An Extension Study to Evaluate the Long-Term Safety and Tolerability of NEOD001 for Treatment of People with Light Chain Amyloidosis

Overview

  • Study type

    Interventional
  • Study phase

    II
  • Study IDs

  • 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.
  • 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.

  • Site IRB
    • Rochester, Minnesota: 16-003114
    NCT ID: NCT02613182
    Sponsor Protocol Number: NEOD001-OLE001

About this study

The purpose of this study is to provide additional treatment with NEOD001 and evaluate long term safety and tolerability for patients wih light chain amyloidosis who completed the previous study NEOD001-001.

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

  • Previously enrolled and treated for at least 9 months in Study NEOD001-001
  • Ability to understand and willingness to sign an informed consent form prior to initiation of any study procedures
  • Has adequate bone marrow reserve, and hepatic and renal function, as demonstrated by
    • Absolute neutrophil count (ANC) ≥1.0 ×109/L
    • Platelet count ≥75 × 109/L
    • Hemoglobin ≥9 g/dL
    • Total bilirubin ≤2 times the upper limit of normal (× ULN)
    • Aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) ≤3 × ULN
    • Estimated glomerular filtration rate ≥30 mL/minute
  • Seated systolic blood pressure 90 to 180 mmHg
  • ECOG Performance Status 0 to 2
  • Women of childbearing potential (WOCBP) must have a negative serum pregnancy test within 28 days prior to the first administration of study drug and agree to use highly effective physician-approved contraception from 30 days prior to the first study drug administration to 90 days following the last study drug administration
  • Male subjects must be surgically sterile or must agree to use highly effective physician-approved contraception from 30 days prior to the first study drug administration to 90 days following the last study drug administration

Exclusion Criteria

  • Any new medical contraindication or clinically significant abnormality on physical, neurological, laboratory, vital signs, or electrocardiogram (ECG) examination such as atrial fibrillation
    • With the exception of subjects for whom the ventricular rate is controlled
  • History of Grade ≥3 infusion-associated adverse events or hypersensitivities to NEOD001 or any of its excipients
  • Treatment with any anticancer therapy (standard or investigational) within the 14 days prior to the first dose of study drug
    • Must have fully recovered (i.e., National Cancer Institute Common Terminology Criteria for Adverse Events [CTCAE] Grade 1 [exception: subjects with prior bortezomib may have CTCAE Grade 2 neuropathy]) from the clinically significant toxic effects of that treatment
  • Received any of the following within the specified time frame prior to the first administration of study drug
    • Hematopoietic growth factors, transfusions of blood or blood products within 1 week
    • Major surgery within 2 weeks
    • Radiotherapy within 2 weeks
    • Transplant within 8 weeks
    • Investigational drug other than NEOD001 within 4 weeks
    • Another experimental anti-amyloid therapy other than NEOD001 within 2 years
  • Uncontrolled symptomatic orthostatic hypotension
  • Myocardial infarction, uncontrolled angina, severe uncontrolled ventricular arrhythmias, or electrocardiographic evidence of acute ischemia, within 6 months prior to the first dose of study drug
  • Uncontrolled infection
  • Secondary malignancy, with the exception of
    • Adequately treated basal cell carcinoma, squamous cell carcinoma, or in situ cervical cancer
    • Adequately treated stage I cancer from which the subject is currently in remission
    • Any other cancer from which the subject has been disease-free for ≥ 3 years
  • Uncontrolled human immunodeficiency virus (HIV), hepatitis B, or hepatitis C infection
  • Women who are lactating

Participating Mayo Clinic locations

Study statuses change often. Please contact us for help.

Mayo Clinic Location Status Contact

Rochester, Minn.

Mayo Clinic principal investigator

Morie Gertz, M.D.

Closed for enrollment

Contact information:

Cancer Center Clinical Trials Referral Office

855-776-0015

.
CLS-20260199

Mayo Clinic Footer