Myelodysplastic Syndrome RAEB-I or RAEB-IINCT00426205
GM-CSF Vaccinations After Allogeneic Blood Stem Cell Transplantation in Patients With Advanced Myeloid Malignancies
A study testing GM-CSF secreting leukemia vaccine in people with Myelodysplastic Syndrome RAEB-I or RAEB-II and Refractory Acute Myeloid Leukemia, run by Dana-Farber Cancer Institute at 1 site in United States.
Trial summary
The purpose of this study is to investigate whether the addition of a vaccine after participants reduced intensity transplant will be safe and beneficial. The vaccine used in this trial, called GVAX, will be made from the participants own leukemia cells, and will be given between 1-4 months after transplant. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelodysplastic Syndrome RAEB-I or RAEB-II, Refractory Acute Myeloid Leukemia, Refractory CML Myeloid Blast Crisis
- What is being tested?
- GM-CSF secreting leukemia vaccine
- Type of treatment
- Vaccines, Injection or infusion
- How many people
- 28 participants
- Where
- 1 siteUnited States
- Study dates
- Jun 2004to Sep 2009
- Phase
- Interventional
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- AML, MDS-RAEB or RAEB-T, CML, myeloid blast crisis not in remission or CML accelerated phase. Subjects must have > 5% blasts in bone marrow or peripheral blood prior to admission for transplant.
- HLA 6/6 matched related or unrelated donor available
- ECOG Performance Statue 0-2
- 18 years of age or older
Who cannot join
- Uncontrolled infection
- Leukemia with active CNS involvement
- Serum creatinine greater than 2.0 mg/dl
- ALT or AST greater than 3 x ULN
- Total bilirubin greater than 2.0 mg/dl
- Positive HIV or HTLV-1 serology
- Prior allogeneic stem cell transplant
Trial locations and contacts
1 site in 1 country.
United States1 site
Dana-Farber Cancer Institute
Boston, Massachusetts, 02115
About this study
Official title: GM-CSF Secreting Leukemia Cell Vaccinations After Allogeneic Non-myeloablative Peripheral Blood Stem Cell Transplantation in Patients With Advanced Myelodysplastic Syndrome or Refractory Acute or Advanced Chronic Myeloid Leukemia
The purpose of this study is to investigate whether the addition of a vaccine after participants reduced intensity transplant will be safe and beneficial. The vaccine used in this trial, called GVAX, will be made from the participants own leukemia cells, and will be given between 1-4 months after transplant. In recent years, researchers have discovered that GVAX vaccine made from the patient's own cancer calls that have been engineered in the laboratory to produce a protein called GM-CSF, can be effective in stimulating a powerful immune response specific to that cancer.
Read the detailed description
- This trial can be divided into three phases: 1) Pre-transplant phase; 2) Reduced intensity transplant phase; 3) Vaccination phase.
- Pre-transplant phase: Once a suitable donor has been identified, the participant will undergo a battery of standard pre-transplant tests and procedure to collect their leukemia cells for vaccine generation. Blood tests, heart function test, pulmonary function test, tuberculosis test, bone marrow aspirate and biopsy, and leukemia cell collection through leukapheresis.
- Allogeneic reduced intensity stem cell transplant phase: The transplant phase of the study will begin when the participant is admitted to the hospital to receive the chemotherapy and stem cell transplant. The minimum duration of hospitalization for the procedure is approximately 8 days. In the week before the participant receives the stem cells, they will be treated with chemotherapy through a central line. The goal of chemotherapy is to both control the cancer and suppress the immune system so that the body will not reject the donor stem cells.
- Just prior to and immediately following the infusion of stem cells, participants will receive medications to help prevent graft-versus-host disease (GVHD), a common complication of transplant where the donor's immune cells attack the body. After the transplant, participants will also take antibiotic medication to help prevent possible infections.
- Sargramostim (GM-CSF, leukine), a white blood cell growth factor, will be given daily subcutaneously starting the day after the stem cell transplant until blood counts have recovered.
- After the stem cell infusion, participants will be examined and have blood tests weekly for 1 month. Between 30-45 days after the transplant, a bone marrow biopsy will be performed to assess the status of the disease and to look for evidence of the donor's cells in the bone marrow.
- Vaccination Phase: After the bone marrow biopsy 30-45 days after the transplant, the participant will begin to receive the vaccinations. The vaccine will be administered subcutaneously and intradermally on the arm, leg, or abdomen 6 times over a period of 9 weeks. The first 3 vaccinations will occur once a week for 3 consecutive weeks, and the last 3 vaccines will be given once every other week over 6 weeks. All vaccinations may be given as an outpatient in the clinic. During this period of time, participants will be closely monitored on a weekly basis to monitor for side effects. Before the first and after the fifth and sixth vaccinations, a small amount of the participants leukemia cells will be injected under the skin to see if the immune system will react against it and cause redness and swelling.
- About 4 weeks after the last vaccination (6th), a bone marrow aspirate and biopsy will be performed to assess the status of the disease.
- After the 1st and 5th vaccinations, a skin biopsy will be performed to assess for response at the vaccine site. These biopsies are relatively simple outpatient procedures.
- Conditions
- Myelodysplastic Syndrome RAEB-I or RAEB-II, Refractory Acute Myeloid Leukemia, Refractory CML Myeloid Blast Crisis
- Being tested
- GM-CSF secreting leukemia vaccine
- Keywords
- GM-CSF, GVAX, Cancer vaccine, blood stem cell transplantation, MDS, AML, CML
- 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.
GM-CSF secreting leukemia vaccineBiological
Vaccine given subcutaneously or intradermally on the leg, arm, or abdomen 6 times. The first three vaccines will be given once a week for three weeks. The last three vaccines will be given once every other week for three doses.
What is being measured
The primary outcome is the main question the study is designed to answer.
Feasibility as measured by ability to generate sufficient vaccine, and ability for this patient population to initiate vaccination between day 30 to day 45 after transplant.
Measured: 3 years
Secondary outcomes (3)
Safety of GVAX vaccination as measured by grade III-IV acute GVHD, and CTC grade 3 or higher non-hematologic toxicity
Measured: 3 years
biologic activity of GVAX vaccination
Measured: 3 years
disease free and overall survival.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Jan 2007 (estimated)
- Study startJun 2004
- Primary completionSep 2007
- Study completionSep 2009
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 Myelodysplastic Syndrome RAEB-I or RAEB-IIStudies of the same condition, open and under way.
- More trials run by Dana-Farber Cancer InstituteThe 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 N/A
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 28 participants (actual)
Sponsor and investigators
- Lead sponsor
- Dana-Farber Cancer Institute
- Sponsor type
- University, hospital or other
- Collaborators
- Brigham and Women's Hospital
- Responsible party
- Principal investigator, Vincent T. Ho, MD, Dana-Farber Cancer Institute
- Investigators
- Vincent Ho, MD, Principal investigator, Dana-Farber Cancer Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00426205
- Sponsor's protocol ID
- 04-023
- Status last verified
- Jan 2022
- First submitted
- 23 Jan 2007
- Last updated
- 11 Feb 2022
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Ho VT, Vanneman M, Kim H, Sasada T, Kang YJ, Pasek M, Cutler C, Koreth J, Alyea E, Sarantopoulos S, Antin JH, Ritz J, Canning C, Kutok J, Mihm MC, Dranoff G, Soiffer R. Biologic activity of irradiated, autologous, GM-CSF-secreting leukemia cell vaccines early after allogeneic stem cell transplantation. Proc Natl Acad Sci U S A. 2009 Sep 15;106(37):15825-30. doi: 10.1073/pnas.0908358106. Epub 2009 Aug 26.
PubMed 19717467
Study at a glance
From the registry record, last updated by the sponsor on 11 Feb 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- GM-CSF Secreting Leukemia Cell Vaccinations After Allogeneic Non-myeloablative Peripheral Blood Stem Cell Transplantation in Patients With Advanced Myelodysplastic Syndrome or Refractory Acute or Advanced Chronic Myeloid Leukemia
- ClinicalTrials.gov ID
- NCT00426205
- Acronym / sponsor ID
- 04-023
- Conditions
- Myelodysplastic Syndrome RAEB-I or RAEB-II, Refractory Acute Myeloid Leukemia, Refractory CML Myeloid Blast Crisis
- Keywords
- GM-CSF, GVAX, Cancer vaccine, blood stem cell transplantation, MDS, AML, CML
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 28 participants (actual)
- Lead sponsor
- Dana-Farber Cancer InstituteUniversity, hospital or other
- Collaborators
- Brigham and Women's Hospital
- Study start
- Jun 2004
- Primary completion
- Sep 2007 (actual)
- Study completion
- Sep 2009 (actual)
- First posted
- 24 Jan 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 purpose of this study is to investigate whether the addition of a vaccine after participants reduced intensity transplant will be safe and beneficial. The vaccine used in this trial, called GVAX, will be made from the participants own leukemia cells, and will be given between 1-4 months after transplant. In recent years, researchers have discovered that GVAX vaccine made from the patient's own cancer calls that have been engineered in the laboratory to produce a protein called GM-CSF, can be effective in stimulating a powerful immune response specific to that cancer.
Detailed description
- This trial can be divided into three phases: 1) Pre-transplant phase; 2) Reduced intensity transplant phase; 3) Vaccination phase.
- Pre-transplant phase: Once a suitable donor has been identified, the participant will undergo a battery of standard pre-transplant tests and procedure to collect their leukemia cells for vaccine generation. Blood tests, heart function test, pulmonary function test, tuberculosis test, bone marrow aspirate and biopsy, and leukemia cell collection through leukapheresis.
- Allogeneic reduced intensity stem cell transplant phase: The transplant phase of the study will begin when the participant is admitted to the hospital to receive the chemotherapy and stem cell transplant. The minimum duration of hospitalization for the procedure is approximately 8 days. In the week before the participant receives the stem cells, they will be treated with chemotherapy through a central line. The goal of chemotherapy is to both control the cancer and suppress the immune system so that the body will not reject the donor stem cells.
- Just prior to and immediately following the infusion of stem cells, participants will receive medications to help prevent graft-versus-host disease (GVHD), a common complication of transplant where the donor's immune cells attack the body. After the transplant, participants will also take antibiotic medication to help prevent possible infections.
- Sargramostim (GM-CSF, leukine), a white blood cell growth factor, will be given daily subcutaneously starting the day after the stem cell transplant until blood counts have recovered.
- After the stem cell infusion, participants will be examined and have blood tests weekly for 1 month. Between 30-45 days after the transplant, a bone marrow biopsy will be performed to assess the status of the disease and to look for evidence of the donor's cells in the bone marrow.
- Vaccination Phase: After the bone marrow biopsy 30-45 days after the transplant, the participant will begin to receive the vaccinations. The vaccine will be administered subcutaneously and intradermally on the arm, leg, or abdomen 6 times over a period of 9 weeks. The first 3 vaccinations will occur once a week for 3 consecutive weeks, and the last 3 vaccines will be given once every other week over 6 weeks. All vaccinations may be given as an outpatient in the clinic. During this period of time, participants will be closely monitored on a weekly basis to monitor for side effects. Before the first and after the fifth and sixth vaccinations, a small amount of the participants leukemia cells will be injected under the skin to see if the immune system will react against it and cause redness and swelling.
- About 4 weeks after the last vaccination (6th), a bone marrow aspirate and biopsy will be performed to assess the status of the disease.
- After the 1st and 5th vaccinations, a skin biopsy will be performed to assess for response at the vaccine site. These biopsies are relatively simple outpatient procedures.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- AML, MDS-RAEB or RAEB-T, CML, myeloid blast crisis not in remission or CML accelerated phase. Subjects must have > 5% blasts in bone marrow or peripheral blood prior to admission for transplant.
- HLA 6/6 matched related or unrelated donor available
- ECOG Performance Statue 0-2
- 18 years of age or older
Key exclusion criteria (7 listed)
- Uncontrolled infection
- Leukemia with active CNS involvement
- Serum creatinine greater than 2.0 mg/dl
- ALT or AST greater than 3 x ULN
- Total bilirubin greater than 2.0 mg/dl
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 |
|---|---|---|
| GM-CSF secreting leukemia vaccineVaccine given subcutaneously or intradermally on the leg, arm, or abdomen 6 times. The first three vaccines will be given once a week for three weeks. The last three vaccines will be given once every other week for three doses. | Biological | – |
Primary outcome
Feasibility as measured by ability to generate sufficient vaccine, and ability for this patient population to initiate vaccination between day 30 to day 45 after transplant.
Time frame: 3 years
3 secondary outcomes
Safety of GVAX vaccination as measured by grade III-IV acute GVHD, and CTC grade 3 or higher non-hematologic toxicity
Time frame: 3 years
biologic activity of GVAX vaccination
Time frame: 3 years
disease free and overall survival.
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 11 Feb 2022.
- Background: PubMed 19717467Ho VT, Vanneman M, Kim H, Sasada T, Kang YJ, Pasek M, Cutler C, Koreth J, Alyea E, Sarantopoulos S, Antin JH, Ritz J, Canning C, Kutok J, Mihm MC, Dranoff G, Soiffer R. Biologic activity of irradiated, autologous, GM-CSF-secreting leukemia cell vaccines early after allogeneic stem cell transplantation. Proc Natl Acad Sci U S A. 2009 Sep 15;106(37):15825-30. doi: 10.1073/pnas.0908358106. Epub 2009 Aug 26.
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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.
Vincent Ho, MD principal investigator
Dana-Farber Cancer Institute
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing GM-CSF secreting leukemia vaccine in people with Myelodysplastic Syndrome RAEB-I or RAEB-II and Refractory Acute Myeloid Leukemia, run by Dana-Farber Cancer Institute 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 4 inclusion and 7 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 11 Feb 2022. 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.
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?
Feasibility as measured by ability to generate sufficient vaccine, and ability for this patient population to initiate vaccination between day 30 to day 45 after transplant. (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 NCT00426205, 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 11 Feb 2022.