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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.

Tracking Information
First Submitted Date  ICMJE October 11, 2012
First Posted Date  ICMJE October 16, 2012
Last Update Posted Date August 28, 2018
Actual Study Start Date  ICMJE April 2013
Actual Primary Completion Date August 9, 2016   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: October 15, 2012)
  • 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
  • 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
Original Primary Outcome Measures  ICMJE
 (submitted: October 12, 2012)
  • Safety and tolerability [ Time Frame: 28 day cycles ]
    • Adverse event profile
    • Dose limiting toxicity and maximum tolerated dose
  • Maximum tolerated dose [ Time Frame: 28 day cycles ]
    • Adverse event profile
    • Dose Limiting Toxicity and maximum tolerated dose
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: October 15, 2012)
  • 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
  • Immunogenicity [ Time Frame: 28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator ]
    • Measurement of anti-NEOD001 antibodies
Original Secondary Outcome Measures  ICMJE
 (submitted: October 12, 2012)
  • Pharmacokinetics [ Time Frame: 28 day cycles ]
    • Pharmacokinetic parameters including Cmax, Tmax, AUC, Cav, Cmin, t½, CL, and Vz
  • Immunogenicity [ Time Frame: 28 day cycles ]
    • Measurement of anti-NEOD001 antibodies
Current Other Pre-specified Outcome Measures
 (submitted: October 15, 2012)
  • Hematologic Response [ Time Frame: 28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator ]
    • Hematologic response
  • 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
Original Other Pre-specified Outcome Measures
 (submitted: October 12, 2012)
  • Hematologic Response [ Time Frame: 28 day cycles ]
    • Hematologic response
  • Organ response [ Time Frame: 28 day cycles ]
    • Organ response
    • Changes in organ function markers
 
Descriptive Information
Brief Title  ICMJE Phase 1/2, Open Label, Dose Escalation Study of NEOD001 in Subjects With Light Chain (AL) Amyloidosis
Official Title  ICMJE A Phase 1/2, Open Label, Dose Escalation Study of Intravenous Administration of Single Agent NEOD001 in Subjects With Light Chain (AL) Amyloidosis
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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Primary Amyloidosis
Intervention  ICMJE Drug: NEOD001
Monoclonal antibody administered by intravenous infusion every 28 days.
Study Arms  ICMJE 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.
Intervention: Drug: NEOD001
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: October 10, 2015)		 69
Original Estimated Enrollment  ICMJE
 (submitted: October 12, 2012)		 50
Actual Study Completion Date  ICMJE August 9, 2016
Actual Primary Completion Date August 9, 2016   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01707264
Other Study ID Numbers  ICMJE NEOD001-001
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Current Responsible Party Prothena Biosciences Ltd.
Original Responsible Party Same as current
Current Study Sponsor  ICMJE Prothena Biosciences Ltd.
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Julia Martinisi Prothena Biosciences Ltd.
PRS Account Prothena Biosciences Ltd.
Verification Date August 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP