Umbilical Cord Blood-Supported Haplo-HSCT for Aplastic Anemia Treatment Study

NCT06650553 | Recruiting DMC

• 1 other identifier: IIT-2024-018-FS02
• Study Type: interventional
• Target: 110
• Locations: 1 country
• Sites: 1

Brief Summary

Aplastic anemia (AA) is a rare bone marrow failure syndrome with an annual incidence of about 0.74/100,000, affecting all ages but more common in the elderly. It's divided into congenital and acquired forms, with the latter being more prevalent. The primary acquired form is linked to T lymphocyte activation and genetic factors. The best treatment is allogeneic hematopoietic stem cell transplantation (allo-HSCT), with a near 90% cure rate. Sibling allo-HSCT is ideal but finding a match is challenging. For those who relapse after immunosuppressive therapy, haploidentical HSCT is a viable option despite risks like graft failure and GVHD. Advances in transplantation have made haplo-HSCT's efficacy comparable to other methods. Recent studies suggest co-transplantation with umbilical cord blood cells can improve outcomes by hastening hematopoietic recovery and prognosis. Our study will evaluate the feasibility and safety of this approach in AA treatment, comparing it to sibling fully matched transplantation, with a focus on infection rates, GVHD incidence, TRM, and EFS, aiming to enhance treatment practices and benefit patients and the medical industry.

Conditions

Aplastic Anemias

Keywords

Aplastic Anemias; Stem Cell Transplantation; Umbilical Cord Blood

Outcome Measures

Primary Outcomes (1)

• Time to neutrophil engraftment

  Neutrophil engraftment was defined as the first of three consecutive days on which the patient's absolute neutrophil count was 500/uL.

  60 days

Secondary Outcomes (6)

• Time to platelet engraftment

  60 days

• Incidence of infections (including CMV and EBV incidence)

  100 days,6 months,12 months,24 months

• Overall survival rate (OS)

  24 months

• Event-free survival (EFS)

  24 months

• Incidence of acute/chronic GvHD

  24 months

Study Arms (1)

Umbilical Cord Blood-Supported Haplo-HSCT for Aplastic Anemia Treatment Study

EXPERIMENTAL

In patients with AA undergoing hematopoietic stem cell transplantation, the feasibility of the umbilical cord blood combined with haploidentical transplantation scheme is assessed by comparing it with sibling fully matched transplantation using a non-inferiority analysis method (with the time of neutrophil engraftment after transplantation as the primary efficacy indicator). At the same time, indicators such as the time of platelet engraftment, infection incidence (including CMV, EBV incidence), acute/chronic GvHD incidence, transplant-related mortality (TRM), event-free survival (EFS), and overall survival (OS) are monitored to evaluate the safety and effectiveness of this scheme.

Procedure: Clinically diagnosed AA patients are divided into HLA-matched HSCT group and umbilical cord blood-supported haplo-HSCT group.

Interventions

Clinically diagnosed AA patients are divided into HLA-matched HSCT group and umbilical cord blood-supported haplo-HSCT group. PROCEDURE

Conditioning regimen consisted of the following1)Severe aplastic anemia(SAA):FC-ATG:Flu 30 mg/m\^2/d ×4d (- 7d- -4d), CTX 22.5 -25mg/kg/d × 4d (-7d- -4d), ATG 7.5mg in total（- 5d- -2d).2)Transfusion-dependent non-severe aplastic anemia（TD-NSAA) and Paroxysmal nocturnal hemoglobinuria (PNH) acquired clonal-aplastic anemia(AA):BU/FC-ATG:Bu 0.8mg/kg(q6h -8d）+FC-ATG. The GVHD prophylaxis program was Application of Anti-CD25 Humanized Monoclonal Antibody 50mg (+1d,+4d);cyclosporine A + mycophenolate mofetil (MMF)+short course methotrexate (MTX).The infusion time of umbilical cord blood (UCB) stem cells in the UCB-assisted haploidentical hematopoietic stem cell transplantation (UCB-Haplo-HSCT) group: 4 hours before the infusion of peripheral blood stem cells.In the follow-up phase, outcome evaluation indicators must be collected according to the follow-up plan, ensuring data quality.

Umbilical Cord Blood-Supported Haplo-HSCT for Aplastic Anemia Treatment Study

Eligibility Criteria

• Age: 6 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• A patient age of 6-75 years
• Patients diagnosed with Severe Aplastic Anemia (SAA) , Transfusion-Dependent Non-Severe Aplastic Anemia (TD-NSAA) or Paroxysmal nocturnal hemoglobinuria (PNH) acquired clonal-aplastic anemia(AA) according to the Chinese Guidelines for the Diagnosis and Treatment of Aplastic Anemia (2022 Edition) and suitable for allo-HSCT
• Subjects (or their legally acceptable representatives) must have signed an informed consent document indicating that they understand the purpose of and procedures required for the study and are willing to participate in the study

You may not qualify if:

• Any abnormality in a vital sign (e.g., heart rate, respiratory rate, or blood pressure)
• Patients with any conditions not suitable for the trial (investigators' decision)

Sponsors & Collaborators

• Shanxi Bethune Hospital: lead

Study Sites (1)

Shanxi Bethune Hospital

Taiyuan, Shanxi, 030000, China

RECRUITING

MeSH Terms

Conditions

Anemia, Aplastic

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases

Study Officials

• Tao Wang, Dr.

  Shanxi Bethune Hospital Regulatory Authority

  STUDY DIRECTOR

Central Study Contacts

Tao Wang, Dr.

CONTACT

13835175119; wangtao99699@163.com

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 28, 2024
• First Posted: October 21, 2024
• Study Start: June 1, 2024
• Primary Completion (Estimated): June 1, 2027
• Study Completion (Estimated): July 1, 2027
• Last Updated: August 14, 2025

Record last verified: 2024-09

Locations

CN (1)