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

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Prothena Therapeutics Ltd.
ClinicalTrials.gov Identifier:
NCT01707264
First received: October 11, 2012
Last updated: August 18, 2015
Last verified: August 2015
  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 25 additional subjects at the maximum tolerated dose.


Condition Intervention Phase
Primary Amyloidosis
Drug: NEOD001
Phase 1
Phase 2

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 1/2, 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 Prothena Therapeutics Ltd.:

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: 55
Study Start Date: April 2013
Estimated Study Completion Date: September 2016
Estimated Primary Completion Date: June 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 25 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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01707264

Locations
United States, California
Stanford University Cancer Center
Palo Alto, California, United States, 94305
United States, Massachusetts
Boston University School of Medicine
Boston, Massachusetts, United States, 02118
Tufts Medical Center
Boston, Massachusetts, United States, 02111
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 Therapeutics Ltd.
Investigators
Study Director: Spencer Guthrie Prothena Therapeutics Ltd.
  More Information

No publications provided

Responsible Party: Prothena Therapeutics Ltd.
ClinicalTrials.gov Identifier: NCT01707264     History of Changes
Other Study ID Numbers: NEOD001-001
Study First Received: October 11, 2012
Last Updated: August 18, 2015
Health Authority: United States: Food and Drug Administration

Keywords provided by Prothena Therapeutics Ltd.:
AL amyloidosis
Primary amyloidosis

Additional relevant MeSH terms:
Amyloidosis
Metabolic Diseases
Proteostasis Deficiencies

ClinicalTrials.gov processed this record on August 30, 2015