Last reviewed · How we verify

NCT01864018

Ixazomib With Cyclophosphamide and Dexamethasone in Patients With Previously Untreated Symptomatic Multiple Myeloma or Light Chain Amyloidosis

Completed Phase 1, PHASE2 Results posted Last updated 24 July 2025
What this trial tests

Phase 1, PHASE2 trial testing Cyclophosphamide in Amyloidosis in 87 participants. Completed in 9 September 2023.

Timeline
20 August 2013
Primary endpoint
30 April 2021
9 September 2023

Quick facts

Lead sponsorMayo Clinic
PhasePhase 1, PHASE2
StatusCompleted
Study typeINTERVENTIONAL
Allocationna
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment87
Start date20 August 2013
Primary completion30 April 2021
Estimated completion9 September 2023
Sites2 locations across United States

Drugs / interventions tested

Conditions studied

Sponsor

Mayo Clinic

Who can join

18 and older, any sex, with Amyloidosis or 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.

Maximum Tolerated (MTD) Dose of Cyclophosphamide With Ixazomib and Dexamethasone (Phase I) Primary · At 28 days

Will be defined as the dose level below the lowest dose that induces dose-limiting toxicity in at least 1/3 of patients, as assessed by National Cancer Institute Common Terminology Criteria for Adverse Events version 4.0.

MTD of ixazomib
GroupValue95% CI
Treatment (Ixazomib Citrate, Cyclophosphamide, Dexamethasone)4
MTD of dexamethasone
GroupValue95% CI
Treatment (Ixazomib Citrate, Cyclophosphamide, Dexamethasone)40
Percentage of Patients With Complete Response or Very Good Partial Response (Phase II, Cohort A) Primary · Up to 48 weeks

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 proportion will be calculated.

GroupValue95% CI
Treatment (Ixazomib Citrate, Cyclophosphamide, Dexamethasone)3522 – 50
Rate of Complete Response, Very Good Partial Response, or Partial Response (Phase II, Cohort B) Primary · Up to 48 weeks

The proportion 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 proportion will be calculated.

GroupValue95% CI
Treatment (Ixazomib Citrate, Cyclophosphamide, Dexamethasone)6345 – 79
Number of Patients Experiencing a Grade 3 or Greater Adverse Event at Least Possibly Related to Treatment as Assessed by the National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0 Secondary · Up to 5 years

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. Adverse events will be graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events version 4.0. A grade 3 event is one that is severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self care ADL.

GroupValue95% CI
Phase I/II, Cohort A36
Phase II, Cohort B16
Progression-free Survival (PFS) Secondary · Time from registration to the earliest date of documentation of disease progression or death due to any cause, assessed up to 5 years

The distribution of PFS will be estimated using the method of Kaplan Meier.

GroupValue95% CI
Phase I/II, Cohort ANA31.3 – NA
Phase II, Cohort BNA39.6 – NA
Survival Time Secondary · Time from registration to death due to any cause, assessed up to 5 years

The distribution of survival time will be estimated using the method of Kaplan-Meier.

GroupValue95% CI
Phase I/II, Cohort ANANA – NA
Phase II, Cohort BNANA – NA
Maximum Tolerated (MTD) Dose of Cyclophosphamide (Phase I) Primary · At 28 days

Will be defined as the dose level below the lowest dose that induces dose-limiting toxicity in at least 1/3 of patients, as assessed by National Cancer Institute Common Terminology Criteria for Adverse Events version 4.0.

GroupValue95% CI
Treatment (Ixazomib Citrate, Cyclophosphamide, Dexamethasone)400

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 I/II, Cohort A
Serious: 16/48 (33%)
Deaths: 2/48
Phase II, Cohort B
Serious: 15/35 (43%)
Deaths: 7/35

Serious adverse events (53 terms)

ReactionSystemPhase I/II, Cohort APhase II, Cohort B
Lymphocyte count decreasedInvestigations
Atrial fibrillationCardiac disorders
Heart failureCardiac disorders
Lung infectionInfections and infestations
Neutrophil count decreasedInvestigations
Platelet count decreasedInvestigations
White blood cell decreasedInvestigations
HyperglycemiaMetabolism and nutrition disorders
SyncopeNervous system disorders
DyspneaRespiratory, thoracic and mediastinal disorders
Rash maculo-papularSkin and subcutaneous tissue disorders
AnemiaBlood and lymphatic system disorders
Cardiac arrestCardiac disorders
Chest pain - cardiacCardiac disorders
Pericardial effusionCardiac disorders
PericarditisCardiac disorders
Ventricular tachycardiaCardiac disorders
DiarrheaGastrointestinal disorders
Gastric hemorrhageGastrointestinal disorders
PancreatitisGastrointestinal disorders
Death NOSGeneral disorders
FatigueGeneral disorders
CholecystitisHepatobiliary disorders
Allergic reactionImmune system disorders
Infections and infestations - Oth specInfections and infestations
Other adverse events (61 terms — click to expand)

ReactionSystemPhase I/II, Cohort APhase II, Cohort B
FatigueGeneral disorders
Lymphocyte count decreasedInvestigations
White blood cell decreasedInvestigations
ConstipationGastrointestinal disorders
Peripheral sensory neuropathyNervous system disorders
Platelet count decreasedInvestigations
DiarrheaGastrointestinal disorders
NauseaGastrointestinal disorders
Neutrophil count decreasedInvestigations
Rash maculo-papularSkin and subcutaneous tissue disorders
Creatinine increasedInvestigations
AnemiaBlood and lymphatic system disorders
VomitingGastrointestinal disorders
Peripheral motor neuropathyNervous system disorders
InsomniaPsychiatric disorders
Edema limbsGeneral disorders
CD4 lymphocytes decreasedInvestigations
Lymphocyte count increasedInvestigations
DyspneaRespiratory, thoracic and mediastinal disorders
HyperglycemiaMetabolism and nutrition disorders
SepsisInfections and infestations
Gastrointestinal disorders - Oth specGastrointestinal disorders
AnorexiaMetabolism and nutrition disorders
MyalgiaMusculoskeletal and connective tissue disorders
Thromboembolic eventVascular disorders
Febrile neutropeniaBlood and lymphatic system disorders
Chest pain - cardiacCardiac disorders
Heart failureCardiac disorders
Hearing impairedEar and labyrinth disorders
CataractEye disorders
Duodenal ulcerGastrointestinal disorders
DyspepsiaGastrointestinal disorders
Gastroesophageal reflux diseaseGastrointestinal disorders
Edema trunkGeneral disorders
PainGeneral disorders
Infections and infestations - Oth specInfections and infestations
Lung infectionInfections and infestations
Upper respiratory infectionInfections and infestations
Alkaline phosphatase increasedInvestigations
HyperuricemiaMetabolism and nutrition disorders

Most-reported serious reactions: Lymphocyte count decreased, Atrial fibrillation, Heart failure, Lung infection, Neutrophil count decreased, Platelet count decreased, White blood cell decreased, Hyperglycemia.

Data from ClinicalTrials.gov NCT01864018 adverse events section.

Sponsor's own description

This phase I/II trial studies the side effects and the best dose of cyclophosphamide when given together with ixazomib citrate and dexamethasone in treating patients with previously untreated symptomatic multiple myeloma or light chain amyloidosis. Drugs used in chemotherapy, such as cyclophosphamide and dexamethasone, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Ixazomib citrate may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving cyclophosphamide together with ixazomib citrate and dexamethasone may be a better treatment for multiple myeloma or light chain amyloidosis.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Spotlight on ixazomib: potential in the treatment of multiple myeloma.
    Muz B, Ghazarian RN, Ou M, Luderer MJ, et al · · 2016 · cited 87× · PMID 26811670 · DOI 10.2147/dddt.s93602
  2. Immunoglobulin light chain amyloidosis diagnosis and treatment algorithm 2018.
    Gertz MA. · · 2018 · cited 59× · PMID 29795248 · DOI 10.1038/s41408-018-0080-9
  3. Light Chain Amyloidosis.
    Milani P, Merlini G, Palladini G. · · 2018 · cited 54× · PMID 29531659 · DOI 10.4084/mjhid.2018.022
  4. An evidence-based review of ixazomib citrate and its potential in the treatment of newly diagnosed multiple myeloma.
    Offidani M, Corvatta L, Caraffa P, Gentili S, et al · · 2014 · cited 40× · PMID 25302026 · DOI 10.2147/ott.s49187
  5. Immunoglobulin light chain amyloidosis diagnosis and treatment algorithm 2021.
    Hasib Sidiqi M, Gertz MA. · · 2021 · cited 35× · PMID 33993188 · DOI 10.1038/s41408-021-00483-7
  6. Trends in patient-reported outcome use in early phase dose-finding oncology trials - an analysis of ClinicalTrials.gov.
    Lai-Kwon J, Yin Z, Minchom A, Yap C. · · 2021 · cited 23× · PMID 34676991 · DOI 10.1002/cam4.4307
  7. Trial Watch: Proteasomal inhibitors for anticancer therapy.
    Obrist F, Manic G, Kroemer G, Vitale I, et al · · 2015 · cited 17× · PMID 27308423 · DOI 10.4161/23723556.2014.974463
  8. Phase 1/2 trial of ixazomib, cyclophosphamide and dexamethasone in patients with previously untreated symptomatic multiple myeloma.
    Kumar SK, Buadi FK, LaPlant B, Halvorson A, et al · · 2018 · cited 15× · PMID 30061664 · DOI 10.1038/s41408-018-0106-3

Verify or expand the search:

Other trials of Cyclophosphamide

Trials testing the same drug.

Other recruiting trials for Amyloidosis

Currently open trials in the same condition.

Other Mayo Clinic trials

Trials by the same sponsor.

Verify against primary sources

Data sources for this page

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/NCT01864018.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing