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, Belgium, France9 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 range

18 Years

Sex

ALL

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).

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

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 range

18 Years

Sex

ALL

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