NCT02342145

Brief Summary

The purpose of this study is to evaluate the basiliximab for prevention of graft-versus-host disease in unrelated allo-genetic hematopoietic stem cell transplantation for thalassemia major. The objective was to evaluate the effect and safety of basiliximab for acute graft-versus-host disease.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
205

participants targeted

Target at P75+ for phase_4

Timeline
Completed

Started Apr 2015

Longer than P75 for phase_4

Geographic Reach
1 country

3 active sites

Status
completed

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

First Submitted

Initial submission to the registry

January 14, 2015

Completed
5 days until next milestone

First Posted

Study publicly available on registry

January 19, 2015

Completed
2 months until next milestone

Study Start

First participant enrolled

April 1, 2015

Completed
7.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 20, 2022

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

August 20, 2022

Completed
Last Updated

June 18, 2025

Status Verified

June 1, 2025

Enrollment Period

7.4 years

First QC Date

January 14, 2015

Last Update Submit

June 15, 2025

Conditions

Beta-Thalassemia MajorGraft-versus-host Disease (GVHD)

Keywords

beta-Thalassemia majorGraft-versus-host diseaseBasiliximabAllogeneic hematopoietic stem cell transplantationmatched unrelated donors

Outcome Measures

Primary Outcomes (1)

  • grade II-IV acute graft-versus-host disease incidence

    The cumulative incidence of grade II-IV acute graft-versus-host disease after matched unrelated donor hematopoietic stem cell transplantation +100d.

    100 days after matched unrelated donor hematopoietic stem cell transplantation.

Secondary Outcomes (6)

  • Implantation rate

    three years

  • Transplanted-related mortality

    three years

  • Infection incidence

    three years

  • Chronic graft-versus-host-disease incidence

    five years

  • Overall survival

    three years

  • +1 more secondary outcomes

Study Arms (2)

group A

EXPERIMENTAL

The patients were used FK506 (0.03mg/kg/day), Methotrexate (15mg/m2 on +1d, 10mg/m2 on +3d, +6d, +11d), Mycophenolate Mofetil (0.25g/d, days-1 to 90) and Basiliximab (use 10mg for weight under 35kg, 20mg for over 35kg, 0d and +4d) for prevention of acute graft-versus-host-disease.

Drug: Basiliximab,Drug: TacrolimusDrug: MethotrexateDrug: Mycophenolate mofetil

group B

ACTIVE COMPARATOR

The patients were used FK506 (0.03mg/kg/day), Methotrexate (15mg/m2 on +1d, 10mg/m2 on +3d, +6d, +11d), Mycophenolate Mofetil (0.25g/d, days-1 to 90) for prevention of acute graft-versus-host-disease.

Drug: TacrolimusDrug: MethotrexateDrug: Mycophenolate mofetil

Interventions

Basiliximab,DRUG

Basiliximab was used on 0d (after transplantation) and +4d (10mg for weight under 35kg, 20mg for over 35kg) .

Also known as: chimeric mouse-human antiCD25
group A
TacrolimusDRUG

Specifically Tacrolimus was used by intravenous drip infusion on 0.03mg/kg dosage from -1d and change to 0.1mg/kg oral when gastrointestinal function recovers. The blood concentrations of cyclosporine A was maintained 5-10 ng/ml.

Also known as: FK506
group Agroup B
MethotrexateDRUG

Methotrexate was used 15mg/m2 on +1d and 10mg/m2 on +3d,+6d,+11d by intravenous for prevention of graft-versus-host-disease.

Also known as: Ametnopterin
group Agroup B
Mycophenolate mofetilDRUG

Mycophenolate mofetil was used 0.25g per day from -1d to 90d for prevention of graft-versus-host-disease.

Also known as: Mycophenolic acid
group Agroup B

Eligibility Criteria

Age2 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • a diagnosis of TM with hemoglobin electrophoresis, a genetic diagnosis of -thalassemia by DNA analysis, and blood transfusion dependence;
  • a cardiac ejection fraction \>50%;
  • normal pulmonary function tests and pulmonary examination results;
  • normal kidney function.

You may not qualify if:

  • aspartate aminotransferase level \>4- fold the upper limit of normal range in our institution's laboratory criteria;
  • uncontrolled bacterial, viral, or fungal infection;
  • positive serology for HIV;
  • cytomegalovirus (CMV) or Epstein-Barr virus (EBV) copy number \>200 copies/mL in blood by quantitative PCR. Patients positive for hepatitis C or hepatitis B virus were also excluded.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (3)

Liuzhou Worker's Hospital

Liuchow, Guangxi, China

Location

The affiliated hospital of guangxi medical university

Nanning, Guangxi, 530021, China

Location

Hainan general Hospital

Haikou, Hainan, China

Location

Related Publications (5)

  • Smiers FJ, Krishnamurti L, Lucarelli G. Hematopoietic stem cell transplantation for hemoglobinopathies: current practice and emerging trends. Pediatr Clin North Am. 2010 Feb;57(1):181-205. doi: 10.1016/j.pcl.2010.01.003.

    PMID: 20307718BACKGROUND

  • Feng Z, Sun E, Lan H, Zhang C, Li Q, Zhu W. Unrelated donor bone marrow transplantation for beta-thalassemia major: an experience from China. Bone Marrow Transplant. 2006 Jan;37(2):171-4. doi: 10.1038/sj.bmt.1705193.

    PMID: 16273116BACKGROUND

  • Hongeng S, Pakakasama S, Chuansumrit A, Sirachainan N, Kitpoka P, Udomsubpayakul U, Ungkanont A, Jootar S. Outcomes of transplantation with related- and unrelated-donor stem cells in children with severe thalassemia. Biol Blood Marrow Transplant. 2006 Jun;12(6):683-7. doi: 10.1016/j.bbmt.2006.02.008.

    PMID: 16737942BACKGROUND

  • Wang HX, Yan HM, Wang ZD, Xue M, Liu J, Guo ZK. Haploidentical hematopoietic stem cell transplantation in hematologic malignancies with G-CSF mobilized bone marrow plus peripheral blood stem cells grafts without T cell depletion: a single center report of 29 cases. Leuk Lymphoma. 2012 Apr;53(4):654-9. doi: 10.3109/10428194.2011.624225. Epub 2011 Dec 5.

    PMID: 21929286BACKGROUND

  • Hu LD, Chen H, Jiang M, Li BT, Yu ZY, Li YH. [The role of CD25 antibody in unrelated hematopoietic stem cell transplantation]. Zhonghua Nei Ke Za Zhi. 2005 Nov;44(11):848-50. Chinese.

    PMID: 16316569BACKGROUND

MeSH Terms

Conditions

beta-ThalassemiaGraft vs Host Disease

Interventions

BasiliximabTacrolimusMethotrexateMycophenolic Acid

Condition Hierarchy (Ancestors)

ThalassemiaAnemia, Hemolytic, CongenitalAnemia, HemolyticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesHemoglobinopathiesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesImmune System Diseases

Intervention Hierarchy (Ancestors)

Antibodies, Monoclonal, HumanizedAntibodies, MonoclonalAntibodiesImmunoglobulinsImmunoproteinsBlood ProteinsProteinsAmino Acids, Peptides, and ProteinsSerum GlobulinsGlobulinsMacrolidesLactonesOrganic ChemicalsAminopterinPterinsPteridinesHeterocyclic Compounds, 2-RingHeterocyclic Compounds, Fused-RingHeterocyclic CompoundsCaproatesAcids, AcyclicCarboxylic AcidsFatty AcidsLipids

Study Officials

  • yongrong lai, PhD

    First Affiliated Hospital of Guangxi Medical University

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 4
Allocation
RANDOMIZED
Masking
NONE
Purpose
PREVENTION
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR INVESTIGATOR
PI Title
The First Affiliated Hospital of Guangxi Medical University

Study Record Dates

First Submitted

January 14, 2015

First Posted

January 19, 2015

Study Start

April 1, 2015

Primary Completion

August 20, 2022

Study Completion

August 20, 2022

Last Updated

June 18, 2025

Record last verified: 2025-06

Locations

CN(3)