RecruitingInterventionalPhase 1

A Phase 1a/1b Study of ELVN-001 for the Treatment of Chronic Myeloid Leukemia

NCT ID: NCT05304377Sponsor: Enliven TherapeuticsLast updated: 2026-04-01

Summary

The purpose of this study is to evaluate the safety, tolerability and determine the recommended dose for further clinical evaluation of ELVN-001 in patients with chronic myeloid leukemia with and without T315I mutations in patients who are relapsed, refractory or intolerant to TKIs.

Detailed description

This first-in-human trial with ELVN-001 is a dose escalation study with the primary purpose to identify the recommended dose(s) for expansion (RDEs) of single agent ELVN-001 in chronic phase CML with or without T315I mutations. The safety, tolerability and pharmacokinetic profile of ELVN-001 will be assessed together with an evaluation of changes in BCR-ABL1 transcript. An understanding of the safety profile, PK and preliminary evidence of anti-CML activity will be used to inform future development of ELVN-001 in adults with CML. By virtue of its predicted pharmacological profile ELVN-001 has the potential to be tolerable and achieve a deep molecular response in patients with CML with or without T315I mutations who do not tolerate or benefit from available TKIs.

Arms & interventions

DrugELVN-001
orally once or twice daily

Outcome measures

Primary

Phase 1a: Incidence of dose limiting toxicities
DLTs will be used to support that the recommended doses for expansion are \</= MTD
Time frame: 28 days
Phase 1a: Incidence of adverse events (AEs)
Adverse events will be used to support that the recommended doses for expansion are likely to be tolerable
Time frame: up to 28 days
Phase 1a: Incidence of clinically significant laboratory abnormalities
Clinically significant laboratory abnormalities will be used to support that the recommended doses for expansion are likely to be tolerable
Time frame: up to 28 days
Phase 1a: Incidence of clinically significant ECG abnormalities
Clinically significant ECG abnormalities will be used to support that the recommended doses for expansion are likely to be tolerable
Time frame: up to 28 days
Phase 1b: Incidence of adverse events
Adverse events will be used to support that the dose(s) evaluated in expansion is tolerable
Time frame: up to 3 years
Phase 1b: Incidence of clinically significant laboratory abnormalities
Clinically significant ECG abnormalities will be used to support that the dose(s) evaluated in expansion is tolerable
Time frame: up to 3 years
Phase 1b: Incidence of clinically significant ECG abnormalities
Clinically significant ECG abnormalities will be used to support that the recommended dose(s) evaluated in expansion is tolerable
Time frame: up to 3 years

Secondary

Phase 1a and 1b: area under the curve
Time frame: 6 months
Phase 1a and 1b: maximum concentration
Time frame: 6 months
Phase 1a and 1b: time of maximum concentration
Time frame: 6 months
Phase 1a and 1b: minimum concentration
Time frame: 6 months
Phase 1a and 1b: Molecular response (MR)
Time frame: up to 3 years
Phase 1b: Duration of Molecular Response
Time frame: up to 3 years
Phase 1b: Complete Hematologic Response (CHR)
Time frame: up to 3 years

Eligibility criteria

Sex: AllAge: 18 Years and olderHealthy volunteers: No

Inclusion Criteria: * BCR-ABL1 positive CML in chronic phase, with or without T315I mutation. * US: The patient has failed or is intolerant to at least one prior second-generation tyrosine kinase inhibitor (TKI) or asciminib. Rest of World: The patient has failed, is intolerant to, or not a candidate for, available therapies known to be active for treatment of their CML (country-specific criteria may vary). * ECOG performance status of 0 to 2. * Adequate hematologic, hepatic and renal function. * Prior bone marrow transplant allowed if ≥ 6 months prior to the first dose of ELVN-001. Exclusion Criteria: * Treatment with anti-cancer or anti-CML therapy within 7 days or 5 half-lives, whichever is longer. * History of acute tyrosine kinase inhibitor (TKI)-related pancreatitis within 6 months of study entry. Active chronic pancreatitis, or pancreatic disease due to any cause. * QTc \>470 ms.

Study locations (4)

Memorial Sloan Kettering Cancer Center

New York, New York, 10065

Recruiting

Michael Mauro, MD · Contact

+1 347 798 9213

Montefiore Medical Center

The Bronx, New York, 10461

Recruiting

David Levitz MD · Contact

718-920-4321

Oregon Health & Science University-Knight Cardiovascular Institute

Portland, Oregon, 97239

Recruiting

Michael Heinrich, MD · Contact

(503) 418-1964

The University of Texas MD Anderson Cancer Center

Houston, Texas, 77030

Recruiting

Fadi Haddad, MD · Contact

877-632-6789