Hematologic MalignanciesPhase 1NCT00477542
A Phase I Trial of Myeloablative Conditioning w/ Clofarabine and HD Busulfan for Pts w/ Refractory Heme Malignancies Undergoing Allo PBSCT
A Phase 1 study testing clofarabine in people with Hematologic Malignancies, run by Indiana University at 1 site in United States.
Trial summary
This is a phase trial to determine the maximum tolerated dose (MTD) of clofarabine in a combination with a myeloablative dose of busulfan. This is an initial step in developing a novel myeloablative preparative regimen for allogeneic hematopoietic stem cell transplantation (HSCT). Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- Hematologic Malignancies
- What is being tested?
- clofarabine
- How many people
- 18 participants
- Where
- 1 siteUnited States
- Study dates
- May 2007to Nov 2010
- 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 18 to 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Documentation of disease. Patients must have one of the following disease types:
- Acute myeloid leukemia (AML) with either:
- Primary refractory to induction chemotherapy
- Relapsed and refractory AML with >5% blasts in bone marrow or extramedullary disease (excluding active disease of the central nervous system).
- Patients in second or subsequent complete remission (CR2, CR3, etc.).
- Acute lymphoblastic leukemia (ALL) with one of the following criteria:
Show all 30Show fewer
- Primary refractory to induction chemotherapy.
- Relapsed and refractory ALL with >5% blasts in bone marrow or extramedullary disease (excluding active disease of the central nervous system).
- Patients in second or subsequent complete remission (CR2, CR3, etc.).
- Myelodysplasia, refractory anemia with excess blasts with 11-20% blasts in the bone marrow (RAEB II).
- Chronic myelogenous leukemia (CML) with one of the following criteria:
- Accelerated phase.
- Patients in blast crisis.
- Patients with aggressive non-Hodgkin's lymphoma (NHL), including diffuse large cell lymphoma, mediastinal B-cell lymphoma, transformed lymphoma, mantle cell lymphoma, and peripheral T cell lymphoma, who also have one of the following criteria:
- Failure to achieve complete remission to primary induction therapy
- Relapsed NHL, refractory to at least one line of salvage systemic therapy
- Patients who relapse < 6 months following autologous stem cell transplantation are not eligible.
- Patient age 18-60 years
- Availability of a consenting HLA-matched donor
- Performance status ECOG 0-1
- No active infection. Patients with active infections requiring oral or intravenous antibiotics are not eligible for enrollment until resolution of infection.
- No HIV disease. Patients with immune dysfunction are at a significantly higher risk of infection from intensive immunosuppressive therapies.
- Non-pregnant and non-nursing. Treatment under this protocol would expose a fetus to significant risks. Women of childbearing potential should have a negative pregnancy test prior to study entry. Women and men of reproductive potential should agree to use an appropriate method of birth control throughout their participation in this study due to the teratogenic potential of the therapy utilized in this trial. Appropriate methods of birth control include oral contraceptives, implantable hormonal contraceptives (Norplant®), or double barrier method (diaphragm plus condom).
- Required baseline laboratory values:
- LVEF > 45% corrected
- DLCO > 50% of predicted value (corrected for hemoglobin)
- Serum creatinine ≤ 2.0 mg/dl or estimated creatinine clearance of ≥60 ml/min
- Bilirubin < 1 x upper limit of normal value
- AST and ALT < 1 x upper limit of normal value
- Signed written informed consent. Patient must be capable of understanding the investigational nature of the investigational nature, potential risks and benefits of the study, and able to provide valid informed consent.
Who cannot join
- Patients who relapse < 6 months following autologous stem cell transplantation are not eligible
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 I Trial of Myeloablative Conditioning Using Clofarabine and High-Dose Busulfan for Patients With Refractory Hematological Malignancies Undergoing Allogeneic HSCT
This is a phase trial to determine the maximum tolerated dose (MTD) of clofarabine in a combination with a myeloablative dose of busulfan. This is an initial step in developing a novel myeloablative preparative regimen for allogeneic hematopoietic stem cell transplantation (HSCT). While this phase I trial will initially develop the regimen in patients with refractory disease, it is expected that it will find its best application in patients with less advanced disease
Read the detailed description
All patients will receive the same dose of busulfan. The dose of clofarabine will be escalated in successive cohorts of patients. Using a standard dose escalation design, successive cohorts of 3 patients will be treated with escalating doses of clofarabine. At the MTD (or highest dose-level if the MTD is not reached), the cohort will be expanded to 10 patients to better investigate correlative studies and give some preliminary idea of efficacy.
- Conditions
- Hematologic Malignancies
- Being tested
- clofarabine
- Keywords
- Clofarabine, allogeneic HSCT, refractory malignancies
- Medical subject headings
- Hematologic Neoplasms, Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
clofarabineDrug
Cohort- n- Clofarabine (mg/m2/day)
-1 3-10 20
- 3-6 30
- 3-6 40
- 3-6 50
- 3-6 60
- 6-10 70
What is being measured
The primary outcome is the main question the study is designed to answer.
Determine the MTD of clofarabine in association with a myeloablative dose of busulfan as a preparative regimen in patients with refractory hematological malignancies undergoing allogeneic HSCT.
Measured: 1 year
Secondary outcomes (1)
• Assessment of the toxicity of the combination of clofarabine and busulfan • Assess clofarabine and busulfan pharmacokinetics with combination therapy • Describe the response rate • Describe relapse rate and event-free survival
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 May 2007 (estimated)
- Study startMay 2007
- Primary completionNov 2010
- Study completionNov 2010
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hematologic MalignanciesStudies of the same condition, open and under way.
- More trials run by Indiana UniversityThe 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 18 participants (actual)
Sponsor and investigators
- Lead sponsor
- Indiana University
- Sponsor type
- University, hospital or other
- Collaborators
- Genzyme, a Sanofi Company
- Responsible party
- Sponsor
- Investigators
- Sherif Farag, MD, PhD, Principal investigator, Indiana University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00477542
- Sponsor's protocol ID
- 0704-30 IUCRO-0186
- Status last verified
- Sep 2014
- First submitted
- 21 May 2007
- Last updated
- 18 Sep 2014
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 18 Sep 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I Trial of Myeloablative Conditioning Using Clofarabine and High-Dose Busulfan for Patients With Refractory Hematological Malignancies Undergoing Allogeneic HSCT
- ClinicalTrials.gov ID
- NCT00477542
- Acronym / sponsor ID
- 0704-30 IUCRO-0186
- Conditions
- Hematologic Malignancies
- Keywords
- Clofarabine, allogeneic HSCT, refractory malignancies
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 18 participants (actual)
- Lead sponsor
- Indiana UniversityUniversity, hospital or other
- Collaborators
- Genzyme, a Sanofi Company
- Study start
- May 2007
- Primary completion
- Nov 2010 (actual)
- Study completion
- Nov 2010 (actual)
- First posted
- 23 May 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.
This is a phase trial to determine the maximum tolerated dose (MTD) of clofarabine in a combination with a myeloablative dose of busulfan. This is an initial step in developing a novel myeloablative preparative regimen for allogeneic hematopoietic stem cell transplantation (HSCT). While this phase I trial will initially develop the regimen in patients with refractory disease, it is expected that it will find its best application in patients with less advanced disease
Detailed description
All patients will receive the same dose of busulfan. The dose of clofarabine will be escalated in successive cohorts of patients. Using a standard dose escalation design, successive cohorts of 3 patients will be treated with escalating doses of clofarabine. At the MTD (or highest dose-level if the MTD is not reached), the cohort will be expanded to 10 patients to better investigate correlative studies and give some preliminary idea of efficacy.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (30 listed)
- Documentation of disease. Patients must have one of the following disease types:
- Patients who relapse < 6 months following autologous stem cell transplantation are not eligible.
- Patient age 18-60 years
- Availability of a consenting HLA-matched donor
- Performance status ECOG 0-1
Key exclusion criteria (1 listed)
- Patients who relapse < 6 months following autologous stem cell transplantation are not eligible
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 |
|---|---|---|
| clofarabineCohort- n- Clofarabine (mg/m2/day) -1 3-10 20 1. 3-6 30 2. 3-6 40 3. 3-6 50 4. 3-6 60 5. 6-10 70 | Drug | – |
Primary outcome
Determine the MTD of clofarabine in association with a myeloablative dose of busulfan as a preparative regimen in patients with refractory hematological malignancies undergoing allogeneic HSCT.
Time frame: 1 year
1 secondary outcome
• Assessment of the toxicity of the combination of clofarabine and busulfan • Assess clofarabine and busulfan pharmacokinetics with combination therapy • Describe the response rate • Describe relapse rate and event-free survival
Time frame: 1 year
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 18 Sep 2014.
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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 1 study testing clofarabine in people with Hematologic Malignancies, run by Indiana University 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 30 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 18 Sep 2014. 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 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?
Determine the MTD of clofarabine in association with a myeloablative dose of busulfan as a preparative regimen in patients with refractory hematological malignancies undergoing allogeneic HSCT. (time frame: 1 year). 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 18 Sep 2014.