Trial Outcomes & Findings for Combination Immunotherapy and Autologous Stem Cell Transplantation for Myeloma (NCT NCT01245673)

NCT ID: NCT01245673

Last Updated: 2020-04-06

Results Overview

To determine whether lenalidomide maintenance plus the late booster immunizations leads to improved myeloma clinical responses between day 180 and day 100 post-transplant.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

28 participants

Primary outcome timeframe

Between day 100 and 180 post transplant

Results posted on

2020-04-06

Participant Flow

Participant milestones

Participant milestones
Measure
MAGE A-3, GM-CSF, Hiltonol®, T Cells, Lenalidomide,
MAGE-A3, GM-CSF, Hiltonol® (Poly-ICLC), PCV vaccine prior to apheresis. Stem cell mobilization. High-dose melphalan, then hematopoietic stem cells on Day 0. Day 2 Activated/costimulated autologous T cells. Days 14, 42 and 90 MAGE-A3, Hiltonol® and PVC. Day 100 start Lenalidomide. Day 120 and 150 MAGE-A3 and PVC.
Enrollment
STARTED
28
Enrollment
COMPLETED
27
Enrollment
NOT COMPLETED
1
Pre Transplant Vaccinations
STARTED
27
Pre Transplant Vaccinations
Pre Transplant Vaccinations 1-3
27
Pre Transplant Vaccinations
COMPLETED
27
Pre Transplant Vaccinations
NOT COMPLETED
0
Activated/Costimulated Autologous T Cell
STARTED
27
Activated/Costimulated Autologous T Cell
COMPLETED
27
Activated/Costimulated Autologous T Cell
NOT COMPLETED
0
MAGE-A3, Hiltonol®, PVC After T Cells
STARTED
27
MAGE-A3, Hiltonol®, PVC After T Cells
COMPLETED
26
MAGE-A3, Hiltonol®, PVC After T Cells
NOT COMPLETED
1
Start Lenalidomide
STARTED
26
Start Lenalidomide
COMPLETED
26
Start Lenalidomide
NOT COMPLETED
0
Maintenance: MAGE-A3, PVC After T Cells
STARTED
26
Maintenance: MAGE-A3, PVC After T Cells
COMPLETED
26
Maintenance: MAGE-A3, PVC After T Cells
NOT COMPLETED
0

Reasons for withdrawal

Reasons for withdrawal
Measure
MAGE A-3, GM-CSF, Hiltonol®, T Cells, Lenalidomide,
MAGE-A3, GM-CSF, Hiltonol® (Poly-ICLC), PCV vaccine prior to apheresis. Stem cell mobilization. High-dose melphalan, then hematopoietic stem cells on Day 0. Day 2 Activated/costimulated autologous T cells. Days 14, 42 and 90 MAGE-A3, Hiltonol® and PVC. Day 100 start Lenalidomide. Day 120 and 150 MAGE-A3 and PVC.
MAGE-A3, Hiltonol®, PVC After T Cells
Off Study due to Disease Progression
1

Baseline Characteristics

Combination Immunotherapy and Autologous Stem Cell Transplantation for Myeloma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Prevnar, T Cells, Lenalidomide, MAGE A-3
n=27 Participants
MAGE-A3, GM-CSF, Hiltonol® (Poly-ICLC), PCV vaccine prior to apheresis. Stem cell mobilization. High-dose melphalan, then hematopoietic stem cells on Day 0. Day 2 Activated/costimulated autologous T cells. Days 14, 42 and 90 MAGE-A3, Hiltonol® and PVC. Day 100 start Lenalidomide. Day 120 and 150 MAGE-A3 and PVC.
Age, Categorical
<=18 years
0 Participants
n=93 Participants
Age, Categorical
Between 18 and 65 years
22 Participants
n=93 Participants
Age, Categorical
>=65 years
5 Participants
n=93 Participants
Age, Continuous
52.48 years
n=93 Participants
Sex: Female, Male
Female
11 Participants
n=93 Participants
Sex: Female, Male
Male
16 Participants
n=93 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=93 Participants
Race (NIH/OMB)
Asian
3 Participants
n=93 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=93 Participants
Race (NIH/OMB)
Black or African American
6 Participants
n=93 Participants
Race (NIH/OMB)
White
18 Participants
n=93 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=93 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
n=93 Participants
Region of Enrollment
United States
27 participants
n=93 Participants

PRIMARY outcome

Timeframe: Between day 100 and 180 post transplant

Population: Subjects that reached D100 and D180

To determine whether lenalidomide maintenance plus the late booster immunizations leads to improved myeloma clinical responses between day 180 and day 100 post-transplant.

Outcome measures

Outcome measures
Measure
Prevnar, T Cells, Lenalidomide, MAGE A-3
n=26 Participants
All patients will receive a priming immunization with a MAGE-A3/GM-CSF vaccine with adjuvant Hiltonol® (Poly-ICLC) along with the pneumococcal conjugate vaccine/PCV control vaccine about 10 days before a steady-state mononuclear cell apheresis. Patients will then undergo hematopoietic stem cell mobilization. All patients will receive high-dose melphalan followed by hematopoietic stem cells on day 0.
Primary Myeloma Endpoint
Subjects in CR D100 and D180
4 Participants
Primary Myeloma Endpoint
Subjects in sCR D100 and 180
1 Participants
Primary Myeloma Endpoint
Subjects in VGPR D100 and D180
1 Participants
Primary Myeloma Endpoint
Subjects in PR D100 and D180
5 Participants
Primary Myeloma Endpoint
Subjects with SD at D+100 and 180
3 Participants
Primary Myeloma Endpoint
Subjects with improved response at D100 and D180
6 Participants
Primary Myeloma Endpoint
Subjects with poorer response at D100 and D180
4 Participants
Primary Myeloma Endpoint
Subjects with no disease response reported
2 Participants

Adverse Events

Prevnar, T Cells, Lenalidomide, MAGE A-3

Serious events: 9 serious events
Other events: 27 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
Prevnar, T Cells, Lenalidomide, MAGE A-3
n=27 participants at risk
MAGE-A3, GM-CSF, Hiltonol® (Poly-ICLC), PCV vaccine prior to apheresis. Stem cell mobilization. High-dose melphalan, then hematopoietic stem cells on Day 0. Day 2 Activated/costimulated autologous T cells. Days 14, 42 and 90 MAGE-A3, Hiltonol® and PVC. Day 100 start Lenalidomide. Day 120 and 150 MAGE-A3 and PVC.
Blood and lymphatic system disorders
Anemia
3.7%
1/27 • Number of events 1 • Adverse event reporting begins at the time of T-cell harvest and continues until day 180 post infusion.
General disorders
Chills
3.7%
1/27 • Number of events 1 • Adverse event reporting begins at the time of T-cell harvest and continues until day 180 post infusion.
Respiratory, thoracic and mediastinal disorders
Coughing/shortness of breath
3.7%
1/27 • Number of events 1 • Adverse event reporting begins at the time of T-cell harvest and continues until day 180 post infusion.
Vascular disorders
Deep Vein Thrombosis
3.7%
1/27 • Number of events 1 • Adverse event reporting begins at the time of T-cell harvest and continues until day 180 post infusion.
Blood and lymphatic system disorders
Febrile neutropenia
3.7%
1/27 • Number of events 1 • Adverse event reporting begins at the time of T-cell harvest and continues until day 180 post infusion.
General disorders
Fever
7.4%
2/27 • Number of events 2 • Adverse event reporting begins at the time of T-cell harvest and continues until day 180 post infusion.
General disorders
Flu like symptoms
3.7%
1/27 • Number of events 1 • Adverse event reporting begins at the time of T-cell harvest and continues until day 180 post infusion.
Metabolism and nutrition disorders
Hypoglycemia
3.7%
1/27 • Number of events 1 • Adverse event reporting begins at the time of T-cell harvest and continues until day 180 post infusion.
Vascular disorders
Hypotension
3.7%
1/27 • Number of events 1 • Adverse event reporting begins at the time of T-cell harvest and continues until day 180 post infusion.
General disorders
Hypothermia
3.7%
1/27 • Number of events 1 • Adverse event reporting begins at the time of T-cell harvest and continues until day 180 post infusion.
Infections and infestations
Influenza B
3.7%
1/27 • Number of events 1 • Adverse event reporting begins at the time of T-cell harvest and continues until day 180 post infusion.
General disorders
Infusion site Extravasation
3.7%
1/27 • Number of events 1 • Adverse event reporting begins at the time of T-cell harvest and continues until day 180 post infusion.
Gastrointestinal disorders
Nausea
3.7%
1/27 • Number of events 1 • Adverse event reporting begins at the time of T-cell harvest and continues until day 180 post infusion.
Gastrointestinal disorders
Nausea/Vomiting
3.7%
1/27 • Number of events 1 • Adverse event reporting begins at the time of T-cell harvest and continues until day 180 post infusion.
Investigations
Neutrophil count decreased
11.1%
3/27 • Number of events 3 • Adverse event reporting begins at the time of T-cell harvest and continues until day 180 post infusion.
Investigations
Platelet count decreased
7.4%
2/27 • Number of events 2 • Adverse event reporting begins at the time of T-cell harvest and continues until day 180 post infusion.
Infections and infestations
Pneumonia
14.8%
4/27 • Number of events 5 • Adverse event reporting begins at the time of T-cell harvest and continues until day 180 post infusion.
Cardiac disorders
Sinus bradycardia
3.7%
1/27 • Number of events 1 • Adverse event reporting begins at the time of T-cell harvest and continues until day 180 post infusion.
Skin and subcutaneous tissue disorders
Skin Indurations w/erythema
3.7%
1/27 • Number of events 1 • Adverse event reporting begins at the time of T-cell harvest and continues until day 180 post infusion.
Infections and infestations
Sterile abscess w/drainage
3.7%
1/27 • Number of events 1 • Adverse event reporting begins at the time of T-cell harvest and continues until day 180 post infusion.
Nervous system disorders
Syncope
3.7%
1/27 • Number of events 1 • Adverse event reporting begins at the time of T-cell harvest and continues until day 180 post infusion.
Musculoskeletal and connective tissue disorders
Tendonitis
3.7%
1/27 • Number of events 1 • Adverse event reporting begins at the time of T-cell harvest and continues until day 180 post infusion.

Other adverse events

Other adverse events
Measure
Prevnar, T Cells, Lenalidomide, MAGE A-3
n=27 participants at risk
MAGE-A3, GM-CSF, Hiltonol® (Poly-ICLC), PCV vaccine prior to apheresis. Stem cell mobilization. High-dose melphalan, then hematopoietic stem cells on Day 0. Day 2 Activated/costimulated autologous T cells. Days 14, 42 and 90 MAGE-A3, Hiltonol® and PVC. Day 100 start Lenalidomide. Day 120 and 150 MAGE-A3 and PVC.
Metabolism and nutrition disorders
Anorexia
33.3%
9/27 • Number of events 14 • Adverse event reporting begins at the time of T-cell harvest and continues until day 180 post infusion.
General disorders
Chills
18.5%
5/27 • Number of events 7 • Adverse event reporting begins at the time of T-cell harvest and continues until day 180 post infusion.
Gastrointestinal disorders
Diarrhea
14.8%
4/27 • Number of events 5 • Adverse event reporting begins at the time of T-cell harvest and continues until day 180 post infusion.
General disorders
Fatigue
25.9%
7/27 • Number of events 7 • Adverse event reporting begins at the time of T-cell harvest and continues until day 180 post infusion.
General disorders
Fever
37.0%
10/27 • Number of events 16 • Adverse event reporting begins at the time of T-cell harvest and continues until day 180 post infusion.
Infections and infestations
Flu-like symptoms
7.4%
2/27 • Number of events 3 • Adverse event reporting begins at the time of T-cell harvest and continues until day 180 post infusion.
Musculoskeletal and connective tissue disorders
Generalized muscle weakness
7.4%
2/27 • Number of events 2 • Adverse event reporting begins at the time of T-cell harvest and continues until day 180 post infusion.
Nervous system disorders
Headache
29.6%
8/27 • Number of events 10 • Adverse event reporting begins at the time of T-cell harvest and continues until day 180 post infusion.
Blood and lymphatic system disorders
Increased eosinophil
7.4%
2/27 • Number of events 3 • Adverse event reporting begins at the time of T-cell harvest and continues until day 180 post infusion.
Skin and subcutaneous tissue disorders
Induration
11.1%
3/27 • Number of events 5 • Adverse event reporting begins at the time of T-cell harvest and continues until day 180 post infusion.
Skin and subcutaneous tissue disorders
Induration - thigh
7.4%
2/27 • Number of events 2 • Adverse event reporting begins at the time of T-cell harvest and continues until day 180 post infusion.
Skin and subcutaneous tissue disorders
Induration at injection site
25.9%
7/27 • Number of events 14 • Adverse event reporting begins at the time of T-cell harvest and continues until day 180 post infusion.
Skin and subcutaneous tissue disorders
Induration/Swelling
14.8%
4/27 • Number of events 7 • Adverse event reporting begins at the time of T-cell harvest and continues until day 180 post infusion.
General disorders
Injection site reaction
29.6%
8/27 • Number of events 36 • Adverse event reporting begins at the time of T-cell harvest and continues until day 180 post infusion.
Nervous system disorders
Loss of energy
29.6%
8/27 • Number of events 13 • Adverse event reporting begins at the time of T-cell harvest and continues until day 180 post infusion.
Musculoskeletal and connective tissue disorders
Muscle aches
40.7%
11/27 • Number of events 17 • Adverse event reporting begins at the time of T-cell harvest and continues until day 180 post infusion.
Gastrointestinal disorders
Nausea
18.5%
5/27 • Number of events 6 • Adverse event reporting begins at the time of T-cell harvest and continues until day 180 post infusion.
Investigations
Neutrophil count decreased
7.4%
2/27 • Number of events 2 • Adverse event reporting begins at the time of T-cell harvest and continues until day 180 post infusion.
Gastrointestinal disorders
Pain at injection site
59.3%
16/27 • Number of events 40 • Adverse event reporting begins at the time of T-cell harvest and continues until day 180 post infusion.
Skin and subcutaneous tissue disorders
Pruritis
7.4%
2/27 • Number of events 3 • Adverse event reporting begins at the time of T-cell harvest and continues until day 180 post infusion.
Skin and subcutaneous tissue disorders
Rash
11.1%
3/27 • Number of events 3 • Adverse event reporting begins at the time of T-cell harvest and continues until day 180 post infusion.
Skin and subcutaneous tissue disorders
Rash - lower extremity
7.4%
2/27 • Number of events 2 • Adverse event reporting begins at the time of T-cell harvest and continues until day 180 post infusion.
General disorders
Swelling at injection site
7.4%
2/27 • Number of events 2 • Adverse event reporting begins at the time of T-cell harvest and continues until day 180 post infusion.
Musculoskeletal and connective tissue disorders
Swelling/Induration
25.9%
7/27 • Number of events 9 • Adverse event reporting begins at the time of T-cell harvest and continues until day 180 post infusion.

Additional Information

Liz Veloso

University of Pennsylvania

Phone: 215

Results disclosure agreements

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