The PRONTO Study, a Global Phase 2b Study of NEOD001 in Previously Treated Subjects With Light Chain (AL) Amyloidosis

Prothena

Status and phase: Completed; Phase 2

Conditions: AL Amyloidosis

Treatments: Drug: NEOD001; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT02632786

NEOD001-201

Details and patient eligibility

About

This is a global, multicenter, Phase 2b, randomized, double-blind, placebo-controlled, two-arm, parallel-group efficacy and safety study of NEOD001 as a single agent administered intravenously in adults with AL amyloidosis who had a hematologic response to previous treatment for their amyloidosis (e.g., chemotherapy, autologous stem cell transplant [ASCT]) and have persistent cardiac dysfunction.

Enrollment: 129 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Age ≥18 years
2. Confirmed diagnosis of systemic AL amyloidosis
3. ≥1 prior systemic plasma cell dyscrasia therapy with at least a partial hematologic response
4. Cardiac involvement
5. NT-proBNP ≥650

Exclusion criteria

1. Non-AL amyloidosis
2. Meets the International Myeloma Working Group (IMWG) definition of Multiple Myeloma
3. NT-proBNP >5000
4. Received Plasma cell directed chemotherapy within 6 months
5. Received autologous stem cell transplant (ASCT) within 12 months

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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129 participants in 2 patient groups, including a placebo group

NEOD001

Experimental group

Description:

Study Drug given IV every 28 days at 24mg/kg

Treatment:

Drug: NEOD001

Placebo

Placebo Comparator group

Description:

Placebo

Treatment:

Drug: Placebo