ITP Clinical Trials

Clinical trials are research investigations in which people volunteer to test new treatments, interventions or tests as a means to prevent, detect, treat or manage various diseases or medical conditions. Some investigations look at how people respond to a new intervention and what side effects might occur while others help to determine the best dose of a particular treatment is effective.

There are a number of Clinical Trials available in Australia for the treatment of Immune Thrombocytopenia, which further information available below.
In addition, there are clinical trials being held internationally. You can review a complete list by visiting ClinicalTrials.gov.

Clinical Trial Register

Status

Trial

Sponsor

Trial Summary

Not yet recruiting.

Locations:
Frankston, VIC
West Perth, WA

Drug: HMPL-523

ClinicalTrials.gov ID: NCT06291415

Pharma Trial ID: 2022-523-GLOB1

Hutchmed

HMPL-523

This is an open-label, multicenter study to evaluate the safety, tolerability, and efficacy of HMPL-523 in adult subjects with ITP.

This study is a Phase 1b, open-label, multicenter, single-arm study to evaluate the safety, tolerability, and preliminary efficacy of HMPL-523 in adult subjects with primary ITP diagnosed at least 3 months prior to enrollment or randomization.

In the dose escalation stage (Part 1), subjects will receive one of 3 dose levels of HMPL-523 to determine the recommended dose of HMPL-523 for the randomized dose optimization- stage (Part 2).

Recruiting.

Locations:
Prahran, VIC
Canberra, ACT

Drug: Ianalumab

ClinicalTrials.gov ID: NCT05885555

Pharma Trial ID: CVAY736Q12201

Novartis Pharmaceuticals

Ianalumab

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 count <30 G/L at screening) previously treated with at least a corticosteroid and a TPO-RA.

The study consists of the screening period, the primary endpoint assessment period, the follow-up period. The screening period will last for up to 14 days prior to the first dose of ianalumab. All eligible participants will be treated with the same dose of ianalumab and will complete the primary endpoint assessment period. After completion of the primary endpoint assessment period, all participants will continue in safety monitoring and those with a platelet count ≥30 G/L in absence of a new line of ITP therapy and rescue therapy will also continue in efficacy monitoring. The study will end once all participants have completed 24 months of safety follow-up since their last dose of ianalumab or discontinued the study earlier.

Recruiting.

Locations:
Douglas, QLD,
Liverpool, NSW
Westmead, NSW
Box Hill, VIC
West Perth, WA

Drug: povetacicept (ALPN-303)

ClinicalTrials.gov ID: NCT05757570

Pharma Trial ID: AIS-D04

Alpine Immune Sciences, Inc.

RUBY-4

The goal of this clinical study is to evaluate povetacicept (ALPN-303) in adults with autoimmune cytopenias of immune thrombocytopenia, autoimmune hemolytic anemia, and cold agglutinin disease to determine if povetacicept is safe and potentially beneficial in treating these diseases. During the study treatment period participants will receive povetacicept approximately every 4 weeks for 6 months, with the possibility of participating in a 6-month study treatment extension period.

Recruiting.

Locations:
Adelaide, SA
Frankston, VIC
Kogarah, NSW
Westmead, NSW
Perth, WA
South Brisbane, QLD

Drug: Rilzabrutinib (PRN1008)

ClinicalTrials.gov ID: NCT04562766

Pharma Trial ID: PRN1008-018

Sanofi

LUNA 3 – Rilzabrutinib

This is a randomized, double-blind study of rilzabrutinib in patients with persistent or chronic ITP, with an average platelet count of <30,000/μL (and no single platelet count >35,000/μL) on two counts at least 5 days apart in the 14 days before treatment begins. Patients will receive rilzabrutinib or placebo 400mg twice daily.

For each patient, the study will last up to 60 weeks from the start of the Screening Period to the End of Study (EOS) visit. This includes Screening (up to 4 weeks) through a 12 to 24-week Blinded Treatment Period followed by a 28-week Open-Label Period. Followed by a 4-week post dose follow-up.

Patients who respond per specified criteria at the end of the Open-Label Period will be able to enter the Long-Term Extension (LTE) where they will continue to receive treatment until the last patient who enters the LTE has completed 12 months.

Recruiting.

Locations:
Kogarah, NSW
Liverpool, NSW
Mount Kuring-Gai, NSW
Waratah, NSW
Bedford Park, SA
Melbourne, VIC
Perth, WA

Drug: PF-06835375

ClinicalTrials.gov ID: NCT05070845

Pharma Trial ID: C1131003

Pfizer

PF-06835375

An Interventional Phase 2, Open-label, multi-centre study to evaluate safety and efficacy of PR-06835375 in adult participants with moderate to severe primary Immune Thrombocytopenia.

This is a Phase 2, open-label, multicenter, multiple subcutaneous injections, safety and efficacy study of PF-06835375 in adult participants with primary immune thrombocytopenia (ITP). This study will focus on participants with persistent (>3 months and ≤12 months) or chronic (>12 months) ITP.

Recruiting.

Locations:
Canberra, ACT
Woolloongabba, QLD
Clayton, VIC
Prahran, VIC

Drug: Ianalumab

ClinicalTrials.gov ID: NCT05653349

Pharma Trial ID: CVAY736I12301

Novartis Pharmaceuticals

Ianalumab

A Phase III, Randomized, Double-blind Study of Ianalumab (VAY736) Versus Placebo in Addition to First-line Corticosteroids in Primary Immune Thrombocytopenia (VAYHIT1)

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.

Recruiting.

Locations:
Clayton, VIC
Parkville, VIC

Drug: Ianalumab

ClinicalTrials.gov ID: NCT05653219

Pharma Trial ID: CVAY736Q12301

Novartis Pharmaceuticals

Ianalumab

A Phase 3 Randomized, Double-blind Study of Ianalumab (VAY736) Versus Placebo in Addition to Eltrombopag in Patients With Primary Immune Thrombocytopenia (ITP) Who Had an Insufficient Response or Relapsed After First Line Steroid Treatment (VAYHIT2).

This is a multicenter, randomized, double-blinded phase 3 study to assess efficacy and safety of two different doses of ianalumab versus placebo in addition to eltrombopag in adults with primary ITP (platelet count <30 G/L) who failed previous first-line treatment with corticosteroids.

Active, Not recruiting.

Locations:
Garran, ACT
Concord, NSW
Woolloongabba, QLD
Clayton, VIC
Parkville, VIC
Perth, WA

Drug: Rilzabrutinib (PRN1008)

ClinicalTrials.gov ID: NCT03395210

Pharma Trial ID: PRN1008-010

Sanofi

Rilzabrutinib – Oral BTK Inhibitor

An Adaptive, Open-Label, Dose-Finding, Phase 1/2 Study Investigating the Safety, Pharmacokinetics, and Clinical Activity of PRN1008, an Oral BTK Inhibitor, in Patients With Relapsed Immune Thrombocytopenia.

This is a 2 part (Part A and B) adaptive, open-label, dose-finding study of PRN1008 in patients with ITP who are refractory or relapsed with no available and approved therapeutic options, with a platelet count <30,000/μL on two counts, no sooner than 7 days apart in the 15 days before treatment begins. The dose-finding portion of the study has been completed. Part B treatment dose is 400 mg twice daily.

Active, Not recruiting.

Locations:
Garran, ACT
West Perth, WA
Westmead, NSW

Drug: Efgartigimod PH20 SC

ClinicalTrials.gov ID: NCT04812925

Pharma Trial ID: ARGX-113-2005

Argenx

ADVANCESC+ Study

A Study to Evaluate the Efficacy and Safety of Efgartigimod PH20 Subcutaneous in Adult Patients With Primary Immune Thrombocytopenia.

This is a phase 3, multicenter, open-label, long-term trial to evaluate the efficacy of Efgartigimod (ARGX-113) PH20 Subcutaneous in adult patients with primary ITP.

Completed.

Locations:
Adelaide, SA
Bedford Park, SA
Box Hill, VIC
Clayton, VIC
Garran, ACT
Hobart, TAS
Perth, WA
West Perth, WA
Westmead, NSW

Drug: Efgartigimod PH20 SC

ClinicalTrials.gov ID: NCT04687072

Pharma Trial ID: ARGX-113-2004

Argenx

ADVANCESC Study

A Study to Evaluate the Efficacy and Safety of Efgartigimod PH20 Subcutaneous in Adult Patients With Primary Immune Thrombocytopenia.

This is a phase 3, multicenter, randomized, double-blinded, placebo-controlled, parallel-group trial to evaluate the efficacy, safety, and effect on QoL/PRO of efgartigimod PH20 SC treatment in adult patients with primary ITP.

Contact ITP Australia and New Zealand today to ensure your Clinical Trial is included in our register.

Platinum Sponsor

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Gold Sponsor

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Bronze Sponsor

Argenx
Sanofi