NCT01707264

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
69

participants targeted

Target at P75+ for phase_1

Timeline
Completed

Started Apr 2013

Typical duration for phase_1

Geographic Reach
1 country

7 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

October 11, 2012

Completed
5 days until next milestone

First Posted

Study publicly available on registry

October 16, 2012

Completed
6 months until next milestone

Study Start

First participant enrolled

April 1, 2013

Completed
3.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 9, 2016

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

August 9, 2016

Completed
Last Updated

August 28, 2018

Status Verified

August 1, 2018

Enrollment Period

3.4 years

First QC Date

October 11, 2012

Last Update Submit

August 24, 2018

Conditions

Primary Amyloidosis

Keywords

AL amyloidosisPrimary amyloidosis

Outcome Measures

Primary Outcomes (2)

  • Safety and tolerability

    * Adverse event profile * Dose limiting toxicity and maximum tolerated dose

    28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator

  • Maximum tolerated dose

    * Adverse event profile * Dose Limiting Toxicity and maximum tolerated dose

    28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator

Secondary Outcomes (2)

  • Pharmacokinetics

    28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator

  • Immunogenicity

    28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator

Other Outcomes (2)

  • Hematologic Response

    28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator

  • Organ response

    28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator

Study Arms (1)

NEOD001

EXPERIMENTAL

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

Interventions

NEOD001DRUG

Monoclonal antibody administered by intravenous infusion every 28 days.

NEOD001

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (7)

Stanford University Cancer Center

Palo Alto, California, 94305, United States

Location

Tufts Medical Center

Boston, Massachusetts, 02111, United States

Location

Boston University School of Medicine

Boston, Massachusetts, 02118, United States

Location

Karmanos Cancer Institute

Detroit, Michigan, 48201, United States

Location

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

Memorial Sloan-Kettering Cancer Center

New York, New York, 10065, United States

Location

Hospital of the University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

Location

Related Publications (1)

  • Gertz MA, Landau H, Comenzo RL, Seldin D, Weiss B, Zonder J, Merlini G, Schonland S, Walling J, Kinney GG, Koller M, Schenk DB, Guthrie SD, Liedtke M. First-in-Human Phase I/II Study of NEOD001 in Patients With Light Chain Amyloidosis and Persistent Organ Dysfunction. J Clin Oncol. 2016 Apr 1;34(10):1097-103. doi: 10.1200/JCO.2015.63.6530. Epub 2016 Feb 8.

    PMID: 26858336DERIVED

MeSH Terms

Conditions

Immunoglobulin Light-chain Amyloidosis

Interventions

birtamimab

Condition Hierarchy (Ancestors)

Neoplasms, Plasma CellNeoplasms by Histologic TypeNeoplasmsAmyloidosisProteostasis DeficienciesMetabolic DiseasesNutritional and Metabolic DiseasesLymphoproliferative DisordersImmunoproliferative DisordersImmune System DiseasesParaproteinemias

Study Officials

  • Julia Martinisi

    Prothena Biosciences Ltd.

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 1
Masking
NONE
Purpose
TREATMENT
Intervention Model
SEQUENTIAL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 11, 2012

First Posted

October 16, 2012

Study Start

April 1, 2013

Primary Completion

August 9, 2016

Study Completion

August 9, 2016

Last Updated

August 28, 2018

Record last verified: 2018-08

Locations

US(7)