Clinical trial NCT06664970: A Multicenter Study on the Treatment of MDS/MPN Overlap Syndrome With AZA or Rux Combined With Selinexor
Official title: Prospective, Multicenter Study on the Treatment of Myelodysplastic Neoplasms (MDS)/Myeloproliferative Neoplasms (MPN) Overlap Syndrome With Azacitidine or Ruxolitinib Combined With Selinexor
Study type: INTERVENTIONAL
Overall status: ENROLLING_BY_INVITATION
Phase: PHASE2
Conditions: MDS
Lead sponsor: Peking Union Medical College Hospital
Sponsor class: OTHER
First posted: 2024-10-30
Enrollment: 39
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: Inclusion Criteria:

1. Meets the diagnostic criteria of MDS/MPN (WHO 2022 edition)
2. Age ≥ 18 years old;
3. There are indications that require treatment, such as symptomatic anemia, decreased blood cells, splenomegaly, and constitutional symptoms;
4. ECOG score 0-2;
5. No stem cell transplantation plan within 6 months;
6. 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;
7. Renal function: Within 21 days before the start of treatment, creatinine clearance rate ≥ 30 mL/min, calculated using Cockcroft and Gault formulas;
8. 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.
9. 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.
10. 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:

1. Those who have undergone major surgery within 4 weeks before the start of treatment,
2. those with severe liver and kidney dysfunction;
3. Patients who have undergone splenectomy in the past;
4. 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;
5. 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.
6. Known active hepatitis A, B, or C infection; Or known to be positive for HCV RNA or HBsAg (HBV surface antigen); Known HIV seropositivity;
7. Patients with obvious gastrointestinal lesions or obstruction, or uncontrolled vomiting or diarrhea.
8. At baseline, peripheral neuropathy was ≥ grade 2 (within 21 days before the first day of the first cycle).
9. History of epileptic seizures, movement disorders, or cerebrovascular accidents (within 1 year before the first day of the first cycle)
10. Evidence of AML (≥ 20% of primitive cells in bone marrow or peripheral blood) according to WHO definition
11. Patients with other active malignant tumors
12. Pregnant and lactating women;
13. 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;
14. Patients without autonomous behavioral ability, such as psychiatric patients; Other situations deemed unsuitable for inclusion by researchers.

Primary outcomes:
measure: ORR at 6 months of treatment
timeFrame: 6 months
Secondary outcomes:
Other outcomes:
Locations:
city: Beijing
country: China
facility: Bing Han
Study officials:
References: