Phase I, Open Label, Dose Escalation Study of NEOD001 in Subjects With 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:

    I 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:

    • Rochester, Minnesota: 12-007863
  • NCT ID:

    NCT01707264
  • Sponsor Protocol Number:

    NEOD001-001

About this study

Dose escalation study to determine the maximum tolerated dose of NEOD001 in approximately 30 subjects with AL amyloidosis. Expansion phase to evaluate safety, efficacy and pharmacokinetics of NEOD001 in 20 additional subjects at the maximum tolerated dose.

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:

  • Males and females aged ≥18 years;
  • ECOG performance status (PS) 0-2;
  • Diagnosis of systemic AL amyloidosis (subjects with non-AL amyloidosis are not eligible);
  • Received at least one prior systemic therapy, which may include stem cell transplant, for AL amyloidosis;
  • Have adequate organ function;
  • Ability to understand and willingness to sign informed consent prior to initiation of any study procedures.

Exclusion Criteria:

  • Secondary or familial amyloidosis;
  • Life expectancy of < 3 months;
  • Symptomatic multiple myeloma;
  • Hypersensitivities to other monoclonal antibodies;
  • Known HIV infection;
  • Women who are lactating;
  • Any other condition or prior therapy, which in the opinion of the PI, would make the subject unsuitable for the study.

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.

Open for enrollment

Cancer Center Clinical Trials Referral Office

855-776-0015