Primary Immune ThrombocytopeniaPhase 2VAY RE-HITNCT07039422

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 testing Ianalumab in people with Primary Immune Thrombocytopenia and Warm Autoimmune Hemolytic Anemia, run by Novartis Pharmaceuticals.

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Official record

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Trial summary

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… Read more about this study

Who is it for?: Ages 18 to 100 years
Conditions the study is for: Primary Immune Thrombocytopenia, Warm Autoimmune Hemolytic Anemia
What is being tested?: Ianalumab
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 0 participants
Where: No sites listed yet
Study dates: 26 Jun 2026to 30 Aug 2030
Phase: Phase 2

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 to 100 years
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

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:

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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

Who cannot join

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.

About this study

Official title: 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.

Read the detailed description

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.

Conditions: Primary Immune Thrombocytopenia, Warm Autoimmune Hemolytic Anemia
Being tested: Ianalumab
Keywords: Primary Immune Thrombocytopenia (ITP), ITP, Warm Autoimmune Hemolytic Anemia (wAIHA), wAIHA, VAY736, Ianalumab, B-cell depletion, B-cell Activating Factor Receptor (BAFF-R) blockade
Medical subject headings: Purpura, Thrombocytopenic, Idiopathic

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

IanalumabBiological
Also called VAY736
Concentrate for solution for infusion for intravenous use

Study groups in NCT07039422
Study group	Role	What they receive
Treatment arm 1	Experimental	Participants will receive ianalumab lower dose Receives: Biological: Ianalumab
Treatment arm 2	Experimental	Participants will receive ianalumab higher dose Receives: Biological: Ianalumab

What is being measured

The primary outcome is the main question the study is designed to answer.

Treatment failure free (yes, no) for participants with Immune Thrombocytopenia (ITP)
Treatment failure free (yes, no) by 12 months after start of second course of ianalumab at assigned dose.
Measured: From treatment start date until the events indicative of treatment failure (up to 2 years after last dose)
Durable response for participants with Warm Autoimmune Hemolytic Anemia (wAIHA)
Durable response (hemoglobin (Hb) ≥10 g/dL and ≥2 g/dL increase from baseline) for a period of at least 8 consecutive weeks, between W9 and W25 in the absence of rescue or prohibited treatment prior to that durable response achievement.
Measured: At least 8 consecutive weeks, between week 9 and week 25

Secondary outcomes (10)

Response rate in participants with ITP only
Percentage of participants with ITP achieving a response among all participants who received a second course of ianalumab, and among cohorts and/or group of cohorts.
Measured: from treatment start to end of study (up to 2 years after last dose)
Complete response rate in participants with ITP only
Percentage of participants with ITP achieving a complete response among all participants who received a second course of ianalumab, and among cohorts and/or group of cohorts.
Measured: from treatment start to end of study (up to 2 years after last dose)
Number and percentage of participants receiving new ITP therapy only
Number and percentage of participants who need new ITP therapy among all the participants who received a second course of ianalumab, and among cohorts and/or group of cohorts.
Measured: from treatment start to end of study (up to 2 years after last dose)
Response and complete response in participants with wAIHA only
Percentage of participants with wAIHA achieving a response and complete response among all participants who received a second course of ianalumab and among cohorts and/or group of cohorts.
Measured: from treatment start to end of study (up to 2 years after last dose)
Number and percentage of participants receiving new wAIHA therapy only
Number and percentage of participants who need new wAIHA therapy among all the participants who received a second course of ianalumab, and among cohorts and/or group of cohorts.
Measured: from treatment start to end of study (up to 2 years after last dose)
Number and percentage of participants receiving rescue treatment
Number and percentage of participants who need rescue treatment among all the participants who received a second course of ianalumab, and among cohorts and/or group of cohorts.
Measured: from treatment start to end of study (up to 2 years after last dose)
Number of severe infections and percentage of participants with severe infection
This is to assess the safety profile of ianalumab.
Measured: from treatment start to end of study (up to 2 years after last dose)
Ianalumab concentration in serum
Assess the pharmacokinetics of a second course of ianalumab treatment.
Measured: after first dose (pre dose 0 and 672 hours) and at study end (up to 2 years after last dose of Ianalumab)
Incidence of anti-ianalumab antibodies in serum (ADA assay) over time
Anti-drug antibodies (ADA) will be evaluated in samples collected from all participants to assess the immunogenicity of Ianalumab.
Measured: up to end of treatment (Week 16), end of study (up to 2 years after last dose of Ianalumab)
Titer of anti-ianalumab antibodies in serum (ADA assay) over time
Assess the immunogenicity of second course of ianalumab treatment.
Measured: up to end of treatment (Week 16), end of study (up to 2 years after last dose of Ianalumab)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

26 Jun 202630 Aug 2030

First posted26 Jun 2025
Study start26 Jun 2026 (estimated)
Primary completion30 Aug 2030 (estimated)
Study completion30 Aug 2030 (estimated)

Why it stopped: The study was discontinued due to evolving scientific \& feasibility considerations that could adversely affect recruitment, thereby reducing the likelihood of generating a sufficiently robust dataset to support reliable efficacy \& safety conclusions.

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Other trials for Primary Immune ThrombocytopeniaStudies of the same condition, open and under way.
More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Parallel groups
Masking: Quadruple blind
Who is masked: Participant, Care provider, Investigator, Outcomes assessor
Enrolment: 0 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT07039422
Sponsor's protocol ID: CVAY736Q12202B
Other IDs: 2024-518231-11-00
Acronym: VAY RE-HIT
Status last verified: Sep 2026
First submitted: 5 May 2025
Last updated: 15 Oct 2026
Results posted: No
Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: Yes: Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com
Expanded access: Available

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

Official record

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