A Phase 3 Study of INCA033989 Versus Best Available Therapy in Participants With Essential Thrombocythemia
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: INCA033989, Best Available Treatment.
- Who it may be relevant to
- Registry conditions: Essential Thrombocythemia. Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Australia, Austria, Belgium, Canada +13
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
A Phase 3, Randomized, Open-Label Study of INCA033989 Versus Best Available Therapy in Participants With Essential Thrombocythemia and a CALR Mutation Previously Treated With Cytoreductive Therapy (EXCALIBUR-ET2)
Overview
This study is being conducted to evaluate INCA033989 versus best available therapy in participants with essential thrombocythemia and a CALR mutation previously treated with cytoreductive therapy.
Interventions
- Drug INCA033989
Administered intravenous (IV) in accordance with the protocol-defined requirements. - Drug Best Available Treatment
Best Available Therapy (BAT) will be selected by the investigator.
Primary outcome measures
- Durable clinicohematologic response (DCR) [Time frame: Week 24]
Secondary outcome measures (12)
- Reduction from baseline in calreticulin exon 9 frameshift mutation(s) (mutCLAR) variant allele frequency (VAF) [Time frame: Week 24]
- Durable clinicohematologic response (DCR) [Time frame: Week 48]
- Durable partial clinicohematologic response (DPR) [Time frame: Week 24]
- Durable partial clinicohematologic response (DPR) [Time frame: Week 48]
- Longest duration of complete hematologic response (CHR) [Time frame: Up to Week 48]
- Number of Participants with Treatment Emergent Adverse Events (TEAE) [Time frame: Up to Week 48 and 60 days after last dose]
- TEAEs leading to dose interruptions, dose reductions or discontinuation of study treatment [Time frame: Up to Week 48 and 60 days after last dose]
- Number of participants with a reduction in mutCALR VAF [Time frame: Week 24 and Week 48]
- Molecular response [Time frame: Week 24 and Week 48]
- Change from baseline in Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) total symptom score (TSS) [Time frame: Up to Week 48]
- Change from baseline in Brief Fatigue Inventory (BFI) fatigue score [Time frame: Up to Week 48]
- Patient Global Impression of Change (PGIC) score [Time frame: Up to Week 48]
Eligibility criteria
Inclusion criteria
- Confirmed diagnosis of high-risk ET.
- Presence of mutCALR.
- Prior treatment with at least 1 cytoreductive therapy.
Exclusion criteria
- Presence of any hematologic malignancy other than ET.
- Major bleeding or thrombosis within the last 3 months prior to study enrollment.
- Any prior allogenic or autologous stem-cell transplantation.
- Unresolved toxicity ≥ Grade 2 from previous therapy except for stable chronic toxicities (Grade 2) not expected to resolve, such as stable Grade 2 peripheral neuropathy.
- Prior nonhematologic malignancy except for the following: Malignancy treated with curative intent and with no evidence of active disease for more than 2 years before screening. Adequately treated carcinoma in situ without current evidence of disease.
Other protocol-defined Inclusion/Exclusion Criteria apply.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
United States · 44 centers
- Banner Md Anderson Cancer Center — Gilbert
- The University of Arizona Cancer Center — Tucson
- City of Hope Medical Center — Duarte
- Marin Cancer Care — Greenbrae
- University of California, Los Angeles Medical Center — Los Angeles
- Stanford Cancer Center — Palo Alto
- UCSF — San Francisco
- Rocky Mountain Cancer Centers-Lone Tree — Lone Tree
- … and 36 more centers
Italy · 16 centers
Center list to be confirmed — check the primary protocol.
Japan · 15 centers
Center list to be confirmed — check the primary protocol.
Spain · 15 centers
Center list to be confirmed — check the primary protocol.
Germany · 13 centers
Center list to be confirmed — check the primary protocol.
Australia · 9 centers
- Blacktown Cancer & Haematology Centre-Blacktown Hospital — Blacktown
- Gosford Hospital (Gh) - Central Coast Cancer Centre — Gosford
- Liverpool Hospital - South Western Sydney Local Health District — Liverpool
- Sunshine Coast University Hospital — Birtinya
- Royal Brisbane and Women'S Hospital — Brisbane
- Peter Maccallum Cancer Centre — Melbourne
- Box Hill Hospital — Melbourne
- The Canberra Hospital — Garran
- … and 1 more center
France · 9 centers
- Chu D'Amiens-Picardie - Hopital Sud — Amiens
- Institut Bergonie - Centre Regional de Lutte Contre Le Cancer de Bordeaux Et Sud Ouest — Bordeaux
- Chu de Brest Hopital de La Cavale Blanche Cic Hematologie — Brest
- Hospital Henri Mondor — Créteil
- … and 5 more centers
United Kingdom · 8 centers
Center list to be confirmed — check the primary protocol.
Canada · 7 centers
- St. Paul'S Hospital (Sph), Providence Health Care, University of British Columbia — Vancouver
- Margaret and Charles Juravinski Cancer Center — Hamilton
- Queens University, Kingston General Hospital (Kgh), Kingston Health Sciences Centre (Khsc) — Kingston
- University Health Network (Uhn) - Princess Margaret Cancer Centre — Toronto
- Sir Mortimer B. Davis - Jewish General Hospital — Montreal
- McGill University Health Centre - Glen Site, Centre For Innovative Medicine — Montreal
- Hotel-Dieu de Levis — Québec
Poland · 7 centers
Center list to be confirmed — check the primary protocol.
South Korea · 7 centers
Center list to be confirmed — check the primary protocol.
Belgium · 6 centers
- Jules Bordet Lnstitute — Anderlecht
- Centre Hospitalier Universitaire (Chu) Brugmann (Brugmann University Hospital (Uh)) - Vict — Brussels
- Universitair Ziekenhuis Leuven — Leuven
- Centre Hospitalier Universitaire de Liege - Sart Tilman — Liège
- Algemeen Ziekenhuis Deltaaretaie — Roeselare
- Universite Catholique de Louvain (Ucl) - Cliniques Universitaires Saint-Luc — Woluwe-Saint-Lambert
Austria · 5 centers
- Lkh - Medizinische Universitaet Graz — Graz
- Landeskrankenhaus (Lkh) Leoben - Standort Leoben — Leoben
- Ordensklinikum Linz Gmbh Elisabethinen — Linz
- Medizinische Universitaet Wien, Akh Wien — Vienna
- Klinikum Wels-Grieskirchen Gmbh — Wels
Czechia · 5 centers
- Fakultni Nemocnice Brno — Brno
- Fakultni Nemocnice Ostrava — Ostrava-poruba
- Faculty Hospital Kralovske Vinohrady — Prague
- Vseobecna Fakultni Nemocnice V Praze — Prague
- Ustav Hematologie A Krevni Transfuze (Uhkt) — Prague
Netherlands · 5 centers
Center list to be confirmed — check the primary protocol.
Switzerland · 4 centers
Center list to be confirmed — check the primary protocol.
Hungary · 3 centers
Center list to be confirmed — check the primary protocol.
Norway · 2 centers
Center list to be confirmed — check the primary protocol.
Identifiers
NCT: NCT07623200 · INCA033989-304 · 2026-525398-38-00