Trial Outcomes & Findings for Proteinase 3 PR1 Peptide Mixed With Montanide ISA-51 VG Adjuvant and Administered With GM-CSF and PEG-INTRON(R) (NCT NCT00415857)

NCT ID: NCT00415857

Last Updated: 2018-08-21

Results Overview

Molecular response rate is number of respondents compared to total participants. Molecular Response is defined as a greater than a one-log reduction of Breakpoint Cluster Region-Abelson Murine Leukemia (BCR-ABL) transcript levels by quantitative polymerase chain reaction (PCR) from the baseline level at the time vaccination was initiated, or a disappearance of BCR-ABL transcripts, as measured by reverse transcription polymerase chain reaction (RT-PCR), occurring within 6 months from the last vaccination. Participants receive a series of 4 vaccinations administered at 3-week intervals and the fourth (final) vaccination administered 3 months after the third vaccination with blood draw to test PCR following every 3 months to test the level of leukemia in the blood and to see if disease is responding to the vaccine.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

5 participants

Primary outcome timeframe

Baseline to 18 weeks, up to 6 months post final vaccination for overall study participation period.

Results posted on

2018-08-21

Participant Flow

Recruitment period: December 20, 2006 to March 13, 2009. All recruitment done at the University of Texas (UT) MD Anderson Center.

Due to the unavailability of the study drug (vaccine) enrollment was halted and the study eventually terminated when additional attempts were unsuccessful to obtain a supply of vaccine.

Participant milestones

Participant milestones
Measure
PR1 + Imatinib
PR1 peptide administered dose 0.5 mg on weeks 0, 3, 6 and 18 for a total of 4 doses, with oral Imatinib at same dose received during last 6 months. Granulocyte-macrophage colony-stimulating factor (GM-CSF) 75 micrograms subcutaneously in same vaccine area with every vaccination.
PR1 + Imatinib + Interferon
PR1 peptide administered dose 0.5 mg on weeks 0, 3, 6 and 18 for a total of 4 doses, with oral Imatinib at same dose received during last 6 months. Granulocyte-macrophage colony-stimulating factor (GM-CSF) 75 micrograms subcutaneously in same vaccine area with every vaccination, and Peginterferon alfa-2b 0.5 microg/kg subcutaneous injection with each PR1 vaccination.
Overall Study
STARTED
2
3
Overall Study
COMPLETED
2
3
Overall Study
NOT COMPLETED
0
0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Proteinase 3 PR1 Peptide Mixed With Montanide ISA-51 VG Adjuvant and Administered With GM-CSF and PEG-INTRON(R)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
PR1 + Imatinib
n=2 Participants
PR1 peptide administered dose 0.5 mg on weeks 0, 3, 6 and 18 for a total of 4 doses, with oral Imatinib at same dose received during last 6 months. Granulocyte-macrophage colony-stimulating factor (GM-CSF) 75 micrograms subcutaneously in same vaccine area with every vaccination.
PR1 + Imatinib + Interferon
n=3 Participants
PR1 peptide administered dose 0.5 mg on weeks 0, 3, 6 and 18 for a total of 4 doses, with oral Imatinib at same dose received during last 6 months. Granulocyte-macrophage colony-stimulating factor (GM-CSF) 75 micrograms subcutaneously in same vaccine area with every vaccination, and Peginterferon alfa-2b 0.5 microg/kg subcutaneous injection with each PR1 vaccination.
Total
n=5 Participants
Total of all reporting groups
Age, Continuous
45 Years
n=93 Participants
46 Years
n=4 Participants
46 Years
n=27 Participants
Sex: Female, Male
Female
1 Participants
n=93 Participants
1 Participants
n=4 Participants
2 Participants
n=27 Participants
Sex: Female, Male
Male
1 Participants
n=93 Participants
2 Participants
n=4 Participants
3 Participants
n=27 Participants
Region of Enrollment
United States
2 participants
n=93 Participants
3 participants
n=4 Participants
5 participants
n=27 Participants

PRIMARY outcome

Timeframe: Baseline to 18 weeks, up to 6 months post final vaccination for overall study participation period.

Molecular response rate is number of respondents compared to total participants. Molecular Response is defined as a greater than a one-log reduction of Breakpoint Cluster Region-Abelson Murine Leukemia (BCR-ABL) transcript levels by quantitative polymerase chain reaction (PCR) from the baseline level at the time vaccination was initiated, or a disappearance of BCR-ABL transcripts, as measured by reverse transcription polymerase chain reaction (RT-PCR), occurring within 6 months from the last vaccination. Participants receive a series of 4 vaccinations administered at 3-week intervals and the fourth (final) vaccination administered 3 months after the third vaccination with blood draw to test PCR following every 3 months to test the level of leukemia in the blood and to see if disease is responding to the vaccine.

Outcome measures

Outcome measures
Measure
PR1 + Imatinib
n=2 Participants
PR1 peptide administered dose 0.5 mg on weeks 0, 3, 6 and 18 for a total of 4 doses, with oral Imatinib at same dose received during last 6 months. Granulocyte-macrophage colony-stimulating factor (GM-CSF) 75 micrograms subcutaneously in same vaccine area with every vaccination.
PR1 + Imatinib + Interferon
n=3 Participants
PR1 peptide administered dose 0.5 mg on weeks 0, 3, 6 and 18 for a total of 4 doses, with oral Imatinib at same dose received during last 6 months. Granulocyte-macrophage colony-stimulating factor (GM-CSF) 75 micrograms subcutaneously in same vaccine area with every vaccination, and Peginterferon alfa-2b 0.5 microg/kg subcutaneous injection with each PR1 vaccination.
Molecular Response Rate
0 percentage of participants
0 percentage of participants

SECONDARY outcome

Timeframe: Period of 18 weeks (i.e., one vaccination each on weeks 0, 3, 6, and 18).

Immunologic Response (immune response) is defined as an increase of ≥ 0.5 PR1-HLA-A2 \[human leukocyte antigen-A2 (HLA-A2)\] tetramer cells / μl at the time of either the 3rd or 4th vaccination compared to the pre study absolute PR1-HLA-A2 tetramer cells / μl. Participants receive a total of 4 vaccinations over a period of 18 weeks (i.e., one vaccination each on weeks 0, 3, 6, and 18). Participants assessed after 3rd and 4th vaccination for immunologic response.

Outcome measures

Outcome measures
Measure
PR1 + Imatinib
n=2 Participants
PR1 peptide administered dose 0.5 mg on weeks 0, 3, 6 and 18 for a total of 4 doses, with oral Imatinib at same dose received during last 6 months. Granulocyte-macrophage colony-stimulating factor (GM-CSF) 75 micrograms subcutaneously in same vaccine area with every vaccination.
PR1 + Imatinib + Interferon
n=3 Participants
PR1 peptide administered dose 0.5 mg on weeks 0, 3, 6 and 18 for a total of 4 doses, with oral Imatinib at same dose received during last 6 months. Granulocyte-macrophage colony-stimulating factor (GM-CSF) 75 micrograms subcutaneously in same vaccine area with every vaccination, and Peginterferon alfa-2b 0.5 microg/kg subcutaneous injection with each PR1 vaccination.
Number of Participants With Immunologic Response
0 participants
0 participants

Adverse Events

PR1 + Imatinib

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

PR1 + Imatinib + Interferon

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

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure
PR1 + Imatinib
n=2 participants at risk
PR1 peptide administered dose 0.5 mg on weeks 0, 3, 6 and 18 for a total of 4 doses, with oral Imatinib at same dose received during last 6 months. Granulocyte-macrophage colony-stimulating factor (GM-CSF) 75 micrograms subcutaneously in same vaccine area with every vaccination.
PR1 + Imatinib + Interferon
n=3 participants at risk
PR1 peptide administered dose 0.5 mg on weeks 0, 3, 6 and 18 for a total of 4 doses, with oral Imatinib at same dose received during last 6 months. Granulocyte-macrophage colony-stimulating factor (GM-CSF) 75 micrograms subcutaneously in same vaccine area with every vaccination, and Peginterferon alfa-2b 0.5 microg/kg subcutaneous injection with each PR1 vaccination.
Skin and subcutaneous tissue disorders
Injection Site Reaction
50.0%
1/2 • Number of events 1 • Participants followed for one month after removal from study. The overall study period was 1 years and 8 months.
33.3%
1/3 • Number of events 1 • Participants followed for one month after removal from study. The overall study period was 1 years and 8 months.

Additional Information

Jorge Cortes M.D./Professor

The University of Texas M. D. Anderson Cancer Center

Phone: 713/792-7305

Results disclosure agreements

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