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

Prothena

Status and phase: Completed; Phase 2; Phase 1

Conditions: Primary Amyloidosis

Treatments: Drug: NEOD001

Study type: Interventional

Funder types: Industry

Identifiers

NCT01707264

NEOD001-001

Details and patient eligibility

About

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.

Full 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.

Enrollment: 69 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

69 participants in 1 patient group

NEOD001

Experimental group

Description:

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.

Treatment:

Drug: NEOD001