Trial Outcomes & Findings for Bortezomib Followed by High-Dose Melphalan and Bortezomib as Conditioning Regimen for Tandem Stem Cell Transplants (NCT NCT00307086)

Results Overview

Median overall survival after first peripheral blood stem cell transplant (PBSCT).

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

30 participants

Primary outcome timeframe

40 months post transplant

Results posted on

2017-03-06

Participants recruited from the Blood and Marrow Transplant (BMT) program at H. Lee Moffitt Cancer Center.

Participant milestones
Measure	Autologous PBSCT bortezomib in combination with high-dose melphalan as a conditioning regimen for autologous peripheral blood stem cell transplant Bortezomib : the maximum tolerated dose (MTD) of bortezomib in combination with high-dose melphalan as a conditioning regimen for autologous stem cell transplant PBSCT : PBSCT #1 Day 0 PBSCT #2 Day 0 (approx 90 days =/- 15 days after PBSCT #1) Melphalan : Day -4 melphalan 100 mg/m2 intravenously over 30 minutes, Day -3 melphalan 100 mg/m2 intravenously over 30 minutes
Overall Study STARTED	30
Overall Study COMPLETED	25
Overall Study NOT COMPLETED	5

Reasons for withdrawal
Measure	Autologous PBSCT bortezomib in combination with high-dose melphalan as a conditioning regimen for autologous peripheral blood stem cell transplant Bortezomib : the maximum tolerated dose (MTD) of bortezomib in combination with high-dose melphalan as a conditioning regimen for autologous stem cell transplant PBSCT : PBSCT #1 Day 0 PBSCT #2 Day 0 (approx 90 days =/- 15 days after PBSCT #1) Melphalan : Day -4 melphalan 100 mg/m2 intravenously over 30 minutes, Day -3 melphalan 100 mg/m2 intravenously over 30 minutes
Overall Study ineligible	5

Bortezomib Followed by High-Dose Melphalan and Bortezomib as Conditioning Regimen for Tandem Stem Cell Transplants

Baseline characteristics by cohort
Measure	Autologous PBSCT n=30 Participants bortezomib in combination with high-dose melphalan as a conditioning regimen for autologous peripheral blood stem cell transplant Bortezomib : the maximum tolerated dose (MTD) of bortezomib in combination with high-dose melphalan as a conditioning regimen for autologous stem cell transplant PBSCT : PBSCT #1 Day 0 PBSCT #2 Day 0 (approx 90 days =/- 15 days after PBSCT #1) Melphalan : Day -4 melphalan 100 mg/m2 intravenously over 30 minutes, Day -3 melphalan 100 mg/m2 intravenously over 30 minutes
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	28 Participants n=5 Participants
Age, Categorical >=65 years	2 Participants n=5 Participants
Age, Continuous	55 years STANDARD_DEVIATION 9.26 • n=5 Participants
Sex: Female, Male Female	15 Participants n=5 Participants
Sex: Female, Male Male	15 Participants n=5 Participants
Region of Enrollment United States	30 participants n=5 Participants

Timeframe: 40 months post transplant

Population: All evaluable participants

Median overall survival after first peripheral blood stem cell transplant (PBSCT).

Outcome measures
Measure	Autologous PBSCT n=25 Participants bortezomib in combination with high-dose melphalan as a conditioning regimen for autologous peripheral blood stem cell transplant Bortezomib : the maximum tolerated dose (MTD) of bortezomib in combination with high-dose melphalan as a conditioning regimen for autologous stem cell transplant PBSCT : PBSCT #1 Day 0 PBSCT #2 Day 0 (approx 90 days =/- 15 days after PBSCT #1) Melphalan : Day -4 melphalan 100 mg/m2 intravenously over 30 minutes, Day -3 melphalan 100 mg/m2 intravenously over 30 minutes
Overall Survival (OS)	40 months Interval 32.0 to 40.0

Serious events: 25 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events
Measure	Autologous PBSCT n=25 participants at risk bortezomib in combination with high-dose melphalan as a conditioning regimen for autologous peripheral blood stem cell transplant Bortezomib : the maximum tolerated dose (MTD) of bortezomib in combination with high-dose melphalan as a conditioning regimen for autologous stem cell transplant PBSCT : PBSCT #1 Day 0 PBSCT #2 Day 0 (approx 90 days =/- 15 days after PBSCT #1) Melphalan : Day -4 melphalan 100 mg/m2 intravenously over 30 minutes, Day -3 melphalan 100 mg/m2 intravenously over 30 minutes
Infections and infestations Febrile Neutropenia	52.0% 13/25 • 2 years post transplant
Infections and infestations Bacteremia	24.0% 6/25 • 2 years post transplant
Metabolism and nutrition disorders Hypokalaemia	28.0% 7/25 • 2 years post transplant
Gastrointestinal disorders Mucositis	16.0% 4/25 • 2 years post transplant
Metabolism and nutrition disorders Hypophosphatemia	12.0% 3/25 • 2 years post transplant
Respiratory, thoracic and mediastinal disorders Pneumonia	8.0% 2/25 • 2 years post transplant

Adverse event data not reported

H. Lee Moffitt Cancer Center

Phone: 813-745-6886