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NEOD001

Phase 2

AL Amyloidosis | Small molecule | Rare Disease |Prothena Corporation plc|Last Updated: Apr 5, 2019

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials1
Total Enrollment129

FDA Designations

No designations recorded

Clinical trial landscape

NEOD001 · 2 trials · 2 indications

Phase 2 1Phase 1 1
NCT02632786The PRONTO Study, a Global Phase 2b Study of NEOD001 in Previously Treated Subjects With Light Chain (AL) AmyloidosisAL Amyloidosis
COMPLETED129 Analytics
PHASE2COMPLETED
The PRONTO Study, a Global Phase 2b Study of NEOD001 in Previously Treated Subjects With Light Chain (AL) Amyloidosis
AL AmyloidosisUnlock trial analytics

Study Endpoints

Primary Endpoints

Number of Participants With Cardiac Response and Non-Response
Baseline through 12 months of treatment

N-terminal pro-brain natriuretic peptide (NT-proBNP ) best response (Response or Non-Response \[Stable, Progression\]) from baseline through 12 months of treatment. Cardiac best response, as assessed by NT-proBNP alone, is defined as the most favorable category among response (ie, decrease in NT-proBNP from baseline of \>30% and \>300 ng/L), stable (ie, neither response nor progression), and progression (ie, increase in NT-proBNP from baseline of \>30% and \>300 ng/L) across all visits after the first infusion of study drug up to and through the end of the study. Subjects are considered non-responders until a response is achieved. Non-response is defined as either stable or progression.

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 Endpoints

SF-36v2 PCS Score
Baseline to 12 months of treatment
6MWT Distance
Baseline to 12 months of treatment
Number of Participants With Renal Best Response and Non-Response
Baseline through 12 months of treatment
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
NEOD001EXPERIMENTALStudy Drug given IV every 28 days at 24mg/kg
PlaceboPLACEBO_COMPARATORPlacebo

Interventions

NameTypeDescription
NEOD001DRUGNEOD001 is a monoclonal antibody directed at soluble and insoluble light chain aggregates
PlaceboDRUGSaline Bag
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites40

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 Myel...

Countries:United StatesAustraliaAustriaFranceGermanyGreeceIsraelItalySpainUnited Kingdom
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Frequently asked questions about NEOD001

What is NEOD001 used for?

NEOD001 is an investigational small molecule being developed for the treatment of AL amyloidosis, also known as primary amyloidosis. It is a rare disease therapeutic that has been studied in clinical trials for this condition.

Who makes NEOD001?

NEOD001 is being developed by Prothena Corporation plc, a biopharmaceutical company. The company's stock is traded under the ticker symbol PRTA.

What phase is NEOD001 in?

NEOD001 has completed Phase 1 and Phase 2 clinical trials. It is an investigational drug, meaning it has not been approved by regulatory authorities and is still in clinical development.

What clinical trials is NEOD001 in?

NEOD001 has been studied in two completed clinical trials. The first, NCT01707264, was a Phase 1/2 open-label dose escalation study in subjects with primary amyloidosis. The second, NCT02632786, was the PRONTO study, a global Phase 2b trial in previously treated subjects with AL amyloidosis.

Is NEOD001 the same as PRONTO?

No, NEOD001 is the drug being studied, while PRONTO is the name of a specific clinical trial, NCT02632786, which evaluated NEOD001 in patients with AL amyloidosis.