Open-label Extension Study of NEOD001 in Subjects With Light Chain (AL) Amyloidosis (OLE)
Primary Purpose
AL Amyloidosis
Status
Terminated
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
NEOD001
Sponsored by
About this trial
This is an interventional treatment trial for AL Amyloidosis
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, 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 (e.g., atrial fibrillation; with the exception of subjects for whom the ventricular rate is controlled) that precludes continued or initiation of treatment with NEOD001 or participation in the study
- History of Grade ≥3 infusion-associated adverse events (AEs) 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. In addition, subjects 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
Sites / Locations
- Stanford Cancer Institute (SCI)
- Tufts Medical Center
- Karmanos Cancer Institute
- Mayo Clinic
- Hospital of the University of Pennsylvania
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Open Label
Arm Description
Open Label Study Drug NEOD001
Outcomes
Primary Outcome Measures
Long-term Safety and Tolerability of NEOD001
Adverse events are defined as any unfavorable and unintended diagnosis, symptom, sign (including an abnormal laboratory finding), syndrome, or disease which either occurs during study, having been absent at baseline, or, if present at baseline, appears to worsen. Serious adverse events are any untoward medical occurrences that result in death, are life threatening, require (or prolong) hospitalization, cause persistent or significant disability/incapacity, result in congenital anomalies or birth defects, or are other conditions which in judgment of investigators represent significant hazards.
Secondary Outcome Measures
Full Information
NCT ID
NCT02613182
First Posted
November 18, 2015
Last Updated
May 14, 2019
Sponsor
Prothena Biosciences Ltd.
1. Study Identification
Unique Protocol Identification Number
NCT02613182
Brief Title
Open-label Extension Study of NEOD001 in Subjects With Light Chain (AL) Amyloidosis
Acronym
OLE
Official Title
Open-label Extension Study to Evaluate the Long-term Safety and Tolerability of NEOD001 in Subjects With Light Chain (AL) Amyloidosis
Study Type
Interventional
2. Study Status
Record Verification Date
May 2019
Overall Recruitment Status
Terminated
Why Stopped
NEOD001 program terminated due to lack of clinical benefit
Study Start Date
February 2016 (undefined)
Primary Completion Date
May 2018 (Actual)
Study Completion Date
May 2018 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Prothena Biosciences Ltd.
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief Summary
The rationale for this study is to provide additional treatment with NEOD001 for subjects who complete Study NEOD001-001, and to continue to evaluate long term safety and tolerability. All subjects in the current NEOD001 trials are being dosed at 24 mg/kg, which will be continued in this study.
Detailed Description
The rationale for this study is to provide additional treatment with NEOD001 for subjects who complete Study NEOD001-001, and to continue to evaluate long term safety and tolerability. All subjects in the current NEOD001 trials are being dosed at 24 mg/kg, which will be continued in this study. The primary objective of the study is to evaluate long-term safety and tolerability of NEOD001 and the secondary objectives are to assess the immunogenicity of NEOD001 and to incorporate serum NEOD001 concentrations in a population pharmacokinetic (PK) analysis.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
AL Amyloidosis
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
34 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Open Label
Arm Type
Experimental
Arm Description
Open Label Study Drug NEOD001
Intervention Type
Drug
Intervention Name(s)
NEOD001
Intervention Description
NEOD001
Primary Outcome Measure Information:
Title
Long-term Safety and Tolerability of NEOD001
Description
Adverse events are defined as any unfavorable and unintended diagnosis, symptom, sign (including an abnormal laboratory finding), syndrome, or disease which either occurs during study, having been absent at baseline, or, if present at baseline, appears to worsen. Serious adverse events are any untoward medical occurrences that result in death, are life threatening, require (or prolong) hospitalization, cause persistent or significant disability/incapacity, result in congenital anomalies or birth defects, or are other conditions which in judgment of investigators represent significant hazards.
Time Frame
From initiation of study drug through the last study visit or up to 30 days after date of last dose, whichever came first, assessed up to 24 months
10. Eligibility
Sex
All
Accepts Healthy Volunteers
No
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, 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 (e.g., atrial fibrillation; with the exception of subjects for whom the ventricular rate is controlled) that precludes continued or initiation of treatment with NEOD001 or participation in the study
History of Grade ≥3 infusion-associated adverse events (AEs) 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. In addition, subjects 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
Facility Information:
Facility Name
Stanford Cancer Institute (SCI)
City
Stanford
State/Province
California
ZIP/Postal Code
94305
Country
United States
Facility Name
Tufts Medical Center
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02111
Country
United States
Facility Name
Karmanos Cancer Institute
City
Detroit
State/Province
Michigan
ZIP/Postal Code
48201
Country
United States
Facility Name
Mayo Clinic
City
Rochester
State/Province
Minnesota
ZIP/Postal Code
55905
Country
United States
Facility Name
Hospital of the University of Pennsylvania
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
12. IPD Sharing Statement
Learn more about this trial
Open-label Extension Study of NEOD001 in Subjects With Light Chain (AL) Amyloidosis
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