Study of Dexamethasone Plus IXAZOMIB or Physician's Choice of Treatment in Relapsed or Refractory Systemic Light Chain (AL) Amyloidosis

  • Study type:

    Interventional What is this?

    Describes the nature of a clinical study. Types include:

    • Observational study — observes people and measures outcomes without affecting results.
    • Interventional study (clinical trial) — studies new tests, treatments, drugs, surgical procedures or devices.
    • Medical records research — uses historical information collected from medical records of large groups of people to study how diseases progress and which treatments and surgeries work best.
  • Study phase:

    III What is this?

    During the early phases (phases 1 and 2), researchers assess safety, side effects, optimal dosages and risks/benefits. In the later phase (phase 3), researchers study whether the treatment works better than the current standard therapy. They also compare the safety of the new treatment with that of current treatments. Phase 3 trials include large numbers of people to make sure that the result is valid. There are also less common very early (phase 0) and later (phase 4) phases. Phase 0 trials are small trials that help researchers decide if a new agent should be tested in a phase 1 trial. Phase 4 trials look at long-term safety and effectiveness, after a new treatment has been approved and is on the market.

Study IDs

  • Site IRB:

    • Scottsdale/Phoenix, Arizona: 12-005223
    • Rochester, Minnesota: 12-005223
  • NCT ID:

    NCT01659658
  • Sponsor Protocol Number:

    C16011

About this study

This is a phase 3, randomized, controlled, open-label, multicenter study of the oral formulation of dexamethasone plus IXAZOMIB compared with treatment chosen by the investigator from a prespecified list of regimens available in clinical practice. Treatment options will include: dexamethasone alone, dexamethasone plus an alkylating agent (melphalan or cyclophosphamide), or dexamethasone plus an immunomodulatory drug (IMiD, thalidomide or lenalidomide) in patients with relapsed or refractory AL amyloidosis. Crossover to the investigational treatment arm is not permitted during participation in this study.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. If you need assistance understanding the eligibility criteria, please contact the study team.

See eligibility criteria

Inclusion Criteria:

  • Male or female patients 18 years or older
  • Biopsy-proven AL amyloidosis with relapsed or refractory disease despite 1 or 2 prior therapies. Patients may be proteasome inhibitor-exposed or naive, but cannot be refractory to proteasome inhibitor therapy
  • Disease requiring further treatment
  • Measurable disease as defined by serum differential free light chain concentration (dFLC)
  • Objective and measurable major organ involvement (ie, cardiac or renal) as defined by the standard International Society of Amyloidosis (ISA) criteria.
  • Eastern Cooperative Oncology Group (ECOG) performance status of less than or equal to 2
  • Meet the clinical laboratories criteria as specified in the protocol
  • Female patients who are post menopausal, surgically sterile, or agree to practice 2 effective methods of contraception through 90 days after the last dose of study treatment or agree to practice true abstinence; must also adhere to the guidelines of any treatment specific pregnancy prevention program
  • Male patients who agree to practice effective barrier contraception through 90 days after the last dose of study treatment or agree to practice true abstinence AND must adhere to the guidelines of any treatment specific pregnancy prevention program
  • Voluntary written consent

Exclusion Criteria:

  • Amyloidosis due to mutations of the transthyretin gene or presence of other non-AL amyloidosis
  • Female patients who are lactating, breastfeeding or pregnant
  • Evidence of current uncontrolled cardiovascular conditions as specified in study protocol
  • Clinically overt multiple myeloma as specified in study protocol
  • Inability to swallow medication, inability or unwillingness to comply with the drug administration requirements, or gastrointestinal (GI) procedure that could interfere with the oral absorption or tolerance of treatment
  • Requirement for other concomitant chemotherapy, immunotherapy, radiotherapy, or any ancillary therapy considered to be investigational or which would be considered as a treatment of AL amyloidosis. However, patients may be on chronic steroids (maximum dose 20 mg/day prednisone or equivalent if they are being given for disorders other than amyloidosis
  • Comorbid systemic illnesses or other severe concurrent disease which, in the judgment of the investigator, would make the patient inappropriate for entry into this study or interfere significantly with the proper assessment of safety and toxicity of the prescribed regimens
  • Ongoing or active infection, known HIV positive, active hepatitis B or C infection
  • Psychiatric illness/social situations that would limit compliance with study requirements
  • Known allergy to any of the study medications, their analogues or excipients
  • Systemic treatment with strong inhibitors of CYP1A2, strong inhibitors of CYP3A, or strong CYP3A inducers, or use of Ginkgo biloba or St. John's wort within 14 days before the first dose of study treatment
  • Diagnosed or treated for another malignancy within 5 years before study enrollment or previously diagnosed with another malignancy and have any evidence of residual disease. Patients with nonmelanoma skin cancer or carcinoma in situ of any type are not excluded if they have undergone complete resection

Participating Mayo Clinic locations

Study statuses change often. Please contact us for help.

Mayo Clinic Location Status Contact

Scottsdale/Phoenix, Ariz.

Mayo Clinic principal investigator

Angela Dispenzieri, M.D.

Closed for enrollment

Cancer Center Clinical Trials Referral Office

855-776-0015

Rochester, Minn.

Mayo Clinic principal investigator

Angela Dispenzieri, M.D.

Open for enrollment

Cancer Center Clinical Trials Referral Office

855-776-0015