Primary Amyloidosis
Conditions
Keywords
AL amyloidosis, Primary 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.
Interventions
DRUGNEOD001
Monoclonal antibody administered by intravenous infusion every 28 days.
Sponsors
Prothena Biosciences Ltd.
Study design
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Safety and tolerability | 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 | 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 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Pharmacokinetics | 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 | 28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator | • Measurement of anti-NEOD001 antibodies |
Other
| Measure | Time frame | Description |
|---|---|---|
| Hematologic Response | 28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator | • Hematologic response |
| Organ response | 28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator | * Organ response * Changes in organ function markers |
Countries
United States
Outcome results
None listed