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

• Sponsor: Asan Medical Center (Other)
• Overall Status: Unknown status
• CT.gov ID: NCT01509300
• Collaborator: (none)
• Enrollment: 10
• Location: 1
• Arm: 1
• Duration (Months): 26
• Patients Per Site Per Month: 0.4

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Acute Leukemia; Myelodysplastic Syndrome; Solid Tumors	Biological: anti-thymocyte globulin; Biological: filgrastim; Radiation: Total body irradiation; Drug: Fludarabine; Drug: cyclophosphamide; Drug: Tacrolimus; Drug: Mycophenolate mofetil; Drug: Rituximab	Phase 1/Phase 2

Study Design

• Study Type: Interventional
• Anticipated Enrollment: 10 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: HLA-haploidentical Allogeneic Hematopoietic Cell Transplantation Using CD3 Depletion for Children and Adolescents With Acute Leukemia, Myelodysplastic Syndrome and Solid Tumors After Conditioning of TBI, Fludarabine, Cyclophosphamide and Antithymocyte Globulin
• Study Start Date: Jan 1, 2012
• Anticipated Primary Completion Date: Dec 1, 2013
• Anticipated Study Completion Date: Mar 1, 2014

Arms and Interventions

Arm

Intervention/Treatment

Experimental: HAPLO

Biological: anti-thymocyte globulin; On days -10 to -9; Biological: filgrastim; Beginning on day 4 and continuing until blood counts recover; Radiation: Total body irradiation; 2Gy D-6 to D-4; Drug: Fludarabine; 30mg/M2 once daily IV on days -8 to -4; Drug: cyclophosphamide; 60 mg/kg IV on day-3 and -2; Drug: Tacrolimus; begin on 0; Drug: Mycophenolate mofetil; begin on 0; Drug: Rituximab; 375mg/m2 on day +21

Outcome Measures

Primary Outcome Measures

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 Outcome Measures

1. Engraftment and graft failure rates [28 days engraftment and graft failure]

   Number of patients who failed to stable engraftment by 28 days

2. Incidence of acute GVHD [100 days post-transplant]

   Number of patients with acute GVHD.

3. Treatment related mortality [100 days post-transplant]

   Number of death after transplantation

4. Relapse rate and overall survival [2 year after transplantation]

Eligibility Criteria

Criteria

• Ages Eligible for Study: N/A to 21 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria

1. 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)

1. No HLA-identical family member or closely matched (8 or 7 of 8 HLA-locus match) unrelated marrow donor available

2. HLA-haploidentical related donor available

Exclusion criteria

1. Active fungal infections

2. HIV positive

3. Pregnant or nursing

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Asan Medical Center	Seoul		Korea, Republic of	138-736

Sponsors and Collaborators

• Asan Medical Center

Investigators

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

Study Documents (Full-Text)

None provided.

More Information

Publications

• 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.
• 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. Review.

• Responsible Party: Ho Joon Im, Principal investigator, Asan Medical Center
• ClinicalTrials.gov Identifier: NCT01509300
• Other Study ID Numbers: AMCPHO-SCT0902
• First Posted: Jan 13, 2012
• Last Update Posted: Jan 13, 2012
• Last Verified: Jan 1, 2012

• Keywords provided by Ho Joon Im, Principal investigator, Asan Medical Center: TBI; Fludarabine; CD3 depletion; Children and adolescents; Haploidentical hematopoietic stem cell transplantation
• Additional relevant MeSH terms: Leukemia; Preleukemia; Myelodysplastic Syndromes; Syndrome; Acute Disease; Mycophenolic Acid; Cyclophosphamide; Rituximab; Fludarabine; Tacrolimus; Antilymphocyte Serum

Study Results

No Results Posted as of Jan 13, 2012