Trial Review

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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT00429598




Registration number
NCT00429598
Ethics application status
Date submitted
14/12/2006
Date registered
18/12/2006
Date last updated
28/10/2021

Titles & IDs
Public title
Single vs Double Umbilical Cord Blood Transplants in Children With High Risk Leukemia and Myelodysplasia (BMT CTN 0501)
Scientific title
Multi-center, Open Label, Randomized Trial Comparing Single Versus Double Umbilical Cord Blood (UCB) Transplantation in Pediatric Patients With High Risk Leukemia and Myelodysplasia (BMT CTN #0501)
Secondary ID [1] 0 0
2U01HL069294
Secondary ID [2] 0 0
BMTCTN0501
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Colitis, Ulcerative 0 0
Acute Myelogenous Leukemia 0 0
Acute Lymphocytic Leukemia 0 0
Chronic Myelogenous Leukemia 0 0
Condition category
Condition code

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Comparator / control treatment
Control group

Outcomes

Eligibility
Key inclusion criteria
* Two partially HLA-matched UCB units. Units must be HLA-matched minimally at 4 of 6 HLA-A and B (at intermediate resolution by molecular typing) and DRB1 (at high resolution by molecular typing) loci with the patient, and the units must be HLA-matched at 3 of 6 HLA- A, B, DRB1 loci with each other (using same resolution of molecular typing as indicated above). Two appropriately HLA-matched units must be available such that one unit delivers a pre-cryopreserved nucleated cell dose of at least 2.5 x 10^7 per kilogram and the second unit at least 1.5 x 10^7 per kilogram.
* Acute myelogenous leukemia (AML) at the following stages:

1. High risk first complete remission (CR1), defined as the following:

* Having preceding myelodysplasia (MDS)
* High risk cytogenetics (high risk cytogenetics: del (5q) -5, -7, abn (3q), t (6;9) complex karyotype [at least 5 abnormalities],)the presence of a high FLT3 ITD-AR (> 0.4)
* Requiring more than 1 cycle of chemotherapy to obtain complete remission (CR);
* FAB M6
2. Second or greater CR
3. First relapse with less than 25% blasts in bone marrow
4. Morphologic complete remission with incomplete blood count recovery
* Therapy-related AML for which prior malignancy has been in remission for at least 12 months
* Acute lymphocytic leukemia (ALL) at the following stages:

1. High risk first remission, defined as one of the following conditions:

* Philadelphia chromosome-positive adult lymphoblastic leukemia (Ph+ ALL)
* Mixed lineage leukemia (MLL) rearrangement with slow early response (defined as having M2 [5-25% blasts] or M3 [more than 25% blasts on bone marrow examination on Day 14 of induction therapy])
* Hypodiploidy (less than 44 chromosomes or DNA index less than 0.81)
* End of induction M3 bone marrow
* End of induction M2 with M2-3 at Day 42
* Evidence of minimal residual disease (MRD). If a patient's only high risk criterion is MRD, approval by a protocol chair or protocol officer is required for enrollment. For COG centers, this will only be for MRD greater than 1 percent by flow MRD at the end of extended induction.
2. High risk second remission, defined as one of the following conditions:

* Philadelphia chromosome-positive adult lymphoblastic leukemia (Ph+ ALL)
* Bone marrow relapse less than 36 months from induction
* T-lineage relapse at any time
* Very early isolated central nervous system (CNS) relapse (6 months from diagnosis)
* Slow reinduction (M2-3 at Day 28) after relapse at any time
* Evidence of minimal residual disease (MRD). If a patient's only high risk criterion is MRD, approval by a protocol chair or protocol officer is required for enrollment. For COG centers, this will only be for MRD greater than 1 percent by flow MRD at the end of extended induction.
3. Any third or subsequent CR
* NK cell lymphoblastic leukemia in any CR
* Biphenotypic or undifferentiated leukemia in any CR or if in first relapse must have less than 25% blasts in bone marrow (BM)
* Myelodysplastic syndrome (MDS) at any stage
* Chronic myelogenous leukemia (CML) in chronic or accelerated phase
* All patients with evidence of CNS leukemia must be treated and be in CNS CR to be eligible for study.
* Patients 16 years old or older must have a Karnofsky score of at least 70% and patients younger than 16 years old must have a Lansky score of at least 70%.
* Patients with adequate physical function as measured by:

1. Cardiac: Left ventricular ejection fraction greater than 40% or shortening fraction greater than 26%
2. Hepatic: Bilirubin no more than 2.5 mg/dL; alanine aminotransferase (ALT), aspartate aminotransferase (AST), and alkaline phosphatase (ALP) no more than 5 times the upper limit of normal (ULN)
3. Renal: Serum creatinine within normal range for age, or if serum creatinine is outside normal range for age, then renal function (creatinine clearance or GFR) greater than 70 mL/min/1.73 m^2
4. Pulmonary: Diffusing capacity of the lung for carbon monoxide (DLCO), forced expiratory volume in one second (FEV1), or forced vital capacity (FVC) greater than 50% of predicted value (corrected for hemoglobin); if unable to perform pulmonary function tests, then O2 saturation greater than 92% of room air
Minimum age
1 Year
Maximum age
21 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Pregnant (ß-positive human chorionic gonadotropin [HCG]) or breastfeeding
* Evidence of HIV infection or HIV positive serology
* Current uncontrolled bacterial, viral, or fungal infection (currently taking medication and progression of clinical symptoms)
* Autologous transplant less than 12 months prior to enrollment
* Prior autologous transplant for the disease for which the UCB transplant will be performed
* Prior allogeneic hematopoietic stem cell transplant
* Active malignancy other than the one for which the UCB transplant is being performed within 12 months of enrollment
* Inability to receive TBI
* Requirement of supplemental oxygen
* HLA-matched related donor able to donate

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/12/2006
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/10/2014
Sample size
Target
Accrual to date
Final
224
Recruitment in Australia
Recruitment state(s)

Funding & Sponsors
Primary sponsor type
Other
Name
Medical College of Wisconsin
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Mary Horowitz, MD, MS
Address 0 0
Center for International Blood and Marrow Transplant Research
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results are available at https://clinicaltrials.gov/study/NCT00429598