NCT02566395

Brief Summary

This clinical pilot trial is intended to evaluate the feasibility, efficacy and safety of hematopoietic stem cell transplantation (HSCT) from Human Leukocyte Antigen (HLA)-mismatched related donors for children and young adults with hematologic malignancies who lack a suitably matched related or unrelated donor. The methodology will be one that has been successfully utilized in adult patients at Thomas Jefferson University.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
10

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started Dec 2014

Longer than P75 for phase_3

Geographic Reach
1 country

1 active site

Status
completed

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

Study Start

First participant enrolled

December 1, 2014

Completed
10 months until next milestone

First Submitted

Initial submission to the registry

September 30, 2015

Completed
2 days until next milestone

First Posted

Study publicly available on registry

October 2, 2015

Completed
6.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 14, 2022

Completed
1 month until next milestone

Study Completion

Last participant's last visit for all outcomes

February 14, 2022

Completed
Last Updated

April 7, 2022

Status Verified

April 1, 2022

Enrollment Period

7.1 years

First QC Date

September 30, 2015

Last Update Submit

April 6, 2022

Conditions

Keywords

Stem Cell TransplantationHaploidentical Donors

Outcome Measures

Primary Outcomes (1)

  • Hematopoietic engraftment

    Absolute neutrophil count \>500/microliter x 3 consecutive days

    Day +30 post-transplantation

Secondary Outcomes (4)

  • 2-Year disease-free survival

    2 years post-transplantation

  • Grade II-IV GvHD

    Day +100 post-transplantatation

  • Grade III-IV GvHD

    Day +100 post-transplantation

  • Relapse rate

    2 years post-transplantation

Study Arms (1)

Haploidentical Stem Cell Transplantation

EXPERIMENTAL

Subjects will receive pretransplantation conditioning of total-body irradiation (1,200 cGy delivered in 8 fractions over 4 days \[Days -9 through -6\] and cyclophosphamide (60 mg/kg IV daily x 2 on Days -3 and -2). Donor lymphocyte infusion will occur on day -6; donor CD34+ cells will be infused on Day 0.

Radiation: RadiationDrug: CyclophosphamideBiological: Donor Lymphocyte Infusion (DLI)Biological: Haploidentical Stem Cell Transplantation

Interventions

RadiationRADIATION

1,200 cGy, delivered in 8 fractions of 150 cGy bid x 4 days

Also known as: Total Body Irradiation
Haploidentical Stem Cell Transplantation

Cyclophosphamide 60 mg/kg IV daily x 2 consecutive days

Also known as: Cytoxan
Haploidentical Stem Cell Transplantation

DLI containing 1 x 10E8/kg donor T-cells

Haploidentical Stem Cell Transplantation

2-10 x 10E6/kg donor CD34+ selected cells

Haploidentical Stem Cell Transplantation

Eligibility Criteria

Age2 Years - 21 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Acute lymphoblastic leukemia
  • Acue myelogenous leukemia
  • Myelodysplastic syndrome
  • Non-Hodgkin lymphoma
  • Chronic myelogenous leukemia
  • Adequate lung, liver, renal, cardiac function
  • Performance status \>70
  • Available related donor who is mismatched at ≥ 2 HLA alleles

You may not qualify if:

  • Available HLA-identical related donor
  • HIV positive
  • Active uncontrolled infection
  • Pregnancy
  • Performance status ≤70

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Cohen Children's Medical Center

New Hyde Park, New York, 11040, United States

Location

Related Publications (1)

  • Grosso D, Gaballa S, Alpdogan O, Carabasi M, Filicko-O'Hara J, Kasner M, Martinez-Outschoorn U, Wagner JL, O'Hara W, Rudolph S, Chervoneva I, Colombe B, Farley PC, Flomenberg P, Pro B, Sharma M, Shi W, Weiss M, Flomenberg N. A two-step approach to myeloablative haploidentical transplantation: low nonrelapse mortality and high survival confirmed in patients with earlier stage disease. Biol Blood Marrow Transplant. 2015 Apr;21(4):646-52. doi: 10.1016/j.bbmt.2014.12.019. Epub 2014 Dec 23.

    PMID: 25542159BACKGROUND

MeSH Terms

Conditions

Precursor Cell Lymphoblastic Leukemia-LymphomaLeukemia, Myeloid, AcuteMyelodysplastic SyndromesLeukemia, Myelogenous, Chronic, BCR-ABL PositiveLymphoma, Non-HodgkinHodgkin Disease

Interventions

RadiationWhole-Body IrradiationCyclophosphamide

Condition Hierarchy (Ancestors)

Leukemia, LymphoidLeukemiaNeoplasms by Histologic TypeNeoplasmsHematologic DiseasesHemic and Lymphatic DiseasesLymphoproliferative DisordersLymphatic DiseasesImmunoproliferative DisordersImmune System DiseasesLeukemia, MyeloidBone Marrow DiseasesMyeloproliferative DisordersChronic DiseaseDisease AttributesPathologic ProcessesPathological Conditions, Signs and SymptomsLymphoma

Intervention Hierarchy (Ancestors)

Physical PhenomenaRadiotherapyTherapeuticsInvestigative TechniquesPhosphoramide MustardsNitrogen Mustard CompoundsMustard CompoundsHydrocarbons, HalogenatedHydrocarbonsOrganic ChemicalsPhosphoramidesOrganophosphorus Compounds

Study Officials

  • Joel A Brochstein, MD

    Cohen Children's Medical Center

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Associate Chief for Cellular Therapy, CCMC

Study Record Dates

First Submitted

September 30, 2015

First Posted

October 2, 2015

Study Start

December 1, 2014

Primary Completion

January 14, 2022

Study Completion

February 14, 2022

Last Updated

April 7, 2022

Record last verified: 2022-04

Locations