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Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation in Children and Adolescents

This study is currently recruiting participants.
Verified March 2017 by Ho Joon Im, Asan Medical Center
Sponsor:
ClinicalTrials.gov Identifier:
NCT02014506
First Posted: December 18, 2013
Last Update Posted: March 28, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Information provided by (Responsible Party):
Ho Joon Im, Asan Medical Center
  Purpose
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.

Condition Intervention Phase
Haploidentical Hematopoietic Stem Cell Transplantation Malignant Disease Non-malignant Disease Drug: Fludarabine Drug: Cyclophosphamide Biological: anti-thymocyte globulin Biological: filgrastim Radiation: Total body irradiation Procedure: TCRαβ-depleted hematopoietic cell transplantation Device: CliniMACS Phase 1 Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation in Children and Adolescents

Further study details as provided by Ho Joon Im, Asan Medical Center:

Primary Outcome Measures:
  • To evaluate tralsplant-related mortality after haploidentical hematopoietic stem cell transplantation using TCRαβ-depleted graft [ Time Frame: 1 year posttransplant ]

Secondary Outcome Measures:
  • To assess engraftment and graft failure [ Time Frame: 28 days posttransplant ]
  • To estimate the risk of acute GVHD [ Time Frame: 100 days posttransplant ]
  • To estimate the incidence of relapse [ Time Frame: 100 days and 1 year post-transplant ]
  • To estimate the incidence and severity of chronic GVHD [ Time Frame: 1 year posttransplant ]
  • To estimate the overall survival [ Time Frame: 1 year posttransplant ]
  • To estimate the incidence of bacterial, fungal and viral infection [ Time Frame: 100 days and 1 year posttransplant ]
  • To estimate the reactivation rate of CMV, EBV [ Time Frame: 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 ]
  • 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 ]
  • To assess event free survival [ Time Frame: 1 year posttransplant ]

Estimated Enrollment: 30
Actual Study Start Date: January 2017
Estimated Study Completion Date: December 2018
Estimated Primary Completion Date: December 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: HAPLO Drug: Fludarabine
40mg/M2 once daily IV on days -7 to -2
Drug: Cyclophosphamide
50 mg/kg IV on day -3 and -2
Biological: anti-thymocyte globulin 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

  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   up to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02014506


Contacts
Contact: Ho Joon Im, MD, PhD 82-2-3010-3371 hojim@amc.seoul.kr

Locations
Korea, Republic of
Asan Medical Center Recruiting
Seoul, Korea, Republic of, 138-736
Contact: Ho Joon Im, MD & PhD    82-2-3010-3371    hojim@amc.seoul.kr   
Principal Investigator: Ho Joon Im, MD & PhD         
Sponsors and Collaborators
Asan Medical Center
Investigators
Principal Investigator: Ho Joon Im, MD, PhD Asan Medical Center
  More Information

Additional Information:
Publications:
Responsible Party: Ho Joon Im, Professor, Asan Medical Center
ClinicalTrials.gov Identifier: NCT02014506     History of Changes
Other Study ID Numbers: AMCPHO-SCT1303
First Submitted: December 4, 2013
First Posted: December 18, 2013
Last Update Posted: March 28, 2017
Last Verified: March 2017

Keywords provided by Ho Joon Im, Asan Medical Center:
Children and adolescents
Malignant disease
Non-malignant disease
TCRαβ depletion
Haploidentical hematopoietic stem cell transplantation

Additional relevant MeSH terms:
Cyclophosphamide
Antilymphocyte Serum
Fludarabine
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antirheumatic Agents
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists