Status unknownNCT02014506Updated Mar 28, 2017

Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation in Children and Adolescents

A Phase 1/2 interventional study of Fludarabine and Cyclophosphamide in Haploidentical Hematopoietic Stem Cell Transplantation, Malignant Disease and Non-malignant Disease, sponsored by Asan Medical Center. Status unknown at 1 site in Korea, Republic of. Open to participants aged Up to 21 Years. Per ClinicalTrials.gov, last updated 2017-03-28.

Sponsored by Asan Medical Center · Phase 1/2, Interventional, and Treatment

The sponsor has not verified this record recently (last verified Mar 2017), so the status shown — last known as Recruiting — may be out of date.

Phase: Phase 1/2
Study type: Interventional
Enrollment: 30
Allocation: Not applicable
Ages: Up to 21 Years
Sex: All

Study summary

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.

Conditions studied

Haploidentical Hematopoietic Stem Cell Transplantation
Malignant Disease
Non-malignant Disease

Keywords

Children and adolescents
Malignant disease
Non-malignant disease
TCRαβ depletion
Haploidentical hematopoietic stem cell transplantation

In context

Lead sponsor

Asan Medical Center is the lead sponsor of 562 studies on the registry; 71 are open to participants now.

Counted across the registry records on this site, refreshed daily.

Who can participate

Ages eligible: Up to 21 Years
Sexes eligible: All
Accepts healthy volunteers: No

Inclusion criteria

A. Disease inclusions

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
Non-hematologic malignancy
- Relapsed or refractory solid tumors including neuroblastoma, rhabdomyosarcoma and so on
Non-malignant hematologic disease
- Acquired severe and very severe aplastic anemia
- Fanconi anemia
- Paroxysmal nocturnal hemoglobinuria
- Congenital dyserythropoietic anemia
- Others
Inherited or metabolic disease
- Hemophagocytic lymphohistiocytosis
- Malignant osteopetrosis
- Storage diseases
- Others B. Recipient inclusions

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

Exclusion Criteria:

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

Study design

Phase: Phase 1 / Phase 2
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrollment: 30 participants (estimated)

Study arms

Experimental
HAPLO
Drug: Fludarabine · Drug: Cyclophosphamide · Biological: anti-thymocyte globulin · Biological: filgrastim · Radiation: Total body irradiation · Procedure: TCRαβ-depleted hematopoietic cell transplantation · Device: CliniMACS

Interventions

DrugFludarabine
40mg/M2 once daily IV on days -7 to -2
DrugCyclophosphamide
50 mg/kg IV on day -3 and -2
Biologicalanti-thymocyte globulin
Biologicalfilgrastim
Beginning on day 4 and continuing until blood counts recover
RadiationTotal 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)
ProcedureTCRαβ-depleted hematopoietic cell transplantation
DeviceCliniMACS
Immunogenetic depletion of TCRαβ cells

What researchers measure

Primary outcomes

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

Secondary outcomes

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

Study locations

1 of 1 sites recruiting

Asan Medical Center
Seoul, 138-736, Korea, Republic of
- Ho Joon Im, MD & PhD · Contact · hojim@amc.seoul.kr · 82-2-3010-3371
- Ho Joon Im, MD & PhD · Principal investigator
Recruiting

References and documents

Publications

Park JA, Koh KN, Choi ES, Jang S, Kwon SW, Park CJ, Seo JJ, Im HJ. Successful rescue of early graft failure in pediatric patients using T-cell-depleted haploidentical hematopoietic SCT. Bone Marrow Transplant. 2014 Feb;49(2):270-5. doi: 10.1038/bmt.2013.163. Epub 2013 Oct 21. PubMed 24141651 ↗
Im HJ, Koh KN, Choi ES, Jang S, Kwon SW, Park CJ, Chi HS, Seo JJ. Excellent outcome of haploidentical hematopoietic stem cell transplantation in children and adolescents with acquired severe aplastic anemia. Biol Blood Marrow Transplant. 2013 May;19(5):754-9. doi: 10.1016/j.bbmt.2013.01.023. Epub 2013 Feb 1. PubMed 23380343 ↗
Schumm M, Lang P, Bethge W, Faul C, Feuchtinger T, Pfeiffer M, Vogel W, Huppert V, Handgretinger R. Depletion of T-cell receptor alpha/beta and CD19 positive cells from apheresis products with the CliniMACS device. Cytotherapy. 2013 Oct;15(10):1253-8. doi: 10.1016/j.jcyt.2013.05.014. PubMed 23993299 ↗

Updates

Tracking since Sep 25, 2026

No changes since tracking began. The registry record was last updated on Mar 28, 2017, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗

Registry details

Key details

Study ID: NCT02014506
Lead sponsor: Asan Medical Center
Responsible party: Ho Joon Im (Professor, Asan Medical Center) — Principal investigator
First posted: Dec 18, 2013
Start date: Jan 2017
Primary completion: Dec 2018 (estimated)
Completion: Dec 2018 (estimated)
Last update: Mar 28, 2017

Study contacts

Ho Joon Im, MD, PhD

Contact

hojim@amc.seoul.kr

82-2-3010-3371

Ho Joon Im, MD, PhD

principal investigator · Asan Medical Center

Oversight

Data monitoring committee: Yes

View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is status unknown, as verified in Mar 2017. You cannot join it, but the record below documents what was studied.

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