Actively Recruiting
Ziftomenib in Combination With Chemotherapy for Children With Relapsed/Refractory Acute Leukemia
Led by PedAL BCU, LLC · Updated on 2025-10-20
20
Participants Needed
20
Research Sites
93 weeks
Total Duration
On this page
Sponsors
P
PedAL BCU, LLC
Lead Sponsor
K
Kura Oncology
Collaborating Sponsor
AI-Summary
What this Trial Is About
The primary objective of the study is to determine the recommended phase 2 dose (RP2D) of ziftomenib in combination with chemotherapy (FLA) in children with relapsed or refractory KMT2A-r, NUP98-r, or NPM1-m acute leukemia based on safety and pharmacokinetics (PK).
CONDITIONS
Official Title
Ziftomenib in Combination With Chemotherapy for Children With Relapsed/Refractory Acute Leukemia
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 0 to 21 years and at least 5 kg body weight, with most participants under 18 years old
- Diagnosis of KMT2A-r, NPM1-m, or NUP98-r acute leukemia in first or greater relapse or refractory to standard treatment
- Bone marrow relapse with defined criteria including 5% leukemic blasts or peripheral blood blast count 1,000 cells/microliter
- Combined extramedullary and bone marrow relapse allowed; isolated extramedullary or isolated CNS relapse not eligible
- Asymptomatic CNS3 disease allowed without isolated CNS3 extramedullary relapse
- Performance status ECOG 0-2 or equivalent Lansky/Karnofsky score
- Adequate organ function including kidney, liver, and heart function per study definitions
- Recovery from prior anti-cancer therapy with specified minimum washout periods
- No prior severe adverse reaction to menin inhibitors except under defined conditions
- Written informed consent and pediatric assent obtained
- Negative pregnancy test for females of childbearing potential
- Agreement to use effective contraception during and after study as specified
- Participants in US and Canada must be enrolled in APAL2020SC trial prior to this study
You will not qualify if you...
- Inability to comply with study requirements
- Diagnosis of Down syndrome
- Isolated extramedullary disease or isolated CNS relapse
- Acute promyelocytic leukemia or juvenile myelomonocytic leukemia
- Conditions preventing oral administration of menin inhibitor
- Use of proton pump inhibitors during study
- Current participation in another investigational drug trial
- Known congenital bone marrow failure syndrome
- Known allergy to study medications
- Active uncontrolled infection at study entry
- Active HIV, hepatitis B, or hepatitis C infection
- Positive pregnancy test or breastfeeding in females
- Pre-existing conditions increasing risk of severe infection
- Use of investigational medications within 30 days prior to study
- Significant congenital cardiovascular disease posing risk
- Medical conditions interfering with treatment or toxicity assessment
- Hypersensitivity to fludarabine or cytarabine
- Recent live vaccinations within 6 months prior to study
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 20 locations
1
Children's Hospital Los Angeles
Los Angeles, California, United States, 90027
Actively Recruiting
2
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
Actively Recruiting
3
Children's Healthcare of Atlanta
Atlanta, Georgia, United States, 30329
Actively Recruiting
4
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
Actively Recruiting
5
Dana-Farber Cancer Institute
Boston, Massachusetts, United States, 02215
Actively Recruiting
6
Memorial Sloan Kettering Cancer Center - New York
New York, New York, United States, 10065
Actively Recruiting
7
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229-3026
Actively Recruiting
8
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
9
St. Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105-3678
Actively Recruiting
10
Texas Children's Hospital
Houston, Texas, United States, 77030
Actively Recruiting
11
Seattle Children's Hospital
Seattle, Washington, United States, 98105
Actively Recruiting
12
Sankt Anna-Kinderspital
Vienna, State of Vienna, Austria, 1090
Actively Recruiting
13
SickKids - The Hospital for Sick Children
Toronto, Ontario, Canada, M5G 1X8
Actively Recruiting
14
CHU de Nantes - Hôpital Femme-Enfant-Adolescent
Nantes, Loire-Atlantique, France, 44093
Actively Recruiting
15
CHU de Reims - Hôpital Robert Debré
Paris, Île-de-France Region, France, 75019
Actively Recruiting
16
Ospedale Pediatrico Bambino Gesù
Roma, Rome, Italy, 00165
Actively Recruiting
17
Fondazione IRCCS San Gerardo dei Tintori (Ospedale San Gerardo)
Monza, Italy, 20900
Actively Recruiting
18
Prinses Maxima Centrum Kinderoncologie
Utrecht, Netherlands, 3584 CS
Actively Recruiting
19
Hospital Universitari Vall d'Hebrón
Barcelona, Spain, 08035
Actively Recruiting
20
Hospital Infantil Universitario Niño Jesús
Madrid, Spain, 28009
Actively Recruiting
Research Team
D
Dr. Branko Cuglievan
CONTACT
D
Dr. Sarah Tasian, MD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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