HLA-haploidentical Hematopoietic Stem Cell Transplantation for Children and Adolescents With Acute Leukemia, Myelodysplastic Syndrome and Solid Tumors

NCT01509300 | Unknown Phase 1

• 1 other identifier: AMCPHO-SCT0902
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief Summary

RATIONALE: Conditioning with total body irradiation (TBI) and fludarabine, cyclophosphamide and anti-thymocyte globulin may induce the engraftment cross the immunologic barrier in the setting of HLA-haploidentical allogeneic hematopoietic cell transplantation. In addition, T-cell depletion may contribute to prevent developing severe acute graft versus host disease (GVHD) in haploidentical transplantation. PURPOSE: This phase I/II trial is to evaluate the safety and efficacy of TBI, fludarabine, cyclophosphamide and antithymocyte globulin with T-cell depleted graft from haploidentical donors in treating patients with acute leukemia and myelodysplastic syndrome.

Conditions

Acute Leukemia; Myelodysplastic Syndrome; Solid Tumors

Keywords

TBI; Fludarabine; CD3 depletion; Children and adolescents; Haploidentical hematopoietic stem cell transplantation

Outcome Measures

Primary Outcomes (1)

• Transplantation-related mortality and overall survival of TBI, Fludarabine, Cyclophosphamide and anti-thymocyte globulin for engraftment of CD3 depleted haploidentical peripheral blood stem cells.

  2 years post-transplant

Secondary Outcomes (4)

• Engraftment and graft failure rates

  28 days engraftment and graft failure

• Incidence of acute GVHD

  100 days post-transplant

• Treatment related mortality

  100 days post-transplant

• Relapse rate and overall survival

  2 year after transplantation

Study Arms (1)

HAPLO

EXPERIMENTAL

Biological: anti-thymocyte globulin; Biological: filgrastim; Radiation: Total body irradiation; Drug: Fludarabine; Drug: cyclophosphamide; Drug: Tacrolimus; Drug: Mycophenolate mofetil; Drug: Rituximab

Interventions

anti-thymocyte globulin BIOLOGICAL

On days -10 to -9

HAPLO

filgrastim BIOLOGICAL

Beginning on day 4 and continuing until blood counts recover

HAPLO

Total body irradiation RADIATION

2Gy D-6 to D-4

HAPLO

Fludarabine DRUG

30mg/M2 once daily IV on days -8 to -4

HAPLO

cyclophosphamide DRUG

60 mg/kg IV on day-3 and -2

HAPLO

Tacrolimus DRUG

begin on 0

HAPLO

Mycophenolate mofetil DRUG

begin on 0

HAPLO

Rituximab DRUG

375mg/m2 on day +21

HAPLO

Eligibility Criteria

• Age: Up to 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Disease characteristics
• Acute lymphoblastic leukemia (first remission, high risk; beyond first remission; refractory)
• Acute myeloblastic leukemia (first remission, high risk; beyond first remission; refractory)
• Myelodysplastic syndrome
• Solid tumors (Refractory/relapse)
• No HLA-identical family member or closely matched (8 or 7 of 8 HLA-locus match) unrelated marrow donor available
• HLA-haploidentical related donor available

You may not qualify if:

• Active fungal infections
• HIV positive
• Pregnant or nursing

Sponsors & Collaborators

• Asan Medical Center: lead

Study Sites (1)

Asan Medical Center

Seoul, 138-736, South Korea

RECRUITING

Related Publications (3)

• Koh KN, Im HJ, Kim BE, Choi ES, Jang S, Kwon SW, Park CJ, Seo JJ. Haploidentical haematopoietic stem cell transplantation using CD3 or CD3/CD19 depletion and conditioning with fludarabine, cyclophosphamide and antithymocyte globulin for acquired severe aplastic anaemia. Br J Haematol. 2012 Apr;157(1):139-42. doi: 10.1111/j.1365-2141.2011.08924.x. Epub 2011 Nov 5. No abstract available.

  PMID: 22055111 BACKGROUND

• Lang P, Handgretinger R. Haploidentical SCT in children: an update and future perspectives. Bone Marrow Transplant. 2008 Oct;42 Suppl 2:S54-9. doi: 10.1038/bmt.2008.285.

  PMID: 18978746 BACKGROUND

• Im HJ, Koh KN, Suh JK, Lee SW, Choi ES, Jang S, Kwon SW, Park CJ, Seo JJ. Refinement of treatment strategies in ex vivo T-cell-depleted haploidentical SCT for pediatric patients. Bone Marrow Transplant. 2015 Feb;50(2):225-31. doi: 10.1038/bmt.2014.232. Epub 2014 Oct 13.

  PMID: 25310303 DERIVED

MeSH Terms

Conditions

Myelodysplastic Syndromes

Interventions

Antilymphocyte Serum; Filgrastim; Whole-Body Irradiation; fludarabine; Cyclophosphamide; Tacrolimus; Mycophenolic Acid; Rituximab

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Immune Sera; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins; Biological Products; Complex Mixtures; Granulocyte Colony-Stimulating Factor; Colony-Stimulating Factors; Glycoproteins; Glycoconjugates; Carbohydrates; Hematopoietic Cell Growth Factors; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Biological Factors; Radiotherapy; Therapeutics; Investigative Techniques; Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Organic Chemicals; Phosphoramides; Organophosphorus Compounds; Macrolides; Lactones; Caproates; Acids, Acyclic; Carboxylic Acids; Fatty Acids; Lipids; Antibodies, Monoclonal, Murine-Derived; Antibodies, Monoclonal

Study Officials

• Ho Joon Im, MD & PhD

  Asan Medical Center

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Ho Joon Im, MD & PhD

CONTACT

82-2-3010-3371; hojim@amc.seoul.kr

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal investigator

Study Record Dates

• First Submitted: January 5, 2012
• First Posted: January 13, 2012
• Study Start: January 1, 2012
• Primary Completion: December 1, 2013
• Study Completion: March 1, 2014
• Last Updated: January 13, 2012

Record last verified: 2012-01

Locations

KR (1)