Acute Myeloid LeukemiaPhase 1NCT01398501
Sorafenib Maintenance Therapy for Patients With AML After Allogeneic Stem Cell Transplant
A Phase 1 study testing Sorafenib in people with Acute Myeloid Leukemia, run by Massachusetts General Hospital at 2 sites in United States.
Trial summary
Sorfenib works by slowing the spread of cancer cells. It has been used in other studies for patients with AML with the FLT3-ITD mutation and information from these studies suggests that sorafenib may help to control leukemia. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Acute Myeloid Leukemia
- What is being tested?
- Sorafenib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 22 participants
- Where
- 2 sitesUnited States
- Study dates
- Aug 2011to Aug 2016
- 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 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Subjects with AML with the FLT3-ITD mutation who have undergone allogeneic HSCT
- Peripheral blood chimerism studies showing >/= 70% of all cells are of donor origin
- Adequate hematologic and hepatic function
- ECOG performance status 0-2
- Able to swallow whole pills
Who cannot join
- Evidence of relapsed/recurrent/residual disease as assessed by bone marrow aspirate and biopsy performed between days 30-60 after HSCT
- Active acute graft vs host disease requiring an equivalent dose of > 0.5 mg/kg/day of prednisone or equivalent or those patients which necessitated the addition of another agent for the treatment of GVHD beyond corticosteroids
- Ongoing uncontrolled infection
- Cardiac disease: congestive heart failure > class II NYHA, unstable angina or new onset angina (began within the last 3 months) or myocardial infarction within the past 6 months
- Cardiac ventricular arrhythmias requiring anti-arrhythmic therapy
- Uncontrolled hypertension
Show all 17Show fewer
- Known HIV infection or chronic hepatitis B or C
- Thrombotic or embolic events such as cerebrovascular accident including transient ischemic attacks within the past 6 months
- Pulmonary hemorrhage/bleeding event > CTCAE v 4.0 Grade 2 within 4 weeks of starting study drug
- Any other hemorrhage/bleeding event > CTCAE v. 4.0 Grade 3 within 4 weeks of starting study drug
- Serious non-healing wound, non-healing ulcer, or bone fracture
- Evidence or history of bleeding diathesis or coagulopathy
- Major surgery or significant traumatic injury within 4 weeks of starting study drug
- Use of St. John's Wort or rifampin (rifampicin)
- Known or suspected allergy to sorafenib
- Pregnant or breast-feeding
- Receiving any other investigational agents
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Massachusetts General Hospital
Boston, Massachusetts, 02114
Dana-Farber Cancer Institute
Boston, Massachusetts, 02214
About this study
Official title: Phase I Trial of Sorafenib Maintenance Therapy for Patients With FLT3-ITD AML After Allogeneic Stem Cell Transplantation
Sorfenib works by slowing the spread of cancer cells. It has been used in other studies for patients with AML with the FLT3-ITD mutation and information from these studies suggests that sorafenib may help to control leukemia. The purpose of this study is to find the highest dose of sorafenib for maintenance therapy that can be safely used in participants with AML who have undergone allogeneic stem cell transplant.
Read the detailed description
Subjects will taken sorafenib orally either once or twice daily. Subjects will come to the Bone Marrow Transplant Clinic 3 times (on Day 8, 15, and 30) during the first month of treatment. After the first month, they will be seen every month for 3 months and then at 9 at 6 and 9 months. Subjects will have a physical exam and be asked questions regarding general health and specific questions about any problems they might be having and any medications they are taking.
Subjects will have standard blood tests every month for 12 months to check liver and kidney function and complete blood count.
Subjects will have research blood tests on Days 8, 15 and 30 during the first month of treatment.
Subjects will have a bone marrow biopsy after 3 months and 12 months of treatment.
Subjects will receive treatment for up to 12 months and be followed for 1 year after completing the study.
- Conditions
- Acute Myeloid Leukemia
- Being tested
- Sorafenib
- Keywords
- AML, FLT3-ITD, Stem cell transplant
- Medical subject headings
- Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SorafenibDrug
Also called BAY 43-9006
Oral, 200 to 400 mg QD or BID
| Study group | Role | What they receive |
|---|---|---|
| Post-SCT Sorafenib | Experimental | Sorafenib will be given as maintenance therapy after allo HCT to patients with FLT3-ITD AML. Receives: Drug: Sorafenib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum Tolerated Dose
To define the maximum tolerated dose (MTD) of maintenance sorafenib after allogeneic HSCT
Measured: 3 years
Secondary outcomes (6)
Median number of days sorafenib tolerated
Define the median number of days of sorafenib tolerated prior to dose-limiting toxicity or disease relapse
Measured: 3 years
Rate of serious infections
Rate of serious infections (bacterial, viral, fungal, or other) after starting sorafenib
Measured: 3 years
Rate of acute GVHD
Rate of grades II-IV acute graft-vs-host disease (GVHD) after starting sorafenib
Measured: 3 years
Rate of chronic GVHD
Rates of significant chronic GVHD after starting sorafenib
Measured: 3 years
Survival
1-year and 2-year progression-free and overall survival after HSCT
Measured: 3 years
Impact of sorafenib on bone marrow and serum levels of FLT3-ITD quantitative PCR
To assess the impact of sorafenib on quantitative bone marrow and serum levels of FLT3-ITD DNA in patients (as measured by PCR) with FLT3-ITD AML after allogeneic SCT
Measured: 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Jul 2011 (estimated)
- Study startAug 2011
- Primary completionSep 2015
- Study completionAug 2016
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.
- More trials run by Massachusetts General HospitalThe 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
- 22 participants (actual)
Sponsor and investigators
- Lead sponsor
- Massachusetts General Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Dana-Farber Cancer Institute
- Responsible party
- Principal investigator, Yi-Bin A. Chen, MD, Massachusetts General Hospital
- Investigators
- Yi-Bin Chen, MD, Principal investigator, Massachusetts General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01398501
- Sponsor's protocol ID
- 11-114
- Status last verified
- Mar 2017
- First submitted
- 19 Jul 2011
- Last updated
- 22 Mar 2017
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Chen YB, Li S, Lane AA, Connolly C, Del Rio C, Valles B, Curtis M, Ballen K, Cutler C, Dey BR, El-Jawahri A, Fathi AT, Ho VT, Joyce A, McAfee S, Rudek M, Rajkhowa T, Verselis S, Antin JH, Spitzer TR, Levis M, Soiffer R. Phase I trial of maintenance sorafenib after allogeneic hematopoietic stem cell transplantation for fms-like tyrosine kinase 3 internal tandem duplication acute myeloid leukemia. Biol Blood Marrow Transplant. 2014 Dec;20(12):2042-8. doi: 10.1016/j.bbmt.2014.09.007. Epub 2014 Sep 17.
PubMed 25239228
Study at a glance
From the registry record, last updated by the sponsor on 22 Mar 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I Trial of Sorafenib Maintenance Therapy for Patients With FLT3-ITD AML After Allogeneic Stem Cell Transplantation
- ClinicalTrials.gov ID
- NCT01398501
- Acronym / sponsor ID
- 11-114
- Conditions
- Acute Myeloid Leukemia
- Keywords
- AML, FLT3-ITD, Stem cell transplant
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 22 participants (actual)
- Lead sponsor
- Massachusetts General HospitalUniversity, hospital or other
- Collaborators
- Dana-Farber Cancer Institute
- Study start
- Aug 2011
- Primary completion
- Sep 2015 (actual)
- Study completion
- Aug 2016 (actual)
- First posted
- 20 Jul 2011 (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.
Sorfenib works by slowing the spread of cancer cells. It has been used in other studies for patients with AML with the FLT3-ITD mutation and information from these studies suggests that sorafenib may help to control leukemia. The purpose of this study is to find the highest dose of sorafenib for maintenance therapy that can be safely used in participants with AML who have undergone allogeneic stem cell transplant.
Detailed description
Subjects will taken sorafenib orally either once or twice daily. Subjects will come to the Bone Marrow Transplant Clinic 3 times (on Day 8, 15, and 30) during the first month of treatment. After the first month, they will be seen every month for 3 months and then at 9 at 6 and 9 months. Subjects will have a physical exam and be asked questions regarding general health and specific questions about any problems they might be having and any medications they are taking.
Subjects will have standard blood tests every month for 12 months to check liver and kidney function and complete blood count.
Subjects will have research blood tests on Days 8, 15 and 30 during the first month of treatment.
Subjects will have a bone marrow biopsy after 3 months and 12 months of treatment.
Subjects will receive treatment for up to 12 months and be followed for 1 year after completing the study.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Post-SCT Sorafenib | Experimental | Drug: SorafenibSorafenib will be given as maintenance therapy after allo HCT to patients with FLT3-ITD AML. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Subjects with AML with the FLT3-ITD mutation who have undergone allogeneic HSCT
- Peripheral blood chimerism studies showing >/= 70% of all cells are of donor origin
- Adequate hematologic and hepatic function
- ECOG performance status 0-2
- Able to swallow whole pills
Key exclusion criteria (17 listed)
- Evidence of relapsed/recurrent/residual disease as assessed by bone marrow aspirate and biopsy performed between days 30-60 after HSCT
- Active acute graft vs host disease requiring an equivalent dose of > 0.5 mg/kg/day of prednisone or equivalent or those patients which necessitated the addition of another agent for the treatment of GVHD beyond corticosteroids
- Ongoing uncontrolled infection
- Cardiac disease: congestive heart failure > class II NYHA, unstable angina or new onset angina (began within the last 3 months) or myocardial infarction within the past 6 months
- Cardiac ventricular arrhythmias requiring anti-arrhythmic therapy
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 |
|---|---|---|
| SorafenibAlso: BAY 43-9006Oral, 200 to 400 mg QD or BID | Drug | Post-SCT Sorafenib |
Primary outcome
Maximum Tolerated Dose
Time frame: 3 years
To define the maximum tolerated dose (MTD) of maintenance sorafenib after allogeneic HSCT
6 secondary outcomes
Median number of days sorafenib tolerated
Time frame: 3 years
Rate of serious infections
Time frame: 3 years
Rate of acute GVHD
Time frame: 3 years
Rate of chronic GVHD
Time frame: 3 years
Survival
Time frame: 3 years
Impact of sorafenib on bone marrow and serum levels of FLT3-ITD quantitative PCR
Time frame: 3 years
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 22 Mar 2017.
- Results publication: PubMed 25239228Chen YB, Li S, Lane AA, Connolly C, Del Rio C, Valles B, Curtis M, Ballen K, Cutler C, Dey BR, El-Jawahri A, Fathi AT, Ho VT, Joyce A, McAfee S, Rudek M, Rajkhowa T, Verselis S, Antin JH, Spitzer TR, Levis M, Soiffer R. Phase I trial of maintenance sorafenib after allogeneic hematopoietic stem cell transplantation for fms-like tyrosine kinase 3 internal tandem duplication acute myeloid leukemia. Biol Blood Marrow Transplant. 2014 Dec;20(12):2042-8. doi: 10.1016/j.bbmt.2014.09.007. Epub 2014 Sep 17.
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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.
Yi-Bin Chen, MD principal investigator
Massachusetts General Hospital
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Sorafenib in people with Acute Myeloid Leukemia, run by Massachusetts General Hospital at 2 sites 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 5 inclusion and 17 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 22 Mar 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites 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?
Maximum Tolerated Dose (time frame: 3 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 NCT01398501, 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 22 Mar 2017.