Study of Dexamethasone in Patients with AL Amyloidosis
Research type
Research Study
Full title
A Phase 3 Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of Dexamethasone Plus MLN9708 or Physician's Choice of Treatment Administered to Patients With Relapsed or Refractory Systemic Light Chain (AL) Amyloidosis
IRAS ID
112563
Contact name
Ashutosh Wechalekar
Eudract number
2011-005468-10
Clinicaltrials.gov Identifier
Research summary
rimary light chain amyloidosis is a very rare disease due to deposition of monoclonal immunoglobulin light chain proteins causing progressive organ failure. Given the rarity, there have not been any randomised research studies in the relapsed disease setting. A phase III trial is needed as there are no approved treatments and data are insufficient to identify an optimal therapy. The treatment of patients with relapsed and/or refractory systemic light-chain (AL) amyloidosis is an area of unmet need.
The aim of therapy in AL amyloidosis is to reduce the amyloidogenic-involved free light chains (proteins that contribute to the disease) – which can improve the clinical course of the disease. The therapies that achieve these results are not curative. Currently, there is no agreed standard chemotherapy when patients relapse/do not responds to first-line therapy. A number of small non-randomised often retrospective studies have identified a number of effective regimes but none have been systematically studied in a randomised controlled fashion to identify the most appropriate and effective regime. Hence, there is no consensus, the choice of treatment currently decided on a fine balance between the perceived effectiveness of the chosen regimen and the individual patient’s expected ability to tolerate the treatment’s toxicity considering their age, organ dysfunction, and pace of disease, and given the limited treatment options, may mean exposure to an agent from the same drug class. Available treatment options in AL amyloidosis have used advances made in the chemotherapy of multiple myeloma (MM), including the use of corticosteroids, alkylating agents, proteasome inhibitors, and immunomodulatory agents (IMiDs), more frequently as combination regimens but also as single drug treatments.
Recognising that proteasome inhibition is an effective anticancer therapeutic approach, Millennium developed MLN9708, with the aim of improving the treatment of this disease. This study compares MLN9708 with investigator’s
REC name
London - Harrow Research Ethics Committee
REC reference
12/LO/1771
Date of REC Opinion
1 Feb 2013
REC opinion
Further Information Favourable Opinion