NCT03171831

Brief Summary

The purpose of the study is to assess the safety and efficacy of haploidentical hematopoietic stem cell transplantation for patients with thalassemia major.

Trial Health

43
At Risk

Trial Health Score

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

Trial has exceeded expected completion date
Enrollment
30

participants targeted

Target at below P25 for phase_4

Timeline
Completed

Started Apr 2017

Longer than P75 for phase_4

Geographic Reach
1 country

1 active site

Status
unknown

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 Start

First participant enrolled

April 1, 2017

Completed
17 days until next milestone

First Submitted

Initial submission to the registry

April 18, 2017

Completed
1 month until next milestone

First Posted

Study publicly available on registry

May 31, 2017

Completed
4.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 31, 2021

Completed
3 months until next milestone

Study Completion

Last participant's last visit for all outcomes

April 1, 2022

Completed
Last Updated

May 31, 2017

Status Verified

May 1, 2017

Enrollment Period

4.8 years

First QC Date

April 18, 2017

Last Update Submit

May 27, 2017

Conditions

Thalassemia Major

Outcome Measures

Primary Outcomes (1)

  • Overall Survival

    2-years overall survival

    2 years

Secondary Outcomes (6)

  • Disease Free Survival

    2 years

  • Engraftment

    30 days

  • Transplant Related Mortality

    1 year

  • Cumulative Incidence of acute Graft Versus Host Disease

    180 days

  • Cumulative Incidence of chronic Graft Versus Host Disease

    2 years

  • +1 more secondary outcomes

Study Arms (1)

Haploidentical HSCT

EXPERIMENTAL

Procedure: Haploidentical hematopoietic stem cell transplantation from a related donor (partially matched sibling, father or mother). Conditioning: Busulfan （4 mg/kg/day,4 days） + Cyclophosphamide （50 mg/kg/day,4 days）+ Fludarabine （50 mg/m2/day,3 days） GVHD Prophylaxis：Mycophenolate mofetil（0.25g/day）+ Tacrolimus（0.03mg/kg/day）+ Methotrexate（15mg/m2 on day +1,10mg/m2 on day +3,+6,+11）+ Thymoglobulin（2.5 mg/kg/day,4 days）+Basiliximab（10mg on day 0 and +4）

Drug: BusulfanDrug: CyclophosphamideDrug: FludarabineDrug: Mycophenolate mofetilDrug: TacrolimusDrug: MethotrexateDrug: ThymoglobulinDrug: Basiliximab

Interventions

BusulfanDRUG

Busulfan（4 mg/kg/day,4 days）

Also known as: Bu
Haploidentical HSCT
CyclophosphamideDRUG

Cyclophosphamide（50 mg/kg/day,4 days）

Also known as: Cy
Haploidentical HSCT
FludarabineDRUG

Fludarabine（50 mg/m2/day,3 days）

Also known as: Flu
Haploidentical HSCT
Mycophenolate mofetilDRUG

Mycophenolate mofetil（0.25g/day）

Also known as: MMF
Haploidentical HSCT
TacrolimusDRUG

Tacrolimus（0.03mg/kg/day）

Also known as: FK506
Haploidentical HSCT
MethotrexateDRUG

Methotrexate（15mg/m2 on day +1,10mg/m2 on day +3,+6,+11）

Also known as: MTX
Haploidentical HSCT
ThymoglobulinDRUG

Thymoglobulin（2.5 mg/kg/day,4 days）

Also known as: ATG
Haploidentical HSCT
BasiliximabDRUG

Basiliximab（10mg on day 0 and +4）

Also known as: Simulect
Haploidentical HSCT

Eligibility Criteria

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

You may qualify if:

  • Diagnosed with thalassemia major
  • Lacking of a HLA-identical sibling donor or unrelated donor
  • Indication of haploidentical hematopoietic stem cell transplantation
  • No restrictions for transplantation

You may not qualify if:

  • Any restriction for transplantation
  • No indication of haploidentical hematopoietic stem cell transplantation

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

First Affiliated Hospital of Guangxi Medical University

Nanning, Guangxi, 510515, China

RECRUITING

MeSH Terms

Conditions

beta-Thalassemia

Interventions

BusulfanCyclophosphamidefludarabineInfluenza VaccinesMycophenolic AcidTacrolimusMethotrexatethymoglobulinBasiliximab

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Butylene GlycolsGlycolsAlcoholsOrganic ChemicalsMesylatesAlkanesulfonatesAlkanesulfonic AcidsAlkanesHydrocarbons, AcyclicHydrocarbonsSulfonic AcidsSulfur AcidsSulfur CompoundsPhosphoramide MustardsNitrogen Mustard CompoundsMustard CompoundsHydrocarbons, HalogenatedPhosphoramidesOrganophosphorus CompoundsViral VaccinesVaccinesBiological ProductsComplex MixturesCaproatesAcids, AcyclicCarboxylic AcidsFatty AcidsLipidsMacrolidesLactonesAminopterinPterinsPteridinesHeterocyclic Compounds, 2-RingHeterocyclic Compounds, Fused-RingHeterocyclic CompoundsAntibodies, Monoclonal, HumanizedAntibodies, MonoclonalAntibodiesImmunoglobulinsImmunoproteinsBlood ProteinsProteinsAmino Acids, Peptides, and ProteinsSerum GlobulinsGlobulins

Study Officials

  • Yongrong Lai, MD

    First Affiliated Hospital of Guangxi Medical University

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Yongrong Lai, MD

CONTACT

+86（0771）5356510laiyongrong@hotmail.com

Meiqing Wu, MD

CONTACT

+86（0771）5356510wumeiqing1309@163.com

Study Design

Study Type
interventional
Phase
phase 4
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Director of the Hematology Department

Study Record Dates

First Submitted

April 18, 2017

First Posted

May 31, 2017

Study Start

April 1, 2017

Primary Completion

December 31, 2021

Study Completion

April 1, 2022

Last Updated

May 31, 2017

Record last verified: 2017-05

Locations

CN(1)