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

This study is currently recruiting participants.
Verified February 2014 by Onclave Therapeutics Limited, a wholly-owned subsidiary of Prothena Corporation plc
Sponsor:
Information provided by (Responsible Party):
Onclave Therapeutics Limited, a wholly-owned subsidiary of Prothena Corporation plc
ClinicalTrials.gov Identifier:
NCT01707264
First received: October 11, 2012
Last updated: February 7, 2014
Last verified: February 2014
  Purpose

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 20 additional subjects at the maximum tolerated dose.


Condition Intervention Phase
Primary Amyloidosis
Drug: NEOD001
Phase 1

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase I, Open Label, Dose Escalation Study of Intravenous Administration of Single Agent NEOD001 in Subjects With Light Chain (AL) Amyloidosis

Resource links provided by NLM:


Further study details as provided by Onclave Therapeutics Limited, a wholly-owned subsidiary of Prothena Corporation plc:

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

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


Secondary Outcome Measures:
  • Pharmacokinetics [ Time Frame: 28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator ] [ Designated as safety issue: No ]
    • Pharmacokinetic parameters including Cmax, Tmax, AUC, Cav, Cmin, t½, CL, and Vz

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


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

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


Estimated Enrollment: 50
Study Start Date: April 2013
Estimated Study Completion Date: September 2016
Estimated Primary Completion Date: May 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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.

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 20 additional evaluable subjects.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
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
Please refer to this study by its ClinicalTrials.gov identifier: NCT01707264

Contacts
Contact: Theresa Neumann, PhD theresa.neumann@prothena.com

Locations
United States, California
Stanford University Cancer Center Recruiting
Palo Alto, California, United States, 94305
Principal Investigator: Michaela Liedtke, MD         
United States, Massachusetts
Tufts Medical Center Recruiting
Boston, Massachusetts, United States, 02111
Contact: Jodi Jensen, RN    617-636-5558    jjensen@tuftsmedicalcenter.org   
Principal Investigator: Raymond Comenzo, MD         
Boston University School of Medicine Recruiting
Boston, Massachusetts, United States
Contact: Anthony Shelton    617-638-5612    anthony.shelton@bmc.org   
Contact: Stephen Lo    617-638-8274    stephen.lo@bmc.org   
Principal Investigator: David C Seldin, MD, PhD         
United States, Michigan
Karmanos Cancer Institute Recruiting
Detroit, Michigan, United States, 48201
Contact: Christiane Houde, BS, CCRP    313-576-9381    houdec@karmanos.org   
Principal Investigator: Jeffrey A Zonder, MD         
United States, Minnesota
Mayo Clinic Recruiting
Rochester, Minnesota, United States, 55905
Contact: Ann Birgin, CRA    507-284-8828      
Principal Investigator: Morris A Gertz, MD         
United States, New York
Memorial Sloan-Kettering Cancer Center Recruiting
New York, New York, United States, 10065
Contact: Heather Landau, MD    212-639-8808    Landauh@mskcc.org   
Contact: Hani Hassoun, MD    (212) 639-3228      
Principal Investigator: Heather Landau, MD         
United States, Pennsylvania
Hospital of the University of Pennsylvania Recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Scott Weber, BA    215-349-8913    scott.weber@uphs.upenn.edu   
Principal Investigator: Brendan Weiss, MD         
Sponsors and Collaborators
Onclave Therapeutics Limited, a wholly-owned subsidiary of Prothena Corporation plc
Investigators
Study Director: Theresa Neumann, PhD Onclave Therapeutics Ltd
  More Information

No publications provided

Responsible Party: Onclave Therapeutics Limited, a wholly-owned subsidiary of Prothena Corporation plc
ClinicalTrials.gov Identifier: NCT01707264     History of Changes
Other Study ID Numbers: NEOD001-001
Study First Received: October 11, 2012
Last Updated: February 7, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Onclave Therapeutics Limited, a wholly-owned subsidiary of Prothena Corporation plc:
AL amyloidosis
Primary amyloidosis

Additional relevant MeSH terms:
Amyloidosis
Proteostasis Deficiencies
Metabolic Diseases

ClinicalTrials.gov processed this record on April 15, 2014