Acute Myeloid LeukemiaPhase 1NCT00667927
Autologous Bone Marrow Transplant for Children With Acute Myelogenous Leukemia (AML) in First Complete Remission
A Phase 1 study testing Busulfan, Cyclophosphamide and Mesna in people with Acute Myeloid Leukemia, run by St. Jude Children's Research Hospital at 1 site in United States.
Trial summary
This study proposes to transfer marker genes (detectable genetic traits or segments of DNA that can be identified and tracked) into aliquots of marrow obtained for Bone Marrow Transplant (BTM) in patients in remission of Acute Myelogenous Leukemia (AML). Read more about this study
- Who is it for?
- Ages 1 year to 18 years
- Conditions the study is for
- Acute Myeloid Leukemia
- What is being tested?
- Busulfan, Cyclophosphamide and Mesna
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 17 participants
- Where
- 1 siteUnited States
- Study dates
- Mar 1991to Jan 2008
- Phase
- Phase 1
- 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 1 year to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients aged between 1 and 18 years at diagnosis with acute nonlymphocytic leukemia in first remission are eligible for this protocol.
- Patients enrolled on the AML-87 study in second or subsequent remission are eligible for this protocol.
Who cannot join
- Has an HLA-matched, MLC-compatible donor(unless parents and/or patient refuses transplant.
- Diagnosis of FAB M3 or FAB M3v (acute progranulocytic leukemia)
- Life expectancy limited by disease other than leukemia
- Significant cardiac disease (echo shortening fraction <25% or MUGA scan <50%)
- Severe renal dysfunction, i.e., creatinine clearance less than 60cc/1.73 m2/min
- Severe restrictive pulmonary disease (FCV less than 40% of predicted)
Show all 14Show fewer
- Severe hepatic disease (bilirubin greater than 3 mg/dl or SGPT greater than 500IU)
- Severe personality disorder or mental illness
- Previous severe cystitis from cyclophosphamide
- Previous total dose of anthracyclines of >450 mg/m2
- Sever infection that on evaluation by the PI precludes ablative chemotherapy or successful transplantation
- Previous autologous transplant
- HIV reactivity
- Karnofsky score <70%
Trial locations and contacts
1 site in 1 country.
United States1 site
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
About this study
Official title: Autologous Bone Marrow Transplant for Children With AML in First Complete Remission: Use of Marker Genes to Investigate the Biology of Marrow Reconstitution and the Mechanism of Relapse
This study proposes to transfer marker genes (detectable genetic traits or segments of DNA that can be identified and tracked) into aliquots of marrow obtained for Bone Marrow Transplant (BTM) in patients in remission of Acute Myelogenous Leukemia (AML).
Read the detailed description
The primary objective of this study was to estimate the continuous complete remission rate at 2 years post transplant for children with AML in first complete remission treated with autologous BMT.
Secondary objectives used transduction of marker genes into autologous marrow to determine the following:
- whether the source of relapse after BMT for AML is residual malignant cells in the harvested marrow or in the patient, and whether marrow purging is therefore rational.
- whether the majority of AML, which lack genetic markers, represent abnormalities in a multi-lineage progenitor cell, and whether therefore, auto grafting/intensified chemotherapy is ever likely to augment the cure rate.
- the mechanisms of autologous reconstitution, and the effects of stimuli which modify the process.
- Conditions
- Acute Myeloid Leukemia
- Being tested
- Busulfan, Cyclophosphamide, Mesna
- Keywords
- Autologous bone marrow transplant, gene marking
- Medical subject headings
- Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BusulfanDrug
Also called Myleran
See Detailed Description section for description of treatment plan.
CyclophosphamideDrug
Also called Cytoxan, CTX
See Detailed Description section for description of treatment plan.
MesnaDrug
Also called MESNCX
See Detailed Description section for description of treatment plan.
| Study group | Role | What they receive |
|---|---|---|
| 1 | Other | Not described Receives: Drug: Busulfan; Drug: Cyclophosphamide; Drug: Mesna |
What is being measured
The primary outcome is the main question the study is designed to answer.
To estimate the continuous complete remission rate at 2 years for children with AML in first complete remission treated with Autologous Bone Marrow Transplant (ABMT).
Measured: 2 years post transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Apr 2008 (estimated)
- Study startMar 1991
- Primary completionJan 1995
- Study completionJan 2008
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid LeukemiaStudies of the same condition, open and under way.
- More trials run by St. Jude Children's Research HospitalThe 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 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 17 participants (actual)
Sponsor and investigators
- Lead sponsor
- St. Jude Children's Research Hospital
- Sponsor type
- University, hospital or other
- Investigators
- Gregory A Hale, MD, Principal investigator, St. Jude Children's Research Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00667927
- Sponsor's protocol ID
- AMLREM
- Status last verified
- Apr 2008
- First submitted
- 24 Apr 2008
- Last updated
- 28 Apr 2008
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Apr 2008. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Autologous Bone Marrow Transplant for Children With AML in First Complete Remission: Use of Marker Genes to Investigate the Biology of Marrow Reconstitution and the Mechanism of Relapse
- ClinicalTrials.gov ID
- NCT00667927
- Acronym / sponsor ID
- AMLREM
- Conditions
- Acute Myeloid Leukemia
- Keywords
- Autologous bone marrow transplant, gene marking
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 17 participants (actual)
- Lead sponsor
- St. Jude Children's Research HospitalUniversity, hospital or other
- Study start
- Mar 1991
- Primary completion
- Jan 1995 (actual)
- Study completion
- Jan 2008 (actual)
- First posted
- 28 Apr 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.
This study proposes to transfer marker genes (detectable genetic traits or segments of DNA that can be identified and tracked) into aliquots of marrow obtained for Bone Marrow Transplant (BTM) in patients in remission of Acute Myelogenous Leukemia (AML).
Detailed description
The primary objective of this study was to estimate the continuous complete remission rate at 2 years post transplant for children with AML in first complete remission treated with autologous BMT.
Secondary objectives used transduction of marker genes into autologous marrow to determine the following:
- whether the source of relapse after BMT for AML is residual malignant cells in the harvested marrow or in the patient, and whether marrow purging is therefore rational.
- whether the majority of AML, which lack genetic markers, represent abnormalities in a multi-lineage progenitor cell, and whether therefore, auto grafting/intensified chemotherapy is ever likely to augment the cure rate.
- the mechanisms of autologous reconstitution, and the effects of stimuli which modify the process.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 1 | Other | Drug: Busulfan, Drug: Cyclophosphamide, Drug: Mesna |
Who may qualify
- Ages
- 1 year to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (2 listed)
- Patients aged between 1 and 18 years at diagnosis with acute nonlymphocytic leukemia in first remission are eligible for this protocol.
- Patients enrolled on the AML-87 study in second or subsequent remission are eligible for this protocol.
Key exclusion criteria (14 listed)
- Has an HLA-matched, MLC-compatible donor(unless parents and/or patient refuses transplant.
- Diagnosis of FAB M3 or FAB M3v (acute progranulocytic leukemia)
- Life expectancy limited by disease other than leukemia
- Significant cardiac disease (echo shortening fraction <25% or MUGA scan <50%)
- Severe renal dysfunction, i.e., creatinine clearance less than 60cc/1.73 m2/min
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: MyleranSee Detailed Description section for description of treatment plan. | Drug | 1 |
| CyclophosphamideAlso: Cytoxan, CTXSee Detailed Description section for description of treatment plan. | Drug | 1 |
| MesnaAlso: MESNCXSee Detailed Description section for description of treatment plan. | Drug | 1 |
Primary outcome
To estimate the continuous complete remission rate at 2 years for children with AML in first complete remission treated with Autologous Bone Marrow Transplant (ABMT).
Time frame: 2 years post transplant
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Apr 2008.
- Related InfoLink provided by the sponsor.
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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.
Gregory A Hale, MD principal investigator
St. Jude Children's Research Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Busulfan, Cyclophosphamide and Mesna in people with Acute Myeloid Leukemia, run by St. Jude Children's Research Hospital 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 2 inclusion and 14 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 Apr 2008. 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 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.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
To estimate the continuous complete remission rate at 2 years for children with AML in first complete remission treated with Autologous Bone Marrow Transplant (ABMT). (time frame: 2 years post transplant). 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 Apr 2008.