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Haploidentical Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major

NCT ID: NCT03171831

Last Updated: 2017-05-31

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Clinical Phase

PHASE4

Total Enrollment

30 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-04-01

Study Completion Date

2022-04-01

Brief Summary

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

Detailed Description

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The only curative therapy for thalassemia major remains the replacement of the defective erythropoiesis by allogeneic hematopoietic stem cell transplantation(HSCT). However, this option is unavailable to many patients as a result of a lack of compatible donors.Haploidentical transplantation has been certified as a valuable alternative for hematological malignancy patients lacking a well-matched donor, with results comparable matched HLA-identical sibling donors or unrelated donors.The objective of this study is to test the feasibility of haploidentical HSCT for patients with thalassemia major.

Conditions

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Thalassemia Major

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Haploidentical HSCT

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）

Group Type EXPERIMENTAL

Busulfan

Intervention Type DRUG

Busulfan（4 mg/kg/day,4 days）

Cyclophosphamide

Intervention Type DRUG

Cyclophosphamide（50 mg/kg/day,4 days）

Fludarabine

Intervention Type DRUG

Fludarabine（50 mg/m2/day,3 days）

Mycophenolate mofetil

Intervention Type DRUG

Mycophenolate mofetil（0.25g/day）

Tacrolimus

Intervention Type DRUG

Tacrolimus（0.03mg/kg/day）

Methotrexate

Intervention Type DRUG

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

Thymoglobulin

Intervention Type DRUG

Thymoglobulin（2.5 mg/kg/day,4 days）

Basiliximab

Intervention Type DRUG

Basiliximab（10mg on day 0 and +4）

Interventions

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Busulfan

Busulfan（4 mg/kg/day,4 days）

Intervention Type DRUG

Cyclophosphamide

Cyclophosphamide（50 mg/kg/day,4 days）

Intervention Type DRUG

Fludarabine

Fludarabine（50 mg/m2/day,3 days）

Intervention Type DRUG

Mycophenolate mofetil

Mycophenolate mofetil（0.25g/day）

Intervention Type DRUG

Tacrolimus

Tacrolimus（0.03mg/kg/day）

Intervention Type DRUG

Methotrexate

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

Intervention Type DRUG

Thymoglobulin

Thymoglobulin（2.5 mg/kg/day,4 days）

Intervention Type DRUG

Basiliximab

Basiliximab（10mg on day 0 and +4）

Intervention Type DRUG

Other Intervention Names

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Bu Cy Flu MMF FK506 MTX ATG Simulect

Eligibility Criteria

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Inclusion Criteria

1. Diagnosed with thalassemia major
2. Lacking of a HLA-identical sibling donor or unrelated donor
3. Indication of haploidentical hematopoietic stem cell transplantation
4. No restrictions for transplantation

Exclusion Criteria

1. Any restriction for transplantation
2. No indication of haploidentical hematopoietic stem cell transplantation
Minimum Eligible Age

2 Years

Maximum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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First Affiliated Hospital of Guangxi Medical University

OTHER

Sponsor Role lead

Responsible Party

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Yongrong Lai

Director of the Hematology Department

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Yongrong Lai, MD

Role: PRINCIPAL_INVESTIGATOR

First Affiliated Hospital of Guangxi Medical University

Locations

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First Affiliated Hospital of Guangxi Medical University

Nanning, Guangxi, China

Site Status RECRUITING

Countries

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China

Central Contacts

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Yongrong Lai, MD

Role: CONTACT

Phone: +86（0771）5356510

Email: [email protected]

Meiqing Wu, MD

Role: CONTACT

Phone: +86（0771）5356510

Email: [email protected]

Facility Contacts

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Yongrong Lai, MD

Role: primary

Other Identifiers

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Guangxi-Haplo-HSCT-2016

Identifier Type: -

Identifier Source: org_study_id