Acute Myeloid LeukemiaPhase 2NCT00593554
Phase 2 Haplotype Mismatched HSCT in Patients With Hematological Malignancies
A Phase 2 study testing Total Body Irradiation, Thiotepa and Fludarabine in people with Acute Myeloid Leukemia and Acute Lymphoblastic Leukemia, run by Sherif S. Farag at 1 site in United States.
Trial summary
The purpose of this study is to determine if haplotype-mismatched HSCT is associated with an improvement in treatment-related mortality (TRM) rate at 6 months. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Myelodysplasia
- What is being tested?
- Total Body Irradiation, Thiotepa and Fludarabine
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 1 siteUnited States
- Study dates
- 7 Aug 2007to 28 Jul 2017
- Phase
- Phase 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 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients must have histologically documented AML, ALL, MDS, CML, Acute myeloid leukemia (AML) with one or more of the following criteria
- CR 1 with poor risk features
- CR 2, or higher order CR
- Acute lymphoblastic leukemia (ALL) with one of the following criteria
- CR 1 with poor risk features
- CR 2, or higher order CR
Show all 10Show fewer
- Myelodysplasia, RAEB I
- Donor has been identified
- Age ≤ 65 years.
- Performance Status 0-1.
Who cannot join
- Patients relapsing <6 months after autologous SCT are not eligible.
- Patients with active infections requiring oral or intravenous antibiotics are not eligible for enrollment until resolution of infection.
- Non-pregnant and non-nursing
Trial locations and contacts
1 site in 1 country.
United States1 site
Indiana University Cancer Center
Indianapolis, Indiana, 46202
About this study
Official title: A Phase II Trial of Haplotype Mismatched Hematopoietic Stem Cell Transplantation Using Highly Purified CD34 Cells in Patients With Hematological Malignancies
The purpose of this study is to determine if haplotype-mismatched HSCT is associated with an improvement in treatment-related mortality (TRM) rate at 6 months.
- Conditions
- Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Myelodysplasia, Chronic Myeloid Leukemia
- Being tested
- Total Body Irradiation, Thiotepa, Fludarabine, Rabbit ATG, Palifermin
- Medical subject headings
- Leukemia, Myeloid, Acute, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Anemia, Refractory, with Excess of Blasts, Leukemia, Myelogenous, Chronic, BCR-ABL Positive
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Total Body IrradiationRadiation
8 Gy on Day -9
ThiotepaDrug
5 mg/kg/d on Day -8 to -7
FludarabineDrug
40 mg/m2/d on Day -6 to -3
Rabbit ATGBiological
Also called Antithymocyte globulin, Thymoglobulin
2.5 mg/kg/d on Day -5 to -2
PaliferminDrug
Also called Recombinant human keratinocyte growth factor, Kepivance
60 ug/kg (actual body weight) on Day -9 to -7 and Day 0 to +2
| Study group | Role | What they receive |
|---|---|---|
| 1 | Active comparator | Total body Irradiation; Thiotepa; Fludarabine; Rabbit ATG; Receives: Radiation: Total Body Irradiation; Drug: Thiotepa; Drug: Fludarabine; Biological: Rabbit ATG |
| 2 | Experimental | Palifermin; Total Body Irradiation; Thiotepa; Fludarabine; Rabbit ATG Receives: Radiation: Total Body Irradiation; Drug: Thiotepa; Drug: Fludarabine; Biological: Rabbit ATG; Drug: Palifermin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Treatment-related Mortality (TRM) Rate at 6 Months After Transplantation
To determine if haplotype-mismatched HSCT is associated with a ≤40% treatment-related mortality (TRM) rate at 6 months after transplantation; a TRM ≥60% being considered unacceptable. The percent of patients with the exact 95% confidence interval who had treatment-related mortality within 6 months of their transplant is presented.
Measured: thru 6 months after transplant
Secondary outcomes (6)
Regimen-related Toxicity
The number of unique patients who had adverse events that were possibly/probably/definitely related to treatment/regimen.
Measured: Up to 1 year
Time to Neutrophil Engraftment
Time to neutrophil engraftment will be analyzed by the Kaplan-Meier method. The time to engraftment of neutrophil is defined as the time from transplant until absolute neutrophil count (ANC) > 500 uL for 3 consecutive days. The median and 95% confidence intervals will be provided.
Measured: Transplant (Day 0) up to 1 year
Time to Platelet Engraftment
Time to platelet engraftment will be analyzed by the Kaplan-Meier method. The time to engraftment of platelets is defined as the time from day 0 to the first of seven consecutive days after transplantation during which the platelet count is at least 20 x109/l without transfusion support. Only patients who achieved engraftment of platelets will be included in the analysis. The median and 95% confidence intervals will be provided.
Measured: Transplant (Day 0) up to 1 year
Acute Graft vs. Host Disease (GvHD)
Number of unique patients who had acute Graft vs. Host Disease (GvHD) diagnosed while on the study.
Measured: Up to 1 year
Chronic Graft vs. Host Disease (GvHD)
Number of unique patients who had chronic Graft vs. Host Disease (GvHD) diagnosed while on the study.
Measured: Up to 1 year
Frequency of Infection
Number of unique patients with bacterial and/or viral infections reported.
Measured: Day 0 through 1 year post transplantation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Jan 2008 (estimated)
- Study start7 Aug 2007
- Primary completion27 Aug 2016
- Study completion28 Jul 2017
Why it stopped: Slow accrual
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 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sherif S. Farag
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Sherif S. Farag, Indiana University
- Investigators
- Sherif Farag, MD/PhD, Principal investigator, Indiana University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00593554
- Sponsor's protocol ID
- 0704-19 IUCRO-0184
- Status last verified
- Mar 2018
- First submitted
- 4 Jan 2008
- Last updated
- 10 Apr 2018
- 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 10 Apr 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Trial of Haplotype Mismatched Hematopoietic Stem Cell Transplantation Using Highly Purified CD34 Cells in Patients With Hematological Malignancies
- ClinicalTrials.gov ID
- NCT00593554
- Acronym / sponsor ID
- 0704-19 IUCRO-0184
- Conditions
- Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Myelodysplasia, Chronic Myeloid Leukemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Slow accrual
- Enrolment
- 9 participants (actual)
- Lead sponsor
- Sherif S. FaragUniversity, hospital or other
- Study start
- 7 Aug 2007 (actual)
- Primary completion
- 27 Aug 2016 (actual)
- Study completion
- 28 Jul 2017 (actual)
- First posted
- 15 Jan 2008 (estimated)
- Results
- Posted 10 Apr 2018
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to determine if haplotype-mismatched HSCT is associated with an improvement in treatment-related mortality (TRM) rate at 6 months.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 1 | Active comparator | Radiation: Total Body Irradiation, Drug: Thiotepa, Drug: Fludarabine, Biological: Rabbit ATGTotal body Irradiation; Thiotepa; Fludarabine; Rabbit ATG; |
| 2 | Experimental | Radiation: Total Body Irradiation, Drug: Thiotepa, Drug: Fludarabine, Biological: Rabbit ATG, Drug: PaliferminPalifermin; Total Body Irradiation; Thiotepa; Fludarabine; Rabbit ATG |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Patients must have histologically documented AML, ALL, MDS, CML, Acute myeloid leukemia (AML) with one or more of the following criteria
- Acute lymphoblastic leukemia (ALL) with one of the following criteria
- Myelodysplasia, RAEB I
- Donor has been identified
- Age ≤ 65 years.
Key exclusion criteria (3 listed)
- Patients relapsing <6 months after autologous SCT are not eligible.
- Patients with active infections requiring oral or intravenous antibiotics are not eligible for enrollment until resolution of infection.
- Non-pregnant and non-nursing
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 |
|---|---|---|
| Total Body Irradiation8 Gy on Day -9 | Radiation | 1, 2 |
| Thiotepa5 mg/kg/d on Day -8 to -7 | Drug | 1, 2 |
| Fludarabine40 mg/m2/d on Day -6 to -3 | Drug | 1, 2 |
| Rabbit ATGAlso: Antithymocyte globulin, Thymoglobulin2.5 mg/kg/d on Day -5 to -2 | Biological | 1, 2 |
| PaliferminAlso: Recombinant human keratinocyte growth factor, Kepivance60 ug/kg (actual body weight) on Day -9 to -7 and Day 0 to +2 | Drug | 2 |
Primary outcome
Treatment-related Mortality (TRM) Rate at 6 Months After Transplantation
Time frame: thru 6 months after transplant
To determine if haplotype-mismatched HSCT is associated with a ≤40% treatment-related mortality (TRM) rate at 6 months after transplantation; a TRM ≥60% being considered unacceptable. The percent of patients with the exact 95% confidence interval who had treatment-related mortality within 6 months of their transplant is presented.
6 secondary outcomes
Regimen-related Toxicity
Time frame: Up to 1 year
Time to Neutrophil Engraftment
Time frame: Transplant (Day 0) up to 1 year
Time to Platelet Engraftment
Time frame: Transplant (Day 0) up to 1 year
Acute Graft vs. Host Disease (GvHD)
Time frame: Up to 1 year
Chronic Graft vs. Host Disease (GvHD)
Time frame: Up to 1 year
Frequency of Infection
Time frame: Day 0 through 1 year post transplantation
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Apr 2018.
- 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.
Sherif Farag, MD/PhD principal investigator
Indiana University
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 Total Body Irradiation, Thiotepa and Fludarabine in people with Acute Myeloid Leukemia and Acute Lymphoblastic Leukemia, run by Sherif S. Farag 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 10 inclusion and 3 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 10 Apr 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 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 10 Apr 2018). 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?
Treatment-related Mortality (TRM) Rate at 6 Months After Transplantation (time frame: thru 6 months 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 10 Apr 2018.