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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03154047




Registration number
NCT03154047
Ethics application status
Date submitted
28/04/2017
Date registered
15/05/2017

Titles & IDs
Public title
Study in Subjects With Light Chain (AL) Amyloidosis
Scientific title
A Phase 2b Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of NEOD001 in Subjects With Light Chain (AL) Amyloidosis Who Were Previously Enrolled in Study NEOD001-201 (PRONTO)
Secondary ID [1] 0 0
NEOD001-OLE251
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
AL Amyloidosis 0 0
Condition category
Condition code
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders
Metabolic and Endocrine 0 0 0 0
Metabolic disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - NEOD001

Experimental: Open label - Open Label Study Drug NEOD001


Treatment: Drugs: NEOD001
humanized monoclonal antibody
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Participants With Adverse Events
Timepoint [1] 0 0
Each subject's study participation may have been up to 36 months or until the study was terminated

Eligibility
Key inclusion criteria
1. Completed the End of Study Visit in Study NEOD001-201
2. 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
3. Systolic blood pressure 80-180 mmHg
4. 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
5. 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
6. Ability to understand and willingness to sign an informed consent form prior to initiation of any study procedures
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. 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
2. 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
3. Myocardial infarction, uncontrolled angina, uncontrolled ventricular arrhythmias, or ECG evidence of acute ischemia, within 6 months prior to the Month 1-Day 1 Visit
4. Severe valvular stenosis (e.g., aortic or mitral stenosis with a valve area <1.0 cm2) or severe congenital heart disease
5. 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])
6. 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.
7. 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)
* Major surgery within 4 weeks (or within 2 weeks following consultation with and approval of Medical Monitor)
* Planned organ transplant during the study
* Any investigational agent, other than NEOD001, within 4 weeks
* Any experimental imaging agent directed at amyloid within 2 weeks
8. Active malignancy with the exception of any of the following:

* 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 and has been in remission for =2 years
* Low-risk prostate cancer with Gleason score <7 and prostate-specific antigen <10 mg/mL
* Any other cancer from which the subject has been disease-free for =2 years
9. History of Grade =3 infusion-related adverse events (AEs) or hypersensitivity to NEOD001
10. History of severe allergy to any of the components of NEOD001 such as histidine/L-Histidine, Trehalose, or Polysorbate 20
11. Currently known uncontrolled bacterial, viral, fungal, HIV, hepatitis B, or hepatitis C infection
12. Women who are breastfeeding
13. Any condition which could interfere with, or the treatment for which might interfere with, the conduct of the study or which would, in the opinion of the Investigator, unacceptably increase the subject's risk by participating in the study
14. Unable or unwilling to adhere to the study-specified procedures and restrictions
15. Subject is under legal custodianship

Study design
Purpose of the study
Treatment
Allocation to intervention
NA
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
14/06/2017
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
30/05/2018
Sample size
Target
Accrual to date
Final
80
Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC
Recruitment hospital [1] 0 0
Westmead Hospital - Sydney
Recruitment hospital [2] 0 0
Princess Alexandra Hospital - Woolloongabba
Recruitment hospital [3] 0 0
Eastern Health (Box Hill Hospital) - Box Hill
Recruitment postcode(s) [1] 0 0
2145 - Sydney
Recruitment postcode(s) [2] 0 0
4102 - Woolloongabba
Recruitment postcode(s) [3] 0 0
3128 - Box Hill
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Colorado
Country [3] 0 0
United States of America
State/province [3] 0 0
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
Illinois
Country [5] 0 0
United States of America
State/province [5] 0 0
Indiana
Country [6] 0 0
United States of America
State/province [6] 0 0
Massachusetts
Country [7] 0 0
United States of America
State/province [7] 0 0
Michigan
Country [8] 0 0
United States of America
State/province [8] 0 0
Minnesota
Country [9] 0 0
United States of America
State/province [9] 0 0
New York
Country [10] 0 0
United States of America
State/province [10] 0 0
North Carolina
Country [11] 0 0
United States of America
State/province [11] 0 0
Ohio
Country [12] 0 0
United States of America
State/province [12] 0 0
Oregon
Country [13] 0 0
United States of America
State/province [13] 0 0
Pennsylvania
Country [14] 0 0
United States of America
State/province [14] 0 0
Tennessee
Country [15] 0 0
United States of America
State/province [15] 0 0
Texas
Country [16] 0 0
United States of America
State/province [16] 0 0
Wisconsin
Country [17] 0 0
Austria
State/province [17] 0 0
Vienna
Country [18] 0 0
France
State/province [18] 0 0
Limoges
Country [19] 0 0
France
State/province [19] 0 0
Paris
Country [20] 0 0
Germany
State/province [20] 0 0
Berlin
Country [21] 0 0
Germany
State/province [21] 0 0
Essen
Country [22] 0 0
Germany
State/province [22] 0 0
Hamburg
Country [23] 0 0
Germany
State/province [23] 0 0
Heidelberg
Country [24] 0 0
Greece
State/province [24] 0 0
Athens
Country [25] 0 0
Greece
State/province [25] 0 0
Patras
Country [26] 0 0
Israel
State/province [26] 0 0
Jerusalem
Country [27] 0 0
Italy
State/province [27] 0 0
Pavia
Country [28] 0 0
Spain
State/province [28] 0 0
Barcelona
Country [29] 0 0
Spain
State/province [29] 0 0
Majadahonda
Country [30] 0 0
United Kingdom
State/province [30] 0 0
England

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Prothena Biosciences Ltd.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Undecided
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT03154047