Acute Myeloid LeukemiaPhase 2NCT00491634
Treosulfan-based Conditioning for Transplantation in AML/MDS
A Phase 2 study testing treosulfan in people with Acute Myeloid Leukemia and Myelodysplastic Syndrome, run by Dr. Avichai Shimoni MD at 1 site in Israel.
Trial summary
The study hypotheses is that the introduction of dose escalated treosulfan, in substitution to busulfan, will reduce toxicity after allogeneic transplantation while improving myeloablation and and disease control in patients with AML and MDS not eligible for standard transplantation. Read more about this study
- Who is it for?
- Ages 18 to 68 years
- Conditions the study is for
- Acute Myeloid Leukemia, Myelodysplastic Syndrome
- What is being tested?
- treosulfan
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 1 siteIsrael
- Study dates
- Jun 2007to Jun 2014
- Phase
- Phase 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 18 to 68 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age less than physiologic 68 years.
- Patients with AML and MDS not eligible for standard TBI- or Busulfan-based myeloablative conditioning due to age, concurrent medical condition, or extensive prior therapy (e.g. age > 55 years for HLA-matched sibling transplants or > 50 for matched unrelated donor transplants, prior / concomitant pulmonary, liver, or other organ complications).
- This study will only include patients with chemo-refractory disease or previously untreated active disease.
- acute myeloid leukemias (AML) according to WHO classification (> 20% myeloblasts in peripheral blood or bone marrow at diagnosis) in induction failure, PR, untreated or chemo-refractory relapse. Patients must have > 10% marrow blasts at the time of transplantation.
- myelodysplastic syndromes (MDS) according to WHO classification (< 20% myeloblasts in peripheral blood and bone marrow at diagnosis), indicated for allogeneic transplantation:
- refractory anaemia with excess blasts (RAEB-1 and RAEB-2) with no prior therapy
- Patients must have an HLA matched related or unrelated donor willing to donate either peripheral blood stem cells or bone marrow. Matching is based on high-resolution class I (HLA-A, -B, -C) and class II (HLA-DRB1, -DQB1) typing. The goal is to transplant > 3 x 106 CD34+ cells per kg body weight of the recipient -
Who cannot join
- Bilirubin > 3.0 mg/dl, transaminases > 3 times upper normal limit
- Creatinine > 2.0 mg/dl
- ECOG-Performance status > 2
- Uncontrolled infection
- Pregnancy or lactation
- Abnormal lung diffusion capacity (DLCO < 40% predicted)
Show all 11Show fewer
- Severe cardiovascular disease
- CNS disease involvement
- Pleural effusion or ascites > 1 liter
- Known hypersensitivity to fludarabine or treosulfan
- Psychiatric conditions/disease that impair the ability to give informed consent or to adequately co-operate -
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Phase II Trial of Fludarabine Combined With Intravenous Treosulfan and Allogeneic Hematopoietic Stem-cell Transplantation in Patients With Chemo-refractory or Previously Untreated Acute Myeloid Leukemia and Myelodysplastic Syndrome.
The study hypotheses is that the introduction of dose escalated treosulfan, in substitution to busulfan, will reduce toxicity after allogeneic transplantation while improving myeloablation and and disease control in patients with AML and MDS not eligible for standard transplantation.
- Conditions
- Acute Myeloid Leukemia, Myelodysplastic Syndrome
- Being tested
- treosulfan
- Keywords
- acute myeloid leukemia, myelodysplastic syndrome, allogeneic stem cell transplantation, reduced-intensity conditioning, treosulfan
- Medical subject headings
- Leukemia, Myeloid, Acute, Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
treosulfanDrug
12 g/m2 x 3 days
TreosulfanDrug
12 g/m2 x 3
| Study group | Role | What they receive |
|---|---|---|
| 1 | Experimental | treosulfan Receives: Drug: treosulfan |
What is being measured
The primary outcome is the main question the study is designed to answer.
disease-free survival
Measured: 2 years after transplantation
Secondary outcomes (1)
treatment-related mortality, GVHD, relapse, overall survival
Measured: 2 year after transplantation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Jun 2007 (estimated)
- Study startJun 2007
- Primary completionJun 2014
- Study completionJun 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Acute Myeloid 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 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 24 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Dr. Avichai Shimoni MD
- Sponsor type
- Other government
- Responsible party
- Sponsor-investigator, Dr. Avichai Shimoni MD, Sheba Medical Center
- Investigators
- Arnon Nagler, MD, Principal investigator, Chaim Sheba Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00491634
- Sponsor's protocol ID
- SHEBA-07-3116-AN-CTIL
- Status last verified
- Nov 2015
- First submitted
- 25 Jun 2007
- Last updated
- 2 Dec 2015
- Results posted
- No
- Data monitoring committee
- No
Publications
Kroger N, Shimoni A, Zabelina T, Schieder H, Panse J, Ayuk F, Wolschke C, Renges H, Dahlke J, Atanackovic D, Nagler A, Zander A. Reduced-toxicity conditioning with treosulfan, fludarabine and ATG as preparative regimen for allogeneic stem cell transplantation (alloSCT) in elderly patients with secondary acute myeloid leukemia (sAML) or myelodysplastic syndrome (MDS). Bone Marrow Transplant. 2006 Feb;37(4):339-44. doi: 10.1038/sj.bmt.1705259.
PubMed 16415898
Study at a glance
From the registry record, last updated by the sponsor on 2 Dec 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Trial of Fludarabine Combined With Intravenous Treosulfan and Allogeneic Hematopoietic Stem-cell Transplantation in Patients With Chemo-refractory or Previously Untreated Acute Myeloid Leukemia and Myelodysplastic Syndrome.
- ClinicalTrials.gov ID
- NCT00491634
- Acronym / sponsor ID
- SHEBA-07-3116-AN-CTIL
- Conditions
- Acute Myeloid Leukemia, Myelodysplastic Syndrome
- Keywords
- acute myeloid leukemia, myelodysplastic syndrome, allogeneic stem cell transplantation, reduced-intensity conditioning, treosulfan
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (estimated)
- Lead sponsor
- Dr. Avichai Shimoni MDOther government
- Study start
- Jun 2007
- Primary completion
- Jun 2014 (actual)
- Study completion
- Jun 2014 (actual)
- First posted
- 26 Jun 2007 (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 hypotheses is that the introduction of dose escalated treosulfan, in substitution to busulfan, will reduce toxicity after allogeneic transplantation while improving myeloablation and and disease control in patients with AML and MDS not eligible for standard transplantation.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 1 | Experimental | Drug: treosulfantreosulfan |
Who may qualify
- Ages
- 18 to 68 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Age less than physiologic 68 years.
- Patients with AML and MDS not eligible for standard TBI- or Busulfan-based myeloablative conditioning due to age, concurrent medical condition, or extensive prior therapy (e.g. age > 55 years for HLA-matched sibling transplants or > 50 for matched unrelated donor transplants, prior / concomitant pulmonary, liver, or other organ complications).
- This study will only include patients with chemo-refractory disease or previously untreated active disease.
- Patients must have an HLA matched related or unrelated donor willing to donate either peripheral blood stem cells or bone marrow. Matching is based on high-resolution class I (HLA-A, -B, -C) and class II (HLA-DRB1, -DQB1) typing. The goal is to transplant > 3 x 106 CD34+ cells per kg body weight of the recipient -
Key exclusion criteria (11 listed)
- Bilirubin > 3.0 mg/dl, transaminases > 3 times upper normal limit
- Creatinine > 2.0 mg/dl
- ECOG-Performance status > 2
- Uncontrolled infection
- Pregnancy or lactation
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 |
|---|---|---|
| treosulfan12 g/m2 x 3 days | Drug | 1 |
| Treosulfan12 g/m2 x 3 | Drug | 1 |
Primary outcome
disease-free survival
Time frame: 2 years after transplantation
1 secondary outcome
treatment-related mortality, GVHD, relapse, overall survival
Time frame: 2 year after transplantation
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 2 Dec 2015.
- Background: PubMed 16415898Kroger N, Shimoni A, Zabelina T, Schieder H, Panse J, Ayuk F, Wolschke C, Renges H, Dahlke J, Atanackovic D, Nagler A, Zander A. Reduced-toxicity conditioning with treosulfan, fludarabine and ATG as preparative regimen for allogeneic stem cell transplantation (alloSCT) in elderly patients with secondary acute myeloid leukemia (sAML) or myelodysplastic syndrome (MDS). Bone Marrow Transplant. 2006 Feb;37(4):339-44. doi: 10.1038/sj.bmt.1705259.
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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.
Arnon Nagler, MD principal investigator
Chaim Sheba Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing treosulfan in people with Acute Myeloid Leukemia and Myelodysplastic Syndrome, run by Dr. Avichai Shimoni MD at 1 site in Israel.
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 7 inclusion and 11 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 2 Dec 2015. 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 Israel. 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 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?
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?
disease-free survival (time frame: 2 years after transplantation). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00491634, which also lists 1 linked publication.
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 2 Dec 2015.