LeukemiaPhase 1NCT00245115
Ex Vivo Expansion of Mafosfamide Purged CD34+ Cells in Patients With Acute Leukemia
A Phase 1 study testing filgrastim, busulfan and cyclophosphamide in people with Leukemia, run by Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins at 1 site in United States.
Trial summary
RATIONALE: Giving colony-stimulating factors, such as G-CSF, and certain chemotherapy drugs, helps stem cells move from the bone marrow to the blood so they can be collected. Treating stem cells collected from the patient's blood or bone marrow with chemotherapy in the laboratory removes any remaining cancer cells. Read more about this study
- Who is it for?
- Ages Up to 70 years
- Conditions the study is for
- Leukemia
- What is being tested?
- filgrastim, busulfan and cyclophosphamide
- Type of treatment
- Surgery or procedure
- How many people
- 7 participants
- Where
- 1 siteUnited States
- Study dates
- Oct 2005to Jan 2012
- 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 Up to 70 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
None listed.
Who cannot join
- Availability of a suitable matched HLA-identical sibling marrow donor
- Intermediate or good risk acute leukemia in CR1 (39)
- Age greater than 70
- Weight less than 10 kg
- Any risk of pregnancy - all female patients must have an effective form of contraception or be infertile due to hysterectomy, fallopian tube, surgery or premature menopause.
- HIV Infection: Patients must be HIV negative for inclusion.
- Poor organ function as defined in the BMT Policies and Procedures Manual.
Trial locations and contacts
1 site in 1 country.
United States1 site
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Baltimore, Maryland, 21231
About this study
RATIONALE: Giving colony-stimulating factors, such as G-CSF, and certain chemotherapy drugs, helps stem cells move from the bone marrow to the blood so they can be collected. Treating stem cells collected from the patient's blood or bone marrow with chemotherapy in the laboratory removes any remaining cancer cells. Chemotherapy or radiation therapy is given to the patient to prepare the bone marrow for stem cell transplant. The treated stem cells are then returned to the patient to replace the blood-forming cells that were destroyed by the chemotherapy.
PURPOSE: This clinical trial is studying how well an autologous peripheral stem cell or bone marrow transplant using laboratory-treated cells works in treating patients with acute leukemia.
Read the detailed description
OBJECTIVES:
- Determine the feasibility of ex vivo expanded mafosfamide-purged CD34-positive cells for autologous peripheral blood stem cell or bone marrow transplantation in patients with acute leukemia.
- Determine the duration of aplasia associated with the use of ex vivo cytokine expanded mafosfamide-purged cells in patients treated with this regimen.
- Determine, preliminarily, the event-free survival of patients treated with this regimen.
OUTLINE: This is a pilot study.
- Mobilization and stem cell collection: Patients receive cyclophosphamide IV and filgrastim (G-CSF) subcutaneously (SC) or IV once daily for 7-14 days followed by leukapheresis to collect peripheral blood stem cells (PBSCs). Some patients may also undergo bone marrow (BM) harvest if sufficient PBSCs are not collected. Patients with a sufficient number of stem cells or BM (5 x 10^6 PBSC/kg or 3 x 10^8 BM cells/kg) proceed to autologous PBSC transplantation (PBSCT) or BM transplantation (BMT).
- CD34-positive cell selection and mafosfamide purging: Collected PBSCs and/or BM are treated in the laboratory to isolate CD34-positive cells. A minimum of 1 x 10^6 nucleated CD34-positive BM cells/kg or 2 x 10^6 nucleated CD34-positive PBSCs/kg must be available after selection to proceed to mafosfamide-purging. The selected cells are then treated in vitro with mafosfamide to purge remaining leukemic cells. One third of the mafosfamide-purged cells are then cryopreserved for future use and 2/3 of the mafosfamide-purged cells proceed to ex vivo expansion.
- Ex vivo expansion: The remaining CD34-positive mafosfamide-purged cells are treated in vitro with stem cell factor, G-CSF, and recombinant human thrombopoietin and incubated for 12-14 days.
- Myeloablative therapy: Patients receive busulfan on days -9 to -6 and cyclophosphamide on days -5 to -2.
- PBSCT or BMT: Patients undergo autologous PBSCT or BMT using CD34-positive mafosfamide-purged cryopreserved cells and ex vivo expanded CD34-positive mafosfamide-purged cells on day 0 followed by G-CSF SC or IV once daily until blood counts recover.
After completion of study treatment, patients are followed periodically for at least 5 years.
PROJECTED ACCRUAL: A total of 25 patients will be accrued for this study.
- Conditions
- Leukemia
- Being tested
- filgrastim, busulfan, cyclophosphamide, in vitro-treated bone marrow transplantation, in vitro-treated peripheral blood stem cell transplantation
- Keywords
- adult acute myeloblastic leukemia without maturation (M1), childhood acute myeloblastic leukemia without maturation (M1), adult acute myeloblastic leukemia with maturation (M2), childhood acute myeloblastic leukemia with maturation (M2), adult acute promyelocytic leukemia (M3), childhood acute promyelocytic leukemia (M3), adult acute myelomonocytic leukemia (M4), childhood acute myelomonocytic leukemia (M4), adult acute monoblastic leukemia (M5a), adult acute monocytic leukemia (M5b), childhood acute monoblastic leukemia (M5a), childhood acute monocytic leukemia (M5b), adult erythroleukemia (M6a), adult pure erythroid leukemia (M6b), childhood acute erythroleukemia (M6), adult acute megakaryoblastic leukemia (M7), childhood acute megakaryocytic leukemia (M7), adult acute lymphoblastic leukemia in remission, adult acute myeloid leukemia in remission, childhood acute lymphoblastic leukemia in remission
- Medical subject headings
- Leukemia, Leukemia, Myeloid, Acute, Leukemia, Promyelocytic, Acute, Leukemia, Myelomonocytic, Acute, Leukemia, Monocytic, Acute, Leukemia, Erythroblastic, Acute, Leukemia, Megakaryoblastic, Acute, Congenital Abnormalities
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
filgrastimBiological
busulfanDrug
cyclophosphamideDrug
in vitro-treated bone marrow transplantationProcedure or surgery
in vitro-treated peripheral blood stem cell transplantationProcedure or surgery
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of patients infused with Mf-treated cells with successful expansion
Number of patients with successful expansion of a CD34 selected, mafosfamide purged autograft.
Measured: 2 weeks
Secondary outcomes (2)
Duration of aplasia as determined by Number of Days to Absolute Neutrophil Count (ANC) recovery
Measured: 1 month
Event free survival
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 posted27 Oct 2005 (estimated)
- Study startOct 2005
- Primary completionDec 2007
- Study completionJan 2012
Why it stopped: Expiration of study supply
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.
- More trials run by Sidney Kimmel Comprehensive Cancer Center at Johns HopkinsThe 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
- 7 participants (actual)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Responsible party
- Sponsor
- Investigators
- B. Douglas Smith, MD, Principal investigator, Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00245115
- Sponsor's protocol ID
- J0563 CDR0000453619
- Other IDs
- P01CA070970, P30CA006973
- Status last verified
- Aug 2018
- First submitted
- 25 Oct 2005
- Last updated
- 23 Aug 2018
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Aug 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Ex Vivo Expansion of Mafosfamide Purged CD34+ Cells in Patients With Acute Leukemia
- ClinicalTrials.gov ID
- NCT00245115
- Acronym / sponsor ID
- J0563 CDR0000453619
- Conditions
- Leukemia
- Keywords
- adult acute myeloblastic leukemia without maturation (M1), childhood acute myeloblastic leukemia without maturation (M1), adult acute myeloblastic leukemia with maturation (M2), childhood acute myeloblastic leukemia with maturation (M2), adult acute promyelocytic leukemia (M3), childhood acute promyelocytic leukemia (M3), adult acute myelomonocytic leukemia (M4), childhood acute myelomonocytic leukemia (M4)
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Expiration of study supply
- Enrolment
- 7 participants (actual)
- Lead sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns HopkinsUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Study start
- Oct 2005
- Primary completion
- Dec 2007 (actual)
- Study completion
- Jan 2012 (actual)
- First posted
- 27 Oct 2005 (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 colony-stimulating factors, such as G-CSF, and certain chemotherapy drugs, helps stem cells move from the bone marrow to the blood so they can be collected. Treating stem cells collected from the patient's blood or bone marrow with chemotherapy in the laboratory removes any remaining cancer cells. Chemotherapy or radiation therapy is given to the patient to prepare the bone marrow for stem cell transplant. The treated stem cells are then returned to the patient to replace the blood-forming cells that were destroyed by the chemotherapy.
PURPOSE: This clinical trial is studying how well an autologous peripheral stem cell or bone marrow transplant using laboratory-treated cells works in treating patients with acute leukemia.
Detailed description
OBJECTIVES:
- Determine the feasibility of ex vivo expanded mafosfamide-purged CD34-positive cells for autologous peripheral blood stem cell or bone marrow transplantation in patients with acute leukemia.
- Determine the duration of aplasia associated with the use of ex vivo cytokine expanded mafosfamide-purged cells in patients treated with this regimen.
- Determine, preliminarily, the event-free survival of patients treated with this regimen.
OUTLINE: This is a pilot study.
- Mobilization and stem cell collection: Patients receive cyclophosphamide IV and filgrastim (G-CSF) subcutaneously (SC) or IV once daily for 7-14 days followed by leukapheresis to collect peripheral blood stem cells (PBSCs). Some patients may also undergo bone marrow (BM) harvest if sufficient PBSCs are not collected. Patients with a sufficient number of stem cells or BM (5 x 10^6 PBSC/kg or 3 x 10^8 BM cells/kg) proceed to autologous PBSC transplantation (PBSCT) or BM transplantation (BMT).
- CD34-positive cell selection and mafosfamide purging: Collected PBSCs and/or BM are treated in the laboratory to isolate CD34-positive cells. A minimum of 1 x 10^6 nucleated CD34-positive BM cells/kg or 2 x 10^6 nucleated CD34-positive PBSCs/kg must be available after selection to proceed to mafosfamide-purging. The selected cells are then treated in vitro with mafosfamide to purge remaining leukemic cells. One third of the mafosfamide-purged cells are then cryopreserved for future use and 2/3 of the mafosfamide-purged cells proceed to ex vivo expansion.
- Ex vivo expansion: The remaining CD34-positive mafosfamide-purged cells are treated in vitro with stem cell factor, G-CSF, and recombinant human thrombopoietin and incubated for 12-14 days.
- Myeloablative therapy: Patients receive busulfan on days -9 to -6 and cyclophosphamide on days -5 to -2.
- PBSCT or BMT: Patients undergo autologous PBSCT or BMT using CD34-positive mafosfamide-purged cryopreserved cells and ex vivo expanded CD34-positive mafosfamide-purged cells on day 0 followed by G-CSF SC or IV once daily until blood counts recover.
After completion of study treatment, patients are followed periodically for at least 5 years.
PROJECTED ACCRUAL: A total of 25 patients will be accrued for this study.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- Up to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (0 listed)
Key exclusion criteria (7 listed)
- Availability of a suitable matched HLA-identical sibling marrow donor
- Intermediate or good risk acute leukemia in CR1 (39)
- Age greater than 70
- Weight less than 10 kg
- Any risk of pregnancy - all female patients must have an effective form of contraception or be infertile due to hysterectomy, fallopian tube, surgery or premature menopause.
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 |
|---|---|---|
| filgrastim | Biological | – |
| busulfan | Drug | – |
| cyclophosphamide | Drug | – |
| in vitro-treated bone marrow transplantation | Procedure or surgery | – |
| in vitro-treated peripheral blood stem cell transplantation | Procedure or surgery | – |
Primary outcome
Number of patients infused with Mf-treated cells with successful expansion
Time frame: 2 weeks
Number of patients with successful expansion of a CD34 selected, mafosfamide purged autograft.
2 secondary outcomes
Duration of aplasia as determined by Number of Days to Absolute Neutrophil Count (ANC) recovery
Time frame: 1 month
Event free survival
Time frame: 5 years
Results and safety
The study was stopped early (reason given: Expiration of study supply). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Aug 2018.
- Individual participant dataSharing planned: No
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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.
B. Douglas Smith, MD principal investigator
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
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 filgrastim, busulfan and cyclophosphamide in people with Leukemia, run by Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins 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 0 inclusion and 7 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: stopped early, as of the sponsor's last update on 23 Aug 2018. 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 was stopped early (reason given: Expiration of study supply). 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.
What does the primary outcome measure?
Number of patients infused with Mf-treated cells with successful expansion (time frame: 2 weeks). 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 Aug 2018.