Trial Outcomes & Findings for PR1 Vaccination in Myelodysplastic Syndrome (MDS) (NCT NCT00893997)

NCT ID: NCT00893997

Last Updated: 2012-07-16

Results Overview

Patients assessed after 4th vaccination for immunologic response categorized as 'Immunologic-Responders' or 'Non-Responders.' Immune response defined as an increase of ≥ 0.5 PR1-HLA-A2 tetramer cells/μl compared to the pre study absolute PR1-HLA-A2 tetramer cells/μl. Time period 29 weeks after study entry, with week 0 corresponding to 1st injection, and 8th injection thus being given at week 25, 29 weeks corresponds to 13 weeks after receipt of a 4th injection.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

12 participants

Primary outcome timeframe

29 weeks

Results posted on

2012-07-16

Participant Flow

Recruitment Period: 01/09/07 through 03/24/08. All participants recruited at UT MD Anderson Cancer Center.

Twelve patients were registered, two patients did not receive drug and were not included in the study group therefore ten patients were evaluable.

Participant milestones

Participant milestones
Measure
PR-1 Vaccine
4 injections of 0.5 mg PR1 peptide vaccine every 3 weeks.
Overall Study
STARTED
10
Overall Study
COMPLETED
10
Overall Study
NOT COMPLETED
0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

PR1 Vaccination in Myelodysplastic Syndrome (MDS)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
PR-1 Vaccine
n=10 Participants
4 injections of 0.5 mg PR1 peptide vaccine every 3 weeks.
Age Continuous
64 years
n=5 Participants
Sex: Female, Male
Female
5 Participants
n=5 Participants
Sex: Female, Male
Male
5 Participants
n=5 Participants
Region of Enrollment
United States
10 participants
n=5 Participants

PRIMARY outcome

Timeframe: 29 weeks

Population: Analysis on patients treated; study terminated early.

Patients assessed after 4th vaccination for immunologic response categorized as 'Immunologic-Responders' or 'Non-Responders.' Immune response defined as an increase of ≥ 0.5 PR1-HLA-A2 tetramer cells/μl compared to the pre study absolute PR1-HLA-A2 tetramer cells/μl. Time period 29 weeks after study entry, with week 0 corresponding to 1st injection, and 8th injection thus being given at week 25, 29 weeks corresponds to 13 weeks after receipt of a 4th injection.

Outcome measures

Outcome measures
Measure
PR-1 Vaccine
n=10 Participants
4 injections of 0.5 mg PR1 peptide vaccine every 3 weeks.
Patient Immunologic Response
Immunological Response
3 participants
Patient Immunologic Response
No Immunological Response
7 participants

PRIMARY outcome

Timeframe: At 29 weeks

Population: Analysis on patients treated; study terminated early.

Clinical response based on the International Working Group (IWG) Response Criteria in myelodysplastic syndromes (MDS): 'Complete Response' or Hematologic Improvement' and 'No Clinical Response'. Clinical responses as assessed by standard criteria with bone marrow biopsy, cytogenetic studies (standard chromosome banding) and molecular studies 3 weeks after the last vaccination.

Outcome measures

Outcome measures
Measure
PR-1 Vaccine
n=10 Participants
4 injections of 0.5 mg PR1 peptide vaccine every 3 weeks.
Number of Patients With Clinical Response
Complete Response
0 participants
Number of Patients With Clinical Response
Hematological Improvement
1 participants
Number of Patients With Clinical Response
No Clinical Response
9 participants

Adverse Events

PR-1 Vaccine

Serious events: 1 serious events
Other events: 6 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
PR-1 Vaccine
n=10 participants at risk
4 injections of 0.5 mg PR1 peptide vaccine every 3 weeks.
General disorders
Allergic Reaction
10.0%
1/10 • Number of events 1 • 1 year and 11 months

Other adverse events

Other adverse events
Measure
PR-1 Vaccine
n=10 participants at risk
4 injections of 0.5 mg PR1 peptide vaccine every 3 weeks.
General disorders
Injection site reaction
40.0%
4/10 • Number of events 5 • 1 year and 11 months
General disorders
Allergic reaction
10.0%
1/10 • Number of events 1 • 1 year and 11 months
General disorders
Fatigue
20.0%
2/10 • Number of events 2 • 1 year and 11 months
Gastrointestinal disorders
Nausea
10.0%
1/10 • Number of events 1 • 1 year and 11 months
Gastrointestinal disorders
Vomiting
10.0%
1/10 • Number of events 1 • 1 year and 11 months
General disorders
Bone pain
20.0%
2/10 • Number of events 2 • 1 year and 11 months
General disorders
Chill
10.0%
1/10 • Number of events 1 • 1 year and 11 months
Skin and subcutaneous tissue disorders
Puritis
10.0%
1/10 • Number of events 1 • 1 year and 11 months
Gastrointestinal disorders
Mucositis
10.0%
1/10 • Number of events 1 • 1 year and 11 months

Additional Information

Guillermo Garcia-Manero, MD / Associate Professor

UT MD Anderson Cancer Center

Phone: 713-792-7305

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place