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

NCT ID: NCT02613182

Last Updated: 2019-05-16

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE2
• Total Enrollment: 34 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2016-02-29
• Study Completion Date: 2018-05-31

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.

Detailed Description

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. The primary objective of the study is to evaluate long-term safety and tolerability of NEOD001 and the secondary objectives are to assess the immunogenicity of NEOD001 and to incorporate serum NEOD001 concentrations in a population pharmacokinetic (PK) analysis.

Conditions

AL Amyloidosis

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Open Label

Open Label Study Drug NEOD001

Group Type: EXPERIMENTAL

NEOD001

Intervention Type: DRUG

NEOD001

Interventions

NEOD001

NEOD001

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

1. Previously enrolled and treated for at least 9 months in Study NEOD001-001

2. Ability to understand and willingness to sign an informed consent form prior to initiation of any study procedures

3. 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

4. Seated systolic blood pressure 90 to 180 mmHg

5. ECOG Performance Status 0 to 2

6. 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

7. 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

Exclusion Criteria

1. 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

2. History of Grade ≥3 infusion-associated adverse events (AEs) or hypersensitivities to NEOD001 or any of its excipients

3. 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

4. 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

5. Uncontrolled symptomatic orthostatic hypotension

6. 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

7. Uncontrolled infection

8. Secondary malignancy, with the exception of:

• Adequately treated basal cell carcinoma, squamous cell carcinoma, or in situ cervical cancer
• Adequately treated stage I cancer from which the subject is currently in remission
• Any other cancer from which the subject has been disease-free for ≥3 years

9. Uncontrolled human immunodeficiency virus (HIV), hepatitis B, or hepatitis C infection

10. Women who are lactating

• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Prothena Biosciences Ltd.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Stanford Cancer Institute (SCI)

Stanford, California, United States

Tufts Medical Center

Boston, Massachusetts, United States

Karmanos Cancer Institute

Detroit, Michigan, United States

Mayo Clinic

Rochester, Minnesota, United States

Hospital of the University of Pennsylvania

Philadelphia, Pennsylvania, United States

Countries

United States

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

NEOD001-OLE001

Identifier Type: -

Identifier Source: org_study_id