Clinical Trials

Multiple clinical trials spanning Phase 1 to Phase 3 evaluate pelabresib (CPI-0610) primarily for hematological malignancies, bone marrow disorders, and solid tumors. Sponsored by industry and academic entities, these studies reflect varying recruitment statuses, including completed, recruiting, withdrawn, and not yet recruiting. Additionally, Phase 1 trials assess pharmacokinetics, drug-drug interactions, and food effects in healthy participants and patients with advanced malignancies.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT07422610 RECRUITING
Advanced Malignancies and Hepatic Impairment
Novartis Pharmaceuticals
 2026-04-24 PHASE1
NCT07357727 RECRUITING
Primary Myelofibrosis (PMF); Post-polycythemia Vera Myelofibrosis (PPV-MF); Post-essential Thrombocythemia Myelofibrosis (PET-MF)
Novartis Pharmaceuticals
 2026-06-16 PHASE3
NCT07340190 RECRUITING
Advanced Malignancies
Novartis Pharmaceuticals
 2026-06-12 PHASE1
NCT06401356 RECRUITING
Hematologic Malignancy; Solid Tumor; Advanced Malignancies
Novartis Pharmaceuticals
 2024-08-13 PHASE3
NCT07340138 RECRUITING
Primary Myelofibrosis (PMF); Post-polycythemia Vera Myelofibrosis (Post-PV MF); Post-essential Thrombocythemia Myelofibrosis (Post-ET MF)
Novartis Pharmaceuticals
 2026-04-15 PHASE1
NCT07783503 NOT_YET_RECRUITING
Healthy Participants
Novartis Pharmaceuticals
 2026-08-27 PHASE1
NCT02158858 COMPLETED
Myelofibrosis; Leukemia, Myelocytic, Acute; Myelodysplastic/Myeloproliferative Neoplasm; Myelodysplastic Syndrome (MDS); Preleukemia; Primary Myelofibrosis; Myeloproliferative Disorders; Bone Marrow Disease; Hematological Disease; Precancerous Conditions; Neoplasms; Leukemia; Neoplasms by Histologic Type; Essential Thrombocytosis
Constellation Pharmaceuticals
 2014-07-16 PHASE1; PHASE2
NCT04603495 ACTIVE_NOT_RECRUITING
Myelofibrosis; Primary Myelofibrosis; Post-polycythemia Vera Myelofibrosis; Post-essential Thrombocythemia Myelofibrosis
Novartis Pharmaceuticals
 2021-04-09 PHASE3
NCT05391022 COMPLETED
Advanced Malignancies; Solid Tumor; Hematological Malignancy
Constellation Pharmaceuticals
 2021-07-20 PHASE1
NCT05391022 Completed
Advanced Malignancies|Solid Tumor|Hematological Malignancy
Constellation Pharmaceuticals
 2021-07-20 Phase 1
NCT02986919 WITHDRAWN
Peripheral Nerve Tumors
University of Texas Southwestern Medical Center
 2017-05-05 PHASE2
NCT01949883 COMPLETED
Lymphoma
Constellation Pharmaceuticals
 2013-09-05 PHASE1
NCT02986919 Withdrawn
Peripheral Nerve Tumors
University of Texas Southwestern Medical Center
 2017-05-05 Phase 2
NCT02157636 COMPLETED
Multiple Myeloma
Constellation Pharmaceuticals
 2014-07-15 PHASE1
NCT02157636 Completed
Multiple Myeloma
Constellation Pharmaceuticals|The Leukemia and Lymphoma Society
 2014-07-15 Phase 1
NCT01949883 Completed
Lymphoma
Constellation Pharmaceuticals|The Leukemia and Lymphoma Society
 2013-09-05 Phase 1

(data from https://clinicaltrials.gov, updated on 2026-08-18)

Check the Pelabresib (CPI-0610) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Pelabresib (CPI-0610) is a potent, selective benzoisoxazoloazepine inhibitor of bromodomain and extra-terminal (BET) proteins, binding BRD4-BD1 with an IC50 of 39 nM to suppress the downstream expression of key target genes such as Nuclear receptor binding SET domain protein 3 (NSD3). This targeted transcriptional blockade disrupts oncogenic gene expression networks and halts cancer cell proliferation, providing therapeutic utility in inhibiting disease progression in hematological malignancies and myelofibrosis.

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.