NCT06378060

Brief Summary

The aim of this study was to evaluate the safety and efficacy of a modified allogeneic hematopoietic stem cell transplantation regimen for aplastic anemia.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
30

participants targeted

Target at P25-P50 for phase_2

Timeline
11mo left

Started Mar 2024

Typical duration for phase_2

Geographic Reach
1 country

1 active site

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress71%
Mar 2024Apr 2027

Study Start

First participant enrolled

March 1, 2024

Completed
2 months until next milestone

First Submitted

Initial submission to the registry

April 17, 2024

Completed
5 days until next milestone

First Posted

Study publicly available on registry

April 22, 2024

Completed
1.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 1, 2026

Completed
1 year until next milestone

Study Completion

Last participant's last visit for all outcomes

April 1, 2027

Expected
Last Updated

April 22, 2024

Status Verified

March 1, 2024

Enrollment Period

2.1 years

First QC Date

April 17, 2024

Last Update Submit

April 19, 2024

Conditions

Severe Aplastic Anemia

Keywords

Severe Aplastic AnemiaModified transplantation systemRuxolitinibMelphalan

Outcome Measures

Primary Outcomes (2)

  • overall survival (OS)

    2-year overall survival (OS) and graft-versus-host disease and relapse-free survival (GRFS) after transplantation

    [Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen]

  • Acute graft-versus-host disease incidence

    [Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen]

Secondary Outcomes (5)

  • Transplantation-related motality

    [Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen]

  • Chronic graft-versus-host disease incidence

    [Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen]

  • incidence of Intensive fungal disease

    [Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen]

  • EB virus reactivation rate

    [Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen]

  • CMV reactivation rate

    [Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen]

Study Arms (1)

Experimental group

EXPERIMENTAL
Drug: Modified transplantation system

Interventions

Modified transplantation systemDRUG

Conditioning regimen: fludarabine 30mg/m2×5\~6d,cyclophosphamide 50mg/kg×2d, melphalan 100mg/m2×1d aGVHD prophylaxis: PTCY 25mg/kg×2d, ATG 4.5mg/kg total, MMF 15mg/kg +5d\~+35d, Ruxolitinib 5mg bid -1d\~+50d and 2.5mg bid +51d\~+110d, CSA

Experimental group

Eligibility Criteria

Age3 Years - 65 Years
Sexall
Healthy VolunteersYes
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Patients with benign or malignant hematological diseases such as leukemia, lymphoma, thalassemia, aplastic anemia, etc. diagnosed by NCCN guidelines, and requiring allogeneic hematopoietic stem cell transplantation as determined by the researchers;
  • Age 3-65 years old;
  • Weight 10Kg-100Kg;
  • Eastern Cooperative Oncology Group (ECOG) score ≤3;
  • No major organ injury (ECG ejection fraction \>45%; bilirubin \< 2 times the upper limit of normal value; AST and ALT \< 3 times the upper limit of normal value; serum creatinine \< 2 times the upper limit of normal value);
  • No severe infection;
  • Subjects voluntarily participated in this clinical trial and signed the informed consent.

You may not qualify if:

  • patients with nonhematologic diseases who are not eligible for transplantation or who do not wish to receive transplantation;
  • patients with an expected survival of less than 1 month;
  • patients with previous autologous or allogeneic hematopoietic stem cell transplantation;
  • pregnant patients;
  • patients with severe mental or neurological disorders that would affect the ability to provide informed consent and/or to report or observe adverse events;
  • other conditions that the investigator determines to be inappropriate for enrollment.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

920th Hospital of Joint Logistics Support Force of People's Liberation Army of China

Kunming, Yunnan, 650000, China

RECRUITING

MeSH Terms

Conditions

Anemia, Aplastic

Condition Hierarchy (Ancestors)

AnemiaHematologic DiseasesHemic and Lymphatic DiseasesBone Marrow Failure DisordersBone Marrow Diseases

Central Study Contacts

wang PI sanbin, Doctor

CONTACT

0871-64774206Wangsanbin2022@126.com

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

April 17, 2024

First Posted

April 22, 2024

Study Start

March 1, 2024

Primary Completion

April 1, 2026

Study Completion (Estimated)

April 1, 2027

Last Updated

April 22, 2024

Record last verified: 2024-03

Data Sharing

IPD Sharing
Will not share

Locations

CN(1)