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:
TBC
Drug: OM336
ClinicalTrials.gov ID: NCT07083960
Pharma Trial ID: OM336-AIC-1001
Ouro Medicines
An early-phase clinical trial evaluating OM336 in adult participants with autoimmune cytopenias.
An open-label, multicenter, ascending dose study, evaluating safety, tolerability, and PK/ADA of OM336 in autoimmune cytopenias, including Immune Thrombocytopenia (ITP), Warm Autoimmune Hemolytic Anemia (wAIHA) and Cold Autoimmune Hemolytic Anemia (cAIHA).
This study is a dose-escalating trial where participants will receive OM336 via subcutaneous injection and will run for 12 weeks, with follow-up over 52 weeks (12 months)
During the study, participants will visit their study clinic several times.
Not Yet Recruiting.
Locations:
TBC
Drug: Mezagitamab
ClinicalTrials.gov ID: NCT06722235
Pharma Trial ID: TAK-079-3002
Takeda
A Study of Mezagitamab in Adults With Chronic Primary Immune Thrombocytopenia
Primary immune thrombocytopenia (ITP) is a condition where the immune system mistakenly destroys platelets, which are cells that help stop bleeding. This leads to a low number of platelets, making it easier to bruise or bleed. The main aim of this study is to learn whether mezagitamab, when given just under the skin (subcutaneously [SC]), is effective in keeping the platelet count of adults with ITP stable when compared to a placebo. A placebo looks like medicine but doesn’t have any active ingredients in it.
The participants will be treated with mezagitamab for up to 6 months.
During the study, participants will visit their study clinic several times.
Recruiting.
Locations:
Adelaide, SA (recruiting soon)
Canberra, ACT (recruiting soon)
Frankston, VIC (recruiting soon)
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).
At the end of Part 1, 2 dose levels will be selected to be used in the dose-optimization stage (Part 2) of the study. In Part 2 of the study, subjects will be randomized in a 1:1 ratio between the 2 dose levels to better understand the exposure/efficacy/toxicity relationship.
At the end of Part 2, the Recommended Phase 3 dose (RP3D) of HMPL-523 will be determined based on the safety, efficacy and PK data.
Recruiting.
Locations:
Douglas, QLD,
Concord, NSW
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:
Canberra, ACT
Woolloongabba, QLD
Clayton, VIC
Melbourne, VIC
Drug: Ianalumab
ClinicalTrials.gov ID: NCT05653349
Pharma Trial ID: CVAY736I12301
Novartis Pharmaceuticals
Ianalumab (VAYHIT1)
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 (VAYHIT2)
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.
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.
Active, Not 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 participants 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. Participants will receive rilzabrutinib or placebo 400mg twice daily.
For each participant, 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.
For adult participants, the maximum duration of the long-term extension (LTE) period will be 12 months from the date of the last adult participant to enter the LTE.
For pediatric participants, the maximum duration of the LTE period will be 12 months from the date of the last pediatric participant to enter the LTE.
Active, Not recruiting.
Locations:
Canberra, 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:
Melbourne, 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.
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.
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