Juvenile Myelomonocytic LeukemiaPhase 1/2NCT00167219
Stem Cell Transplant for Juvenile Myelomonocytic Leukemia (JMML)
A Phase 1/2 study testing Stem Cell Transplant and Preparative Regimen in people with Juvenile Myelomonocytic Leukemia, run by Masonic Cancer Center, University of Minnesota at 1 site in United States.
Trial summary
The investigators hypothesize that long-term disease-free survival (DFS) in patients with JMML can be achieved with a treatment of busulfan (BU), cyclophosphamide (CY) and melphalan (L-PAM) followed by hematopoietic cell transplantation (HCT). Read more about this study
- Who is it for?
- Ages Up to 18 years
- Conditions the study is for
- Juvenile Myelomonocytic Leukemia
- What is being tested?
- Stem Cell Transplant and Preparative Regimen
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 siteUnited States
- Study dates
- 18 Nov 1999to 9 Aug 2023
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients must have a diagnosis of JMML and fulfill these minimal criteria (International diagnostic criteria for JMML):
- Leukocytosis (> 13,000) with absolute monocytosis (> 1,000)
- The presence of immature myeloid cells in the peripheral blood
- Less than 30% marrow blasts
- Absence of t(9:22) or BCR-ABL transcript
- Adequate major organ function including:
Show all 11Show fewer
- Cardiac: ejection fraction > 45%
- Hepatic: no clinical evidence of hepatic failure (e.g. coagulopathy, ascites)
- Karnofsky performance status > 70% or Lansky score > 50%
- Creatinine must be < 2 x normal for age
- Written informed consent.
Who cannot join
- Active uncontrolled infection within one week of HCT.
Trial locations and contacts
1 site in 1 country.
United States1 site
Masonic Cancer Center, University of Minnesota
Minneapolis, Minnesota, 55455
About this study
Official title: Hematopoietic Cell Transplantation in Children With Juvenile Myelomonocytic Leukemia
The investigators hypothesize that long-term disease-free survival (DFS) in patients with JMML can be achieved with a treatment of busulfan (BU), cyclophosphamide (CY) and melphalan (L-PAM) followed by hematopoietic cell transplantation (HCT).
Read the detailed description
Prior to transplantation, subjects will receive BUSULFAN via the central venous line, six times a day for four days, CYCLOPHOSPHAMIDE via the central venous line once a day for two days, and MELPHALAN via the central venous line for one day. Busulfan, cyclophosphamide, and melphalan are given to destroy the subject's leukemia. As well, these drugs will destroy the subject's own immune system to help ensure the new bone marrow takes and grows after transplantation.
On the day of transplantation, bone marrow or umbilical cord blood from the donor will arrive to the bone marrow transplant unit and be transfused via venous line. These new cells will replace the subject's bone marrow.
- Conditions
- Juvenile Myelomonocytic Leukemia
- Being tested
- Stem Cell Transplant, Preparative Regimen
- Keywords
- Stem cell transplant, long term survival, retinoic acid
- Medical subject headings
- Leukemia, Myelomonocytic, Juvenile
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Stem Cell TransplantBiological
Also called Bone marrow transplantation
Transplantation on Day 0.
Preparative RegimenDrug
- Busulfan
- Cyclophosphamide
- Mesna
- Melphalan
- Anti-thymocyte Globulin (ATG)
| Study group | Role | What they receive |
|---|---|---|
| Intent-to-Treat | Experimental | Patients receiving study regimen. Receives: Biological: Stem Cell Transplant; Drug: Preparative Regimen |
What is being measured
The primary outcome is the main question the study is designed to answer.
Determine Probability of Long-term Disease Free Survival in JMML
Measured: at 1 year after transplant
Secondary outcomes (1)
Combined Incidence of Neutrophil Engraftment, Graft-versus-host Disease (GVHD), Regimen-related Toxicity, and Relapse.
Measured by AGVHD Grades 2 - 4.
Measured: at 1 year after transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Sep 2005 (estimated)
- Study start18 Nov 1999
- Primary completion12 Jul 2022
- Study completion9 Aug 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Juvenile Myelomonocytic LeukemiaStudies of the same condition, open and under way.
- More trials run by Masonic Cancer Center, University of MinnesotaThe 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Margaret MacMillan, MD, Principal investigator, Masonic Cancer Center, University of Minnesota
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00167219
- Sponsor's protocol ID
- 1999LS073
- Other IDs
- MT1999-20, 9911M24961
- Status last verified
- Nov 2023
- First submitted
- 9 Sep 2005
- Last updated
- 7 Nov 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Nov 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Hematopoietic Cell Transplantation in Children With Juvenile Myelomonocytic Leukemia
- ClinicalTrials.gov ID
- NCT00167219
- Acronym / sponsor ID
- 1999LS073
- Conditions
- Juvenile Myelomonocytic Leukemia
- Keywords
- Stem cell transplant, long term survival, retinoic acid
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- Masonic Cancer Center, University of MinnesotaUniversity, hospital or other
- Study start
- 18 Nov 1999 (actual)
- Primary completion
- 12 Jul 2022 (actual)
- Study completion
- 9 Aug 2023 (actual)
- First posted
- 14 Sep 2005 (estimated)
- Results
- Posted 7 Nov 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The investigators hypothesize that long-term disease-free survival (DFS) in patients with JMML can be achieved with a treatment of busulfan (BU), cyclophosphamide (CY) and melphalan (L-PAM) followed by hematopoietic cell transplantation (HCT).
Detailed description
Prior to transplantation, subjects will receive BUSULFAN via the central venous line, six times a day for four days, CYCLOPHOSPHAMIDE via the central venous line once a day for two days, and MELPHALAN via the central venous line for one day. Busulfan, cyclophosphamide, and melphalan are given to destroy the subject's leukemia. As well, these drugs will destroy the subject's own immune system to help ensure the new bone marrow takes and grows after transplantation.
On the day of transplantation, bone marrow or umbilical cord blood from the donor will arrive to the bone marrow transplant unit and be transfused via venous line. These new cells will replace the subject's bone marrow.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Intent-to-Treat | Experimental | Biological: Stem Cell Transplant, Drug: Preparative RegimenPatients receiving study regimen. |
Who may qualify
- Ages
- Up to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (11 listed)
- Patients must have a diagnosis of JMML and fulfill these minimal criteria (International diagnostic criteria for JMML):
- Written informed consent.
Key exclusion criteria (1 listed)
- Active uncontrolled infection within one week of HCT.
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 |
|---|---|---|
| Stem Cell TransplantAlso: Bone marrow transplantationTransplantation on Day 0. | Biological | Intent-to-Treat |
| Preparative Regimen* Busulfan * Cyclophosphamide * Mesna * Melphalan * Anti-thymocyte Globulin (ATG) | Drug | Intent-to-Treat |
Primary outcome
Determine Probability of Long-term Disease Free Survival in JMML
Time frame: at 1 year after transplant
1 secondary outcome
Combined Incidence of Neutrophil Engraftment, Graft-versus-host Disease (GVHD), Regimen-related Toxicity, and Relapse.
Time frame: at 1 year after transplant
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Nov 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Margaret MacMillan, MD principal investigator
Masonic Cancer Center, University of Minnesota
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 Stem Cell Transplant and Preparative Regimen in people with Juvenile Myelomonocytic Leukemia, run by Masonic Cancer Center, University of Minnesota 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 11 inclusion and 1 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 7 Nov 2023. 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?
Yes. Results are posted in the registry (first posted 7 Nov 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Determine Probability of Long-term Disease Free Survival in JMML (time frame: at 1 year after 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 7 Nov 2023.