Ianalumab plus a thrombopoietin receptor agonist for adults with ITP or Evans syndrome

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

Adults with primary immune thrombocytopenia (and an exploratory cohort with primary Evans syndrome) who are on a stable thrombopoietin receptor agonist will receive ianalumab (9 mg/kg) alongside their chosen TPO-RA to record tolerability and safety. The schedule includes a 28-day screening period, a 16-week treatment period, a TPO-RA tapering period with 16 weeks of monitoring, and a 60-week long-term safety follow-up. Participants must have received between one and four prior treatments for ITP and have had no recent change in their TPO-RA dose. The trial is open at selected U.S. clinical sites and is sponsored by Novartis Pharmaceuticals.

Why it matters

Who can join: the full rules

Key Inclusion Criteria:

* Signed informed consent must be obtained prior to participation in the study.
* Patients aged 18 years and older on the day of signing the informed consent.
* ITP cohort only: Confirmed diagnosis of primary ITP that has previously responded to corticosteroid treatment or IVIG treatment but the response was not sustained (response is defined as a platelet count ≥ 50 G/L).
* ITP cohort only: Received at least one prior treatment for ITP.
* ITP cohort only: Patients with a platelet count \< 100 G/L who are receiving a TPO-RA. Patients may already be receiving a TPO-RA or may start a TPO-RA at the time of screening. All patients should be on a stable dose of TPO-RA for at least 14 days prior to first dose of ianalumab. Note: during the screening period, a documented assessment of platelets \< 100 G/L is mandatory for enrollment. For patients who received rescue medication before screening, platelet count results obtained prior to the start of the rescue therapy should be used to assess eligibility if collected within 14 days prior to screening.
* ES cohort only: Patients with clinical diagnosis of primary ES with active thrombocytopenia (\< 100 G/L) with warm autoimmune hemolytic anemia (wAIHA) for whom a TPO-RA is appropriate per Investigator.
* ES cohort only: Inadequate response to or relapse after treatment with corticosteroid therapy.
* ES cohort only: Diagnosis confirmed by current or past positive direct antiglobulin test (DAT) (IgG+, with or without C3+) and evidence of hemolysis.
* ES cohort only: any supportive care treatment administered for wAIHA must be stable for at least 4 weeks prior to enrollment.

Key Exclusion Criteria:

* Patients being treated with TPO-RA for \> 6 months.
* Current life-threatening bleeding (related to thrombocytopenia).
* Prior splenectomy within 6 months of first administration of ianalumab.
* Patients with the following laboratory abnormalities:

  * Neutrophils: \< 1000/mm3
  * Serum creatinine \> 1.5 × upper limit of normal (ULN)
  * Aspartate aminotransferase (AST) \> 3.0 × ULN
  * Alanine aminotransferase (ALT) \> 3.0 × ULN
  * Immunoglobulin G (IgG) \< 5 g/L
  * ITP cohort only: hemoglobin \< 10 g/L, total bilirubin \> 1.5 × ULN
* Patients with significantly compromised liver disease (Child-Pugh 7 to 9) and decompensated liver disease (Child-Pugh 10 to 15).
* Treatment with a B-cell depleting therapy (e.g. rituximab or anti-B cell Activating Factor (e.g. belimumab) within 12 weeks prior to the first administration of ianalumab. Patients who are refractory to rituximab will be excluded from this trial, where refractory is defined as:

  \~ Patients who have not achieved a response (defined as platelet count ≥ 30 G/L and at least doubling from baseline within 12 weeks in the absence of rescue therapy) following completion of a standard course of rituximab
* History of hypersensitivity to any of the study treatments or its excipients or to drugs of similar chemical classes.
* Known history of primary or secondary immunodeficiency, or a positive human immunodeficiency virus (HIV) enzyme-linked immunosorbent assay (ELISA) and Western blot) test result.
* Patients exposed to more than 4 prior treatments for ITP.
* ITP cohort only: Diagnosis of secondary thrombocytopenia.
* ITP cohort: Use of immunosuppressant drugs other than corticosteroids or rituximab.
* ES cohort only: Diagnosis of secondary ES.
* ES cohort only: Life-threatening hemolysis.
* ES cohort only: patients with autoimmune hemolytic anemia other than wAIHA

Where it's running

• Ironwood Cancer and Research Centers
  Chandler, Arizona, United States Recruiting
• Highlands Oncology Group
  Fayetteville, Arkansas, United States Recruiting
• Harbor-UCLA Medical Center
  Torrance, California, United States Recruiting
• Yale Cancer Center
  New Haven, Connecticut, United States Recruiting
• Stamford Hospital
  Stamford, Connecticut, United States Recruiting
• Baptist MD Anderson Cancer Center
  Jacksonville, Florida, United States Recruiting

Study contacts

• Study coordinator: Novartis Pharmaceuticals
• Email: novartis.email@novartis.com
• Phone: 1-888-669-6682

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Ianalumab vs placebo in adults with Sjögren's disease and high symptom burden

  A 52-week study will test whether monthly 300 mg ianalumab injections help adults with Sjögren's disease who have high symptom burden.

  Recruiting at 72 sites Phase 3 Novartis · Peoria, Arizona and 71 other locations · NCT07621809

• High-dose dexamethasone plus orelabrutinib versus placebo for newly diagnosed adult ITP

  This trial tests whether adding orelabrutinib to high-dose dexamethasone helps adults newly diagnosed with primary ITP raise and maintain their platelet counts compared with placebo.

  Recruiting Phase 3 Shandong University · Jinan, Shandong · NCT07559331

• Intravenous immunoglobulin (5%) treatment for primary immune thrombocytopenia

  This study tests whether 5% intravenous immunoglobulin given daily for five days safely raises platelet counts in adults (18–65) with chronic primary ITP and platelet counts below 30 × 10^9/L.

  Recruiting at 9 sites Phase 3 Grand Shuyang Life Sciences (Chengdu) Co., Ltd. · Ankara, Altındağ and 8 other locations · NCT07233213

• Romiplostim combined with glucocorticoids for treating newly diagnosed adult ITP

  This study is testing if a new treatment combining romiplostim and glucocorticoids can help adults newly diagnosed with immune thrombocytopenia feel better compared to just using glucocorticoids alone.

  Recruiting Phase 2 Institute of Hematology & Blood Diseases Hospital, China · Tianjin, Tianjin Municipality · NCT06992128

• Testing STSA-1301 injection for treating immune thrombocytopenia

  This study is testing a new injection called STSA-1301 to see if it can safely help people with immune thrombocytopenia improve their platelet levels.

  Recruiting at 7 sites Phase 1/2 Jiangsu BioJeTay Biotechnology Co., Ltd. · Bengbu, Anhui and 6 other locations · NCT06929299

• Registry for patients with Primary Immune Thrombocytopenia in Spain

  This study is collecting information from patients with Primary Immune Thrombocytopenia in Spain to better understand the condition and improve how it's treated.

  Recruiting at 29 sites Fundación Española de Hematología y Hemoterapía · A Coruña, A Coruña and 35 other locations · NCT06919341

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.