NCT00067002

Brief Summary

The goal of this clinical research study is to learn if combining cord blood units to make the cells "take" faster in recipients will help to improve the results of cord blood transplants.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
110

participants targeted

Target at P50-P75 for phase_2

Timeline
Completed

Started Apr 2003

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

Study Start

First participant enrolled

April 1, 2003

Completed
4 months until next milestone

First Submitted

Initial submission to the registry

August 8, 2003

Completed
3 days until next milestone

First Posted

Study publicly available on registry

August 11, 2003

Completed
12.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 1, 2016

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

July 1, 2016

Completed
2.8 years until next milestone

Results Posted

Study results publicly available

May 1, 2019

Completed
Last Updated

May 1, 2019

Status Verified

April 1, 2019

Enrollment Period

13.3 years

First QC Date

August 8, 2003

Results QC Date

July 31, 2017

Last Update Submit

April 10, 2019

Conditions

Keywords

ALLLeukemia, Lymphocytic, AcuteAMLLeukemia, Myelocytic, AcuteCMLLeukemia, Myeloid, ChronicNHLLymphoma, Non-Hodgkindouble cord blood transplantexpanded cord blood transplantRituxanRituximabMelphalanAlkeranThiotepaFludarabineFludarabine phosphateFludaraCyclophosphamideCytoxanNeosarMesnaMesnex

Outcome Measures

Primary Outcomes (2)

  • Time To Neutrophil Engraftment

    Engraftment is defined as a sustained ANC \> 0.5 x 10\^9/L for at least 3 consecutive days. Engraftment date is the first of the 3 days with sustained absolute neutrophil count (ANC) \>/= 0.5 x 10\^9/L.

    First 100 days, evaluation and blood tests twice weekly

  • Number of Participants With Engraftment

    Engraftment defined as a sustained absolute neutrophil count (ANC) \> 0.5 x 10\^9/L for at least 3 consecutive days. Engraftment Failure defined as ANC \<500/ul by day +42 and participant has no evidence of donor chimerism on bone marrow examination.

    First 100 days, evaluation and blood tests twice weekly

Secondary Outcomes (3)

  • Rate of Acute Graft Versus Host Disease (GVHD)

    Review over first 100 days

  • Rate of Chronic GVHD

    Up to one year

  • Number of Participants Severity of Acute GVHD by Treatment Arm

    Following first 100 days, up to one year

Study Arms (2)

Double Cord Blood Transplant Group

EXPERIMENTAL

Two Unmanipulated Cord Blood units. Rituxan 375 mg/m2 by vein for patients with CD20 + malignancies. Melphalan 140 mg/m2 by vein on Day -8. Thiotepa 5 mg/Kg by vein on Day -7. Fludarabine 40 mg/m2 by vein on Days -6 to -3.

Procedure: Expanded allogeneic cord blood (CB)Drug: RituxanDrug: MelphalanDrug: ThiotepaDrug: Fludarabine

One Expanded Cord Blood Transplant Group

EXPERIMENTAL

One Unmanipulated and One Expanded Cord Blood Unit. Fludarabine 40 mg/m2 by vein on Days -6 to -3. Cyclophosphamide 50 mg/kg by vein on Day -6. Mesna 10 mg/kg by vein before the 1st dose of Cyclophosphamide, then 10 mg/kg every 4 hours for four more doses (total of 50 mg/Kg). Total body irradiation (TBI) given on Day -1 at 2 Gy.

Procedure: One Unmanipulated and One Expanded Cord Blood UnitDrug: RituxanDrug: FludarabineDrug: CyclophosphamideDrug: MesnaRadiation: Total body irradiation (TBI)

Interventions

Transplantation of Two Unmanipulated Cord Blood Units.

Also known as: double Cord blood transplant.
Double Cord Blood Transplant Group

Transplantation of One Unmanipulated and One Expanded Cord Blood Unit.

Also known as: Expanded Cord Blood Transplant
One Expanded Cord Blood Transplant Group

375 mg/m2 by vein on Day - 9 for patients with CD20 + malignancies.

Also known as: Rituximab
Double Cord Blood Transplant GroupOne Expanded Cord Blood Transplant Group

140 mg/m2 by vein on Day -8.

Also known as: Alkeran
Double Cord Blood Transplant Group

5 mg/Kg by vein on Day -7.

Double Cord Blood Transplant Group

40 mg/m2 by vein on Days -6 to -3.

Also known as: Fludarabine Phosphate, Fludara
Double Cord Blood Transplant GroupOne Expanded Cord Blood Transplant Group

50 mg/kg by vein on Day -6.

Also known as: Cytoxan, Neosar
One Expanded Cord Blood Transplant Group
MesnaDRUG

10 mg/kg by vein before the 1st dose of Cyclophosphamide, then 10 mg/kg every 4 hours for four more doses (total of 50 mg/Kg).

Also known as: Mesnex
One Expanded Cord Blood Transplant Group

Total body irradiation (TBI) given on Day -1 at 2 Gy.

Also known as: TBI, XRT
One Expanded Cord Blood Transplant Group

Eligibility Criteria

Age1 Month - 80 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Disease-Specific Eligibility Requirements: Patients must have one of the following hematologic malignancies: 1. Acute Myelogenous Leukemia (AML), Myelodysplastic Syndrome (MDS) 2. Acute Lymphoblastic Leukemia (ALL) 3. Chronic Myelogenous Leukemia (CML) 4. Non-Hodgkin's Lymphoma (NHL) 5. Hodgkin's Disease (HD) 6. Chronic Lymphocytic Leukemia (CLL) 7. Chronic eosinophilic leukemia or Philadelphia chromosome negative CML.
  • Greater than 1 month old and \<=60 years old for full myeloablative therapy.
  • Patients must have two CB units available which are matched with the patient at 4, 5, or 6/6 HLA class I (serological) and II (molecular) antigens. Each cord must contain at least 1E7 total nucleated cells/Kg recipient body weight in the pre-thawed fraction.
  • Patient must be willing to undergo bone marrow harvest or peripheral blood progenitor cell (PBPC) collection for use in case of engraftment failure. If the patient is unable or fails to successfully undergo the collection, a family member must be identified to donate hematopoietic stem cells for haploidentical transplant in case of engraftment failure. If autologous hematopoietic stem cells cannot be procured due to marrow contamination by malignancy, or due to harvest failure, and a haploidentical relative is not available or not willing to donate, two cord blood units can be used as the back-up graft.
  • Continuation to Criteria # 4: These units will be identified prior to enrollment in this study.
  • Regimen 1 (Myeloablative mel/thiotepa/fludarabine): 1.Patients with ALL, HD, NHL, AML, MDS, CML, CLL and Chronic eosinophilic leukemia who are candidates for full myeloablative therapy. 2.Performance score of at least 60% by Karnofsky (age \>= 12 years), or Lansky Play-Performance Scale (age \<12 years). 3.Age \>=1 month \<=60 years (high-dose).
  • Continuation to Criteria # 6: 4.Adequate major organ system function as demonstrated by:a. Left ventricular ejection function of at least 40%. b.Pulmonary function test demonstrating a diffusion capacity of at least 50%. predicted (high-dose). c.Creatinine \< 1.6 mg/dL. d.serum glutamate pyruvate transaminase (SGPT)/bilirubin \<= to 2.0 x normal (high-dose).
  • Eligibility for Regimen 2 (Non-myeloablative Cy-Flu-TBI): 1. Patients with ALL, AML, MDS, CML, NHL, CLL, Chronic eosinophilic leukemia and HD who are not candidates for full myeloablative therapy. All patients who received a prior autologous transplant are eligible. 2. Performance score of at least 60% by Karnofsky or PS \< 3 (ECOG) (age \>= 12 years), or Lansky Play-Performance Scale (age \<12 years) 3. Age \>= 1 month \<=80 years
  • Continuation to Criteria # 8: 4. Left ventricular ejection function of at least 30%; 5. Pulmonary function test demonstrating a diffusion capacity of at least 40% predicted; 6. Creatinine \< 3.0 mg/dL; 7. SGPT \<= to 4.0 x normal.
  • Regimen 3 (Myeloablative VP16-TBI): 1. Patients with ALL who are candidates for myeloablative therapy, and require a TBI-containing regimen. 2. Performance score of at least 60% by Karnofsky or PS \< 2 (ECOG) (age \>= 12 years), or Lansky Play-Performance Scale (age \<12 years). 3. Age \>= 1 month \<=50 years. 4. Organ function requirements: a. Left ventricular ejection function of at least 50%. b. Pulmonary function test demonstrating a diffusion capacity of at least 50% predicted. c. Creatinine \< 1.6 mg/dL. d. SGPT \<= 2.0 x normal.

You may not qualify if:

  • HIV positive.
  • Pregnancy.
  • Serious medical Condition.
  • Patients with signs \& symptoms leading to positive lumbar puncture (malignant cells in the CSF) or to documented metastatic parenchymal disease are ineligible for this study.
  • Availability of appropriate, willing, HLA-matched related donor.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

University of Texas MD Anderson Cancer Center

Houston, Texas, 770030, United States

Location

Related Links

MeSH Terms

Conditions

Precursor Cell Lymphoblastic Leukemia-LymphomaLeukemia, Myeloid, AcuteLeukemia, Myelogenous, Chronic, BCR-ABL PositiveLymphoma, Non-Hodgkin

Interventions

RituximabMelphalanThiotepafludarabinefludarabine phosphateCyclophosphamideMesnaWhole-Body Irradiation

Condition Hierarchy (Ancestors)

Leukemia, LymphoidLeukemiaNeoplasms by Histologic TypeNeoplasmsHematologic DiseasesHemic and Lymphatic DiseasesLymphoproliferative DisordersLymphatic DiseasesImmunoproliferative DisordersImmune System DiseasesLeukemia, MyeloidMyeloproliferative DisordersBone Marrow DiseasesChronic DiseaseDisease AttributesPathologic ProcessesPathological Conditions, Signs and SymptomsLymphoma

Intervention Hierarchy (Ancestors)

Antibodies, Monoclonal, Murine-DerivedAntibodies, MonoclonalAntibodiesImmunoglobulinsImmunoproteinsBlood ProteinsProteinsAmino Acids, Peptides, and ProteinsSerum GlobulinsGlobulinsNitrogen Mustard CompoundsMustard CompoundsHydrocarbons, HalogenatedHydrocarbonsOrganic ChemicalsPhenylalanineAmino Acids, AromaticAmino Acids, CyclicAmino AcidsPhosphoramidesOrganophosphorus CompoundsTriethylenephosphoramideAziridinesAzirinesHeterocyclic Compounds, 1-RingHeterocyclic CompoundsPhosphoramide MustardsAlkanesulfonatesAlkanesulfonic AcidsAlkanesHydrocarbons, AcyclicSulfhydryl CompoundsSulfur CompoundsSulfonic AcidsSulfur AcidsRadiotherapyTherapeuticsInvestigative Techniques

Results Point of Contact

Title
Simrit Parmar, MD/Associate Professor, Stem Cell Transplantation
Organization
UT MD Anderson Cancer Center

Study Officials

  • Simrit Parmar, MD

    M.D. Anderson Cancer Center

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
Yes

Study Design

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

Study Record Dates

First Submitted

August 8, 2003

First Posted

August 11, 2003

Study Start

April 1, 2003

Primary Completion

July 1, 2016

Study Completion

July 1, 2016

Last Updated

May 1, 2019

Results First Posted

May 1, 2019

Record last verified: 2019-04

Locations