The VITAL Amyloidosis Study, a Global Phase 3, Efficacy and Safety Study of NEOD001 in Patients With AL Amyloidosis

Prothena

Status and phase: Terminated; Phase 3

Conditions: Primary Systemic (AL) Amyloidosis

Treatments: Drug: NEOD001; Other: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT02312206

NEOD001-CL002

Details and patient eligibility

About

This is a multi-center, international, randomized, double-blind, placebo-controlled, two-arm efficacy and safety study in subjects newly diagnosed with AL amyloidosis. Subjects will remain on-study until study completion, which will occur when all primary endpoint events (all-cause mortality or cardiac hospitalizations) have been reached.

Full description

This is a multi-center, international, randomized, double-blind, placebo-controlled, two-arm efficacy and safety study in subjects newly diagnosed with AL amyloidosis. Approximately 236 subjects will be enrolled in ~60 centers, with approximately 118 subjects per arm.

This is an event driven trial, therefore subjects will remain on-study until study completion, which will occur when all primary endpoint events (all-cause mortality or cardiac hospitalizations) have been reached. All subjects who discontinue will be followed until the last event is adjudicated. The estimated overall study duration is approximately 42 months, including the enrollment and treatment periods

Study drug will be administered once every 28 days as a 60-120 minute IV infusion.

First-line chemotherapy must be a bortezomib-containing regimen, with bortezomib administered weekly. The number of cycles of first-line chemotherapy that are administered are at the discretion of the Investigator, and subsequent chemotherapy regimens may be prescribed as per standard of care at the Investigator's discretion.

An independent Data Monitoring Committee (DMC) will review data on a regular basis.

Enrollment: 260 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

1. Age ≥ 18 years
2. Newly diagnosed, AL amyloidosis treatment naïve
3. Bone marrow consistent with plasma cell dyscrasia
4. Confirmed diagnosis of AL amyloidosis
5. Cardiac involvement
6. Planned first-line chemotherapy contains a proteasome-inhibiting agent administered weekly
7. Adequate bone marrow reserve, hepatic and renal function

Key Exclusion Criteria:

1. Non-AL amyloidosis
2. Meets diagnostic criteria for symptomatic multiple myeloma
3. Subject is eligible for and plans to undergo ASCT
4. History of Grade ≥ 3 infusion-associated AEs or hypersensitivity to another monoclonal antibody, or known hypersensitivity to diphenhydramine or acetaminophen

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

---

260 participants in 2 patient groups, including a placebo group

NEOD001

Experimental group

Description:

24 mg/kg (maximum dose of 2500 mg) of NEOD001 administered once every 28 days.

Treatment:

Drug: NEOD001

Placebo

Placebo Comparator group

Description:

Placebo will be administered as a 250 mL bag of normal saline once every 28 days.

Treatment:

Other: Placebo