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Summary

Eligibility
for people ages 18 years and up
Location
at San Francisco, California and other locations
Dates
study started
estimated completion:

Description

Summary

This is a multicenter, Phase 2b, randomized, double-blind, placebo-controlled, two-arm, parallel-group efficacy and safety study of NEOD001 as a single agent administered intravenously in adults with AL amyloidosis who have a maintained hematologic response to their most recent treatment for AL amyloidosis (e.g., chemotherapy, autologous stem cell transplant [ASCT]) and have persistent renal dysfunction.

Official Title

The RAIN Study, a Multicenter Randomized Double-blind Phase 2b Study of NEOD001 in Previously Treated Subjects With Systemic Light-chain (AL) Amyloidosis and Persistent Renal Involvement

Details

This is a multicenter, Phase 2b, randomized, double-blind, placebo-controlled, two-arm, parallel-group efficacy and safety study of NEOD001 as a single agent administered intravenously in adults with AL amyloidosis who have a maintained hematologic response to their most recent treatment for AL amyloidosis (e.g., chemotherapy, autologous stem cell transplant [SCT]) and have persistent renal dysfunction. Subject screening will occur during the 28 days prior to the first administration of study drug (i.e. month 1 day 1). If screening assessments are completed and all eligibility requirements are met, the subject will be enrolled. Study visits will occur every 28 days based on scheduling from month 1 day 1. A ±5-day window is allowed for visits starting after month 1. Subjects may receive up to 12 infusions of study drug. Subjects who discontinue study drug before the initial End of Study (EOS) visit should have an Early Treatment Discontinuation (ETD) Visit 30 (±5) days after their final administration of study drug. After completing 12 months of treatment and the confirmatory EOS visit, a subject may enter an open-label extension (OLE) study, during which subjects will receive active treatment with NEOD001 for 12 months and may receive concurrent chemotherapy.

Keywords

Amyloidosis NEOD001 RAIN

Eligibility

You can join if…

Open to people ages 18 years and up

  1. 18 years of age or older
  2. Biopsy-proven diagnosis of AL amyloidosis by immunohistochemistry or mass spectroscopy of a tissue biopsy excluding bone marrow
  3. Screening renal biopsy for RAIN confirming AL amyloidosis as exclusive or dominant cause of renal damage
  4. Persistent renal involvement from diagnosis with proteinuria (predominantly albumin)> 500mg/day in a 24-hour urine collection
  5. CKD 1 to 3 (eGFR> 30)
  6. ≥1 prior systemic hematologic therapy for a free light chain (FLC) producing hematologic malignancy underlying the initial diagnosis of AL amyloidosis with at least a partial FLC response (PR, VGPR, CR) to treatment deemed stable and not requiring further treatment
  7. ECOG Performance Status ≤ 2
  8. Clinical laboratory values:
  9. Absolute neutrophil count> 1000/μL
  10. Platelet count> 75,000/μL
  11. Total bilirubin ≤ 1.5X ULN
  12. Alkaline phosphatase ≤ 5X ULN
  13. NT-proBNP < 1800 pg/mL
  14. Voluntary written consent must be given before performance of any study-related procedure not part of standard medical care with the understanding that consent may be withdrawn by the patient at any time without prejudice to future medical care

You CAN'T join if...

  1. Amyloidosis due to mutations of the transthyretin gene or presence of other non-AL amyloidosis
  2. Female patients who are lactating, breastfeeding, or pregnant
  3. Patients who have not been treated or who have received chemotherapy within 6 months,or SCT within 12 months, for the light-chain producing hematologic disease causing AL amyloidosis, at the time of the first dose of NEOD001 (month 1 day 1)
  4. Patients who at initial diagnosis or later met the International Myeloma Working Group(IMWG) definition of active multiple myeloma requiring therapy (Appendix 3)
  5. Patients whose screening renal biopsies for RAIN show dominant causes of renal damage not related to AL amyloidosis
  6. Medically documented cardiac syncope, uncompensated congestive heart failure,myocardial infarction within the previous 6 months, unstable angina pectoris,clinically significant repetitive atrial or ventricular arrhythmias despite antiarrhythmic treatment, or severe orthostatic hypotension or clinically significant uncompensated autonomic insufficiency
  7. 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
  8. Ongoing or active infection, known HIV positive, known to be hepatitis B surface antigen-positive or has known or suspected active hepatitis C infection.
  9. Psychiatric illness/social situations that would limit compliance with study requirements

Locations

  • Mayo Clinic- Arizona not yet accepting patients
    Scottsdale, Arizona, 85259, United States
  • Mayo Clinic- Minnesota not yet accepting patients
    Rochester, Minnesota, 55905, United States
  • Vanderbilt University Medical Center not yet accepting patients
    Nashville, Tennessee, 37232, United States
  • Karmanos Cancer Institute not yet accepting patients
    Detroit, Michigan, 48201, United States
  • Mayo Clinic- Florida not yet accepting patients
    Jacksonville, Florida, 32224, United States
  • Hospital of the University of Pennsylvania not yet accepting patients
    Philadelphia, Pennsylvania, 19104, United States
  • Memorial Sloan-Kettering Cancer Center not yet accepting patients
    New York, New York, 10065, United States
  • Tufts Medical Center accepting new patients
    Boston, Massachusetts, 02111, United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Tufts Medical Center
ID
NCT03168906
Phase
Phase 2
Study Type
Interventional
Last Updated
July 1, 2017
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