immune thrombocytopenia (ITP) MedDRA version: 23.0 Level: PT Classification code 10083842 Term: Immune thrombocytopenia System Organ Class: 10005329 - Blood and lymphatic system disorders
Conditions
Sponsors
Novartis Pharma AG
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Male or female patients aged 18 years and older on the day of signing the informed consent. 2. A signed informed consent must be obtained prior to participation in the study. 3. A diagnosis of primary ITP, with insufficient response to, or relapse after a first-line corticosteroid therapy± IVIG. 4. Patients with platelet count =65 years) yes F.1.3.1 Number of subjects for this age range 38
Exclusion criteria
Exclusion criteria: 1. ITP patients who received second-line ITP treatments (other than corticosteroid therapy± IVIG) including splenectomy. However, patients exposed to thrombopoietin receptor agonists (TPO-RAs) for a limited time (max one week) before screening are eligible. 2. Patients with key lab abnormalities and patients with Evans syndrome or any other cytopenia(patients with low grade anemia related to bleeding or iron deficiency are eligible) 3. Patients with history of clinically significant hematological disorders, or with marked altered hematologic parameters 4. Patients with current or history of life-threatening bleeding 5. Patient that are Human Immunodeficiency Virus (HIV), Hepatitis C Virus (HCV), Hepatitis B surface Antigen (HBsAg)/ Hepatitis B core antibody (HBcAB)-positive. HBcAb positive patients can be enrolled if HBsAg negative, HBV negative, HBV DNA negative, no pre-existing liver fibrosis is present and antiviral prophylaxis is given. 6. Patients with known active or uncontrolled infection requiring systemic treatment during screening period 7. Patients with hepatic impairment 8. Patients with concurrent coagulation disorders and/or receiving antiplatelet or anticoagulant medication(with an exemption of low dose of acetylsalicylic acide(=150 mg daily) 9. Female patients who are pregnant or nursing Other protocol defined criteria may apply
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To demonstrate that the addition of ianalumab (either dose) to standard of care, eltrombopag, prolongs Time to Treatment Failure (TTF) compared to eltrombopag alone in participants with primary ITP who have had an insufficient response to or relapsed after first-line treatment with corticosteroids;Secondary Objective: 1. To assess quality, time to and duration of response in each treatment arm 2. To assess the rate of participants who successfully taper and discontinue eltrombopag in each treatment arm 3. To assess the incidence and severity of bleeding in each treatment arm 4. To assess the need for rescue treatments in each treatment group 5. To evaluate treatment effects on ITP related symptoms, functioning, and health-related quality of life (HFRQoL) 6. To assess B-Cell levels and immunoglobulin (lg) levels 7. To assess ianalumab pharmacokinetics (PK) 8. To assess the immunogenicity of ianalumab;Primary end point(s): Time from randomization date until the first of the following events indicative of treatment failure: -platelet count below 30 G/L -start of a new ITP treatment -need for a rescue treatment -ineligibility to taper or inability to discontinue eltrombopag -death;Timepoint(s) of evaluation of this end point: Randomization to end of study (up to 39 months after randomization of last participant) | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. Percentage of participants with any platelet count of at least 100 G/L in the absence of rescue treatment or new ITP treatment 2. Percentage of participants with any platelet count of at least 50 G/L in the absence of rescue treatment or new ITP treatment 3. Percentage of participants with a best response rate by either response or complete response 4. Time from randomization to date of first response and time from randomization to date of first complete response 5. Time from achievement of response to treatment failure 6. Stable response in each treatment group 7. Time from achievement of complete response to loss of complete response 8. Probability to be treatment failure-free (as defined for the primary efficacy endpoint) after the end of treatment period 9. Percentage of participants reporting bleeding events according to WHO bleeding scale 10. Number of participants who are in need of rescue treatment in each treatment arm 11. Percentage of participants who are in need of rescue treatment 12. The Patient-Reported Outcomes Measurement Information System (PROMIS) Short Form v1.0 Fatigue 13a includes 13 items that assess fatigue in adults. 13. The ITP-PAQ is a 44 item scale for measuring HRQoL in adults with ITP across ten scales: Symptoms, BotherPhysical Health, Fatigue/Sleep, Activity, Fear, Psychological Health, Work, Social Activity, Women's Reproductive Health, overall Qol. Each item is rated on a Likert type scale. Each scale is scored from O to 100. Higher scores represent better HRQoL 14. Change from baseline in the frequency (percentage within CD45) and absolute number of CD19+ B-cell counts 15. Time to B-cell recovery defined as =80% of baseline or = 50 cells/µL 16. Change from baseline in immunoglobulin levels 17. AUClast: Area under the curve from time zero to the last measurable concentration sampling time (tlast) AUCtau: Area under the curve calculated to the end of a dosing interval (tau) 18. Cmax: Maximum (peak) observed | — |
Countries
Argentina, Australia, Austria, Belgium, China, Czechia, Czech Republic, France, Germany, Hungary, India, Italy, Japan, Korea, Republic of, Malaysia, Mexico, Netherlands, Norway, Philippines, Romania, Singapore, Spain, Taiwan, Thailand, Türkiye, United Kingdom, United States
Contacts
Public ContactClinical Trial Information Desk
Novartis Pharma AG
Outcome results
None listed