Clinical trial NCT04547049: A Study Comparing Haploidentical Hematopoietic Stem Cell Transplantations (HSCTs) From Young Non-first-degree and Older First-degree Donors in Hematological Malignancies
Official title: An Open, Multi-center, Randomized Trial Comparing Haploidentical HSCTs From Young Non-first-degree and Older First-degree Donors in Hematological Malignancies
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Leukemia
Keywords: hematopoietic stem cell transplantation
Lead sponsor: First Affiliated Hospital of Zhejiang University
Sponsor class: OTHER
First posted: 2020-09-14
Enrollment: 176
Sex: ALL
Minimum age (years): 13
Maximum age (years): 78
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: An open, multi-center, randomized trial comparing haploidentical HSCTs from young non-first-degree and older first-degree donors in hematological malignancies

Detailed description: This is an open, multi-center, randomized trial comparing the clinical outcomes of haploidentical HSCTs from young non-first-degree and older first-degree donors in hematological malignancies. This study is indicated for patients with hematological malignancies including acute leukemias and MDS who are eligible to haploidentical HSCTs. 2 groups of patients will be enrolled with 88 in each group. The clinical criteria including survival, relapse, transplantation-related mortality will be monitored.

Eligibility criteria: Patient Inclusion Criteria:

* Patient age 13-78 years
* Absence of a suitable HLA identical related or unrelated hematopoietic stem cell donor
* Absence of a suitable partially HLA-mismatched (haploidentical), first-degree related donor aged between 18 and 50
* Presence of both HLA haploidentical young non-first-degree (age ≤ 40) and older first-degree (age \>50) donors

Eligible diagnoses:

AML（excluding APL） with at least one of the following:

* median- or high- risk according to the WHO prognostic stratification system
* failure to achieve CR after 2 cycles of induction chemotherapy
* AML arising from MDS or a myeloproliferative disorder, or secondary AML
* patients in CR2 or beyond

Mixed-phenotype acute leukemia (MPAL) in morphological remission Acute lymphoblastic leukemia (T or B) in morphological remission

MDS with at least one of the following:

* IPSS score of INT-2 or greater
* IPSS score of INT-1 with life-threatening cytopenias, including those generally requiring greater than weekly transfusions

  * A first allo-HCT
  * Adequate end-organ function
  * ECOG performance status \< 2
  * No other contraindications for HSCT
  * Signature of the informed consent

Patient Exclusion Criteria

* Availability of suitable HLA identical related or unrelated hematopoietic stem cell donors
* Availability of suitable partially HLA-mismatched (haploidentical), first-degree related donor aged between 18 and 50
* Not the first allo-HCT
* Presence of uncontrolled bacterial, viral, or fungal infection
* Patients with severe heart, lung, liver and kidney insufficiency
* HIV-positive patients
* Women of childbearing potential who are pregnant (β-HCG+) or breast feeding
* Patients with a psychiatric history
* ECOG performance status ≥ 3
* Patients with malignancies other than the primary disease
* Refusal to sign the informed consent

Donor Inclusion Criteria:

* The donor and recipient must be HLA haploidentical
* Meets institutional selection criteria and medically fit to donate
* Lack of recipient anti-donor HLA antibody

Donor Exclusion Criteria:

* The donor and recipient are HLA identical
* Has not donated blood products to recipient

Primary outcomes:
measure: Cumulative incidence of transplant-related nonrelapse mortality (NRM)
timeFrame: 2 years
Secondary outcomes:
measure: Overall survival (OS)
timeFrame: 2 years
measure: Progression-free survival (PFS)
timeFrame: 2 years
measure: Cumulative incidence of disease relapse or progression
timeFrame: 2 years
measure: GVHD-free, relapse-free survival (GRFS)
timeFrame: 2 years
measure: Cumulative incidence of acute grade II-IV GVHD
timeFrame: 180 days
measure: Cumulative incidence of chronic GVHD
timeFrame: 2 years
Other outcomes:
Locations:
city: Changsha
country: China
facility: Xiangya Hospital Central South University
city: Chongqing
country: China
facility: Xinqiao Hospital, State Key Laboratory of Trauma and Chemical Poisoning, Army Medical University
city: Hangzhou
country: China
facility: The First Affiliated Hospital, College of Medicine, Zhejiang University
city: Hangzhou
country: China
facility: Zhejiang Provincial People's Hospital
city: Nanjing
country: China
facility: The First Affiliated Hospital of Nanjing Medical University
city: Ningbo
country: China
facility: The Affiliated People's Hospital of Ningbo University
city: Ningbo
country: China
facility: The First Affiliated Hospital of Ningbo University
city: Soochow
country: China
facility: The First Affiliated Hospital of Soochow University
Study officials:
affiliation: First Affiliated Hospital of Zhejiang University
name: Yi Luo, MD
role: PRINCIPAL_INVESTIGATOR
References: