A Multicenter Study on the Treatment of MDS/MPN Overlap Syndrome With AZA or Rux Combined With Selinexor

Chinese Academy of Medical Sciences & Peking Union Medical College

Status and phase

Invitation-only

Phase 2

Conditions

MDS

Treatments

Drug: Azacitidine or Ruxolitinib combined with Selinexor

Study type

Interventional

Funder types

Other

Identifiers

NCT06664970

K6812

Details and patient eligibility

About

This study is a prospective, multicenter, open label cohort study involving MDS/MPN patients. The enrolled patients have symptoms that require treatment, which are classified according to their clinical conditions as follows: those with MDS as the main manifestation are treated with Azacitidine combined with Selinexor; For those with MPN as the main manifestation, treatment with Selinexor combined with Ruxolitinib is used.

Enrollment

39 estimated patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Meets the diagnostic criteria of MDS/MPN (WHO 2022 edition)
Age ≥ 18 years old;
There are indications that require treatment, such as symptomatic anemia, decreased blood cells, splenomegaly, and constitutional symptoms;
ECOG score 0-2;
No stem cell transplantation plan within 6 months;
Liver function: Within 21 days before the start of treatment, total bilirubin<2 times the upper limit of normal (ULN), alanine aminotransferase (ALT)<2.5 times ULN;
Renal function: Within 21 days before the start of treatment, creatinine clearance rate ≥ 30 mL/min, calculated using Cockcroft and Gault formulas;
Patients receiving erythropoietin therapy or rituximab must be at a stable dosage and have stable transfusion therapy or hemoglobin levels within 8 weeks prior to entering the study.
Female patients with fertility must agree to the use of dual contraception methods and have a negative serum pregnancy test during screening. Male patients who have sexual intercourse with women with fertility must use effective barrier contraception methods.
Those who meet the requirements of the ethics committee and sign the informed consent form; Willing and able to comply with clinical visit and research related procedures.

Exclusion criteria

Those who have undergone major surgery within 4 weeks before the start of treatment,
those with severe liver and kidney dysfunction;
Patients who have undergone splenectomy in the past;
Patients with unstable cardiovascular function: symptomatic cardiac ischemia, uncontrolled significant conduction abnormalities, congestive heart failure (CHF) with NYHA functional class ≥ 3 or myocardial infarction within 6 months, unstable angina, unstable arrhythmia;
Uncontrolled active infections require systemic use of antibiotics, antiviral drugs, or antifungal drugs within one week prior to the first administration; Allow the use of prophylactic antibiotics.
Known active hepatitis A, B, or C infection; Or known to be positive for HCV RNA or HBsAg (HBV surface antigen); Known HIV seropositivity;
Patients with obvious gastrointestinal lesions or obstruction, or uncontrolled vomiting or diarrhea.
At baseline, peripheral neuropathy was ≥ grade 2 (within 21 days before the first day of the first cycle).
History of epileptic seizures, movement disorders, or cerebrovascular accidents (within 1 year before the first day of the first cycle)
Evidence of AML (≥ 20% of primitive cells in bone marrow or peripheral blood) according to WHO definition
Patients with other active malignant tumors
Pregnant and lactating women;
Fertility capable women or men who are unwilling to take effective contraceptive measures before the first dose/treatment, during the study period, and for at least 6 months after the last dose;
Patients without autonomous behavioral ability, such as psychiatric patients; Other situations deemed unsuitable for inclusion by researchers.