Try our beta test site
IMPORTANT: Listing of a study on this site does not reflect endorsement by the National Institutes of Health. Talk with a trusted healthcare professional before volunteering for a study. Read more...
Trial record 1 of 1 for:    NCT02312206
Previous Study | Return to List | Next Study

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

This study is currently recruiting participants. (see Contacts and Locations)
Verified November 2016 by Prothena Therapeutics Ltd.
Sponsor:
Information provided by (Responsible Party):
Prothena Therapeutics Ltd.
ClinicalTrials.gov Identifier:
NCT02312206
First received: December 2, 2014
Last updated: November 9, 2016
Last verified: November 2016
  Purpose
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.

Condition Intervention Phase
Primary Systemic (AL) Amyloidosis
Drug: NEOD001
Other: Placebo
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double Blind (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 3, Randomized, Multicenter, Double-Blind, Placebo-Controlled, 2-Arm, Efficacy and Safety Study of NEOD001 Plus Standard of Care Versus Placebo Plus Standard of Care in Subjects With Light Chain (AL) Amyloidosis

Resource links provided by NLM:


Further study details as provided by Prothena Therapeutics Ltd.:

Primary Outcome Measures:
  • Time to composite of all-cause mortality or cardiac hospitalization [ Time Frame: From date of randomization until the date of death or cardiac hospitalization, whichever came first, assessed for up to approximately 42 months ]

Secondary Outcome Measures:
  • NT-proBNP best response [ Time Frame: Baseline to Month 9 ]
  • Time to cardiac mortality or cardiac hospitalization [ Time Frame: Baseline to Month 9 ]
  • Change in the 6 Minute Walk Test [ Time Frame: Baseline to Month 9 ]
  • Change in the Short Form-36 Questionnaire [ Time Frame: Baseline to Month 9 ]
  • Change in the Kansas City Cardiomyopathy Questionnaire [ Time Frame: Baseline to Month 9 ]
  • Renal best response [ Time Frame: Baseline to Month 9 ]
    As assessed using Palladini et al, 2014 criteria

  • Hepatic best response assessed using Comenzo et al, 2012 criteria [ Time Frame: Baseline to Month 9 ]
    As assessed using Comenzo et al, 2012 criteria


Estimated Enrollment: 236
Study Start Date: February 2015
Estimated Study Completion Date: August 2018
Estimated Primary Completion Date: August 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: NEOD001
24 mg/kg (maximum dose of 2500 mg) of NEOD001 administered once every 28 days.
Drug: NEOD001
NEOD001, is a humanized immunoglobulin G1 monoclonal antibody, which specifically targets misfolded light chain aggregates and amyloid deposits. NEOD001 is proposed for use to target the misfolded light chain protein in subjects with AL amyloidosis.
Placebo Comparator: Placebo
Placebo will be administered as a 250 mL bag of normal saline once every 28 days.
Other: Placebo
Placebo

Detailed 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 proteasome inhibitor-containing regimen, with the proteasome inhibitor 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.

  Eligibility

Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02312206

Contacts
Contact: Deb Chapman Clinicaltrials@prothena.com

  Show 79 Study Locations
Sponsors and Collaborators
Prothena Therapeutics Ltd.
  More Information

Responsible Party: Prothena Therapeutics Ltd.
ClinicalTrials.gov Identifier: NCT02312206     History of Changes
Other Study ID Numbers: NEOD001-CL002
Study First Received: December 2, 2014
Last Updated: November 9, 2016

Additional relevant MeSH terms:
Amyloidosis
Proteostasis Deficiencies
Metabolic Diseases

ClinicalTrials.gov processed this record on April 24, 2017