Trial Outcomes & Findings for Vaccine Therapy in Treating Patients With Myelodysplastic Syndromes (NCT NCT00361296)

Last Updated: 2023-03-23

Results Overview

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.

Recruitment status

TERMINATED

Study phase

EARLY_PHASE1

Target enrollment

9 participants

Primary outcome timeframe

Baseline, week 21 post-intervention

Results posted on

2023-03-23

Participant Flow

2 participants were screen failures. 2 additional participants were removed from study by physician decision prior to receiving protocol intervention, so they never started the study.

Participant milestones

Participant milestones
Measure	K562/GM-CSF Cell Vaccine Vaccinations of 1x10\^8 cells are given to participants at weeks 0, 3, 6, 9, and 17. K562/GM-CSF cell vaccine
Overall Study STARTED	5
Overall Study COMPLETED	2
Overall Study NOT COMPLETED	3

Reasons for withdrawal

Reasons for withdrawal
Measure	K562/GM-CSF Cell Vaccine Vaccinations of 1x10\^8 cells are given to participants at weeks 0, 3, 6, 9, and 17. K562/GM-CSF cell vaccine
Overall Study Physician Decision	1
Overall Study Lack of Efficacy	2

Baseline Characteristics

Vaccine Therapy in Treating Patients With Myelodysplastic Syndromes

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	K562/GM-CSF Cell Vaccine n=5 Participants Vaccinations of 1x10\^8 cells are given to participants at weeks 0, 3, 6, 9, and 17. K562/GM-CSF cell vaccine
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	3 Participants n=5 Participants
Age, Categorical >=65 years	2 Participants n=5 Participants
Age, Continuous	64 years n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants
Sex: Female, Male Male	4 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	5 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	2 Participants n=5 Participants
Race (NIH/OMB) White	3 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline, week 21 post-intervention

Outcome measures

Outcome measures
Measure	K562/GM-CSF Cell Vaccine n=5 Participants Vaccinations of 1x10\^8 cells are given to participants at weeks 0, 3, 6, 9, and 17. K562/GM-CSF cell vaccine
Hematologic Response Rate as Assessed by Number of Participants Achieving a Major Hematologic Response	1 Participants

PRIMARY outcome

Timeframe: Week 21

Cytogenetic response is defined as normalization of pretreatment cytogenetic abnormalities.

Outcome measures

Outcome measures
Measure	K562/GM-CSF Cell Vaccine n=5 Participants Vaccinations of 1x10\^8 cells are given to participants at weeks 0, 3, 6, 9, and 17. K562/GM-CSF cell vaccine
Cytogenetic Response Rate as Assessed by Number of Participants Achieving a Cytogenetic Response	0 Participants

SECONDARY outcome

Timeframe: Baseline, week 21 post-intervention

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.

Outcome measures

Outcome measures
Measure	K562/GM-CSF Cell Vaccine n=5 Participants Vaccinations of 1x10\^8 cells are given to participants at weeks 0, 3, 6, 9, and 17. K562/GM-CSF cell vaccine
Immune Response Rate as Assessed by Number of Participants Who Exhibit Induced Immune Response to WT-1, Survivin, or Proteinase-3	0 Participants

SECONDARY outcome

Timeframe: Week 21 post-intervention

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.

Outcome measures

Outcome measures
Measure	K562/GM-CSF Cell Vaccine n=5 Participants Vaccinations of 1x10\^8 cells are given to participants at weeks 0, 3, 6, 9, and 17. K562/GM-CSF cell vaccine
Combined Immune and Clinical Response Rate	0 Participants

Adverse Events

K562/GM-CSF Cell Vaccine

Serious events: 0 serious events

Other events: 5 other events

Deaths: 1 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	K562/GM-CSF Cell Vaccine n=5 participants at risk Vaccinations of 1x10\^8 cells are given to participants at weeks 0, 3, 6, 9, and 17. K562/GM-CSF cell vaccine
Investigations Thrombocytopenia	40.0% 2/5 • Number of events 4 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
Nervous system disorders Tinnitus	20.0% 1/5 • Number of events 1 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
Immune system disorders Transfusion reaction	20.0% 1/5 • Number of events 2 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
Nervous system disorders Vision changes	20.0% 1/5 • Number of events 1 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
Gastrointestinal disorders Vomiting	20.0% 1/5 • Number of events 1 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
General disorders Weight gain	20.0% 1/5 • Number of events 1 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
Eye disorders Xerophthalmia	20.0% 1/5 • Number of events 1 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
Skin and subcutaneous tissue disorders Xerosis	20.0% 1/5 • Number of events 1 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
Infections and infestations Zoster infection	20.0% 1/5 • Number of events 2 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
Gastrointestinal disorders Abdominal distension	20.0% 1/5 • Number of events 1 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
Immune system disorders Adenopathy	40.0% 2/5 • Number of events 2 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
Investigations ALT elevated	40.0% 2/5 • Number of events 2 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
Nervous system disorders Altered mental status	20.0% 1/5 • Number of events 1 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
Investigations Anemia	20.0% 1/5 • Number of events 2 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
General disorders Anorexia	20.0% 1/5 • Number of events 1 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
Musculoskeletal and connective tissue disorders Arthralgia	40.0% 2/5 • Number of events 2 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
Investigations AST elevated	40.0% 2/5 • Number of events 2 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
Infections and infestations Bacteremia	20.0% 1/5 • Number of events 1 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
Eye disorders Bleeding - eye	20.0% 1/5 • Number of events 1 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
General disorders Bruising	20.0% 1/5 • Number of events 1 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
Cardiac disorders Heart failure	20.0% 1/5 • Number of events 1 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
Gastrointestinal disorders Constipation	40.0% 2/5 • Number of events 2 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
Respiratory, thoracic and mediastinal disorders Cough	80.0% 4/5 • Number of events 4 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
Investigations Creatinine elevated	20.0% 1/5 • Number of events 1 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
Nervous system disorders Dizziness	40.0% 2/5 • Number of events 2 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
Respiratory, thoracic and mediastinal disorders Dyspnea	40.0% 2/5 • Number of events 2 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
Cardiac disorders Edema	60.0% 3/5 • Number of events 5 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
Skin and subcutaneous tissue disorders Erythema - mouth	20.0% 1/5 • Number of events 1 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
General disorders Fatigue	20.0% 1/5 • Number of events 2 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
General disorders Fever	20.0% 1/5 • Number of events 1 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
General disorders Flu-like symptoms	20.0% 1/5 • Number of events 3 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
General disorders Flushing	20.0% 1/5 • Number of events 1 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
Gastrointestinal disorders Gastroenteritis	20.0% 1/5 • Number of events 1 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
General disorders Headache	80.0% 4/5 • Number of events 6 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
Gastrointestinal disorders Hemorrhoids	20.0% 1/5 • Number of events 1 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
Investigations Hyperbilirubinemia	20.0% 1/5 • Number of events 1 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
Investigations Hyperglycemia	20.0% 1/5 • Number of events 1 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
Investigations Hyperphosphatemia	20.0% 1/5 • Number of events 1 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
Investigations Hyperuricemia	20.0% 1/5 • Number of events 1 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
Immune system disorders Injection site reactions	100.0% 5/5 • Number of events 25 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
General disorders Insomnia	40.0% 2/5 • Number of events 2 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
Investigations Leukopenia	20.0% 1/5 • Number of events 1 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
Infections and infestations Lyme disease	20.0% 1/5 • Number of events 1 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
Respiratory, thoracic and mediastinal disorders Nasal congestion	40.0% 2/5 • Number of events 2 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
Gastrointestinal disorders Nausea	60.0% 3/5 • Number of events 4 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
Nervous system disorders Neuropathy	40.0% 2/5 • Number of events 2 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
Investigations Neutropenia	20.0% 1/5 • Number of events 1 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
General disorders Epistaxis	20.0% 1/5 • Number of events 1 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
Musculoskeletal and connective tissue disorders Pain - flank	20.0% 1/5 • Number of events 1 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
General disorders Night sweats	20.0% 1/5 • Number of events 1 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
Skin and subcutaneous tissue disorders Rash	40.0% 2/5 • Number of events 2 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
Infections and infestations Respiratory infection	40.0% 2/5 • Number of events 3 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
General disorders Sore throat	20.0% 1/5 • Number of events 1 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
General disorders Swollen gums	20.0% 1/5 • Number of events 1 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
Cardiac disorders Tachycardia	20.0% 1/5 • Number of events 1 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
Nervous system disorders Taste changes	20.0% 1/5 • Number of events 2 • Up to 18 months Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.

Additional Information

Doug Smith, MD

Johns Hopkins University

Phone: 4102872935

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place