Overview

This trial is active, not recruiting.

Condition primary amyloidosis
Treatment neod001
Phase phase 1/phase 2
Target amyloid fibrils
Sponsor Prothena Therapeutics Ltd.
Start date April 2013
End date June 2016
Trial size 69 participants
Trial identifier NCT01707264, NEOD001-001

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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Endpoint classification safety/efficacy study
Intervention model single group assignment
Masking open label
Primary purpose treatment
Arm
(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.
neod001
Monoclonal antibody administered by intravenous infusion every 28 days.

Primary Outcomes

Measure
Safety and tolerability
time frame: 28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator
Maximum tolerated dose
time frame: 28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator

Secondary Outcomes

Measure
Pharmacokinetics
time frame: 28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator
Immunogenicity
time frame: 28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator

Eligibility Criteria

Male or female participants at least 18 years old.

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.

Additional Information

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
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.
Trial information was received from ClinicalTrials.gov and was last updated in October 2015.
Information provided to ClinicalTrials.gov by Prothena Therapeutics Ltd..
Location data was received from the National Cancer Institute and was last updated in August 2016.