Trial Outcomes & Findings for Bortezomib, Cyclophosphamide, and Dexamethasone in Treating Patients With Primary Systemic Light Chain Amyloidosis (NCT NCT01072773)

Results Overview

Response that was confirmed on 2 consecutive evaluations during treatment. Complete Response(CR): Complete disappearance of M-protein from serum and urine on immunofixation, normalization of Free Light Chain (FLC) ratio and \<5% plasma cells in bone marrow. Very Good Partial Response(VGPR): \>=90% reduction in serum M-component; Urine M-Component \<=100 mg per 24 hours. Partial Response(PR): \>=50% reduction in serum M-component and/or Urine M-Component \>=90% reduction or \<200 mg per 24 hours; or \>=50% decrease in difference between involved and uninvolved FLC levels.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

2 participants

Primary outcome timeframe

Duration of treatment (up to 12 cycles/months)

Results posted on

2014-04-02

Participant Flow

Participant milestones

Participant milestones
Measure	Bortez/Cyc/Dex Bortezomib IV on days 1, 8, and 15, oral cyclophosphamide and oral dexamethasone once daily on days 1, 8, 15, and 22.
Overall Study STARTED	2
Overall Study COMPLETED	2
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Bortezomib, Cyclophosphamide, and Dexamethasone in Treating Patients With Primary Systemic Light Chain Amyloidosis

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Bortez/Cyc/Dex n=2 Participants Bortezomib IV on days 1, 8, and 15, oral cyclophosphamide and oral dexamethasone once daily on days 1, 8, 15, and 22.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants
Age, Categorical >=65 years	2 Participants n=5 Participants
Age, Continuous	69 years n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants
Sex: Female, Male Male	0 Participants n=5 Participants
Region of Enrollment United States	2 participants n=5 Participants
Prior Treatment	6.5 Prior Treatments n=5 Participants

PRIMARY outcome

Timeframe: Duration of treatment (up to 12 cycles/months)

Response that was confirmed on 2 consecutive evaluations during treatment. Complete Response(CR): Complete disappearance of M-protein from serum and urine on immunofixation, normalization of Free Light Chain (FLC) ratio and \<5% plasma cells in bone marrow. Very Good Partial Response(VGPR): \>=90% reduction in serum M-component; Urine M-Component \<=100 mg per 24 hours. Partial Response(PR): \>=50% reduction in serum M-component and/or Urine M-Component \>=90% reduction or \<200 mg per 24 hours; or \>=50% decrease in difference between involved and uninvolved FLC levels.

Outcome measures

Outcome measures
Measure	Bortez/Cyc/Dex n=2 Participants Bortezomib IV on days 1, 8, and 15, oral cyclophosphamide and oral dexamethasone once daily on days 1, 8, 15, and 22.
Number of Participants With a Confirmed Hematologic Response Complete Response (CR)	0 participants
Number of Participants With a Confirmed Hematologic Response Very Good Partial Response (VGPR)	0 participants
Number of Participants With a Confirmed Hematologic Response Partial Response (PR)	0 participants

SECONDARY outcome

Timeframe: Duration on treatment (up to 12 cycles/months)

Adverse events (AE) that are classified as either possibly, probably, or definitely related to study treatment according to the National Cancer Institute Common Toxicity Criteria for Adverse Events (NCI CTCAE version 3.0). The maximum grade for each type of AE will be recorded for each patient. Grade refers to the severity of the AE. Grade 1: Mild AE, Grade 2: Moderate AE, Grade 3: Severe AE, Grade 4: Life-threatening or disabling AE, Grade 5: Death related AE Adverse events will be assessed using NCI Common Toxicity Criteria for Adverse Effects (CTCAE) v3.0.

Outcome measures

Outcome measures
Measure	Bortez/Cyc/Dex n=2 Participants Bortezomib IV on days 1, 8, and 15, oral cyclophosphamide and oral dexamethasone once daily on days 1, 8, 15, and 22.
Number of Participants With Treatment Related Adverse Events. Grade 1-2	2 participants
Number of Participants With Treatment Related Adverse Events. Grade 3-4	0 participants
Number of Participants With Treatment Related Adverse Events. Grade 5	0 participants

SECONDARY outcome

Timeframe: Duration on treatment (up to 12 cycles/months)

The number of patients that acheived a response in an affected organ.

Outcome measures

Outcome measures
Measure	Bortez/Cyc/Dex n=2 Participants Bortezomib IV on days 1, 8, and 15, oral cyclophosphamide and oral dexamethasone once daily on days 1, 8, 15, and 22.
Number of Participants With an Organ Response.	0 participants

SECONDARY outcome

Timeframe: Duration of Study (up to 5 years)

Survival time is defined as the time from registration to death due to any cause.

Outcome measures

Outcome measures
Measure	Bortez/Cyc/Dex n=2 Participants Bortezomib IV on days 1, 8, and 15, oral cyclophosphamide and oral dexamethasone once daily on days 1, 8, 15, and 22.
Overall Survival	1.45 Months Interval 1.35 to 1.54

SECONDARY outcome

Timeframe: Duration of Study (up to 5 years)

Time to disease progression is defined as the time from registration to the earliest date of documented disease progression. If a patient dies without a documentation of disease progression the patient will be considered to have had tumor progression at the time of their death

Outcome measures

Outcome measures
Measure	Bortez/Cyc/Dex n=2 Participants Bortezomib IV on days 1, 8, and 15, oral cyclophosphamide and oral dexamethasone once daily on days 1, 8, 15, and 22.
Time to Disease Progression	1.45 Months Interval 1.35 to 1.54

SECONDARY outcome

Timeframe: Duration of Study (up to 5 years)

Population: Neither participant achieved a response. Therefore, no duration of response calculation was performed.

Duration of response will be calculated from the date of first evidence of response until the date of progression in the subset of patients with confirmed hematologic responses.

Outcome measures

Outcome data not reported

Adverse Events

Bortez/Cyc/Dex

Serious events: 1 serious events

Other events: 2 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Bortez/Cyc/Dex n=2 participants at risk Bortezomib IV on days 1, 8, and 15, oral cyclophosphamide and oral dexamethasone once daily on days 1, 8, 15, and 22.
Cardiac disorders Atrial fibrillation	50.0% 1/2 • Number of events 1
Infections and infestations Lung infection	50.0% 1/2 • Number of events 1
Investigations Lymphocyte count decreased	50.0% 1/2 • Number of events 1
Investigations Neutrophil count decreased	50.0% 1/2 • Number of events 1
Investigations White blood cell decreased	50.0% 1/2 • Number of events 1
Respiratory, thoracic and mediastinal disorders Bronchopulmonary hemorrhage	50.0% 1/2 • Number of events 1
Respiratory, thoracic and mediastinal disorders Dyspnea	50.0% 1/2 • Number of events 1
Respiratory, thoracic and mediastinal disorders Respiratory failure	50.0% 1/2 • Number of events 1

Other adverse events

Other adverse events
Measure	Bortez/Cyc/Dex n=2 participants at risk Bortezomib IV on days 1, 8, and 15, oral cyclophosphamide and oral dexamethasone once daily on days 1, 8, 15, and 22.
Gastrointestinal disorders Constipation	50.0% 1/2 • Number of events 1
Gastrointestinal disorders Diarrhea	50.0% 1/2 • Number of events 1
General disorders Edema limbs	100.0% 2/2 • Number of events 2
General disorders Fatigue	100.0% 2/2 • Number of events 2
Infections and infestations Urinary tract infection	50.0% 1/2 • Number of events 1
Investigations Creatinine increased	100.0% 2/2 • Number of events 3
Investigations Platelet count decreased	50.0% 1/2 • Number of events 1
Metabolism and nutrition disorders Hypokalemia	50.0% 1/2 • Number of events 1
Metabolism and nutrition disorders Hyponatremia	50.0% 1/2 • Number of events 1
Nervous system disorders Peripheral motor neuropathy	50.0% 1/2 • Number of events 1

Additional Information

Dr. Shaji Kumar

Mayo Clinic

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place