Trial Outcomes & Findings for WT1 Vaccine Treatment of Patients With Multiple Myeloma After Autologous Stem Cell Transplantation (NCT NCT01827137)
NCT ID: NCT01827137
Last Updated: 2024-09-19
Results Overview
Number and percentage of participants with CD4 and/or CD8 immune responses against WT1m measured 12-14 weeks after first GPS administration (6 x administrations)
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
20 participants
Primary outcome timeframe
12 weeks after the initial GPS vaccine (end of first series [GPS x 6 administrations])
Results posted on
2024-09-19
Participant Flow
Participant milestones
| Measure |
Galinpepimut-S + Montanide + GM-CSF
Galinpepimut-S (GPS) vaccinations are started 12-22 days following autologous stem cell transplantation (ASCT). GPS and Montanide (in a 1 mL emulsion) are administered subcutaneously (s.c.) Q2W on weeks 0, 2, 4, 6, 8, and 10. GM-CSF (70 μg) are administered s.c. one to two days before and on the day of GPS/Montanide administration. Participants whose disease has not progressed and who are clinically stable (no active infection with fevers and no cardiovascular/respiratory compromise) may receive up to 6 additional monthly vaccinations. The use of post-ASCT maintenance therapy is allowed starting 3 months after ASCT.
Galinpepimut-S: Galinpepimut-S admixed with the adjuvant Montanide following specified schedule
GM-CSF: subcutaneous injection
Montanide: adjuvant
lenalidomide: optional post-ASCT therapy
bortezomib: optional post-ASCT therapy
|
|---|---|
|
Overall Study
STARTED
|
20
|
|
Overall Study
COMPLETED
|
19
|
|
Overall Study
NOT COMPLETED
|
1
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
WT1 Vaccine Treatment of Patients With Multiple Myeloma After Autologous Stem Cell Transplantation
Baseline characteristics by cohort
| Measure |
Galinpepimut-S + Montanide + GM-CSF
n=19 Participants
Galinpepimut-S (GPS) vaccinations are started 12-22 days following autologous stem cell transplantation (ASCT). GPS and Montanide (in a 1 mL emulsion) are administered subcutaneously (s.c.) Q2W on weeks 0, 2, 4, 6, 8, and 10. GM-CSF (70 μg) are administered s.c. one to two days before and on the day of GPS/Montanide administration. Participants whose disease has not progressed and who are clinically stable (no active infection with fevers and no cardiovascular/respiratory compromise) may receive up to 6 additional monthly vaccinations. The use of post-ASCT maintenance therapy is allowed starting 3 months after ASCT.
Galinpepimut-S: Galinpepimut-S admixed with the adjuvant Montanide following specified schedule
GM-CSF: subcutaneous injection
Montanide: adjuvant
lenalidomide: optional post-ASCT therapy
bortezomib: optional post-ASCT therapy
|
|---|---|
|
Age, Continuous
|
61.3 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
11 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
8 Participants
n=5 Participants
|
|
Type of myeloma
IgG
|
12 Participants
n=5 Participants
|
|
Type of myeloma
IgA
|
4 Participants
n=5 Participants
|
|
Type of myeloma
Light chain
|
3 Participants
n=5 Participants
|
|
Prior therapies
|
2 number of therapies
n=5 Participants
|
|
Post ASCT therapy
Lenalidomide alone or in combination
|
18 Participants
n=5 Participants
|
|
Post ASCT therapy
Other (bortezomib)
|
1 Participants
n=5 Participants
|
|
Prior ASCT
1
|
17 Participants
n=5 Participants
|
|
Prior ASCT
2
|
2 Participants
n=5 Participants
|
|
Cytogenetics at diagnosis
High risk
|
15 Participants
n=5 Participants
|
|
Cytogenetics at diagnosis
Average risk
|
4 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 12 weeks after the initial GPS vaccine (end of first series [GPS x 6 administrations])Population: Only 15 patients completed 6 x GPS administrations
Number and percentage of participants with CD4 and/or CD8 immune responses against WT1m measured 12-14 weeks after first GPS administration (6 x administrations)
Outcome measures
| Measure |
Galinpepimut-S + Montanide + GM-CSF
n=15 Participants
Galinpepimut-S (GPS) vaccinations are started 12-22 days following autologous stem cell transplantation (ASCT). GPS and Montanide (in a 1 mL emulsion) are administered subcutaneously (s.c.) Q2W on weeks 0, 2, 4, 6, 8, and 10. GM-CSF (70 μg) are administered s.c. one to two days before and on the day of GPS/Montanide administration. Participants whose disease has not progressed and who are clinically stable (no active infection with fevers and no cardiovascular/respiratory compromise) may receive up to 6 additional monthly vaccinations. The use of post-ASCT maintenance therapy is allowed starting 3 months after ASCT.
Galinpepimut-S: Galinpepimut-S admixed with the adjuvant Montanide following specified schedule
GM-CSF: subcutaneous injection
Montanide: adjuvant
lenalidomide: optional post-ASCT therapy
bortezomib: optional post-ASCT therapy
|
|---|---|
|
WT1 T-cell Immune Response (IR) 12-14 Weeks After First GPS Administration (6 x Administrations)
|
9 Participants
|
SECONDARY outcome
Timeframe: 3 years and 8 monthsPFS measured from ASCT to time of myeloma progression (as defined by the International Myeloma Working Group \[IMW\] consensus criteria or subject death.
Outcome measures
| Measure |
Galinpepimut-S + Montanide + GM-CSF
n=19 Participants
Galinpepimut-S (GPS) vaccinations are started 12-22 days following autologous stem cell transplantation (ASCT). GPS and Montanide (in a 1 mL emulsion) are administered subcutaneously (s.c.) Q2W on weeks 0, 2, 4, 6, 8, and 10. GM-CSF (70 μg) are administered s.c. one to two days before and on the day of GPS/Montanide administration. Participants whose disease has not progressed and who are clinically stable (no active infection with fevers and no cardiovascular/respiratory compromise) may receive up to 6 additional monthly vaccinations. The use of post-ASCT maintenance therapy is allowed starting 3 months after ASCT.
Galinpepimut-S: Galinpepimut-S admixed with the adjuvant Montanide following specified schedule
GM-CSF: subcutaneous injection
Montanide: adjuvant
lenalidomide: optional post-ASCT therapy
bortezomib: optional post-ASCT therapy
|
|---|---|
|
Progression-free Survival (PFS) From Autologous Stem Cell Transplant (ASCT)
|
717 days
Interval 286.0 to
Upper 95%CI not reached due to end of study monitoring duration
|
SECONDARY outcome
Timeframe: 3 years and 8 monthsOS measured from time of autologous stem cell transplant (ASCT) to the time of subject death
Outcome measures
| Measure |
Galinpepimut-S + Montanide + GM-CSF
n=19 Participants
Galinpepimut-S (GPS) vaccinations are started 12-22 days following autologous stem cell transplantation (ASCT). GPS and Montanide (in a 1 mL emulsion) are administered subcutaneously (s.c.) Q2W on weeks 0, 2, 4, 6, 8, and 10. GM-CSF (70 μg) are administered s.c. one to two days before and on the day of GPS/Montanide administration. Participants whose disease has not progressed and who are clinically stable (no active infection with fevers and no cardiovascular/respiratory compromise) may receive up to 6 additional monthly vaccinations. The use of post-ASCT maintenance therapy is allowed starting 3 months after ASCT.
Galinpepimut-S: Galinpepimut-S admixed with the adjuvant Montanide following specified schedule
GM-CSF: subcutaneous injection
Montanide: adjuvant
lenalidomide: optional post-ASCT therapy
bortezomib: optional post-ASCT therapy
|
|---|---|
|
Overall Survival (OS) From Autologous Stem Cell Transplant (ASCT)
|
NA days
Median OS and 95% CI not reached due to end of the study monitoring period (3 years and 8 months)
|
POST_HOC outcome
Timeframe: 38 weeks after the initial GPS vaccine (end of booster series [GPS x 12 administrations])Population: 12 patients completed all 12 x GPS administrations
Number and percentage of participants with CD4 and/or CD8 immune responses against WT1 measured after all GPS administrations (12 x administrations)
Outcome measures
| Measure |
Galinpepimut-S + Montanide + GM-CSF
n=12 Participants
Galinpepimut-S (GPS) vaccinations are started 12-22 days following autologous stem cell transplantation (ASCT). GPS and Montanide (in a 1 mL emulsion) are administered subcutaneously (s.c.) Q2W on weeks 0, 2, 4, 6, 8, and 10. GM-CSF (70 μg) are administered s.c. one to two days before and on the day of GPS/Montanide administration. Participants whose disease has not progressed and who are clinically stable (no active infection with fevers and no cardiovascular/respiratory compromise) may receive up to 6 additional monthly vaccinations. The use of post-ASCT maintenance therapy is allowed starting 3 months after ASCT.
Galinpepimut-S: Galinpepimut-S admixed with the adjuvant Montanide following specified schedule
GM-CSF: subcutaneous injection
Montanide: adjuvant
lenalidomide: optional post-ASCT therapy
bortezomib: optional post-ASCT therapy
|
|---|---|
|
WT1 T-cell Immune Response (IR) After Completion of All GPS Administrations
|
12 Participants
|
Adverse Events
Galinpepimut-S + Montanide + GM-CSF
Serious events: 0 serious events
Other events: 20 other events
Deaths: 2 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Galinpepimut-S + Montanide + GM-CSF
n=20 participants at risk
Galinpepimut-S (GPS) vaccinations are started 12-22 days following autologous stem cell transplantation (ASCT). GPS and Montanide (in a 1 mL emulsion) are administered subcutaneously (s.c.) Q2W on weeks 0, 2, 4, 6, 8, and 10. GM-CSF (70 μg) are administered s.c. one to two days before and on the day of GPS/Montanide administration. Participants whose disease has not progressed and who are clinically stable (no active infection with fevers and no cardiovascular/respiratory compromise) may receive up to 6 additional monthly vaccinations. The use of post-ASCT maintenance therapy is allowed starting 3 months after ASCT.
Galinpepimut-S: Galinpepimut-S admixed with the adjuvant Montanide following specified schedule
GM-CSF: subcutaneous injection
Montanide: adjuvant
lenalidomide: optional post-ASCT therapy
bortezomib: optional post-ASCT therapy
|
|---|---|
|
Blood and lymphatic system disorders
Anemia
|
100.0%
20/20 • 4.5 years
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
100.0%
20/20 • 4.5 years
|
|
Metabolism and nutrition disorders
Hypoalbuminemia
|
100.0%
20/20 • 4.5 years
|
|
Investigations
Lymphocyte count decreased
|
100.0%
20/20 • 4.5 years
|
|
Investigations
Neutrophil count decreased
|
100.0%
20/20 • 4.5 years
|
|
Investigations
Platelet count decreased
|
100.0%
20/20 • 4.5 years
|
|
Investigations
White blood cell decreased
|
100.0%
20/20 • 4.5 years
|
|
Metabolism and nutrition disorders
Hypocalcemia
|
95.0%
19/20 • 4.5 years
|
|
Investigations
Alanine aminotransferase increased
|
70.0%
14/20 • 4.5 years
|
|
Investigations
Aspartate aminotransferase increased
|
60.0%
12/20 • 4.5 years
|
|
Metabolism and nutrition disorders
Hypokalemia
|
60.0%
12/20 • 4.5 years
|
|
Metabolism and nutrition disorders
Hypomagnesemia
|
50.0%
10/20 • 4.5 years
|
|
General disorders
Fatigue
|
45.0%
9/20 • 4.5 years
|
|
Metabolism and nutrition disorders
Hypophosphatemia
|
45.0%
9/20 • 4.5 years
|
|
Investigations
Alkaline phosphatase increased
|
40.0%
8/20 • 4.5 years
|
|
Investigations
Blood bilirubin increased
|
40.0%
8/20 • 4.5 years
|
|
Metabolism and nutrition disorders
Hyponatremia
|
40.0%
8/20 • 4.5 years
|
|
Metabolism and nutrition disorders
Hypernatremia
|
35.0%
7/20 • 4.5 years
|
|
Gastrointestinal disorders
Diarrhea
|
30.0%
6/20 • 4.5 years
|
|
Gastrointestinal disorders
Nausea
|
30.0%
6/20 • 4.5 years
|
|
Investigations
Cholesterol high
|
25.0%
5/20 • 4.5 years
|
|
Investigations
Creatinine increased
|
25.0%
5/20 • 4.5 years
|
|
Metabolism and nutrition disorders
Hypertriglyceridemia
|
25.0%
5/20 • 4.5 years
|
|
Metabolism and nutrition disorders
Hypoglycemia
|
25.0%
5/20 • 4.5 years
|
|
Metabolism and nutrition disorders
Anorexia
|
20.0%
4/20 • 4.5 years
|
|
Investigations
INR increased
|
20.0%
4/20 • 4.5 years
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
20.0%
4/20 • 4.5 years
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
20.0%
4/20 • 4.5 years
|
|
Psychiatric disorders
Anxiety
|
15.0%
3/20 • 4.5 years
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
15.0%
3/20 • 4.5 years
|
|
Metabolism and nutrition disorders
Hypermagnesemia
|
15.0%
3/20 • 4.5 years
|
|
Nervous system disorders
Nervous system disorders - Other,
|
15.0%
3/20 • 4.5 years
|
|
Metabolism and nutrition disorders
Dehydration
|
10.0%
2/20 • 4.5 years
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
10.0%
2/20 • 4.5 years
|
|
Musculoskeletal and connective tissue disorders
Generalized muscle weakness
|
10.0%
2/20 • 4.5 years
|
|
Metabolism and nutrition disorders
Hyperkalemia
|
10.0%
2/20 • 4.5 years
|
|
General disorders
Injection site reaction
|
10.0%
2/20 • 4.5 years
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
10.0%
2/20 • 4.5 years
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
10.0%
2/20 • 4.5 years
|
|
Nervous system disorders
Peripheral sensory neuropathy
|
10.0%
2/20 • 4.5 years
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place