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Phase 1/2, Open Label, Dose Escalation Study of NEOD001 in Subjects With Light Chain (AL) Amyloidosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01707264
Recruitment Status : Completed
First Posted : October 16, 2012
Last Update Posted : August 28, 2018
Sponsor:
Information provided by (Responsible Party):
Prothena Biosciences Ltd.

Study Description
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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.

Condition or disease Intervention/treatment Phase
Primary Amyloidosis Drug: NEOD001 Phase 1 Phase 2

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.

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 69 participants
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : April 2013
Actual Primary Completion Date : August 9, 2016
Actual Study Completion Date : August 9, 2016

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Amyloidosis

Arms and Interventions
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Arm Intervention/treatment
Experimental: NEOD001
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.
 Drug: NEOD001
Monoclonal antibody administered by intravenous infusion every 28 days.


Outcome Measures
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Primary Outcome Measures :
  1. Safety and tolerability [ Time Frame: 28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator ]
    • Adverse event profile
    • Dose limiting toxicity and maximum tolerated dose

  2. Maximum tolerated dose [ Time Frame: 28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator ]
    • Adverse event profile
    • Dose Limiting Toxicity and maximum tolerated dose


Secondary Outcome Measures :
  1. Pharmacokinetics [ Time Frame: 28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator ]
    • Pharmacokinetic parameters including Cmax, Tmax, AUC, Cav, Cmin, t½, CL, and Vz

  2. Immunogenicity [ Time Frame: 28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator ]
    • Measurement of anti-NEOD001 antibodies


Other Outcome Measures:
  1. Hematologic Response [ Time Frame: 28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator ]
    • Hematologic response

  2. Organ response [ Time Frame: 28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator ]
    • Organ response
    • Changes in organ function markers


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

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

Exclusion Criteria:

  1. Secondary or familial amyloidosis;
  2. Life expectancy of < 3 months;
  3. Symptomatic multiple myeloma;
  4. Hypersensitivities to other monoclonal antibodies;
  5. Known HIV infection;
  6. Women who are lactating;
  7. Any other condition or prior therapy, which in the opinion of the PI, would make the subject unsuitable for the study.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01707264


Locations
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United States, California
Stanford University Cancer Center
Palo Alto, California, United States, 94305
United States, Massachusetts
Tufts Medical Center
Boston, Massachusetts, United States, 02111
Boston University School of Medicine
Boston, Massachusetts, United States, 02118
United States, Michigan
Karmanos Cancer Institute
Detroit, Michigan, United States, 48201
United States, Minnesota
Mayo Clinic
Rochester, Minnesota, United States, 55905
United States, New York
Memorial Sloan-Kettering Cancer Center
New York, New York, United States, 10065
United States, Pennsylvania
Hospital of the University of Pennsylvania
Philadelphia, Pennsylvania, United States, 19104
Sponsors and Collaborators
Prothena Biosciences Ltd.
Investigators
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Study Director: Julia Martinisi Prothena Biosciences Ltd.
More Information
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Responsible Party: Prothena Biosciences Ltd.
ClinicalTrials.gov Identifier: NCT01707264    
Other Study ID Numbers: NEOD001-001
First Posted: October 16, 2012    Key Record Dates
Last Update Posted: August 28, 2018
Last Verified: August 2018
Keywords provided by Prothena Biosciences Ltd.:
AL amyloidosis
Primary amyloidosis
Additional relevant MeSH terms:
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Immunoglobulin Light-chain Amyloidosis
Amyloidosis
Proteostasis Deficiencies
Metabolic Diseases
Neoplasms, Plasma Cell
Neoplasms by Histologic Type
 Neoplasms
Lymphoproliferative Disorders
Immunoproliferative Disorders
Immune System Diseases
Paraproteinemias