Myelodysplastic SyndromesEarly Phase 1NCT00361296
Vaccine Therapy in Treating Patients With Myelodysplastic Syndromes
A Early Phase 1 study testing K562/GM-CSF cell vaccine in people with Myelodysplastic Syndromes, run by Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins at 1 site in United States.
Trial summary
RATIONALE: Vaccines made from cancer cells may help the body build an effective immune response to kill abnormal cells. PURPOSE: This clinical trial is studying how well vaccine therapy works in treating patients with myelodysplastic syndromes (MDS). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelodysplastic Syndromes
- What is being tested?
- K562/GM-CSF cell vaccine
- Type of treatment
- Vaccines
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 1 siteUnited States
- Study dates
- Sep 2007to Jan 2010
- Phase
- Early Phase 1
- 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
DISEASE CHARACTERISTICS:
- Pathologically confirmed myelodysplastic syndromes (MDS), including any of the following:
- Refractory anemia (RA)
- RA with ringed sideroblasts
- Refractory cytopenias with multilineage dysplasia (RCMD)
- RCMD with ringed sideroblasts
- RA with excess blasts 1 (5-9% blasts)
- RA with excess blasts 2 (10-19% blasts)
- Must have poor-risk MDS, defined by the following:
- At least 2 lineages involved
- Unfavorable cytogenetics (i.e., abnormalities of chromosome 5 or 7, 11q23, t[6;9], trisomy 8, inv3, or multiple/complex karyotype)
- Transfusion requirement of > 2 units of packed red blood cells monthly
- No chronic myelomonocytic leukemia
- No transformation to acute myeloid leukemia
PATIENT CHARACTERISTICS:
- ECOG performance status 0-2
- Creatinine < 2.5 mg/dL
- Bilirubin < 2.5 mg/dL (unless due to Gilbert's syndrome)
- Room air oxygen saturation ≥ 94% at rest
- Fertile patients must use effective contraception
- Negative pregnancy test
- No other malignancy within the past 5 years except in situ cervical cancer or adequately treated nonmelanoma skin cancer
- No active autoimmune disease or history of autoimmune disease requiring systemic immunosuppressants including, but not limited to, any of the following:
- Autoimmune hemolytic anemia
- Idiopathic thrombocytopenia purpura
- Inflammatory bowel disease
- Vasculitis
- Thyroiditis
- Rheumatic illnesses
- No known HIV serum antibody positivity
- No other disease requiring long-term corticosteroids or other immunosuppressants, such as severe chronic obstructive pulmonary disease or asthma
PRIOR CONCURRENT THERAPY:
- At least 2 weeks since prior systemic corticosteroids or other immunosuppressants (e.g., cyclosporine, azathioprine, tacrolimus, or mycophenolate mofetil)
- At least 3 weeks since prior growth factors
- At least 2 months since prior azacitidine for MDS
- No prior bone marrow or other organ transplantation
- No concurrent cytotoxic-based therapy for MDS
- No other concurrent growth factors, including epoetin alfa, filgrastim (G-CSF), or sargramostim (GM-CSF)
Trial locations and contacts
1 site in 1 country.
United States1 site
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Baltimore, Maryland, 21231
About this study
Official title: K562/GM-CSF Vaccination in Patients With Myelodysplastic Syndrome
RATIONALE: Vaccines made from cancer cells may help the body build an effective immune response to kill abnormal cells.
PURPOSE: This clinical trial is studying how well vaccine therapy works in treating patients with myelodysplastic syndromes (MDS).
Read the detailed description
OBJECTIVES:
Primary
- Determine the safety of GM-K562 cell vaccine in patients with myelodysplastic syndromes.
- Determine the hematologic and cytogenetic response in patients treated with this vaccine.
Secondary
- Determine if vaccination with GM-K562 cell vaccine can induce an immune response to common myeloid antigens (e.g., Wilms' tumor-1 [WT-1], survivin, or proteinase-3), as defined by a 30% increase from baseline in specific cytotoxic T-cells measured by Elispot assay, in patients with myelodysplastic syndromes.
- Determine if immune response correlates with any clinical responses (e.g., hematologic response, resolution of cytogenetic abnormalities, or decrease in other parameters, such as WT-1 mRNA levels).
OUTLINE: This is an open-label study.
Patients receive GM-K562 cell vaccine subcutaneously once in weeks 0, 3, 6, 9, and 17 in the absence of disease progression or unacceptable toxicity.
Blood and tissue samples are collected periodically for correlative and biomarker studies. Samples are analyzed by cytogenetic studies, fluorescent in situ hybridization (FISH), and flow cytometry. Elispot is used to quantify cellular cytotoxic T-cell response to Wilms' tumor-1 (WT-1), survivin, and proteinase 3.
After completion of study treatment, patients are followed every 3 months for 1 year.
PROJECTED ACCRUAL: A total of 15 patients will be accrued for this study.
- Conditions
- Myelodysplastic Syndromes
- Being tested
- K562/GM-CSF cell vaccine
- Keywords
- refractory anemia with excess blasts, refractory anemia with ringed sideroblasts, refractory anemia, refractory cytopenia with multilineage dysplasia, de novo myelodysplastic syndromes, previously treated myelodysplastic syndromes, secondary myelodysplastic syndromes
- Medical subject headings
- Myelodysplastic Syndromes, Anemia, Refractory, with Excess of Blasts, Anemia, Refractory
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
K562/GM-CSF cell vaccineBiological
| Study group | Role | What they receive |
|---|---|---|
| K562/GM-CSF cell vaccine | Experimental | Vaccinations of 1x10^8 cells are given to participants at weeks 0, 3, 6, 9, and 17. Receives: Biological: K562/GM-CSF cell vaccine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Hematologic Response Rate as Assessed by Number of Participants Achieving a Major Hematologic Response
A major hematologic response is defined as any of the following: hemoglobin increase >= 2 g/dL from baseline; platelet increase >= 30k/mcL from baseline; or neutrophil increase >= 100% or >= 500/mcL from baseline.
Measured: Baseline, week 21 post-intervention
Cytogenetic Response Rate as Assessed by Number of Participants Achieving a Cytogenetic Response
Cytogenetic response is defined as normalization of pretreatment cytogenetic abnormalities.
Measured: Week 21
Secondary outcomes (2)
Immune Response Rate as Assessed by Number of Participants Who Exhibit Induced Immune Response to WT-1, Survivin, or Proteinase-3
Immune response to WT-1, survivin, or proteinase-3 as defined by a 30% increase from baseline in cytotoxic T cells measured by Elispot analysis.
Measured: Baseline, week 21 post-intervention
Combined Immune and Clinical Response Rate
Number of participants who exhibited both an immune response as defined by Outcome 3 and a hematologic or cytogenetic response as defined by Outcomes 1 and 2, respectively.
Measured: Week 21 post-intervention
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Aug 2006 (estimated)
- Study startSep 2007
- Primary completionOct 2009
- Study completionJan 2010
Why it stopped: Loss of funding
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 Myelodysplastic SyndromesStudies of the same condition, open and under way.
- More trials run by Sidney Kimmel Comprehensive Cancer Center at Johns HopkinsThe 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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI), Alliance for Cancer Gene Therapy
- Responsible party
- Sponsor
- Investigators
- B. Douglas Smith, MD, Principal investigator, Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00361296
- Sponsor's protocol ID
- J05115
- Other IDs
- P30CA006973, NA_00001530
- Status last verified
- Oct 2018
- First submitted
- 4 Aug 2006
- Last updated
- 23 Mar 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Robinson TM, Prince GT, Thoburn C, Warlick E, Ferguson A, Kasamon YL, Borrello IM, Hess A, Smith BD. Pilot trial of K562/GM-CSF whole-cell vaccination in MDS patients. Leuk Lymphoma. 2018 Dec;59(12):2801-2811. doi: 10.1080/10428194.2018.1443449. Epub 2018 Apr 4.
PubMed 29616857
Study at a glance
From the registry record, last updated by the sponsor on 23 Mar 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- K562/GM-CSF Vaccination in Patients With Myelodysplastic Syndrome
- ClinicalTrials.gov ID
- NCT00361296
- Acronym / sponsor ID
- J05115
- Conditions
- Myelodysplastic Syndromes
- Keywords
- refractory anemia with excess blasts, refractory anemia with ringed sideroblasts, refractory anemia, refractory cytopenia with multilineage dysplasia, de novo myelodysplastic syndromes, previously treated myelodysplastic syndromes, secondary myelodysplastic syndromes
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Loss of funding
- Enrolment
- 9 participants (actual)
- Lead sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns HopkinsUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI), Alliance for Cancer Gene Therapy
- Study start
- Sep 2007 (actual)
- Primary completion
- Oct 2009 (actual)
- Study completion
- Jan 2010 (actual)
- First posted
- 8 Aug 2006 (estimated)
- Results
- Posted 16 Nov 2018
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
RATIONALE: Vaccines made from cancer cells may help the body build an effective immune response to kill abnormal cells.
PURPOSE: This clinical trial is studying how well vaccine therapy works in treating patients with myelodysplastic syndromes (MDS).
Detailed description
OBJECTIVES:
Primary
- Determine the safety of GM-K562 cell vaccine in patients with myelodysplastic syndromes.
- Determine the hematologic and cytogenetic response in patients treated with this vaccine.
Secondary
- Determine if vaccination with GM-K562 cell vaccine can induce an immune response to common myeloid antigens (e.g., Wilms' tumor-1 [WT-1], survivin, or proteinase-3), as defined by a 30% increase from baseline in specific cytotoxic T-cells measured by Elispot assay, in patients with myelodysplastic syndromes.
- Determine if immune response correlates with any clinical responses (e.g., hematologic response, resolution of cytogenetic abnormalities, or decrease in other parameters, such as WT-1 mRNA levels).
OUTLINE: This is an open-label study.
Patients receive GM-K562 cell vaccine subcutaneously once in weeks 0, 3, 6, 9, and 17 in the absence of disease progression or unacceptable toxicity.
Blood and tissue samples are collected periodically for correlative and biomarker studies. Samples are analyzed by cytogenetic studies, fluorescent in situ hybridization (FISH), and flow cytometry. Elispot is used to quantify cellular cytotoxic T-cell response to Wilms' tumor-1 (WT-1), survivin, and proteinase 3.
After completion of study treatment, patients are followed every 3 months for 1 year.
PROJECTED ACCRUAL: A total of 15 patients will be accrued for this study.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| K562/GM-CSF cell vaccine | Experimental | Biological: K562/GM-CSF cell vaccineVaccinations of 1x10^8 cells are given to participants at weeks 0, 3, 6, 9, and 17. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
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 |
|---|---|---|
| K562/GM-CSF cell vaccine | Biological | K562/GM-CSF cell vaccine |
Primary outcome
Hematologic Response Rate as Assessed by Number of Participants Achieving a Major Hematologic Response
Time frame: Baseline, week 21 post-intervention
A major hematologic response is defined as any of the following: hemoglobin increase >= 2 g/dL from baseline; platelet increase >= 30k/mcL from baseline; or neutrophil increase >= 100% or >= 500/mcL from baseline.
Cytogenetic Response Rate as Assessed by Number of Participants Achieving a Cytogenetic Response
Time frame: Week 21
Cytogenetic response is defined as normalization of pretreatment cytogenetic abnormalities.
2 secondary outcomes
Immune Response Rate as Assessed by Number of Participants Who Exhibit Induced Immune Response to WT-1, Survivin, or Proteinase-3
Time frame: Baseline, week 21 post-intervention
Combined Immune and Clinical Response Rate
Time frame: Week 21 post-intervention
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Mar 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 29616857Robinson TM, Prince GT, Thoburn C, Warlick E, Ferguson A, Kasamon YL, Borrello IM, Hess A, Smith BD. Pilot trial of K562/GM-CSF whole-cell vaccination in MDS patients. Leuk Lymphoma. 2018 Dec;59(12):2801-2811. doi: 10.1080/10428194.2018.1443449. Epub 2018 Apr 4.
- Individual participant dataSharing planned: No
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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.
B. Douglas Smith, MD principal investigator
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing K562/GM-CSF cell vaccine in people with Myelodysplastic Syndromes, run by Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins 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?
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 23 Mar 2023. 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?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
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 16 Nov 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?
Hematologic Response Rate as Assessed by Number of Participants Achieving a Major Hematologic Response (time frame: Baseline, week 21 post-intervention). The study lists 2 primary outcomes in all. 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 NCT00361296, 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 23 Mar 2023.