A Study of PRT543 in Participants With Advanced Solid Tumors and Hematologic Malignancies

Prelude Therapeutics

Status and phase: Completed; Phase 1

Conditions: Relapsed/Refractory Myelodysplasia; Relapsed/Refractory Mantle Cell Lymphoma; Relapsed/Refractory Diffuse Large B-cell Lymphoma; Adenoid Cystic Carcinoma; Relapsed/Refractory Myelofibrosis; Relapsed/Refractory Advanced Solid Tumors; Relapsed/Refractory Acute Myeloid Leukemia; Refractory Chronic Myelomonocytic Leukemia

Treatments: Drug: PRT543

Study type: Interventional

Funder types: Industry

Identifiers

NCT03886831

PRT543-01

Details and patient eligibility

About

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.

Full description

This is a multicenter, open-label, sequential-cohort, dose-escalation, dose-expansion Phase 1 study of PRT543 in patients with advanced cancers who have exhausted available treatment options. Enrollment will take place concurrently into two distinct patient groups (one for solid tumors/lymphomas and one for hematological malignancies). The study will consist of 2 parts, a dose escalation part, and once the recommended phase 2 dose (RP2D) has been determined, a cohort expansion part involving up to ten separate cohorts. For patients, the study will include a screening phase, a treatment phase, and a post treatment follow-up phase. An end-of-study visit will be conducted within 30 days after the last dose of PRT543.

Enrollment: 232 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Sequential Assignment

Masking: None (Open label)

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232 participants in 1 patient group

PRT543

Experimental group

Description:

PRT543 will be administered orally

Treatment:

Drug: PRT543