Juvenile myelomonocytic leukemia

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ORPHA:86834OMIM:607785C93.3
Who is this for?
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1FDA treatments1Active trials54Specialists8Treatment centers

Where are you in your journey?

UniteRare data is sourced from FDA.gov, ClinicalTrials.gov, Orphanet, OMIM, and NORD.
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Overview

Also known as:

Orphanet ↗OMIM ↗NORD ↗

FDA & Trial Timeline

6 events
Apr 2026ONUREG: New indication approved
FDAcompleted
May 2022

Azacitidine: FDA approved

Treatment of pediatric patients aged one month and older with newly diagnosed juvenile myelomonocytic leukemia (JMML)

FDAcompleted
May 2021ONUREG: New indication approved
FDAcompleted
Sep 2020ONUREG: FDA approved
FDAcompleted
Jul 2020Inqovi: FDA approved

Indicated for the treatment of adult patients with myelodysplastic syndromes (MDS), including previously treated and untreated, de novo and secondary MDS with the following French-American-British subtypes (refractory anemia, refractory anemia with ringed sideroblasts, refractory anemia with excess blasts, and chronic myelomonocytic leukemia [CMML]) and intermediate-1, intermediate-2, and high-risk International Prognostic Scoring System groups.

FDAcompleted
Sep 2018Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic Leukemia

National Cancer Institute (NCI) — PHASE2

TrialACTIVE NOT RECRUITING

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Treatments

1 available

Inqovi

CEDAZURIDINE AND DECITABINE· Taiho Pharmaceutical Co., Ltd.Orphan Drug
Indicated for the treatment of adult patients with myelodysplastic syndromes (MDS), including previously treated and untreated, de novo and secondary MDS with the following French-American-British sub

Indicated for the treatment of adult patients with myelodysplastic syndromes (MDS), including previously treated and untreated, de novo and secondary MDS with the following French-American-British subtypes (refractory anemia, refractory anemia with ringed sideroblasts, refractory anemia with excess blasts, and chronic myelomonocytic leukemia [CMML]) and intermediate-1, intermediate-2, and high-risk International Prognostic Scoring System groups.

Clinical Trials

1 recruitingView all trials with filters →
Phase 21 trial
Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic Leukemia
Phase 2
Active Prior treatment eligible
PI: Elliot Stieglitz (Children's Oncology Group) · Sites: Birmingham, Alabama; Phoenix, Arizona +18 more · Age: 121 yrs

Specialists

Showing 25 of 54View all specialists →
ES
Elliot Stieglitz
Birmingham, Alabama
Specialist

Rare Disease Specialist

2 Juvenile myelomonocytic leukemia publications
ME
Miriam Erlacher
Specialist
5 Juvenile myelomonocytic leukemia publications
MS
Marion Strullu
Specialist
5 Juvenile myelomonocytic leukemia publications
SB
Sheila Bohler
Specialist
3 Juvenile myelomonocytic leukemia publications
HM
Hideki Muramatsu
Specialist
5 Juvenile myelomonocytic leukemia publications
CF
Christian Flotho
Specialist
6 Juvenile myelomonocytic leukemia publications
HC
Hélène Cavé
Specialist
5 Juvenile myelomonocytic leukemia publications
JM
Julia Meyer
Specialist
5 Juvenile myelomonocytic leukemia publications
ST
Sarah K Tasian
PHILADELPHIA, PA
Specialist
5 Juvenile myelomonocytic leukemia publications
CN
Charlotte M Niemeyer
Specialist
7 Juvenile myelomonocytic leukemia publications
JR
Jovana Rajak
Specialist
3 Juvenile myelomonocytic leukemia publications
AM
Abhay Singh, MD, MPH
Cleveland, Ohio
Specialist

Rare Disease Specialist

PI on 1 active trial
TM
Thomas G. Martin, MD
Specialist
PI on 4 active trials
MS
Mikkael A Sekeres
MIAMI, FL
Specialist
PI on 1 active trial3 Juvenile myelomonocytic leukemia publications
SM
Steven Devine, MD
LA CROSSE, WI
Specialist
PI on 4 active trials
GM
Guillermo M Bravo, MD
CHANDLER, AZ
Specialist
PI on 1 active trial1 Juvenile myelomonocytic leukemia publication
SM
Susan K. Parsons, MD, MRP
Specialist
PI on 1 active trial
FG
Francis Giles
SAN ANTONIO, TX
Specialist
PI on 4 active trials
PM
Paul Martin
Specialist
PI on 3 active trials73 Juvenile myelomonocytic leukemia publications

Treatment Centers

8 centers
⚗️ Trial Site

Children's Hospital Colorado

📍 Aurora, Colorado

👤 Zachary Grinspan, MD

⚗️ Trial Site

Arkansas Children's Hospital

📍 Little Rock, Arkansas

⚗️ Trial Site

Children's Hospital of Orange County

📍 Orange, California

👤 Richard Neibeger, MD

⚗️ Trial Site

Children's National Medical Center

📍 Washington D.C., District of Columbia

👤 Richard Neibeger, MD

⚗️ Trial Site

Connecticut Children's Medical Center

📍 Hartford, Connecticut

⚗️ Trial Site

University of Miami Miller School of Medicine-Sylvester Cancer Center

📍 Miami, Florida

👤 Ann (Annie) W Silk

🔬 UDN

Harvard/MGH UDN Clinical Site

Massachusetts General Hospital

📍 Boston, MA

🔬 UDN

Baylor College of Medicine UDN Clinical Site

Baylor College of Medicine

📍 Houston, TX

Travel Grants

No travel grants are currently matched to Juvenile myelomonocytic leukemia.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Juvenile myelomonocytic leukemia

Disease timeline:

New trial: Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic Leukemia

Phase PHASE2 trial recruiting. Bone Marrow Aspiration and Biopsy

Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

Mental Health Support

Rare disease caregiving can be isolating. Connect with counseling and peer support.

Family & Caregiver Grants

Financial assistance programs specifically for caregivers of rare disease patients.

Social Security Disability

Learn how rare disease patients may qualify for SSDI/SSI benefits.

Common questions about Juvenile myelomonocytic leukemia

Are there clinical trials for Juvenile myelomonocytic leukemia?

Yes — 1 recruiting clinical trial is currently listed for Juvenile myelomonocytic leukemia on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat Juvenile myelomonocytic leukemia?

25 specialists and care centers treating Juvenile myelomonocytic leukemia are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.