A phase 3, randomized, double-blind, study of ianalumab (VAY736) versus placebo in addition to eltrombopag in primary immune thrombocytopenia (ITP) patients who failed steroids

JRCT ID: jRCT2031220623

Registered date:09/02/2023

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: primary immune thrombocytopenia
• Date of first enrollment: 14/08/2023
• Target sample size: 15
• Countries of recruitment: Singapore,Japan
• Study type: Interventional

Intervention(s)

Experimental: Treatment arm 1
Participants will receive eltrombopag and ianalumab lower dose

Experimental: Treatment arm 2
Participants will receive eltrombopag and ianalumab higher dose

Placebo Comparator: Treatment arm 3
Participants will receive eltrombopag and placebo

Outcome(s)

Primary Outcome

Time from randomization until treatment failure [ Time Frame: Randomization to until end of study (up to 39 months after randomization of last participant) ]
Time from randomization until treatment failure is defined as the 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

Key inclusion & exclusion criteria

• Age minimum: >= 18age old
• Age maximum: Not applicable
• Gender: Both

Include 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. Platelet count <30 G/L and assessed need for treatment (per physician's discretion).

Exclude criteria

1. ITP patients who received second-line ITP treatments (other than steroid 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
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
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
9. Female patients who are pregnant or nursing

Related Information

• Primary Sponsor: Yamauchi Kyosuke
• Secondary ID(s): NCT05653219