Ianalumab + Corticosteroids for Low Platelet Count

(VAYHIT1 Trial)

Recruiting at 174 trial locations

Overseen By Novartis Pharmaceuticals

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Novartis Pharmaceuticals

Must be taking: Corticosteroids

Must not be taking: B-cell depleting, Anticoagulants

Disqualifiers: Evans syndrome, Coagulation disorders, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: Up to 33 weeks

226 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called ianalumab, combined with standard corticosteroids, to determine its effectiveness for people with primary ITP, a condition where the immune system attacks platelets, causing low platelet counts. The study compares two doses of ianalumab and a placebo (a substance with no active drug) to identify which best maintains safe platelet levels. Ideal participants are adults diagnosed with primary ITP within the last three months who have experienced low platelet counts. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to potentially groundbreaking treatment advancements.

Do I need to stop my current medications to join the trial?

The trial does not specify if you need to stop your current medications, but it does mention that participants should not have prior ITP treatments other than corticosteroids or IVIG. If you are on anti-platelet or anticoagulant medication, you may need to stop unless it's a low dose of acetylsalicylic acid.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that ianalumab yields promising results for treating primary immune thrombocytopenia (ITP). In earlier studies, patients taking ianalumab maintained safe platelet levels for longer periods, which is the main goal in managing ITP. Importantly, these studies did not find any major safety issues. Most patients tolerated the treatment well, experiencing no severe side effects.

Ianalumab is in an advanced testing phase, indicating it has already demonstrated safety in earlier studies. While no treatment is without risk, the data so far suggests that ianalumab is generally safe for people with primary ITP. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Researchers are excited about Ianalumab because, unlike traditional treatments for low platelet count, which mainly rely on corticosteroids, Ianalumab introduces a new mechanism of action. It targets the immune system more precisely by blocking BAFF (B-cell Activating Factor), which plays a crucial role in the survival of B cells that can destroy platelets. This targeted approach could potentially lead to better management of platelet levels with fewer side effects compared to standard therapies. Additionally, Ianalumab is administered intravenously, which might allow for more controlled dosing and faster onset of action.

What evidence suggests that this trial's treatments could be effective for low platelet count in primary ITP?

Research has shown that ianalumab, when combined with corticosteroids, can help treat low platelet counts in primary ITP, a blood disorder characterized by a low number of platelets. In this trial, participants will receive either a higher or lower dose of ianalumab, both with corticosteroids. Some studies have found that patients receiving ianalumab experience longer-lasting improvements in their platelet counts. Other research indicates that ianalumab increases platelet levels and speeds up recovery. Additionally, evidence suggests more stable platelet levels and a delay in treatment failure. These findings indicate that ianalumab, especially when combined with corticosteroids, could provide significant benefits for patients with primary ITP. ^{1 2 5 6 7}

Who Is on the Research Team?

Novartis Pharmaceuticals

Principal Investigator

Novartis Pharmaceuticals

Are You a Good Fit for This Trial?

Adults diagnosed with primary ITP within the last 3 months, having a platelet count below 30 G/L before treatment, and who've responded to corticosteroids can join. Excluded are those with other blood disorders, neutrophil counts below 1.0 G/L, life-threatening bleeding, previous ITP treatments (other than specific first-line therapies), or use of B-cell depleting therapy.

Inclusion Criteria

My platelet count increased to 50 G/L or more after steroid treatment.

Signed informed consent prior to participation in the study

I was diagnosed with ITP less than 3 months ago and haven't started treatment yet.

Exclusion Criteria

Absolute neutrophil count below 1.0 G/L at randomization

I am experiencing severe bleeding right now.

I have previously received treatment that targets B-cells.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive ianalumab or placebo in addition to first-line corticosteroids

Up to 33 weeks

Follow-up

Participants are monitored for efficacy and safety after treatment

Up to 39 months

What Are the Treatments Tested in This Trial?

Interventions

• Corticosteroids
• Ianalumab
• Placebo

Trial Overview The trial is testing two doses of Ianalumab versus a placebo in combination with corticosteroids to see if they help maintain platelet counts above 30 G/L in adults with primary ITP. Participants will be randomly assigned to receive either Ianalumab or a placebo.

How Is the Trial Designed?

Group I: Ianalumab Lower dose Experimental Treatment 2 Interventions

Lower dose of ianalumab administered intravenously with corticosteroids oral or parental (if clinically justified)

Group II: Ianalumab Higher dose Experimental Treatment 2 Interventions

Higher dose of ianalumab administered intravenously with corticosteroids oral or parental (if clinically justified)

Group III: Placebo Placebo Group 2 Interventions

Placebo administered intravenously with corticosteroids oral or parental (if clinically justified)

Who Is Running the Clinical Trial?

Novartis Pharmaceuticals

Lead Sponsor

Trials

2,963

Recruited

4,275,000+

Founded

1996

Headquarters

Basel, Switzerland

Known For

Precision medicine

Top Products

Gleevec, Cosentyx, Entresto, Kisqali

Dr. Vas Narasimhan

Novartis Pharmaceuticals

Chief Executive Officer since 2018

MD from Harvard Medical School

Dr. Shreeram Aradhye

Novartis Pharmaceuticals

Chief Medical Officer since 2021

MD

Published Research Related to This Trial

The novel drug delivery system, CD41-VCR-PLT, effectively targets macrophages and significantly increases the accumulation of vincristine in these cells, leading to a high inhibition of macrophage viability and increased apoptosis rates, which are crucial for treating immune thrombocytopenia (ITP).

In a mouse model of ITP, CD41-VLP not only improved platelet counts significantly compared to vincristine alone but also reduced systemic and peripheral neurotoxicity, suggesting a safer and more effective treatment option for patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Vincristine-loaded platelets coated with anti-CD41 mAbs: a new macrophage targeting proposal for the treatment of immune thrombocytopenia. Xu, P., Jiang, Y., Zuo, H., et al.[2020]

In a study of 35 patients with idiopathic thrombocytopenic purpura (ITP), high-dose intravenous immunoglobulin (IVIG) significantly increased platelet counts, but these counts typically returned to baseline levels quickly.

Following IVIG treatment, the addition of immunosuppressive therapy with azathioprine and glucocorticoid prednisolone helped most patients maintain their platelet counts, especially those who previously responded to prednisolone, indicating a potential effective strategy for long-term management of ITP.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Effects of maintenance treatment after high-dose intravenous gamma-globulin for idiopathic thrombocytopenic purpura]. Yasunaga, K., Kokawa, T., Fujitake, H., et al.[2021]

In a study of 65 patients with immune thrombocytopenia (ITP), the combination of high-dose dexamethasone and low-dose rituximab resulted in an impressive 81.5% response rate after one month, with sustained complete responses in 63.1% of patients after 12 months.

The treatment was particularly effective in patients who were already dependent on glucocorticoids, and importantly, it was well-tolerated with no serious adverse reactions reported.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Efficacy of high-dose dexamethasone plus low-dose rituximab as a second-line treatment in 65 patients with primary immune thrombocytopenia]. Yan, Z., Li, Z., Zhang, H., et al.[2020]

Recent advancements in the treatment of immune thrombocytopenic purpura (ITP) include the use of intravenous immunoglobulin (IVIg) and intravenous Rh immunoglobulin (IV RhIg), which have shown efficacy comparable to corticosteroids for increasing and maintaining platelet counts with reduced toxicity.

Rituximab (anti-CD20) has shown promise in inducing sustained remissions in chronic ITP patients with minimal toxicity, potentially offering a new spleen-sparing treatment option for those who are refractory to conventional therapies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Immune thrombocytopenic purpura - current management practices. Sandler, SG., Tutuncuoglu, SO.[2019]

In a study of 12 adults with newly diagnosed immune thrombocytopenia (ITP), the combination of eltrombopag and dexamethasone resulted in 100% of patients achieving a platelet count increase of at least 30 × 10(9)/L after treatment.

After 6 months, 50% of patients maintained a complete response with platelet counts of 100 × 10(9)/L or higher, indicating that this combination therapy is a promising frontline treatment for ITP.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Eltrombopag and high-dose dexamethasone as frontline treatment of newly diagnosed immune thrombocytopenia in adults. Gómez-Almaguer, D., Herrera-Rojas, MA., Jaime-Pérez, JC., et al.[2021]

Citations

1. docwirenews.com docwirenews.com/post/novartis-ianalumab-phase-3-trial-shows-longer-remission-in-itp

Novartis Ianalumab Phase 3 Trial Shows Longer ... Novartis says the ianalumab arms showed a clear delay in treatment failure. The company also points to better stable platelet responses at 6 ...

2. ashpublications.org ashpublications.org/blood/article/144/Supplement%201/552/531133/Ianalumab-an-ADCC-Enhanced-Anti-BAFF-Receptor

Ianalumab, an ADCC-Enhanced Anti-BAFF-Receptor ... Compared with the ITP control, Ianalumab treatment resulted in less thrombocytopenia with an earlier time to recovery, and improved morbidity ...

3. novartis.com novartis.com/news/media-releases/novartis-ianalumab-phase-iii-trial-meets-primary-endpoint-itp-demonstrating-statistically-significant-improvement-time-treatment-failure

Novartis ianalumab Phase III trial meets primary endpoint ... In VAYHIT2, patients treated with ianalumab plus eltrombopag experienced a significantly higher rate of sustained improvements in platelet count ...

4. clinicaltrials.gov clinicaltrials.gov/study/NCT05885555

NCT05885555 | A Study of Ianalumab (VAY736) in ... This is a phase 2, open-label, single-arm study to evaluate the efficacy, safety and tolerability of ianalumab in participants with primary ITP (platelet ...

5. sciencedirect.com sciencedirect.com/science/article/pii/S0006497124032993

Ianalumab, an ADCC-Enhanced Anti-BAFF-Receptor ... Compared with the ITP control, Ianalumab treatment resulted in less thrombocytopenia with an earlier time to recovery, and improved morbidity ...

6. bloodcancerstoday.com bloodcancerstoday.com/post/ianalumab-delivers-encouraging-outcomes-in-heavily-pretreated-itp

Ianalumab Delivers Encouraging Outcomes in Heavily ... At data cutoff (February 2025), 44% of patients achieved a confirmed response (platelet count ≥50 109/L on at least two consecutive assessments ...

7. clinicaltrials.gov clinicaltrials.gov/study/NCT05653349

NCT05653349 | Study of Ianalumab Versus Placebo in ... This is a multi-center, randomized, double-blind Phase 3 study to assess the efficacy and safety of two different doses of ianalumab compared to placebo in ...