Study in Subjects With Light Chain (AL) Amyloidosis

NCT03154047 | Terminated Phase 2 Results FDA Drug

• 1 other identifier: NEOD001-OLE251
• Study Type: interventional
• Target: 80
• Locations: 9 countries
• Sites: 33

Brief Summary

The objective of this study is to evaluate the long-term safety and efficacy of NEOD001 in subjects with AL amyloidosis who have completed Study NEOD001-201.

Conditions

AL Amyloidosis

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Adverse Events

  AEs are defined as any unfavorable and unintended diagnosis, symptom, sign (including an abnormal laboratory finding), syndrome, or disease which either occurs during study, having been absent at baseline, or, if present at baseline, appears to worsen. Serious adverse events are any untoward medical occurrences that result in death, are life threatening, require (or prolong) hospitalization, cause persistent or significant disability/incapacity, result in congenital anomalies or birth defects, or are other conditions which in judgment of investigators represent significant hazards.

  Each subject's study participation may have been up to 36 months or until the study was terminated

Study Arms (1)

Open label

EXPERIMENTAL

Open Label Study Drug NEOD001

Drug: NEOD001

Interventions

NEOD001 DRUG

humanized monoclonal antibody

Open label

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Completed the End of Study Visit in Study NEOD001-201
• Adequate bone marrow reserve, hepatic and renal function, as demonstrated by:
• Absolute neutrophil count (ANC) ≥1.0 × 109/L
• Platelet count ≥75 × 109/L
• Hemoglobin ≥9 g/dL
• Total bilirubin ≤2 × upper limit of normal (ULN)
• Aspartate aminotransferase (AST) ≤3 × ULN
• Alanine aminotransferase (ALT) ≤3 × ULN
• Alkaline phosphatase (ALP) ≤5 × ULN (except for subjects with hepatomegaly and isozymes specific to liver, rather than bone)
• Estimated glomerular filtration rate (eGFR) ≥25 mL/min/1.73 m2 as estimated by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation, or measured GFR ≥25 mL/min/1.73 m2
• Systolic blood pressure 80-180 mmHg
• Women of childbearing potential must have a negative pregnancy test during Screening and must agree to use highly effective physician-approved contraception from Screening to 90 days following the last study drug administration
• Male subjects must be surgically sterile or must agree to use highly effective physician-approved contraception from Screening to 90 days following the last study drug administration
• Ability to understand and willingness to sign an informed consent form prior to initiation of any study procedures

You may not qualify if:

• Any new medical contraindication or clinically significant abnormality on physical, neurological, laboratory, vital signs, or electrocardiographic (ECG) examination (e.g., atrial fibrillation; with the exception of subjects for whom the ventricular rate is controlled) that precludes continuation or initiation of treatment with NEOD001 or participation in the study
• Symptomatic orthostatic hypotension that in the medical judgment of the Investigator would interfere with subject's ability to safely receive treatment or complete study assessments
• Myocardial infarction, uncontrolled angina, uncontrolled ventricular arrhythmias, or ECG evidence of acute ischemia, within 6 months prior to the Month 1-Day 1 Visit
• Severe valvular stenosis (e.g., aortic or mitral stenosis with a valve area \<1.0 cm2) or severe congenital heart disease
• ECG evidence of acute ischemia or active conduction system abnormalities with the exception of any of the following:
• First degree atrioventricular (AV) block
• Second degree AV block Type 1 (Mobitz Type 1/ Wenckebach type)
• Right or left bundle branch block
• Atrial fibrillation with a controlled ventricular rate (uncontrolled \[i.e., \>110 bpm\] ventricular rate is not allowed \[determined by an average of three beats in Lead II or 3 representative beats if Lead II is not representative of the overall ECG\])
• Has not recovered (i.e., equivalent to a Common Terminology Criteria for Adverse Events \[CTCAE\] ≥Grade 2) from the clinically significant toxic effects of prior anticancer therapy. Exception: subjects who have received treatment with a proteasome inhibitor such as bortezomib may have CTCAE Grade 2 neuropathy.
• Received any of the following within the specified time frame prior to the Month 1-Day 1 Visit:
• Oral or IV antibiotics, antifungals, or antivirals within 1 week, with the exception of prophylactic oral agents. Note: In the event that a subject requires the chronic use of antivirals, Medical Monitor permission is required for entry into the study.
• Hematopoietic growth factors, transfusions of blood or blood products within 1 week
• Chemotherapy, radiotherapy, HDAC inhibitors, or other plasma cell directed therapy within 2 weeks
• ASCT within 4 weeks (i.e., ASCT is allowed if it occurred before enrollment in Study NEOD001-201 or after completion of Study NEOD001-201 if it was at least 4 weeks before Month 1-Day 1 of this study)

Sponsors & Collaborators

• Prothena Biosciences Ltd.: lead

Study Sites (34)

City of Hope

Duarte, California, 91010, United States

Location

Colorado Blood Cancer Institute

Denver, Colorado, 80218, United States

Location

Mayo Clinic Hospital - Florida

Jacksonville, Florida, 32224, United States

Location

University of Chicago Medicine

Chicago, Illinois, 60637, United States

Location

Indiana University Simon Cancer Center

Indianapolis, Indiana, 46202, United States

Location

Tufts Medical Center

Boston, Massachusetts, 02111, United States

Location

Karmanos Cancer Institute

Detroit, Michigan, 48201, United States

Location

Mayo Clinic - Minnesota

Rochester, Minnesota, 48201, United States

Location

Memorial Sloan-Kettering Cancer Center

New York, New York, 10065, United States

Location

Duke University Medical Center

Durham, North Carolina, 27705, United States

Location

Cleveland Clinic

Cleveland, Ohio, 44195, United States

Location

Oregon Health & Science University

Portland, Oregon, 97239, United States

Location

Hospital of the University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

Location

Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States

Location

The University of Texas MD Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Medical College of Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

Westmead Hospital

Sydney, New South Wales, 2145, Australia

Location

Princess Alexandra Hospital

Woolloongabba, Queensland, 4102, Australia

Location

Eastern Health (Box Hill Hospital)

Box Hill, Victoria, 3128, Australia

Location

Medizinische Universität Wien

Vienna, 1090, Austria

Location

Hôpital Dupuytren - CHU Limoges

Limoges, 87042, France

Location

Hôpital Pitié-Salpêtrière

Paris, 75651, France

Location

Charité - Universitätsmedizin Berlin

Berlin, 12200, Germany

Location

University of Duisburg-Essen

Essen, 45122, Germany

Location

Universitätsklinikum Hamburg-Eppendorf (UKE)

Hamburg, 20246, Germany

Location

Universitätsklinikum Heidelberg

Heidelberg, 69120, Germany

Location

Alexandra General Hospital of Athens

Athens, 11528, Greece

Location

University Hospital of Patras

Pátrai, Greece

Location

Hadassah Medical Center (HMC)

Jerusalem, 91120, Israel

Location

Fondazione IRCCS Policlinico San Matteo

Pavia, 27100, Italy

Location

Hospital Clínic de Barcelona

Barcelona, 08036, Spain

Location

Hospital Universitario Puerta de Hierro - Majadahonda

Majadahonda, 28222, Spain

Location

Queen Elizabeth Hospital

Birmingham, England, B15 2GW, United Kingdom

Location

The Royal Free Hospital

London, England, NW3 2QG, United Kingdom

Location

MeSH Terms

Conditions

Immunoglobulin Light-chain Amyloidosis

Interventions

birtamimab

Condition Hierarchy (Ancestors)

Neoplasms, Plasma Cell; Neoplasms by Histologic Type; Neoplasms; Amyloidosis; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases; Lymphoproliferative Disorders; Immunoproliferative Disorders; Immune System Diseases; Paraproteinemias

Results Point of Contact

• Title: Wendy Curlin
• Organization: Prothena Biosciences

clinicaltrials@prothena.com

650 837 8550

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 28, 2017
• First Posted: May 15, 2017
• Study Start: June 14, 2017
• Primary Completion: May 30, 2018
• Study Completion: May 30, 2018
• Last Updated: March 26, 2019
• Results First Posted: December 12, 2018

Record last verified: 2019-03

Locations

DE (4); IL (1); FR (2); GR (2); AU (3); ES (2); US (16); IT (1); GB (2)