NCT01328496

Brief Summary

In this study, participants with high-risk hematologic malignancies undergoing hematopoietic cell transplantation (HCT), who do not have a suitable human leukocyte antigen (HLA)-matched related/sibling donor (MSD), matched unrelated donor (MURD) or killer-immunoglobulin receptors (KIR) ligand mismatched haploidentical donor identified, will receive an umbilical cord blood transplantation (UCBT) using a myeloablative preparative regimen. The preparative regimen includes fludarabine (75 mg/m2), fractionated total body irradiation (TBI) (10.0 Gy), and cyclophosphamide (120mg/kg) with mesna. Fludarabine will be given once a day at 25 mg/m2 for three days on day -10 to day -8, TBI will be given twice a day at 150 cGy for four days on day -7 to day -4, and cyclophosphamide will be given once a day for at 60mg/kg for two days on day -3 and day -2. Post-transplantation immunosuppression with cyclosporine and MMF will begin on day -3. Cord Blood infusion will occur on day 0 and G-CSF will start on day +1.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
14

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Jun 2011

Longer than P75 for phase_2

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

First Submitted

Initial submission to the registry

March 31, 2011

Completed
4 days until next milestone

First Posted

Study publicly available on registry

April 4, 2011

Completed
2 months until next milestone

Study Start

First participant enrolled

June 15, 2011

Completed
5.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 31, 2016

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

October 31, 2016

Completed
6 months until next milestone

Results Posted

Study results publicly available

May 5, 2017

Completed
Last Updated

June 5, 2017

Status Verified

March 1, 2017

Enrollment Period

5.4 years

First QC Date

March 31, 2011

Results QC Date

March 23, 2017

Last Update Submit

May 4, 2017

Conditions

Hematologic MalignanciesDisorder Related to TransplantationHematopoietic Malignancy

Keywords

Hematologic MalignanciesUmbilical Cord Blood transplantationHematopoietic Cell Transplantation

Outcome Measures

Primary Outcomes (1)

  • Event Free Survival (EFS) for Research Participants

    Estimate EFS for research participants at one-year post transplant by using single unit umbilical cord blood. The event is defined as relapse, graft failure, death due to any cause. The number of participants who did not experience any of those events (relapse, graft failure, death due to any cause) at year 1 post-transplant was given.

    1 year post-transplant

Secondary Outcomes (9)

  • Number of Observational Arm Participants Engrafted

    1 year

  • Number of Observational Arm Patients Who Relapsed

    1 year

  • Number of Deaths of Observational Arm Patients

    1 year

  • Number of Observational Arm Patients With Transplant-related Mortality (TRM)

    First 100 days

  • Number of Participants With Acute GVHD

    1 year

  • +4 more secondary outcomes

Study Arms (2)

Research Arm

OTHER

Participant with high-risk hematologic malignancies undergoing Hematopoietic Cell Transplantation, who do not have a suitable Human Leukocyte Antigen -matched related/sibling donor, Matched Unrelated Donor or Killer immunoglobulin receptors ligand mismatched haploidentical donor identified, will receive a single UCB unit. Intervention: Preparative Regimen

Drug: Preparative Regimen

Observation Arm

OTHER

Patients requiring two UCB units will be eligible for UCBT01 on the observational arm. Intervention: Preparative Regimen

Drug: Preparative Regimen

Interventions

Preparative RegimenDRUG

Fludarabine (75 mg/m2), fractionated total body irradiation (TBI) (12.0 Gy), and cyclophosphamide (120mg/kg) with mesna. Fludarabine will be given once a day at 25 mg/m2 for three days on day -10 to day -8, TBI will be given twice a day at 150 cGy for four days on day -7 to day -4, and cyclophosphamide will be given once a day for at 60mg/kg for two days on day -3 and day -2. Post-transplantation immunosuppression with cyclosporine and MMF will begin on day -3. Cord Blood infusion will occur on day 0 and G-CSF will start on day +1.

Observation ArmResearch Arm

Eligibility Criteria

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

You may qualify if:

  • Age less than or equal to 21 years old.
  • Has a partially HLA-matched single or double UCB product
  • High-risk hematologic malignancy.
  • High risk ALL in CR1, ALL in High risk CR2, ALL in CR3 or subsequent.
  • AML in high risk CR1, AML in CR2 or subsequent
  • AML in first relapse with \< 25% blasts in BM
  • Therapy related AML, with prior malignancy in CR \> 12mo
  • MDS, primary or secondary
  • NK cell, biphenotypic, or undifferentiated leukemia in CR1 or subsequent.
  • CML in accelerated phase, or in chronic phase with persistent molecular positivity or intolerance to tyrosine kinase inhibitor.
  • Hodgkin lymphoma in CR2 or subsequent after failure of prior autologous HCT, or unable to mobilize stem cells for autologous HCT.
  • Non-Hodgkin lymphoma in CR2 or subsequent after failure of prior autologous HCT, or unable to mobilize stem cells for autologous HCT.
  • JMML
  • All patients with evidence of CNS leukemia must be treated and be in CNS CR to be eligible for study.
  • Patient must fulfill pre-transplant evaluation:
  • +7 more criteria

You may not qualify if:

  • Patient has a suitable MSD, volunteer MURD, or KIR mismatched haploidentical donor available in the necessary time for stem cell donation.
  • Patient has any other active malignancy other than the one for which HCT is indicated.
  • Patient had a prior allogeneic HCT
  • Patient had an autologous HCT within the previous 12 months.
  • Patient is pregnant as confirmed by positive serum or urine pregnancy test within 14 days prior to enrollment.
  • Patient is lactating
  • Patient has Down Syndrome
  • Patient has a current uncontrolled bacterial, fungal, or viral infection per the judgment of the PI.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

St. Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States

Location

Related Links

MeSH Terms

Conditions

Hematologic Neoplasms

Condition Hierarchy (Ancestors)

Neoplasms by SiteNeoplasmsHematologic DiseasesHemic and Lymphatic Diseases

Results Point of Contact

Title
Amr Qudeimat, MD
Organization
St. Jude Children's Research Hospital
amr.qudeimat@stjude.org
901-595-8342

Study Officials

  • Amr Qudeimat, MD

    St. Jude Children's Research Hospital

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
Yes

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

March 31, 2011

First Posted

April 4, 2011

Study Start

June 15, 2011

Primary Completion

October 31, 2016

Study Completion

October 31, 2016

Last Updated

June 5, 2017

Results First Posted

May 5, 2017

Record last verified: 2017-03

Locations

US(1)