NCT01624701

Brief Summary

This is a pilot clinical trial to assess the feasibility and efficacy of expanding umbilical cord blood derived blood stem cells for transplantation using a combination of chemical factors and stromal co-culture. Bone marrow (BM) mesenchymal stromal cells (MSC) will be obtained from a separate bone marrow donor. One cord blood unit will be expanded by this method while another cord blood unit will be infused without manipulation. The expanded cord blood unit will help boost the initial recovery of blood counts after transplantation, though it is expected that the unexpanded cord blood unit will provide the cells which will lead to long term engraftment of blood stem cells. A third cord blood unit will be identified for standby should the cord blood unit expansion fail.

Trial Health

57
Monitor

Trial Health Score

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

Enrollment
3

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Mar 2012

Longer than P75 for phase_1

Geographic Reach
1 country

1 active site

Status
terminated

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

Trial Relationships

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

March 1, 2012

Completed
19 days until next milestone

First Submitted

Initial submission to the registry

March 20, 2012

Completed
3 months until next milestone

First Posted

Study publicly available on registry

June 21, 2012

Completed
3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 1, 2015

Completed
1 year until next milestone

Study Completion

Last participant's last visit for all outcomes

July 1, 2016

Completed
Last Updated

November 30, 2015

Status Verified

November 1, 2015

Enrollment Period

3.3 years

First QC Date

March 20, 2012

Last Update Submit

November 26, 2015

Conditions

Acute LeukemiaChronic LeukemiaMyelodysplastic SyndromeLymphomaMyeloma

Keywords

Cord bloodunrelated donorallogeneiccell dose

Outcome Measures

Primary Outcomes (1)

  • Safety

    Number of subjects with infusional toxicities (including fever, renal dysfunction within 72 hours of infusion) and potential immunologic competition (absent chimerism of donor cells by 21 days post transplantation).

    1 month

Secondary Outcomes (1)

  • Engraftment

    2 months

Study Arms (1)

Expanded

EXPERIMENTAL

'Ex vivo expanded cord blood cells

Other: Ex vivo expanded cord blood cells

Interventions

Ex vivo expanded cord blood cellsOTHER

Cord blood cells will be expanded ex vivo using a combination of stem cell factor (SCF), thrombopoietin (TPO), Flt3 ligand and IGFBP2 with mesenchymal stromal cell (MSC) co-culture.

Expanded

Eligibility Criteria

Age12 Years - 60 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Patients aged 12 years to 60 years.
  • Patient has no currently available HLA-A, B, C, DRB1 and DQB1 matched related or unrelated donor.
  • Patient must have a hematologic malignancy requiring allogeneic haematopoietic stem cell transplantation as further defined below (from National Marrow Donor Program and American Society of Blood and Marrow Transplantation Guidelines) and as further agreed upon by a panel of at least three hematologists specializing in transplantation.
  • Acute myelogenous leukemia (AML): High-risk AML including:
  • Antecedent hematological disease (e.g., myelodysplasia (MDS))
  • Treatment-related leukemia
  • Induction failure
  • st complete remission (CR1) with poor-risk cytogenetics or molecular markers (e.g. Flt 3 mutation, 11q23 etc)
  • nd complete remission (CR2) and beyond
  • Acute lymphoblastic leukemia (ALL)
  • CR1 up to age 35
  • High-risk over age 35 including:
  • Poor-risk cytogenetics (e.g., Philadelphia chromosome t(9;22)or 11q23 rearrangements)
  • High white cell count (\> 30,000/mm3) at diagnosis
  • CNS or testicular leukemia
  • +24 more criteria

You may not qualify if:

  • Inadequate Organ Function as defined by:
  • Renal: Creatinine clearance \> 60ml/min
  • Hepatic: Bilirubin, AST/ALT \< 2x upper limit of normal
  • Pulmonary function: DLCOcorr \> 50% normal
  • Cardiac: left ventricular ejection fraction \> 45%
  • Karnofsky score (adults) \< 70% or Lansky score \< 50% (pediatrics)

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Singapore General Hospital

Singapore, 169608, Singapore

Location

MeSH Terms

Conditions

Myelodysplastic SyndromesLymphomaNeoplasms, Plasma Cell

Condition Hierarchy (Ancestors)

Bone Marrow DiseasesHematologic DiseasesHemic and Lymphatic DiseasesNeoplasms by Histologic TypeNeoplasmsLymphoproliferative DisordersLymphatic DiseasesImmunoproliferative DisordersImmune System Diseases

Study Officials

  • William YK Hwang, MBBS, FRCP

    Singapore General Hospital

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 20, 2012

First Posted

June 21, 2012

Study Start

March 1, 2012

Primary Completion

July 1, 2015

Study Completion

July 1, 2016

Last Updated

November 30, 2015

Record last verified: 2015-11

Locations

SG(1)