Back to resultsPhase 1/2, Open Label, Dose Escalation Study of NEOD001 in Subjects With Light Chain (AL) Amyloidosis
Primary Purpose
Primary Amyloidosis
Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
NEOD001
Sponsored by
About this trial
This is an interventional treatment trial for Primary Amyloidosis focused on measuring AL amyloidosis, Primary amyloidosis
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.
Sites / Locations
- Stanford University Cancer Center
- Tufts Medical Center
- Boston University School of Medicine
- Karmanos Cancer Institute
- Mayo Clinic
- Memorial Sloan-Kettering Cancer Center
- Hospital of the University of Pennsylvania
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
NEOD001
Arm Description
NEOD001 will be administered intravenously once every 28 days. The starting dose will be 0.5 mg/kg. Dose escalation will continue until the the maximum tolerated dose is determined for single agent NEOD001. Approximately 20 additional subjects will be treated with the maximum tolerated dose.
Outcomes
Primary Outcome Measures
Safety and tolerability
Adverse event profile
Dose limiting toxicity and maximum tolerated dose
Maximum tolerated dose
Adverse event profile
Dose Limiting Toxicity and maximum tolerated dose
Secondary Outcome Measures
Pharmacokinetics
• Pharmacokinetic parameters including Cmax, Tmax, AUC, Cav, Cmin, t½, CL, and Vz
Immunogenicity
• Measurement of anti-NEOD001 antibodies
Full Information
NCT ID
NCT01707264
First Posted
October 11, 2012
Last Updated
August 24, 2018
Sponsor
Prothena Biosciences Ltd.
1. Study Identification
Unique Protocol Identification Number
NCT01707264
Brief Title
Phase 1/2, Open Label, Dose Escalation Study of NEOD001 in Subjects With Light Chain (AL) Amyloidosis
Official Title
A Phase 1/2, Open Label, Dose Escalation Study of Intravenous Administration of Single Agent NEOD001 in Subjects With Light Chain (AL) Amyloidosis
Study Type
Interventional
2. Study Status
Record Verification Date
August 2018
Overall Recruitment Status
Completed
Study Start Date
April 2013 (Actual)
Primary Completion Date
August 9, 2016 (Actual)
Study Completion Date
August 9, 2016 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Prothena Biosciences Ltd.
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
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 25 additional subjects at the maximum tolerated dose.
Detailed Description
The purpose of the dose escalation phase of the study is to determine the maximum tolerated dose/Phase 2 recommended dose of NEOD001 when given as a single agent intravenously in approximately 30 subjects with AL amyloidosis.
The purpose of the expansion phase of the study is to evaluate the safety, preliminary efficacy and pharmacokinetics of single agent NEOD001 at the maximum tolerated dose/Phase 2 recommended dose in approximately 25 additional evaluable subjects.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Primary Amyloidosis
Keywords
AL amyloidosis, Primary amyloidosis
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Sequential Assignment
Masking
None (Open Label)
Enrollment
69 (Actual)
8. Arms, Groups, and Interventions
Arm Title
NEOD001
Arm Type
Experimental
Arm Description
NEOD001 will be administered intravenously once every 28 days. The starting dose will be 0.5 mg/kg. Dose escalation will continue until the the maximum tolerated dose is determined for single agent NEOD001. Approximately 20 additional subjects will be treated with the maximum tolerated dose.
Intervention Type
Drug
Intervention Name(s)
NEOD001
Intervention Description
Monoclonal antibody administered by intravenous infusion every 28 days.
Primary Outcome Measure Information:
Title
Safety and tolerability
Description
Adverse event profile
Dose limiting toxicity and maximum tolerated dose
Time Frame
28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator
Title
Maximum tolerated dose
Description
Adverse event profile
Dose Limiting Toxicity and maximum tolerated dose
Time Frame
28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator
Secondary Outcome Measure Information:
Title
Pharmacokinetics
Description
• Pharmacokinetic parameters including Cmax, Tmax, AUC, Cav, Cmin, t½, CL, and Vz
Time Frame
28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator
Title
Immunogenicity
Description
• Measurement of anti-NEOD001 antibodies
Time Frame
28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator
Other Pre-specified Outcome Measures:
Title
Hematologic Response
Description
• Hematologic response
Time Frame
28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator
Title
Organ response
Description
Organ response
Changes in organ function markers
Time Frame
28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Julia Martinisi
Organizational Affiliation
Prothena Biosciences Ltd.
Official's Role
Study Director
Facility Information:
Facility Name
Stanford University Cancer Center
City
Palo Alto
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
Boston University School of Medicine
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02118
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
Memorial Sloan-Kettering Cancer Center
City
New York
State/Province
New York
ZIP/Postal Code
10065
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
Phase 1/2, Open Label, Dose Escalation Study of NEOD001 in Subjects With Light Chain (AL) Amyloidosis
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