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

Study Overview
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Brief Summary
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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
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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.

Official Title
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A Phase 1/2, Open Label, Dose Escalation Study of Intravenous Administration of Single Agent NEOD001 in Subjects With Light Chain (AL) Amyloidosis

Conditions
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Primary Amyloidosis

Intervention / Treatment
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* Drug: NEOD001


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 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. Exclusion Criteria: 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.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Sequential Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: NEOD001<br>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<br>* Monoclonal antibody administered by intravenous infusion every 28 days.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Pharmacokinetics | • Pharmacokinetic parameters including Cmax, Tmax, AUC, Cav, Cmin, t½, CL, and Vz | 28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator | 
| Immunogenicity | • Measurement of anti-NEOD001 antibodies | 28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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AL amyloidosis, Primary amyloidosis