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Compensation: Varies

Number of Visits: Unknown

Duration: Up to 33 weeks

225 Participants Needed

Ianalumab + Corticosteroids for Low Platelet Count
(VAYHIT1 Trial)

Recruiting at 153 trial locations

Overseen ByNovartis 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 DataThis treatment has passed at least one previous human trial

Trial Summary

What is the purpose of this trial?

The purpose of this study is to evaluate the effect of two different doses of ianalumab versus placebo in addition to first-line corticosteroids in maintaining platelet count ≥30 G/L in adult participants with primary ITP.

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.

How does the drug Ianalumab + Corticosteroids differ from other treatments for low platelet count?

Ianalumab combined with corticosteroids is unique because it targets the immune system to help maintain platelet counts, potentially offering a new approach compared to traditional treatments like corticosteroids alone or IVIG, which may not sustain platelet levels effectively.¹ ² ³ ⁴ ⁵

Research Team

Novartis Pharmaceuticals

Principal Investigator

Novartis Pharmaceuticals

Eligibility Criteria

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.

See 2 more

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.

See 3 more

Timeline

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

Treatment Details

Interventions

Corticosteroids
Ianalumab
Placebo

Trial OverviewThe 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.

Participant Groups

3Treatment groups

Experimental Treatment

Placebo Group

Group I: Ianalumab Lower doseExperimental Treatment2 Interventions

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

Group II: Ianalumab Higher doseExperimental Treatment2 Interventions

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

Group III: PlaceboPlacebo Group2 Interventions

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

Find a Clinic Near You

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

Findings from Research

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]

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]

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]