ClinicalTrials.gov record NCT07039422
Study of Ianalumab in Adults With Primary Immune Thrombocytopenia (ITP) and Warm-antibody Autoimmune Hemolytic Anemia (wAIHA) Who Have Previously Benefited From Ianalumab
A Phase 2 Study of Ianalumab in Adults With Primary Immune Thrombocytopenia (ITP) and Warm-antibody Autoimmune Hemolytic Anemia (wAIHA) Who Have Previously Benefited From Ianalumab.
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AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This Phase 2 interventional study gives a second course of ianalumab (intravenous infusion) to adults with primary ITP or wAIHA who previously benefited from ianalumab. Participants are assigned to one of two dose groups and receive the same dose they had before. The trial monitors safety, immune response, and clinical outcomes with safety and efficacy follow-up after treatment. The study is non-randomized, parallel-assignment and quadruple-masked.
What it is studying. The study tests the efficacy and safety of a second course of ianalumab in adults with ITP or wAIHA who previously benefited from ianalumab.
What the researchers hope to learn. To explore the efficacy and safety of a second course of ianalumab in adults with ITP and wAIHA who previously benefited from the drug. Key scientific goals include measuring treatment-failure-free status for ITP (by 12 months), durable hemoglobin response for wAIHA (sustained improvement between weeks 9 and 25), pharmacokinetics of a second course, and the incidence and titer of anti-ianalumab antibodies and severe infections.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
This study is intended to explore the efficacy and safety of a second course of ianalumab after experiencing treatment failure in the pivotal Primary Immune Thrombocytopenia (ITP) trials (CVAY736I12301, CVAY736Q12301) and after loss of durable response in the pivotal Warm Autoimmune Hemolytic Anemia (wAIHA) trial (CVAY736O12301).
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Biological
- Ianalumab
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion criteria
- Signed informed consent obtained prior to participation in the study.
- Male or female participants aged 18 years and older on the day of signing informed consent
- Primary ITP patients:
- Previously enrolled and treated either with ianalumab/placebo in addition to first-line corticosteroids on protocol CVAY736I12301 or with ianalumab/placebo in addition to eltrombopag in the second line on protocol CVAY736Q12301, and who experienced treatment failure (TF) by parent trial definition ≥ 2 years after the last infusion of ianalumab/placebo
- Rescue medication and/or bridging therapy are allowed to be started within the 28 days prior to screening; platelet count results obtained prior to the start of the therapy must be used to assess eligibility and have to be collected within 30 days prior to screening
- For Primary or Secondary wAIHA patients:
- Previously documented by a positive direct antiglobulin test (DAT) specific for anti-IgG or anti-IgA, previously enrolled and treated with ianalumab/placebo in blinded cohort or placebo followed by crossover to open label ianalumab in protocol CVAY736O12301, having experienced durable response lasting beyond 2 years from the last infusion of ianalumab/placebo in blinded cohorts or a durable response beyond week 20 from last dose of first course of ianalumab in the crossover arm.
- Relapsed wAIHA with hemoglobin concentration ≥5 g/dL and <10 g/dL and presence of symptoms related to anemia during screening or within 14 days before screening window or within 28 days before screening window if rescue medication/bridging therapy has been initiated.
- Rescue medication and/or bridging therapy are allowed to be started during the screening and within 28 days prior to screening; hemoglobin level result for eligibility assessment needs to be obtained prior to the start of the treatment within 30 days prior to screening
- Supportive care is allowed in the case the participant received it in the parent trial when the relapse occurred and has remained stable at least 4 weeks prior screening
Exclusion criteria
- Evans syndrome or any cytopenia other than thrombocytopenia (for ITP participants) or anemia (for wAIHA), except for grade 1 anemia due to blood loss or iron deficiency.
- Secondary wAIHA with BM involvement for wAIHA patients
- Current life-threatening bleeding or history of life-threatening bleeding due to thrombocytopenia
- Therapy for ITP or wAIHA other than ianalumab/placebo, bridging/rescue therapies and supportive care prior to the beginning of the screening window
- After primary analysis of each respective parent trial, participants whose treatment was unblinded and who received placebo only will be excluded.
- ITP participants only: Participants with concurrent coagulation disorders and/or receiving anti-platelet or anti-coagulant medication except for low dose of acetylsalicylic acid (≤150 mg per day)
- Other protocol-defined inclusion/exclusion criteria may apply.
Study description and what participation involves
The study's detailed description, as published on ClinicalTrials.gov.
Detailed description from the registry
This is a multicenter, Phase 2 exploratory study to assess the efficacy and safety of a second course of ianalumab in adults with ITP and wAIHA who have previously benefited from ianalumab. All participants will receive ianalumab at the dose of study treatment from which they previously derived benefit. After completion of the screening period, participants who do not meet treatment failure criteria during treatment period will enter the safety and efficacy follow-up, whereas participants who reported a treatment failure during the treatment period will enter the safety follow-up only. The study will end once all participants have completed safety and/or efficacy follow-up since their last dose of ianalumab or discontinued the study earlier.
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AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
After screening, all participants receive a second course of ianalumab by intravenous infusion at the dose they previously benefited from. They will attend treatment visits (infusions) and scheduled follow-up visits for safety and efficacy; if a participant meets treatment-failure criteria during treatment they will move to safety follow-up only. The study involves regular blood tests to check platelets or hemoglobin, monitoring for infections and other side effects, and blood sampling to measure drug levels and anti‑drug antibodies. Follow-up assessments may continue for up to 2 years after the last dose.
How the study is structured. Phase 2 interventional treatment trial. Non-randomized, parallel assignment into two experimental arms (lower and higher ianalumab doses). Quadruple-masked. All participants receive intravenous ianalumab at the dose from which they previously derived benefit, then enter safety and/or efficacy follow-up; participants are followed up to about 2 years after their last dose.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- The listed sites are in Argentina, Belgium, China, Czechia, France, Germany, Hungary, Italy, Malaysia, Romania, Singapore, Spain, Turkey, United Kingdom. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on autoimmune hemolytic anemia
Important dates
- Study start
- Jun 26, 2026 (estimated)
- Primary completion
- Aug 30, 2030 (estimated)
- Study completion
- Aug 30, 2030 (estimated)
- Target enrollment
- 60 participants (estimated)
- Last updated on registry
- Jul 17, 2026
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- Novartis Pharmaceuticals
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Novartis Pharmaceuticals 📞 1-888-669-6682 ✉ novartis.email@novartis.com
- Novartis Pharmaceuticals 📞 +41613241111
Participating sites (25)
- Novartis Investigative SiteWithdrawn
Buenos Aires, C1039AAC · Argentina
- Novartis Investigative SiteRecruiting
Roeselare, West-Vlaanderen, 8800 · Belgium
- Novartis Investigative SiteRecruiting
Guangzhou, Guangdong, 510515 · China
- Novartis Investigative SiteRecruiting
Dalian, Liaoning, 116023 · China
- Novartis Investigative SiteRecruiting
Tianjin, 300020 · China
- Novartis Investigative SiteRecruiting
Ostrava, Poruba, 708 52 · Czechia
- Novartis Investigative SiteRecruiting
Caen, 14033 · France
- Novartis Investigative SiteRecruiting
Rennes, 35033 · France
- Novartis Investigative SiteRecruiting
Jena, Thuringia, 07740 · Germany
- Novartis Investigative SiteRecruiting
Debrecen, Hajdu Bihar Megye, 4032 · Hungary
Terms used on this page · explanations by MedicalUpdates.ai
- informed consent
- The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
- placebo
- A treatment that looks like the study treatment but contains no active ingredient. Some participants receive it instead of the study treatment, usually without knowing which they got.
- corticosteroids
- Steroid medicines, such as prednisone, that reduce inflammation. Studies often require a stable dose at entry or measure whether the study treatment allows the dose to be reduced.
- prior to screening
- The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
- blinded
- Who does not know which intervention a participant is receiving. It can apply to participants, the people giving the treatment, those assessing results, or those analysing the data.
- cohort
- A group of people who share a characteristic, such as a diagnosis, and are studied together. 'Prospective' means they are followed forward from enrolment; 'retrospective' means existing records are reviewed.
- crossover
- Each participant receives more than one intervention, one after the other, usually with a gap (a washout) in between. Participants act as their own comparison.
- open label
- No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
- Relapsed
- A condition that improved with treatment and has since returned or worsened.
- exclusion criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
- Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- follow-up
- The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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