ITP Clinical Trials
Clinical trials are research investigations in which people volunteer to test new treatments, interventions, or tests 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 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, with further information available below.
In addition, clinical trials are being held internationally. You can review a complete list by visiting ClinicalTrials.gov.
Trial
Drug: OM336
ClinicalTrials.gov ID: NCT07083960
Pharma Trial ID: OM336-AIC-1001
Sponsor
Sponsor: Ouro Medicines
Locations
- Canberra Hospital, ACT
- Liverpool Hospital, NSW
- ICON Cancer Care Centre, South Brisbane, QLD
Trial Summary
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.
Trial
Drug: Tafasitamab (INCA000585)
ClinicalTrials.gov ID: NCT07104565
Pharma Trial ID: INCA000585-201
Sponsor: Incyte Corporation
Locations
- St Vincent’s Hospital, Darlinghurst, NSW
- Townsville University Hospital, QLD
- Princess Alexandra Hospital, Woolloongabba, QLD
- Box Hill Hospital, VIC
- Monash University Hospital, Clayton, VIC
- The Alfred Hospital, Melbourne, VIC
Trial Summary
A Phase 2a, Open-Label, Multicenter Study of Tafasitamab in Adult Participants With Primary Autoimmune Blood Cell Disorders.
This study will evaluate the safety and efficacy of tafasitamab in adult participants with primary autoimmune blood cell disorders, including Immune Thrombocytopenia and Warm Autoimmune Hemolytic Anemia (wAIHA).
Trial
Drug: Mezagitamab
ClinicalTrials.gov ID: NCT06948318
Pharma Trial ID: TAK-079-3002
Sponsor: Takeda
Locations
- Canberra Hospital, ACT
- Concord Repatriation General Hospital, NSW (Coming Soon)
- St George Hospital, NSW
- University of NSW – Liverpool Hospital – Liverpool Cancer Therapy Centre, NSW
- Westmead Hospital, NSW
- Peter MacCallum Cancer Centre, VIC (Coming Soon)
- Monash University and The Alfred Hospital, VIC
- Fiona Stanley Hospital, WA
- Perth Blood Institute, WA
Trial Summary
A Follow-up Study of Mezagitamab in Adults With Chronic Primary Immune Thrombocytopenia
Primary immune thrombocytopenia (ITP) is a condition in which the immune system mistakenly destroys platelets, the cells that help stop bleeding. This leads to a lower number of platelets, making it easier to bruise or bleed. The main aim of this study is to check how safe mezagitamab is and how well it is tolerated by adults with chronic primary ITP, if given over a longer time. Other aims are to learn how effective treatment with mezagitamab is and how the body processes it (called pharmacokinetics or PK) over a longer time.
Participants in the following previous mezagitamab studies will be invited to join this continuation study: TAK-079-3002 and TAK-079-1004. In this continuation study, participants will receive mezagitamab if they meet certain protocol criteria.
During the study, participants will visit their study clinic several times.
Trial
Drug: Mezagitamab
ClinicalTrials.gov ID: NCT06722235
Pharma Trial ID: TAK-079-3002
Sponsor: Takeda
Locations
- Canberra Hospital, ACT
- Concord Repatriation General Hospital, NSW
- St George Hospital, NSW
- University of NSW – Liverpool Hospital – Liverpool Cancer Therapy Centre
- Westmead Hospital, NSW
- Peter MacCallum Cancer Centre, VIC (recruiting soon)
- Monash University – Australia Centre for Blood Diseases, VIC
- The Alfred Hospital, VIC
- Fiona Stanley Hospital, WA
- Perth Blood Institute, WA
Trial Summary
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.
Trial
Drug: PF-06835375
ClinicalTrials.gov ID: NCT05070845
Pharma Trial ID: C1131003
Sponsor: Pfizer
Locations
- St George Hospital, NSW
- Liverpool Hospital, NSW
South West Radiology, NSW - Slade Pharmacy, Mount Kuring-Gai NSW
- Calvary Mater Newcastle, NSW
- Flinders Medical Centre, Bedford Park, SA
- ICON Cancer Centre, Kurralta Park, SA
- The Alfred Hospital, VIC
- Royal Perth Hospital, WA
Trial Summary
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.
Trial
Drug: Velinotamig
ClinicalTrials.gov ID: NCT07738822
Pharma Trial ID:
Sponsor: Cullinan Therapeutics
Locations
- To be confirmed
Trial Summary
A Phase 1b/2a, open-label dose escalation and dose expansion study of subcutaneously (SC) administered velinotamig for the treatment of adults with relapsing and refractory immune thrombocytopenia (ITP) and warm autoimmune hemolytic anemia (wAIHA) to evaluate safety and tolerability.
Trial
Drug: povetacicept (ALPN-303)
ClinicalTrials.gov ID: NCT05757570
Pharma Trial ID: AIS-D04
Sponsor: Alpine Immune Sciences, Inc.
Locations
- Douglas, QLD,
- Concord, NSW
- Liverpool, NSW
- Westmead, NSW
- Box Hill, VIC
- West Perth, WA
Trial Summary
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.
Trial
Drug: Ianalumab
ClinicalTrials.gov ID: NCT05653349
Pharma Trial ID: CVAY736I12301
Sponsor: Novartis Pharmaceuticals
Locations
- Canberra, ACT
- Woolloongabba, QLD
- Clayton, VIC
- Melbourne, VIC
Trial Summary
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.
Trial
Drug: Ianalumab
ClinicalTrials.gov ID: NCT05885555
Pharma Trial ID: CVAY736Q12301
Sponsor: Novartis Pharmaceuticals
Locations
- Canberra Hospital, ACT
- Melbourne, VIC
Trial Summary
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 trial includes an option to offer a second course of ianalumab treatment to participants who achieved confirmed response during the initial course of ianalumab and later lost response to explore the benefit of the second course of treatment. The study will end once all participants have completed 24 months of safety follow-up since their last dose of ianalumab (including the optional second course of ianalumab treatment),or discontinued the study earlier.
Trial
Drug: Ianalumab
ClinicalTrials.gov ID: NCT05653219
Pharma Trial ID: CVAY736Q12301
Sponsor: Novartis Pharmaceuticals
Locations
- Clayton, VIC
- Parkville, VIC
Trial Summary
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.
Trial
Drug: Rilzabrutinib (PRN1008)
ClinicalTrials.gov ID: NCT04562766
Pharma Trial ID: PRN1008-018
Sponsor: Sanofi
Locations
- Adelaide, SA
- Frankston, VIC
- Kogarah, NSW
- Westmead, NSW
- Perth, WA
- South Brisbane, QLD
Trial Summary
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.
Trial
Drug: Rilzabrutinib (PRN1008)
ClinicalTrials.gov ID: NCT03395210
Pharma Trial ID: PRN1008-010
Sponsor: Sanofi
Locations
- Canberra, ACT
- Concord, NSW
- Woolloongabba, QLD
- Clayton, VIC
- Parkville, VIC
- Perth, WA
Trial Summary
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.
Trial
Drug: Efgartigimod PH20 SC
ClinicalTrials.gov ID: NCT04812925
Pharma Trial ID: ARGX-113-2005
Sponsor: Argenx
Locations
- Garran, ACT
- West Perth, WA
- Westmead, NSW
Trial Summary
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.
Trial
Drug: Efgartigimod PH20 SC
ClinicalTrials.gov ID: NCT04687072
Pharma Trial ID: ARGX-113-2004
Sponsor: Argenx
Locations
- Adelaide, SA
- Bedford Park, SA
- Box Hill, VIC
- Clayton, VIC
- Garran, ACT
- Hobart, TAS
- Perth, WA
- West Perth, WA
- Westmead, NSW
Trial Summary
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.
Trial
Drug: HMPL-523
ClinicalTrials.gov ID: NCT06291415
Pharma Trial ID: 2022-523-GLOB1
Sponsor: Hutchmed
Locations
- Adelaide, SA (recruiting soon)
- Canberra, ACT (recruiting soon)
- Frankston, VIC (recruiting soon)
- West Perth, WA
Trial Summary
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.
Gold Sponsor
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