Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation in Children and Adolescents

March 24, 2017 updated by: Ho Joon Im, Asan Medical Center
Purpose of study: This phase I/II trial is to evaluate the safety and feasibility of TCRαβ-depleted graft from haploidentical family donors in treating children and adolescents with malignant or non-malignant diseases.

Study Overview

Status

Unknown

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Anticipated)

30

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Korea, Republic of
      • Seoul, Korea, Republic of, 138-736
        • Recruiting
        • Asan Medical Center
        • Contact:
        • Principal Investigator:
          • Ho Joon Im, MD & PhD

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

No older than 21 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

A. Disease inclusions

  1. Hematologic malignancy:

    • Acute lymphoblastic leukemia including induction failure, CR1 (Ph+, t(4:11), hypodiploid and other very high risk features), ≥ CR2, infant ALL with MLL or other unfavorable features
    • Acute myeloid leukemia excluding CR1 with t(8:21), inv(16), t(15:17), and Down syndrome
    • Myelodysplastic syndrome: RCC with -7 or RCC in need of transfusion
    • Chronic myeloid leukemia in AP
    • Juvenile myelomonocytic leukemia
    • Malignant lymphoma, NHL or HD, after failed autologous HSCT
    • Other

  2. Non-hematologic malignancy

    • Relapsed or refractory solid tumors including neuroblastoma, rhabdomyosarcoma and so on

  3. Non-malignant hematologic disease

    • Acquired severe and very severe aplastic anemia
    • Fanconi anemia
    • Paroxysmal nocturnal hemoglobinuria
    • Congenital dyserythropoietic anemia
    • Others

  4. Inherited or metabolic disease

    • Hemophagocytic lymphohistiocytosis
    • Malignant osteopetrosis
    • Storage diseases
    • Others B. Recipient inclusions

1. Age < 21 years 2. No HLA-identical stem cell donor available 3. Lansky-Play performance score >60 4. No active infection at the time of transplantation

Exclusion Criteria:

  1. HIV-infection
  2. Presence of active and serious infection
  3. Cardiac ejection fraction <35% on echocardiography
  4. Severe pulmonary dysfunction (DLCO <30%)
  5. Liver function abnormalities with bilirubin >4mg/dL and elevation of transaminases > 400U/L
  6. Concurrent severe or uncontrolled medical disease
  7. Patients who are pregnant
  8. Patients unwilling or unable to comply with the protocol or unable to give informed consent

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: HAPLO
Biological: anti-thymocyte globulin
Drug: Fludarabine
40mg/M2 once daily IV on days -7 to -2
Drug: Cyclophosphamide
50 mg/kg IV on day -3 and -2
Biological: filgrastim
Beginning on day 4 and continuing until blood counts recover
Radiation: Total body irradiation
200 cGy per day on D-6 to -4 (eligible disease except aplastic anemia) 200 cGy per day on D-5 & -4 (severe aplastic anemia)
Procedure: TCRαβ-depleted hematopoietic cell transplantation
Device: CliniMACS
Immunogenetic depletion of TCRαβ cells

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
To evaluate tralsplant-related mortality after haploidentical hematopoietic stem cell transplantation using TCRαβ-depleted graft
Time Frame: 1 year posttransplant
1 year posttransplant

Secondary Outcome Measures

Outcome Measure
Time Frame
To assess engraftment and graft failure
Time Frame: 28 days posttransplant
28 days posttransplant
To estimate the risk of acute GVHD
Time Frame: 100 days posttransplant
100 days posttransplant
To estimate the incidence of relapse
Time Frame: 100 days and 1 year post-transplant
100 days and 1 year post-transplant
To estimate the incidence and severity of chronic GVHD
Time Frame: 1 year posttransplant
1 year posttransplant
To estimate the overall survival
Time Frame: 1 year posttransplant
1 year posttransplant
To estimate the incidence of bacterial, fungal and viral infection
Time Frame: 100 days and 1 year posttransplant
100 days and 1 year posttransplant
To estimate the reactivation rate of CMV, EBV
Time Frame: 100 days and 1 year posttransplant
100 days and 1 year posttransplant
To evaluate the immune reconstitution of T, B, and NK cells
Time Frame: days 7, 14, 21, 28, 60, 90, 180, 270, and 365 days post-transplant
days 7, 14, 21, 28, 60, 90, 180, 270, and 365 days post-transplant
To evaluate the lineage-specific chimerism using flow cytomery of CD3+, CD19, CD56, TCR αβ, and TCRγδ at pre-transplant
Time Frame: days 7, 10, 14, 21, 28, 60, 90, 180, 270 and 365 post-transplant
days 7, 10, 14, 21, 28, 60, 90, 180, 270 and 365 post-transplant
To assess event free survival
Time Frame: 1 year posttransplant
1 year posttransplant

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Asan Medical Center

Investigators

  • Principal Investigator: Ho Joon Im, MD, PhD, Asan Medical Center

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 1, 2017

Primary Completion (Anticipated)

December 1, 2018

Study Completion (Anticipated)

December 1, 2018

Study Registration Dates

First Submitted

December 4, 2013

First Submitted That Met QC Criteria

December 12, 2013

First Posted (Estimate)

December 18, 2013

Study Record Updates

Last Update Posted (Actual)

March 28, 2017

Last Update Submitted That Met QC Criteria

March 24, 2017

Last Verified

March 1, 2017

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • AMCPHO-SCT1303

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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