Study of Oral Ixazomib in Adult Participants With Relapsed or Refractory Light Chain Amyloidosis
Condition: Light-Chain Amyloidosis
MeSH terms: Amyloidosis
Intervention: Ixazomib; Dexamethasone
Intervention description: Ixazomib capsules.; Dexamethasone tablets.
Intervention MeSH terms: Dexamethasone; Ixazomib
Phase: Phase 1
Study type: Interventional
Primary purpose: Treatment
Allocation: Non-Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Enrollment: 27.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: Drug therapy

Summary:

      This study will include participants with previously treated systemic relapsed or refractory
      light-chain (AL) amyloidosis who require further therapy and will be aimed at determining the
      safety profile and the maximum tolerated dose/recommended phase 2 dose of MLN9078 (Ixazomib)
      administered orally.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Male or female participants 18 years or older

          -  Biopsy-proven systemic relapsed or refractory light-chain (AL) amyloidosis, which
             after at least 1 prior therapy, in the investigator's opinion, requires further
             treatment

          -  If received stem cell transplant, must be at least 3 months posttransplantation and
             recovered from side effects

          -  Must have measurable disease defined as serum differential free light chain
             concentration ≥ 40 mg/L

          -  Must have objective measurable organ (heart or kidney) amyloid involvement

          -  Must have cardiac biomarker risk stage I or II disease

          -  Must have adequate hematologic, hepatic, and renal function

          -  Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2

          -  Female participants who are postmenopausal, surgically sterile, or agree to practice 2
             effective methods of contraception or agree to abstain from heterosexual intercourse

          -  Male participants who agree to practice effective barrier contraception or agree to
             abstain from heterosexual intercourse

          -  Voluntary written consent

        Exclusion Criteria

          -  Peripheral neuropathy that is greater or equal to Grade 2

          -  Cardiac status as described in protocol

          -  Severe diarrhea (≥ Grade 3) not controllable with medication or requires
             administration of total parenteral nutrition

          -  Known gastrointestinal condition or procedure that could interfere with swallowing or
             the oral absorption of tolerance of MLN9708

          -  Uncontrolled infection requiring systematic antibiotics

          -  Known human immunodeficiency virus (HIV) positive, hepatitis B surface
             antigen-positive status, or known or suspected active hepatitis C infection

          -  Presence of other active malignancy with the exception of nonmelanoma skin cancer,
             cervical cancer, treated early-stage prostate cancer provided that prostate-specific
             antigen is within normal limit, or any completely resected carcinoma in situ

          -  Female participants who are lactating or pregnant

          -  Major surgery within 14 days before the first dose of study drug

          -  Serious medical or psychiatric illness that could, in the investigator's opinion,
             potentially interfere with the completion of treatment according to the protocol