Vaccine Therapy in Treating Patients With Myelodysplastic Syndromes

NCT00361296 | Terminated Early Phase 1 Results FDA Drug

• 3 other identifiers: J05115P30CA006973NA_00001530
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 1

Brief 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).

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

Outcome Measures

Primary Outcomes (2)

• 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.

  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.

  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

  Baseline, week 21 post-intervention

• Combined Immune and Clinical Response Rate

  Week 21 post-intervention

Study Arms (1)

K562/GM-CSF cell vaccine

EXPERIMENTAL

Vaccinations of 1x10\^8 cells are given to participants at weeks 0, 3, 6, 9, and 17.

Biological: K562/GM-CSF cell vaccine

Interventions

K562/GM-CSF cell vaccine BIOLOGICAL

K562/GM-CSF cell vaccine

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

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)

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins: lead
• National Cancer Institute (NCI): collaborator
• Alliance for Cancer Gene Therapy: collaborator

Study Sites (1)

Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

Baltimore, Maryland, 21231, United States

Location

Related Publications (1)

• 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.

  PMID: 29616857 RESULT

MeSH Terms

Conditions

Myelodysplastic Syndromes; Anemia, Refractory, with Excess of Blasts; Anemia, Refractory

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Anemia

Results Point of Contact

• Title: Doug Smith, MD
• Organization: Johns Hopkins University

smithdo@jhmi.edu

4102872935

Study Officials

• B. Douglas Smith, MD

  Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 4, 2006
• First Posted: August 8, 2006
• Study Start: September 1, 2007
• Primary Completion: October 1, 2009
• Study Completion: January 1, 2010
• Last Updated: March 23, 2023
• Results First Posted: November 16, 2018

Record last verified: 2018-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)