Clinical trial NCT05807659: Fractionated Busulfan Conditioning Regimen for Allo-HSCT in Non-remission Myeloid Malignancies
Official title: Fractionated Busulfan Combined With Chidamide/Fludarabine/Cytarabine (ChiFAB) With Allogeneic Hematopoietic Stem-Cell Transplantation in Non-remission Myeloid Malignancies: a Phase II Study
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Myeloid Malignancies
Keywords: myeloid malignancies
allo-HSCT
Lead sponsor: Sichuan University
Sponsor class: OTHER
First posted: 2023-04-11
Enrollment: 18
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The goal of this single-arm phase II study is to test in patients with non-remission myeloid malignancies undergoing allogeneic hemopoietic stem-cell transplantation (allo-HSCT). The main question it aims to answer are:

* The safety and efficacy of fractionated busulfan Combined With Chidamide/Fludarabine/Cytarabine(ChiFAB) conditioning regimen in increasing the overall survival rate in patients with non-remission myeloid malignancies after allo-HSCT.
* The efficacy of fractionated busulfan conditioning regimen in reducing the recurrence rate in patients with non-remission myeloid malignancies after allo-HSCT.

Participants will receive fractionated busulfan-based ChiFAB conditioning regimen (busulfan 3.2mg/kg d-13, -12, 1.6mg/kg d-6\~-3, fludarabine 35mg/m2 d-6\~-2, cytarabine 1g/m2,d-6\~-2, chidamide 30mg d-13,-10,-6,-3) before allo-HSCT.


Eligibility criteria: Inclusion Criteria:

1. Age ≥ 18 years old and ≤ 65 years old when signing the Informed Consent Form (ICF);
2. KPS score \> 60 or ECOG score 0-2;
3. The expected survival period \> 3 months;
4. Those who did not achieve complete remission after 2 or more chemotherapy regimens. The proportion of blasts on bone marrow smears before transplantation was ≥5%.
5. Those who have no central nervous system involvement or serious functional damage to important organs of the body;
6. Fully understand and be informed of this study and sign the ICF; willing to follow and have the ability to complete all test procedures;

Exclusion Criteria:

1. Serious basic diseases of important organs: such as myocardial infarction, chronic cardiac insufficiency, decompensated hepatic insufficiency, renal function, gastrointestinal insufficiency, etc.;
2. Uncontrolled active infection (including bacterial, fungal, or viral infection), and drug treatment is ineffective;
3. Participating in other clinical studies, or planning to start treatment in this study and less than 4 weeks before the end of treatment in the previous clinical study;
4. Combined with other malignant tumors and require treatment;
5. Pregnant or lactating females;
6. Patients with known history of human immunodeficiency virus (HIV) virus infection and/or acquired immunodeficiency syndrome;
7. Patients with active chronic hepatitis B or active hepatitis C;
8. History of prolonged QT syndrome;
9. Patients considered by other researchers to be unsuitable for this study

Primary outcomes:
measure: Overall survival (OS)
timeFrame: 2 years
Secondary outcomes:
measure: Non-relapse mortality (NRM)
timeFrame: 6 months
measure: Relapse rate
timeFrame: 2 years
measure: Cumulative incidence of acute graft versus host disease (aGVHD)
timeFrame: Day +100
measure: Cumulative incidence of chronic graft versus host disease (cGVHD)
timeFrame: 2 years
Other outcomes:
Locations:
city: Chendu
country: China
facility: West China Hospital, Sichuan University
state: Sichuan
Study officials:
References: