine), dapsone, danazol, chemotherapy (apart from vincristine as rescue therapy) or splenectomy. Short treatment with any thrombopoietic agent is allowed if given for a limited duration of a maximum of 2 weeks and not given within the last two weeks before inclusion, as rescue therapy for quick elevation of platelet count in emergency situations e.g. high risk of bleeding or ongoing bleeding or surgery; Patients in a severely immune compromised state; Pregnancy or lactation.

EU CTIS 2023-505573-32-00