Clinical trial NCT01707264: Phase 1/2, Open Label, Dose Escalation Study of NEOD001 in Subjects With Light Chain (AL) Amyloidosis
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Primary Amyloidosis
Keywords: AL amyloidosis
Primary amyloidosis
Lead sponsor: Prothena Biosciences Ltd.
Sponsor class: INDUSTRY
First posted: 2012-10-16
Enrollment: 69
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Safety and tolerability
timeFrame: 28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator
measure: Maximum tolerated dose
timeFrame: 28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator
Secondary outcomes:
measure: Pharmacokinetics
timeFrame: 28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator
measure: Immunogenicity
timeFrame: 28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator
Other outcomes:
measure: Hematologic Response
timeFrame: 28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator
measure: Organ response
timeFrame: 28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator
Locations:
city: Palo Alto
country: United States
facility: Stanford University Cancer Center
state: California
city: Boston
country: United States
facility: Tufts Medical Center
state: Massachusetts
city: Boston
country: United States
facility: Boston University School of Medicine
state: Massachusetts
city: Detroit
country: United States
facility: Karmanos Cancer Institute
state: Michigan
city: Rochester
country: United States
facility: Mayo Clinic
state: Minnesota
city: New York
country: United States
facility: Memorial Sloan-Kettering Cancer Center
state: New York
city: Philadelphia
country: United States
facility: Hospital of the University of Pennsylvania
state: Pennsylvania
Study officials:
affiliation: Prothena Biosciences Ltd.
name: Julia Martinisi
role: STUDY_DIRECTOR
References: