Study in Subjects With Light Chain (AL) Amyloidosis

Stopped early · Phase 2

Conditions studied: AL Amyloidosis

In brief

The objective of this study is to evaluate the long-term safety and efficacy of NEOD001 in subjects with AL amyloidosis who have completed Study NEOD001-201.

Key facts

Study ID

NCT03154047

Run by

Prothena Biosciences Ltd.

People needed

80

Starts

2017-06-14

Expected to finish

2018-05-30

Last updated by the study team

2019-03-26

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Completed the End of Study Visit in Study NEOD001-201
• Adequate bone marrow reserve, 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 × upper limit of normal (ULN)
• Aspartate aminotransferase (AST) ≤3 × ULN
• Alanine aminotransferase (ALT) ≤3 × ULN
• Alkaline phosphatase (ALP) ≤5 × ULN (except for subjects with hepatomegaly and isozymes specific to liver, rather than bone)
• Estimated glomerular filtration rate (eGFR) ≥25 mL/min/1.73 m2 as estimated by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation, or measured GFR ≥25 mL/min/1.73 m2
• Systolic blood pressure 80-180 mmHg
• Women of childbearing potential must have a negative pregnancy test during Screening and must agree to use highly effective physician-approved contraception from Screening 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 Screening to 90 days following the last study drug administration
• Ability to understand and willingness to sign an informed consent form prior to initiation of any study procedures

You may not qualify if…

• Any new medical contraindication or clinically significant abnormality on physical, neurological, laboratory, vital signs, or electrocardiographic (ECG) examination (e.g., atrial fibrillation; with the exception of subjects for whom the ventricular rate is controlled) that precludes continuation or initiation of treatment with NEOD001 or participation in the study
• Symptomatic orthostatic hypotension that in the medical judgment of the Investigator would interfere with subject's ability to safely receive treatment or complete study assessments
• Myocardial infarction, uncontrolled angina, uncontrolled ventricular arrhythmias, or ECG evidence of acute ischemia, within 6 months prior to the Month 1-Day 1 Visit
• Severe valvular stenosis (e.g., aortic or mitral stenosis with a valve area <1.0 cm2) or severe congenital heart disease
• ECG evidence of acute ischemia or active conduction system abnormalities with the exception of any of the following:
• First degree atrioventricular (AV) block
• Second degree AV block Type 1 (Mobitz Type 1/ Wenckebach type)
• Right or left bundle branch block
• Atrial fibrillation with a controlled ventricular rate (uncontrolled [i.e., >110 bpm] ventricular rate is not allowed [determined by an average of three beats in Lead II or 3 representative beats if Lead II is not representative of the overall ECG])
• Has not recovered (i.e., equivalent to a Common Terminology Criteria for Adverse Events [CTCAE] ≥Grade 2) from the clinically significant toxic effects of prior anticancer therapy. Exception: subjects who have received treatment with a proteasome inhibitor such as bortezomib may have CTCAE Grade 2 neuropathy.
• Received any of the following within the specified time frame prior to the Month 1-Day 1 Visit:
• Oral or IV antibiotics, antifungals, or antivirals within 1 week, with the exception of prophylactic oral agents. Note: In the event that a subject requires the chronic use of antivirals, Medical Monitor permission is required for entry into the study.
• Hematopoietic growth factors, transfusions of blood or blood products within 1 week
• Chemotherapy, radiotherapy, HDAC inhibitors, or other plasma cell directed therapy within 2 weeks
• ASCT within 4 weeks (i.e., ASCT is allowed if it occurred before enrollment in Study NEOD001-201 or after completion of Study NEOD001-201 if it was at least 4 weeks before Month 1-Day 1 of this study)
• Major surgery within 4 weeks (or within 2 weeks following consultation with and approval of Medical Monitor)
• Planned organ transplant during the study
• Any investigational agent, other than NEOD001, within 4 weeks
• Any experimental imaging agent directed at amyloid within 2 weeks
• Active malignancy with the exception of any of the following:
• 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 and has been in remission for ≥2 years
• Low-risk prostate cancer with Gleason score <7 and prostate-specific antigen <10 mg/mL
• Any other cancer from which the subject has been disease-free for ≥2 years
• History of Grade ≥3 infusion-related adverse events (AEs) or hypersensitivity to NEOD001

Where it is running

• City of Hope — Duarte, California, United States
• Colorado Blood Cancer Institute — Denver, Colorado, United States
• Mayo Clinic Hospital - Florida — Jacksonville, Florida, United States
• University of Chicago Medicine — Chicago, Illinois, United States
• Indiana University Simon Cancer Center — Indianapolis, Indiana, United States
• Tufts Medical Center — Boston, Massachusetts, United States
• Karmanos Cancer Institute — Detroit, Michigan, United States
• Mayo Clinic - Minnesota — Rochester, Minnesota, United States
• Memorial Sloan-Kettering Cancer Center — New York, New York, United States
• Duke University Medical Center — Durham, North Carolina, United States
• Cleveland Clinic — Cleveland, Ohio, United States
• Oregon Health & Science University — Portland, Oregon, United States
• Hospital of the University of Pennsylvania — Philadelphia, Pennsylvania, United States
• Vanderbilt University Medical Center — Nashville, Tennessee, United States
• The University of Texas MD Anderson Cancer Center — Houston, Texas, United States
• Medical College of Wisconsin — Milwaukee, Wisconsin, United States
• Westmead Hospital — Sydney, New South Wales, Australia
• Princess Alexandra Hospital — Woolloongabba, Queensland, Australia
• Eastern Health (Box Hill Hospital) — Box Hill, Victoria, Australia
• Medizinische Universität Wien — Vienna, Austria
• Hôpital Dupuytren - CHU Limoges — Limoges, France
• Hôpital Pitié-Salpêtrière — Paris, France
• Charité - Universitätsmedizin Berlin — Berlin, Germany
• University of Duisburg-Essen — Essen, Germany
• Universitätsklinikum Hamburg-Eppendorf (UKE) — Hamburg, Germany

Full record on ClinicalTrials.gov

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