Ianalumab for Low Platelet Count

Not currently recruiting at 45 trial locations

Overseen By Novartis Pharmaceuticals

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Novartis Pharmaceuticals

Must be taking: Corticosteroids, TPO-RA

Must not be taking: Rituximab, Belimumab

Disqualifiers: Secondary thrombocytopenia, Neutrophils <1000/mm3, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

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

Compensation: Varies

Number of Visits: Unknown

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests ianalumab, a potential treatment for individuals with primary immune thrombocytopenia (ITP), which causes low platelet counts and can lead to easy bruising or bleeding. Researchers aim to evaluate the effectiveness and safety of ianalumab for those who have not responded to other treatments, such as corticosteroids and TPO-RAs. Suitable participants have had ITP for an extended period, have tried specific medications, and continue to experience low platelets that impact their daily life. As a Phase 2 trial, this research focuses on assessing the treatment's effectiveness in an initial, smaller group of participants.

Will I have to stop taking my current medications?

The trial does not specify if you must stop taking your current medications, but you need to be on a stable dose of a corticosteroid or TPO-RA for at least 14 days before starting the trial. Some medications, like B-cell depleting therapies, must be stopped 12 weeks before the trial.

Is there any evidence suggesting that ianalumab is likely to be safe for humans?

Research has shown that ianalumab is generally well-tolerated by patients with primary immune thrombocytopenia (ITP). Earlier studies demonstrated its promise in raising platelet counts, which are crucial for blood clotting. Importantly, no new or unexpected safety issues have been reported.

Initial findings suggest that ianalumab quickly and effectively increases platelet levels, reducing bleeding risks in ITP patients. The safety of ianalumab is considered manageable, with most side effects being manageable.

The fact that ianalumab is in a Phase 2 trial indicates that earlier studies have already provided some evidence of its safety in humans. This is promising, although ongoing studies are needed to fully understand the treatment's safety and effectiveness. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Most treatments for low platelet count, like corticosteroids or immunosuppressants, work by broadly dampening the immune system to prevent it from attacking platelets. But Ianalumab works differently, targeting a specific protein called BAFF (B-cell activating factor) that plays a role in the survival of B cells, which are part of the immune response that can lead to low platelet levels. This targeted approach could mean fewer side effects compared to the broader immune suppression seen with existing treatments. Researchers are excited because this specificity might offer a more effective way to increase platelet counts with potentially better safety and tolerability.

What evidence suggests that ianalumab might be an effective treatment for low platelet count?

Research has shown that ianalumab can help treat low platelet counts in people with primary immune thrombocytopenia (ITP). In one study, ianalumab maintained safe platelet levels during and after treatment, potentially reducing the need for other treatments and improving overall health. Another study found that ianalumab accelerated recovery from low platelet counts and reduced related health issues. These findings suggest ianalumab could be a promising option for those dealing with 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?

This trial is for adults with primary ITP who have low platelet counts and have tried at least two treatments, including corticosteroids and TPO-RAs. Participants must be over 18, provide consent, and not have had certain other treatments like B-cell depleting therapy or a splenectomy recently.

Inclusion Criteria

Signed informed consent obtained prior to participation in the study

My last treatment for ITP didn't work or caused side effects.

My platelet count is below 30 G/L and I need treatment.

Exclusion Criteria

I have had my spleen removed.

I have been diagnosed with a low platelet count not caused by an initial disease.

I haven't had blood transfusions or specific treatments within the last 14 days.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2 weeks

Primary Endpoint Assessment

Participants receive ianalumab and are assessed for the primary endpoint

25 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

24 months

Second Course (Optional)

Participants who lost response may receive a second course of ianalumab

25 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Ianalumab

Trial Overview The study tests the effectiveness of Ianalumab in treating ITP after previous therapies. It aims to see how safe and tolerable this drug is for patients who've already used steroids and TPO-RA but still need treatment.

How Is the Trial Designed?

Group I: Single-arm Experimental Treatment 1 Intervention

All eligible participants will receive ianalumab at the same dose.

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

In a 52-week study involving 234 adults with immune thrombocytopenia, romiplostim significantly increased the rate of platelet response compared to standard care, with a response rate 2.3 times higher (P<0.001).

Patients receiving romiplostim experienced fewer treatment failures and required splenectomy less often (11% vs. 30% for standard care, P<0.001), along with lower rates of bleeding events and improved quality of life.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Romiplostim or standard of care in patients with immune thrombocytopenia. Kuter, DJ., Rummel, M., Boccia, R., et al.[2016]

Citations

1. 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 ... Ianalumab prolonged the duration of safe platelet levels during and after treatment in patients with primary immune thrombocytopenia (ITP) ...

2. 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 ...

3. 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 ...

4. novartis.com novartis.com/clinicaltrials/study/nct05653349

Study of Ianalumab Versus Placebo in Addition to First-line ... The purpose of this study is to evaluate the effect of two different doses of ianalumab versus placebo in addition to first-line ...

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

Novartis Ianalumab Phase 3 Trial Shows Longer ... It included recurrent low platelet counts after week 8, need for rescue therapy, starting a new ITP treatment, inability to taper or discontinue ...

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

Ianalumab Delivers Encouraging Outcomes in Heavily ... Interim VAYHIT3 data show ianalumab delivers rapid, lasting platelet responses and reduced bleeding in refractory ITP, with tolerable safety.

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 ...