Trial reportBlood2017
A phase 1/2 study of the oral proteasome inhibitor ixazomib in relapsed or refractory AL amyloidosis.
Trial report in Blood, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. It is linked to 2 registered trials, which are not on this map. Cited by 57 papers, 2 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
An Open-Label, Dose-Escalation, Phase 1 Study of the Oral Formulation of MLN9708 Administered Weekly in Adult Patients With Relapsed or Refractory Light-Chain (AL) Amyloidosis Who Require Further Treatment
A Phase 3, Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of Dexamethasone Plus MLN9708 or Physicians Choice of Treatment Administered to Patients With Relapsed or Refractory Systemic Light Chain (AL) Amyloidosis
Who cites it
57 citing papers in PubMed, 2 syntheses or guidelines pooled it, 122 citations in OpenAlex.
- Cardiac Toxicity in the Treatment of Light Chain Amyloidosis: Systematic Review of Clinical Studies.Current drug safety · 2024Pooled it
- The proteasome as a druggable target with multiple therapeutic potentialities: Cutting and non-cutting edges.Pharmacology & therapeutics · 2020Pooled it
- Phase 1/2 study of ixazomib with cyclophosphamide and dexamethasone in newly diagnosed AL amyloidosis.Blood advances · 2022Trial
- Phase 2 trial of ixazomib, cyclophosphamide, and dexamethasone for previously untreated light chain amyloidosis.Blood advances · 2022Trial
- A randomized phase 3 study of ixazomib-dexamethasone versus physician's choice in relapsed or refractory AL amyloidosis.Leukemia · 2022Trial
- Marked progress in AL amyloidosis survival: a 40-year longitudinal natural history study.Blood cancer journal · 2021Trial
- Bendamustine With Dexamethasone in Relapsed/Refractory Systemic Light-Chain Amyloidosis: Results of a Phase II Study.Journal of clinical oncology : official journal of the American Society of Clinical Oncology · 2020Trial
- Trial
- Dose and Schedule Selection of the Oral Proteasome Inhibitor Ixazomib in Relapsed/Refractory Multiple Myeloma: Clinical and Model-Based Analyses.Targeted oncology · 2017Trial
- Amyloid Light Chain Proteins in Cardiovascular Disease: Pathogenesis and Emerging Therapies for Cardiac Amyloidosis.Pharmaceutical research · 2025Review
- High-dose melphalan-autologous hematopoietic cell transplantation in systemic AL amyloidosis following daratumumab-based frontline therapy.Bone marrow transplantation · 2024Article
- Roles of the gut microbiota in human neurodevelopment and adult brain disorders.Frontiers in neuroscience · 2024Review
- Safety and efficacy of teclistamab in systemic immunoglobulin light chain amyloidosis.Blood cancer journal · 2023Article
- Echocardiographic phenotype for refined risk stratification and treatment selection in light chain amyloidosis with heart failure.Journal of cancer research and clinical oncology · 2023Article
- Molecular bottlebrush prodrugs as mono- and triplex combination therapies for multiple myeloma.Nature nanotechnology · 2023Article
- The management of light chain (AL) amyloidosis in Europe: clinical characteristics, treatment patterns, and efficacy outcomes between 2004 and 2018.Blood cancer journal · 2023Observational
- Cardiovascular toxicity from therapies for light chain amyloidosis.Frontiers in cardiovascular medicine · 2023Review
- Contrasting presentations of the same disease: A comparison of two cases of amyloidosis presenting with eyelid involvement.American journal of ophthalmology case reports · 2022Article
- Article
- The Role of Microbiome in Brain Development and Neurodegenerative Diseases.Molecules (Basel, Switzerland) · 2022Review
Corrections and comments
- Erratum issued
Authors and funding
15 authors at 12 institutions in 5 countries.
Funding
Abstract
This phase 1/2 study assessed the safety, tolerability, and preliminary efficacy of the oral proteasome inhibitor (PI) ixazomib in patients with relapsed/refractory immunoglobulin light chain (AL) amyloidosis. Ixazomib was administered to adult patients with relapsed/refractory AL amyloidosis after 1 or more prior lines of therapy (including bortezomib) on days 1, 8, and 15 of 28-day cycles, for up to 12 cycles. Patients with less than partial response after 3 cycles received oral dexamethasone (40 mg, days 1-4) from cycle 4. A 3+3 dose-escalation phase was followed by 2 expansion cohorts (PI-naive and PI-exposed patients) at the maximum tolerated dose (MTD). Twenty-seven patients were enrolled: 11 during dose escalation (6 at 4.0 mg and 5 at 5.5 mg) and 16 during dose expansion (4.0 mg). Three patients experienced dose-limiting toxicities: 1 at 4.0 mg and 2 at 5.5 mg; the MTD was determined as 4.0 mg. Most common adverse events (AEs) included nausea, skin and subcutaneous tissue disorders (SSTD), diarrhea, and fatigue; grade 3 or higher AEs included dyspnea, fatigue, and SSTD. Overall, the hematologic response rate was 52% in patients treated at the MTD (n = 21). Organ responses were seen in 56% of patients (5 cardiac, 5 renal). Median hematologic progression-free survival was 14.8 months; 1-year progression-free and overall survival rates were 60% and 85%, respectively (median follow-up, 16.9 months). Weekly oral ixazomib appears to be active in patients with relapsed/refractory AL amyloidosis, with a generally manageable safety profile. The study was registered at clinicaltrials.gov as #NCT01318902 A phase 3 study is ongoing (#NCT01659658).
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.