Study of Dexamethasone Plus IXAZOMIB (MLN9708) or Physicians Choice of Treatment in Relapsed or Refractory Systemic Light Chain (AL) Amyloidosis
Summary
The purpose of this study is to provide continued access of ixazomib and/or other study medications and to continue collecting relevant safety data to monitor participant's safety, determine whether dexamethasone plus IXAZOMIB improves hematologic response, 2-year vital organ (that is, heart or kidney) deterioration and mortality rate versus a physician's choice of a chemotherapy regimen in participants diagnosed with relapsed or refractory systemic light chain (AL) amyloidosis.
Timeline
- Start
- 2012-12-26
- Primary completion
- 2022-07-11
- Completion
- 2022-07-11
Outcome
Missed primary endpoint
Stopped: “Sponsor's decision”
registry analysis (superiority test); Arm A: Ixazomib + Dexamethasone vs Arm B: Dexamethasone + Melphalan vs Arm B: Dexamethasone + Cyclophosphamide vs Arm B: Dexamethasone + Thalidomide vs Arm B: Dexamethasone + Lenalidomide; p =0.7623 NCT01659658 ↗
registry analysis (superiority test); Arm A: Ixazomib + Dexamethasone vs Arm B: Dexamethasone + Melphalan/Cyclophosphamide/Thalidomide/Lenalidomide; p =0.3510 NCT01659658 ↗
release “The TOURMALINE-AL1 trial did not meet the first of the two primary endpoints of significant improvement in overall hematologic response, as reported in June 2019.” takeda.com ↗
Publications
- Sanchorawala V, Wechalekar AD, Kim K, Schonland SO, Landau HJ, Kwok F, Suzuki K, Dispenzieri A, Merlini G, Comenzo RL, Cherepanov D, Hayden VC, Kumar A, Labotka R, Faller DV, Kastritis E. Quality of life and symptoms among patients with relapsed/refractory AL amyloidosis treated with ixazomib-dexamethasone versus physician's choice. Am J Hematol. 2023 May;98(5):720-729. doi: 10.1002/ajh.26866. Epub 2023 Feb 14.
- Sanchorawala V, Palladini G, Kukreti V, Zonder JA, Cohen AD, Seldin DC, Dispenzieri A, Jaccard A, Schonland SO, Berg D, Yang H, Gupta N, Hui AM, Comenzo RL, Merlini G. A phase 1/2 study of the oral proteasome inhibitor ixazomib in relapsed or refractory AL amyloidosis. Blood. 2017 Aug 3;130(5):597-605. doi: 10.1182/blood-2017-03-771220. Epub 2017 May 26.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Comparator | Cyclophosphamide | Other / unclassified | 500 mg | Oral |
| Subject | Ixazomib | Unknown | 4 mg | Oral |
| Comparator | Lenalidomide | Other / unclassified | 15 mg | Oral |
| Comparator | Melphalan | Small molecule | 0.22 mg/kg | Oral |
| Comparator | Thalidomide | Other / unclassified | 200 mg | Oral |
| Background | Dexamethasone | Small molecule | 20 mg | Oral |