Active — Not RecruitingPhase 1/2

First in Human Study of Ziftomenib in Relapsed or Refractory Acute Myeloid Leukemia

United States, Belgium, Canada263 participantsStarted 2019-09-12

Plain-language summary

In this trial, ziftomenib, a menin-MLL(KMT2A) inhibitor, will be tested in patients for the first time. The trial includes a Main Study and four sub-studies. In the Main Study (including Phase 1a, Phase 1b, and Phase 2 portions), ziftomenib will be evaluated in patients with relapsed or refractory (R/R) acute myeloid leukemia (AML). The main study has completed enrollment. In Sub-studies 1 and 2, the effects of taking ziftomenib and other common drugs at the same time will be investigated in AML patients. In Sub-study 3, ziftomenib will be evaluated in patients with R/R acute lymphoblastic leukemia (ALL). In Sub-study 4, ziftomenib will be evaluated in patients with R/R AML with certain genetic mutations.

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

. Phase 1b:
. Phase 2:
. Sub-studies:
. ≥ 18 years of age.
. Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2, and a life expectancy of at least 2 months.
. Adequate liver and kidney function according to protocol requirements.
. Peripheral white blood cell (WBC) counts ≤ 30,000/μL. Patients may receive hydroxyurea to control and maintain white blood cell count prior to enrollment.
. Women of childbearing potential must be willing to use a highly effective method of contraception throughout the study and for at least 187 days after the last dose of study treatment.

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

Phase 1a: Maximum tolerated dose (MTD) and/or the recommended Phase 2 dose (RP2D)

Timeframe: Dose Limiting Toxicities (DLTs) will be evaluated during the first 28 days (1 cycle)

Phase 1b: Number of patients who experience Adverse Events (AEs) and Serious Adverse Events (SAEs)

Timeframe: During treatment and up to approximately 28 days after treatment discontinuation, or until immediately before the initiation of another anticancer therapy, whichever occurs first.

Phase 1b: Minimum biologically effective dose

Timeframe: For at least 12 months following end of treatment

Phase 1a, 1b, and 2: Evidence of anti-leukemia activity

Timeframe: For at least 12 months following end of treatment

Sub-study 1: Time to observed maximum plasma concentration (Tmax) of ziftomenib and midazolam

Timeframe: Cycle 1 on Days 1 and 15 at predose and postdose

Sub-study 1: Area under the plasma concentration-time curve from time 0 to time t (AUC0-t) of ziftomenib and midazolam

Timeframe: Cycle 1 on Days 1 and 15 at predose and postdose

Trial details

NCT IDNCT04067336

SponsorKura Oncology, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2028-10-16

View on ClinicalTrials.gov More Advanced Malignant Neoplasm 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

. Phase 1b:
. Phase 2:
. Sub-studies:
. ≥ 18 years of age.
. Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2, and a life expectancy of at least 2 months.
. Adequate liver and kidney function according to protocol requirements.
. Peripheral white blood cell (WBC) counts ≤ 30,000/μL. Patients may receive hydroxyurea to control and maintain white blood cell count prior to enrollment.
. Women of childbearing potential must be willing to use a highly effective method of contraception throughout the study and for at least 187 days after the last dose of study treatment.

What they're measuring

Phase 1a: Maximum tolerated dose (MTD) and/or the recommended Phase 2 dose (RP2D)

Timeframe: Dose Limiting Toxicities (DLTs) will be evaluated during the first 28 days (1 cycle)

Phase 1b: Number of patients who experience Adverse Events (AEs) and Serious Adverse Events (SAEs)

Timeframe: During treatment and up to approximately 28 days after treatment discontinuation, or until immediately before the initiation of another anticancer therapy, whichever occurs first.

Phase 1b: Minimum biologically effective dose

Timeframe: For at least 12 months following end of treatment

Phase 1a, 1b, and 2: Evidence of anti-leukemia activity

Timeframe: For at least 12 months following end of treatment

Sub-study 1: Time to observed maximum plasma concentration (Tmax) of ziftomenib and midazolam

Timeframe: Cycle 1 on Days 1 and 15 at predose and postdose

Sub-study 1: Area under the plasma concentration-time curve from time 0 to time t (AUC0-t) of ziftomenib and midazolam

Timeframe: Cycle 1 on Days 1 and 15 at predose and postdose

First in Human Study of Ziftomenib in Relapsed or Refractory Acute Myeloid Leukemia

Plain-language summary

Who can participate

Inclusion criteria

Questions worth asking your doctor

Questions for the trial coordinator

What they're measuring

Trial details

First in Human Study of Ziftomenib in Relapsed or Refractory Acute Myeloid Leukemia

Plain-language summary

Who can participate

Inclusion criteria

Questions worth asking your doctor

Questions for the trial coordinator

What they're measuring

Trial details

Exclusion criteria