LeukemiaPhase 1/2NCT00679536
Allogeneic Transplantation for Pediatric Leukemias With Unrelated Donors
A Phase 1/2 study testing Busulfan, Fludarabine and Thymoglobulin in people with Leukemia, run by Ann & Robert H Lurie Children's Hospital of Chicago at 1 site in United States.
Trial summary
The study proposes the use of Fludarabine, Busulfan, Anti Thymocyte Globulin Rabbit (ATG) and Total Body Irradiation as a preparative regimen before hematopoietic stem cell transplant from unrelated donor peripheral blood stem cells (PBSC). Read more about this study
- Who is it for?
- Ages Up to 21 years
- Conditions the study is for
- Leukemia
- What is being tested?
- Busulfan, Fludarabine and Thymoglobulin
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteUnited States
- Study dates
- May 2008to May 2015
- Phase
- Phase 1/2
- 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 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Ages 0-21
- AML in one of the following stages:
- Having preceding myelodysplasia (MDS)
- High Risk cytogenetics
- Requiring > 2 cycles chemotherapy to obtain complete remission
- High allelic ratio FLT3/ITD+,
Show all 34Show fewer
- Standard risk cytogenetics with positive MRD at end of Induction
- Second or greater CR
- First relapse with < 25% blasts in bone marrow
- With therapy-related AML whose prior malignancy has been in remission for at least 12 months
- ALL in one of the following stages:
- High risk first remission, defined as:
- Ph+ ALL; or,
- 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 marrow or MRD>1% with M2-3 marrow or MRD>1% at Day 42.
- High-risk infant ALL defined as age <6 months at diagnosis with MLL (11q23) translocation.
- High risk second remission, defined as:
- Bone marrow relapse < 36 months from induction; or >36 mths if a matched sibling donor is available
- T-lineage relapse at any time; or,
- Very early isolated CNS relapse (<18 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 first relapse must have < 25% blasts in bone marrow
- MDS at any stage; prior therapies allowed
- CML in chronic or accelerated phase; prior therapies allowed
- Patient also must have the following organ requirements:
- Adequate renal function defined as serum creatinine <2x normal, or creatinine clearance > 40 ml/min/m^2 or 70 ml/min.
- Adequate liver function as defined by total bilirubin less than or equal to 2 times normal and AST and ALT less than or equal to 4 times normal.
- Adequate cardiac function as defined by: shortening fraction > 24% by echocardiogram, or ejection fraction > 30% by radionuclide angiogram.
- Adequate pulmonary function as defined by DLCO, FEV1/FVC > 60% by pulmonary function tests. For children who are uncooperative for PFTs and have no evidence of dyspnea at rest or exercise intolerance, pulse oximetry > 94% on room air is considered acceptable, with a normal chest xray.
- Adequate venous access; a double lumen central vascular access device or its equivalent and an additional PICC line will be required for all patients.
- Women of childbearing potential and sexually active males should use effective contraception while on study.
Who cannot join
- Inability to give informed consent or assent
- Inability to obtain a suitable donor
- Patient who is HIV-positive
- Patient who has active Hepatitis B
- Patient who is pregnant
- Patient who is otherwise considered unsuitable for transplant at the discretion of the principal investigator.
Trial locations and contacts
1 site in 1 country.
United States1 site, 1 recruiting
- Recruiting
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Sonali Chaudhury, MD, 312-227-4863, schaudhury@luriechildrens.org
Colleen E Rosen, BS, RN, 312-227-4870, crosen@luriechildrens.org
About this study
Official title: Allogeneic Transplantation for Pediatric Leukemias With Unrelated Donors Using Fludarabine, Busulfan, 400 cGy Total Body Irradiation, and Thymoglobulin
The study proposes the use of Fludarabine, Busulfan, Anti Thymocyte Globulin Rabbit (ATG) and Total Body Irradiation as a preparative regimen before hematopoietic stem cell transplant from unrelated donor peripheral blood stem cells (PBSC). The hypothesis states that the 100 day mortality after this type of transplant will be significantly below the accepted standards, which is about 30% for unrelated donors.
Read the detailed description
The primary objective of this study is to evaluate the toxicity (as measured as 100 day survival) after hematopoietic stem cell transplant from an unrelated donor with a novel preparative regimen of Fludarabine, Busulfan, Anti-Thymocyte Globulin, and Total Body Irradiation for pediatric patients with leukemia. The secondary objectives are to evaluate the relapse-free and overall survival after hematopoietic stem cell transplant as well as to evaluate the incidence of acute and chronic graft-versus-host disease after this preparative regimen.
- Conditions
- Leukemia
- Being tested
- Busulfan, Fludarabine, Thymoglobulin, Total Body Irradiation
- Keywords
- Reduced Toxicity, Toxicity, Event Free Survival, Graft vs Host Disease
- Medical subject headings
- Leukemia, Graft vs Host Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BusulfanDrug
Also called IV Busulfex
Patient will receive a Test Dose of Busulfan on either Day -10 or Day -9. Patient will receive their Regimen Dose of Busulfan on Day -5 to Day -2. The regimen dose of Busulfan will be based off of the findings from their Test Dose.
FludarabineDrug
Also called Fludara
Patient will receive Fludarabine from Day -6 to Day -2. The dose of Fludarabine will be 30 mg/m^2/day.
ThymoglobulinDrug
The patient will also receive Thymoglobulin (rabbitATG) on Day -4 to Day -2. Each dose of rabbitATG will be 1.5 mg/kg/day.
Total Body IrradiationRadiation
On Day -1 the patient will receive a total of 400 cGy of Total Body Irradiation.
| Study group | Role | What they receive |
|---|---|---|
| A | Experimental | All patients on this trial will receive a conditioning regimen of Busulfan, Fludarabine, Anti-Thymocyte Globulin and Total Body Irradiation (400 cGy) Receives: Drug: Busulfan; Drug: Fludarabine; Drug: Thymoglobulin; Radiation: Total Body Irradiation |
What is being measured
The primary outcome is the main question the study is designed to answer.
To evaluate the toxicity (as measures by 100 day survival) after hematopoietic stem cell transplant from an unrelated donor with a novel preparative regimen.
Measured: 100 day mortality
Secondary outcomes (2)
To evaluate the relapse-free and overall survival after hematopoietic stem cell transplant with Fludarabine/Busulfan/ATG/TBI preparative regimen for pediatric patients with leukemia.
Measured: 5 years
To evaluate the incidence of acute and chronic graft-versus-host disease after hematopoietic stem cell transplant
Measured: 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 May 2008 (estimated)
- Study startMay 2008
- Primary completionMay 2015 (estimated)
- Study completionMay 2015 (estimated)
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
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Sonali Chaudhury, MD, Ann & Robert H Lurie Children's Hospital of Chicago
- Investigators
- Sonali Chaudhury, MD, Principal investigator, Ann & Robert H Lurie Children's Hospital of Chicago
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00679536
- Sponsor's protocol ID
- SCT 0208
- Status last verified
- Jan 2014
- First submitted
- 15 May 2008
- Last updated
- 23 Jan 2014
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 23 Jan 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Allogeneic Transplantation for Pediatric Leukemias With Unrelated Donors Using Fludarabine, Busulfan, 400 cGy Total Body Irradiation, and Thymoglobulin
- ClinicalTrials.gov ID
- NCT00679536
- Acronym / sponsor ID
- SCT 0208
- Conditions
- Leukemia
- Keywords
- Reduced Toxicity, Toxicity, Event Free Survival, Graft vs Host Disease
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Ann & Robert H Lurie Children's Hospital of ChicagoUniversity, hospital or other
- Study start
- May 2008
- Primary completion
- May 2015 (estimated)
- Study completion
- May 2015 (estimated)
- First posted
- 19 May 2008 (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 study proposes the use of Fludarabine, Busulfan, Anti Thymocyte Globulin Rabbit (ATG) and Total Body Irradiation as a preparative regimen before hematopoietic stem cell transplant from unrelated donor peripheral blood stem cells (PBSC). The hypothesis states that the 100 day mortality after this type of transplant will be significantly below the accepted standards, which is about 30% for unrelated donors.
Detailed description
The primary objective of this study is to evaluate the toxicity (as measured as 100 day survival) after hematopoietic stem cell transplant from an unrelated donor with a novel preparative regimen of Fludarabine, Busulfan, Anti-Thymocyte Globulin, and Total Body Irradiation for pediatric patients with leukemia. The secondary objectives are to evaluate the relapse-free and overall survival after hematopoietic stem cell transplant as well as to evaluate the incidence of acute and chronic graft-versus-host disease after this preparative regimen.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| A | Experimental | Drug: Busulfan, Drug: Fludarabine, Drug: Thymoglobulin, Radiation: Total Body IrradiationAll patients on this trial will receive a conditioning regimen of Busulfan, Fludarabine, Anti-Thymocyte Globulin and Total Body Irradiation (400 cGy) |
Who may qualify
- Ages
- Up to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (34 listed)
- Ages 0-21
- AML in one of the following stages:
- ALL in one of the following stages:
- High risk first remission, defined as:
- High risk second remission, defined as:
Key exclusion criteria (6 listed)
- Inability to give informed consent or assent
- Inability to obtain a suitable donor
- Patient who is HIV-positive
- Patient who has active Hepatitis B
- Patient who is pregnant
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: IV BusulfexPatient will receive a Test Dose of Busulfan on either Day -10 or Day -9. Patient will receive their Regimen Dose of Busulfan on Day -5 to Day -2. The regimen dose of Busulfan will be based off of the findings from their Test Dose. | Drug | A |
| FludarabineAlso: FludaraPatient will receive Fludarabine from Day -6 to Day -2. The dose of Fludarabine will be 30 mg/m^2/day. | Drug | A |
| ThymoglobulinThe patient will also receive Thymoglobulin (rabbitATG) on Day -4 to Day -2. Each dose of rabbitATG will be 1.5 mg/kg/day. | Drug | A |
| Total Body IrradiationOn Day -1 the patient will receive a total of 400 cGy of Total Body Irradiation. | Radiation | A |
Primary outcome
To evaluate the toxicity (as measures by 100 day survival) after hematopoietic stem cell transplant from an unrelated donor with a novel preparative regimen.
Time frame: 100 day mortality
2 secondary outcomes
To evaluate the relapse-free and overall survival after hematopoietic stem cell transplant with Fludarabine/Busulfan/ATG/TBI preparative regimen for pediatric patients with leukemia.
Time frame: 5 years
To evaluate the incidence of acute and chronic graft-versus-host disease after hematopoietic stem cell transplant
Time frame: 5 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Jan 2014.
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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.
Sonali Chaudhury, MD principal investigator
Ann & Robert H Lurie Children's Hospital of Chicago
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Busulfan, Fludarabine and Thymoglobulin in people with Leukemia, run by Ann & Robert H Lurie Children's Hospital of Chicago 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?
The sponsor lists 34 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: status not confirmed, as of the sponsor's last update on 23 Jan 2014. 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.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
Contact Sonali Chaudhury, MD (312-227-4863, schaudhury@luriechildrens.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To evaluate the toxicity (as measures by 100 day survival) after hematopoietic stem cell transplant from an unrelated donor with a novel preparative regimen. (time frame: 100 day mortality). 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 23 Jan 2014.