Lymphoid Hematological MalignanciesUCALLNCT01247688
Umbilical Cord Blood Transplant for Children With Lymphoid Hematological Malignancies
A study testing Cytoxan, Fludarabine and Total Body Irradiation (TBI) in people with Lymphoid Hematological Malignancies and Umbilical Cord Blood Transplant, run by Baylor College of Medicine.
Trial summary
The purpose of this study is to determine the safety and effectiveness of UCBT to treat patients with lymphoid hematological diseases and to see if this treatment can decrease the incidence of leukemia relapse, GVHD and infections. These patients have a type of blood cell disorder that is very hard to cure. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Lymphoid Hematological Malignancies, Umbilical Cord Blood Transplant
- What is being tested?
- Cytoxan, Fludarabine and Total Body Irradiation (TBI)
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- Nov 2010to Dec 2013
- Phase
- Interventional
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Potential recipients of umbilical cord blood transplantation (i.e. HLA (human leukocyte antigen) matched or mismatched / related or unrelated) with a lymphoid hematologic malignancy (acute lymphoblastic leukemia, hodgkin/non-hodgkin lymphoma) unlikely to be cure by standard chemotherapy. This includes patients who have relapsed after standard chemotherapy treatments and patients in first remission with unfavorable prognostics features.
- Related or Unrelated Umbilical Cord Blood Unit with 0-1 antigen mismatch, Total Nucleated cell dose of greater than or equal to 4 x 10^7/kg.
- Lansky/Karnofsky scores greater than or equal to 60
- Negative pregnancy test (if applicable)
- Written informed consent and/or signed assent line from patient, parent or guardian.
Who cannot join
- Patients with uncontrolled infections. For bacterial infections, patients must be receiving definitive therapy and have no signs of progressing infection for 72 hours prior to enrollment. For fungal infections patients must be receiving definitive systemic antifungal therapy and have no signs of progressing infection for 1 week prior to enrollment. Progressing infection is defined as hemodynamic instability attributable to sepsis or new symptoms, worsening physical signs or radiographic findings attributable to infection. Persisting fever without other signs or symptoms will not be interpreted as progressing infection.
- Severe renal disease (Creatinine > x 3 normal for age)
- Severe hepatic disease (direct bilirubin > 3 mg/dl or SGOT (Serum glutamic oxaloacetic transaminase) > 500)
- Patient has DLCO (Diffusing Capacity of the Lung for Carbon Monoxide) < 50% predicted or FEV1 (Forced expiratory volume ) < 50% of predicted, if applicable
- Patients with symptomatic cardiac failure unrelieved by medical therapy or evidence of significant cardiac dysfunction by echocardiogram (shortening fraction<20%).
- HIV positive
About this study
Official title: Umbilical Cord Blood Transplant for Children With Lymphoid Hematological Malignancies (UCALL)
The purpose of this study is to determine the safety and effectiveness of UCBT to treat patients with lymphoid hematological diseases and to see if this treatment can decrease the incidence of leukemia relapse, GVHD and infections.
These patients have a type of blood cell disorder that is very hard to cure. This treatment that is being used in this trial is known as a stem cell transplant. This treatment might help the patient live longer without the disease. It uses much stronger doses of drugs and radiation to kill the diseased cells that could be given without the transplant. We also think that the healthy cells from the donor may help fight any diseased cells left after the transplant.
Read more
For the transplant to take place, we will administer stem cells from a 'donor' whose cells best 'match' the patient's. In this study umbilical cords will be the source of the stem cells. Before the transplant, two very strong drugs plus total body irradiation will be given to as preconditioning. This treatment will kill most of your blood-forming cells in the bone marrow. The patient will then get then healthy stem cells.
If the patient has the disease in the central nervous system (CNS), they will receive radiation to the head and spine before starting the conditioning. This is to try to get disease control in the CNS. Radiation will not be given for children under 2 yrs old.
Currently, many umbilical cord blood units are available in public banks for transplantation in patients lacking bone marrow donors. UCB transplants (UCBT) may offer several advantages over adult bone marrow or peripheral blood stem cell transplants, including:
- rapid availability,
- absence of donor risk,
- low risk of transmissible infectious diseases,
- low risk of acute GvHD (Graft vs. Host Disease)
The three main causes of death after umbilical cord blood transplantation for these kind of disorders are graft failure, infection and disease relapse.
In this study we are trying to address these three problems:
To help improve engraftment we will add the drug Fludarabine to Cytoxan and total body irradiation. Fludarabine is a very strong medicine. We will try to decrease infections and reduce leukemia relapse by using fludarabine instead of antithymocyte globulin (ATG).
Read the detailed description
After the eligible criterion for treatment has been met and a suitable UCB stem donor has been found, the patient will have a central line placed.
Research Therapy:
After placement of the central line, the following chemotherapy will be given to after admission to the hospital and before the infusion of the umbilical cord blood stem cells:
- 9 - 6 days before the infusion: Total Body Irradiation (TBI) in two fractions ("doses") per days.
- 5 - 2 days before the infusion: Cytoxan given daily for 4 days, over 1 hour as an intravenous infusion. Mesna will be given per standards. Mesna is a drug given to decrease the side effects of Cytoxan. It will be given daily as an intravenous infusion while the patient receives the Cytoxan.
- 4 - 2 days before the infusion: Fludarabine given daily for 3 days over 1 hour as an intravenous infusion.
Stem cell transplant (intravenous infusion of the UCB stem cells) - defined as day 0 of the treatment. All other "numbered" days relate to this infusion date. For example, Day 1 is the first day after the stem cell transplant.
The following medications will be given to help decrease side effects from the chemotherapy and UCB infusion: Cyclosporine A (CSA) will be given starting 3 days prior to the stem cell infusion. It will be given daily over 2 hours every 12 hours, after the infusion and then tapered if no GVHD is present.
Administration of Mycophenolate mofetil (MMF) will start on the day the stem cell infusion is completed and will continue daily for 45 days, unless the patient develops GvHD.
Intravenous immunoglobulin's (IVIG) will be given monthly until GVHD therapy is stopped and there is evidence that the patient's body is producing antibodies.
Study Evaluations: Various study evaluations will be performed before and during the trial.
Follow Up: After year 1, the patient will be asked to return to the clinic once a year for consultations and bone marrow tests. A follow up bone marrow biopsy and aspirate will be done 1 and 2 years after transplant. Consultations with specialists will be similar to the ones the patient had before the transplant.
- Being tested
- Cytoxan, Fludarabine, Total Body Irradiation (TBI), Cord Blood Stem Cell Infusion
- Keywords
- Lymphoid, hematologic malignancy, UCB, Umbilical Cord Blood Transplant, Total Body Irradiation, Cytoxan, Cyclophosphamide, Fludarabine
- Medical subject headings
- Hematologic Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CytoxanDrug
Also called Cyclophophamide
50 mg/kg/dose will be given IV on day -5,-4, -3, -2 over 1 hour. The total dose to be given over 4 days is 200 mg/kg.
FludarabineDrug
Also called Fludara
40mg/m2/day will be given IV over 1 hour on day -4, -3, -2
Total Body Irradiation (TBI)Radiation
1.75Gy/fraction will be given on day -9, -8, -7, -6
Cord Blood Stem Cell InfusionProcedure or surgery
The cord blood stem cells will be infused on day 0
| Study group | Role | What they receive |
|---|---|---|
| Umbilical Cord Blood Transplant Treatment Plan | Experimental | Cytoxan, Fludarabine, Total Body Irradiation (TBI), Cord Blood Stem Cell Infusion Receives: Drug: Cytoxan; Drug: Fludarabine; Radiation: Total Body Irradiation (TBI); Procedure: Cord Blood Stem Cell Infusion |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Survival at 1 year after UCB transplant in pediatric patients
To determine the survival rate at 1 year after umbilical cord blood transplant in pediatric patients with lymphoid hematological malignancies
Measured: 1 year
Secondary outcomes (7)
Assessment of Platelet Count Recovery
To assess platelet recovery at day 42 post transplant
Measured: Day 42
Number of participants with severe acute GVHD grade III-IV as an assessment of safety
To estimate the risk of severe grade III-IV acute GvHD at day 100.
Measured: Day 100
Number of participants with chronic GvHD as an assessment of safety
To estimate the risk of chronic GvHD at 1 year
Measured: 1 year
Assess relapse rate after transplant
To assess relapse rate at 1 and 3 years after transplant.
Measured: 1 and 3 years
Assessment of neutrophil count recovery
To assess neutrophil count recovery at day 42 post transplant
Measured: Day 42
To evaluate cell recovery and function
To evaluate T/B/NK cell recovery and function at day 100, 6, 12, and 24 months after transplant.
Measured: day 100, 6, 12, and 24 months
Assess leukemia free survival
To assess leukemia free survival at 1 and 3 years.
Measured: 1 and 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Nov 2010 (estimated)
- Study startNov 2010
- Primary completionDec 2013
- Study completionDec 2013
Why it stopped: This study was withdrawn due to low accrual.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Lymphoid Hematological MalignanciesStudies of the same condition, open and under way.
- More trials run by Baylor College of MedicineThe 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
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Baylor College of Medicine
- Sponsor type
- University, hospital or other
- Collaborators
- Center for Cell and Gene Therapy, Baylor College of Medicine
- Responsible party
- Principal investigator, Caridad Martinez, Baylor College of Medicine
- Investigators
- Caridad A Martinez, MD, Principal investigator, Baylor College of MedicineRobert A Krance, MD, Principal investigator, Baylor College of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01247688
- Sponsor's protocol ID
- 26336-UCALL
- Acronym
- UCALL
- Status last verified
- Dec 2013
- First submitted
- 17 Nov 2010
- Last updated
- 12 Dec 2013
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 12 Dec 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Umbilical Cord Blood Transplant for Children With Lymphoid Hematological Malignancies (UCALL)
- ClinicalTrials.gov ID
- NCT01247688
- Acronym / sponsor ID
- UCALL / 26336-UCALL
- Keywords
- Lymphoid, hematologic malignancy, UCB, Umbilical Cord Blood Transplant, Total Body Irradiation, Cytoxan, Cyclophosphamide, Fludarabine
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: This study was withdrawn due to low accrual.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Baylor College of MedicineUniversity, hospital or other
- Collaborators
- Center for Cell and Gene Therapy, Baylor College of Medicine
- Study start
- Nov 2010
- Primary completion
- Dec 2013 (actual)
- Study completion
- Dec 2013 (actual)
- First posted
- 24 Nov 2010 (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.
The purpose of this study is to determine the safety and effectiveness of UCBT to treat patients with lymphoid hematological diseases and to see if this treatment can decrease the incidence of leukemia relapse, GVHD and infections.
These patients have a type of blood cell disorder that is very hard to cure. This treatment that is being used in this trial is known as a stem cell transplant. This treatment might help the patient live longer without the disease. It uses much stronger doses of drugs and radiation to kill the diseased cells that could be given without the transplant. We also think that the healthy cells from the donor may help fight any diseased cells left after the transplant.
For the transplant to take place, we will administer stem cells from a 'donor' whose cells best 'match' the patient's. In this study umbilical cords will be the source of the stem cells. Before the transplant, two very strong drugs plus total body irradiation will be given to as preconditioning. This treatment will kill most of your blood-forming cells in the bone marrow. The patient will then get then healthy stem cells.
If the patient has the disease in the central nervous system (CNS), they will receive radiation to the head and spine before starting the conditioning. This is to try to get disease control in the CNS. Radiation will not be given for children under 2 yrs old.
Currently, many umbilical cord blood units are available in public banks for transplantation in patients lacking bone marrow donors. UCB transplants (UCBT) may offer several advantages over adult bone marrow or peripheral blood stem cell transplants, including:
- rapid availability,
- absence of donor risk,
- low risk of transmissible infectious diseases,
- low risk of acute GvHD (Graft vs. Host Disease)
The three main causes of death after umbilical cord blood transplantation for these kind of disorders are graft failure, infection and disease relapse.
In this study we are trying to address these three problems:
To help improve engraftment we will add the drug Fludarabine to Cytoxan and total body irradiation. Fludarabine is a very strong medicine. We will try to decrease infections and reduce leukemia relapse by using fludarabine instead of antithymocyte globulin (ATG).
Detailed description
After the eligible criterion for treatment has been met and a suitable UCB stem donor has been found, the patient will have a central line placed.
Research Therapy:
After placement of the central line, the following chemotherapy will be given to after admission to the hospital and before the infusion of the umbilical cord blood stem cells:
- 9 - 6 days before the infusion: Total Body Irradiation (TBI) in two fractions ("doses") per days.
- 5 - 2 days before the infusion: Cytoxan given daily for 4 days, over 1 hour as an intravenous infusion. Mesna will be given per standards. Mesna is a drug given to decrease the side effects of Cytoxan. It will be given daily as an intravenous infusion while the patient receives the Cytoxan.
- 4 - 2 days before the infusion: Fludarabine given daily for 3 days over 1 hour as an intravenous infusion.
Stem cell transplant (intravenous infusion of the UCB stem cells) - defined as day 0 of the treatment. All other "numbered" days relate to this infusion date. For example, Day 1 is the first day after the stem cell transplant.
The following medications will be given to help decrease side effects from the chemotherapy and UCB infusion: Cyclosporine A (CSA) will be given starting 3 days prior to the stem cell infusion. It will be given daily over 2 hours every 12 hours, after the infusion and then tapered if no GVHD is present.
Administration of Mycophenolate mofetil (MMF) will start on the day the stem cell infusion is completed and will continue daily for 45 days, unless the patient develops GvHD.
Intravenous immunoglobulin's (IVIG) will be given monthly until GVHD therapy is stopped and there is evidence that the patient's body is producing antibodies.
Study Evaluations: Various study evaluations will be performed before and during the trial.
Follow Up: After year 1, the patient will be asked to return to the clinic once a year for consultations and bone marrow tests. A follow up bone marrow biopsy and aspirate will be done 1 and 2 years after transplant. Consultations with specialists will be similar to the ones the patient had before the transplant.
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 Transplant Treatment Plan | Experimental | Drug: Cytoxan, Drug: Fludarabine, Radiation: Total Body Irradiation (TBI), Procedure: Cord Blood Stem Cell InfusionCytoxan, Fludarabine, Total Body Irradiation (TBI), Cord Blood Stem Cell Infusion |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Potential recipients of umbilical cord blood transplantation (i.e. HLA (human leukocyte antigen) matched or mismatched / related or unrelated) with a lymphoid hematologic malignancy (acute lymphoblastic leukemia, hodgkin/non-hodgkin lymphoma) unlikely to be cure by standard chemotherapy. This includes patients who have relapsed after standard chemotherapy treatments and patients in first remission with unfavorable prognostics features.
- Related or Unrelated Umbilical Cord Blood Unit with 0-1 antigen mismatch, Total Nucleated cell dose of greater than or equal to 4 x 10^7/kg.
- Lansky/Karnofsky scores greater than or equal to 60
- Negative pregnancy test (if applicable)
- Written informed consent and/or signed assent line from patient, parent or guardian.
Key exclusion criteria (6 listed)
- Patients with uncontrolled infections. For bacterial infections, patients must be receiving definitive therapy and have no signs of progressing infection for 72 hours prior to enrollment. For fungal infections patients must be receiving definitive systemic antifungal therapy and have no signs of progressing infection for 1 week prior to enrollment. Progressing infection is defined as hemodynamic instability attributable to sepsis or new symptoms, worsening physical signs or radiographic findings attributable to infection. Persisting fever without other signs or symptoms will not be interpreted as progressing infection.
- Severe renal disease (Creatinine > x 3 normal for age)
- Severe hepatic disease (direct bilirubin > 3 mg/dl or SGOT (Serum glutamic oxaloacetic transaminase) > 500)
- Patient has DLCO (Diffusing Capacity of the Lung for Carbon Monoxide) < 50% predicted or FEV1 (Forced expiratory volume ) < 50% of predicted, if applicable
- Patients with symptomatic cardiac failure unrelieved by medical therapy or evidence of significant cardiac dysfunction by echocardiogram (shortening fraction<20%).
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 |
|---|---|---|
| CytoxanAlso: Cyclophophamide50 mg/kg/dose will be given IV on day -5,-4, -3, -2 over 1 hour. The total dose to be given over 4 days is 200 mg/kg. | Drug | Umbilical Cord Blood Transplant Treatment Plan |
| FludarabineAlso: Fludara40mg/m2/day will be given IV over 1 hour on day -4, -3, -2 | Drug | Umbilical Cord Blood Transplant Treatment Plan |
| Total Body Irradiation (TBI)1.75Gy/fraction will be given on day -9, -8, -7, -6 | Radiation | Umbilical Cord Blood Transplant Treatment Plan |
| Cord Blood Stem Cell InfusionThe cord blood stem cells will be infused on day 0 | Procedure or surgery | Umbilical Cord Blood Transplant Treatment Plan |
Primary outcome
Overall Survival at 1 year after UCB transplant in pediatric patients
Time frame: 1 year
To determine the survival rate at 1 year after umbilical cord blood transplant in pediatric patients with lymphoid hematological malignancies
7 secondary outcomes
Assessment of Platelet Count Recovery
Time frame: Day 42
Number of participants with severe acute GVHD grade III-IV as an assessment of safety
Time frame: Day 100
Number of participants with chronic GvHD as an assessment of safety
Time frame: 1 year
Assess relapse rate after transplant
Time frame: 1 and 3 years
Assessment of neutrophil count recovery
Time frame: Day 42
To evaluate cell recovery and function
Time frame: day 100, 6, 12, and 24 months
Assess leukemia free survival
Time frame: 1 and 3 years
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Dec 2013.
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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.
Caridad A Martinez, MD principal investigator
Baylor College of Medicine
Robert A Krance, MD principal investigator
Baylor College of Medicine
Questions about this trial
What is this clinical trial studying?
A study testing Cytoxan, Fludarabine and Total Body Irradiation (TBI) in people with Lymphoid Hematological Malignancies and Umbilical Cord Blood Transplant, run by Baylor College of Medicine.
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 5 inclusion and 6 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: withdrawn, as of the sponsor's last update on 12 Dec 2013. Status can change between updates, so confirm with the study contact before making plans.
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?
The study was withdrawn before enrolling participants, so there are no results.
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?
Overall Survival at 1 year after UCB transplant in pediatric patients (time frame: 1 year). 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 12 Dec 2013.