LeukemiaNCT00423826
Umbilical Cord Blood Stem Cell Transplant in Treating Patients With Hematologic Cancer or Other Disease
A study testing Busulfan, Cytarabine and Fludarabine phosphate in people with Leukemia and Lymphoma, run by Barbara Ann Karmanos Cancer Institute at 1 site in United States.
Trial summary
RATIONALE: Giving low doses of chemotherapy and total-body irradiation before a donor umbilical cord blood stem cell transplant helps stop the growth of cancer or abnormal cells. It also stops the patient's immune system from rejecting the donor's stem cells. Read more about this study
- Who is it for?
- Ages Up to 69 years
- Conditions the study is for
- Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- What is being tested?
- Busulfan, Cytarabine and Fludarabine phosphate
- Type of treatment
- Pill or oral medicine, Injection or infusion, Surgery or procedure
- How many people
- Not given
- Where
- 1 siteUnited States
- Study dates
- Jan 2007to Jan 2015
- Phase
- Expanded access
- Are there results?
- None posted in the registry
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 69 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
DISEASE CHARACTERISTICS:
- Histologically confirmed diagnosis of 1 of the following:
- Acute myeloid leukemia meeting the following criteria:
- M0-M7 histologic subtypes by French-American-British classification
- Previously treated disease
- Meets 1 of the following criteria:
- Persistent disease as evidenced by 5-30% persistent blasts in bone marrow after induction or salvage therapy
- In second or subsequent complete remission (CR)
- In first CR with 1 of the following high-risk features:
- Philadelphia chromosome present
- Noncore-binding factor type of chromosomal abnormalities
- Myelodysplastic syndromes with 1 of the following International Prognostic Scoring System (IPSS) scores:
- Intermediate-1
- Intermediate-2
- High-risk score with transfusion dependence
- Chronic myelogenous leukemia meeting 1 of the following criteria:
- In accelerated or blastic phase
- Failed prior imatinib mesylate therapy
- Acute lymphoblastic leukemia meeting 1 of the following criteria:
- In first CR with any of the following high-risk features:
- Philadelphia chromosome present
- Translocation t(4;11) present
- WBC > 30,000/mm³ (adult patients)
- More than 4 weeks from initiation of treatment was required to achieve CR (adult patients)
- DNA index of near haploid (N=23 chromosomes) (pediatric patients)
- In second or subsequent CR
- Persistent disease as evidenced by 5-20% persistent blasts in bone marrow after induction or salvage therapy
- Hodgkin's or non-Hodgkin's lymphoma meeting the following criteria:
- Recurrent or refractory disease
- Tumor ≤ 5 cm in diameter
- Myeloma or plasma cell neoplasm meeting 1 of the following staging criteria:
- Stage III at presentation
- Stage I-II at presentation
- Not responding OR progressed after first-line therapy
- Chronic lymphocytic leukemia or Waldenstrom's macroglobulinemia with refractory or progressive disease after first-line therapy
- No 5-6/6 HLA-matched related or 7-8/8 HLA-matched unrelated marrow or peripheral blood stem cell donor available
- No single 4-6/6 HLA-A, -B, or -DRB1-matched umbilical cord blood unit ≥ 3.5 x 10^7 nucleated cells/kg available
PATIENT CHARACTERISTICS:
- ECOG performance status (PS) 0-2 OR Karnofsky or Lansky PS 70-100%
- Not pregnant
- Fertile patients must use effective contraception prior to and during study participation
- HIV negative
- Bilirubin < 3.0 mg/dL
- AST and ALT ≤ 3 times upper limit of normal
- Creatinine < 2.0 mg/dL OR creatinine clearance > 50 mL/min
- Cardiac ejection fraction > 50% by echocardiogram OR shortening fraction > 27%
- No uncontrolled symptomatic congestive heart failure
- No unstable angina pectoris
- No cardiac arrhythmia
- FEV_1 > 50% of normal
- Forced vital capacity > 50% of normal
- DLCO normal
- Oxygen saturation > 92% on room air (for patients < 5 years of age)
- No history of allergic reactions attributed to compounds of similar chemical or biological composition to busulfan and fludarabine phosphate
- No ongoing or active infection
- No psychiatric illness or social situation that would preclude study compliance
- No other uncontrolled illness
PRIOR CONCURRENT THERAPY:
- See Disease Characteristics
- At least 4 weeks since prior and no concurrent surgery
- At least 4 weeks since prior and no other concurrent investigational or commercial agents or therapies for the malignancy, including chemotherapy, biologic therapy, or radiotherapy
Trial locations and contacts
1 site in 1 country.
United States1 site
Barbara Ann Karmanos Cancer Institute
Detroit, Michigan, 48201-1379
About this study
Official title: A Pilot Study of Double Cord Blood Stem Cell Transplantation in Patients With Hematologic Malignancies
RATIONALE: Giving low doses of chemotherapy and total-body irradiation before a donor umbilical cord blood stem cell transplant helps stop the growth of cancer or abnormal cells. It also stops the patient's immune system from rejecting the donor's stem cells. The donated stem cells may replace the patient's immune cells and help destroy any remaining cancer or abnormal cells (graft-versus-tumor effect). Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. Giving tacrolimus and mycophenolate mofetil before the transplant may stop this from happening.
PURPOSE: This clinical trial is studying how well umbilical cord blood stem cell transplant works in treating patients with hematologic cancer or other disease.
Read the detailed description
OBJECTIVES:
Primary
- Determine the efficacy of double umbilical cord blood stem cell transplantation using a conditioning regimen comprising lower doses of busulfan and fludarabine phosphate and low-dose total body irradiation, in terms of stem cell engraftment at 60 days post transplantation, in patients with hematologic cancer or other diseases.
- Determine the merits of conducting a larger, comparative study of this regimen.
Secondary
- Determine mortality within 100 days of transplantation in these patients.
OUTLINE: This is a pilot study.
- Reduced-intensity conditioning regimen: Patients receive busulfan IV over 3 hours on days -9 to -8 and fludarabine phosphate IV on days -7 to -3. Patients then undergo low-dose total body irradiation on day 0.
- Graft-versus-host disease prophylaxis: Patients receive tacrolimus IV twice daily and mycophenolate orally or IV three times daily beginning on day -3.
- CNS prophylaxis and/or treatment: Patients with a history of CNS involvement receive prophylactic cytarabine (Ara-C) intrathecally (IT) prior to transplant. Patients also undergo lumbar puncture (LP) to test for active CNS disease. Patients with cerebrospinal fluid positive for leukemia receive Ara-C IT every 2-3 days until a repeat LP shows no remaining leukemic cells. Three days after the last LP and after one final dose of Ara-C, patients begin the conditioning regimen.
- Double umbilical cord blood (UCB) donor stem cell transplantation (SCT): Patients undergo double UCB donor SCT on day 0.
PROJECTED ACCRUAL: A total of 30 patients will be accrued for this study.
- Conditions
- Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Precancerous Condition, Secondary Myelofibrosis
- Being tested
- Busulfan, Cytarabine, Fludarabine phosphate, mycophenolate mofetil, tacrolimus
- Keywords
- accelerated phase chronic myelogenous leukemia, blastic phase chronic myelogenous leukemia, childhood chronic myelogenous leukemia, relapsing chronic myelogenous leukemia, adult acute lymphoblastic leukemia in remission, recurrent adult acute lymphoblastic leukemia, childhood acute lymphoblastic leukemia in remission, recurrent childhood acute lymphoblastic leukemia, adult acute myeloid leukemia in remission, adult acute myeloid leukemia with 11q23 (MLL) abnormalities, adult acute myeloid leukemia with inv(16)(p13;q22), adult acute myeloid leukemia with t(15;17)(q22;q12), adult acute myeloid leukemia with t(16;16)(p13;q22), adult acute myeloid leukemia with t(8;21)(q22;q22), recurrent adult acute myeloid leukemia, adult acute minimally differentiated myeloid leukemia (M0), adult acute myeloblastic leukemia without maturation (M1), adult acute myeloblastic leukemia with maturation (M2), adult acute promyelocytic leukemia (M3), adult acute myelomonocytic leukemia (M4)
- Medical subject headings
- Leukemia, Lymphoma, Multiple Myeloma, Neoplasms, Plasma Cell, Myelodysplastic Syndromes, Precancerous Conditions, Leukemia, Myeloid, Accelerated Phase, Blast Crisis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BusulfanDrug
Also called Busulfex®, Myleran®
3 mg/kg intravenously over 3 hours
CytarabineDrug
Also called DepoCyt(TM), Liposomal Ara-C
Patients with previous history of CNS involvement will receive pre-transplant intrathecal Cytarabine (Ara-C) (30 mg/M2) therapy.
Also called Fludara
25 mg/M2/day IV
Also called Cellcept
Orally at the dose of 1 gm every 8 hours.
tacrolimusDrug
Also called Advagraf, Prograf, Protopic
0.015 mg/kg IV every 12 hours by continuous infusion.
allogeneic hematopoietic stem cell transplantationProcedure or surgery
10 days post drug intervention
umbilical cord blood transplantationProcedure or surgery
10 days post drug intervention
total-body irradiationRadiation
10 days post drug intervention
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Jan 2007 (estimated)
- Study startJan 2007
- Primary completionJan 2015
- Study completionJan 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for LeukemiaStudies of the same condition, open and under way.
- 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
- Expanded access
Sponsor and investigators
- Lead sponsor
- Barbara Ann Karmanos Cancer Institute
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Responsible party
- Principal investigator, Voravit Ratanatharathorn, Barbara Ann Karmanos Cancer Institute
- Investigators
- Voravit Ratanatharathorn, MD, Study chair, Barbara Ann Karmanos Cancer Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00423826
- Sponsor's protocol ID
- CDR0000518230
- Other IDs
- P30CA022453, WSU-2006-059, WSU-112506MP2F
- Status last verified
- Feb 2016
- First submitted
- 16 Jan 2007
- Last updated
- 29 Feb 2016
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Feb 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Study of Double Cord Blood Stem Cell Transplantation in Patients With Hematologic Malignancies
- ClinicalTrials.gov ID
- NCT00423826
- Acronym / sponsor ID
- CDR0000518230
- Conditions
- Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Precancerous Condition, Secondary Myelofibrosis
- Keywords
- accelerated phase chronic myelogenous leukemia, blastic phase chronic myelogenous leukemia, childhood chronic myelogenous leukemia, relapsing chronic myelogenous leukemia, adult acute lymphoblastic leukemia in remission, recurrent adult acute lymphoblastic leukemia, childhood acute lymphoblastic leukemia in remission, recurrent childhood acute lymphoblastic leukemia
- Study type
- Expanded access
- Recruitment status
- No Longer Available
- Lead sponsor
- Barbara Ann Karmanos Cancer InstituteUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Study start
- Jan 2007
- Primary completion
- Jan 2015 (actual)
- Study completion
- Jan 2015 (actual)
- First posted
- 18 Jan 2007 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
RATIONALE: Giving low doses of chemotherapy and total-body irradiation before a donor umbilical cord blood stem cell transplant helps stop the growth of cancer or abnormal cells. It also stops the patient's immune system from rejecting the donor's stem cells. The donated stem cells may replace the patient's immune cells and help destroy any remaining cancer or abnormal cells (graft-versus-tumor effect). Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. Giving tacrolimus and mycophenolate mofetil before the transplant may stop this from happening.
PURPOSE: This clinical trial is studying how well umbilical cord blood stem cell transplant works in treating patients with hematologic cancer or other disease.
Detailed description
OBJECTIVES:
Primary
- Determine the efficacy of double umbilical cord blood stem cell transplantation using a conditioning regimen comprising lower doses of busulfan and fludarabine phosphate and low-dose total body irradiation, in terms of stem cell engraftment at 60 days post transplantation, in patients with hematologic cancer or other diseases.
- Determine the merits of conducting a larger, comparative study of this regimen.
Secondary
- Determine mortality within 100 days of transplantation in these patients.
OUTLINE: This is a pilot study.
- Reduced-intensity conditioning regimen: Patients receive busulfan IV over 3 hours on days -9 to -8 and fludarabine phosphate IV on days -7 to -3. Patients then undergo low-dose total body irradiation on day 0.
- Graft-versus-host disease prophylaxis: Patients receive tacrolimus IV twice daily and mycophenolate orally or IV three times daily beginning on day -3.
- CNS prophylaxis and/or treatment: Patients with a history of CNS involvement receive prophylactic cytarabine (Ara-C) intrathecally (IT) prior to transplant. Patients also undergo lumbar puncture (LP) to test for active CNS disease. Patients with cerebrospinal fluid positive for leukemia receive Ara-C IT every 2-3 days until a repeat LP shows no remaining leukemic cells. Three days after the last LP and after one final dose of Ara-C, patients begin the conditioning regimen.
- Double umbilical cord blood (UCB) donor stem cell transplantation (SCT): Patients undergo double UCB donor SCT on day 0.
PROJECTED ACCRUAL: A total of 30 patients will be accrued for this study.
Study design
Who may qualify
- Ages
- Up to 69 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
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 |
|---|---|---|
| BusulfanAlso: Busulfex®, Myleran®3 mg/kg intravenously over 3 hours | Drug | – |
| CytarabineAlso: DepoCyt(TM), Liposomal Ara-CPatients with previous history of CNS involvement will receive pre-transplant intrathecal Cytarabine (Ara-C) (30 mg/M2) therapy. | Drug | – |
| Fludarabine phosphateAlso: Fludara25 mg/M2/day IV | Drug | – |
| mycophenolate mofetilAlso: CellceptOrally at the dose of 1 gm every 8 hours. | Drug | – |
| tacrolimusAlso: Advagraf, Prograf, Protopic0.015 mg/kg IV every 12 hours by continuous infusion. | Drug | – |
| allogeneic hematopoietic stem cell transplantation10 days post drug intervention | Procedure or surgery | – |
| umbilical cord blood transplantation10 days post drug intervention | Procedure or surgery | – |
| total-body irradiation10 days post drug intervention | Radiation | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Feb 2016.
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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.
Voravit Ratanatharathorn, MD study chair
Barbara Ann Karmanos Cancer Institute
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Busulfan, Cytarabine and Fludarabine phosphate in people with Leukemia and Lymphoma, run by Barbara Ann Karmanos Cancer Institute at 1 site 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?
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: status not confirmed, as of the sponsor's last update on 29 Feb 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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?
Results are not available in the source record.
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.
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 29 Feb 2016.