TerminatedPhase 1

Study of Elenestinib (BLU-263) in Advanced Systemic Mastocytosis (AdvSM) and and Other KIT Altered Hematologic Malignancies

Stopped: Terminated - alignment of strategic priorities

United States, Belgium9 participantsStarted 2023-09-25

Plain-language summary

The goal of this clinical trial is to evaluate elenestinib (BLU-263) in participants with Advanced Systemic Mastocytosis (AdvSM), SM with an associated hematologic neoplasm (SM-AHN), and other hematologic malignancies. The main questions it aims to answer are: * Determine Recommended Dose of elenestinib (BLU-263) monotherapy for participants with AdvSM * Safety and tolerability of elenestinib (BLU-263) monotherapy * Efficacy of elenestinib (BLU-263) monotherapy in participants with AdvSM * Determine Recommended Dose of elenestinib (BLU-263) in combination with azacitidine in participants with AdvSM * Safety and tolerability of elenestinib (BLU-263) in combination with azacitidine * Efficacy of elenestinib (BLU-263) in combination with azacitidine in participants with AdvSM The estimated study duration for each participant will be approximately 4 years: 2 years of treatment followed by 2 years of follow-up. Participants may be required to attend monthly visits for the first six months, followed by quarterly visits for the remainder of the study.

Who can participate

Age range18 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Aggressive SM (ASM).
✓. SM-AHN that in the opinion of the Investigator is not considered to be a candidate for Hypomethylating agent (HMA) monotherapy. Incidental indolent, low-grade lymphoid AHNs (eg, chronic lymphocytic leukemia) not requiring treatment are eligible.
✓. Mast cell leukemia (MCL), including diagnoses with an AHN component, that does not require a C-finding.
✓. Upon discussion with the Sponsor, other relapsed or refractory hematologic neoplasms with evidence of aberrant KIT or PDGFR may be considered for enrollment. (eg, participants with chronic myeloid neoplasms, such as subvariants of MDS/MPN that harbor activating KIT exon 17 mutations but do not fulfill the diagnostic criteria of SM-AHN, and participants with myeloid/lymphoid neoplasms with PDGFRa/b fusion genes and mutations conferring resistance to imatinib, such as T674I or D842V).

What they're measuring

Dose Escalation: Number of Dose-limiting Toxicities (DLTs) (monotherapy only)

Timeframe: 28 Days

Dose Escalation: Number of DLTs (combination therapy only)

Timeframe: 28 Days

Dose Escalation and Expansion: Pure Pathological Response (PPR) Rate for SM in Selective KIT Inhibitor-naïve Participants (monotherapy only)

Timeframe: Up to approximately 4 years

Dose Escalation and Expansion: Number of Participants with Adverse Events (AEs)

Timeframe: Up to approximately 4 years

Dose Escalation and Expansion: Number of Participants with Serious Adverse Events (SAEs)

Timeframe: Up to approximately 4 years

Trial details

NCT IDNCT05609942

SponsorBlueprint Medicines Corporation

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2025-01-14

View on ClinicalTrials.gov More Advanced Systemic Mastocytosis trials

Plain-language summary

Who can participate

Age range18 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Aggressive SM (ASM).
✓. SM-AHN that in the opinion of the Investigator is not considered to be a candidate for Hypomethylating agent (HMA) monotherapy. Incidental indolent, low-grade lymphoid AHNs (eg, chronic lymphocytic leukemia) not requiring treatment are eligible.
✓. Mast cell leukemia (MCL), including diagnoses with an AHN component, that does not require a C-finding.
✓. Upon discussion with the Sponsor, other relapsed or refractory hematologic neoplasms with evidence of aberrant KIT or PDGFR may be considered for enrollment. (eg, participants with chronic myeloid neoplasms, such as subvariants of MDS/MPN that harbor activating KIT exon 17 mutations but do not fulfill the diagnostic criteria of SM-AHN, and participants with myeloid/lymphoid neoplasms with PDGFRa/b fusion genes and mutations conferring resistance to imatinib, such as T674I or D842V).

What they're measuring

Dose Escalation: Number of Dose-limiting Toxicities (DLTs) (monotherapy only)

Timeframe: 28 Days

Dose Escalation: Number of DLTs (combination therapy only)

Timeframe: 28 Days

Dose Escalation and Expansion: Pure Pathological Response (PPR) Rate for SM in Selective KIT Inhibitor-naïve Participants (monotherapy only)

Timeframe: Up to approximately 4 years

Dose Escalation and Expansion: Number of Participants with Adverse Events (AEs)

Timeframe: Up to approximately 4 years

Dose Escalation and Expansion: Number of Participants with Serious Adverse Events (SAEs)

Timeframe: Up to approximately 4 years