Ianalumab for Low Platelet Count

Recruiting at 36 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

41 Participants Needed

Trial Summary

What is the purpose of this trial?

The purpose of this study is to evaluate the therapeutic efficacy, safety and tolerability of ianalumab in adult patients with primary ITP previously treated with at least one corticosteroid and one TPO-RA.

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.

What data supports the effectiveness of the drug Ianalumab for low platelet count?

While there is no direct data on Ianalumab, other drugs like eltrombopag and avatrombopag, which are thrombopoietin receptor agonists (drugs that help increase platelet production), have shown effectiveness in increasing platelet counts in conditions like chronic immune thrombocytopenia and chemotherapy-induced thrombocytopenia. This suggests that treatments targeting platelet production can be effective for low platelet counts. ^{1 2 3 4 5}

How is the drug Ianalumab different from other treatments for low platelet count?

Ianalumab is unique because it targets the immune system in a specific way, potentially offering a novel approach compared to existing treatments that focus on increasing platelet production or reducing antiplatelet antibodies. This could provide a new option for patients who do not respond well to current therapies. ^{6 7 8 9 10}

Research Team

Novartis Pharmaceuticals

Principal Investigator

Novartis Pharmaceuticals

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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

Findings from Research

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]

References

1. United States pubmed.ncbi.nlm.nih.gov

Real-world experience with caplacizumab in the management of acute TTP. [2021]

2. Spain pubmed.ncbi.nlm.nih.gov

Eltrombopag in chronic idiopathic thrombocytopenic purpura and HCV-related thrombocytopenia. [2017]

3. England pubmed.ncbi.nlm.nih.gov

Avatrombopag for chemotherapy-induced thrombocytopenia in patients with non-haematological malignancies: an international, randomised, double-blind, placebo-controlled, phase 3 trial. [2022]

4. England pubmed.ncbi.nlm.nih.gov

Additional efficacy analysis of avatrombopag phase III data for the treatment of adults with immune thrombocytopenia. [2023]

5. United States pubmed.ncbi.nlm.nih.gov

Romiplostim or standard of care in patients with immune thrombocytopenia. [2016]

6. England pubmed.ncbi.nlm.nih.gov

The effect of anti-CD40 ligand in immune thrombocytopenic purpura. [2019]

7. United States pubmed.ncbi.nlm.nih.gov

Anchoring IgG-degrading enzymes to the surface of platelets selectively neutralizes antiplatelet antibodies. [2023]

8. England pubmed.ncbi.nlm.nih.gov

Clinical efficacy and safety of Flebogammadif, a new high-purity human intravenous immunoglobulin, in adult patients with chronic idiopathic thrombocytopenic purpura. [2022]

9. Turkey pubmed.ncbi.nlm.nih.gov

A Multi-Center Study on the Efficacy of Eltrombopag in Management of Refractory Chronic Immune Thrombocytopenia: A Real-Life Experience [2020]

10. Spain pubmed.ncbi.nlm.nih.gov

Caplacizumab to treat immune-mediated thrombotic thrombocytopenic purpura. [2019]