LeukemiaPhase 1/2NCT00943319

Busulfan-fludarabine Conditioning and T-cell Depleted Allogeneic Stem Cell Transplantation for Patients With Advanced Hematologic Malignancies

A Phase 1/2 study testing Busulfan, Fludarabine and Campath in people with Leukemia and Lymphoma, run by University of Chicago at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

The purpose of this study is: 1. To establish the maximally tolerated dose (MTD) of intravenous busulfan (Busulfan®) in combination with fludarabine as conditioning regimen for transplantation with in-vivo T-cell depletion. 2. Read more about this study

Who is it for?: Ages 18 years and older
Conditions the study is for: Leukemia, Lymphoma, Myeloma
What is being tested?: Busulfan, Fludarabine and Campath
Type of treatment: Injection or infusion, Surgery or procedure
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 50 participants
Where: 1 siteUnited States
Study dates: Mar 2012to Dec 2018
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 18 years and older
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

Phase I portion:
Relapsed or refractory acute myelogenous or lymphoid leukemia.
Chronic myelogenous leukemia in accelerated phase or blast-crisis.
Recurrent or refractory malignant lymphoma or Hodgkin's disease
Recurrent or refractory multiple myeloma.
Chronic lymphocytic leukemia, relapsed or with poor prognostic features.

Show all 11Show fewer

Myeloproliferative disorder (polycythemia vera, myelofibrosis) with transformation
Myelodysplastic syndromes with more than 5% blasts.
Phase II portion:
AML with active disease or beyond CR2.
MDS with more than 5% blasts.

Who cannot join

Clinical progression. Such patients may be treated on other treatment protocols or at the investigator's discretion. Such patients will continue to be monitored for survival and, may be asked to continue to provide specimens for studies of minimal residual disease and immune reconstitution as other treatments are recommended.

Trial locations and contacts

1 site in 1 country.

United States1 site

The University of Chicago
Chicago, Illinois, 60637

Other trials in United States

About this study

Official title: A Phase I-II Study of Busulfan-fludarabine Conditioning and T-cell Depleted Allogeneic Stem Cell Transplantation for Patients With Advanced Hematologic Malignancies

The purpose of this study is:

To establish the maximally tolerated dose (MTD) of intravenous busulfan (Busulfan®) in combination with fludarabine as conditioning regimen for transplantation with in-vivo T-cell depletion.
To evaluate disease free and overall survival after this conditioning regimen in patients with advanced acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS).
To evaluate potential pharmacogenomic determinants of toxicity of this regimen.
To evaluate potential pharmacogenomic determinants of efficacy of this regimen.

Conditions: Leukemia, Lymphoma, Myeloma
Being tested: Busulfan, Fludarabine, Campath, Stem Cell Transplant
Keywords: Cancers of the blood
Medical subject headings: Leukemia, Lymphoma, Neoplasms, Plasma Cell

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

BusulfanDrug
Daily intravenous dosing to target AVC
FludarabineDrug
Fludarabine dosing will be based on actual body weight. Fludarabine will be infused over 30 minutes before busulfan treatment dose.
CampathDrug
All patients will receive premedication for Campath (daily doses of 20 mg are repeated for up to five times).
Stem Cell TransplantProcedure or surgery
Infusion of bone marrow and donors(related/ unrelated).

Study groups in NCT00943319
Study group	Role	What they receive
Busulfan and fludarabine	Experimental	Intravenous busulfan (Busulfan®) in combination with fludarabine Receives: Drug: Busulfan; Drug: Fludarabine; Drug: Campath; Procedure: Stem Cell Transplant

What is being measured

The primary outcome is the main question the study is designed to answer.

Maximum Tolerated Dose
Maximally tolerated area under the curve of intravenous busulfan (Busulfan®) in combination with fludarabine as conditioning regimen for transplantation with in-vivo T-cell depletion. The number reported will be an Area Under the Curve (AUC) measure reported in µmol-min/L.
Measured: 5 years

Secondary outcomes (2)

Overall Survival
Overall Survival measured as median survival in days
Measured: 5 years
Disease Free Survival
Disease Free Survival measured by median survival time in days
Measured: 5 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Mar 2012Dec 2018

First posted22 Jul 2009 (estimated)
Study startMar 2012
Primary completionDec 2017
Study completionDec 2018

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.
Publications (1)Papers the sponsor has linked to this study.
Other trials for LeukemiaStudies of the same condition, open and under way.
More trials run by University of ChicagoThe 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: 50 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00943319
Sponsor's protocol ID: 12-0132
Status last verified: Feb 2019
First submitted: 20 Jul 2009
Last updated: 4 Feb 2019
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes

Publications

O'Donnell PH, Artz AS, Undevia SD, Pai RK, Del Cerro P, Horowitz S, Godley LA, Hart J, Innocenti F, Larson RA, Odenike OM, Stock W, Van Besien K. Phase I study of dose-escalated busulfan with fludarabine and alemtuzumab as conditioning for allogeneic hematopoietic stem cell transplant: reduced clearance at high doses and occurrence of late sinusoidal obstruction syndrome/veno-occlusive disease. Leuk Lymphoma. 2010 Dec;51(12):2240-9. doi: 10.3109/10428194.2010.520773. Epub 2010 Oct 4.
PubMed 20919852

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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Study at a glance

From the registry record, last updated by the sponsor on 4 Feb 2019. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase I-II Study of Busulfan-fludarabine Conditioning and T-cell Depleted Allogeneic Stem Cell Transplantation for Patients With Advanced Hematologic Malignancies
ClinicalTrials.gov ID: NCT00943319
Acronym / sponsor ID: 12-0132
Conditions: Leukemia, Lymphoma, Myeloma
Keywords: Cancers of the blood
Phase: Phase 1/2
Study type: Interventional
Recruitment status: Completed
Enrolment: 50 participants (actual)
Lead sponsor: University of ChicagoUniversity, hospital or other
Study start: Mar 2012
Primary completion: Dec 2017 (actual)
Study completion: Dec 2018 (actual)
First posted: 22 Jul 2009 (estimated)
Results: Posted 4 Feb 2019

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 establish the maximally tolerated dose (MTD) of intravenous busulfan (Busulfan®) in combination with fludarabine as conditioning regimen for transplantation with in-vivo T-cell depletion.
To evaluate disease free and overall survival after this conditioning regimen in patients with advanced acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS).
To evaluate potential pharmacogenomic determinants of toxicity of this regimen.
To evaluate potential pharmacogenomic determinants of efficacy of this regimen.

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Busulfan and fludarabine	Experimental	Drug: Busulfan, Drug: Fludarabine, Drug: Campath, Procedure: Stem Cell TransplantIntravenous busulfan (Busulfan®) in combination with fludarabine

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (11 listed)

Relapsed or refractory acute myelogenous or lymphoid leukemia.
Chronic myelogenous leukemia in accelerated phase or blast-crisis.
Recurrent or refractory malignant lymphoma or Hodgkin's disease
Recurrent or refractory multiple myeloma.
Chronic lymphocytic leukemia, relapsed or with poor prognostic features.

Key exclusion criteria (1 listed)

Clinical progression. Such patients may be treated on other treatment protocols or at the investigator's discretion. Such patients will continue to be monitored for survival and, may be asked to continue to provide specimens for studies of minimal residual disease and immune reconstitution as other treatments are recommended.

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
BusulfanDaily intravenous dosing to target AVC	Drug	Busulfan and fludarabine
FludarabineFludarabine dosing will be based on actual body weight. Fludarabine will be infused over 30 minutes before busulfan treatment dose.	Drug	Busulfan and fludarabine
CampathAll patients will receive premedication for Campath (daily doses of 20 mg are repeated for up to five times).	Drug	Busulfan and fludarabine
Stem Cell TransplantInfusion of bone marrow and donors(related/ unrelated).	Procedure or surgery	Busulfan and fludarabine

Primary outcome

Maximum Tolerated Dose
Time frame: 5 years
Maximally tolerated area under the curve of intravenous busulfan (Busulfan®) in combination with fludarabine as conditioning regimen for transplantation with in-vivo T-cell depletion. The number reported will be an Area Under the Curve (AUC) measure reported in µmol-min/L.

2 secondary outcomes

Overall Survival
Time frame: 5 years
Disease Free Survival
Time frame: 5 years

Results and safety

Results posted to the registry on 4 Feb 2019, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Busulfan and Fludarabine	50	49

Primary outcome: Maximum Tolerated Dose

Time frame: 5 years. Number. Unit: mmol-min/L

Measure	Busulfan and Fludarabine n = 36
Value	6800

Population analysed: The 36 patients refer to the phase I portion of the study only.

2 secondary outcomes are reported in the full results.

Adverse events

Time frame: 10 years. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Busulfan and Fludarabine	1 of 49 (2%)	1 of 49 (2%)	45 of 49

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 4 Feb 2019.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 20919852O'Donnell PH, Artz AS, Undevia SD, Pai RK, Del Cerro P, Horowitz S, Godley LA, Hart J, Innocenti F, Larson RA, Odenike OM, Stock W, Van Besien K. Phase I study of dose-escalated busulfan with fludarabine and alemtuzumab as conditioning for allogeneic hematopoietic stem cell transplant: reduced clearance at high doses and occurrence of late sinusoidal obstruction syndrome/veno-occlusive disease. Leuk Lymphoma. 2010 Dec;51(12):2240-9. doi: 10.3109/10428194.2010.520773. Epub 2010 Oct 4.

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Enrolling by invitationObservational study
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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.

Andrew Artz, MD principal investigator

University of Chicago

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 Busulfan, Fludarabine and Campath in people with Leukemia and Lymphoma, run by University of Chicago 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 4 Feb 2019. 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 4 Feb 2019). 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?

Maximum Tolerated Dose (time frame: 5 years). 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 NCT00943319, 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 4 Feb 2019.