NCT02014506

Brief 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.

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
30

participants targeted

Target at P25-P50 for phase_1

Timeline
Completed

Started Jan 2017

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

December 4, 2013

Completed
14 days until next milestone

First Posted

Study publicly available on registry

December 18, 2013

Completed
3 years until next milestone

Study Start

First participant enrolled

January 1, 2017

Completed
1.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2018

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2018

Completed
Last Updated

March 28, 2017

Status Verified

March 1, 2017

Enrollment Period

1.9 years

First QC Date

December 4, 2013

Last Update Submit

March 24, 2017

Conditions

Haploidentical Hematopoietic Stem Cell TransplantationMalignant DiseaseNon-malignant Disease

Keywords

Children and adolescentsMalignant diseaseNon-malignant diseaseTCRαβ depletionHaploidentical hematopoietic stem cell transplantation

Outcome Measures

Primary Outcomes (1)

  • To evaluate tralsplant-related mortality after haploidentical hematopoietic stem cell transplantation using TCRαβ-depleted graft

    1 year posttransplant

Secondary Outcomes (10)

  • To assess engraftment and graft failure

    28 days posttransplant

  • To estimate the risk of acute GVHD

    100 days posttransplant

  • To estimate the incidence of relapse

    100 days and 1 year post-transplant

  • To estimate the incidence and severity of chronic GVHD

    1 year posttransplant

  • To estimate the overall survival

    1 year posttransplant

  • +5 more secondary outcomes

Study Arms (1)

HAPLO

EXPERIMENTAL
Drug: FludarabineDrug: CyclophosphamideBiological: anti-thymocyte globulinBiological: filgrastimRadiation: Total body irradiationProcedure: TCRαβ-depleted hematopoietic cell transplantationDevice: CliniMACS

Interventions

FludarabineDRUG

40mg/M2 once daily IV on days -7 to -2

HAPLO
CyclophosphamideDRUG

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

HAPLO
anti-thymocyte globulinBIOLOGICAL
HAPLO
filgrastimBIOLOGICAL

Beginning on day 4 and continuing until blood counts recover

HAPLO
Total body irradiationRADIATION

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)

HAPLO
TCRαβ-depleted hematopoietic cell transplantationPROCEDURE
HAPLO
CliniMACSDEVICE

Immunogenetic depletion of TCRαβ cells

HAPLO

Eligibility Criteria

AgeUp to 21 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • 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
  • +6 more criteria

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Asan Medical Center

Seoul, 138-736, South Korea

RECRUITING

Related Publications (3)

  • 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.

    PMID: 24141651BACKGROUND

  • 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.

    PMID: 23380343BACKGROUND

  • 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.

    PMID: 23993299BACKGROUND

Related Links

MeSH Terms

Interventions

fludarabineCyclophosphamideAntilymphocyte SerumFilgrastimWhole-Body Irradiation

Intervention Hierarchy (Ancestors)

Phosphoramide MustardsNitrogen Mustard CompoundsMustard CompoundsHydrocarbons, HalogenatedHydrocarbonsOrganic ChemicalsPhosphoramidesOrganophosphorus CompoundsImmune SeraAntibodiesImmunoglobulinsImmunoproteinsBlood ProteinsProteinsAmino Acids, Peptides, and ProteinsSerum GlobulinsGlobulinsBiological ProductsComplex MixturesGranulocyte Colony-Stimulating FactorColony-Stimulating FactorsGlycoproteinsGlycoconjugatesCarbohydratesHematopoietic Cell Growth FactorsCytokinesIntercellular Signaling Peptides and ProteinsPeptidesBiological FactorsRadiotherapyTherapeuticsInvestigative Techniques

Study Officials

  • Ho Joon Im, MD, PhD

    Asan Medical Center

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Ho Joon Im, MD, PhD

CONTACT

82-2-3010-3371hojim@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
Professor

Study Record Dates

First Submitted

December 4, 2013

First Posted

December 18, 2013

Study Start

January 1, 2017

Primary Completion

December 1, 2018

Study Completion

December 1, 2018

Last Updated

March 28, 2017

Record last verified: 2017-03

Locations

KR(1)