Acute myeloid leukemia and myelodysplastic syndromes related to alkylating agent

Last reviewed

🖨 Print for my doctorAdvocacy Hub →
ORPHA:102379C92.8
Who is this for?
Show terms as
131Active trials240Specialists8Treatment centers

Where are you in your journey?

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

Overview

Also known as:

Orphanet ↗NORD ↗

FDA & Trial Timeline

10 events
Dec 2027A Phase 1b/2 Open-label, Dose-ranging Safety and Efficacy Study of Oral Cladribine in Patients With Acute Myeloid Leukemia (AML)

M.D. Anderson Cancer Center — PHASE1, PHASE2

TrialNOT YET RECRUITING
Dec 2026FLT3-ITD Targeted Therapy in Fit AML Patients

European Organisation for Research and Treatment of Cancer - EORTC — PHASE2, PHASE3

TrialNOT YET RECRUITING
Oct 2026Comparing Different Treatment Lengths for Venetoclax in Older People With Newly Diagnosed Acute Myeloid Leukemia (A MyeloMATCH Treatment Trial)

National Cancer Institute (NCI) — PHASE2

TrialNOT YET RECRUITING
Aug 2026Phase 1/1b Trial Of Olutasidenib And Ziftomenib For NPM1 And IDH1 Co-Mutated Acute Myeloid Leukemia

M.D. Anderson Cancer Center — PHASE1

TrialNOT YET RECRUITING
May 2026High-dose Ascorbate (HDA) in Combination With Standard of Care Azacitidine and Venetoclax in Acute Myeloid Leukemia (AML)

Kittika Poonsombudlert — PHASE1

TrialNOT YET RECRUITING
May 2026Study of an Innovative Therapy Using CAR-T Cells Targeting IL-1RAP in Patients With High-Risk Myelodysplastic Syndromes (MDS

University Hospital, Grenoble — NA

TrialNOT YET RECRUITING
Apr 2026Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms

National Cancer Institute (NCI) — PHASE1, PHASE2

TrialRECRUITING
Apr 2026A Study to Compare Elritercept With Epoetin Alfa to Treat Anemia in Adults With Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS) Who Need Regular Blood Transfusions

Takeda — PHASE3

TrialRECRUITING
Apr 2026A Study of Rebecsinib for Patients With Relapsed/Refractory Secondary Acute Myeloid Leukemia or High Risk Myelofibrosis

Aspera Biomedicines, Inc. — PHASE1

TrialNOT YET RECRUITING
Apr 2026Phase I/II Clinical Trial of Universal Donor CD33 CAR Natural Killer Cells for AML

Nationwide Children's Hospital — PHASE1, PHASE2

TrialNOT YET RECRUITING

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

Treatments

No FDA-approved treatments are currently listed for Acute myeloid leukemia and myelodysplastic syndromes related to alkylating agent.

20 clinical trialsare actively recruiting — trials can provide access to cutting-edge therapies.

View clinical trials →

Clinical Trials

20 recruitingView all trials with filters →
Phase 38 trials
Reduced Intensity Conditioning Regimens for Acute Myeloid Leukemia and Myelodysplastic Syndrome
Phase 3
Enrolling by Invitation
PI: Linghui Xia, Professor (Department of Hematology, Wuhan Union Hospital, To) · Sites: Wuhan, Hubei · Age: 1899 yrs
A Study to Compare Elritercept With Epoetin Alfa to Treat Anemia in Adults With Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS) Who Need Regular Blood Transfusions
Phase 3
Actively Recruiting
PI: Study Director (Takeda) · Sites: Orange, California; Tamarac, Florida +144 more · Age: 1899 yrs
Ivosidenib (IVO) Monotherapy and Azacitidine (AZA) Monotherapy in Patients With Hypomethylating Agent (HMA) Naive Myelodysplastic Syndromes (MDS) With an IDH1 Mutation
Phase 3
Actively Recruiting
· Sites: Denver, Colorado; Chicago, Illinois +60 more · Age: 1899 yrs
CHIP-AML22/Master: An Open Label Complex Clinical Trial in Newly Diagnosed Pediatric de Novo AML Patients
Phase 3
Actively Recruiting
PI: Gertjan Kaspers, Prof. Dr. (Pediatric Oncologist) · Sites: Utrecht, Utrecht · Age: 018 yrs
Efficacy of Intermediate-Dose Cytarabine Induction Regimen in Adult AML
Phase 3
Active
PI: Jianxiang Wang, Dr (Institute of Hematology & Blood Diseases Hospital,) · Sites: Tianjin, Tianjin Municipality · Age: 1455 yrs
Randomised Study of Oral Azacitidine vs Placebo Maintenance in AML or MDS Patients After Allo-SCT
Phase 3
Active
PI: Charles Craddock (University of Birmingham) · Sites: Birmingham; Bristol +18 more · Age: 1699 yrs
Phase III Study of Induction and Consolidation Chemotherapy With Venetoclax in Patients With Newly Diagnosed AML or MDS-EB-2
Phase 3
Actively Recruiting
PI: Hartmut Doehner, MD (University of Ulm) · Sites: Innsbruck; Linz +89 more · Age: 1875 yrs
Study of AG-120 (Ivosidenib) vs. Placebo in Combination With Azacitidine in Participants With Previously Untreated Acute Myeloid Leukemia With an IDH1 Mutation
Phase 3
Active
· Sites: Louisville, Kentucky; Boston, Massachusetts +88 more · Age: 1899 yrs
Phase 42 trials
Olutasidenib DDI Study in Patients With IDH1 Mutation Positive Malignancies
Phase 4
Actively Recruiting
· Sites: Orange, California; New York, New York · Age: 1899 yrs
RIC Allo-HSCT vs. Venetoclax-Based Consolidation in Elderly AML Patients After First CR
Phase 4
Actively Recruiting
· Sites: Hangzhou, Zhejiang · Age: 6075 yrs
Other10 trials
A Study of c-Kit Mutation as MRD in Acute Myeloid Leukemia
Actively Recruiting
· Sites: Tianjin · Age: 1499 yrs
OBServatory of Compassionate Use of IVOsidenib in France for Patients With Acute Myeloid Leukemia
Actively Recruiting
PI: Pierre PETERLIN, Dr (French Innovative Leukemia Organisation) · Sites: Amiens; Angers +19 more · Age: 1899 yrs
Monitoring Minimal Residual Disease of Patients With Acute Myelogenous Leukemia or High Grade Myelodysplastic Syndrome
Actively Recruiting
PI: Michael W Becker, MD (University of Rochester) · Sites: Rochester, New York · Age: 1899 yrs
Registry of Patients Having Received oNKord®
Actively Recruiting
· Sites: Hanover; Basel · Age: 1899 yrs
Defining Inflammatory Markers of Cardiovascular Disease in Patients With Myelodysplastic Syndromes
Actively Recruiting
· Sites: Burlington, Vermont · Age: 1899 yrs
Muscle Dysfunction in Patients With Haematological Diseases
Active
· Sites: Copenhagen · Age: 1899 yrs
A Study to Asses Effectiveness and Treatment Management of Oral Venetoclax Tablets + Subcutaneously Injected Azacitidine in Adult Participants Ineligible for Intensive Chemotherapy With de Novo Acute Myeloid Leukemia (AML) in Italy
Active
PI: ABBVIE INC. (AbbVie) · Sites: Lecce, Apulia; Florence, Firenze +26 more · Age: 1899 yrs
Clinical Registration Study of Haplo-HSCT for Elderly Patients With Acute Leukemia/ Myelodysplastic Syndrome
Actively Recruiting
· Sites: Beijing, Beijing Municipality · Age: 5570 yrs
The Patient Cohort of the National Center for Precision Medicine in Leukemia
Actively Recruiting
· Sites: Bobigny; Paris +1 more
Institut Paoli Calmettes Myelodysplastic Syndromes Database
Actively Recruiting
PI: Norbert Vey, Pr (Institut Paoli-Calmettes) · Sites: Marseille, PACA · Age: 1899 yrs

Specialists

Showing 25 of 240View all specialists →
HH
He Huang
Specialist
PI on 11 active trials3 Acute myeloid leukemia and myelodysplastic syndromes related to alkylating agent publications
HM
Hagop Kantarjian, MD
HOUSTON, TX
Specialist
PI on 7 active trials
GL
Global Clinical Leader
Specialist
PI on 44 active trials4 Acute myeloid leukemia and myelodysplastic syndromes related to alkylating agent publications
OM
Olivier Hermine, MD
Specialist
PI on 3 active trials
FM
Farhad Ravandi-Kashani, MD
HOUSTON, TX
Specialist
PI on 8 active trials
TK
Tapan M Kadia
Houston, Texas
Specialist

Rare Disease Specialist

PI on 9 active trials
JC
Jorge Cortes
Specialist
PI on 7 active trials8 Acute myeloid leukemia and myelodysplastic syndromes related to alkylating agent publications
RV
Ravi Vij
SAINT LOUIS, MO
Specialist
PI on 1 active trial
AA
Aref Al-Kali
ROCHESTER, MN
Specialist
PI on 4 active trials1 Acute myeloid leukemia and myelodysplastic syndromes related to alkylating agent publication
MP
Michael Andreeff, MD, PhD
HOUSTON, TX
Specialist
PI on 2 active trials
KM
Kenneth G Lucas, MD
Specialist
PI on 3 active trials

Treatment Centers

8 centers
🏥 NORD

Baylor College of Medicine Rare Disease Center

Baylor College of Medicine

📍 Houston, TX

🏥 NORD

Stanford Medicine Rare Disease Center

Stanford Medicine

📍 Stanford, CA

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program

National Institutes of Health

📍 Bethesda, MD

🔬 UDN

UCLA UDN Clinical Site

UCLA Health

📍 Los Angeles, CA

🔬 UDN

Baylor College of Medicine UDN Clinical Site

Baylor College of Medicine

📍 Houston, TX

🔬 UDN

Harvard/MGH UDN Clinical Site

Massachusetts General Hospital

📍 Boston, MA

🏥 NORD

Mayo Clinic Center for Individualized Medicine

Mayo Clinic

📍 Rochester, MN

👤 Mayo Clinic Center for Individualized Medicine

🏥 NORD

UCLA Rare Disease Day Program

UCLA Health

📍 Los Angeles, CA

Travel Grants

No travel grants are currently matched to Acute myeloid leukemia and myelodysplastic syndromes related to alkylating agent.

Search all travel grants →NORD Financial Assistance ↗

Community

Open Acute myeloid leukemia and myelodysplastic syndromes related to alkylating agentForum →

No community posts yet. Be the first to share your experience with Acute myeloid leukemia and myelodysplastic syndromes related to alkylating agent.

Start the conversation →

Latest news about Acute myeloid leukemia and myelodysplastic syndromes related to alkylating agent

2 articles
Clinical trialUNITERAREApr 5, 2026
New Recruiting Trial: Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms
Researchers are testing a new drug called pacritinib in people ages 12 and older who have myelodysplastic syndromes or myelodysplastic/myeloproliferative neopla
Clinical trialUNITERAREApr 3, 2026
New Recruiting Trial: A Study to Compare Elritercept With Epoetin Alfa to Treat Anemia in Adults With Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS) Who Need Regular Blood Transfusions
Researchers are testing a new drug called elritercept to see if it works better than a standard treatment (epoetin alfa) for anemia in adults with myelodysplast
See all news about Acute myeloid leukemia and myelodysplastic syndromes related to alkylating agent

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 Acute myeloid leukemia and myelodysplastic syndromes related to alkylating agent

Are there clinical trials for Acute myeloid leukemia and myelodysplastic syndromes related to alkylating agent?

Yes — 20 recruiting clinical trials are currently listed for Acute myeloid leukemia and myelodysplastic syndromes related to alkylating agent on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat Acute myeloid leukemia and myelodysplastic syndromes related to alkylating agent?

25 specialists and care centers treating Acute myeloid leukemia and myelodysplastic syndromes related to alkylating agent are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.