LeukemiaNCT02007863
Umbilical Cord Blood (UCB) Transplantation in Pediatric Patients With High Risk Leukemia and Myelodysplasia
A study testing Umbilical Cord Blood Transfusion, Fludarabine and Busulfan in people with Leukemia and Myelodysplasia, run by University of Miami at 2 sites in United States.
Trial summary
Unrelated Cord Blood (UCB) transplant in children is a viable stem cell transplant modality for patients with leukemia and myelodysplasia. UCB is now considered "Standard Of Care" in cases where a suitable living bone marrow donor is not available. The survival of UCB is similar to Matched Unrelated Marrow Transplant. Read more about this study
- Who is it for?
- Ages Up to 21 years
- Conditions the study is for
- Leukemia, Myelodysplasia, Acute Lymphocytic Leukemia
- What is being tested?
- Umbilical Cord Blood Transfusion, Fludarabine and Busulfan
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 2 participants
- Where
- 2 sitesUnited States
- Study dates
- Aug 2008to Jun 2014
- Phase
- Interventional
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients must be up to 21 years of age
- Patients cannot receive total body irradiation (TBI) because of:
- Young age - < 2 years at diagnosis of leukemia resulting in an age < 4 years at transplantation (due to risk of severe growth retardation and brain damage).
- Inability to tolerate TBI because of prior radiation or organ toxicity.
- Refractory/multiply relapsed leukemia, for which a traditional TBI/cyclophosphamide regimen would unlikely lead to a successful outcome.
- Patients must have a partially human leukocyte antigen (HLA)-matched UCB unit. Unit 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. The unit must deliver a pre-cryopreserved nucleated cell dose of at least 2.5 x 10^7 per kilogram.
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- Acute myelogenous leukemia (AML) at the following stages:
- High risk first complete remission (CR1), defined as:
- Having preceding myelodysplasia (MDS)
- High risk cytogenetics (High-risk cytogenetics: del (5q) -5, -7, abn (3q), t (6;9) complex karyotype (>= 5 abnormalities)
- Requiring > 2 cycles chemotherapy to obtain CR;
- Second or greater CR.
- First relapse with < 25% blasts in bone marrow.
- Patients with therapy-related AML whose prior malignancy has been in remission for at least 12 months.
- Acute lymphocytic leukemia (ALL) at the following stages:
- High risk first remission, defined as:
- Ph+ ALL; or,
- Myeloid/lymphoid leukemia (MLL) rearrangement with slow early response [defined as having M2 (5-25% blasts) or M3 (>25% blasts on bone marrow examination on Day 14 of induction therapy)]; or,
- Hypodiploidy (< 44 chromosomes or DNA index < 0.81); or,
- End of induction M3 bone marrow; or,
- End of induction M2 with M2-3 at Day 42.
- High risk second remission, defined as:
- Bone marrow relapse < 36 months from induction; or,
- T-lineage relapse at any time; or,
- Very early isolated central nervous system (CNS) relapse (6 months from diagnosis); or,
- Slow reinduction (M2-3 at Day 28) after relapse at any time.
- Any third or subsequent CR.
- Biphenotypic or undifferentiated leukemia in any CR or if in 1st relapse must have < 25% blasts in bone marrow (BM).
- 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 must have a Karnofsky score ≥ 70% and patients < 16 years old must have a Lansky score ≥ 70%.
- Signed informed consent.
- Patients with adequate physical function as measured by:
- Cardiac: Left ventricular ejection fraction at rest must be > 40%, or shortening fraction > 26%
- Hepatic: Bilirubin ≤ 2.5 mg/dL; and alanine transaminase (ALT), aspartate transaminase (AST) and Alkaline Phosphatase ≤ 5 x upper limit of normal (ULN)
- Renal: Serum creatinine within normal range for age, or if serum creatinine outside normal range for age, then renal function (creatinine clearance or GFR) > 70 mL/min/1.73 m2.
- Pulmonary: Diffusing capacity of the lungs for carbon monoxide (DLCO), Forced expiratory volume in 1 second (FEV1), Forced vital capacity (FVC) (diffusion capacity) > 50% of predicted (corrected for hemoglobin); if unable to perform pulmonary function tests, then O2 saturation > 92% of room air.
Who cannot join
- Pregnant (B-positive 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 < 6 months prior to enrollment.
- Prior autologous transplant for the disease for which the UCB transplant will be performed.
- Allogeneic hematopoietic stem cell transplant < 6 months prior to enrollment.
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- Active malignancy other than the one for which the UCB transplant is being performed within 12 months of enrollment
- Active CNS leukemia.
- Requirement of supplemental oxygen.
- HLA-matched related donor able to donate.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Jackson Memorial Hospital
Miami, Florida, 33136
University of Miami Sylvester Comprehensive Cancer Center
Miami, Florida, 33136
About this study
Official title: Treatment Protocol: Umbilical Cord Blood (UCB) Transplantation in Pediatric Patients With High Risk Leukemia and Myelodysplasia Using Conditioning Regimen Without Radiation
Unrelated Cord Blood (UCB) transplant in children is a viable stem cell transplant modality for patients with leukemia and myelodysplasia. UCB is now considered "Standard Of Care" in cases where a suitable living bone marrow donor is not available. The survival of UCB is similar to Matched Unrelated Marrow Transplant. This study is considered "Research" since UCB is not a licensed product and requires investigational new drug (IND). THERE ARE NO SPECIFIC RESEARCH QUESTIONS IN THIS PROTOCOL. This protocol merely provides UCB as a stem cell treatment modality to pediatric patients who may require it after a conditioning regimen that excludes Total Body Irradiation.
Read the detailed description
The preparative regimen will consist of:
- Fludarabine: 25 mg/m2/day IV x 5 doses on Days -13, to -9
- Busulfan 1mg/kg IV every 6 hrs x 16 doses on Days -8 to -5
- Melphalan 45 mg/m2/day IV x 3 doses on days -4 to -2
- ATGAM 30mg/kg/day x 3 doses on Days -3 to -1
- Day 0 will be the day of the UCB Transplant
- The graft-versus-host-disease (GVHD) prophylaxis will be Cyclosporin A to maintain level 200-400 beginning on Day -3, through 200-400. Solumedrol at 1mg/kg/day on Day 1 until D+4, then solumedrol 2mg/kg/day until Day +19 or till absolute neutrophil count (ANC) reaches 500/mm2, then taper by 0.2 mg/kg/week.
- Being tested
- Umbilical Cord Blood Transfusion, Fludarabine, Busulfan, Melphalan
- Keywords
- Leukemia, Myelodysplasia, Acute lymphocytic leukemia, ALL, Acute myelogenous leukemia, AML, Pediatric
- Medical subject headings
- Leukemia, Anemia, Refractory, with Excess of Blasts, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Umbilical Cord Blood TransfusionProcedure or surgery
Also called UCB
Following the administration of the preparative therapy, all subjects will undergo UCB transplantation. Umbilical Cord Blood Transfusion will occur on Day 0
FludarabineDrug
Also called Fludara
Fludarabine 25 mg/m2/day will be administered over 30-60 minutes intravenous infusion on Days -13 through -9 for a total of 5 doses. Fludarabine will not be dose adjusted for body weight.
BusulfanDrug
Also called Busulfex
Busulfan IV (Busulfex) will be administered IV every 6 hours on days -8 through -5 for a total of 16 doses. Seizure prophylaxis prior to first dose of busulfan till Day -3 will be administered.
MelphalanDrug
Also called Alkeran
Melphalan 45 mg/m2/day will be administered over 60 minutes intravenous infusion on Days -4 through -2 for a total of 3 doses.
| Study group | Role | What they receive |
|---|---|---|
| Umbilical Cord Blood + Chemotherapy | Experimental | Umbilical Cord Blood transfusion + Chemotherapy (Fludarabine + Busulfan + Melphalan) Receives: Procedure: Umbilical Cord Blood Transfusion; Drug: Fludarabine; Drug: Busulfan; Drug: Melphalan |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Successful Unrelated Cord Blood (UCB) Transplants
The number of patients who received successful UCB transplants as evidenced by absolute neutrophil recovery.
Measured: 2 Years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Dec 2013 (estimated)
- Study startAug 2008
- Primary completionJun 2014
- Study completionJun 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for LeukemiaStudies of the same condition, open and under way.
- More trials run by University of MiamiThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Miami
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Martin Andreansky, University of Miami
- Investigators
- Martin Andreasky, MD, PhD, Principal investigator, University of Miami
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02007863
- Sponsor's protocol ID
- 20080774
- Status last verified
- Nov 2015
- First submitted
- 3 Dec 2013
- Last updated
- 28 Dec 2015
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 28 Dec 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Treatment Protocol: Umbilical Cord Blood (UCB) Transplantation in Pediatric Patients With High Risk Leukemia and Myelodysplasia Using Conditioning Regimen Without Radiation
- ClinicalTrials.gov ID
- NCT02007863
- Acronym / sponsor ID
- 20080774
- Keywords
- Leukemia, Myelodysplasia, Acute lymphocytic leukemia, ALL, Acute myelogenous leukemia, AML, Pediatric
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 2 participants (actual)
- Lead sponsor
- University of MiamiUniversity, hospital or other
- Study start
- Aug 2008
- Primary completion
- Jun 2014 (actual)
- Study completion
- Jun 2014 (actual)
- First posted
- 11 Dec 2013 (estimated)
- Results
- Posted 28 Dec 2015
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Unrelated Cord Blood (UCB) transplant in children is a viable stem cell transplant modality for patients with leukemia and myelodysplasia. UCB is now considered "Standard Of Care" in cases where a suitable living bone marrow donor is not available. The survival of UCB is similar to Matched Unrelated Marrow Transplant. This study is considered "Research" since UCB is not a licensed product and requires investigational new drug (IND). THERE ARE NO SPECIFIC RESEARCH QUESTIONS IN THIS PROTOCOL. This protocol merely provides UCB as a stem cell treatment modality to pediatric patients who may require it after a conditioning regimen that excludes Total Body Irradiation.
Detailed description
The preparative regimen will consist of:
- Fludarabine: 25 mg/m2/day IV x 5 doses on Days -13, to -9
- Busulfan 1mg/kg IV every 6 hrs x 16 doses on Days -8 to -5
- Melphalan 45 mg/m2/day IV x 3 doses on days -4 to -2
- ATGAM 30mg/kg/day x 3 doses on Days -3 to -1
- Day 0 will be the day of the UCB Transplant
- The graft-versus-host-disease (GVHD) prophylaxis will be Cyclosporin A to maintain level 200-400 beginning on Day -3, through 200-400. Solumedrol at 1mg/kg/day on Day 1 until D+4, then solumedrol 2mg/kg/day until Day +19 or till absolute neutrophil count (ANC) reaches 500/mm2, then taper by 0.2 mg/kg/week.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Umbilical Cord Blood + Chemotherapy | Experimental | Procedure: Umbilical Cord Blood Transfusion, Drug: Fludarabine, Drug: Busulfan, Drug: MelphalanUmbilical Cord Blood transfusion + Chemotherapy (Fludarabine + Busulfan + Melphalan) |
Who may qualify
- Ages
- Up to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (38 listed)
- Patients must be up to 21 years of age
- Patients cannot receive total body irradiation (TBI) because of:
- Patients must have a partially human leukocyte antigen (HLA)-matched UCB unit. Unit 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. The unit must deliver a pre-cryopreserved nucleated cell dose of at least 2.5 x 10^7 per kilogram.
- Acute myelogenous leukemia (AML) at the following stages:
- Patients with therapy-related AML whose prior malignancy has been in remission for at least 12 months.
Key exclusion criteria (10 listed)
- Pregnant (B-positive 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 < 6 months prior to enrollment.
- Prior autologous transplant for the disease for which the UCB transplant will be performed.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Umbilical Cord Blood TransfusionAlso: UCBFollowing the administration of the preparative therapy, all subjects will undergo UCB transplantation. Umbilical Cord Blood Transfusion will occur on Day 0 | Procedure or surgery | Umbilical Cord Blood + Chemotherapy |
| FludarabineAlso: FludaraFludarabine 25 mg/m2/day will be administered over 30-60 minutes intravenous infusion on Days -13 through -9 for a total of 5 doses. Fludarabine will not be dose adjusted for body weight. | Drug | Umbilical Cord Blood + Chemotherapy |
| BusulfanAlso: BusulfexBusulfan IV (Busulfex) will be administered IV every 6 hours on days -8 through -5 for a total of 16 doses. Seizure prophylaxis prior to first dose of busulfan till Day -3 will be administered. | Drug | Umbilical Cord Blood + Chemotherapy |
| MelphalanAlso: AlkeranMelphalan 45 mg/m2/day will be administered over 60 minutes intravenous infusion on Days -4 through -2 for a total of 3 doses. | Drug | Umbilical Cord Blood + Chemotherapy |
Primary outcome
Number of Successful Unrelated Cord Blood (UCB) Transplants
Time frame: 2 Years
The number of patients who received successful UCB transplants as evidenced by absolute neutrophil recovery.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Dec 2015.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Martin Andreasky, MD, PhD principal investigator
University of Miami
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Umbilical Cord Blood Transfusion, Fludarabine and Busulfan in people with Leukemia and Myelodysplasia, run by University of Miami at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 38 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 28 Dec 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Yes. Results are posted in the registry (first posted 28 Dec 2015). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Successful Unrelated Cord Blood (UCB) Transplants (time frame: 2 Years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Dec 2015.