NCT02310997

Brief Summary

This trial is looking at using umbilical cord blood from unrelated donors after high dose chemotherapy. It is for people who have cancer of the bone marrow or lymphatic system including leukaemia and lymphoma, or a blood disorder called myelodysplastic syndrome (MDS). The trial is for babies over 4 weeks old, children, and adults up to the age of 45.

Trial Health

57
Monitor

Trial Health Score

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

Enrollment
11

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Jul 2011

Typical duration for phase_2

Geographic Reach
1 country

12 active sites

Status
terminated

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

July 1, 2011

Completed
3.2 years until next milestone

First Submitted

Initial submission to the registry

September 18, 2014

Completed
3 months until next milestone

First Posted

Study publicly available on registry

December 8, 2014

Completed
5 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 1, 2015

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

May 1, 2015

Completed
Last Updated

May 27, 2015

Status Verified

May 1, 2015

Enrollment Period

3.8 years

First QC Date

September 18, 2014

Last Update Submit

May 26, 2015

Conditions

Outcome Measures

Primary Outcomes (1)

  • Non-relapse mortality at 100 days post transplant

    100 days post transplant

Secondary Outcomes (5)

  • Overall survival at 1 year

    1 year post transplant

  • Time course of mixed chimerism established by the donor graft

    28, 60, 100 days, 6 months, 1 year post transplant

  • Recovery from neutropenia and cytopenia

    up to 1 year post transplant

  • Incidence of acute and chronic GVHD

    100 Days and 1 year post transplant

  • Incidence of relapse

    1 year post transplant

Study Arms (2)

Fludarabine + Cyclophosphamide + TBI

EXPERIMENTAL

Patients aged 2-45 years (excluding AML, JMML and MDS patients aged \<16 years) will receive an umbilical cord blood transplant using a myeloablative conditioning regimen comprising: Fludarabine 25mg/m\^2/day day -8 to -6 (total 75mg/m\^2) Cyclophosphamide 60mg/kg day -7 and -6 (total 120mg/kg) Total body irradiation 13 - 14.4Gy in 6-8 fractions

Other: Umbilical Cord Blood TransplantDrug: CyclophosphamideRadiation: Total body irradiationDrug: Fludarabine

Busulfan + Cyclophosphamide + Melphalan

EXPERIMENTAL

Patients aged \< 2 years and AML, JMML and MDS patients \<16 years will receive an umbilical cord blood transplant using a myeloablative conditioning regimen comprising: Busulfan 3.2mg/kg/day in 2 or 4 doses per day, days -9 to -6 (total 12.8mg/kg). Cyclophosphamide 60mg/kg days -4 and -3 (total 120mg/kg) Melphalan 140mg/m\^2 day -2

Other: Umbilical Cord Blood TransplantDrug: CyclophosphamideDrug: BusulfanDrug: Melphalan

Interventions

Busulfan + Cyclophosphamide + MelphalanFludarabine + Cyclophosphamide + TBI
Busulfan + Cyclophosphamide + MelphalanFludarabine + Cyclophosphamide + TBI
Fludarabine + Cyclophosphamide + TBI
Busulfan + Cyclophosphamide + Melphalan
Busulfan + Cyclophosphamide + Melphalan
Fludarabine + Cyclophosphamide + TBI

Eligibility Criteria

Age28 Days - 45 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Subjects must be \< 45 years of age and ≥ 28 days old
  • Patients with high risk, advanced or poorly responding haematological disease for which there is published evidence that haematopoietic stem cell transplantation using myeloablative conditioning is likely to be effective
  • The individual patient's disease status is such that there is no alternative therapy likely to achieve cure or provide a significant prolongation of disease-free survival
  • Left ventricular ejection fraction \>45%
  • Transaminases and bilirubin \< twice the upper limit of the normal range
  • Creatinine clearance \> 60mls/min
  • Comorbidity index of 0 or 1

You may not qualify if:

  • Patients with a suitably matched sibling donor or 10/10 unrelated volunteer donor available within an acceptable time period.
  • Pregnancy or breastfeeding.
  • Evidence of HIV or HTLV (I+II) infection or known HIV or HTLV positive serology
  • Current active serious infection, in particular uncontrolled fungal infection -Previous irradiation that precludes the safe administration of an additional dose of 13- 14.4 Gy of total body irradiation (TBI) in patients aged 2-45 years
  • Prior autograft
  • CML in first chronic phase responding to Imatinib or refractory blast crisis
  • Patients with acute leukaemia in morphological relapse/ persistent disease (defined as \> 5% blasts in normocellular bone marrow)
  • Patients with acute myeloid leukaemia with relapse/persistent disease unresponsive to re-induction chemotherapy (defined as \>20% blasts in normocellular bone marrow)
  • Malignant disease that is refractory to or progressive on salvage therapy
  • Myelofibrosis.
  • Aplastic anaemia

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (12)

University College London Hospital

London, Greater London, W1T 4TJ, United Kingdom

Location

University Hospital Birmingham

Birmingham, B15 2TH, United Kingdom

Location

Bristol Haematology & Oncology Centre

Bristol, BS2 8ED, United Kingdom

Location

Addenbrooke's Hospital

Cambridge, CB2 0QQ, United Kingdom

Location

St James' University Hospital

Leeds, LS9 7TF, United Kingdom

Location

St Bartholomew's Hospital

London, EC1A 7BE, United Kingdom

Location

Great Ormond Street Hospital for Children

London, WC1N 3JH, United Kingdom

Location

Manchester Royal Infirmary

Manchester, M13 9WL, United Kingdom

Location

Christie Hospital NHS Foundation Trust

Manchester, M20 4BX, United Kingdom

Location

Nottingham City Hospital

Nottingham, NG5 1PB, United Kingdom

Location

Royal Hallamshire Hospital

Sheffield, S10 2JF, United Kingdom

Location

Royal Marsden Hospital

Sutton, SM2 5PT, United Kingdom

Location

MeSH Terms

Conditions

LeukemiaLymphoma

Interventions

CyclophosphamideWhole-Body IrradiationBusulfanMelphalanfludarabine

Condition Hierarchy (Ancestors)

Neoplasms by Histologic TypeNeoplasmsHematologic DiseasesHemic and Lymphatic DiseasesLymphoproliferative DisordersLymphatic DiseasesImmunoproliferative DisordersImmune System Diseases

Intervention Hierarchy (Ancestors)

Phosphoramide MustardsNitrogen Mustard CompoundsMustard CompoundsHydrocarbons, HalogenatedHydrocarbonsOrganic ChemicalsPhosphoramidesOrganophosphorus CompoundsRadiotherapyTherapeuticsInvestigative TechniquesButylene GlycolsGlycolsAlcoholsMesylatesAlkanesulfonatesAlkanesulfonic AcidsAlkanesHydrocarbons, AcyclicSulfonic AcidsSulfur AcidsSulfur CompoundsPhenylalanineAmino Acids, AromaticAmino Acids, CyclicAmino AcidsAmino Acids, Peptides, and Proteins

Study Officials

  • Rachael Hough

    University College London Hospital

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

September 18, 2014

First Posted

December 8, 2014

Study Start

July 1, 2011

Primary Completion

May 1, 2015

Study Completion

May 1, 2015

Last Updated

May 27, 2015

Record last verified: 2015-05

Locations