A Phase I/II Study of Trametinib and Azacitidine for Patients With Newly Diagnosed Juvenile Myelomonocytic Leukemia

Part of paid clinical trials in Phoenix, Arizona.

Sponsor
Therapeutic Advances in Childhood Leukemia Consortium
Study ID
NCT05849662
Phase
PHASE1/PHASE2
Status
Recruiting
Apply to this trial

Conditions

  • CBL Syndrome
  • JCML
  • JMML
  • Leukemia, Juvenile Myelomonocytic
  • Neurofibromatosis 1

Eligibility Criteria

Sex
ALL
Age
1 Month - 21 Years
Healthy Volunteers
Not accepted

Interventions

  • Trametinib — DRUG
    PO or NG QD Days 1-28 For patients age \< 6 years: 0.032 mg/kg/day at max dose = 2mg/day For patients age ≥ 6 years: 0.025 mg/kg/day at max dose = 2 mg/day
  • Azacitidine — DRUG
    IV over 30 minutes Days 1-5 Age \< 1 year or weight \<10kg: 2.5 mg/kg/day Age ≥ 1 year and weight ≥ 10kg: 75 mg/m2/day
  • Fludarabine — DRUG
    IV over 30 minutes Days 6-10 30 mg/m2/day (1mg/kg if \<12 kg)
  • Cytarabine — DRUG
    IV over 3 hours Days 6-10 2000 mg/m2/day (67mg/kg if \<12 kg)

Study Details

This clinical trial will test the safety and efficacy of combining trametinib and azacitidine in patients with juvenile myelomonocytic leukemia (JMML). Newly diagnosed lower-risk JMML patients will receive trametinib and azacitidine. High-risk JMML patients will receive trametinib, azacitidine, fludarabine, and cytarabine.

Key Dates

Start date
Oct 11, 2024
Status verified
Jun 2025
Primary completion
Dec 31, 2028
Completion
Dec 31, 2029

Study Design

Enrollment
58 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: Lower-risk patients
    Lower-risk patients are defined as having a mutational burden of one clonal alteration AND a low DNA methylation classification.
  • Experimental: High-risk patients
    High-risk patients are defined as having as having a mutational burden of more than one clonal alteration AND/OR an intermediate or high DNA methylation classification.

Primary Outcome Measure

To determine the safety of combining trametinib with azacitidine for patients with newly diagnosed lower-risk JMML. [ Time Frame: At the end of the evaluation period of Cycle 1 (defined as 28 day cycle of therapy plus 30 days following the last dose of study therapy) ]

Central Contacts

Locations (19)

FacilityCityStateZIPSite coordinators
Phoenix Children's HospitalPhoenixArizona85016
Dana Salzberg, MD
Children's Hospital Los AngelesLos AngelesCalifornia900027
Jaime Stokke, MD
University of California San FranciscoSan FranciscoCalifornia94158
Elliot Stieglitz, MD
Children's Hospital of ColoradoDenverColorado80045
Kelly Faulk, MD
Children's National Medical CenterWashington D.C.District of Columbia20010
Reuven Schore, MD
University of MiamiMiamiFlorida33136
Julio Barredo, MD
Children's Hospital of AtlantaAtlantaGeorgia30322
Melinda Pauly, MD
Lurie Children's Hospital of ChicagoChicagoIllinois60611
Jenna Rossoff, MD
Indiana University/Riley Hospital for ChildrenIndianapolisIndiana46202
Sandeep Batra, MD
Sidney Kimmel Cancer Center at Johns HopkinsBaltimoreMaryland21231
Stacy Cooper, MD
C.S. Mott Children's HospitalAnn ArborMichigan48109
Jennifer Agrusa, MD
Children's Mercy HospitalKansas CityMissouri64108
Keith August, MD
Memorial Sloan Kettering Cancer CenterNew YorkNew York10065
Kavitha Ramaswamy, MD
Cincinnati Children's Hospital Medical CenterCincinnatiOhio45229
Lauren Pommert, MD
Oregon Health & Science UniversityPortlandOregon97239
Bill Chang, MD
Children's Hospital of PhiladelphiaPhiladelphiaPennsylvania19104
Sarah Tasian, MD
St. Jude Children's Research Hospital MemphisMemphisTennessee38105
Seth Karol, MD
Primary Children's HospitalSalt Lake CityUtah84113
Mallorie Heneghan, MD
Seattle Children's HospitalSeattleWashington98105
Mignon Loh, MD

Find similar trials in Phoenix, AZ

By research site
Phoenix Children's Hospital· Phoenix, AZChildren's Hospital Los Angeles· Los Angeles, CAUniversity of California San Francisco· San Francisco, CAChildren's Hospital of Colorado· Denver, COChildren's National Medical Center· Washington D.C., DCUniversity of Miami· Miami, FL

Related Studies