Amyloidosis Research

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Amyloidosis Clinical Trials

The key to finding better treatments for rare diseases for which the standard of care is not acceptable is the constant collaboration between scientists, pharmaceutical companies and clinical investigators. This partnership is the only way to move therapies swiftly from our on-campus research labs to patients. The quality and frequency of this interaction allows us to get potentially life-extending therapies to our patients quickly.

Clinical Trials for Newly Diagnosed Patients

  • IRB No. 20342: Phase 3, double-blind, multicenter study to evaluate the efficacy and safety of CAEL-101 and plasma cell dyscrasia treatment versus placebo and plasma cell dyscrasia treatment in plasma cell dyscrasia treatment-naive patients with Mayo Stage 3b amyloidosis
    Principal investigator: Michael Rosenzweig, M.D., M.S.
  • IRB No. 20343: Phase 3, double-blind, multicenter study to evaluate the efficacy and safety of CAEL-101and plasma cell dyscrasia treatment versus placebo and plasma cell dyscrasia treatment in plasma cell dyscrasia treatment-naive patients with Mayo Stage 3a amyloidosis
    Principal investigator: Michael Rosenzweig, M.D., M.S.
  • IRB No. 21747: Phase 2, multicohort study of daratumumab-based therapies in participants with amyloid light chain amyloidosis
    Protocol 54767414AMY2009: relapsed disease
  • IRB No. 20563: Phase1, open-label, dose-escalation study of the safety and efficacy of an anti-CD38 antibody drug conjugate (STl-6129) in patients with relapsed or refractory systemic amyloidosis
    Principal investigator: Michael Rosenzweig, M.D., M.S.
  • IRB No. 21395: A Phase 1/la study of venetoclax, MLN9708 (ixazomib citrate) and dexamethasone for relapsed or refractory light chain amyloidosis
    Principal investigator: Michael Rosenzweig, M.D., M.S.

Amyloidosis Publications

  • Rosenzweig M, Comenzo RL. Liver and Gastrointestinal Involvement. Hematol Oncol Clin North Am. 2020 Dec;34(6):1081-1090

  • Viola D, Dona A, Caserta E, Troadec E, Besi F, McDonald T, Ghoda L, Gunes EG, Sanchez JF, Khalife J, Martella M, Karanes C, Htut M, Wang X, Rosenzweig M, Chowdhury A, Sborov D, Miles RR, Yazaki PJ, Ebner T, Hofmeister CC, Forman SJ, Rosen ST, Marcucci G, Shively J, Keats JJ, Krishnan A, Pichiorri F. Daratumumab induces mechanisms of immune activation through CD38+ NK cell targeting. Leukemia. 2021 Jan;35(1):189-200.

  • Premkumar VJ, Lentzsch S, Pan S, Bhutani D, Richter J, Jagannath S, Liedtke M, Jaccard A, Wechalekar AD, Comenzo R, Sanchorawala V, Royer B, Rosenzweig M, Valent J, Schonland S, Fonseca R, Wong S, Kapoor P. Venetoclax induces deep hematologic remissions in 1(11;14) relapsed/refractory AL amyloidosis. Blood Cancer J. 2021 Jan 11;11(1):10.

  • Jamal F, Rosenzweig M. Amyloidosis with Cardiac Involvement: Identification, Characterization, and Management. Curr Hematol Malig Rep. 2021 Aug;16(4):357-366. doi: 10.1007/sll899-021-00626-4. Epub 2021Jun 9.

  • Kastritis E, Palladini G, Minnema MC, Wechalekar AD, Jaccard A, Lee HC, Sanchorawala V, Gibbs S, Mollee P, Venner CP, Lu J, Schonland S, Galt ME, Suzuki K, Kim K, Cibeira MT, Beksac M, Libby E, Valent J, Hungria V, Wong SW, Rosenzweig M, Summa N, Huart A, Dimopoulos MA, Bhutani D, Waxman AJ, Goodman SA, Zonder JA, Lam S, Song K, Hansen T, Manier S, Roeloffzen W, Jamroziak K, Kwok F, Shimazaki C, Kim JS, Crusoe E, Ahmadi T, Tran N, Qin X, Vasey SY, Tromp B, Schecter JM, Weiss BM, Zhuang SH, Vermeulen J, Merlini G, Comenzo RL. ANDROMEDA Trial Investigators. Daratumumab-Based Treatment for lmmunoglobulin Light-Chain Amyloidosis. N Engl J Med. 2021 Jul 1;385(1)46-58.