Thiotepa-Containing Conditioning Regimen for Allogeneic HSCT in Chronic Myelomonocytic Leukemia
Trial statusRecruiting
Trial phaseNot applicable
Trial typeInterventional
Biological sexAll
Age18+
SponsorPeking University People's Hospital
About this trial
This is a prospective, single-arm clinical study. It aims to evaluate the effectiveness and safety of a conditioning regimen containing thiotepa (in combination with busulfan and fludarabine, with or without ATG) of allogeneic hematopoietic stem cell transplantation (allo-HSCT) in patients with chronic myelomonocytic leukemia (CMML) who have an intermediate-2 or high-risk prognosis.
The main goal is to evaluate 1 year RFS and OS. Other goals include assessing engraftment, overall survival, transplant-related complications, and side effects. A total of 31 participants will be enrolled.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Age ≥ 18 years, any sex/gender.
Confirmed diagnosis of chronic myelomonocytic leukemia (CMML) according to the 2022 WHO classification.
Intermediate-2 or high-risk CMML based on CPSS or CPSS-mol score, and planned to receive allo-HSCT.
For haploidentical donor: at least 5/10 HLA match at HLA-A, -B, -C, -DQB1, and -DRB1.
Disqualifiers
Previous allogeneic HSCT for CMML that later relapsed.
Unwilling or unable to receive the study treatment regimen.
Active hepatitis B or C, or chronic active hepatitis; known human immunodeficiency virus (HIV) infection.
Active uncontrolled infection, including: hemodynamic instability related to infection, new or worsening signs/symptoms of infection, new infection lesions on imaging, or persistent fever without explanation despite no symptoms/signs.
Trial design
Design model
Single group
Treatments tested in this trial
TBF regimen
DrugIntravenous thiotepa 5 mg/kg/d on days -11,-10; busulfan 3.2 mg/kg/d on days -8,-7,-6; fludarabine 30 mg/m²/d on days -6 to -2; ATG per donor type (haplo/unrelated: 2.5 mg/kg/d days -5 to -2; MSD: 1.125 mg/kg/d days -5 to -2). Allogeneic stem cells infused on day 0. GVHD prophylaxis: CsA, MMF, MTX per protocol.
Treatment groups
31 Participants
are divided into 1 treatment groupGroup A: Single Arm: Thiotepa + Busulfan + Fludarabine ± ATGExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
One-year relapse-free survival (RFS) after allogeneic hematopoietic stem cell transplantation
Time from stem cell infusion to hematologic/extramedullary relapse or death, censored at 12 months for event-free participants.
Time frame
At 12 months after allogeneic hematopoietic stem cell transplantation.Secondary outcomes
1
One-year overall survival (OS)
Overall survival is defined as the time from stem cell infusion to death from any cause. Participants alive at 12 months are censored at the last follow-up.
Time frame
At 12 months after allogeneic hematopoietic stem cell transplantation.2
One-year cumulative incidence of relapse (RR)
Relapse is defined as recurrence of CMML . Non-relapse mortality is treated as a competing risk.
Time frame
At 12 months after allogeneic hematopoietic stem cell transplantation.3
One-year non-relapse mortality (NRM)
Non-relapse mortality is defined as death from any cause other than disease relapse or progression. Relapse is treated as a competing risk.
Time frame
At 12 months after allogeneic hematopoietic stem cell transplantation.4
Regimen toxicity at day +30
Toxicity is graded according to NCI CTCAE v5.0. Any grade 3-5 non-hematologic toxicity occurring within 30 days after stem cell infusion is reported.
Time frame
Up to day +30 post-transplantSponsors and contacts
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Source ClinicalTrials.gov