Trial Outcomes & Findings for Escalating Doses of Thalidomide in Conjunction With Bortezomib and HIgh Dose Melphalan for BSCT (NCT NCT01242267)

Last Updated: 2023-01-03

Results Overview

Maximum tolerated dose of thalidomide used in conjunction with dose-intense melphalan, bortezomib and autologous (syngeneic) HSC support in the salvage therapy of patients who failed a prior treatment with dose-intense melphalan

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

29 participants

Primary outcome timeframe

Dose escalation will be based on the assessment of tolerability determined after the last patient of each cohort reaches day +21.

Results posted on

2023-01-03

Participant Flow

Participant milestones

Participant milestones
Measure	Phase I - Thalidomide Dose Level 600 mg Phase I - Thalidomide Dose Level 600 mg	Phase I - Thalidomide Dose Level 800 mg Phase I - Thalidomide Dose Level 800 mg	Phase I - Thalidomide Dose Level 1000 mg Phase I - Thalidomide Dose Level 1000 mg	Phase 2 Phase 2 Thalidomide Dose Level 1000 mg
Overall Study STARTED	3	3	3	20
Overall Study COMPLETED	3	3	3	20
Overall Study NOT COMPLETED	0	0	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Escalating Doses of Thalidomide in Conjunction With Bortezomib and HIgh Dose Melphalan for BSCT

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Phase I - Thalidomide Dose Level 600 mg n=3 Participants Phase I - Thalidomide Dose Level 600 mg	Phase I - Thalidomide Dose Level 800 mg n=3 Participants Phase I - Thalidomide Dose Level 800 mg	Phase I - Thalidomide Dose Level 1000 mg n=3 Participants Phase I - Thalidomide Dose Level 1000 mg	Phase II n=20 Participants Phase II Thalidomide Dose Level 1000 mg	Total n=29 Participants Total of all reporting groups
Age, Customized Range	66 years n=93 Participants	59 years n=4 Participants	52 years n=27 Participants	55 years n=483 Participants	56 years n=36 Participants
Sex: Female, Male Female	1 Participants n=93 Participants	2 Participants n=4 Participants	3 Participants n=27 Participants	9 Participants n=483 Participants	15 Participants n=36 Participants
Sex: Female, Male Male	2 Participants n=93 Participants	1 Participants n=4 Participants	0 Participants n=27 Participants	11 Participants n=483 Participants	14 Participants n=36 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=93 Participants	0 Participants n=4 Participants	1 Participants n=27 Participants	1 Participants n=483 Participants	2 Participants n=36 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	3 Participants n=93 Participants	3 Participants n=4 Participants	2 Participants n=27 Participants	19 Participants n=483 Participants	27 Participants n=36 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants	0 Participants n=483 Participants	0 Participants n=36 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants	0 Participants n=483 Participants	0 Participants n=36 Participants
Race (NIH/OMB) Asian	1 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants	0 Participants n=483 Participants	1 Participants n=36 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants	0 Participants n=483 Participants	0 Participants n=36 Participants
Race (NIH/OMB) Black or African American	0 Participants n=93 Participants	0 Participants n=4 Participants	1 Participants n=27 Participants	4 Participants n=483 Participants	5 Participants n=36 Participants
Race (NIH/OMB) White	2 Participants n=93 Participants	3 Participants n=4 Participants	2 Participants n=27 Participants	14 Participants n=483 Participants	21 Participants n=36 Participants
Race (NIH/OMB) More than one race	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants	0 Participants n=483 Participants	0 Participants n=36 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants	2 Participants n=483 Participants	2 Participants n=36 Participants
Region of Enrollment United States	3 participants n=93 Participants	3 participants n=4 Participants	3 participants n=27 Participants	20 participants n=483 Participants	29 participants n=36 Participants

PRIMARY outcome

Timeframe: Dose escalation will be based on the assessment of tolerability determined after the last patient of each cohort reaches day +21.

Outcome measures

Outcome measures
Measure	Phase 1 n=9 Participants Phase 1 Dose Level 600, 800, 1000 mg of Thalidomide	Phase 2 n=20 Participants Phase 2 Dose Level 1000 mg of Thalidomide	Phase I - Thalidomide Dose Level 1000 mg Phase I - Thalidomide Dose Level 1000 mg	Phase 2 Phase 2 Thalidomide Dose Level 1000 mg
Maximum Tolerated Dose of Thalidomide	1000 mg	1000 mg	—	—

SECONDARY outcome

Timeframe: Assessments will be made from Day +28 after transplantation for 3 months and then at 3 month intervals until demonstration of disease progression and initiation of further therapy, an average of 9 months

The complete response (CR) and very good partial response (VgPR) rate in patients undergoing ASCT using thalidomide, bortezomib and melphalan

Outcome measures

Outcome measures
Measure	Phase 1 n=3 Participants Phase 1 Dose Level 600, 800, 1000 mg of Thalidomide	Phase 2 n=3 Participants Phase 2 Dose Level 1000 mg of Thalidomide	Phase I - Thalidomide Dose Level 1000 mg n=3 Participants Phase I - Thalidomide Dose Level 1000 mg	Phase 2 n=20 Participants Phase 2 Thalidomide Dose Level 1000 mg
Complete Response (CR) and Very Good Partial Response (VgPR) Rate	0 Participants	3 Participants	1 Participants	13 Participants

SECONDARY outcome

Timeframe: Patients will be hospitalized or in the outpatient transplant facility until engraftment and resolution of serious adverse events, a median of 16 (range, 11-24) days

Assessment of the toxicities resulting from administration of combinations of thalidomide, bortezomib and melphalan

Outcome measures

Outcome measures
Measure	Phase 1 n=3 Participants Phase 1 Dose Level 600, 800, 1000 mg of Thalidomide	Phase 2 n=3 Participants Phase 2 Dose Level 1000 mg of Thalidomide	Phase I - Thalidomide Dose Level 1000 mg n=3 Participants Phase I - Thalidomide Dose Level 1000 mg	Phase 2 n=20 Participants Phase 2 Thalidomide Dose Level 1000 mg
Toxicity Assessment	1 Serious Adverse Events	0 Serious Adverse Events	0 Serious Adverse Events	3 Serious Adverse Events

SECONDARY outcome

Timeframe: PFS was defined as the time from ASCT to disease progression or death from any cause, an average of 9 months

Population: Data no longer available, only manuscript information where PFS reported for single Arm/Group of all participants

The treatment-free interval after treatment with the combination of thalidomide, bortezomib and melphalan

Outcome measures

Outcome measures
Measure	Phase 1 n=29 Participants Phase 1 Dose Level 600, 800, 1000 mg of Thalidomide	Phase 2 Phase 2 Dose Level 1000 mg of Thalidomide	Phase I - Thalidomide Dose Level 1000 mg Phase I - Thalidomide Dose Level 1000 mg	Phase 2 Phase 2 Thalidomide Dose Level 1000 mg
Treatment Free Interval/PFS	9.3 months Standard Deviation 0	—	—	—

Adverse Events

Phase I - Thalidomide Dose Level 600 mg

Serious events: 1 serious events

Other events: 3 other events

Deaths: 0 deaths

Phase I - Thalidomide Dose Level 800 mg

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

Phase I - Thalidomide Dose Level 1000 mg

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

Phase 2

Serious events: 3 serious events

Other events: 20 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Phase I - Thalidomide Dose Level 600 mg n=3 participants at risk Phase I - Thalidomide Dose Level 600 mg	Phase I - Thalidomide Dose Level 800 mg n=3 participants at risk Phase I - Thalidomide Dose Level 800 mg	Phase I - Thalidomide Dose Level 1000 mg n=3 participants at risk Phase I - Thalidomide Dose Level 1000 mg	Phase 2 n=20 participants at risk Phase 2 Thalidomide Dose Level 1000 mg
Blood and lymphatic system disorders Engraftment Syndrome with GI Invovlement	33.3% 1/3 • Number of events 1 The limited number of patients limited our ability to analyze groups separately. As a result, all subjects in the Phase I portion were combined to produce a meaningful analysis.	0.00% 0/3 The limited number of patients limited our ability to analyze groups separately. As a result, all subjects in the Phase I portion were combined to produce a meaningful analysis.	0.00% 0/3 The limited number of patients limited our ability to analyze groups separately. As a result, all subjects in the Phase I portion were combined to produce a meaningful analysis.	0.00% 0/20 The limited number of patients limited our ability to analyze groups separately. As a result, all subjects in the Phase I portion were combined to produce a meaningful analysis.
Immune system disorders Rheumatoid Arthritis Flare	0.00% 0/3 The limited number of patients limited our ability to analyze groups separately. As a result, all subjects in the Phase I portion were combined to produce a meaningful analysis.	0.00% 0/3 The limited number of patients limited our ability to analyze groups separately. As a result, all subjects in the Phase I portion were combined to produce a meaningful analysis.	0.00% 0/3 The limited number of patients limited our ability to analyze groups separately. As a result, all subjects in the Phase I portion were combined to produce a meaningful analysis.	5.0% 1/20 • Number of events 1 The limited number of patients limited our ability to analyze groups separately. As a result, all subjects in the Phase I portion were combined to produce a meaningful analysis.
Gastrointestinal disorders Diarrhea	0.00% 0/3 The limited number of patients limited our ability to analyze groups separately. As a result, all subjects in the Phase I portion were combined to produce a meaningful analysis.	0.00% 0/3 The limited number of patients limited our ability to analyze groups separately. As a result, all subjects in the Phase I portion were combined to produce a meaningful analysis.	0.00% 0/3 The limited number of patients limited our ability to analyze groups separately. As a result, all subjects in the Phase I portion were combined to produce a meaningful analysis.	5.0% 1/20 • Number of events 1 The limited number of patients limited our ability to analyze groups separately. As a result, all subjects in the Phase I portion were combined to produce a meaningful analysis.
Metabolism and nutrition disorders Tumor Lysis Syndrome	0.00% 0/3 The limited number of patients limited our ability to analyze groups separately. As a result, all subjects in the Phase I portion were combined to produce a meaningful analysis.	0.00% 0/3 The limited number of patients limited our ability to analyze groups separately. As a result, all subjects in the Phase I portion were combined to produce a meaningful analysis.	0.00% 0/3 The limited number of patients limited our ability to analyze groups separately. As a result, all subjects in the Phase I portion were combined to produce a meaningful analysis.	5.0% 1/20 • Number of events 1 The limited number of patients limited our ability to analyze groups separately. As a result, all subjects in the Phase I portion were combined to produce a meaningful analysis.

Other adverse events

Additional Information

Joshua Zenreich

Hackensack Meridan Health

Phone: 5519964248

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place