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Clinical Trials/NCT03654703
NCT03654703UnknownPhase 2

Improved Post-Transplant Cyclophosphamide Regimens for Unmanipulated Haploidentical Transplant in Pediatric Patients With Refractory Acute Myeloid Leukemia

Capital Research Institute of Pediatrics1 site in 1 country100 target enrollmentStarted: March 1, 2015Last updated:
Conditions
Interventions
Drugs

Trial Snapshot

Phase
Phase 2
Sponsor
Enrollment
100
Locations
1
Primary Endpoint
Disease status

Study Overview

Brief Summary

No more datas about post-transplant cyclophosphamide (PT/Cy) used in pediatric refractory acute myeloid leukemia (R-AML)patients. Investigators reasoned that this group of patients if they have been treated with ablative conditioning regimens for HSCT combined with PT/Cy, under the rapid of immune reconstitution will have better outcomes.

Detailed Description

Haploidentical stem cell transplantation (HSCT) is a potentially curative therapy for patients with high risk or refractory acute myeloid leukemia (R-AML). Graft-versus-host disease (GVHD)is a major barrier to achieve success for patients with HSCT. High dose cyclophosphamide given after HLA-matched related and unrelated allogeneic stem cell transplantation for patients with hematologic malignancies is effective single agent (GVHD) prophylaxis in adults and in pediatric benign patients. Data describing outcomes for pediatric maligant patients has not been reported. Investigators have recruited 26 pediatric patients (3 years < age <18 years) between March 2015 to July 2018 with primary induction R-AML treated in investigators' institution for R-AML with modified myeloablative regimen, post-transplant cyclophosphamide (PTCy) has been used as GVHD prophylaxis.

Conditioning regimen of the group of patient consisted Idrabine(IDA,10mg/m2/day) which was administered intravenously for 2 days, from days -14 to -13, total body irritation (TBI, 9 Gy) which was divided into 3 fractions and 3 days from -12 to -10, thymoglobulin (2.5mg/kg/day) was administered for 3 days, from -9 to -7, etoposide (VP-16, 300mg/m2) which was infused intravenously on day -6, cladribine (10mg/m2/day) which was administered for 3 days from -5 to -2 .

Study Design

Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel
Primary Purpose
Treatment
Masking
Double (Participant, Investigator)

Eligibility Criteria

Ages
3 Years to 18 Years (Child, Adult)
Sex
All
Accepts Healthy Volunteers
No

Inclusion Criteria

  • refractory AML

Exclusion Criteria

  • complete remission AML

Arms & Interventions

Cyclophophamide

Experimental

Cyclophosphamide 50mg/kg/day on day+3,+4 after HSCT. Intervention: drugs:Cyclophosphamide.

Intervention: Cyclophosphamide (Drug)

Placebo

Experimental

5% GLS(Placebo) 50ml/day on day+3,+4 after HSCT. Intervention: drugs: Placebo other name: placebo (for Cyclophosphamide) 5%Glugose in water 50ml or normal saline

Intervention: Cyclophosphamide (Drug)

Outcomes

Primary Outcomes

Disease status

Time Frame: 1 year

Disease status can be measured by test the of (minimal residual disease) MRD via flow cytometry one time each month until one year after HSCT. MRD\<0.0001 (complete remission), if not, relapse.

Secondary Outcomes

No secondary outcomes reported

Investigators

Sponsor
Capital Research Institute of Pediatrics
Sponsor Class
Other Gov
Responsible Party
Principal Investigator
Principal Investigator

Yan Yue

MD,PhD

Capital Research Institute of Pediatrics

Study Sites (1)

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