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NCT01842308

Carfilzomib and Melphalan Before Stem Cell Transplant in Treating Patients With Multiple Myeloma

Completed Phase 1, PHASE2 Results posted Last updated 1 September 2020
What this trial tests

Phase 1, PHASE2 trial testing Autologous Bone Marrow Transplantation in DS Stage I Plasma Cell Myeloma in 50 participants. Completed in 28 October 2019.

Timeline
4 June 2013
Primary endpoint
11 October 2017
28 October 2019

Quick facts

Lead sponsorMayo Clinic
PhasePhase 1, PHASE2
StatusCompleted
Study typeINTERVENTIONAL
Allocationna
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment50
Start date4 June 2013
Primary completion11 October 2017
Estimated completion28 October 2019
Sites2 locations across United States

Drugs / interventions tested

Conditions studied

Sponsor

Mayo Clinic

Who can join

18 and older, any sex, with DS Stage I Plasma Cell Myeloma or DS Stage II Plasma Cell Myeloma. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Determine Maximum Tolerated Dose by the Number of Patients With a DLT Per Dose Level Primary · Up to day 30

Will be defined as the dose level below the lowest dose that induces dose-limiting toxicity(DLT) in at least one-third of patients. For this study, a DLT is any of the following during the first 2 cycles of treatment; Absolute neutrophil count engraftment\* delayed beyond day 21 or Platelet engraftment\* delayed beyond day 30, grade 3+ related sensory or motor neuropathy, or grade 4+ related non-neurologic or non-hematologic adverse event(excluding nausea, vomiting, and diarrhea. Reported below are the number of patients who experienced a DLT.

GroupValue95% CI
Phase 1: Dose Level 31
Phase 1: Dose Level 20
Phase 1: Dose Level 10
Phase 1: Dose Level 00
Percentage of Patients With Complete Responses, Defined as a Complete Response Noted as the Objective Status on Two Consecutive Evaluations (Phase II) Primary · Up to 5 years

The percentage of successes will be estimated by the number of successes divided by the total number of evaluable patients. Exact binomial 95% confidence intervals for the true success percentages will be calculated.

GroupValue95% CI
Dose Level 321.9510.56 – 37.61
Adverse Event Rate, Graded According to National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0 Secondary · Up to 5 years

These results are reported in the adverse events section. The maximum grade for each type of adverse event will be recorded for each patient, and frequency tables will be reviewed to determine patterns. Additionally, the relationship of the adverse event(s) to the study treatment will be taken into consideration.

GroupValue95% CI
Phase 1: Dose Level 36
Phase 1: Dose Level 23
Phase 1: Dose Level 13
Phase 1: Dose Level 02
Phase 2: Dose Level 335
Complete Response Rate at Day 100 Secondary · At day 100

Complete response rate (CRR) is defined as the percentage of complete responses estimated by the total number of patients who achieve a complete response by day 100 post-transplant divided by the total number of evaluable patients. Exact binomial 95% confidence intervals for the true complete response rate at day 100 will be calculated.

GroupValue95% CI
Dose Level 317.077.15 – 32.06
Progression Free Percentage at 1 and 2 Years Post Registration Secondary · At 1 year and 2 years

Progression is an Increase of 25% from lowest value in any of the following (A "25% increase" refers to M protein, FLC and bone marrow results and does not refer to bone lesions, soft tissue plasmacytoma or hypercalcemia. The lowest value does not need to be a confirmed value. If the lowest serum Mprotein is ≥ 5 g/dL, an increase in serum M-protein of ≥ 1 g/dL is sufficient to define disease progression.), (In the case where a value is felt to be a spurious result per physician discretion (for example, a possible lab error), that value will not be considered when determining the lowest value.)

1 year
GroupValue95% CI
Dose Level 390.2476.87 – 97.28
2 years
GroupValue95% CI
Dose Level 375.6159.70 – 87.64

Adverse events — posted to ClinicalTrials.gov

Time frame: 5 years. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Phase 1; Dose Level 3
Serious: 3/6 (50%)
Deaths: 0/6
Phase 1; Dose Level 2
Serious: 2/3 (67%)
Deaths: 0/3
Phase 1; Dose Level 1
Serious: 1/3 (33%)
Deaths: 0/3
Phase 1; Dose Level 0
Serious: 2/2 (100%)
Deaths: 0/2
Phase 2
Serious: 16/35 (46%)
Deaths: 0/35

Serious adverse events (33 terms)

ReactionSystemPhase 1; Dose Level 3Phase 1; Dose Level 2Phase 1; Dose Level 1Phase 1; Dose Level 0Phase 2
NauseaGastrointestinal disorders
VomitingGastrointestinal disorders
Aspartate aminotransferase increasedInvestigations
Creatinine increasedInvestigations
PresyncopeNervous system disorders
SyncopeNervous system disorders
HypoxiaRespiratory, thoracic and mediastinal disorders
AnemiaBlood and lymphatic system disorders
Atrial fibrillationCardiac disorders
Sinus bradycardiaCardiac disorders
Supraventricular tachycardiaCardiac disorders
Abdominal painGastrointestinal disorders
ColitisGastrointestinal disorders
Mucositis oralGastrointestinal disorders
FatigueGeneral disorders
FeverGeneral disorders
CholecystitisHepatobiliary disorders
Infections and infestations - Other, specifyInfections and infestations
Upper respiratory infectionInfections and infestations
Spinal fractureInjury, poisoning and procedural complications
Alanine aminotransferase increasedInvestigations
Alkaline phosphatase increasedInvestigations
Blood bilirubin increasedInvestigations
White blood cell decreasedInvestigations
DehydrationMetabolism and nutrition disorders
Other adverse events (46 terms — click to expand)

ReactionSystemPhase 1; Dose Level 3Phase 1; Dose Level 2Phase 1; Dose Level 1Phase 1; Dose Level 0Phase 2
Platelet count decreasedInvestigations
Lymphocyte count decreasedInvestigations
White blood cell decreasedInvestigations
AnemiaBlood and lymphatic system disorders
Neutrophil count decreasedInvestigations
Peripheral sensory neuropathyNervous system disorders
HypotensionVascular disorders
AlopeciaSkin and subcutaneous tissue disorders
FatigueGeneral disorders
Creatinine increasedInvestigations
HypophosphatemiaMetabolism and nutrition disorders
HypokalemiaMetabolism and nutrition disorders
NauseaGastrointestinal disorders
VomitingGastrointestinal disorders
DiarrheaGastrointestinal disorders
AnorexiaMetabolism and nutrition disorders
HyperglycemiaMetabolism and nutrition disorders
HypocalcemiaMetabolism and nutrition disorders
Febrile neutropeniaBlood and lymphatic system disorders
Peripheral motor neuropathyNervous system disorders
EsophagitisGastrointestinal disorders
Mucositis oralGastrointestinal disorders
Blood bilirubin increasedInvestigations
HypoalbuminemiaMetabolism and nutrition disorders
HypertensionVascular disorders
Gastroesophageal reflux diseaseGastrointestinal disorders
FeverGeneral disorders
CD4 lymphocytes decreasedInvestigations
DehydrationMetabolism and nutrition disorders
Atrial fibrillationCardiac disorders
Cardiac disorders - Other, specifyCardiac disorders
Abdominal painGastrointestinal disorders
DyspepsiaGastrointestinal disorders
Gastrointestinal disorders - Other, specifyGastrointestinal disorders
PainGeneral disorders
Infections and infestations - Other, specifyInfections and infestations
Lung infectionInfections and infestations
Weight lossInvestigations
HyponatremiaMetabolism and nutrition disorders
Bone painMusculoskeletal and connective tissue disorders

Most-reported serious reactions: Nausea, Vomiting, Aspartate aminotransferase increased, Creatinine increased, Presyncope, Syncope, Hypoxia, Anemia.

Data from ClinicalTrials.gov NCT01842308 adverse events section.

Sponsor's own description

This phase I/II trial studies the side effects and best dose of carfilzomib when given together with melphalan and to see how well they work in treating patients with multiple myeloma before stem cell transplant. Carfilzomib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as melphalan, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Giving carfilzomib together with melphalan may kill more cancer cells.

Publications & conference data

No peer-reviewed publications indexed yet for this trial. Completed trials usually publish results within 12-18 months.

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Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT01842308.

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