Open-label Extension Study of NEOD001 in Subjects With Light Chain (AL) Amyloidosis

NCT02613182 | Terminated Phase 2 Results

• 1 other identifier: NEOD001-OLE001
• Study Type: interventional
• Target: 34
• Locations: 1 country
• Sites: 5

Brief Summary

The rationale for this study is to provide additional treatment with NEOD001 for subjects who complete Study NEOD001-001, and to continue to evaluate long term safety and tolerability. All subjects in the current NEOD001 trials are being dosed at 24 mg/kg, which will be continued in this study.

Conditions

AL Amyloidosis

Outcome Measures

Primary Outcomes (1)

• Long-term Safety and Tolerability of NEOD001

  Adverse events 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.

  From initiation of study drug through the last study visit or up to 30 days after date of last dose, whichever came first, assessed up to 24 months

Study Arms (1)

Open Label

EXPERIMENTAL

Open Label Study Drug NEOD001

Drug: NEOD001

Interventions

NEOD001 DRUG

NEOD001

Open Label

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Previously enrolled and treated for at least 9 months in Study NEOD001-001
• Ability to understand and willingness to sign an informed consent form prior to initiation of any study procedures
• Has 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 times the upper limit of normal (× ULN)
• Aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) ≤3 × ULN
• Estimated glomerular filtration rate ≥30 mL/minute
• Seated systolic blood pressure 90 to 180 mmHg
• ECOG Performance Status 0 to 2
• Women of childbearing potential (WOCBP) must have a negative serum pregnancy test within 28 days prior to the first administration of study drug and agree to use highly effective physician-approved contraception from 30 days prior to the first study drug administration 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 30 days prior to the first study drug administration to 90 days following the last study drug administration

You may not qualify if:

• Any new medical contraindication or clinically significant abnormality on physical, neurological, laboratory, vital signs, or electrocardiogram (ECG) examination (e.g., atrial fibrillation; with the exception of subjects for whom the ventricular rate is controlled) that precludes continued or initiation of treatment with NEOD001 or participation in the study
• History of Grade ≥3 infusion-associated adverse events (AEs) or hypersensitivities to NEOD001 or any of its excipients
• Treatment with any anticancer therapy (standard or investigational) within the 14 days prior to the first dose of study drug. In addition, subjects must have fully recovered (i.e., National Cancer Institute Common Terminology Criteria for Adverse Events \[CTCAE\] Grade 1 \[exception: subjects with prior bortezomib may have CTCAE Grade 2 neuropathy\]) from the clinically significant toxic effects of that treatment
• Received any of the following within the specified time frame prior to the first administration of study drug:
• Hematopoietic growth factors, transfusions of blood or blood products within 1 week
• Major surgery within 2 weeks
• Radiotherapy within 2 weeks
• Transplant within 8 weeks
• Investigational drug other than NEOD001 within 4 weeks
• Another experimental anti-amyloid therapy other than NEOD001 within 2 years
• Uncontrolled symptomatic orthostatic hypotension
• Myocardial infarction, uncontrolled angina, severe uncontrolled ventricular arrhythmias, or electrocardiographic evidence of acute ischemia, within 6 months prior to the first dose of study drug
• Uncontrolled infection
• Secondary malignancy, with the exception of:
• Adequately treated basal cell carcinoma, squamous cell carcinoma, or in situ cervical cancer

Sponsors & Collaborators

• Prothena Biosciences Ltd.: lead

Study Sites (5)

Stanford Cancer Institute (SCI)

Stanford, California, 94305, United States

Location

Tufts Medical Center

Boston, Massachusetts, 02111, United States

Location

Karmanos Cancer Institute

Detroit, Michigan, 48201, United States

Location

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

Hospital of the University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

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

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: November 18, 2015
• First Posted: November 24, 2015
• Study Start: February 1, 2016
• Primary Completion: May 1, 2018
• Study Completion: May 1, 2018
• Last Updated: May 16, 2019
• Results First Posted: May 16, 2019

Record last verified: 2019-05

Locations

US (5)