Clinical Trials

Prelude Therapeutics has sponsored several completed Phase I clinical trials evaluating PRT543 for dose escalation and expansion in patients with advanced malignancies. These studies investigated the compound across relapsed or refractory solid tumors, adenoid cystic carcinoma, and various hematologic neoplasms, including diffuse large B-cell lymphoma, mantle cell lymphoma, acute myeloid leukemia, chronic myelomonocytic leukemia, myelodysplasia, and myelofibrosis. Overall, these early-stage clinical evaluations established initial clinical parameters for PRT543 in heavily pretreated patient populations.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT03886831 COMPLETED
Relapsed/Refractory Advanced Solid Tumors; Relapsed/Refractory Diffuse Large B-cell Lymphoma; Relapsed/Refractory Myelodysplasia; Relapsed/Refractory Myelofibrosis; Adenoid Cystic Carcinoma; Relapsed/Refractory Mantle Cell Lymphoma; Relapsed/Refractory Acute Myeloid Leukemia; Refractory Chronic Myelomonocytic Leukemia
Prelude Therapeutics
 2019-02-11 PHASE1
NCT03886831 Completed
Relapsed/Refractory Advanced Solid Tumors|Relapsed/Refractory Diffuse Large B-cell Lymphoma|Relapsed/Refractory Myelodysplasia|Relapsed/Refractory Myelofibrosis|Adenoid Cystic Carcinoma|Relapsed/Refractory Mantle Cell Lymphoma|Relapsed/Refractory Acute Myeloid Leukemia|Refractory Chronic Myelomonocytic Leukemia
Prelude Therapeutics
 2019-02-11 Phase 1

(data from https://clinicaltrials.gov, updated on 2023-03-28)

Check the PRT543 product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

PRT543 selectively binds to protein arginine methyltransferase 5 (PRMT5) and inhibits the enzymatic activity of the PRMT5/MEP50 complex with an IC50 of 10.8 nM, thereby blocking arginine dimethylation on protein substrates to induce cell cycle arrest and apoptosis. This inhibition of oncogenic arginine methylation underlies the compound's therapeutic activity in relapsed or refractory advanced solid tumors and hematologic malignancies, including diffuse large B-cell lymphoma and acute myeloid leukemia.

Appendix RUO and cGMP Quality Standards

Quality Dimension RUO (Research Use Only) cGMP (Current Good Manufacturing Practice)
Clinical Applicability Prohibited in human clinical trials or medical diagnostics. Mandatory for human clinical trials (Phase I–III) and therapies.
Regulatory Status Non-regulated grade; exempt from drug manufacturing laws. Legally enforced by health authorities (e.g., FDA, EMA, NMPA).
Facility Environment Unclassified analytical or research laboratories. Validated Cleanrooms (ISO Class 5–8) with continuous monitoring.
Quality Control Basic purity and activity testing. Rigorous safety release testing (Sterility, Endotoxin, Mycoplasma).
Process Validation Basic equipment calibration; no process validation required. Full qualification (IQ/OQ/PQ) and complete batch records.
Quality Assurance Vendor self-declared without required formal QMS. Mandatory QA/QC unit, Change Control, CAPA, and vendor audits.
Regulatory Impact High risk of IND rejection if used as a critical raw material. Required for IND/NDA filings, supported by Drug Master Files (DMF).

Footnotes

Regulatory Note: Governed by FDA (21 CFR Parts 210/211/312), EMA (EudraLex Vol 4), ICH Guidelines (E6/Q7/Q9/Q10), and compendial standards (USP <71>/<85>/<1043>).

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Note: Technical data last updated: Sep 1, 2026.