Clinical trial · Interventional
A Study of PRT543 in Participants With Advanced Solid Tumors and Hematologic Malignancies
A Phase 1, Open-Label, Multicenter, Dose Escalation, Dose Expansion Study of PRT543 in Patients With Advanced Solid Tumors and Hematologic Malignancies
NCT03886831CI-TRIAL-00065070completedPhase 1ClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
This is a Phase 1 cohort, dose-escalation, dose-expansion study of PRT543 in patients with advanced cancers who have exhausted available treatment options. The purpose of this study is to define a safe dose and schedule to be used in subsequent development of PRT543.
Conditions
Conditions (8)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Adenoid Cystic Carcinoma | Adenoid Cystic Carcinoma | ONTOLOGY_EXACT | 0.98 |
| Refractory Chronic Myelomonocytic Leukemia | Chronic Myelomonocytic Leukemia | CURATED_BROADER | 0.78 |
| Relapsed/Refractory Acute Myeloid Leukemia | — | UNRESOLVED | — |
| Relapsed/Refractory Advanced Solid Tumors | — | UNRESOLVED | — |
| Relapsed/Refractory Diffuse Large B-cell Lymphoma | — | UNRESOLVED | — |
| Relapsed/Refractory Mantle Cell Lymphoma | — | UNRESOLVED | — |
| Relapsed/Refractory Myelodysplasia | — | UNRESOLVED | — |
| Relapsed/Refractory Myelofibrosis | — | UNRESOLVED | — |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| PRT543 | Drug | — | UNRESOLVED |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- PRT543
- description
- PRT543 will be administered orally
- interventionNames
- Drug: PRT543
Primary outcomes (3)
- measure
- To describe dose limiting toxicities (DLT) of PRT543
- timeFrame
- Baseline through Day 28.
- description
- Dose limiting toxicities (DLTs) will be evaluated during the first cycle
- measure
- To determine the maximally tolerated dose (MTD)
- timeFrame
- Baseline through approximately 2 years.
- description
- The maximum tolerated dose (MTD) will be established for further investigation in participants with advanced malignancies who have failed prior treatments.
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
Show eligibility criteria text
Inclusion Criteria: * Metastatic or advanced solid tumor; or advanced diffuse large B-cell lymphoma; or advanced mantle cell lymphoma; or relapsed myelodysplastic syndrome, acute myeloid leukemia or chronic myelomonocytic leukemia; or relapsed myelofibrosis. All malignancies must be refractory to established therapies * Biomarker-selected solid tumors * Eastern Cooperative Oncology Group (ECOG) Performance Score of 0 or 1 * Adequate organ function (bone marrow, hepatic, renal, cardiovascular) * Female patients of childbearing potential must have a negative pregnancy test within 7 days of the start of treatment and must agree to use an effective method of contraception during the trial Exclusion Criteria: * Primary malignancies of the Central Nervous System(CNS) or uncontrolled CNS metastases * Requirement of pharmacologic doses of glucocorticoids * Prior treatment with chimeric antigen receptor T cells (CAR-T cells) * HIV positive; known active hepatitis B or C * Known hypersensitivity to any of the components of PRT543 * Prior allogeneic bone marrow transplant; autologous hematopoietic transplantation less than 100 days since transplantation
References
Publications (2)
- DERIVEDMaron MI, Abdel-Wahab O. Outstanding Questions to Understand and Target Splicing Factor-Mutant Blood Cancers. Blood Cancer Discov. 2026 May 5;7(3):348-352. doi: 10.1158/2643-3230.BCD-26-0047. PMID 42013378
- DERIVEDFerrarotto R, Swiecicki PL, Zandberg DP, Baiocchi RA, Wesolowski R, Rodriguez CP, McKean M, Kang H, Monga V, Nath R, Palmisiano N, Babbar N, Sun W, Hanna GJ. PRT543, a protein arginine methyltransferase 5 inhibitor, in patients with advanced adenoid cystic carcinoma: An open-label, phase I dose-expansion study. Oral Oncol. 2024 Feb;149:106634. doi: 10.1016/j.oraloncology.2023.106634. Epub 2023 Dec 20. PMID 38118249